[Federal Register Volume 69, Number 28 (Wednesday, February 11, 2004)]
[Rules and Regulations]
[Pages 6788-6854]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 04-2912]
[[Page 6787]]
-----------------------------------------------------------------------
Part III
Department of Health and Human Services
-----------------------------------------------------------------------
Food and Drug Administration
-----------------------------------------------------------------------
21 CFR Part 119
Final Rule Declaring Dietary Supplements Containing Ephedrine Alkaloids
Adulterated Because They Present an Unreasonable Risk; Final Rule
Federal Register / Vol. 69, No. 28 / Wednesday, February 11, 2004 /
Rules and Regulations
[[Page 6788]]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
21 CFR Part 119
[Docket No. 1995N-0304]
RIN 0910-AA59
Final Rule Declaring Dietary Supplements Containing Ephedrine
Alkaloids Adulterated Because They Present an Unreasonable Risk
AGENCY: Food and Drug Administration, HHS.
ACTION: Final rule.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA, we, our) is issuing a
final regulation declaring dietary supplements containing ephedrine
alkaloids adulterated under the Federal Food, Drug, and Cosmetic Act
(the act) because they present an unreasonable risk of illness or
injury under the conditions of use recommended or suggested in
labeling, or if no conditions of use are suggested or recommended in
labeling, under ordinary conditions of use. We are taking this action
based upon the well-known pharmacology of ephedrine alkaloids, the
peer-reviewed scientific literature on the effects of ephedrine
alkaloids, and the adverse events reported to have occurred in
individuals following consumption of dietary supplements containing
ephedrine alkaloids.
DATES: This rule is effective on April 12, 2004.
FOR FURTHER INFORMATION CONTACT: Wayne Amchin, Center for Food Safety
and Applied Nutrition (HFS-007), Food and Drug Administration, 5600
Fishers Lane, Rockville, MD 20857, 301-827-6733.
SUPPLEMENTARY INFORMATION:
Table of Contents
I. Introduction
A. Why Have We Concluded That Dietary Supplements Containing
Ephedrine Alkaloids Present an Unreasonable Risk?
B. What Are the Ephedrine Alkaloids and Where Do They Come From?
C. What Regulatory Actions Have We Taken Regarding Dietary
Supplements Containing Ephedrine Alkaloids?
D. Petitions Received Relating to Dietary Supplement Containing
Ephedrine Alkaloids
II. Summary of Letters and Comments
III. Finding of Adulteration
A. What Does the Final Rule Do?
B. What Products are Covered?
IV. Legal Issues
A. What Is Our Legal Authority Under the Act?
B. Do the Ephedrine Alkaloid-Containing Products Covered by this
Rule Fall Within the Definition of Dietary Supplement Under the Act?
C. Administrative Procedures
V. Scientific Evaluation
A. How Did We Evaluate the Evidence?
B. What Are the Known and Reasonably Likely Risks Presented by
Dietary Supplements Containing Ephedrine Alkaloids?
1. Pharmacology
2. Other Safety Data
3. Comparison with Drug Products Containing Ephedrine Alkaloids
4. Abuse and Misuse
5. Traditional Asian Medicine
6. Adverse Events
C. What Are the Known and Reasonably Likely Benefits of Dietary
Supplements Containing Ephedrine Alkaloids?
1. Weight Loss
2. Enhancement of Athletic Performance
3. Eased Breathing
4. Other Uses
D. Do Dietary Supplements Containing Ephedrine Alkaloids Present
an Unreasonable Risk?
1. What Does ``Unreasonable Risk'' Mean?
2. Do Dietary Supplements Containing Ephedrine Alkaloids Present
an Unreasonable Risk for Labeled or Ordinary Conditions of Use?
3. Conclusion
VI. Why We Conclude that Other Restrictions Would Not Adequately
Protect Consumers from the Risks Presented by Dietary Supplements
Containing Ephedrine Alkaloids
A. Warning Statement Alone
B. Multiple Restrictions
C. Self-Regulation
D. More Education
E. Nonbinding Guidance
F. Targeted Enforcement Actions
VII. Miscellaneous Issues
A. Freedom of Choice/FDA Bias
B. Conduct of the Advisory Committee Meetings
VIII. Analysis of Impacts
A. Benefit-Cost Analysis
1. Introduction
2. Regulatory Options
3. Summary of Conclusions
4. Option One--Take No New Regulatory Action
5. Option Two--Remove Dietary Supplements Containing Ephedrine
Alkaloids from the Market
6. Option Three--Require the 2003 Proposed Warning Statement
7. Option Four--Require the Proposed Warning Statement, But
Modify it or Require it Only on Certain Products
8. Option Five--Generate Additional Information or Take Some
Action Other Than Removing Dietary Supplements Containing Ephedrine
Alkaloids From the Market or Requiring Warning Statements
9. Benefit-Cost Analysis: Summary
B. Small Entity Analysis
IX. Environmental Impact
X. Paperwork Reduction Act
XI. Federalism
XII. References
I. Introduction
A. Why Have We Concluded That Dietary Supplements Containing Ephedrine
Alkaloids Present an Unreasonable Risk?
We conclude that dietary supplements containing ephedrine alkaloids
are adulterated under section 402(f)(1)(A) (21 U.S.C. 342(f)(1)(A)) of
the act because they present an unreasonable risk of illness or injury
under the conditions of use recommended or suggested in labeling, or if
no conditions of use are suggested or recommended in labeling, under
ordinary conditions of use. Dietary supplements containing ephedrine
alkaloids are most often used for weight loss, energy, or to enhance
athletic performance.
By its plain language, section 402(f)(1)(A) of the act requires
evidence of ``significant or unreasonable risk'' of illness or injury.
There is no requirement that there be evidence proving that the product
has caused actual harm to specific individuals, only that scientific
evidence supports the existence of risk. The Government's burden of
proof for ``unreasonable risk'' is met when a product's risks outweigh
its benefits in light of the claims and directions for use in the
product's labeling or, if the labeling is silent, under ordinary
conditions of use. ``Unreasonable risk,'' thus, represents a relative
weighing of the product's known and reasonably likely risks against its
known and reasonably likely benefits. In the absence of a sufficient
benefit, the presence of even a relatively small risk of an important
adverse health effect to a user may be unreasonable. Because it is not
reasonable to conclude that a product is too risky in the absence of
any significant evidence, some weight of evidence of risk is required
to meet this standard. For example, isolated adverse events alone might
not be expected to constitute substantiation of risk, but adverse event
reports combined with pharmacological and other clinical evidence might
be expected to do so.
In considering whether dietary supplements containing ephedrine
alkaloids present an unreasonable risk, we considered evidence from
three principal sources: (1) The well-known, scientifically established
pharmacology of ephedrine alkaloids; (2) peer-reviewed scientific
literature on the effects of ephedrine alkaloids; and (3) the adverse
events (including published case reports) reported to have occurred
following consumption of dietary supplements containing ephedrine
alkaloids.
[[Page 6789]]
Ephedrine alkaloids are members of a large family of
pharmacological compounds called sympathomimetics. Sympathomimetics
mimic the effects of epinephrine and norepinephrine, which occur
naturally in the human body. Multiple studies demonstrate that dietary
supplements containing ephedrine alkaloids, like other
sympathomimetics, raise blood pressure and increase heart rate. These
products expose users to several risks, including the consequences of
increased blood pressure (e.g., serious adverse events such as stroke,
heart attack, and death) and increased morbidity and mortality from
worsened heart failure and pro-arrhythmic effects. Based on the best
available scientific data and the known pharmacology of ephedrine
alkaloids and similar compounds, we conclude that dietary supplements
containing ephedrine alkaloids pose short-term and long-term risks.
This is clearest in long-term use, where sustained increased blood
pressure in any population will increase the risk of stroke, heart
attack, and death, but there is also evidence of risk from shorter-term
use in patients with heart failure or underlying coronary artery
disease.
The data do not indicate that these products provide a health
benefit sufficient to outweigh these risks. The best clinical evidence
for a benefit is for weight loss, but even there the evidence supports
only a modest short-term weight loss, insufficient to positively affect
cardiovascular risk factors or health conditions associated with being
overweight or obese. Even if long-term weight loss could be achieved
with the use of dietary supplements containing ephedrine alkaloids, we
believe that the risks posed by these products when used continuously
in the long term generally could not be adequately mitigated except
through physician supervision. Other possible benefits, such as
enhanced athletic performance, enhanced energy, or a feeling of
alertness, lack scientific support and/or provide only temporary
benefits that we consider trivial compared to the risks of these
products, which may include long-term or permanent consequences like
heart attack, stroke, and death. Therefore, we have determined that the
risks of dietary supplements containing ephedrine alkaloids, when used
for their labeled indications or under ordinary conditions of use,
outweigh the benefits of these products. We do not believe these risks
can be adequately mitigated through other regulatory measures available
to FDA for dietary supplements, such as warnings in labeling.
As with other sympathomimetics, we believe that the risks posed by
dietary supplements containing ephedrine alkaloids, when used
continuously over the long term, generally cannot be adequately
mitigated except through physician supervision. Similar to over-the-
counter (OTC) single ingredient ephedrine and pseudoephedrine products,
we expect that dietary supplements containing ephedrine alkaloids could
be marketed without physician supervision for a very temporary,
episodic use that provides a benefit that outweighs the known and
reasonably likely risks of these products. However, we are currently
unaware of any such use, and our experience with ephedrine alkaloid-
containing OTC drug products suggests that such benefits will be
demonstrable only for disease uses.
B. What Are the Ephedrine Alkaloids and Where Do They Come From?
The ephedrine alkaloids, including, among others, ephedrine,
pseudoephedrine, norephedrine, methylephedrine, norpseudoephedrine,
methylpseudoephedrine, are chemical stimulants that occur naturally in
some botanicals (Refs. 1 through 5), but can be synthetically derived.
The ingredient sources of the ephedrine alkaloids in dietary
supplements include raw botanicals (i.e., plants) and extracts from
botanicals. Ma huang, Ephedra, Chinese Ephedra, and epitonin are
several names used for botanical ingredients, primarily from Ephedra
sinica Stapf, Ephedra equisetina Bunge, Ephedra intermedia var.
tibetica Stapf and Ephedra distachya L. (the Ephedras), that are
sources of ephedrine alkaloids (Refs. 1, 6, and 7). Other plant sources
that contain ephedrine alkaloids include Sida cordifolia L. and
Pinellia ternata (Thunb.) Makino (Refs. 8 and 9). Common names that
have been used for the various plants that contain ephedrine alkaloids
include sea grape, yellow horse, joint fir, popotillo, and country
mallow. The names desert herb, squaw tea, Brigham tea, and Mormon tea
refer to North American species of Ephedra that do not contain
ephedrine alkaloids but have been misused to identify ephedrine
alkaloid containing ingredients. Although the proportions of the
various ephedrine alkaloids in botanical species vary from one species
to another, in most species used commercially, ephedrine is typically
the predominant alkaloid in the raw material (Ref. 10).
Dietary supplements containing ephedrine alkaloids are widely sold
in the United States (Refs. 11 through 13).\1\ Over the last decade,
dietary supplements containing ephedrine alkaloids have been labeled
and used primarily for weight loss, energy, or to enhance athletic
performance. Additional scientific evidence, and numerous reports of
serious adverse events, including death, following consumption of
dietary supplements containing ephedrine alkaloids, have raised
concerns about their safety. Consequently, we have taken a number of
actions in an attempt to protect the public from the risks of these
products.
---------------------------------------------------------------------------
\1\ We use the term ``dietary supplements containing ephedrine
alkaloids'' in this final rule to refer to dietary supplements
containing botanical sources of ephedrine alkaloids. We use the term
``ephedra'' to refer to botanical sources of ephedrine alkaloids,
whether derived from a member of the Ephedra genus or another
botanical, such as Sida cordifolia L. or Pinellia ternata (Thunb.)
Makino. We use the term ``Ephedra'' to refer specifically to the
Ephedra genus of plants.
---------------------------------------------------------------------------
C. What Regulatory Actions Have We Taken Regarding Dietary Supplements
Containing Ephedrine Alkaloids?
In the Federal Register of June 4, 1997 (62 FR 30678) (June 1997
proposal), we published a proposed rule on dietary supplements
containing ephedrine alkaloids. In this document, we proposed to make a
finding, with the force and effect of law, that a dietary supplement is
adulterated if it contains 8 milligrams (mg) or more of ephedrine
alkaloids per serving, or if its labeling suggests or recommends
conditions of use that would result in an intake of 8 mg or more in a
6-hour period or a total daily intake of 24 mg or more of ephedrine
alkaloids. The June 1997 proposal would also have required that the
label of dietary supplements containing ephedrine alkaloids state that
the product should not be used for more than 7 days. We also proposed
to prohibit the use of ephedrine alkaloids in dietary supplements with
other ingredients that have a known stimulant effect that may interact
with ephedrine alkaloids, and to prohibit labeling claims, such as
weight loss or body building, that require long-term intake to achieve
the purported effect. In addition, the June 1997 proposal would have
required a statement accompanying claims that encourage short-term
excessive intake to enhance a purported effect, such as an increase in
energy, that taking more than the recommended serving may result in
serious adverse health effects. We also proposed to require that the
labels of all dietary supplements containing ephedrine alkaloids bear a
statement warning consumers not to use the product if they are taking
certain drugs;
[[Page 6790]]
advising them to contact a health care professional before use if they
have certain diseases or health conditions; and warning them to stop
use and call a health care professional if they develop certain signs
or symptoms. We proposed these actions in response to reports of
serious illnesses and injuries, including a number of deaths,
associated with the use of dietary supplements containing ephedrine
alkaloids and our investigations and assessment of these illnesses and
injuries. These actions were also supported by many of the
recommendations made during the October 1995 meeting of an ad hoc
Working Group of the FDA Advisory Committee (Working Group) and the
August 1996 meeting of the Food Advisory Committee (FAC) and the
Working Group concerning the potential public health problems
associated with the use of dietary supplements containing ephedrine
alkaloids and what action FDA should take to address the serious health
concerns associated with their use (Refs. 14 and 15).
The comment period for the June 4, 1997, proposed rule ended on
August 18, 1997. In a document published in the Federal Register of
August 20, 1997 (62 FR 44247), we announced our intent to reopen the
comment period after we corrected a number of inadvertent omissions in
the administrative record. Subsequently on September 18, 1997 (62 FR
48968), we reopened the comment period until December 2, 1997.
During this second comment period, the Commission on Dietary
Supplement Labels (the Commission) released its final report on
November 24, 1997. The Commission, an independent agency established by
section 12 of the Dietary Supplement Health and Education Act of 1994
(DSHEA) (Public Law 103-417), was charged with conducting a study on,
and providing recommendations for, the regulation of label claims and
statements for dietary supplements. The Commission's members included
several scientists from academia and industry. In its report, the
Commission divided its conclusions into three categories: findings,
guidance, and recommendations. The Commission Report defined
``findings'' as conclusions reached by the Commission based on
information and data it received during its deliberations. The
Commission defined ``guidance'' that was directed to FDA as advice that
we should consider as we developed or implemented activities related to
the availability of dietary supplements in the marketplace. The
Commission defined ``recommendations'' as suggested changes to FDA
regulations or the development of new regulations governing dietary
supplements.
One guidance statement in the Commission Report pertains to the
safety of dietary supplements containing ephedrine alkaloids. In the
report, the Commission urges FDA to use its authority under DSHEA to
take swift enforcement action to address potential safety issues such
as those posed recently by products containing ephedrine alkaloids.
While it is expected that a responsible industry will avoid marketing
unsafe products and that the industry will react promptly to remove
products shown to be associated with significant or serious adverse
events, in the final analysis there must be a strong and reliable
enforcement system to back up the safety provisions of DSHEA. Failure
by FDA to act when strong enforcement is needed undermines public
confidence in the ability of not only the Federal Government but also
the dietary supplement industry to ensure safety and avoid harm to the
public (Ref. 16 at p. VII of Executive Summary).
In a notice published in the Federal Register on April 29, 1998 (63
FR 23633), we announced our views on the recommendations and guidance
of the Commission, as presented in the Commission's report. In this
notice, we stated that we take seriously our public health protection
mission and are committed to removing unsafe dietary supplements from
the market (63 FR 23633 at 23634). The direction taken in the current
rulemaking on dietary supplements containing ephedrine alkaloids is
consistent with the Commission's advice.
In September 1998, the U.S. General Accounting Office (GAO) began a
study on FDA's June 1997 proposal. GAO's work culminated in the
issuance of a July 1999 report (Ref. 17). GAO concluded that the
evidence supported concern that ephedrine alkaloid-containing
supplements can cause serious health problems and it recommended
further data collection and review. At the same time, GAO criticized
FDA's reliance on adverse event reports (AERs) as the basis for the
proposed restrictions on dosage, frequency and duration of use.
In the Federal Register of April 3, 2000 (65 FR 17474, April 3,
2000), we withdrew parts of the June 1997 proposal. More specifically,
we withdrew the proposed finding that a dietary supplement is
adulterated if it contains 8 mg or more of ephedrine alkaloids per
serving, or if its labeling suggests or recommends conditions of use
that would result in the intake of 8 mg or more in a 6-hour period or a
total daily intake of 24 mg or more of ephedrine alkaloids; the
proposed compliance procedures (regarding the analytical method FDA
would use to determine the level of ephedrine alkaloids in a dietary
supplement); the proposed label statement ``Do not use this product for
more than 7 days;'' the proposed prohibition on labeling claims for
uses that encourage long-term intake; and the proposed label statement
to accompany claims for short-term uses (``Taking more than the
recommended serving may cause heart attack, stroke, seizure, or
death.'').
We stated in our 2000 partial withdrawal of the June 1997 proposal
that we continued to have a public health concern about the use of
dietary supplements containing ephedrine alkaloids and that we would
continue to monitor and provide appropriate followup on adverse events
associated with the use of these products. We also stated that
withdrawal of certain provisions of the June 1997 proposal did not
limit our discretion to initiate enforcement actions with respect to
dietary supplements containing ephedrine alkaloids.
On the same day as the 2000 partial withdrawal of the June 1997
proposal, we announced the availability of certain documents to update
the administrative docket of the proposed rule (65 FR 17509, April 3,
2000). The documents consisted of additional information about some of
the 270 adverse event reports (AERs) received by FDA between February
and September 1997. In a separate Federal Register notice also issued
on April 3, 2000, we announced the availability of additional AERs and
related information received after publication of the proposed rule.
The additional information included the analyses of these new AERs by
experts both inside and outside the agency; review of labels of
products associated with these adverse events; review of the use of
Ephedra species in traditional Asian medicine; analysis of the
likelihood and factors affecting the reporting of adverse events; and
summaries of the known physiological, pharmacological, and toxic
effects of ephedrine alkaloids (Ref. 18). This announcement was made in
part to prepare for a meeting convened by the U.S. Department of Health
and Human Services (HHS) Office of Women's Health (OWH) in August 2000
to discuss information about the safety of dietary supplements
containing ephedrine alkaloids. Shortly before that meeting, FDA
announced (65 FR 46721, July 31, 2000) that it would again reopen the
comment period for the June 1997
[[Page 6791]]
proposal from August 10, 2000 (the day after the OWH meeting) until
September 30, 2000. In that notice, we also announced the availability
of a report on phenylpropanolomine and hemorrhagic stroke (Ref. 19).
In April 2001, HHS's Office of the Inspector General issued a
report entitled ``Adverse Event Reporting For Dietary Supplements: An
Inadequate Safety Valve'' (Ref. 20) that assessed the effectiveness of
FDA's Adverse Event Reporting System. This report found that adverse
event reporting systems typically detect only a small proportion of the
events that actually occur.
In the Federal Register of March 5, 2003 (68 FR 10417), we
published a notice making available new information about dietary
supplements containing ephedrine alkaloids and requesting public
comment on the new information and on regulation of these products (68
FR 10417, March 5, 2003) (March 2003 notice). We specifically sought
comments on whether, in light of current information, we should
determine that dietary supplements containing ephedrine alkaloids are
adulterated because they present a significant or unreasonable risk of
illness or injury under the conditions of use recommended or suggested
in labeling or under ordinary conditions of use if the labeling is
silent. The notice also sought comment on a revised version of the
warning statement first proposed on June 4, 1997. The revised warning
statement had two components, a short warning that would be required to
appear on the principal display panel (PDP) and a longer warning that
could appear elsewhere in labeling. The proposed PDP warning stated
that strokes, heart attacks, seizures, and death have been reported
after consumption of dietary supplements containing ephedrine alkaloids
and that the risks of adverse events increase with strenuous exercise
and with use of other stimulants, including caffeine. The longer
proposed warning included more detailed information about risks
associated with the use of the product and recommended that consumers
avoid using the product and/or consult a doctor under certain
circumstances.
In the March 2003 notice, we asked for public comment on all
additional evidence developed since the publication of the June 1997
proposal. One such study was a report by the Southern California
Evidenced Based Practice Center (the RAND report, RAND, or RAND Corp.),
commissioned by the National Institutes of Health (NIH) (Refs. 21 and
22). RAND reviewed recent evidence on the risks and benefits of ephedra
and ephedrine\2\ and found that dietary supplements containing
ephedrine alkaloids are associated with higher risks of mild to
moderate side effects such as heart palpitations, psychiatric effects,
and upper gastrointestinal effects, and symptoms of autonomic
hyperactivity such as tremor and insomnia, especially when they are
taken with other stimulants. The RAND report identified 21 ``sentinel
events'' among the adverse event reports it reviewed, including stroke,
heart attack, and death.\3\ RAND also found limited evidence of an
effect of ephedra on short-term weight loss. Furthermore, RAND found
limited evidence that synthetic ephedrine and caffeine in combination
have a short-term enhancement effect on athletic performance in certain
physical activities. RAND concluded that the scientific literature does
not support an effect of ephedrine alone on athletic performance, and
there were no clinical trials on the effects of dietary supplements
containing botanical ephedrine alkaloids on athletic performance. One
of the studies reviewed by RAND, a study by Boozer, et al. (2002),
though frequently relied on by the dietary supplement industry to
demonstrate the safety of ephedrine alkaloids, raised additional
concerns about the effects of dietary supplements containing ephedrine
alkaloids on blood pressure. This evidence, discussed in section V.B of
this document, added significantly to the evidence suggesting that
dietary supplements containing ephedrine alkaloids as currently
marketed are associated with unreasonable safety risks.
---------------------------------------------------------------------------
\2\ The RAND report uses the term ``ephedra'' to refer to
ephedrine alkaloids from botanical sources, whether or not they are
contained in dietary supplements. RAND uses the term ``ephedrine''
to refer to pharmaceutical sources of ephedrine.
\3\ RAND defined a ``sentinel event'' as a case that met all
three of the following criteria: (1) Documentation of an adverse
event that met the selection criteria; (2) documentation that the
person having the adverse event took an ephedra-containing
supplement or ephedrine within 24 hours prior to the event (for
cases of death, myocardial infarction [heart attack], stroke, or
seizure); and, (3) documentation that alternative explanations for
the adverse event were investigated and were excluded with
reasonable certainty. These criteria were subject to procedures
which included the following (among other procedures): medical
record documentation that an adverse event had occurred;
documentation that the subject had consumed ephedra or ephedrine
within 24 hours prior to the adverse event, or that a toxicological
examination revealed ephedrine or one of its associated products in
the blood or urine. Cases with no such documentation were not
reviewed further. For the Metabolife cases, ephedra was assumed to
have been used within the prior 24 hours for all but psychiatric
events. All cases of stroke that met the criterion of having
consumed ephedra or ephedrine within 24 hours were reviewed in more
detail; to be classified as a ``sentinel event,'' reports of
thrombotic stroke needed to have an assessment for a hypercoagulable
state and vasculitis, reports of embolic stroke needed to have an
embolic evaluation performed, and reports of hemorrhagic stroke
required an examination to assess structural problems with the
circulatory system of the brain.
---------------------------------------------------------------------------
At about the same time as we published the March 2003 notice, we
issued warning letters to 26 firms for making unsubstantiated claims
concerning the use of dietary supplements containing ephedrine
alkaloids to enhance athletic performance. We also issued warning
letters to firms promoting dietary supplements containing ephedrine
alkaloids as alternatives to illicit street drugs.
In July 2003, GAO testified at a House Subcommittee hearing on
issues relating to dietary supplements containing ephedrine alkaloids.
GAO's testimony discussed and updated some of its findings from its
prior 1999 report on dietary supplements containing ephedrine alkaloids
(Ref. 23). The testimony provided new information, including an
evaluation of Metabolife International's records of health-related
calls from consumers of Metabolife 356 (Ref. 24). GAO noted that the
types of adverse events identified in the health-related call records
from Metabolife International were consistent with the types of adverse
events reported to us, as well as with the scientifically documented
physiological effects of ephedrine alkaloids. GAO also noted that
despite the limited information contained in most of the call records,
14,684 call records contained reports of at least one adverse event
among consumers of Metabolife 356. The GAO testimony identified 92
serious events that included heart attacks, strokes, seizures, and
deaths and emphasized that these findings were similar to other reviews
of the call records, including those done by Metabolife International
and its consultants. The GAO testimony noted that, in those call
records where age was documented, many of the serious adverse events
occurred in relatively young consumers, with more than one-third being
under the age of 30. Furthermore, for those call records in which
quantity of use and/or frequency and duration of use were noted, most
of the serious adverse events occurred among Metabolife 356 users who
used the product within the recommended guidelines, i.e., they did not
take more of the product nor consume it for a longer period of time
than the product label recommended.
[[Page 6792]]
D. Petitions Received Relating to Dietary Supplement Containing
Ephedrine Alkaloids
We received three petitions relating to dietary supplements
containing ephedrine alkaloids. The first petition, dated August 27,
1998, was submitted by the American Obesity Association and requested
that we issue a final rule on dietary supplements containing ephedrine
alkaloids that adopts the regulations in the June 1997 proposal. The
second petition, dated October 25, 2000, was filed jointly by the
American Herbal Products Association, the Consumer Healthcare Products
Association, the National Nutritional Foods Association, and the Utah
Natural Products Alliance and requested that we withdraw the remaining
portions of our June 1997 proposal and adopt and implement in its place
an industry-developed standard for the labeling and marketing of
dietary supplements containing ephedrine alkaloids.
The third petition, dated September 5, 2001, was submitted by
Public Citizen. This petition requested that we declare dietary
supplements containing ephedrine alkaloids adulterated because they
present a significant or unreasonable risk of illness or injury under
section 402(f) of the act and ban, all production and sales of these
products under section 301(a) (21 U.S.C. 331(a)) of the act. The
petition also requested that we issue an advisory to stop the use of
dietary supplements containing ephedrine alkaloids due to the
established risks of injury.
The information cited in support of this petition included:
Summaries of the updated numbers and types of
adverse events reported to us for ephedrine-alkaloid containing dietary
supplements compared to the lower incidence of the same types of
adverse events reported for all other dietary supplements;
An FDA preliminary analysis of data collected by
and purchased from the American Association of Poison Control Centers
(AAPCC) that showed an increase in the number of ephedrine alkaloid-
related AERS from 211 in 1997 to 407 in 1999; and
Adverse events reported to Public Citizen.
The petition also cited the known pharmacological and toxicological
properties of ephedrine alkaloids, recent published articles and case
reports, the fact that adverse events are invariably underreported, and
the lack of any evidence of long-term benefits for the products.
We have considered the information submitted by these petitions, as
well as the comments received in response to these petitions and all
other information in the docket. For the reasons summarized in section
I.A of this document, we have concluded that dietary supplements
containing ephedrine alkaloids are adulterated.
II. Summary of Letters and Comments
We have received more than 48,000 comments in three dockets
pertaining to ephedrine alkaloids, Docket Nos. 1995N-0304, 2000N-1200,
and 2001P-0396. These comments include all letters received prior to
the June 1997 proposal, all comments received in response to Federal
Register notices, and all submissions related to public meetings
pertaining to dietary supplements containing ephedrine alkaloids. The
48,000 comments include more than 41,000 form letters received in the
1997 docket. Many comments submitted identical or nearly identical
statements to more than one docket or in response to more than one
Federal Register notice. Most of the comments were submitted by
individual consumers who use dietary supplements containing ephedrine
alkaloids or by independent distributors of these products. Other
comments were received from persons who had, or who knew persons who
had, suffered adverse events or who were reporting adverse events
associated with the use of an ephedrine alkaloid-containing dietary
supplement. The remaining comments included those submitted by medical
professionals, scientists, medical or scientific associations, State or
local health departments, Government agencies, members of Congress,
dietary supplement manufacturers, traditional Asian medicine
practitioners and associations, dietary supplement industry trade
associations, public health associations, and consumer groups.
The form letters, while not submitting substantive evidence or
analyses, expressed strong views about our regulation of these
products. Most of these letters opposed further federal regulation of
dietary supplements containing ephedrine alkaloids. More than 13,000
comments opposed a ban of these products and indicated that further
restrictions on these products would infringe on personal choice.
Thousands of comments requested that FDA not impose stricter
regulations on dietary supplements containing ephedrine alkaloids than
those imposed on OTC drugs that contain synthetic ephedrine alkaloids.
Hundreds of comments requested that we not ban or reclassify ephedra as
a prescription drug because, they claimed, such action would result in
illegitimate profits for the pharmaceutical companies. Many expressed
the view that we should only ban supplements containing excessive
amounts of ephedrine alkaloids and those marketed to adolescents and
children or to others who may abuse and misuse these products.
Some form letters supported further regulation of these dietary
supplement products. Several stated that dietary supplements containing
ephedrine alkaloids are dangerous and asked us to ban them. Others
requested that we impose more stringent requirements such as mandatory
warning labels and maximum dosage levels. Thousands of form letters
stated that DSHEA provides us with the necessary authority to protect
the public health and that we do not need additional authority.
Numerous comments criticized us for failing to exercise the enforcement
powers authorized by DSHEA. Numerous form letters requested that
ephedrine alkaloids be allowed for professional use by traditional
Asian medicine practitioners and dispensed by licensed health care
professionals.
We have also received approximately 2,500 individual comments that,
although not form letters, did not contain substantive information,
analyses, or data. Many of these individual comments raised the same
issues as raised in the form letters. Many comments were personal
testimonials of how dietary supplements containing ephedrine alkaloids
are effective for weight control, improving stamina, or treating
medical conditions, and should not be banned or further restricted.
Several comments stated that the June 1997 proposal lacked scientific
basis and that there are many legitimate studies that support the
responsible use of dietary supplements containing ephedrine alkaloids;
however, these comments did not submit any additional scientific
evidence. Others stated that dietary supplements containing ephedrine
alkaloids are safe when used appropriately. Others were personal
testimonials of adverse events related to these products that urged a
ban or tighter restrictions of these products. Some comments criticized
the proposed label warning as too long and ineffective.
Other comments came from members of Congress, with many echoing the
issues raised by the form letters. Several congressional
representatives commented that Americans are increasingly turning to
dietary supplements to improve their health and that Congress passed
DSHEA to ensure that these products are regulated
[[Page 6793]]
as foods rather than drugs. They cited our own statements that DSHEA
gives FDA sufficient authority to remove unsafe dietary supplements
from the market. Many urged us to ensure that there was ample
opportunity to submit scientific evidence related to dietary
supplements containing ephedrine alkaloids. Many urged us to base our
decisions on sound science and not rely too heavily on AERs. Some
expressed concern about alleged FDA bias against dietary supplements
containing ephedrine alkaloids. Others passed on concerns expressed by
constituents about adverse health effects from these products. Several
comments from members of Congress expressed concern about consumers'
ability to read and properly use labels and warnings.
Many of the substantive comments submitted data and other
information regarding the use of ephedrine alkaloids. Some comments
contained legal analyses of DSHEA and other provisions of the act. Many
comments related to provisions of the June 1997 proposal that were
withdrawn in 2000 or that have become moot as a result of the action
taken in this final rule and, therefore, do not require a response.
Examples of moot issues are the proposed prohibition on claims that
encourage long-term use and the proposed label statement that the
product should not be used for more than 7 days. Other comments
addressed issues outside the scope of the rulemaking (e.g., comments
about the diversion of ephedrine alkaloids for the illegal manufacture
of methamphetamine and methcathinone) and will also not be addressed in
this document.
A summary of all relevant comments and our responses to those
comments follow. To make it easier to identify comments and our
responses, the word ``Comment,'' in parentheses, will appear before the
comment summary and the word ``Response,'' in parentheses, will appear
before our response. We have also numbered each comment summary to help
distinguish between different comment summaries. The number assigned to
each comment summary is purely for organizational purposes and does not
signify the comments' value or importance or the order in which they
were received.
III. Finding of Adulteration
A. What Does the Final Rule Do?
This final rule declares dietary supplements containing ephedrine
alkaloids to be adulterated under section 402(f)(1)(A) of the act. We
have determined that these products present an unreasonable risk of
illness or injury under the conditions of use recommended or suggested
in labeling or, if no conditions of use are suggested or recommended in
labeling, under ordinary conditions of use. We are taking this action
based upon the well-known and scientifically established pharmacology
of ephedrine alkaloids, the peer-reviewed scientific literature about
the effects of ephedrine alkaloids, published case reports of adverse
events, and the adverse events reported to us that have occurred in
individuals using products containing ephedrine alkaloids, particularly
dietary supplements. We have concluded that dietary supplements
containing ephedrine alkaloids pose a risk of serious adverse events,
including heart attack, stroke, and death, and that these risks are
unreasonable in light of any benefits that may result from the use of
these products under their labeled conditions of use, or under ordinary
conditions of use if the labeling is silent. We are not addressing the
issue of whether these products present a ``significant'' risk under
section 402(f)(1)(A) of the act.
B. What Products are Covered?
This final rule applies to dietary supplements containing ephedrine
alkaloids, including, but not limited to, those from the botanical
species Ephedra sinica Stapf, Ephedra equisetina Bunge, Ephedra
intermedia var. tibetica Stapf, Ephedra distachya L., Sida cordifolia
L. and Pinellia ternata (Thunb.) Makino or their extracts. The
ingredient sources of the ephedrine alkaloids include raw botanicals
and extracts from botanical sources. Although synthetic ephedrine (in
the form of ephedrine hydrochloride) has been found in products labeled
as dietary supplements, ephedrine hydrochloride was approved for use as
a human drug as early as the late 1940s and, to the best of our
knowledge there is no evidence that it was marketed prior to that time
as a dietary supplement or food. Furthermore, ephedrine hydrochloride
and other synthetic sources of ephedrine cannot be dietary ingredients
because they are not constituents or extracts of a botanical, nor do
they qualify as any other type of dietary ingredient. For these
reasons, products containing synthetic ephedrine cannot be legally
marketed as dietary supplements (See section 201(ff)(1) and
201(ff)(3)(B) of the act (21 U.S.C. 321(ff)(1) and (ff)(3)(B))). In
October 2001, we brought a seizure action against $2.8 million worth of
finished drug products containing synthetic ephedrine hydrochloride
that were labeled as dietary supplements (United States v. 1009 Cases *
* * E'ola International AMP II), No. 2:01CV-820C (D. Utah filed October
22, 2001)). As a result of this seizure, in 2002, the manufacturer
signed a consent decree agreeing to the condemnation and destruction of
the seized products and prohibiting it from manufacturing or
distributing violative ephedrine hydrochloride products. In other
actions, we have sent warning letters to multiple firms that were
marketing products containing synthetic ephedrine alkaloids as dietary
supplements, resulting in the removal of the illegal products from the
market.
The final rule does not apply to conventional food products that
contain ephedrine alkaloids. Substances intentionally added to a
conventional food are generally considered to be food additives under
section 201(s) of the act. Ephedrine alkaloids contained in
conventional foods would generally be considered unsafe food additives
(see section 409 of the act (21 U.S.C. 348)). A food that contains an
unsafe food additive is adulterated under section 402(a)(2)(C) of the
act.
This final rule also does not include OTC or prescription drugs
that contain ephedrine alkaloids. The use of ephedrine or
pseudoephedrine for the treatment of asthma, colds, allergies, or any
other disease is beyond the scope of this final rule. Ephedrine is
allowed as an active ingredient in oral OTC bronchodilator drugs for
use in the treatment of medically diagnosed mild asthma (Sec. 341.16
(21 CFR 341.16)), when used within the established dosage limits and
when the product is labeled in accordance with the required statements
of identity, indications, warnings, and directions for use found in
Sec. 341.76. In the near future, we intend to propose revisions to Sec.
341.76 to reflect current scientific information about the risks of
ephedrine. Both ephedrine (topical) and pseudoephedrine (oral) are
permitted as active ingredients for use as nasal decongestants (Sec.
341.20), when they are used within the dosage limits established by and
labeled in accordance with Sec. 341.80. The topical use of ephedrine
will not be further discussed in this rule because it is not relevant
to oral consumption of ephedrine in dietary supplements. The use of
ephedrine alkaloids in drug products is discussed in more detail in
section V.B.3 of this document.
Several Ephedra species (including those known as ma huang) have a
long history of use in traditional Asian medicine. These products are
beyond the scope of this rule because they are
[[Page 6794]]
not marketed as dietary supplements. The use of ephedrine alkaloids in
traditional Asian medicine is discussed in more detail in section V.B.5
of this document. As we describe there, this rule does not change how
these products are regulated under the act.
(Comment 1) One comment stated that we coined the term ``ephedrine
alkaloids'' to improperly broaden the scope of the published scientific
literature and AERs cited in the June 1997 proposal. The comment
pointed out that ephedrine, pseudoephedrine, and phenylpropanolamine
(PPA) are all different chemical entities and stated the opinion that
only data on ephedrine are relevant to the June 1997 proposal.
(Response) Although we agree that the terms ephedrine,
pseudoephedrine, and PPA refer to different chemical entities, we
disagree with the rest of the comment and its conclusions. The term
``ephedrine alkaloids'' refers to a class of naturally occurring
compounds structurally related to ephedrine, and the term has been used
in that manner in the scientific literature (Refs. 25 and 26). We chose
this particular term, rather than several alternatives, such as
``Ephedra bases'' and ``ephedrine type alkaloids,'' to limit the scope
of the June 1997 proposal to those compounds that are natural
constituents of the aerial parts of the Ephedra plant or other
botanical sources of ephedrine and related alkaloids. We also defined
the term by listing the six principal natural alkaloids in the June
1997 proposal and other FDA documents (Refs. 6 and 27). The ephedrine
alkaloids in botanicals include l-ephedrine, d-pseudoephedrine, l-
norephedrine, l-methylephedrine, d-norpseudoephedrine, d-
methylpseudoephedrine, and minor related alkaloids. All of these
compounds are pharmacologically active substances in the plant.
Therefore, we considered all of them in our evaluation of the risks
associated with the use of the botanical or extracts from the
botanical. However, as discussed in the response to comment 24 in
section VI.B.1 of this document, we recognize that there are some
differences between ephedrine and PPA.
(Comment 2) Several comments asked whether North American species
of Ephedra (e.g., Mormon Tea) are covered in this rulemaking.
(Response) Most North American species of Ephedra (e.g., Mormon
tea) do not contain ephedrine alkaloids (Refs. 2 and 26). Nonetheless,
any dietary supplement that contains ephedrine alkaloids from any
botanical source, including from a North American species of Ephedra,
is subject to this rulemaking.
IV. Legal Issues
A. What Is Our Legal Authority Under the Act?
We are issuing this final regulation under sections 402(f)(1)(A)
and 701(a) of the act (21 U.S.C. 371(a)). Section 402(f)(1)(A) of the
act deems a food to be adulterated for the following reasons:
If it is a dietary supplement or contains a dietary ingredient
that--
(A) presents a significant or unreasonable risk of illness or
injury under--
(i) conditions of use recommended or suggested in labeling, or
(ii) if no conditions of use are suggested or recommended in the
labeling, under ordinary conditions of use.
This regulation makes a finding that dietary supplements containing
ephedrine alkaloids are adulterated because they present an
unreasonable risk within the meaning of section 402(f)(1)(A) of the
act. This finding is based on our conclusion that the risks of these
products outweigh their benefits. Our legal interpretation of
``unreasonable risk'' is discussed in detail in section V.D.1 of this
document. This regulation does not address the meaning of ``significant
risk'' or whether dietary supplements containing ephedrine alkaloids
present a significant risk under section 402(f)(1(A) of the act.
Section 701(a) of the act gives FDA authority to issue regulations
for the efficient enforcement of the act. We are using this rulemaking
authority for dietary supplements containing ephedrine alkaloids
because we are articulating a standard for unreasonable risk under
402(f)(1)(A) of the act for the first time and because it is more
efficient to declare these products adulterated as a category than to
remove them from the market in individual enforcement actions in which
we would have to establish, for each individual product, that they
present a significant or unreasonable risk.
The March 2003 notice asked about the adequacy of FDA's authority
to regulate dietary supplements containing ephedrine alkaloids. More
specifically, we sought comments on ``what additional legislative
authorities, if any, would be necessary or appropriate to enable us to
address this issue most effectively'' (68 FR 10417 at 10420).
(Comment 3) Many comments expressed the view that we already have
the authority we need to take action against dietary supplements
containing ephedrine alkaloids. These comments cited our authority to
declare these supplement products to be a significant or unreasonable
risk or imminent hazard under section 402(f)(1) of the act or to
regulate the products as containing a poisonous or deleterious
substance that may render them injurious to health under section
402(a). The comments differed as to whether we had the necessary
evidence to utilize these provisions. Several comments opposed any
additional authority and criticized us for allegedly not fully
implementing the authority we already have.
(Response) We agree that we have the authority to take action
against dietary supplements that contain ephedrine alkaloids. All three
authorities mentioned by the comments are available to us when
circumstances warrant. In this instance, we have chosen to proceed
under the adulteration standard in section 402(f)(1)(A) of the act. We
believe that we have sufficient evidence to meet this standard.
(Comment 4) In contrast, other comments stated that our legal
authority should be strengthened. Several comments expressed the view
that DSHEA needs to be amended because it cannot adequately protect
public health. One public interest group noted that our delay in acting
reflects the difficulty we encounter implementing DSHEA. Several
comments offered suggestions for amendments that would strengthen our
legal authority, including mandatory reporting of adverse events,
certain sales restrictions (e.g., restricting sales to behind the
counter only, prohibiting sales to individuals under the age of 18),
special labeling requirements for dietary supplements containing
ephedrine alkaloids, registration and listing, premarket approval for
safety and efficacy (particularly for all new stimulants and steroid
substitutes), and repeal of the de novo review provision so that we
would receive judicial deference on adulteration issues. A few comments
suggested that dietary supplements be regulated as drugs. One comment
suggested new legislation to classify dietary supplements according to
a risk-based regulatory scheme.
(Response) We must regulate dietary supplements under our existing
authority. Accordingly, we are unable to take action regarding
suggestions for amendments to DSHEA because any such amendments must
result from congressional action rather than rulemaking. Therefore, we
are not addressing those suggestions in this rule.
(Comment 5) One comment stated that conventional food safety
standards, i.e., the generally recognized as safe (GRAS) standard or
the standard for
[[Page 6795]]
FDA approval as a food additive, do not apply to dietary ingredients.
(Response) We agree that the standards referred to in this comment
do not apply to dietary ingredients. Premarket approval is required of
substances that are food additives as defined in section 201(s) of the
act. Substances that would otherwise fall under the food additive
definition but are generally recognized as safe by experts are not food
additives and do not require premarket approval. Dietary ingredients
contained in, or intended for use in, a dietary supplement are
explicitly excluded from the food additive definition in section
201(s)(6) of the act. Therefore, neither the premarket approval regime
for food additives nor the GRAS standard applies to dietary
ingredients. We are instead basing this final rule on the dietary
supplement adulteration standard set forth in section 402(f)(1)(A) of
the act.
(Comment 6) One comment stated we are violating the First Amendment
of the U.S. Constitution and the Administrative Procedure Act (APA) by
requiring a much higher standard of safety for dietary supplements than
for conventional foods. Another comment also raised concerns about the
First Amendment limits of FDA's authority to regulate dietary
supplements containing ephedrine alkaloids.
(Response) We disagree with these comments. There are a number of
different safety standards for foods (see, e.g., section 402(a)(1) and
section 402(a)(2)(C) of the act), and whether these standards are
higher or lower than the ``significant or unreasonable risk'' standard
for dietary supplements in section 402(f)(1)(A) of the act is not
relevant to the legal sufficiency of this rule. To the extent that we
regulate dietary supplements and conventional foods differently, these
differences are justified by the differences in the statutory
provisions that apply to these two categories of products. Although
some parts of the act apply to both dietary supplements and
conventional foods, other provisions apply only to one or the other.
Where Congress expressly provided for dietary supplements to be subject
to a requirement or standard that does not apply to conventional foods,
we may implement that provision without violating the APA. Further,
this final rule does not violate the First Amendment. This rule does
not restrict speech; rather, it makes a finding of adulteration that
results in a prohibition on the distribution and sale of a product that
presents unreasonable health risks. Such restrictions on purely
commercial, nonexpressive conduct are not subject to First Amendment
scrutiny. See, e.g., United States v. O'Brien, 391 U.S. 367, 376
(1968).
(Comment 7) Several comments expressed the view that these products
should be regulated as drugs under our existing authority. Some
comments stated that we should make these products available only by
prescription, arguing that the potential health hazards associated with
dietary supplements containing ephedrine alkaloids are too serious for
OTC use and that restricting access by requiring a prescription would
insert trained medical professionals into a case-by-case decision on
the appropriateness of these products to an individual consumer.
Further, one comment recommended that if the frequency of adverse
events under prescription status does not improve, more restrictive
action should be implemented, including the withdrawal of all products
containing ephedrine alkaloids from the market.
(Response) We do not agree that all dietary supplements containing
ephedrine alkaloids may be regulated as drugs under our existing
authority. Products are drugs only if they meet the definition of drug
in section 201(g)(1) of the act. Products containing ephedrine
alkaloids are regulated as drugs if they are intended to be used in the
diagnosis, cure, mitigation, treatment, or prevention of disease
(section 201(g)(1)(B) of the act). Without evidence of intended use for
such purposes, the product is not a drug under the act. Some dietary
supplements containing ephedrine alkaloids are promoted for disease
uses, e.g., to treat obesity. In such instances, we can and have taken
action against certain dietary supplement products as drugs. Under the
act, considerations such as potential risks to health, need for medical
supervision, and pharmacology of a product that meets the dietary
supplement definition are not by themselves sufficient to subject the
product to regulation as a drug.
To the extent that comments suggest that these products could
somehow remain dietary supplements but be available only by
prescription, we note that we do not have authority to take such
action. The act gives us the authority to restrict drugs and devices to
prescription use; it does not give us the authority to restrict dietary
supplements to prescription use.
(Comment 8) One comment stated that the generally accepted
definition of safety for a drug, i.e., a low incidence of adverse
reactions or significant side effects under appropriate conditions of
use, and a low potential for harm, which might result from abuse
situations, is equally applicable to dietary supplements or food.
(Response) We do not agree that the safety standards for drugs
apply to dietary supplements or other foods. As explained previously,
dietary supplements are not drugs unless they meet the definition of
drug in section 201(g)(1) of the act. The same is true for conventional
foods. We are basing this final rule on the dietary supplement
adulteration standard set forth in section 402(f)(1)(A) of the act. The
adulteration standard for dietary supplements set forth in section
402(f)(1)(A) of the act implies a risk-benefit calculus. While we also
use a risk-benefit evaluation in the drug evaluation process (see Sec.
312.21(c), Sec. 314.50(c)(5)(viii), and Sec. 330.10(a)(4) (21 CFR
312.21(c), 314.50(c)(5)(viii), and 330.10(a)(4))), the act creates
different evidentiary standards for dietary supplements and drugs.
Therefore, we are not applying the drug safety standard to dietary
supplements.
B. Do the Ephedrine Alkaloid-Containing Products Covered by this Rule
Fall Within the Definition of Dietary Supplement Under the Act?
A threshold issue is whether the products covered by this rule meet
the definition of a dietary supplement under section 201(ff) of the
act.
(Comment 9) One comment from a State department of health stated
the opinion that dietary supplements containing ephedrine alkaloids
present significant risks when they are consumed as a regular part of
the diet and do not fall within section 201(ff)(1) of the act. The
comment explained that because these products cannot be used on a daily
basis without presenting significant risks they cannot be ``intended to
supplement the diet'' and are not dietary supplements within the
meaning of the act. A related comment expressed the opinion that, for a
substance to be a dietary supplement, it must be proven that the human
body needs the substance to establish a need for supplementation.
(Response) We agree with these comments in part and disagree in
part. We agree that dietary supplements containing ephedrine alkaloids
present a risk when consumed as a regular part of the diet; as
discussed in section V.B of this document, they present a risk to some
users even when consumed occasionally. We do not agree, however, that
dietary supplements containing botanical ephedrine alkaloids do not
fall within the definition of a dietary supplement in section 201(ff)
of the act. Section 201(ff)(1) of the act, added by
[[Page 6796]]
DSHEA, provides, in part, that the term ``dietary supplement'' means a
product ``intended to supplement the diet'' that bears or contains one
or more dietary ingredients. Among the dietary ingredients listed in
section 201(ff)(1) of the act are herbs and other botanicals.
Therefore, botanical sources of ephedrine alkaloids, such as Ephedra
sinica Stapf and the other botanicals described in section III.B. of
this document, are dietary ingredients. Further, we do not agree that
the phrase ``intended to supplement the diet'' authorizes the exclusion
of a product from the dietary supplement definition solely on the basis
of risk. Given the explicit references to risk in section 402 of the
act and the inclusion of botanicals as a category of dietary
ingredients in section 201(ff)(1) of the act, it seems clear that
Congress intended us to regulate botanical products as dietary
supplements (provided that they are not drugs and otherwise meet the
dietary supplement definition) and to evaluate their risks under the
adulteration provisions in section 402 of the act.
We also do not agree that, under the dietary supplement definition,
it must be proven that the human body needs a particular substance to
establish a need for supplementation. Under DSHEA, a substance does not
necessarily have to be shown to be essential to human nutrition to be
marketed as a dietary supplement. Although no provision in the act or
legislative history directly addresses this issue, section 201(ff) of
the act lists classes of dietary ingredients (e.g., botanicals) that
are not essential for growth or to maintain good health (Ref. 28). The
fact that Congress classified such substances as dietary ingredients is
clear evidence that Congress did not intend to limit dietary
ingredients to substances that have been deemed to be essential in
human nutrition.
(Comment 10) Several comments, including one from an industry
medical consultant, stated that herbal products should not be regulated
under DSHEA because they have physiologic effects and significant
potential for toxicity. The comment encouraged us to work with industry
to establish an appropriate regulatory category for botanicals.
(Response) Under the act (as amended by DSHEA), botanicals can be
marketed as dietary supplements provided that they otherwise meet the
dietary supplement definition, and are safe and properly labeled. If
botanicals meet the drug definition in section 201(g) of the act, they
are properly regulated as drugs. In this regard, we published a final
rule entitled ``Additional Criteria and Procedures for Classifying
Over-the-Counter Drugs as Generally Recognized as Safe and Effective
and Not Misbranded'' (67 FR 3060, January 23, 2002). This rule defines
the term ``botanical drug substance'' and explains how to submit a time
and extent application to request that a botanical drug substance be
included in an OTC drug monograph (see Sec. 330.14). In addition, we
recognize, and are addressing, the current need for guidance for
manufacturers seeking to develop botanicals as either OTC or
prescription drug products under the applicable statutory and
regulatory requirements. (See Guidance for Industry: Botanical Drug
Products (Draft Guidance) (August 2000) (available at http://www.fda.gov/cder/guidance/1221dft.pdf).)
C. Administrative Procedures
(Comment 11) Several comments stated that it is premature to
request comments on whether dietary supplements containing ephedrine
alkaloids present a significant or unreasonable risk before we define
that standard. These comments urged us to undertake a rulemaking, or a
guidance document, on this new standard so that it can be applied in
the future to all dietary supplements posing health concerns. One
comment suggested that defining ``significant or unreasonable risk''
may require new legislation.
(Response) We do not agree that we must define the term
``unreasonable risk'' standard through regulation or guidance before
taking action against dietary supplements containing ephedrine
alkaloids based upon this standard. An agency may interpret a statutory
provision through rulemaking or case-by-case adjudication (SEC v.
Chenery, 332 U.S. 194 (1947)). We conclude, based upon available
evidence discussed in section V of this document, that dietary
supplements containing ephedrine alkaloids present an unreasonable risk
of illness or injury because their risks outweigh their benefits, and
that these products are therefore adulterated under section
402(f)(1)(A) of the act. We are using our general rulemaking authority
to issue regulations for the efficient enforcement of the act (section
701(a) of the act) to issue a regulation applying the standard in the
context of a particular category of dietary supplements--those that
contain botanical ephedrine alkaloids. We are not required to issue a
separate rule or guidance defining the 402(f)(1)(A) standard before
issuing such a regulation. Similarly, lack of a regulation or guidance
defining the standard neither prevents us from taking enforcement
action against dietary supplements that present an ``unreasonable
risk,'' nor is it new legislation necessary for us to interpret the
meaning of ``unreasonable risk.'' If Congress has clearly spoken to a
question of statutory interpretation, the agency charged with
administering the statute must implement the unambiguous intent of
Congress (``Chevron step one'') (Chevron U.S.A., Inc. v. Natural
Resource Defense Council, 467 U.S. 837, 842-843 (1984)). If a statute
is silent or ambiguous on the question, however, the agency may
interpret the ambiguous provision (``Chevron step two'') Id. at 843-
844. When such administrative interpretations are made through
rulemaking, they will be upheld as long as they are reasonable and
consistent with the statute's purpose and legislative history
(Christensen v. Harris County, 529 U.S. 576, 587 (2000); Chevron
U.S.A., Inc. v. FERC, 193 F.Supp.2d 54, 68 (D.D.C. 2002)). As discussed
in the response to comment 59 in section V.D.1 of this document, we
have concluded under Chevron step one that the phrase ``unreasonable
risk'' clearly directs FDA to conduct a risk-benefit analysis. Even if
a court were to find that phrase ambiguous, however, our interpretation
is reasonable under Chevron step two.
(Comment 12) Several comments urged us not to act against all
dietary supplements containing ephedrine alkaloids because all such
products are different and must be considered individually. The
comments cited differences in dosages, formulations, labeling, etc.,
across products and, thus, each product must be analyzed on its own
merits. One industry comment argued that we exceeded our statutory
authority in trying to regulate all dietary supplements containing
ephedrine alkaloids through notice and comment rulemaking.
(Response) We do not agree that we may not regulate the entire
category of dietary supplements containing ephedrine alkaloids through
rulemaking. We recognize that there are differences between different
dietary supplements containing ephedrine alkaloids. However, we
conclude, based on available science, that all dietary supplements
containing ephedrine alkaloids present an unreasonable risk of illness
or injury, regardless of how they are formulated or labeled, because
the risks outweigh any benefits that may result from use of the
products. Therefore, we may issue a rule finding the entire class of
products adulterated.
(Comment 13) A few comments noted that we bear the burden of proof
to show
[[Page 6797]]
dietary supplements are adulterated under section 402(f)(1) of the act.
(Response) We agree with this comment. Section 402(f)(1) of the act
clearly states that in any proceeding under that provision, ``the
United States shall bear the burden on each element to show that a
dietary supplement is adulterated.'' We have met that burden in this
rulemaking.
(Comment 14) Several comments discussed our ability to declare
dietary supplements containing ephedrine alkaloids an imminent hazard
under section 402(f)(1)(C) of the act.
(Response) We are not addressing these comments because we have
chosen to proceed under section 402(f)(1)(A).
(Comment 15) One industry comment stressed that comments to the
June 1997 proposal may not be used to authorize other final
regulations. The comment expressed concern that comments to a proposed
warning statement would be used as a basis for another FDA action to
regulate these supplements.
(Response) We disagree with this comment. FDA may issue this final
regulation based on a finding that dietary supplements containing
ephedrine alkaloids are adulterated because they present an
unreasonable risk under section 402(f)(1)(A) of the act. APA requires
agencies to provide the public with notice and an opportunity for
comment before issuing a new regulation (5 U.S.C. 553(b) and (c)). In
keeping with this requirement, a final rule may differ from a proposed
rule if the final rule is a ``logical outgrowth'' of a proposed rule
(Small Refiner Lead Phase-Down Task Force v. EPA, 705 F.2d 506, 547
(D.C. Cir. 1983)). The inquiry into whether a final rule is a logical
outgrowth of the proposed rule is often stated as whether the regulated
party ``should have anticipated that such a requirement might be
imposed'' (Small Refiner, 705 F.2d at 549). Agencies ``undoubtedly have
authority to promulgate a final rule that differs in some particulars
from its proposed rule* * * `[a] contrary rule would lead to the
absurdity that * * * the agency can learn from the comments on its
proposals only at the peril of starting a new procedural round of
commentary''' (Small Refiner, 705 F.2d at 546-547 (quoting
International Harvester Co. v. Ruckelshaus, 478 F.2d 615, 632 n.51
(D.C. Cir.1973))). The D.C. Circuit has also stated: ``The APA notice
requirement is satisfied if the notice fairly apprises interested
person of the subjects and issues the agency is considering; `the
notice need not specifically identify ``every precise proposal which
[the agency] may adopt as a final rule''' (Chemical Manufacturers
Association Waste Mfrs. v. EPA, 870 F.2d 177, 203 (5th Cir. 1989)
(quoting United Steelworkers of Am. v. Schuylkill Metals, 828 F.2d 314,
317 (5th Cir. 1987) (internal citations omitted))).
Our June 1997 proposal, along with our March 5, 2003 Federal
Register notice, provided a sufficient basis to allow the public to
anticipate our actions in this final rule. Through our proposed actions
on dietary supplements containing ephedrine alkaloids, the public was
properly notified of the possibility that we would find such products
to be adulterated under section 402(f)(1)(A) of the act. In fact, our
March 2003 notice specifically asked for comment on whether dietary
supplements containing ephedrine alkaloids present a significant or
unreasonable risk under section 402(f)(1)(A) of the act. We also sought
comment on new evidence concerning the safety of dietary supplements
containing ephedrine alkaloids (68 FR 10417 at 10420). In addition, the
restriction on ephedrine alkaloid/stimulant combinations proposed in
1997, which was unaffected by the 2000 partial withdrawal proposal, was
based in part on a finding of adulteration under section 402(f)(1)(A)
of the act (62 FR 30678 at 30696). Though we did not specifically
propose to codify a finding of adulteration based on significant or
unreasonable risk in the March 2003 notice, it was clear that we were
contemplating the possibility that dietary supplements containing
ephedrine alkaloids were adulterated under section 402(f)(1)(A) of the
act. Courts have upheld final rules that contained new elements when
the public was made aware that the agency was contemplating such a
change (See Chem. Mfrs. Ass'n. , 870 F.2d 202-203). Furthermore, we
received several comments regarding the possibility of a finding that
all dietary supplements containing ephedrine alkaloids would be deemed
adulterated under section 402(f)(1)(A) of the act. Though not
determinative of logical outgrowth in and of themselves, comments on
the issue are evidence that the public received adequate notice of our
final rule (Shell Oil Co. v. EPA, 950 F.2d 741, 757 (D.C. Cir. 1991)).
Based upon our explicit request for comments on the adulteration issue
in our March 2003 notice, our reference to the section 402(f)(1)(A) of
the act adulteration standard as a basis for our June 1997 proposal,
and the fact that a number of parties commented on whether dietary
supplements containing ephedrine alkaloids present a significant or
unreasonable risk, there was adequate notice to the public of our
actions in this final rule.
(Comment 16) Several comments cited language in section 402(f)(1)
of the act providing that courts must review any determination under
section 402(f)(1) of the act de novo and further stated that we would
not get judicial deference in any court review. The comments argued
that, under this provision, it would make no difference whether we
brought our case initially in court or whether we proceeded through
rulemaking that was subsequently challenged in court. One trade
association noted that such de novo review is a novel approach in that
usually a court would just review the administrative record.
(Response) Section 402(f)(1) of the act states that a court will
decide any issue under that paragraph on a de novo basis. We agree that
the de novo standard of review applies to our factual findings under
section 402(f)(1) of the act, but do not agree that it applies to our
conclusion under Chevron U.S.A., Inc., that ``unreasonable risk'' means
a risk-benefit analysis (see section V.D.1 of this document). This
interpretation of the de novo provision of section 402(f)(1) of the act
is consistent with case law on the Toxic Substances Control Act (TSCA),
which contains an unreasonable risk standard coupled with a
``substantial evidence'' standard of review, analogous to the act's
unreasonable risk standard coupled with a de novo standard of review.
In Chem. Mfrs. Ass'n v. EPA, 859 F.2d 977 (D.C. Cir. 1988), the D.C.
Circuit distinguished EPA's legal interpretation of unreasonable risk,
which received deference under Chevron U.S.A., Inc. v. Natural
Resources Defense Council, 467 U.S. 837 (1984), from its burden of
showing with ``substantial evidence'' in the record that it has met the
standard. The court stated: ``This fairly rigorous standard of record
review should not * * * be confused with the substantive statutory
standard * * * '' (859 F.2d at 992). Thus, the court in Chem. Mfrs.
Ass'n. held that the ``substantial evidence'' standard of record review
applied to the factual basis of EPA's decision but not to its
interpretation of the statutory standard. In applying Chevron U.S.A.,
Inc., we have concluded that Congress unambiguously intended that
unreasonable risk entails a risk-benefit calculus. If a court were to
find the phrase ``unreasonable risk'' ambiguous, however, our
interpretation of unreasonable risk as meaning a risk-benefit calculus
should receive Chevron U.S.A., Inc. deference, like EPA's
[[Page 6798]]
interpretation of the statutory standard in Chem. Mfrs. Ass'n.. The
requirement for de novo review should be applied only to the factual
basis of FDA's determination.
Regardless of which standard applies, however, our determination
that dietary supplements containing ephedrine alkaloids present an
unreasonable risk under section 402(f)(1)(A) of the act should be
sustained by a court. Our conclusion that ``unreasonable risk'' entails
a risk-benefit analysis is consistent with the express intent of
Congress. The scientific evidence regarding the pharmacology of
products containing ephedrine alkaloids, clinical studies showing that
these products raise blood pressure, published case reports, and AERs,
when compared with the evidence regarding the very modest benefits
conferred by these supplements, forms a strong factual basis for
finding that the known and reasonably likely risks of dietary
supplements containing ephedrine alkaloids outweigh the known and
reasonably likely benefits of these products. Therefore, dietary
supplements containing ephedrine alkaloids present an unreasonable risk
of injury or illness under section 402(f)(1)(A) of the act.
(Comment 17) One comment submitted by a trade association noted
that, before requesting the Department of Justice to take any civil
action against dietary supplements containing ephedrine alkaloids, we
must give appropriate notice and opportunity to present oral and
written arguments at least 10 days prior to the request.
(Response) We agree with this comment in part and disagree in part.
Section 402(f)(2) of the act provides that ``the person against whom
such proceeding would be initiated'' must be given notice and the
opportunity to present views, orally and in writing, 10 days before we
report a violation of section 402(f)(1)(A) of the act (the
``significant or unreasonable risk'' provision) to the Department of
Justice for a civil proceeding. By the plain language of this
provision, it applies to proceedings against persons, not to
proceedings against products. Thus, the requirement applies to
injunction actions, which are brought against a corporate or individual
person, but not to seizures, which are brought against a product.
Therefore, if we were to refer a seizure of dietary supplements
containing ephedrine alkaloids to the Department of Justice, the notice
requirement would not apply. We further note that the current
proceeding is a rulemaking, not a civil action being referred to the
Department of Justice, and therefore the 10-day notice requirement does
not apply.
(Comment 18) One industry comment stated that the stringent 30-day
timeframe allowed for comments in response to the March 2003 notice did
not provide the industry with a fair opportunity to review the
administrative record and fairly respond to ``any alleged new evidence
and analyses'' by FDA. This comment urged us to allow for a comment
period of 180 days. The comment stated that this procedural lapse would
render the entire rulemaking process arbitrary and capricious.
(Response) We disagree with this comment. We believe that the 30-
day comment period on the March 2003 notice provided interested persons
with an adequate opportunity for review and comment. The information
placed in the public docket at that time was limited, consisting of the
RAND report plus six recent studies. APA requires only that an agency
``give interested persons an opportunity to participate in the
rulemaking through submission of written data, views, or arguments * *
*'' This opportunity to participate is all that the APA requires. There
is no statutory requirement concerning how many days we must allow for
comment, nor is there a requirement that we extend the comment period
at the request of an interested person (See Phillips Petroleum Co. v.
EPA, 803 F.2d 545, 559 (10th Cir. 1986)). Moreover, given that we first
opened a docket on the issue of dietary supplements containing
ephedrine alkaloids in 1995 and sought comments on this issue several
times between then and 2003 (see section I.C of this document), there
has been ample opportunity for all those interested to submit
information and views.
V. Scientific Evaluation
A. How Did We Evaluate the Evidence?
To determine whether a dietary supplement presents an unreasonable
risk of illness or injury, the agency performs a risk/benefit analysis
to ascertain whether the risks of the product outweigh its benefits.
The risks and benefits of a dietary supplement must be evaluated in
light of the claims and directions for use in the product's labeling
or, if the labeling is silent, under ordinary conditions of use
(section 402(f)(1)(A) of the act). Labeling claims for dietary
supplements must be substantiated. Unless the manufacturer has
substantiation that a labeling claim promoting a dietary supplement for
a purported benefit is truthful and non-misleading, the claim misbrands
the product (See section 403(a)(1) and 403(r)(6) of the act. We note
that the standards for substantiating the efficacy of a drug for a
labeled indication (i.e., the generally recognized as effective (GRAE)
standard for OTC monograph ingredients and the substantial evidence
standard for new drugs) do not apply to dietary supplements.
Substantiation of a benefit may not be necessary to lawfully market
a dietary supplement if its labeling does not include a claim, and the
product poses little or no risk. In weighing risks and benefits to
determine whether dietary supplements containing ephedrine alkaloids
present an unreasonable risk under section 402(f)(1)(A) of the act, we
considered only known and reasonably likely benefits, not speculative
benefits. A reasonably likely benefit is one that is supported by a
meaningful totality of the evidence, given the current state of
scientific knowledge, though the evidence need not necessarily meet the
approval standard for a prescription drug.
Although Congress placed the burden on FDA to show ``unreasonable
risk,'' once a danger is identified, we do not believe that Congress
intended us to delay action until double-blind, placebo-controlled
clinical studies could be conducted or that no action be taken if such
clinical studies are infeasible or unethical (see the response to
comment 19 of this document). While such studies are the ``gold
standard'' for determining effectiveness, they are not always available
for dietary supplements because DSHEA does not require companies to
conduct such studies before marketing a dietary supplement. DSHEA also
does not require postmarketing safety and adverse event reporting from
dietary supplement manufacturers. Accordingly, FDA is relying on the
available scientific data and literature to support its conclusion that
dietary supplements containing ephedrine alkaloids present an
``unreasonable risk.'' The government's burden of proof for
``unreasonable risk'' can be met with any science-based evidence of
risk and does not require a showing that the substance has actually
caused harm in particular cases.
For example, there is clear scientific evidence that a sustained
increase in blood pressure increases the risks of cardiovascular
disease (Refs. 29, 29a, and 30). Thus, a dietary supplement that caused
a sustained rise in blood pressure across the population would increase
the risk of cardiovascular events including stroke, heart attack, or
death to that population. Even risks that
[[Page 6799]]
may not be detectable in small studies or studies of short duration
(which are not designed to detect such risks at a statistically
significant level) could, over time, and on a population-wide basis,
result in thousands of adverse health events.
In making a determination, we consider studies using closely
related products. In considering the risks of a product, such as
dietary supplements containing ephedrine alkaloids, it is appropriate
to consider the safety of closely related products, such as those with
the same active ingredient (e.g., synthetic ephedrine products) or
closely related ingredients (such as other sympathomimetics) because we
would expect that dietary supplements containing ephedrine alkaloids
will exhibit pharmacological effects similar to those other products
and, therefore, pose similar risks. It is more difficult to extrapolate
conclusions regarding the benefits between an ephedrine drug product
and a dietary supplement containing ephedrine alkaloids since the
ephedrine drug product is a well defined product with a known dose of
ephedrine, while in the latter there is a complex mixture with,
possibly, an unknown quantity of ephedrine plus other ephedrine
alkaloids, and sometimes other active ingredients, many of which may
not be fully characterized. We would need to know how the two products
compare with regard to systemic delivery of ephedrine (e.g., the
pharmacokinetics profile) to make any judgments about comparable
benefits of the two products. If ephedrine pharmacokinetics were the
same in a synthetic and plant-derived product and there were no
ingredients or components other than ephedrine, one might conclude that
the plant-derived and synthetic products would behave similarly. In
actual fact, that is not the case because plant derived ephedra
products contain other ephedrine alkaloids in addition to ephedrine
itself (e.g. pseudoephedrine, methylephedrine, and others listed in
section I.B of this document). Moreover, if there were other active and
inactive ingredients in the plant-derived product, their properties
would need to be explored.
In evaluating whether dietary supplements containing ephedrine
alkaloids present an unreasonable risk, we looked at the seriousness of
the risks and the quality and persuasiveness of the totality of the
evidence to support the presence of those risks. We then weighed the
risks against the importance of the benefits and the quality and
persuasiveness of the totality of the evidence to support the existence
of those benefits. We give more weight to benefits that improve health
outcomes, especially in the long term, than to benefits that are
temporary or rely on subjective measures such as feeling or looking
better. For example, sustained, long-term weight loss in an obese or
overweight person is a much more important benefit than short-term
weight loss because long-term weight loss in these individuals reduces
the risk of serious morbidity and mortality (e.g., heart attacks and
strokes), while short-term weight loss does not.
In sections V.B, C, and D of this document, we describe the
evidence FDA evaluated to reach its determination that dietary
supplements containing ephedrine alkaloids present an unreasonable risk
of illness or injury.
(Comment 19) Many comments stated that any assessment of
unreasonable risk must be based on sound science. Several comments
stated that a conclusion about the safety and efficacy of dietary
supplements containing ephedrine alkaloids is premature and that
additional prospective or retrospective case controlled studies are
needed to determine causality. A few comments recommended that FDA,
NIH, or other parts of the federal government conduct such research to
address unresolved issues of causation. Another trade association urged
the government to collaborate with industry to design future controlled
studies. Several of these comments cited RAND in support of the need
for further research. Several comments noted that the National Center
for Complementary and Alternative Medicine/NIH Working Group evaluated
the RAND report and suggested a multi-site case-control study to assess
the risks associated with these products, although it stated that such
a study would take 4 to 8 years and cost $2 to $4 million per year
(Ref. 31).
In contrast, several comments asserted that conducting clinical
trials of ephedrine alkaloids would be unethical in light of the risks
to the human subjects. A professional association stated that FDA
regulations that govern drug development and approval would not allow
such research, given the absence of information to suggest a benefit
that would outweigh the risks. A few comments suggested that any study
that could be approved by a human subjects committee would be required
to exclude patients at risk and therefore, would not be useful in
evaluating risk when the products are taken by the general population
without medical supervision. Other comments expressed concern that the
additional research recommended by RAND would delay efforts or render
it virtually impossible to safeguard public health.
(Response) We recognize the value of properly conducted clinical
trials to answer questions regarding the safety and effectiveness of
FDA-regulated products. It is not clear, however, that clinical trials
to evaluate the adverse effects of ephedrine alkaloids can be
conducted. It would not be ethical to study the arrhythmogenic
potential of ephedrine alkaloids in patients with coronary artery
disease, the adverse effects of ephedrine alkaloids in people with
heart failure, or the consequences of raising blood pressure in various
populations. Moreover, there is now sufficient evidence, generated
through multiple sources, including clinical trials, published
literature, and other information, to reach the conclusion that dietary
supplements containing ephedrine alkaloids have effects on blood
pressure and other pharmacological risks that predict adverse effects
in users. After considering the best available information, we conclude
that these products present an unreasonable risk because the benefits
that may result from use of these products are outweighed by the risks
associated with such use (see discussion in section V.D of this
document). Because of the nature of these risks, we do not believe it
is appropriate to delay action until further clinical studies can be
conducted to evaluate the safety of dietary supplements containing
ephedrine alkaloids in the general population. We would, however,
support the conduct of clinical investigations (carried out under the
Investigational New Drug (IND) regulations with careful screening to
exclude subjects at risk and careful safety monitoring during the
trials) that examine the safety and efficacy of ephedrine alkaloids,
with or without caffeine, as drugs such as for the treatment of obesity
(see 21 CFR part 312).
(Comment 20) Two comments stated that there is an accepted
scientific methodology for determining whether, and at what level, a
food additive, dietary ingredient, OTC or prescription drug, or
biologic may be hazardous to human health. The stated components of
this methodology include reviews of the following reports: (1) The
existing scientific literature on the substance, to determine what is
known about the substance's risk, particularly at the levels to be used
in a product; (2) clinical studies involving the substance; (3)
available animal studies on the substance and, if necessary, the
conduct of additional studies; and (4) adverse event reports caused by
the substance.
[[Page 6800]]
In addition, the methodology includes a determination of whether
individuals who consume the products suffer from a statistically
significantly greater number of adverse (or beneficial) events than
those who do not. One comment stated that the absence of premarket
approval authority for dietary supplements does not preclude reliance
on traditional methods of evaluating safety when making a decision
about levels that are not safe.
(Response) We do not agree with the comments stating that there is
a single accepted method of evaluation to determine when a food
ingredient or dietary ingredient in a dietary supplement presents a
hazard to the public health. In any evaluation of the risks presented
by a substance in a product in the marketplace, the method of
evaluating the risk must be applied on a case-by-case basis that is
based on the available data concerning the substance being evaluated.
We believe that our method of evaluation for ephedrine alkaloids is,
however, consistent with that used for other substances. The scientific
methodology we used to evaluate the risks associated with the use of
dietary supplements containing ephedrine alkaloids consisted of a
review and evaluation of the available scientific literature (including
literature on pharmacology), clinical studies, published case reports,
and other data, including adverse event reports. This is the same type
of scientific methodology that is applied in the evaluation of adverse
effects associated with other FDA-regulated products (Ref. 32), and
includes most of the steps listed in the comments summarized above.
(Comment 21) A number of comments focused on FDA's obligation to
ensure that its regulatory assessments are science-based. Two comments
raised concern regarding our compliance with a statutory provision
popularly known as the Data Quality Act (section 515 of the
Consolidated Appropriations Act, 2001, Public Law 106-554, 44 U.S.C.A.
3516 note). One comment stated that we are vulnerable to challenge
under the Data Quality Act because there is a disconnect between our
proposed actions and the conclusions of the RAND report. Another
comment pointed to our related guidance entitled ``Guidelines for
Ensuring the Quality of Information Disseminated to the Public''
(http://www.hhs.gov/infoquality/fda.html[numsign]i). FDA's guidance,
which describes how we intend to meet our obligations under the Data
Quality Act and the implementing Office of Management and Budget (OMB)
guidelines, states that we are committed to ensuring that our
regulatory decisions are based on objective information and notes our
commitment to using the best available science conducted in accordance
with sound and objective scientific practices, including peer reviewed
science and supporting studies when available. This comment also cited
the Center for Food Safety and Applied Nutrition's report ``Initiation
and Conduct of All `Major' Risk Assessments within a Risk Analysis
Framework'' (http://www.cfsan.fda.gov/dms/rafw-toc.html), which
similarly stresses the importance of data quality and scientific
objectivity in regulatory decisionmaking. Finally, this comment
suggested that in evaluating the safety of dietary supplements
containing ephedrine alkaloids, we should apply a rigorous scientific
standard such as that used to evaluate whether a new drug application
(NDA) should be approved or whether a health claim should be authorized
under the significant scientific agreement standard (See Sec.Sec.
314.125 and 314.126) (NDAs); Guidance for Industry: Significant
Scientific Agreement in the Review of Health Claims for Conventional
Foods and Dietary Supplements (http://www.cfsan.fda.gov/dms/
ssaguide.html) (health claims).
(Response) We agree that we have an obligation to base regulatory
assessments, including our regulatory assessment of the safety of
dietary supplements containing ephedrine alkaloids, on sound science.
We have spent a great deal of time and effort compiling and evaluating
the best available scientific evidence relevant to this rulemaking, and
our decision is based on a careful, objective analysis of the most
current information, including peer reviewed studies. In considering
whether dietary supplements containing ephedrine alkaloids present an
unreasonable risk, we considered evidence from three principal sources:
(1) The well-known, scientifically established pharmacology of
ephedrine alkaloids; (2) peer-reviewed scientific literature on the
effects of ephedrine alkaloids; and (3) the adverse events (including
published case reports) reported to have occurred following consumption
of dietary supplements containing ephedrine alkaloids. We believe that
this final rule, and the data considered, are consistent with the
principles set forth in the Data Quality Act and related guidances
cited in the comments. We do not agree, however, that we should apply
the same standard of scientific proof to a determination of
adulteration under section 402(f)(1)(A) of the act, the ``significant
or unreasonable risk'' provision, as we would apply to a decision
whether to approve an NDA or authorize a health claim under other
provisions of the act. Although our decision on dietary supplements
containing ephedrine alkaloids must be based on sound science, that
decision is not subject to, and need not meet, the very specific
evidentiary requirements set out in the new drug and health claim
provisions of the act (See 21 U.S.C. 355(d) and 21 U.S.C.
343(r)(3)(B)(i)).
B. What Are the Known and Reasonably Likely Risks Presented by Dietary
Supplements Containing Ephedrine Alkaloids?
1. Pharmacology
We have reviewed numerous studies and other data related to the
safety of dietary supplements containing ephedrine alkaloids. Evidence
about the pharmacology of ephedrine alkaloids--as well as other
evidence in the docket--shows that these products present a risk of
serious adverse health effects. Information submitted to the docket in
an effort to establish the safety of these products is inadequate to
rebut the evidence of risk.
(Comment 22) Several comments focused on the known pharmacological
and toxicological effects of ephedrine/ephedra on the cardiovascular
and nervous systems, explaining that ephedra contains vasopressor
amines that excite the heart and constrict the blood vessels, which in
turn increases heart rate and raises blood pressure. The comments
contended that, because of these effects, adverse events such as
hypertensive episodes, arrhythmias (abnormal heart rhythms), heart
attacks, seizures, and strokes can be anticipated and expected when
millions of people are exposed to such products. Various comments
maintained that dietary supplements containing ephedrine alkaloids have
the same pharmacological and toxicological activity as prescription and
OTC ephedrine alkaloid drugs and, thus, present the same risks. One
comment emphasized that Chen and Middleton (Ref. 33) warned about
ephedrine alkaloid-induced thromboembolism (blood clots that travel in
the body) in 1927 and thereafter, reports of toxicity appeared in the
medical literature, accompanied by warnings against indiscriminate use
by doctors and sale to consumers. These early reports are relevant to
current reports of myocardial infarctions (heart attacks) and stroke
associated with products containing ephedrine alkaloids.
[[Page 6801]]
One comment stated that ephedra presents a danger of prolonged
bleeding in those who undergo surgery, and that patients and doctors
may not be aware of this potential complication. Another comment cited
a review article (Ref. 2) that described myocardial depression
occurring with repeated dosing of ephedrine, and cited a reference from
a pharmacological textbook documenting ephedrine's tendencies to cause
atrial and ventricular arrhythmias. Another comment suggested that we
should not ignore the other ingredients commonly found in dietary
supplements containing ephedrine alkaloids, such as caffeine,
laxatives, and diuretics, because these ingredients can alter
electrolyte levels and increase the risk of arrhythmias. One comment,
citing a study by Haller et al., contended that the apparent causal
role of ephedrine alkaloids in severe adverse effects could be related
to the additive stimulant effects of caffeine (Ref. 34). One comment
submitted by a manufacturer attributed the good safety record of its
product to, among other reasons, the absence of caffeine and other
stimulants.
(Response) We agree that dietary supplements containing ephedrine
alkaloids present risks of adverse physiological and pharmacological
effects. Based on the best available scientific data and the known
pharmacology of ephedrine alkaloids and other sympathomimetics,
ephedrine alkaloids--including dietary supplements containing ephedrine
alkaloids--pose short-term and long-term risks. This is clearest in
long-term use, where increased blood pressure in any population will
clearly increase the risk of stroke, heart attack, and death, but there
is also evidence of increased risk from shorter-term use in patients
with heart failure or underlying coronary artery disease.
Ephedrine alkaloids are members of a large family of
sympathomimetic compounds that include dobutamine and amphetamine.
Members of this family increase blood pressure and heart rate by
binding to alpha- and beta-adrenergic receptors present in many parts
of the body, including the heart and blood vessels (Refs. 35, 36, and
37). These compounds are called sympathomimetics because they mimic the
effects of epinephrine and norepinephrine, which occur naturally in the
human body. In addition to their direct pharmacological effects, many
of these compounds also stimulate the release of norepinephrine from
nerve endings. The release of norepinephrine further increases the
sympathomimetic effects of these compounds, at least transiently.
Sympathomimetic effects raise three concerns. First, sympathomimetics
can induce cardiac arrhythmias in susceptible people, such as those
with underlying coronary artery disease. Second, increased mortality
has been observed in patients with congestive heart failure who were
treated with sympathomimetic drugs, such as beta-agonists (early
studies using such drugs as albuterol led to adverse outcomes) and
xamoterol (Ref. 38), as well as phosphodiesterase inhibitors, which
potentiate (increase the effect of) the effects of beta-agonists,
including milrinone (Ref. 39) and enoximone (Ref. 40). The studies that
showed these adverse effects occurred in about 3 months of product use.
Third, sympathomimetics can raise blood pressure (Ref. 41).
Based on clinical data, the ephedrine alkaloids present in dietary
supplements would be expected to have the same or similar effects as
other sympathomimetics on heart rate and blood pressure. Controlled
clinical trials using products containing ephedrine alkaloids confirm
their typical sympathomimetic effects. Single-dose studies of dietary
supplements containing ephedrine alkaloids show that these products
cause increases in both heart rate and blood pressure in healthy
subjects (Refs. 42, 43, and 44). In one such study of a dietary
supplement containing ephedrine alkaloids, the peak increase in blood
pressure following a single oral dose of ephedrine alkaloids and
caffeine (20 mg/200 mg) was 14 millimeters of mercury (mm Hg) systolic
and 6 mm Hg diastolic, occurring about 2 hours after the single dose
was taken (Ref. 42).
The findings from these studies are complicated by the presence of
caffeine in the dietary supplements used because caffeine is also known
to have acute effects on blood pressure and heart rate. However, the
effect of caffeine on blood pressure is transient and is lost within 2
weeks of continued use (Refs. 45 and 46). Evidence that ephedrine
independently causes an increase in blood pressure when coadministered
with caffeine comes from two sources. First, there are studies in which
ephedrine and caffeine were tested separately so that their effects
could be compared. In a study by Jacobs et al., a group of healthy
subjects received ephedrine (E, 0.1 mg/kilogram (kg) orally), caffeine
(C, 4 mg/kg orally), the combination, or a placebo (P) (Ref. 47).
Although caffeine caused a small increase in systolic blood pressure
(average 3 to 6 mm Hg), ephedrine alone gave a 12 mm Hg effect, and
when added to caffeine, increased systolic blood pressure by an
additional 15 mm Hg (C+E = 156 +/- 29 mm Hg; E = 150 +/- 14; C = 141 +/
- 16; P = 138 +/- 14) (Refs. 47 and 48). Second, ephedrine has been
shown in a clinical study to increase blood pressure and heart rate
acutely when administered intravenously to children to maintain blood
pressure during surgery (Ref. 37). Therefore, these studies show a
blood pressure effect from ephedrine itself, independent of any
additional effect from caffeine.
In a multiple-dose controlled trial, Boozer et al. (2002) compared
the effects of a combination of ephedrine alkaloids (from Ephedra) and
caffeine (from kola nut) with placebo over a 6-month period in a highly
selected population of obese and overweight individuals, who were
carefully screened by medical history and medical evaluation to
eliminate cardiovascular and other acute or chronic disorders (Ref.
49). The study measured sitting blood pressure in the clinic using the
cuff method for all 6 months (at weeks 1, 2, 3, 4, and every 4 weeks
thereafter) of the study; these cuff measurements were not taken
throughout the day so they reflect only a snapshot of the blood
pressure at the time of measurement. The study also measured changes in
blood pressure throughout the day at weeks 1, 2 and 4 using an
automated blood pressure monitoring device (ABPM); the ABPM method
provides more frequent measurements of blood pressure and is,
therefore, better able to evaluate blood pressure effects over time.
The ephedrine alkaloids and caffeine-treated subjects did not show a
difference in the blood pressure measurements taken at the clinic, but
did show statistically significant higher average blood pressure
measurements over 24 hours at week 4 measured by ABPM (approximately 4
mm Hg for both systolic and diastolic blood pressure) when compared to
placebo treated subjects. The ABPM results are shown in a table in the
paper. The difference in blood pressure between the two groups
represented the sum of small downward changes in the placebo group
(compared to baseline) and small upward changes, or no change, in the
ephedra group. Boozer et al. reported numerous breakdowns of these data
(e.g., 6 a.m. to midnight and midnight to 6 a.m.) and characterized the
difference between the ephedra and placebo groups as small (about 3 mm
Hg) but for the most common ABPM measure, 24-hour value, the difference
was 4/4 mm Hg. The observation that this difference (shown in table 2
of the paper) (Ref. 49) reflected a fall in blood
[[Page 6802]]
pressure in the placebo group as much as a rise in blood pressure in
the ephedra group is not relevant. The only controlled and, therefore,
reliable observation is the comparison of the two groups. Small changes
from baseline can occur for a wide variety of reasons and are commonly
observed in placebo and treated groups. Therefore, the ABPM data are
important because they demonstrate that the effect of the ephedrine
alkaloids, including dietary supplements containing ephedrine
alkaloids, on blood pressure is not transient, but is still evident
after 1 month of continued exposure (when measured by ABPM) and,
therefore, would be expected to persist long term. The effect reported
in the Boozer, et al. (2002) study cannot be attributed to the caffeine
because the effect of caffeine on blood pressure (discussed previously)
is transient, and the acute effect of caffeine to increase blood
pressure is lost within 2 weeks of continued use (Refs. 45 and 46).
While some effects of sympathomimetics show tachyphylaxis (i.e.,
decrease in response following repetitive administration of a
pharmacologically active substance http://www.stedmans.com/)
tachyphylaxis usually occurs rapidly. (FDA has verified the Web site
address, but FDA is not responsible for any subsequent changes to the
nonFDA Web sites after this document publishes in the Federal
Register.) Therefore, we believe, based upon these data and our
experience, that the blood pressure effects of ephedrine alkaloids seen
after 4 weeks of continued use will persist.
The Boozer et al. (2002) study (Ref. 49) was reviewed at our
request by three outside scientific experts, Norman M. Kaplan, M.D.
(Ref. 50), Richard L. Atkinson, M.D. (Ref. 51), and Mark Espeland,
Ph.D. (Ref. 52). These experts were asked to give their independent,
scientific opinion of whether the study provides adequate data to
assess safety of ephedrine alkaloids and caffeine for weight loss--
considering, among other things, the design and duration of the trial
and subject selection--and whether further studies are needed. In
general, the experts concluded that the safety of ephedrine alkaloid
and caffeine containing products could not be established by this study
because the study used a highly selected population (i.e., carefully
screened by medical history and medical evaluation to eliminate
cardiovascular and other acute or chronic disorders) and had relatively
few subjects. One of the experts also concluded that the duration of
the study was inadequate to establish safety. In general, the reviewers
found that the results raised safety concerns. Dr. Kaplan, one of the
reviewers, raised the concern that the size of the change in blood
pressure observed with ABPM, when applied to a large population, could
translate into a significant increase in the incidence of strokes and
heart attacks. Dr. Kaplan's concern reflects the potential consequence
of long-term use of ephedra (i.e., the consequence of a population
increase in blood pressure). A short-term increase (e.g., 1 to 2
months) would not be expected to have such an effect. Approximately one
in four adults has high blood pressure. Of those with high blood
pressure, 31 percent are unaware that they have it (Ref. 53). A
relative increase in blood pressure in any population, even individuals
with ``normal'' blood pressure, will increase the risk of heart attack,
stroke, and death in that population (Refs. 29, 29a, and 54).
The extremely high prevalence of diagnosed and undiagnosed
hypertension in the U.S. population and the likelihood that blood
pressure in obese patients is already elevated make the 4 mm Hg effect
shown by the Boozer et al. (2002) study (Ref. 49) one of great concern.
Reductions in blood pressure of this magnitude (i.e., around 4 mm Hg
diastolic or systolic) are clearly associated with substantial long-
term reductions in the occurrence of heart attack, stroke and death, as
seen in meta-analyses of antihypertensive drug trials (Refs. 55 and
56). While these trials were conducted in patients with hypertension,
increasing blood pressure in any population, even in individuals with
``normal'' blood pressure, will increase the risk of cardiovascular
disease (Ref. 29).
Epidemiological studies support a graded and continuous
relationship between increased blood pressure and risk of stroke, heart
attack, and sudden death, even when the increase is within the normal
range (i.e., less than 140 mm Hg systolic and less than 90 mm Hg
diastolic) (Refs. 29 and 30). This indicates that many people would be
at an increased risk with long-term use of dietary supplements
containing ephedrine alkaloids. Studies of hypertension treatments
suggest that this increase in risk would occur fairly quickly in
hypertensive individuals. Anti-hypertensive drugs that lower blood
pressure by 4 to 6 mm Hg have been shown to significantly decrease the
occurrence of cardiovascular morbidity (stroke, heart attack) and
mortality (Refs. 55, 57, and 58). This effect is evident within 6 to 12
months in large outcome studies (Refs. 29 and 30). FDA is concerned
about the adverse health effects that can occur with the use of agents
that raise blood pressure, such as dietary supplements containing
ephedrine alkaloids, for short- or long-term use. Even in the case of a
controlled clinical trial of a possible hypertension treatment where
subjects are closely monitored, we advise sponsors to limit the length
of time subjects can be in a placebo/untreated group to about 8 weeks
to minimize their exposure to cardiovascular risks from the absence of
treatment.
As noted previously, the pharmacological effects of ephedrine
alkaloids also present increased short-term risks of adverse health
events in susceptible populations. For example, there is evidence from
peer-reviewed scientific literature that a wide range of drugs with
sympathomimetic activity, including beta-agonists, phosphodiesterase
inhibitors, and dobutamine, have adverse effects (increased mortality
due to heart failure and sudden death) in patients studied with
congestive heart failure. These effects have been seen in relatively
short-term studies (Refs. 59, 60, and 61) Similarly, there are studies
that document that people with coronary artery disease are more
susceptible to the well-known pro-arrhythmic effects of
sympathomimetics (Refs. 62, 63, and 64) The occurrence of such an
arrhythmic event is not one that requires prolonged exposure but would
represent a risk associated with each use, including the first. Many
individuals are unaware that they have coronary artery disease or early
heart failure because these conditions may not cause prominent symptoms
until later in the course of these conditions. As a result, we are
concerned that such individuals will not know that they are at an
increased risk for developing significant cardiovascular adverse events
from even short-term use of dietary supplements containing ephedrine
alkaloids. Overweight and obese individuals are particularly prone to
hypertension, coronary artery disease, and/or heart failure, as
overweight and obesity are associated with these conditions (Refs. 65
and 66). These conditions may not manifest clinically until later in
the course of the condition and, therefore, individuals, including
overweight and obese individuals, may be unaware they have these
conditions. As a population, the overweight and obese are, thus, at a
greater risk even from short-term use of sympathomimetics.
As summarized previously, the comments cited certain literature
suggesting the possibility of additional adverse effects of ephedrine
alkaloids,
[[Page 6803]]
such as prolonged bleeding in those who undergo surgery. Given the
clear scientific evidence of this cardiovascular risks presented by
dietary supplements containing ephedrine alkaloids, we have not relied
on these other possible adverse effects noted in the comments in our
determination of unreasonable risk.
(Comment 23) Various comments did not agree that there are risks
with products containing ephedrine alkaloids and stated the opinion
that cardiovascular side effects associated with products containing
ephedrine alkaloids in several blinded studies were not significantly
different in control and treatment groups. Several comments maintained
that there is no evidence from clinical studies that ephedrine
``supplementation'' increases peak heart rate, peak blood pressure, or
the prevalence of cardiac arrhythmias. Another comment contended that
``clinically relevant doses'' of ephedra have no clinically significant
effect on pulse or blood pressure, and produce no measurable
alterations in myocardial function. A number of comments noted that
changes in heart rate and blood pressure are transient and similar to
those produced by exercise. Several comments stated that the effects of
ephedra combined with caffeine on blood pressure are modest and
generally subside over the first few days of use. Other comments stated
that, although dietary supplements containing ephedrine alkaloids have
a relatively high incidence of subjective and cardiovascular side
effects with first use, the side effects diminish with continued use
due to tachyphylaxis. Several comments noted that the literature,
including the obesity studies we cited in the June 1997 proposal (Refs.
36 and 67 through 80), indicated that tachyphylaxis sets in within a
few days, at the most a few weeks, and results in a dramatic decrease
in the likelihood of adverse events. Another comment suggested that
pharmacological studies showed that peak ephedrine levels are reached
within 1 to 4 days and that no further accumulation occurs thereafter.
Another comment suggested that this fact means ephedrine alkaloids pose
no risk of long-term toxicity.
One comment noted that ephedrine alkaloids are not toxic in the
classic sense, that is, do not cause organ changes or damage to the
metabolism. Other comments suggested that the available pathology data
do not show any pattern consistent with ephedrine alkaloids as a cause
of death.
(Response) We do not agree that ephedrine alkaloids pose no risk of
adverse consequences. The suggestion that the cardiovascular effects of
ephedrine alkaloids persist for only a few days is not supported by the
Boozer et al. (2002) study (Ref. 49), which demonstrated a higher blood
pressure (compared with placebo) at the end of 1 month of therapy (Ref.
80a). This difference was observed when blood pressure was measured
throughout the day, using ABPM, but not with cuff blood pressure
measurements (a less sensitive measure). This difference in results
using different measurement methods may have confused some readers and
led them to conclude that ephedrine alkaloids do not have a clinically
meaningful effect on blood pressure. The fact that an effect on blood
pressure (as measured using ABPM, which follows measurements throughout
the day) was still present at 1 month strongly indicates that
tachyphylaxis to the effects of ephedrine does not occur. As discussed
in the response to comment 22 of this document, tachyphylaxis tends to
occur rapidly, as with caffeine, whose blood pressure raising effect is
lost within 2 weeks. Therefore, FDA does not agree with the comments
expressing assurances that adverse effects will disappear with
continued use of ephedrine alkaloids because of tachyphylaxis.
Additionally, some of the studies cited by the comments apparently
measured cuff blood pressure only around the time of dosing, when
minimal serum concentrations of ephedrine alkaloids and effects on
blood pressure would be expected. Absence of an effect at this time
cannot be seen as evidence that ephedrine alkaloids do not increase
blood pressure.
The suggestion that ``clinically relevant'' or ``clinically
significant'' doses of ephedrine have no effects on blood pressure is
unsupported by the available data. What constitutes a ``clinically
relevant or significant'' dose is undefined (and unlikely to be
definable given the nature of the available efficacy data for ephedrine
alkaloids). The difficulties in using the available clinical data to
obtain such reassurance with regard to the safe use of ephedrine are
discussed in the response to comment 26 of this document.
We do not agree that the clinical studies establish that ephedrine
does not have adverse pharmacological and clinical effects. The
published controlled studies of the use of ephedrine alkaloid products
for weight loss cited by these comments cannot establish the safety
profile of these products. First, many of the most serious risks, such
as strokes or heart attacks (consequences of elevated blood pressure),
arrhythmias, or worsened heart failure, are relatively infrequent or
are delayed and, therefore, will not be detected in studies using small
populations (such as under 100 patients per group) as these studies
did. Second, these studies often had other important design
limitations, such as lack of adequate controls (including the absence
of placebo groups in some studies), and inadequate information about
the causes that led to participants dropping out of the trial. In
addition, persons with known cardiovascular disease or cardiovascular
risks were usually excluded. Thus, these studies were not designed to
detect serious adverse effects in susceptible individuals, nor to
detect adverse effects that occur infrequently. As discussed in the
following paragraphs, these studies were also not adequately designed
to assess blood pressure effects. Given these limitations, it is not
surprising that these published studies do not report serious adverse
events (Refs. 21, 22, 50, 52, and 81).
These trials also would not have been able to detect effects on
blood pressure because of other design limitations. For example, when
sponsors of drug products seek to detect a drug-induced decrease in
blood pressure in patients with hypertension, the trial is specifically
designed to perform the following functions: (1) Assess the blood
pressure effects at both peak and trough levels of the drug in the
blood, and (2) measure blood pressure in a consistent and reproducible
manner. This typically requires the enrollment of at least 100 patients
to detect a difference from placebo of around 4 to 6 mm Hg systolic,
multiple measures at each time point and careful attention to how blood
pressure is measured. These design features are either lacking or not
described in the publications cited by the comments summarized above,
significantly limiting the trials' ability to detect any differences
between the treatment and placebo groups with regard to blood pressure
or heart rate. With regard to the timing of the measurement, the blood
pressure measures appear to have been made at (or shortly after) the
administration of the product containing ephedrine for almost all of
the published trials. Absorption of the new dose would be minimal or
incomplete and the dose taken the day before (8 to 12 hours earlier)
would have been substantially removed from the circulation, given
ephedrine's approximately 4-hour half-life. Blood levels of ephedrine
would
[[Page 6804]]
thus be at or near their lowest values of the day (``trough level''), a
time when minimal effects on blood pressure would be anticipated.
Measurements made only at trough level might well miss a significant
effect on blood pressure that would have been seen at or near peak
concentrations of ephedrine. Thus, although some published studies on
the cardiovascular effects of ephedrine (especially blood pressure)
over a period of weeks or months have reported little or no effect of
ephedrine on blood pressure and a variable effect on heart rate, these
studies are severely limited in their ability to establish safety
because of the clinical trial design limitations (Refs. 81a, 81b, and
81c), such that the true effects of ephedrine on heart rate and blood
pressure cannot have been adequately assessed.
We do not agree with the comments that state that ephedrine
alkaloids are not toxic because they do not induce specific organ
pathology. Persistently elevated blood pressure can result in defined
cardiovascular toxicity (Refs. 29, 29a, and 54), as can ephedrine's
sympathomimetic effects in people with coronary artery disease or heart
failure, but the kinds of damage seen in humans from these effects
would look the same as similar damage that occurs from the underlying
disease or from raised blood pressure or arrhythmia due to another
cause.
(Comment 24) A number of comments discussed the relevance of PPA to
regulatory decisions on dietary supplements containing ephedrine
alkaloids. Several comments stated that PPA is a metabolite of
ephedrine. Various comments contended that ephedrine and PPA are both
partial agonists and that adverse events associated with dietary
supplements containing ephedrine alkaloids are of the same type and
greater in number than those associated with PPA, which was voluntarily
withdrawn from the U.S. market for safety reasons. Other comments
maintained that we should not use PPA data to support the hazards of
dietary supplements containing ephedrine alkaloids. Several such
comments stated that because PPA differs in pharmacological,
pharmacokinetic, and pharmacotoxic effects from ephedrine or
pseudoephedrine, it is scientifically inappropriate for us to assume
that all ephedrine alkaloids are equivalent. Other comments asserted
that the various isomers of ephedrine alkaloids have different actions,
different favorable and adverse effects, different activation of
receptors, and different effects on human tissues. Several comments
indicated that norephedrine (an ephedrine alkaloid that makes up one
component of PPA) is a metabolite of ephedrine and that interactions of
the multiple ephedrine alkaloids in Ephedra and other botanicals and
their in vivo metabolites should be considered in a safety evaluation
of these ingredients and products containing them.
A few comments asserted that the Hemorrhagic Stroke Project (HSP)
(Ref. 19) was not designed to assess ephedra exposure. These comments
maintained that the HSP is limited by significant issues relating to
observation bias, selection bias, and confounding. One comment
complained that we reopened the ephedra docket requesting comment on
the HSP, but we did not place in the docket, or request comment on, the
many published and unpublished clinical studies submitted by one trade
organization to support PPA's safety. The comment asserted that our
review of the pharmacology of ephedrine alkaloids did not include most
of the pivotal information on PPA submitted to us by the Consumer
Healthcare Products Association (CHPA). Another comment expressed the
view that, in our review of safety data related to ephedra, we should
avoid relying on safety data concerning other ingredients.
(Response) The substance, l-norephedrine, also known as (-)-
norephedrine, refers to the isomeric portion of PPA that occurs
naturally in Ephedra and as a metabolite of ephedrine in the body. We
agree that the l-norephedrine in racemic PPA is a metabolite of
ephedrine, and further that ephedrine and its metabolites have potent
vasoactive properties, reinforcing the view that dietary supplements
containing ephedrine alkaloids have the pharmacological properties
described in the response to comment 22 of this document. These
properties, in turn, are linked to predictable adverse clinical
outcomes both in the general population (e.g., increased blood
pressure) and in susceptible populations (e.g., cardiac arrhythmias).
Although there are some similarities between PPA and ephedrine, there
are also differences. PPA shows tachyphylaxis to rises in blood
pressure within approximately 24 hours and usage has been linked to
hemorrhagic strokes (bleeding strokes due to a ruptured blood vessel).
Ephedrine does not show such tachyphylaxis. In addition, use of
ephedrine has been associated with ischemic strokes (a blood clot
blocking off an artery causing a lack of oxygen to portions of the
brain), but not hemorrhagic strokes. The major alkaloid in most dietary
supplements containing ephedrine alkaloids is generally ephedrine, and
not norephedrine (Ref. 82).
Therefore, we have not relied on the HSP or spontaneous reports of
hemorrhagic stroke in patients receiving PPA for any of our conclusions
about the risks of ephedrine alkaloids, and data regarding PPA is not
as informative for drawing conclusions about the benefits and risks of
dietary supplements containing ephedrine alkaloids as data on
ephedrine. Of course, those supplements that contain meaningful amounts
of PPA would pose additional serious risks expected from the use of
PPA-containing products, such as hemorrhagic strokes. This adverse
event can occur in healthy individuals with one dose of PPA. Reopening
the docket to request comment on these data is unnecessary as we have
not relied on the data for our determination in this final rule.
(Comment 25) One comment stated that l-ephedrine is both a direct
and indirect-acting isomer with both alpha- and beta-agonist activity,
while d-pseudoephedrine acts indirectly on both receptors. PPA, which
is racemic (i.e., contains both the (+) and (-) forms of the chemical),
is a direct and indirect agonist for alpha-receptors but has weaker
beta-receptor activity. The comment suggested that ephedrine,
pseudoephedrine, and PPA elevate blood pressure, but only l-ephedrine
and d-pseudoephedrine increase heart rate. The comment cited Chua and
Benrimoj (Ref. 83) stating that d-pseudoephedrine has half of the
bronchodilator activity compared to l-ephedrine and one-quarter of the
vasopressor effect. The comment argued that we cannot use the
pharmacokinetic and toxicokinetic properties of any isomer to predict
that of other ephedrine isomers.
(Response) Given that Ephedra and other botanicals used as dietary
ingredients contain a mixture of ephedrine alkaloids, and given the
small database on the supposed selective effects of the isomers, we
cannot draw any reassurance from the possibility that one alkaloid has
more or less of an effect on the vasculature (or organ systems) than
another alkaloid. Further, the reported differences in receptor binding
affinity or other in vitro tests cannot eliminate concern about the
effects of ephedrine alkaloids in humans, because there is clinical
evidence that ephedrine alkaloids have important pharmacological
effects (e.g., increased blood pressure, heart rate) that persist,
particularly in the case of ephedrine, through at least 1 month of use.
As noted previously in this document, the
[[Page 6805]]
major alkaloid in most dietary supplements containing ephedrine
alkaloids is generally ephedrine (Ref. 82). The comments pointing to
evidence of differences in the effects of different ephedrine alkaloids
do not provide a basis to conclude that dietary supplements containing
ephedrine alkaloids do not present an unreasonable risk of illness or
injury.
(Comment 26) Some comments argued that the scientific literature
indicates that single doses of ephedrine up to 60 mg generally do not
increase blood pressure (Ref. 83). Other comments cited a handbook of
intravenous drug therapy for nurses that states that ephedrine is of
low toxicity. One comment stated that the scientific literature
describing the effects of ephedrine in doses of 50 to 150 mg does not
support the contention that ephedrine in dosages of 50 to 150 mg per
day would represent a health hazard. Many comments stated that reviews
of the literature and other data by independent experts reflect the
scientific consensus that ephedrine alkaloids at 25 mg per dose are
safe. One comment cited a clinical study of 98 elderly patients
undergoing hip surgery who received 0.6mg/kg ephedrine by intramuscular
injection. One out of 48 patients in the placebo group and two out of
50 in the ephedrine group experienced increased heart rate or increased
systolic blood pressure greater than 20 percent from baseline. The
comment concluded that the dosages used are greater than the dosages
found in any dietary supplement containing ephedrine alkaloids and that
the results of the study are consistent with the conclusion that, as
also asserted by other comments, no significant injury has been clearly
associated with dietary supplements containing ephedrine alkaloids when
used as directed.
We received numerous other comments dealing with the issue of
``safe'' doses for ephedrine alkaloids in dietary supplement products.
Many expressed the view that low doses of ephedrine alkaloids in
dietary supplements do not pose a safety concern and should remain on
the market.
(Response) We do not agree that the scientific literature indicates
that there is a dose of ephedrine or ephedrine alkaloids that does not
present a risk of adverse events. Although dosages vary in dietary
supplements containing ephedrine alkaloids, most products are labeled
with 20-25 mg ephedrine alkaloids per recommended serving and 100-150
mg ephedrine alkaloids per day. Some of the doses described in the
comments as safe (50 to 150 mg ephedrine alkaloids per day) are in the
range studied by Boozer et al. (90 mg ephedrine alkaloids per day)
(Ref. 49) and, thus, could cause an increase in blood pressure, a
significant health concern (see previous discussion). We also do not
agree that some lower dose of ephedrine has been demonstrated not to
increase blood pressure and heart rate. The relationship between a
given dose of ephedrine and changes in heart rate and blood pressure
has been poorly characterized, although it is clear that ephedrine is
capable of increasing both. As discussed in the response to comment 23
of this document, the published studies that have found no effects on
blood pressure and/or heart rate have had methodological deficiencies
that limited their ability to detect such changes. With respect to the
clinical study of 98 elderly patients, the failure to find serious
adverse events is understandable, as the study was designed to
demonstrate that intramuscular ephedrine was effective to prevent
hypotension related to spinal anesthesia. The concern that led to the
study was adverse events related to an expected decrease in blood
pressure resulting from the anesthesia. As would be expected based on
the pharmacology of ephedrine, the study showed that ephedrine is
effective in maintaining blood pressure in patients receiving spinal
anesthesia.
We do not agree with comments that suggest that low doses of
ephedrine alkaloids in dietary supplements do not present an
unreasonable risk and should remain on the market. Because this issue
was raised in comments responding to the June 1997 proposal, we
commissioned a scientific review that was placed in the 2000 docket
(Refs. 84 and 85). This review concluded that a ``safe dose'' of
ephedrine alkaloids cannot be identified. The review determined that
even ``a dose of 1.5 mg every 4 hours (a daily dose of 9 mg) would
produce cardiovascular effects that may be dangerous alone, or in
association with risk factors* * *'' (Ref. 84 at p. 6). We also note
that in the 1996 FAC meeting, several committee members stated that,
based on the available data, no safe level of ephedrine alkaloids could
be identified for use in dietary supplements (Ref. 86). Consequently,
they recommended removing dietary supplements containing ephedrine
alkaloids from the market (Ref. 87). Although the CANTOX Health
Sciences International (CANTOX) review attempted to establish a level
of ephedrine alkaloids at which there were no adverse effects, we do
not consider the information submitted sufficient to establish a
``safe'' dose (see discussion of CANTOX in the response to comment 32
of this document).
(Comment 27) Many comments raised the issue of the safety of
dietary supplements containing ephedrine alkaloids for use in sensitive
or special populations. A number of comments indicated that certain
individuals may be relatively more sensitive to the stimulant effects
of ephedrine alkaloids, and as a result, at greater risk for adverse
health consequences. One comment from a physician noted that he does
not recommend the use of ephedra products by pregnant women. Another
comment indicated a particular safety concern with the use of dietary
supplements containing ephedrine alkaloids in older persons; according
to the comment, many elderly persons take medications for which the use
of dietary supplements containing ephedrine alkaloids would be
contraindicated. Citing a survey that indicated that shift workers
frequently use stimulants, including ephedrine alkaloids, in
combination with coffee, depressants and/or pain relievers that contain
caffeine, one comment expressed the view that ephedrine alkaloids pose
a significant health risk to the shift worker population (Ref. 88). The
comment further submitted that 69 percent of shift workers are
overweight, that shift work is likely to involve physical labor, often
performed in hot conditions, and that these factors increase the risks
of adverse cardiovascular effects when shift workers use ephedrine
alkaloids. Other comments stated that the presence or absence of a
susceptible population cannot be determined with the available data.
Several comments stated that dietary supplements containing ephedrine
alkaloids are not for everyone, and consumers should consult a
physician prior to use if they have specified preexisting health
conditions.
(Response) We agree with the comments that expressed concern about
the effects of ephedrine alkaloids on susceptible populations and have
previously discussed long-term and short-term risks to susceptible
populations in the response to comment 22 of this document. There is
every reason to expect that certain populations will be more
susceptible to the adverse effects of ephedrine alkaloids and that many
such people will not be aware of their greater susceptibility. As noted
previously, people with coronary artery disease, early congestive heart
failure, and high blood pressure, all of which are more
[[Page 6806]]
common in obese individuals, are often unaware of these risk factors.
Thus, the recommendations contained in the comments regarding the
suitability of dietary supplements containing ephedrine alkaloids for
certain populations and the need to consult a physician if the consumer
has certain preexisting conditions are ineffective to mitigate the risk
that dietary supplements containing ephedrine alkaloids pose to these
susceptible populations.
(Comment 28) Several comments stated that warning labels on dietary
supplements containing ephedrine alkaloids are not sufficient to
protect the public health because many individuals are not aware they
have medical conditions or individual sensitivities that put them at
greater risk for experiencing serious adverse effects.
The comments stated that warnings are ineffective for individuals
who are not aware that they have disease conditions such as high blood
pressure or other cardiovascular diseases, hyperactive thyroid
function, undiagnosed cerebrovascular abnormalities, or a propensity
for cardiac arrhythmia, seizure or certain psychiatric disorders. The
same comments maintained that even small amounts of ephedrine alkaloids
can be potentially dangerous to otherwise healthy individuals who may
have a genetically predetermined sensitivity to ephedrine alkaloids or
other sympathomimetic agents. Other comments asserted that warning
labels are ineffective because serious adverse events have occurred
after the initial or first few uses.
(Response) We generally agree with the comments. Warning labels may
be beneficial when people are able to identify the risk factors about
which they are being warned. As explained in section V.B.3 of this
document, OTC drug products containing ephedrine or pseudoephedrine
bear warnings that they should not be used by certain populations.
Despite the identified risks of these products, we have determined that
the demonstrated health benefits for the labeled OTC drug uses outweigh
their risks for certain temporary, episodic disease uses when
appropriate warnings are contained in the product labeling. While
dietary supplements containing ephedrine alkaloids present the same
risks, there are no health benefits for the labeled uses sufficient to
outweigh their risks (see discussion in sections V.C and V.D of this
document). A more detailed discussion on why a warning label would be
insufficient to make the risks of dietary supplements containing
ephedrine alkaloids reasonable appears in section VI.A of this
document.
(Comment 29) A number of comments indicated that ephedrine
alkaloids could only be used safely under the supervision of a health
professional or that products containing ephedrine alkaloids should be
restricted to prescription use only. Reasons given for these opinions
included the potential for interactions between dietary supplements
containing ephedrine alkaloids and caffeine or other commonly available
products (predominantly drugs) that might not be identified by the
typical consumer. Other comments stated that consumers could not self
diagnose many of the conditions where the use of ephedrine alkaloids
would either be contraindicated or pose a potential safety concern.
In contrast, a physician who used dietary supplements containing
ephedrine alkaloids in his practice stated that he was as comfortable
with people using dietary supplements containing ephedrine alkaloids on
their own, as he was with people using an OTC drug product on their
own.
(Response) We generally believe that the risks posed by dietary
supplements containing ephedrine alkaloids when used continuously,
particularly in obese patients who may already have underlying
illnesses that can be aggravated by these products (such as
hypertension), cannot be adequately mitigated without physician
supervision. Sustained high blood pressure has significant
consequences, including increased risk of stroke, heart attack, and
death. As noted previously, even short-term use of dietary supplements
containing ephedrine alkaloids poses certain risks, such as arrhythmias
in patients with coronary artery disease. While we allow ephedrine and
pseudoephedrine in OTC drugs for temporary, episodic uses, such as the
temporary relief of symptoms (shortness of breath, tightness of chest,
and wheezing) of certain diseases (e.g., colds, allergies, previously
diagnosed bronchial asthma, colds, allergies) individuals who use
dietary supplements containing ephedrine alkaloids for reasons other
than to improve their health (e.g., to lose weight for improved
appearance) obtain no health benefits and at the same time are at risk
for the types of adverse events that can occur with both short and
long-term use of ephedrine alkaloids. As discussed more thoroughly in
section V.C.1 of this document, use for relatively short term weight
loss would give, at best, a weight loss of a few pounds, which would
not be sufficient to result in any health benefit. However, use for
weight loss is likely to be longer term, giving a sustained increase in
blood pressure in addition to the short-term risks. If these products
met prescription drug standards, then it is possible that the risks of
use for weight loss could be mitigated by a physician's evaluation of
the patient's medical history and appropriate monitoring during
treatment. We note that manufacturers can conduct clinical
investigations of ephedrine alkaloids under an IND application and can
seek approval of ephedrine alkaloid-containing products as new drugs
for the treatment of obesity or other diseases under a NDA if
sufficient evidence is provided to support such use. It is also
possible that products containing ephedrine alkaloids might not present
an unreasonable risk, even without physician supervision, if they were
marketed as dietary supplements for a use that results in a meaningful
health benefit and that requires only temporary, episodic use to
achieve the benefit. However, based on the information we have now, we
believe that it is unlikely that any such nondisease use could be
identified.
(Comment 30) Another comment, citing a study by Haller et al.,
contended that the apparent causal role of ephedrine alkaloids in
severe adverse effects could be related to the additive stimulant
effects of caffeine (Ref. 34). One comment submitted by a manufacturer
attributed the good safety record of its product to, among other
reasons, the absence of caffeine and other stimulants.
(Response) While caffeine would be expected to have additive
effects with ephedrine alkaloids, acute administration of ephedrine
alone increases blood pressure and heart rate (Refs. 37 and 47). The
available evidence shows that chronic use of caffeine has no effect on
blood pressure that persists beyond 2 weeks (Refs. 45 and 46), in
contrast to ephedrine, which does have a persistent effect (Boozer)
(Ref. 49).
(Comment 31) Many comments contended that we failed to consider the
differences among ephedrine alkaloids from the raw botanical; extracts
from the raw botanical that contain unaltered proportions of alkaloids
and other substances; concentrated and/or otherwise manipulated ephedra
extracts such that naturally occurring proportions and/or quantities of
ephedrine alkaloids are changed; and synthetic or pure isolated
ephedrine (extracted as a single entity from the plant). Because these
products have chemical differences and differences in
[[Page 6807]]
potency, toxicity, pharmacokinetics, and pharmacological and
physiological effects, the comments maintained they should be
considered separately in scientific, medical, and regulatory contexts.
Other comments, citing a study by White et al., stated that other
natural constituents, including other alkaloids and ephedradines in the
raw botanical, modify or attenuate the physiological and
pharmacological effects of the ephedrine contained in dietary
supplements (Ref. 43). Numerous comments maintained that raw Ephedra
and/or Ephedra extracts are safer than ephedrine that is synthetic or
that has been isolated and that serious adverse events associated with
the appropriate use of ephedra have been rare. Several comments
asserted that the ephedradines have hypotensive effects and are found
in ephedra roots, rather than the aerial portions of the plant. One
comment maintained that ephedradines are thought to occur in small
amounts in Ephedra stems. One comment stated that ephedra extract is
safer than pharmaceutical ephedrine based on the fact that the
LD50 is higher for the botanical extract (5.4g/kg) when
compared to the LD50 for pharmaceutical ephedrine (64.9 mg/
kg) (``LD50'' refers to the amount of a material that causes
death in 50 percent of test animals).
Several comments stated that pharmaceutical ephedrine is more
potent than ephedrine from botanical sources because ephedrine
comprises only 30 to 90 percent of the total alkaloids of the raw
botanical, with the remaining portion containing potentially less
potent stimulants such as pseudoephedrine. Several comments claimed
that the various ephedrine alkaloids from botanical sources have a
slower rate of absorption due to the plant matrix as compared to the
rate of absorption for pharmaceutical ephedrine (Ref. 43). These
comments stated that delayed effects diminish side effects and provide
for the cardiovascular adaptation of effects, thereby diminishing
cardiovascular response. One comment stated that except for absorption
rate, ephedrine alkaloids from the plant have the same pharmacokinetics
as pharmaceutical ephedrine (Ref. 43). Other comments note that
botanical ephedrine from formulations containing whole Ephedra is
absorbed more slowly than dietary supplements formulated with
standardized extracts (Ref. 44). A few comments suggested that ephedra
extract has higher neurocytotoxic (toxic effect on nerve cells)
potential than synthetic ephedrine hydrochloride due to combinations of
different ephedrine alkaloids or other unknown compounds found in
ephedra extract that are not found in ephedrine hydrochloride (Ref.
89).
Other comments maintained that there is no difference between blood
levels of ephedrine from botanical sources and ephedrine contained in
OTC drugs. Comments from a State Board of Pharmacy stated that
ephedrine from botanical sources is neither safer than, nor different
from, pharmaceutical ephedrine. One comment objected to our including
clinical studies using pharmaceutical ephedrine in our evaluation. A
number of comments suggested that naturally occurring ephedrine is more
potent than its synthetic counterpart. A few comments stated that the
presence of varying amounts, proportions and chemical configurations of
ephedrine alkaloids in crude Ephedra and prepared Ephedra extracts, as
well as the presence of unknown compounds, leads to uncertainty in
dose, purity, and composition and a greater risk for adverse effects.
Comments noted that this variability is not an issue for synthetic or
pure isolated ephedrine alkaloids.
(Response) The data are wholly inadequate to demonstrate that any
differences among forms of naturally occurring ephedrine alkaloids and
synthetic ephedrine have a meaningful impact on risks to health. The
overall database of clinical trials, including trials using both
natural and synthetic ephedrine, does not lead to the conclusion that
one form of ephedrine is safer than the other form.
We are not persuaded by any of the available evidence that
ephedrine from botanical sources is materially different from ephedrine
from pharmaceuticals with respect to chemistry, potency, or
physiological and pharmacological effects. Chemically, any isomer with
the same conformation from one source, including botanical sources, is
identical to the same isomer from another source. For example, (-)-
ephedrine from Ephedra (Ephedra sinica Stapf) is chemically
indistinguishable from synthetic (-)-ephedrine manufactured by a
pharmaceutical company.
Regarding the ephedradines, we are not aware of any evidence in the
scientific literature, nor were any data provided in the comments, that
indicate that these compounds are present in Ephedra, in other
botanical sources of ephedrine alkaloids, or in extracts from these
botanicals. The ephedradines are known constituents of the roots of the
species Ephedra sinica Stapf (Ref. 90). In traditional Asian medicine,
the roots and rhizome of the plant are referred to as ``ma huang gen,''
while the aerial parts of the plant are referred to as ``ma huang''
(Ref. 3). The ephedradines are not ephedrine alkaloids. Nor are they
present in the aerial parts of the plant that are used in dietary
supplements. The scientific evidence, thus, does not support the
opinion that the other ephedradrines in the raw botanical act to modify
or attenuate the physiological and pharmacological effects of the
ephedrine alkaloids contained in these products.
We do not agree, therefore, that current evidence establishes that
ephedrine alkaloids from botanical sources, including botanical
extracts, are different from, or are any safer than, pharmaceutical
ephedrine alkaloids. With regard to the comment asserting that ephedra
extract is safer than pharmaceutical ephedrine because the
LD50 is higher for the botanical extract than the
LD50 for pharmaceutical ephedrine, we note that scientific
views on this point differ. Another scientific reference suggests that
a mixture of ephedrine alkaloids from a botanical extract may be more
toxic, based on LD50 calculations, than an equal amount of
pharmaceutical ephedrine (Ref. 91). While there is not enough
scientific evidence to draw a conclusion, we acknowledge the
possibility that other components in the concentrated extracts (e.g.,
tannins derived from the botanical) may affect the toxicity of
botanical preparations of ephedrine alkaloids (Refs. 89 and 92).
2. Other Safety Data
(Comment 32) Many comments cited multiple data and information
sources as support for the safety of dietary supplements containing
ephedrine alkaloids. These cited sources have been submitted to the
docket and include the CANTOX review, RAND Report, the Ad Hoc Committee
on the Safety of Ma Huang report and the Ad Hoc Committee on the Safety
of Dietary Supplements, Ephedra Education Council Expert Panel Report,
and a 6-month clinical trial by Boozer et al. (2002) (Refs. 21, 49, 93,
94, and 95). Some comments also claimed that the toxicological database
supports clinical evidence of safety; that no serious adverse events
have been reported in controlled clinical trials using products
containing ephedrine alkaloids for weight loss, and that few or no
serious adverse events have been reported to manufacturers of dietary
supplements containing ephedrine alkaloids.
One trade association commented that a valid and quantitative
scientific process is needed to identify intakes
[[Page 6808]]
and conditions of use that do not cause significant or unreasonable
risk, and urged us to adopt scientific conclusions based on the CANTOX
risk assessment, which was based on methods developed by the Institute
of Medicine (IOM) (Ref. 28). A number of comments argued that the
results of the CANTOX review established that dietary supplements
containing ephedrine alkaloids are safe when used in accordance with
the industry standard.
One comment stated that the methods employed by CANTOX were not
appropriate for use in evaluating the safety of dietary supplements
containing ephedrine alkaloids. Several comments stated that there are
no data that establish that ephedrine alkaloids are an ordinary
component of food, that there is a need for ephedrine alkaloids in the
diet, or that some deficiency state exists when ephedrine alkaloids are
not a normal component of the diet.
(Response) We do not agree with the methodology or conclusions of
the risk assessment performed by CANTOX. The CANTOX review, sponsored
by an industry trade group, was a quantitative risk assessment that
used IOM methods to determine a safe upper level (called the No
Observed Adverse Effect Level (NOAEL)) for botanical ephedrine
alkaloids as used in dietary supplements. We believe that this review
cannot be used to establish a NOAEL for ephedrine alkaloids used in
dietary supplements because it was flawed. Its flaws include use of an
inappropriate risk assessment model and deviation from the criteria and
procedures established by IOM, including relying on abstracts and
unpublished articles, using an unsuitable definition of ``Tolerable
Upper Intake Level'' (UL), and using an overly narrow definition of
``adverse effect.''
The IOM model referenced by CANTOX is the Food and Nutrition
Board's report entitled ``Dietary Reference Intakes: A Risk Assessment
Model For Establishing Upper Intake Levels For Nutrients.'' The
introduction to this report states that dietary reference intakes are
being established for ``nutrients and food components'' which include
nutrients, dietary antioxidants, micronutrients including electrolytes
and fluid, macronutrients, ``and other food components not
traditionally classified as ``nutrients,'' but purported to play a
beneficial role in human diets'' (Ref. 28 at pp. 1 and 2). The IOM
report defined dietary reference intakes, in part, as ``reference
values that are quantitative estimates of nutrient intakes to be used
for planning and assessing diets for healthy people. They include both
recommended intakes and [tolerable upper intake levels] as reference
values'' (Ref. 28 at p. 2). The report defined ``Tolerable Upper Intake
Level'' (UL) as ``the highest level of daily nutrient intake that is
likely to pose no risk of adverse health effects to almost all
individuals in the general population. As intake increases above the
UL, the risk of adverse effects increases'' (Ref. 28 at p. 3). The
rationale for establishing such a risk assessment model is that
nutrients are an essential part of the diet and deficiency states
result when they are absent from the diet or are available in too low
of a concentration.
CANTOX claimed that the use of this model was appropriate for
ephedrine alkaloids in dietary supplements because nutrients, like all
chemical agents, can produce adverse health effects if intakes are
excessive. However, ephedrine alkaloids are not nutrients. The CANTOX
report did not include any data establishing that there is a need for
ephedrine alkaloids in the diet, or that some deficiency state exists
when ephedrine alkaloids are not present in the diet. Therefore, we
conclude that the use of the IOM risk assessment method based on the
model of a nutrient is inappropriate for the evaluation of the safety
of dietary supplements containing ephedrine alkaloids.
Even if the IOM dietary reference intakes model were an appropriate
risk assessment model for dietary supplements containing ephedrine
alkaloids, we note that CANTOX deviated from the IOM's criteria and
procedures in several important ways. For instance, the IOM report used
studies published in peer-reviewed journals as the principal sources of
data for its evaluations. In contrast, while CANTOX did use some
publications, it also relied on abstracts and unpublished studies. For
example, CANTOX cited the study by Boozer, et al. as the pivotal study
demonstrating the safety of dietary supplements containing ephedrine
alkaloids and the establishment of the NOAEL. However, the Boozer (Ref.
96) study was only available in abstract form at the time of the CANTOX
review. Abstracts are not subject to the same rigorous peer review that
full manuscripts go through. Further, abstracts do not contain
sufficient information to enable a reader fully to evaluate a study's
methodology or independently to interpret or verify a study's results.
As a result, abstracts should not be given the same weight as the full
reports of studies themselves. In the case of the Boozer study, the
abstract did not provide details on the exclusion or inclusion criteria
for the study, so a reader could not determine how the subjects were
selected or how they were monitored during the study. The CANTOX
authors also did not acknowledge the significance of the blood pressure
findings in the Boozer et al. As we have discussed extensively in
section V.B.1 of this document, this study by Boozer et al. (Ref. 49)
clearly demonstrates a higher blood pressure in ephedra plus caffeine
treated subjects (compared to placebo), which translates into serious
long-term risks in the general population and serious short-term risks
in susceptible populations. Furthermore, as stated by outside
scientific experts who reviewed this study, the Boozer et al. (2002)
study cannot establish the safety of dietary supplements containing
botanical ephedrine alkaloids and caffeine because the study used a
highly selected population, had relatively few subjects and was carried
out for too short a period of time. Rather, the Boozer study raises
questions about the safety of these products.
Indeed, of the 20 studies that CANTOX considered in identifying the
NOAEL, four were abstracts, and two were unpublished reports. Thus,
unlike the IOM report's reliance on peer-reviewed journal articles, a
significant proportion of the CANTOX ``studies'' were not subject to
peer review.
We also note a number of other deviations from the IOM's
application of its risk assessment model (Ref. 28). Compared to the
definition in the IOM report, CANTOX expanded the definition of the UL
and narrowed the population to which it applies. As noted earlier, the
IOM report defined the UL, in part, as ``the highest level of daily
nutrient intake that is likely to pose no risk of adverse health
effects to almost all individuals in the general population.'' The IOM
report stated that the term ``tolerable'' was chosen ``because it
connotes a level of intake that can, with high probability, be
tolerated biologically by individuals; it does not imply acceptability
of that level in any other sense.'' The IOM report also noted that
``the UL is not intended to be a recommended level of intake'' (Ref. 28
at pp. 3, 4, and 5). The IOM report also stated that ``the critical
endpoint used to establish a UL is the adverse biological effect
exhibiting the lowest NOAEL (for example, the most sensitive indicator
of a nutrient or food toxicity). The derivation of a UL based on the
most sensitive endpoint will ensure protection against all other
adverse effects'' (Ref. 28 at p. 18). The IOM report also explained
that, ``When possible, the UL is based on a NOAEL, which is the highest
intake (or
[[Page 6809]]
experimental oral dose) of a nutrient at which no adverse effects have
been observed in the individuals studied. This is identified for a
specific circumstance in the hazard identification and dose-response
assessment steps of the risk assessment'' (Ref. 28 at p. 10).
Although CANTOX defined the UL as ``the maximum level of chronic
daily intake of a substance judged unlikely to pose a risk to the most
sensitive members of the health population,'' their UL determination
was based upon the ``specified conditions of use,'' which includes
label warnings that these products not be used by many in the general
population (including those under 18 years, pregnant or lactating
women, and persons with certain health conditions, including those most
sensitive to the effects of these products, e.g., persons with
hypertension and coronary artery disease). In contrast, the IOM concept
of the UL is the highest level of intake likely to pose no risk of
adverse health effects to almost all individuals in the general
population. Thus, the CANTOX UL is less protective than the IOM UL
because it removes from its risk assessment the members of the
population who would be most at risk for adverse effects of dietary
supplements containing ephedrine alkaloids.) (Ref. 93 at p. 5).
It also appears that CANTOX deviated from the IOM model in its
assessment of what constituted an ``adverse effect.'' Although the
CANTOX report failed to define the endpoints (potential adverse
effects) that were considered in the determination of a NOAEL, the
report stated that ``the selection of 90 mg/day is an appropriate value
for a NOAEL for ephedra in light of the evidence of no significant
increases in frequency of adverse effects or changes in heart rate or
blood pressure at or below this level leading to cardiac arrhythmias.''
Thus, it appears that CANTOX did not consider changes in heart rate or
blood pressure to be ``adverse effects,'' although these biological
effects can lead to serious adverse health consequences, such as
arrhythmias and strokes. In addition, in discussing the Boozer et al.
study, the CANTOX report described the statistically significant 4 mm
Hg elevation in systolic blood pressure in the ephedra plus caffeine
treated group as compared to the placebo group, as well as other self-
reported symptoms (dry mouth, heartburn and insomnia) in the treated
group, as ``minimal side effects.'' This choice of terminology suggests
that CANTOX did not consider the well-described pharmacological effects
of ephedrine alkaloids to have potentially serious adverse health
effects. This difference would affect the NOAEL, which, in turn, would
lead to different UL determinations. We further address the
definitional issue of adverse events versus side effects later in
section V.B.6. of this document.
We also note that CANTOX's stated study objective, ``to provide and
justify a safe upper intake level for ephedrine alkaloids from ephedra
used as a dietary supplement,'' appears to assume that such a safe dose
exists. This assumption indicates a bias towards finding a safe dose,
rather than an unbiased assessment of whether any safe dose exists.
Finally, we discuss the inadequacies of the publications used by
CANTOX to assess the safety of ephedrine alkaloids in section V.B.2 of
this document. Whatever methods are employed, these deficiencies in the
data used in CANTOX's analysis significantly undermine any conclusions
reached in the CANTOX report.
(Comment 33) Several comments objected that we did not consider
animal studies using ephedrine alkaloids to evaluate the safety of
ephedrine alkaloids as dietary ingredients, as several comments noted
had been done in the CANTOX review. One comment stated that the results
of the National Toxicology Program's long-term rodent studies on
ephedrine showed that a lethal dose of ephedrine alkaloids for most
animal species, translated into human consumption, was between 200 and
400 25 mg tablets. A related comment referred to toxicity
(LD50) studies comparing pharmaceutical ephedrine with ma
huang in mice, emphasizing lesser toxicity of ma huang: The
LD50 for ephedrine alkaloids from ma huang was 5300 mg/kg
body weight versus 689 mg/kg for pharmaceutical ephedrine. A related
point from this comment was that wild and domestic animals consume
Ephedra shrubs and there are no reports of adverse effects in these
animals. One comment included data from rat, mouse, and dog toxicity
studies on a specific ephedrine alkaloid-containing dietary supplement.
The results and their interpretation by consultants were offered as
demonstrating a very low toxicity for the supplement. One comment
stated that no animal study suggests that the ephedrine alkaloids would
be harmful at human doses of 25 mg per serving. One comment stated that
animal and laboratory testing may be informative on some issues but, in
and of itself, cannot answer the human causation question.
(Response) We recognize the value of animal studies in identifying
or predicting the toxicological properties of substances for human
exposure. In fact, animal studies do identify the sympathomimetic
effects of ephedrine that underlie our concern. These would not be
expected to lead to harm in healthy laboratory animals because these
animals do not have coronary artery disease or other susceptibility to
arrhythmias or congestive heart failure. An effect of elevated blood
pressure, if large and sustained, might perhaps show effects in very
large, long-term animal studies, but there is no reason to think that a
modest effect, one that would increase hypertensive risk in humans but
still lead to a low overall risk in any individual, would be detectable
in animals. The animal data are, therefore, not at all reassuring. The
discussion of the consumption of wild Ephedra species by wild and
domestic animals contributes no relevant safety information, since
these animals also lack pertinent human risk factors (coronary artery
disease, heart failure, elevated blood pressure). Also, were these
animals to have an adverse effect, there would be no way to identify
it. However, we believe, as stated previously, that there is sufficient
scientific evidence from multiple sources, including clinical trials
and the published literature pertaining to use of ephedrine alkaloids
in humans, to conclude that dietary supplements containing ephedrine
alkaloids pose serious risks of illness or injury.
3. Comparison with Drug Products Containing Ephedrine Alkaloids
(Comment 34) One comment asserted that our proposal to treat
dietary supplements more restrictively than OTC drugs containing
ephedrine and pseudoephedrine is in violation of the Administrative
Procedure Act's prohibition on rulemaking that is arbitrary and
capricious. According to the comment, OTC ephedrine and pseudoephedrine
products contain higher doses of ephedrine alkaloids and therefore are
potentially more dangerous than dietary supplements that contain these
substances at lower levels.
(Response) Our decision in this rulemaking to treat dietary
supplements that contain ephedrine alkaloids differently from OTC drugs
that contain ephedrine or pseudoephedrine is not arbitrary or
capricious. Our decision is based on differences in the intended uses
of these products, as well as differences in the scientific evidence
available to support the risk-benefit ratio for the products. The risk-
benefit ratio is dependent on several factors, including the product's
intended use, the product's benefits, if any, and the
[[Page 6810]]
availability of adequate measures to control risk.
As discussed previously, dietary supplements containing ephedrine
alkaloids present an unreasonable risk of illness or injury because
their risks outweigh their benefits. Like dietary supplements
containing ephedrine alkaloids, OTC drug products containing ephedrine
or pseudoephedrine have risks related to these ingredients. However,
unlike dietary supplements, such OTC drug products have demonstrated
benefits in the treatment and mitigation of disease. Through the OTC
drug review process, we have determined that drug products containing
ephedrine are GRASE for OTC use as a bronchodilator for the temporary
relief or symptomatic control of bronchial asthma (see Sec.Sec. 341.16
and 341.76), and that drug products containing pseudoephedrine are
GRASE for OTC use as a nasal decongestant for the temporary relief of
nasal congestion due to the common cold or hay fever (allergic
rhinitis) (See Sec.Sec. 341.20 and 341.80). Based on controlled
clinical investigations (See Sec. 330.10(a)(4)(ii)), we have determined
that the benefits associated with the use of OTC drug products
containing ephedrine and pseudoephedrine for these disease indications
outweigh the risks and justify the use of these products despite their
risks. However, such uses for disease mitigation and treatment are
beyond the scope of permissible dietary supplement uses.
Moreover, we do not agree that dietary supplements containing
ephedrine alkaloids are safer than OTC drugs containing ephedrine or
pseudoephedrine based on the relative doses of ephedrine alkaloids in
these products. We consider an OTC drug product's safety in the context
of its conditions of use (See Sec. 330.10(a)(4)(i)). OTC drugs
containing ephedrine and pseudoephedrine are marketed to persons with
specific disease conditions or symptoms for temporary, episodic relief.
In fact, OTC ephedrine bronchodilator drug products are required to
bear a warning limiting the use of these products to persons who have
been diagnosed with asthma by a doctor (See Sec. 341.76(c)(1)).
Additionally, although drug products containing ephedrine and
pseudoephedrine are permitted to be marketed OTC at specific doses,
these doses have been determined based on the specific indications of
these drugs. As previously discussed, the indications and benefits
applicable to OTC drugs containing ephedrine and pseudoephedrine do not
apply to dietary supplements. Thus, the safety of dietary supplements
containing ephedrine alkaloids cannot be established merely by showing
that the level of ephedrine alkaloids in these products falls within or
under the dose ranges permitted for OTC drug products. Furthermore,
these dietary supplements contain several ephedrine alkaloids, making
it difficult to draw any conclusions about benefits from studies using
OTC drug products that contain a single ephedrine alkaloid.
(Comment 35) Several comments pointed out that we have concluded
that the ephedrine levels permitted in OTC drugs are generally
recognized as safe. Other comments maintained that the long-term
marketing and favorable safety record of OTC drugs containing ephedrine
alkaloids is evidence of the safety of dietary supplements containing
ephedrine alkaloids. Several comments asserted that there is a lack of
serious AERs for both traditional Asian herbal products and OTC
ephedrine drugs with dosages based on FDA's monograph (less than or
equal to 25 mg per serving and less than or equal to 150 mg in a 24-
hour period) and that these dosages are, thus, safe.
One comment maintained that the nonserious events identified by
RAND are consistent with the side effects of caffeine and OTC ephedrine
listed in the OTC drug review and do not pose an unreasonable risk.
Other comments referred to statements made during the 1996 FDA Food
Advisory Committee that there are no serious adverse effects reported
with drugs containing ephedrine alkaloids within the allowable dosage
range and to a February 28, 2003 FDA press release relating to ephedra
that stated there are fewer AERs linked to OTC ephedrine drug products
than to dietary supplements containing ephedrine alkaloids.
(Response) We do not agree that the safety of dietary supplements
containing ephedrine alkaloids can be established by reference to the
safety of OTC drug products containing ephedrine or pseudoephedrine,
two ephedrine alkaloids currently included in OTC drug monographs.
As discussed previously, all sympathomimetics may pose risks for
adverse events even after a single dose. GRASE status does not mean
that an OTC drug product may not cause adverse events. In fact, there
have been adverse events reported to FDA concerning ephedrine- and
pseudoephedrine-containing OTC drugs. There are also numerous adverse
event reports for dietary supplements containing ephedrine alkaloids.
The incidence and type of adverse event reports related to dietary
supplements containing ephedrine alkaloids are discussed in section
V.B.6 of this document, which also contains our discussion on the
significance of these AERs in our determination of unreasonable risk.
As part of our OTC drug review, we have determined that ephedrine
and pseudoephedrine are GRASE OTC drug ingredients for certain
indications. Ephedrine is GRASE for the temporary relief or symptomatic
control of bronchial asthma (See Sec.Sec. 341.16 and 341.76).
Pseudoephedrine is GRASE for the temporary relief of nasal congestion
due to the common cold or hay fever (allergic rhinitis) (See Sec.Sec.
341.20 and 341.80). OTC ephedrine and pseudoephedrine drug products
have been studied in controlled trials that establish their safe and
effective dose for specific disease indications (labeled uses) (41 FR
38312 at 38371 and 38402 to 38403, September 9, 1976) (Refs. 97 and
98). These OTC drug products provide health benefits when used by the
population experiencing the particular disease. We note that these OTC
drug products bear warnings that certain populations should not use
them, and they are not risk free. However, we have determined that the
demonstrated benefits for the labeled OTC drug uses outweigh their
risks (See Sec. 330.10(a)(4)(iii)). The labeling of OTC ephedrine and
pseudoephedrine drug products warns consumers not to use the products
if they have heart disease, high blood pressure, thyroid disease,
diabetes, or difficulty in urination due to an enlargement of the
prostate gland unless directed by a doctor (Sec.Sec. 341.76(c)(2) and
341.80(c)(1)(C)). In addition, OTC ephedrine bronchodilator drug
products are labeled with a warning not to use the product unless a
diagnosis of asthma has been made by a doctor (Sec. 341.76(c)(1)).
Moreover, the labeling directs users not to continue to use ephedrine
drug products but to seek medical assistance immediately if symptoms
are not relieved within 1 hour or become worse (Sec. 341.76(c)(5)). As
discussed in the response to comment 34 of this document, the benefits
of ephedrine and pseudoephedrine drug products for disease claims are
different from the benefits of dietary supplement products for
nondisease claims, so it would be inappropriate to conclude based on
OTC drug product information that these dietary supplements do not
present an unreasonable risk. No data demonstrate that dietary
supplements containing ephedrine alkaloids provide a meaningful health
benefit to a particular
[[Page 6811]]
population for any specific use and for short periods of time, as is
the case for OTC ephedrine or pseudoephedrine drug products. Therefore,
we have determined that the risks presented by dietary supplements
containing ephedrine alkaloids (including heart attack, stroke, and
death) outweigh their benefits, and that these products are adulterated
regardless of what warnings are included in their labeling. We note
that dietary supplements containing ephedrine alkaloids may also
present other, less serious risks listed in the required warnings for
OTC drugs containing ephedrine and pseudoephedrine; however, because we
are removing these dietary supplement products from the market based on
their cardiovascular risks, we are not addressing these other risks in
this rule.
With regard to the comments that discussed safety data for OTC
ephedrine bronchodilator drugs specifically, we note that the studies
used to evaluate ephedrine for the treatment of asthma and those using
ephedrine alkaloids for weight loss and other nondisease uses enrolled
different populations and used different study designs, endpoints, and
monitoring protocols. Therefore, comparisons across patient populations
or indications (e.g., asthma treatment versus weight loss) for a risk
benefit analysis is not justified. FDA's 1986 final rule finding
ephedrine GRASE as a bronchodilator was based on the 1976
recommendation of the Advisory Review Panel on OTC Cold, Cough,
Allergy, Bronchodilator, and Antiasthmatic Drug Products (the Panel)
(See 51 FR 35326, October 2, 1986 and 41 FR 38312 at 38370 to 38372,
September 9, 1976). The Panel relied on data from studies conducted in
1973 and 1975 (Refs. 97 and 98). These studies were designed to examine
the efficacy of terbutaline as a bronchodilator. The patient population
enrolled in these studies were not only clinically stable (i.e. normal
electrocardiogram, blood pressure, and pulse) but also had no apparent
history of adverse events related to treatment with other stimulant
bronchodilators used at the time. These studies support the use of
ephedrine for patients with asthma who are otherwise clinically stable
(i.e. not found by a physician to have high blood pressure or other
cardiovascular risk); however, they do not support the safety or
efficacy of dietary supplements containing ephedrine alkaloids for
weight loss or other nondisease uses.
(Comment 36) Several comments asserted that it is misleading to
compare the safety and efficacy of ephedra to OTC drugs because all
drugs are toxic to some individuals and all products must be evaluated
on the basis of their benefits relative to their risks. These comments
expressed the view that dietary supplements containing ephedrine
alkaloids have only limited benefit for weight loss over placebo and
that this modest weight loss has never been shown to reduce the
increased morbidity that is associated with obesity.
(Response) We agree that dietary supplements containing ephedrine
alkaloids and OTC drug products must be evaluated based on a comparison
of their risks and benefits. It should be noted, however, that the
evidentiary standards for evaluating these two categories of products
are different. We have done a risk-benefit analysis for dietary
supplements containing ephedrine alkaloids for weight loss, as well as
other uses, and have discussed our analysis and conclusions regarding
weight loss in section V.C.1 of this document.
(Comment 37) Numerous comments asserted that herbal medicines,
including ephedra, have a favorable safety record when compared to
approved pharmaceuticals. Several comments cited the numbers of serious
adverse events associated with approved pharmaceuticals, including
deaths, among the U.S. population that are not due to medication
errors. For example, various authorities estimate that more than
100,000 deaths per annum are associated with approved pharmaceuticals
(Refs. 99 and 100). One comment stated that the rate of severe adverse
reactions to prescription drugs, without necessarily including misuse,
ranks as the fourth to sixth leading cause of death in the United
States (Ref. 100). The comment expressed the view that ephedrine
alkaloids do not carry a significant or unreasonable risk of harm when
compared to the high incidence of serious adverse effects with
prescription drugs.
(Response) While we agree that serious adverse events can occur
with the use of prescription drugs, that fact does not change our
determination that dietary supplements containing ephedrine alkaloids
present an unreasonable risk. Prescription medications, although
considered safe and effective for their labeled indications, are not
free from all risks. However, the benefit of using prescription
medications outweighs such risks for particular patients with
particular disease conditions, in part because the risk is managed
through the physician supervision required for the use of prescription
medications. Although dietary supplements need not be free of risks to
be lawfully marketed, the risks of using dietary supplements containing
ephedrine alkaloids are not outweighed by any benefit. Moreover, it
would not be surprising to see more AERs for prescription drugs than
for dietary supplements. Healthcare professionals, who are aware of the
drugs prescribed for their patients, are the primary source of drug
AERs reported to us directly or through manufacturers. They may not be
similarly aware of their patients' use of dietary supplements. In
addition, there are no mandatory reporting requirements for dietary
supplement manufacturers, unlike for prescription drug manufacturers.
Finally, the comments and literature cited pertain to adverse events
for all prescription drugs combined. This information has no meaningful
bearing on whether dietary supplements containing ephedrine alkaloids
present risks.
(Comment 38) One comment contended that dietary supplements
containing ephedrine alkaloids should be banned because we have already
banned OTC drugs containing ephedrine in combination with caffeine.
Numerous other comments stated that our November 18, 1983 (48 FR
52513), prohibition of ephedrine alkaloids combined with caffeine and
other stimulants (48 FR 52513) was due to such products' potential for
abuse and misuse as illicit street drug alternatives and not because of
safety issues. One comment stated that our proposal (60 FR 38643, July
27, 1995) (July 1995 proposal) to amend the final monograph for OTC
bronchodilator drug products to remove the ingredients ephedrine,
ephedrine hydrochloride, ephedrine sulfate, and racephedrine
hydrochloride and to classify these ingredients as not generally
recognized as safe and effective for OTC use was proposed to restrict
the OTC availability of ephedrine because of its illicit use as the
primary precursor in the synthesis of the controlled substances
methamphetamine and methcathinone. The comment stated that the July
1995 proposal does not discuss the safety of the use of ephedrine and
thus does not support our actions.
(Response) We do not agree that our July 1995 proposal did not
discuss the safety of OTC bronchodilator drug products containing
ephedrine alkaloids (60 FR 38643 at 38644). In any event, comments
about the basis and scope of our 1983 prohibition on ephedrine and
caffeine combinations in OTC drug products and the 1995 ephedrine drug
product proposal are not relevant to this rulemaking because we are not
relying on those actions as a basis for the
[[Page 6812]]
removal of dietary supplements containing ephedrine alkaloids.
4. Abuse and Misuse
(Comment 39) Many comments asserted that we must consider
directions for use, warnings, and other labeling when making an
assessment of significant or unreasonable risk. The comments stated
that we cannot consider misuse or abuse of properly labeled dietary
supplements. One comment urged that any evaluation of significant or
unreasonable risk be based on the standards specified in the American
Herbal Products Association's (AHPA) Ephedra Trade Recommendation,
which recommends that dietary supplements containing ephedrine
alkaloids be formulated to contain no more than 25 mg of ephedrine
alkaloids per serving, that such products bear a warning statement and
that directions for use limit consumption to 100 mg of ephedrine
alkaloids per day (Ref. 101).
(Response) We agree that directions for use, warnings, and other
labeling must be considered when making an assessment of significant or
unreasonable risk. Section 402(f)(1)(A) of the act provides that
whether a dietary ingredient or dietary supplement presents a
significant or unreasonable risk must be evaluated ``under conditions
of use recommended or suggested in labeling,'' except that ordinary
conditions of use may be considered if the labeling is silent on
conditions of use. Thus, for purposes of the ``significant or
unreasonable risk'' provision, unless no conditions of use are
recommended or suggested in labeling, we must consider a dietary
supplement's labeled use rather than its actual use. We do not agree,
however, that our evaluation of significant or unreasonable risk should
be based on the standards specified in AHPA's Ephedra Trade
Recommendation (Ref. 101). These standards are voluntary
recommendations by a trade association and are not universally
followed. We must consider all dietary supplements containing ephedrine
alkaloids, not just those formulated and labeled in accordance with the
Ephedra Trade Recommendation. In this instance, we conclude that all
dietary supplements containing ephedrine alkaloids present an
unreasonable risk, regardless of whether they are formulated and
labeled in accordance with the Ephedra Trade Recommendation, based on
our evaluation of the totality of the evidence and a weighing of the
risks and benefits of the products. As discussed in section VI.A of
this document, the presence of a warning label or of directions
recommending a limit on daily consumption of ephedrine alkaloids does
not sufficiently reduce the risks of dietary supplements containing
ephedrine alkaloids to allow them to continue to be marketed as
currently labeled or under ordinary conditions of use, and the risks of
these products outweigh their benefits regardless of labeling.
(Comment 40) Several comments compared the effects of ephedra to
other sympathomimetics such as cocaine or amphetamine. Several other
comments stated that while ephedrine, PPA, and amphetamine are similar
in chemical structure, they differ in physiological effect, and that
amphetamines have much stronger reinforcing effects and a much higher
liability for abuse than ephedrine. One comment stated that the
subjective effects of ephedrine more closely resemble caffeine. Another
comment stated that amphetamines do not have direct agonist properties,
but promote release of neurotransmitters and inhibit their deactivation
and reuptake. One comment from a manufacturer of a dietary supplement
containing ephedrine alkaloids stated that its product label warns
consumers not to take the product longer than 12 weeks because it can
be habit forming and to take it longer runs the danger of ``getting
hooked.''
Several comments expressed the opinion that ephedrine alkaloid
dependence is similar to amphetamine dependence, as are the
psychological effects of abuse such as psychosis, paranoia, and the
potential to cause mania in susceptible individuals. Comments from
several individuals and the founder of a consumer advocacy Web site
included anecdotal reports of individuals who reported dependence or
apparent addiction associated with use of ephedrine and dietary
supplements containing ephedrine alkaloids. Several other comments
cited the German Commission E monograph's instructions to limit the use
of ephedra preparations to short-term because of the danger of
addiction. (The Commission E was a division of the German Federal
Health Agency established in 1978 to evaluate the safety and efficacy
of herbal medicines sold in Germany. It produced official monographs
for botanicals and botanical formulations sold in German pharmacies.)
(Response) We agree that ephedrine alkaloids and amphetamines share
some pharmacological and physiological properties that may be
associated with abuse and dependence. Psychostimulant effects that have
been reported with sympathomimetic agents include drug tolerance,
dependence, or addiction, although these psychostimulant effects are
better recognized for cocaine and amphetamines (Refs. 102 and 103 of
English abstract), Ephedrine alkaloids exhibit physiological effects
common to the amphetamines, but differ in the relative intensity of
these effects. We agree that amphetamines and cocaine have been shown
to have much greater reinforcing effects and higher liability for abuse
than products containing ephedrine alkaloids, but also agree that the
development of dependence from the use of ephedrine alkaloids has been
noted with both pharmaceutical and botanical products (Refs. 104, 105,
and 106). The greater possibility of dependence and abuse of
amphetamine-containing and cocaine-containing drug products marketed in
the United States is recognized by the placement of these substances in
Schedule II of the Controlled Substances Act (CSA). Ephedrine-
containing drug products are not scheduled under the CSA; however,
ephedrine, its salts, optical isomers, and salts of optical isomers are
List I chemicals under the CSA (See 21 U.S.C. 802(34)) because they are
chemical precursors of methamphetamine (Schedule II) and are used in
its illicit manufacture. As List I chemicals, these substances are
subject to various Drug Enforcement Administration (DEA) requirements,
including recordkeeping, reporting, and sale behind the counter (See 21
CFR 1310.03 through 1310.07). While we are concerned about the
potential for abuse, we did not rely on evidence of abuse or dependence
to make our determination under section 402(f)(1)(A) of the act.
(Comment 41) Some comments advocated use of ephedra as an
alternative to more dangerous street drugs. They postulated that
banning dietary supplements containing ephedrine alkaloids would push
those products underground or drive consumers to seek out more
dangerous drugs for stimulant effects.
(Response) No data were submitted with these comments to support
their conclusions. We have no information regarding the extent of use
of ephedra, or dietary supplements containing ephedrine alkaloids, as
an alternative to more dangerous street drugs, nor do we have any
information about whether users of ephedrine alkaloids would be likely
to use other substances were ephedra to become unavailable. Regardless,
such information would not affect the determination we have made that
dietary supplements containing ephedrine alkaloids present an
unreasonable risk.
[[Page 6813]]
(Comment 42) Several comments stated that we cannot stop the abuse
of substances by regulation. Some comments cited tobacco and alcohol as
examples. Another comment stated that if we regulated products that
caused injury because of their potential for abuse, then common
household products, such as aerosol paint, would be banned.
(Response) Our conclusion that dietary supplements containing
ephedrine alkaloids present an unreasonable risk is based not on abuse
or misuse but rather on evidence supporting the presence of risks under
conditions of use recommended or suggested in the labeling, or if the
labeling is silent, under ordinary conditions of use. Abuse or misuse
of other products is not relevant to our determination that dietary
supplements containing ephedrine alkaloids present an unreasonable
risk.
(Comment 43) Several comments stated the opinion that we do not
appear to distinguish between dietary supplements containing ephedrine
alkaloids marketed for weight loss or energy from those products
marketed as alternatives to illicit street drugs or as ``legal highs.''
(Response) We do not agree with these comments. Beginning with the
June 1997 proposal on dietary supplements containing ephedrine
alkaloids, we have repeatedly warned industry and the public that we do
not consider products marketed as street drug alternatives to be
dietary supplements because they are intended for recreational purposes
to affect psychological states (e.g., to get high) and are not intended
to be used to augment the diet or to promote health (62 FR 30678 at
30699 and 306700). Since 1997, we have issued a series of warning
letters to firms for marketing ephedrine alkaloid-containing products
as street drug alternatives and warned consumers not to purchase or
consume such products. In March 2000, we issued a guidance document
stating that street drug alternatives are unapproved and misbranded
drugs that are subject to regulatory action, including seizure and
injunction (available at http://www.fda.gov/cder/guidance/3602fnl.pdf).
Our position was that street drug alternatives are drugs, not dietary
supplements, was upheld in United States v. Undetermined Quantities of
Articles of Drug (Street Drug Alternatives), 145 F. Supp. 2d 692 (D.
Md. 2001). That case involved a seizure of numerous street drug
alternatives marketed as dietary supplements, including four products
containing botanical ephedrine alkaloids. In January 2003, we witnessed
the voluntary destruction of $4 million worth of illegally marketed
street drug alternative products containing ephedrine alkaloids. We
continue to address the street drug alternatives with appropriate
regulatory actions. We have determined that the appropriate regulatory
action for dietary supplements containing ephedrine alkaloids--i.e.,
products marketed for weight loss, athletic performance, energy
enhancement, or other nonstreet drug alternative uses--is to issue a
final rule finding that these products present an unreasonable risk of
illness or injury.
5. Traditional Asian Medicine
(Comment 44) Many comments stated that the use of ephedrine
alkaloids in dietary supplements is safe based on its traditional use
in Asian medicine for thousands of years. Several comments asserted
that few or no adverse effects have been recorded with the use of
Ephedra in traditional Asian medicine. Numerous other comments,
including those by traditional Asian medicine practitioners, disagreed
with these comments about dietary supplements, highlighting the
differences in the products themselves and how they are used from what
is used in traditional medicine.
Several comments suggested that the raw Ephedra and Ephedra
extracts used in traditional Asian medicine formulae differ in potency,
toxicity, pharmacokinetics, and pharmacological and physiological
effects from many dietary supplements containing ephedrine alkaloids
and, therefore, that these formulations should be considered distinct
in scientific, medical, and regulatory contexts. Comments stated that
``Ephedra'' properly refers to dried aerial parts of medicinal plants,
or crude extracts thereof, not to isolated alkaloidal constituents.
Several comments further distinguished the various products containing
Ephedra as follows: Herb and extracts of raw herb of medicinal Ephedra
plants containing naturally occurring alkaloids and other compounds
without further manipulation, concentration, or adulteration; Ephedra
extracts that are concentrated, manipulated, or adulterated such that
naturally occurring proportions and/or quantities of ephedrine
alkaloids are altered; products containing ephedrine alkaloids combined
with other agents such as caffeine, caffeine-containing herbs,
salicylate-containing herbs, synephrine, and other substances; and
traditional Asian herbal medicinal formulae.
Several comments asserted that traditional Asian medicine Ephedra
formulae often deliver lower amounts of ephedrine alkaloids compared to
other types of ephedrine alkaloid-containing products and that
traditional formulae rarely contain more than 15 percent Ephedra in the
herb mixture. Comments also asserted that Ephedra in traditional
formulae is usually combined with other botanicals that typically
modify Ephedra's inherent stimulant effects. Another comment attributed
the relative safety of Ephedra to the mixture of ephedrine alkaloid
isomers not present in purified or synthetic alkaloids. One comment
suggested that the established therapeutic dose range of Ephedra sinica
in herbal medicine formulae is 60 to 90 mg total alkaloids per day
(adults), which falls within the dosage range established for OTC
ephedrine/pseudoephedrine-containing drugs (150 mg and 240 mg alkaloids
daily, respectively), and the recommendations of the Germany Commission
E (maximum daily Ephedra alkaloid dose of 300 mg daily). Other comments
asserted that infusions or teas of Ephedra are effective in relieving
respiratory symptoms but have fewer side effects and are safer than
formulations containing isolated or synthetic ephedrine alkaloids or
prescription drugs. Another comment stated that supplements in a liquid
tea form greatly reduce the risk of excess acute consumption by the
public.
In contrast, several other comments stated that the presence of
varying amounts, proportions, and chemical configurations of ephedrine
alkaloids in crude Ephedra and prepared Ephedra extracts, as well as
the presence of unknown compounds, leads to uncertainty as to dose,
purity, and composition and to a greater risk of adverse effects.
Comments noted that this variability is not an issue for synthetic or
pure isolated ephedrine alkaloids.
Numerous comments, including those by traditional Asian medicine
practitioners, also noted differences in how the products are used.
Several comments stated that most traditional Asian uses of Ephedra are
the same as the indications for OTC ephedrine and pseudoephedrine drugs
(e.g., short-term use to improve respiratory function) and that few if
any adverse effects have been recorded. Several comments stated that
use of Ephedra (ma huang) for weight control or for its stimulating
effects, for more than a short period of time, in combination with
caffeine and other botanical stimulants, and without the supervision of
a health care provider, is irresponsible and dangerous. A number of
traditional Asian medicine practitioners maintained that many
[[Page 6814]]
consumers experienced adverse effects because of this improper use,
over-dosage, or conflict with their illnesses.
Because of these differences, many practitioners of traditional
Asian medicine commented that they support our June 1997 proposal
except to the extent that it would restrict their use of Ephedra in
traditional Asian medicine. Several comments asserted that since most
serious adverse effects involve use of ephedrine alkaloids and not
whole herb or whole herb extracts of Ephedra, any rule must exempt
whole herb Ephedra or whole herb Ephedra extracts that contain no added
ephedrine alkaloids. Furthermore, ephedrine alkaloid-free species of
Ephedra should also be exempted.
Numerous comments asserted that because traditional Asian herbal
products are prescribed by appropriate practitioners (licensed,
certified, and registered acupuncturists, herbalists, and naturopathic
physicians) and because these products are not associated with serious
adverse effects, the products do not appear to constitute a public
health risk and their use should not be prohibited. Many traditional
Asian medicine practitioners stated that Ephedra is an essential
medicine and requested an exemption from the final rule for use of
Ephedra by traditional Asian medicine practitioners and acupuncturists.
A few comments asserted that Ephedra should not be used commercially,
but be restricted to professional use, to be dispensed by licensed
health care professionals trained in the appropriate use of traditional
Asian medicine.
(Response) This final rule does not affect the use of Ephedra
preparations in traditional Asian medicine, although we considered the
comments' views and information on the use of Ephedra in traditional
Asian medicine in the context of their possible relevance to the risks
of dietary supplements containing ephedrine alkaloids. This rule
applies only to products regulated as dietary supplements (See 62 FR
30678 at 30691). Traditional Asian medicine practitioners do not
typically use products marketed as dietary supplements.
With respect to the absence of adverse effects recorded with the
use of traditional Asian medicine, as we stated in the June 1997
proposal, we are not aware of any systematic collection of data related
to adverse effects occurring in individuals treated with Ephedra in
traditional Asian medicine. The absence of recorded adverse events with
the use of Ephedra, therefore, may be related to the lack of a
mechanism for reporting. Under these circumstances, there are no data
to evaluate. We note that the potential for adverse effects resulting
from the traditional Asian use of Ephedra is implied in several
reference texts that list precautions and contraindications for the use
of the botanical Ephedra in traditional Asian medicine preparations
(Refs. 3, 107, and 108). Moreover, even if we could say that the
absence of recorded adverse events with the use of Ephedra in
traditional Asian medicine was due to its safety for that use rather
than due to a lack of mechanism for reporting, the history of use of
Ephedra in traditional Asian medicine primarily for the treatment or
mitigation of respiratory illness cannot provide assurance about the
safety of dietary supplements containing ephedrine alkaloids for other
uses.
6. Adverse Events
AERs involving drugs include those submitted to us voluntarily by
consumers or healthcare professionals and those submitted by
manufacturers who are required to report them to us. However, there is
no required reporting of AERs to us for dietary supplements, including
those containing ephedrine alkaloids. Depending on other information we
may have about the event or about the suspect product, AERs can be hard
to interpret. AERs may raise concerns about a product, as well as
buttress a finding that a particular dietary supplement represents an
unreasonable risk based on other types of evidence. Some AERs can be
reasonably persuasive on their own. For example, individual cases of
adverse events where dechallenge (discontinued use) and rechallenge
(restarting use) have been linked to the abatement and recurrence of
the events, strongly support the association between exposure to the
product and occurrence of the adverse event. FDA, and others, have
reviewed and analyzed the AERs in depth to add to the body of evidence
and to ensure that all relevant evidence is considered (Refs. 109
through 115). Despite the limitations of such reports, a detailed
review of the AERs submitted to us for dietary supplements containing
ephedrine alkaloids and comparison of those AERs to scientific data
about the pharmacology of these substances establishes that the AERs
are consistent with the known and expected pharmacological effects of
these products considered (Refs. 109, 115, and 116).
In the preamble to the June 1997 proposal, we stated that there
were more than 800 reports of illnesses and injuries associated with
the use of dietary supplements containing ephedrine alkaloids. Since
that time, we have received more than 2,200 additional AERs submitted
directly to us plus approximately 16,000 reports from call records
submitted by Metabolife International, one of the largest distributors
of dietary supplements containing ephedrine alkaloids. These records
have been placed in the record for this rulemaking in redacted form.
A Congressional subcommittee minority report (Ref. 117), posted at
http://www.house.gov/reform/min/pdfs/
pdf--inves/pdf--dietary--ephedra
--metabolife--rep.pdf \4\ noted that the call records from Metabolife
International contain nearly 2,000 reports of significant AERs for its
products, including 3 deaths, 20 heart attacks, 24 strokes, 40
seizures, 465 episodes of chest pain, and 966 reports of heart rhythm
disturbances. In addition to these cardiac and neurological events,
psychiatric symptoms were also reported. These reports include 46
reports of hospitalization following use of their products, and 82
additional reports of emergency room care. The report stated that in
more than 90 percent of the most serious AERs-- stroke, heart attack,
seizure, and psychosis--where dosage information is documented in the
call record, the consumer had followed the manufacturer's dosage
recommendations. It also stated that among those most significant
adverse event reports for which age was noted, 50 percent of the
consumers were under 35 and many of the consumers were reported as
being in good health with no prior medical problems. Despite the
limited information provided in Metabolife International's call
records, we note that these types of adverse events reported are
consistent with the scientifically documented effects and potential
risks of ephedrine alkaloids in those cases where appropriate
information was available to make a medical evaluation of the reported
event.
---------------------------------------------------------------------------
\4\ FDA has verified the Web site address, but FDA is not
responsible for any subsequent changes to the nonFDA Web sites after
this document publishes in the Federal Register.
---------------------------------------------------------------------------
(Comment 45) Many comments criticized our system for collecting and
evaluating adverse events and our use of AERs. A number of comments
criticized the reporting system, stating that many of the received
reports were insufficiently documented and lacked critical information
necessary for appropriate evaluation. Other comments stated that the
reports were anecdotal
[[Page 6815]]
and that no scientific standards were used in their evaluation.
Several comments stated that our attempt to rely on AERs for
attributing adverse events to dietary supplements containing ephedrine
alkaloids is in conflict with established scientific principles and FDA
policy. The comments cited the criticism of our reliance on AER in the
July 1999 GAO Report, our bases for regulation of Yellow No. 5 which
included AERs and multiple clinical studies, and the opinion that our
AER review system was biased and lacked scientific rigor.
Several comments stated that our methods of data collection might
have affected the integrity of the data. The comments explained that we
included in the database AERs that had not been verified. Many of these
comments also stated that adverse events were frequently reported by
family members and FDA officials rather than by physicians, health care
facilities, and dietary supplement manufacturers. Some comments stated
that certain products that did not contain ephedrine alkaloids were
reported to be associated with adverse events. Several comments
expressed the opinion that the AER database must be corrected to remove
AERs that relate to products that do not contain ephedrine alkaloids
prior to any rulemaking.
(Response) Because there is no mandatory requirement for submission
of adverse event reports involving foods (including dietary
supplements) to us, we rely on voluntary adverse event reporting from
consumers, physicians and other health care professionals, product
manufacturers, poison control centers, and State health agencies as a
monitoring tool in our identification of potentially serious public
health concerns that may be associated with a particular ingredient,
product, or type of product. As with other passive surveillance
systems, we acknowledge that voluntarily submitted adverse event
reports do not always include adequate descriptions of the event and
important elements of medical history, such as preexisting illness or
other therapy. Our concerns about the risks of dietary supplements
containing ephedrine alkaloids are based primarily on the known
pharmacological effects of sympathomimetics and clinical studies using
botanical and/or synthetic ephedrine alkaloids. Based on these
pharmacological effects, we have identified a likelihood of potentially
fatal arrhythmias, increased mortality in heart failure, and an
increased rate of the consequences of elevated blood pressure, such as
heart attack, stroke, and death. All of these events have been reported
to be associated with consumption of dietary supplements containing
ephedrine alkaloids. Because these events also occur spontaneously,
specific occurrences of the events generally cannot be definitively
attributed to dietary supplements containing ephedrine alkaloids,
although they are compatible with the expected effects of these
products. The AERs were, thus, only one component of our evaluation,
which primarily relied on review of the best available scientific
literature, such as peer-reviewed controlled clinical trials. The AERs
are consistent with events expected from ephedrine alkaloids based on
known pharmacological effects and other evidence in the scientific
literature, and the AERs support our findings concerning the risks of
dietary supplements containing ephedrine alkaloids.
a. Definitional issues.
(Comment 46) Some comments argued that only ``life-threatening''
adverse events should have been considered as the basis for the
rulemaking. Another comment pointed out that a ``serious event'' is
described in FDA's publication entitled ``Clinical Impact of Adverse
Event Reporting'' (Ref. 32) as an event that is fatal, life-
threatening, permanently/significantly disabling, requires or prolongs
hospitalization, causes a congenital anomaly, or requires intervention
to prevent permanent impairment or damage. The comment stated that any
event that fails to meet any of these criteria must then be nonserious,
reasonable, or insignificant. The comment also pointed out that an
``adverse effect'' is an unwanted effect and does not necessarily imply
``serious.'' The comment further stated that we should define key
terms, including ``serious,'' ``unreasonable,'' ``significant,''
``adverse effect,'' and ``side effect.''
Several comments also noted that the vast majority of complaints
received by Metabolife International were mild and common. As such, one
comment stated that some of the complaints were more accurately termed
``side effects,'' not ``adverse events.'' One Metabolife International
consultant who reviewed the call records noted that there is no FDA
guidance to define ``significant effect.''
(Response) We do not agree that we should consider only ``serious''
or ``life-threatening'' adverse events in our evaluation of AERs for
dietary supplements containing ephedrine alkaloids. In considering
reports of adverse effects of ephedra, we have focused on the reports
themselves and their implications, not how they were designated. Thus,
a report of tachycardia, not necessarily serious in itself, indicates a
sympathomimetic response that in some patients could be dangerous.
Marked increases in blood pressure would have similar implications and
could suggest greater sensitivity to sympathomimetic effects in
particular individuals. Reports of serious events like stroke, death or
ventricular tachycardia are important, of course, but as noted earlier,
can be difficult to interpret outside of a controlled trial or
epidemiologic investigation. Concerns about ephedra arise principally
because it has effects known to put particular individuals at risk
(those with coronary artery disease or heart failure) or to pose a risk
to any individual with continued use (increased blood pressure).
Nonserious events that suggest sympathomimetic effects of ephedra are
therefore important and need evaluation.
There is no real distinction between side effects and adverse
effects. In either case, they are unwanted effects of the product. The
description of the reported event is what is critical. Although we
agree that the term ``adverse effect'' means there is an unwanted
effect and does not necessarily imply that the event is serious, that
does not mean it is insignificant. Such effects could be indicative of
more serious cardiovascular risks if use of the product is continued.
When considered with the scientific literature and other data, the less
clinically significant effects may provide evidence that the use of a
dietary supplement or dietary ingredient presents a significant or
unreasonable risk of illness or injury.
In the case of dietary supplements containing ephedrine alkaloids,
our evaluation indicates that serious adverse cardiovascular effects
(e.g., heart attack, stroke, worsened heart failure) can be expected to
occur with the use of these products by the general population. Such
events are relevant even if they may be expected to occur because they
are known to be related to a substance, or combination of substances,
contained in the product. Under section 402(f)(1)(A) of the act, a
dietary supplement is adulterated if it presents a significant or
unreasonable risk of illness or injury based on the conditions of use
in its labeling (or under ordinary conditions of use if the labeling is
silent). Therefore, if the labeled use of a dietary supplement
containing ephedrine alkaloids would be expected to result in a risk of
illness or injury, we must consider that risk in evaluating whether the
dietary supplement is adulterated. For these reasons, we
[[Page 6816]]
considered all types of adverse events associated with the use of
dietary supplements containing ephedrine alkaloids, even those that
would not be considered ``serious'' or ``life-threatening.''
(Comment 47) Some comments stated that the AERs were anecdotal and
by their nature do not allow for statistical evaluation. Other comments
stated that AERs cannot establish a causal relationship between ephedra
use and adverse events. Some comments cited the RAND report as support
for the view that a causal relationship has not been shown.
Many comments stated that, without a control group, it is
impossible to predict the number of persons who could experience the
same type of adverse events that occur in the population not exposed to
the product. Several comments argued that we may be detecting
coincidental adverse events, which could have occurred whether or not
consumers used an ephedrine alkaloid-containing dietary supplement.
Many comments also stated, and pointed out that we have stated, that
AERs cannot be used to calculate incidence rates of adverse events
(i.e., the expected rate of adverse events occurring in the population
using a product) because the actual number of persons exposed to the
product is unknown, as is the actual number of adverse events that
occur with use of these products.
(Response) As noted in the comments, the rate of occurrence of
serious adverse events associated with a particular product or
substance cannot be calculated based simply on the number of adverse
events reported. Furthermore, we agree that the RAND report did not
conclude that a causal relationship between ephedra and the reported
adverse events had been shown. Despite the limitations of AERs,
however, they can be of value in an evaluation of whether a dietary
supplement presents a significant or unreasonable risk. Such reports
can be important as signals of potential problems. Moreover, they can
be more or less persuasive as to the strength of association between
exposure to a product and occurrence of an event, depending, in part,
on how likely the event is in the general population in the absence of
the product. Thus, spontaneous reports have repeatedly signaled the
ability of drugs to cause hepatic injury (e.g., bromfenac,
troglitizone) because the events seen were rarely witnessed in the
absence of hepatotoxic drug or viral illness (which could be ruled
out). Similarly, spontaneous reports have shown drug-caused torsade de
pointes-type arrhythmias, which are also rare in the population. For
more common events (e.g., stroke, heart attack, headache), single
reports may be harder to interpret. As previously discussed, the AERs
for dietary supplements containing ephedrine alkaloids are consistent
with events expected based on the scientific evidence, and the AERs
support our findings.
(Comment 48) One comment urged us to disregard an e-mail memorandum
from Dr. Paul Shekelle (Ref. 118) of the RAND Corp. that responds to
our questions about the level of scientific proof that supports a
causal relationship between the use of ephedrine-containing products
and serious adverse events. The comment maintained that the opinions
expressed in the e-mail are speculative, not objective, and not
consistent with the peer-reviewed findings of the RAND report. The
comment expressed concerns that we and others will interpret the e-mail
as an extension or interpretation of the RAND report.
(Response) We are not treating the e-mail by Dr. Shekelle as an
extension or interpretation of the RAND report. In seeking information
from Dr. Shekelle, we were attempting to clarify the basis for RAND's
conclusion regarding evidence of a causal relationship between dietary
supplements containing ephedrine alkaloids and serious adverse events.
We do not consider the Shekelle e-mail and Dr. Shekelle's subsequent
publication (Ref. 119) as influencing the validity or interpretation of
the RAND report, which is the document on which we rely.
(Comment 49) Several comments objected that we did not consider
``denominator data'' in our evaluation. Several comments stated that
when the number of AERs we received is compared to the number of units
sold and the population of users, the incidence of injury is
insignificant or below the threshold for spontaneous illness (e.g., the
incidence of an adverse event in the general population) and that the
level of risk is acceptable. Several related comments argued that if we
made a statistical comparison of the number of AERs to the number of
servings used, we could find the number of AERs to be statistically
insignificant. Several comments made such a statistical comparison. For
example, one comment estimated the annual number of servings of dietary
supplements containing ephedrine alkaloids based on its own sales
figures and an estimate of their share of the market, and concluded
that the 800 AERs represent one adverse event occurring with every 8
million servings. The comments concluded that if the AER rate is
statistically insignificant, the risk would be considered to be
``insignificant'' under the act.
Several comments requested that we consider industry evidence of
the safe use of dietary supplements containing ephedrine alkaloids.
Several of these comments were from manufacturers and distributors of
dietary supplements containing ephedrine alkaloids that discussed the
AERs their companies had received. One comment stated that the number
of serious adverse events that the company received was statistically
insignificant. Other manufacturers and distributors claimed that they
had not received reports of adverse events related to the use of their
dietary supplements containing ephedrine alkaloids when the products
were used according to labeled directions or that lawsuits had not been
filed against them. Comments from several dietary supplement trade
groups or industry committees submitted survey information about the
number of users of particular products or the number of units sold for
particular products and the number of adverse events that were reported
during the survey. These comments indicated that there were no or few
adverse events (and these were mostly of a minor nature) in contrast to
the millions of doses sold.
Many comments noted the experience of firms with respect to the
number of complaints or lawsuits they had received on products
containing particular amounts of ephedrine alkaloids, sometimes in
conjunction with particular amounts of caffeine, and labeled for use
for various levels of time. Some of these comments included information
on the amount of product sold or the number of people consuming the
product in a specified time period.
Several comments suggested that the number of adverse events
estimated from the AERs is inconsistent with international data. For
example, one comment noted that the Committee on Safety of Medicine
(U.K.) indicated that there were only 22 reported adverse events on a
product sold in the U.K. that contains a mixture of ephedrine alkaloids
and caffeine in the 40 years or more that the product has been
available. Similarly, some comments noted that Danish investigators
estimated that 9.6 million doses of a product containing a combination
of ephedrine and caffeine had been sold in Denmark in 1991 and 1992 and
that only 86 reportable adverse events, defined as reactions which
necessitated stopping the therapy, had been reported to the authorities
during that time, despite relatively ``high dosage levels''.
[[Page 6817]]
(Response) We are not persuaded that the lack, or limited numbers,
of adverse events reported to a limited subset of dietary supplement
manufacturers and distributors demonstrates that the use of dietary
supplements containing ephedrine alkaloids is safe. In contrast to the
absence or low number of AERs described in some of the comments, we
have received a total of more than 18,000 AERs directly, through
dietary supplement firms, and from other sources. The AERs and
international data discussed by the manufacturers and distributors in
their comments are consistent with other adverse event reports we have
received. We note that the Danish product referred to by some comments
has been withdrawn from the market for safety reasons, including
serious adverse event reports documenting cardiovascular and nervous
system effects (Refs. 120 and 121).
There is little doubt that dietary supplement adverse events are
underreported (Ref. 20). There is no requirement that manufacturers of
dietary supplements report such events to FDA. Moreover, the usual
reporters of AERs, physicians, are often unaware of the events
themselves or the person's history of dietary supplement use. We
therefore agree with the comments that the number of AERs reported to
us cannot be used to calculate incidence rates. To calculate the
incidence rate of an adverse event in the general population or in a
subgroup of the general population, both numerator (i.e., the number of
times a specific adverse event occurred with the use of a particular
product over a given time period) and denominator (i.e., the total
number of persons using the product over the same time period) data are
needed. For reasons described previously, the adverse events that are
actually reported are likely only a small fraction of the actual number
of adverse events that occur with the use of these products. In
addition, we have no reliable data on the use of these products by the
general population or subgroups of the population. We could not
evaluate the information from industry surveys on the number of people
who use dietary supplements containing ephedrine alkaloids or the
number of units of these products sold because this information was in
summary form only (e.g., the raw data were not submitted). Therefore,
we do not know the actual number of persons who have used the product.
In addition, because we do not have reliable information on the actual
number of adverse events occurring with these products and on the size
of the population exposed to dietary supplements containing ephedrine
alkaloids, we cannot calculate the rate of adverse events occurring in
the population using these products (i.e., incidence rate). Although we
have done rough estimates for the purpose of calculating a potential
economic impact, these estimates cannot be used to determine the
precise incidence rates of adverse events for dietary supplements
containing ephedrine alkaloids. However, we do not believe it is
necessary to calculate the incidence rate to determine that dietary
supplements containing ephedrine alkaloids present an unreasonable
risk. Such a determination does not require us to find actual harm,
only that a product's risk of illness or injury outweighs its benefits
in light of the claims and directions for use in the product's labeling
or, if the labeling is silent, under ordinary conditions of use.
b. Reporting issues, including underreporting.
(Comment 50) Although many comments agreed that the adverse events
for dietary supplements containing ephedrine alkaloids were
underreported, a number of comments disagreed with our estimates in the
June 1997 proposal. Some comments believed that adverse events were
less underreported than we estimated, while others thought they were
more underreported. One manufacturer stated that it does not report the
complaints it receives to us but rather keeps them for its own records.
(Response) As discussed in the response to comment 49 of this
document, we continue to believe that adverse events are underreported
due to the voluntary nature of the adverse event reporting system for
dietary supplements and other factors. The manufacturer comment
confirms that at least some firms in the dietary supplement industry
receive AERs that they do not share with us. We commissioned a study
that estimated that adverse events reported to us represent less than 1
percent of all of the adverse events associated with dietary
supplements (Ref. 122). Our preliminary evaluation of data purchased
from the American Association of Poison Control Centers, covering the
years 1997 through 1999, indicated more adverse events than we had
received for the same years (Ref. 123). In addition, the Office of the
Inspector General of HHS determined that the number of dietary
supplement adverse event reports we received was significantly less
than the number of dietary supplement adverse event reports received by
Poison Control Centers (Ref. 20 at p. 9).
In section VIII.A.5.a.i, we discuss in detail how we estimated
rates of adverse event reporting for purposes of our impact analysis
for this final rule.
(Comment 51) One comment stated that, despite underreporting,
incomplete reports, and inadequate staff, there is no credible evidence
that our reporting system makes errors in detection of adverse event
signals. The comment asserted the validity of an association between
AERs and risks presented by ephedrine alkaloids. The comment argued
that this conclusion is confirmed by the known pharmacology of
ephedrine alkaloids and the types of reports seen in ephedrine clinical
trials and with drugs that have a similar pharmacological action. The
comment noted that 26 percent of the reports over a four-year period
documented dechallenge and 4 percent documented positive rechallenge,
providing additional evidence supporting causation.
(Response) We agree that our spontaneous reporting system detected
the potential health risks associated with dietary supplement products
containing ephedrine alkaloids and that these health risks are
consistent with those documented in the scientific literature and with
the known pharmacology of these products. As stated in the July 1999
GAO report entitled ``Uncertainties in Analyses Underlying FDA's
Proposed Rule on Ephedrine Alkaloids'' (Ref. 124), AERs surveillance
can be important as an early alert to potential problems.
In considering the comments that disputed our estimates of adverse
event reporting rates, it is important to note that we are not relying
on the number of AERs for dietary supplements containing ephedrine
alkaloids to demonstrate quantitatively that these products present an
unreasonable risk. Rather, we are relying on the AERs as supportive
evidence of the risks. Although the fact that we received many AERs for
these products is relevant, an exact count of the number of AERs
associated with consumption of dietary supplements containing ephedrine
alkaloids is not necessary to our determination that these products
present an unreasonable risk.
c. Interpretation of AERs as supporting the existence of public
health risks.
(Comment 52) Several comments stated that the number of AERs does
not raise a public health concern. One comment asserted that AERs with
appropriate use of ephedra are rare. Other comments stated that there
is no
[[Page 6818]]
association between the use of dietary supplements containing ephedrine
alkaloids and serious adverse events when used with appropriate
dosages, including the American Herbal Products Association (AHPA)
trade recommendations. One comment noted that some of the AERs appear
to be related to high amounts of ephedrine (i.e., in excess of 500 mg/
day) and that the relationship of intake to adverse events with the use
of lower amounts consumed is unknown.
(Response) We disagree with these comments. Public health concerns
were initially raised by the number of AERs following consumption of
dietary supplements containing, or suspected to contain, ephedrine
alkaloids in comparison to the number of AERs for all other dietary
supplements; the type of adverse event (e.g. cardiovascular system and
nervous system effects); and the severity of the adverse events
associated with the use of these products. The type, severity, and
number of adverse events reported to us prompted us to investigate
further. In many of these AERs, including those designated as ``most
significant'' in the Congressional minority report (Ref. 117), the
dietary supplement products were consumed as directed on the
manufacturer's label. Although we do not endorse any current trade
recommendations for the use of dietary supplements containing ephedrine
alkaloids, we note that in many of the AERs, the amounts of ephedrine
alkaloids consumed were within the ranges listed in trade
recommendations or in product labeling. In addition, we note that the
ephedrine alkaloid daily dose limit recommended by AHPA (Ref. 101) is
higher than the dose administered to the treatment group in Boozer et
al. (2002), which resulted in significantly higher blood pressure
measured by ABPM when compared to the placebo group.
(Comment 53) Several comments cited the 1999 GAO report (Ref. 124)
to support their criticisms of our the June 1997 proposal. These
comments state that GAO criticized the validity of serious AERs
reported for ephedra, particularly when used according to trade
recommendations.
(Response) We do not agree that the July 1999 GAO report found the
serious AERs reported for ephedra to be invalid (Ref. 124). Although
the July 1999 GAO report criticized our use of adverse event reports to
support the serving size and duration of use limits in the June 1997
proposal, it also emphasized that the adverse events reported to us
were serious enough to warrant FDA's further investigation of the
safety of dietary supplements containing ephedrine alkaloids. In
addition, the report concluded that scientific information indicates
that ephedrine alkaloids can affect the cardiovascular and nervous
systems, citing (among others) published case reports that suggest
ephedrine alkaloids can increase blood pressure in persons with normal
and high blood pressure; predispose certain individuals to tachycardia
(rapid heart rate), and cause cardiomyopathy (disease of the heart
muscle), stroke, or myocardial necrosis (death of cells in the heart).
The 1999 GAO report also noted that adverse events associated with
dietary supplements containing ephedrine alkaloids include effects on
the central nervous system, such as mania, paranoid psychoses, and
seizures.
GAO's 2003 testimony before the Subcommittee on Oversight and
Investigation of the House Committee on Energy and Commerce discussed
and updated some of GAO's findings from its 1999 report on dietary
supplements containing ephedrine alkaloids and provided new
information, including an evaluation of Metabolife International's
records of health-related calls from consumers of Metabolife 356 (Refs.
23 and 24). The 2003 GAO testimony noted that the types of adverse
events identified in the health-related call records from Metabolife
International were consistent with the types of adverse events reported
to us, as well as with the scientifically documented pharmacological
and physiological effects of ephedrine alkaloids. The 2003 GAO
testimony noted that despite the limited information contained in most
of the call records, approximately 14,684 call records contained
reports of at least one adverse event among consumers of Metabolife
356. The 2003 GAO testimony identified 92 serious events that included
heart attacks, strokes, seizures, and deaths and emphasized that these
findings were similar to other reviews of the call records, including
those done by Metabolife International and its consultants. The 2003
GAO testimony noted that, in those call records where age was
documented, many of the serious adverse events occurred in relatively
young consumers, with more than one-third of such adverse event
occurring in individuals under the age of 30. Furthermore, for those
call records in which quantity of use and/or frequency and duration of
use were noted, most of the serious adverse events occurred among
Metabolife 356 users who used the product within the recommended
guidelines, i.e., they did not take more of the product nor consume it
for a longer period of time than the product label recommended. These
findings are consistent with our evaluations of AERs that we have
received regarding dietary supplements containing ephedrine alkaloids
(Refs. 27 and 109).
The 2003 GAO testimony noted that the adverse event reports are
important sources of information concerning health risks of dietary
supplements containing ephedrine alkaloids because the regulatory
framework for dietary supplements is basically one of postmarketing
surveillance and does not require premarket approval. The testimony
stressed that despite the limited information obtained from the
Metabolife International call records, the types of adverse events
reviewed were consistent with the known risks of ephedrine alkaloids,
including serious adverse events such as five reports of death.
Finally, the testimony noted that several years earlier, we had
concluded that dietary supplements containing ephedrine alkaloids
present a ``significant public health hazard'' based upon the adverse
event reports received and the consistency of those reports with the
known pharmacological effects of ephedrine alkaloids.
C. What Are the Known and Reasonably Likely Benefits of Dietary
Supplements Containing Ephedrine Alkaloids?
1. Weight Loss
(Comment 54) Numerous comments, including those from manufacturers
and industry trade groups, stated that the results of the RAND report
and other evidence, including the CANTOX review and the Boozer et al.
clinical studies (Refs. 49 and 125), support or establish the safety
and efficacy of dietary supplements containing ephedrine alkaloids for
weight loss. Several comments stated that RAND concludes that dietary
supplements containing ephedrine alkaloids have proven benefits for
weight loss purposes. Several comments stated that RAND shows that
dietary supplements containing ephedrine alkaloids provide a
statistically significant increase in short-term weight loss compared
to placebo of about 2 pounds per month for up to 6 months.
(Response) We agree that the RAND report found evidence that
supported an association between short-term use of ephedrine, ephedrine
plus caffeine, or dietary supplements containing ephedrine alkaloids
with or without botanicals containing caffeine and a statistically
significant increase in short-term weight loss compared to placebo.
RAND found that combinations of
[[Page 6819]]
botanical ephedrine alkaloids plus botanical sources of caffeine, or
synthetic ephedrine plus caffeine, were more effective in promoting
short-term weight loss than ephedra or ephedrine alone. The RAND report
concluded that ephedrine alkaloid containing products, in combination
with caffeine, resulted in a modest weight loss of approximately two
pounds per month greater than that with placebo over a period of 4 to 6
months.
We also agree that this modest weight loss effect may be perceived
as a benefit by consumers who seek to lose weight for nonhealth related
purposes (e.g., to look slimmer). We do not agree, however, that these
studies demonstrate the long-term weight loss necessary to provide
health benefits. While the improvements in obesity/overweight and the
accompanying risk factors may be demonstrated in as few as 1 to 2
months, the improvements must be maintained for years to achieve a
reduction in risk (Refs. 66, 126, 127, and 128). We note that dietary
supplements cannot be lawfully marketed for the treatment of obesity, a
disease with serious health consequences. From a health perspective,
the goal of weight loss is to prevent the substantial morbidity and
mortality associated with overweight and obesity (Refs. 66, 129, and
130). Obesity itself adversely impacts multiple cardiovascular risk
factors, or comorbidities, including hypertension, dyslipidemia (high
cholesterol), and insulin resistance with glucose intolerance. Clinical
studies have demonstrated improvements in these risk markers with even
modest sustained weight loss (i.e., approximately 5 to 10 percent of
initial body weight). Clinical studies have also demonstrated that both
the weight loss and the improvements in the comorbidities take time to
accrue (i.e., months) and that, as a rule, weight is regained and the
comorbidities worsened when the intervention, pharmacological or
behavioral, is discontinued. Thus, interventions necessary for
successful weight maintenance must be long term. As discussed in
greater detail below in the response to comment 56 of this document,
the reasonably well-documented moderate, short-term weight loss from
use of ephedrine alkaloids, with or without caffeine, does not prevent
or decrease substantial, obesity-related irreversible morbidity and
mortality. We have not found evidence that demonstrates long-term
weight loss with these products.
We note that, to the extent these comments raise the issue of
safety, we address those issues in section V.B of this document.
(Comment 55) A number of comments from manufacturers, distributors,
industry experts, and trade groups were critical of the methodology
used for the RAND report or the conclusions of this review. One comment
stated that RAND does not take a sufficiently quantitative approach in
its review of the data in contrast to the review performed by CANTOX.
The comment also objected that RAND did not perform an efficacy
comparison for ephedra-caffeine and that its dose-response assessment
excludes the medium dosage range (40 to 90 mg), which includes the 6-
month Boozer et al. (2002) study. Consequently, the comment argued that
these omissions preclude any assessment of the degree of agreement or
disagreement between RAND and CANTOX.
Other comments objected to RAND's criteria for study inclusion in
the evaluation process, stating that RAND failed to consider all
relevant and applicable trials. In particular, one comment criticized
RAND's decision to consider only human weight loss trials that lasted
at least 8 weeks, noting that 20 of 46 identified studies were excluded
for this reason, and an additional six studies for other ``alleged''
reasons. Several comments objected to RAND's conclusions that weight
loss research on ephedra, ephedrine, and caffeine (6-month data) is
``short-term'' only and not sufficient to demonstrate long-term weight
loss, and cited additional studies to support this view. One comment
stated that 6 months is longer than the period of time recommended by
FDA's Advisory Review Panel on OTC Miscellaneous Internal Drug Products
with respect to evaluating weight loss ingredients used in OTC drugs.
The comment stated that, by these standards, RAND's 6-month weight loss
efficacy data ``exceeds the scientific requirement for evaluating OTC
weight loss drugs recommended by FDA's advisory panel by 3 months.''
Other comments stated that, from a scientific perspective, there is no
reason to believe the weight loss from dietary supplements containing
ephedrine alkaloids would cease after a 6-month period (Refs. 70, 79,
and 131).
(Response) RAND, using the principles of evidence-based medicine,
established the scope of the review and methodology used in its
assessment of the currently available data. The RAND reviewers limited
their evaluation to those randomized or controlled clinical trials of a
minimum study duration (8 weeks) that provided adequate information,
including sufficient protocol design and safety information on the
basis that shorter treatment durations were insufficient to assess
long-term weight loss. We believe that RAND's study selection criteria
were appropriate. Further, we note that in the absence of statutory
requirements for dietary supplement manufacturers to submit well-
designed, long-term, placebo-controlled studies to us, the available
body of well-controlled clinical data is limited. We believe that RAND
appropriately screened the available data and reviewed all relevant
studies and adverse event reports meeting their stated minimum standard
criteria, and thus we consider the results and conclusions of this
assessment valid. Exclusion of studies not directed toward weight loss
or obesity was appropriate for this evaluation in that these studies
were designed to examine the efficacy of these agents for asthma and
related pulmonary indications, rather than their safety.
We have reviewed the additional studies cited in the comments to
support the effectiveness of dietary supplements containing ephedrine
alkaloids for long-term weight loss (Refs. 68, 79, and 131). The
results of the Filozof study have been presented only in abstract form
and, therefore, neither details of the protocol nor data were available
for review. The Daly et al. study enrolled only 24 subjects for 8 weeks
in a placebo-controlled trial. After that period, 8 subjects were
followed in an open label study for varying durations (1 subject was
followed for 26 months). These additional studies were not evaluated in
the RAND assessment because they did not meet RAND's screening
criteria, and we find these studies to be either irrelevant or
inadequate to change the conclusions stated in the RAND report.
Therefore, we find that the Boozer 2002 study remains the longest (6-
month) placebo-controlled study using ephedrine alkaloids.
Consequently, we agree with RAND's conclusion that there are no studies
showing an effect of dietary supplements containing ephedrine alkaloids
on weight loss for more than 6 months.
Concerning the comment that referenced the Advisory Review Panel on
OTC Miscellaneous Internal Drug Products with respect to evaluating
weight loss ingredients used in OTC drugs, we note that the 1979 report
of this panel was discussed in an advance notice of proposed rulemaking
published in the Federal Register on February 26, 1982 (47 FR 8466).
Based on the standard of practice at that time, the Advisory Review
Panel
[[Page 6820]]
recommended that non-monograph weight loss ingredients (i.e., those not
classified as GRASE) be studied for a period of 12 weeks to demonstrate
effectiveness.
The treatment of obesity has evolved over the past 50 or so years
(Refs. 127 and 128). In the 1960s, the mainstay of obesity treatment
was behavioral modification and drugs were approved for short-term
treatment to ``jump start'' patients' weight loss. There was a paradigm
shift in the 1990s, with the realization that obesity is a chronic
disease requiring long-term treatment, both with behavior modification
and long-term drug therapy, when appropriate, in addition to diet and
exercise. This shift is reflected in our draft guidance published in
1996 recommending the performance of clinical trials with a minimum 12-
month treatment duration (see FDA Draft Guidance for The Clinical
Evaluation of Weight-Control Drugs, Division of Metabolic and Endocrine
Drug Products, issued on September 24, 1996) (Ref. 129). Therefore,
because the treatment of obesity has evolved over time, the 1982 OTC
Advisory Panel recommendations do not reflect current scientific
understanding of effective treatment of obesity. There are currently no
GRASE OTC drug products for weight loss or management.
(Comment 56) Many comments stated that obesity is a disease with
serious health consequences. Numerous comments from consumers and
physicians contained personal testimonials regarding the efficacy of
dietary supplements containing ephedrine alkaloids for weight loss.
Several physicians noted that patients who used these products were
able to achieve long-term weight loss with an overall improvement of
health, including improved cholesterol levels and lower blood pressure.
No data were submitted, however, to support these statements. Several
comments stated that ephedrine alkaloids are an effective tool to fight
obesity. Several comments expressed the view that there are health
benefits from short-term weight loss. Several other comments stated
that dietary supplements containing ephedrine alkaloids are as--or
more--effective for weight loss than some prescription drugs (e.g.,
amphetamine, phentermine, sibutramine, phendimetrazine). Another
comment stated that the evidence suggested that ephedra/ephedrine-
caffeine supplements are as effective as OTC drugs for weight
management. One comment stated that other modalities used to promote
weight loss are very difficult, very dangerous, or very unsuccessful.
A comment by an industry trade group stated that the amount of
weight loss identified by RAND for dietary supplements containing
ephedrine alkaloids (approximately 2 pounds per month greater than
placebo) is similar to that reported for approved obesity drugs (citing
Ref. 128). Further, the comment asserted that ``similar to ephedra-
containing supplements, there is no long-term information [on weight
loss] for any but the two most recently approved drugs [sibutramine and
orlistat]'' and that few studies of drugs approved for weight loss have
extended to 6 months or beyond. One comment stated that double-blind
placebo-controlled studies, including Boozer et al. (2002) (Ref. 49)
have addressed the safety and efficacy of the dietary supplements
containing ephedrine alkaloids, and further stated that the low cost of
these products is beneficial, especially for low income groups where
maintenance of a good diet is a challenge.
In contrast, other comments from physicians and medical societies,
while acknowledging the results of the RAND report showing modest, but
statistically significant short-term weight loss, questioned such a
weight loss effect in light of the risks of these products. One comment
indicated that this modest degree of ``drug-induced weight loss'' has
never been shown to reduce the increased morbidity observed in obese
patients. Several comments stated that there is no evidence for
efficacy or safety of chronic treatment with ephedra. One medical
association stated that the very modest benefits of ephedra combined
with caffeine on short-term weight loss are far outweighed by the
adverse effects observed in the clinical trials and the serious risks
reported with the use of dietary supplements containing ephedrine
alkaloids.
Several other comments, including those from an herbalist
association and an herbal product manufacturer, stated that the use of
these supplements, although effective, is not a sensible or healthy
approach to long-term, sustainable weight management. The comment from
the herbalist association also stated that obesity, with its higher
risk for cardiovascular disease, is more likely to be a
contraindication rather than an indication for the use of ephedra. A
comment from a medical association said that NIH guidelines for the
pharmacological treatment of adult obesity state that herbal
preparations, including ephedra-containing products, are not
recommended as part of a weight-loss program (Ref. 66).
Several comments, including one by a trade association and a
medical society, while acknowledging the conclusions of the RAND report
with regard to ephedrine alkaloids and weight loss, said that this
effect should not be construed to imply that dietary supplements
containing ephedrine alkaloids can treat diseases. One comment
expressed the view that we should consistently state that obesity is a
disease and, therefore, should only be treated with drugs that have
been approved as safe and effective for that disease. These comments
stated that use of dietary supplements to ``treat'' obesity is
inappropriate.
(Response) As stated previously, we agree that obesity is a disease
with serious health consequences; however, as some comments noted,
treatment of a disease is outside the scope of the uses authorized for
dietary supplements under DSHEA. Consequently, although dietary
supplements containing ephedrine alkaloids could, if they did not
present an unreasonable risk of illness or injury, be labeled for
ordinary weight loss, they are subject to regulation as drugs if
promoted for the treatment of obesity (65 FR 1000 at 1026 and 1027,
January 6, 2000). We agree with the comments stating that obesity
should be treated only with drugs that have been approved as safe and
effective for that use.
We do not agree with the comments comparing the effectiveness of
dietary supplements containing ephedrine alkaloids for weight loss to
approved prescription drugs. The drugs mentioned by the comments are
approved for the treatment of obesity, which is a use for which dietary
supplements cannot be marketed. Furthermore, we are unaware of any data
that have made direct comparisons between dietary supplements
containing ephedrine alkaloids for weight loss and drugs approved for
the treatment of obesity. As discussed previously, prescription drugs
for the treatment of obesity are no longer approved on the basis of
short-term data or for short-term use. Of note, the few prescription
drugs that were approved for short-term use to ``jump-start'' weight
loss are all stimulants and are controlled substances, the first group
being approved in 1939 (amphetamine) and the last being approved in
1979 (phendimetrazine). The use of the majority of these drugs has
fallen out of favor or the drugs have been withdrawn from the U.S.
market. Whether the remainder of these drugs with indications for
short-term use should be withdrawn is beyond the scope of this
rulemaking. The rationale for requiring
[[Page 6821]]
long-term studies (1 to 2 years) to evaluate drugs intended to treat
obesity was thoroughly discussed in the 1995 FDA/Center for Drug
Evaluation and Research (CDER) Endocrinologic and Metabolic Drugs
Advisory Committee Meeting. In that meeting, the panel discussed the
duration of trials for evaluating both efficacy and safety of drugs for
the treatment of obesity and used the example of Fluoxetine as a drug
that demonstrated efficacy for weight loss at 6 months but did not
promote additional weight loss or maintain previous weight loss in
longer term (1-year) studies, although the risk for experiencing
adverse effects still persisted.
Alleged economic benefits of these products are not considered as a
component of our evaluation of their risks and benefits. Therefore,
comments suggesting an economic benefit from using dietary supplements
containing ephedrine alkaloids as an alternative to drugs for weight
loss are not relevant to whether dietary supplements containing
ephedrine alkaloids present an unreasonable risk. We also note that
there are currently no stimulant-containing OTC drugs (including those
with phenylpropanolamine) legally marketed for weight management and
that amphetamine is no longer labeled for weight loss. There are no
existing final OTC drug monographs for any weight control drug
products, although one nonstimulant ingredient (benzocaine) remains to
be evaluated for this use as part of FDA's OTC drug review and can
continue to be marketed pending the outcome of that review.
The comments that mentioned health benefits from short-term weight
loss submitted no data to support this contention, and we are not aware
of any studies that indicate any meaningful health benefit from short-
term weight loss. In the longest controlled study to date on the effect
of ephedrine alkaloid containing products on weight loss by Boozer et
al. (2002) (Ref. 49), subjects treated with placebo, plus diet and
exercise recommendations, lost an average of approximately 6 pounds
over a period of 6 months (Ref. 49). Subjects treated with a
proprietary blend of herbal ephedra and kola nut (a source of
caffeine), plus diet and exercise recommendations, lost an average of
approximately 12 pounds during the same time period. As described
previously in the response to comment 22 of this document, on balance
this trial did not show a favorable effect on cardiovascular risk
factors. To the contrary, there was a statistically significant
increase in heart rate in the ephedra/kola nut (i.e., herbal ephedrine
alkaloids/caffeine) treated subjects compared to the control group.
Moreover, 24-hour measurements of blood pressure measured by ABPM at 1
month showed that the ephedrine alkaloid/caffeine treated subjects had
blood pressure that was approximately 4 mm Hg higher than the placebo-
treated subjects for both systolic and diastolic blood pressure.
While the authors report small but statistically significant
decreases in total cholesterol and low density lipoproteins (LDL)
cholesterol, the clinical significance of the net 3 mg/dl and 8 mg/dl
decreases, respectively, cannot be determined from this study. In
studies designed to assess modifications in cardiovascular risk
factors, cholesterol changes are reported as percentage change from
baseline. These data are not available from the Boozer et al. (2002)
study (Ref. 49).
(Comment 57) A number of comments stated that the Danish experience
using ephedrine/caffeine in a prescription drug for the treatment of
obesity supported the use of dietary supplements containing ephedrine
alkaloids for weight loss. One comment from a manufacturer of dietary
supplements containing ephedrine alkaloids shared the opinion that the
effectiveness of ephedrine alkaloids ``to support one's diet'' has been
demonstrated in numerous studies, involving hundreds of patients in
well-controlled environments, and that efficacy has also been
demonstrated by extensive use data in the United States and Denmark. A
comment from a medical association stated that, in Denmark, ephedrine
is available to treat obesity, but only by prescription. Another
comment stated that the Danish ephedrine-caffeine product (Letigen) has
been banned and withdrawn from the market because of safety issues.
(Response) We agree with the comments that the product used in
Denmark, Letigen, was a prescription drug and that this drug has been
withdrawn from the market for safety reasons, including serious adverse
event reports documenting cardiovascular and nervous system effects
(Refs. 120 and 121). We note that certain studies from Denmark using
the ephedrine-caffeine combination found in Letigen were considered as
part of the RAND report. We do not agree with the comment that numerous
studies have demonstrated the effectiveness of ephedrine alkaloids to
support weight loss for the treatment of obesity, as discussed
previously. The use of dietary supplements containing ephedrine
alkaloids has been shown to produce a small, short-term weight loss,
but no studies showing long-term weight loss with accompanying benefits
to health have been conducted. In any case, if botanical ephedrine
alkaloid products could be shown effective in long-term treatment of
obesity or for long-term weight loss in people who are not obese, they
would need to be marketed as prescription drugs and meet the standards
of safety and effectiveness legally mandated for such products because
physician supervision would be necessary to adequately mitigate the
risks of using these products continuously in the long term.
2. Enhancement of Athletic Performance
(Comment 58) Several comments discussed the effects of ephedrine
alkaloids on athletic performance. One comment noted that, while RAND
states that ephedrine is a good surrogate for evaluation of dietary
supplements containing ephedrine alkaloids, RAND does not make this
extrapolation for athletic performance. Many other comments stated that
there are few data to support the use of synthetic ephedrine alkaloids,
and no data to support the use of dietary supplements containing
ephedrine alkaloids to enhance athletic performance. Therefore, these
comments do not consider the enhancement of athletic performance to be
an appropriate use for dietary supplements containing ephedrine
alkaloids. According to some comments, RAND concluded that there are
insufficient data to support use for enhancement of athletic
performance. One comment asserted that any effect on athletic
performance is more likely due to the caffeine in ephedrine-caffeine
dietary supplements. According to another comment, the few studies that
have assessed the effect of ephedrine for this use support a modest
effect of ephedrine plus caffeine on very short-term (1 to 2 hours
after a single dose) athletic performance in a highly selected,
physically fit population, but no studies have assessed the effect of
dietary supplements containing ephedrine alkaloids.
(Response) We generally agree with these comments. The RAND report
provides the most comprehensive, currently available review of efficacy
studies for ephedrine alkaloid containing products, focusing on two
popular uses of these products--athletic performance and weight loss
(see section V.C.1 of this document). (Note that the RAND report did
not consider the effectiveness data for ephedrine alkaloid containing
products marketed as drugs for other uses, such as to treat asthma, or
for other dietary supplement uses of such products). The effect of
synthetic ephedrine on athletic
[[Page 6822]]
performance was assessed in seven studies that were reviewed in the
RAND report. The RAND report noted that the effects of ephedrine on
exercise performance were most often studied acutely (e.g., 1 to 2
hours after a single dose) (Refs. 21 and 22). The RAND report could
identify no studies that assessed the effect of dietary supplements
containing ephedrine alkaloids on athletic performance. While the RAND
report found that existing data supported a modest effect of synthetic
ephedrine alkaloid containing products plus caffeine on athletic
performance enhancement in healthy males in the very short term, no
data support a sustained improvement in athletic performance over any
significant time period. In these studies, the performance enhancement
effect was demonstrated only with a combination of synthetic ephedrine
and caffeine, not with ephedrine alone. Therefore, since the available
evidence does not indicate that ephedrine itself enhances athletic
performance, there is no need to address the issue as to whether
ephedrine is a good surrogate for ephedra in evaluating athletic
performance enhancement with the use of dietary supplements containing
ephedrine alkaloids.
We determined that certain labeling claims made by manufacturers of
dietary supplements containing ephedrine alkaloids for athletic
performance enhancement were unsubstantiated in light of the findings
in the RAND report. These claims were the subject of warning letters
sent to various manufacturers in February and March 2003 (available at
http://www.fda.gov/bbs/topics/NEWS/ephedra/letterslist.html (list of
firms) and http://www.fda.gov/bbs/topics/NEWS/ephedra/warning.html
(sample letter).
3. Eased Breathing
We are aware that there are teas and other types of dietary
supplements containing ephedrine alkaloids marketed with claims such as
``eased breathing'' or ``better breathing.'' There are no data that
support a benefit to breathing from dietary supplements containing
ephedrine alkaloids in healthy people. Moreover, because healthy people
are able to breathe without difficulty, we do not believe there is any
respiratory benefit in the absence of a disease state (e.g., asthma or
a respiratory infection). We note that claims to treat or mitigate a
disease, or the effects of a disease, subject a product to regulation
as a drug under the act.
4. Other Uses
We are also aware that dietary supplements containing ephedrine
alkaloids are promoted for other uses, such as to ``feel better,''
``feel more alert,'' and ``energized.'' Effects such as ``feel better''
are subjective in nature and difficult to quantify. The agency is
unaware of any data substantiating these types of subjective effects.
Effects such as ``alertness'' and ``energy'' are consistent with the
pharmacological properties of ephedrine alkaloids, although we are not
aware of any studies evaluating ephedrine alkaloid products for these
uses. Effects like alertness and energy may be of modest benefit to the
individual (if they occur), but such effects are temporary and do not
improve health. Any such temporary benefits must be weighed against the
health risks discussed in section V.B of this document, which can
result in long-term or permanent, serious adverse health effects.
D. Do Dietary Supplements Containing Ephedrine Alkaloids Present an
Unreasonable Risk?
1. What Does ``Unreasonable Risk'' Mean?
A threshold issue is the legal standard of ``significant or
unreasonable risk of illness or injury'' (section 402(f)(1)(A) of the
act). By its plain language, this standard requires evidence of
``significant or unreasonable risk of illness or injury'' (emphasis
added).'' There is no requirement that there be evidence conclusively
demonstrating causation of actual harm in specific individuals. In our
evaluation of ``significant or unreasonable risk,'' we can consider any
relevant evidence, including scientific data about the toxicological
properties of a dietary ingredient or its mechanisms of action;
scientific information about the well-known effects of
pharmacologically-related compounds, including those regulated as
drugs; the results of clinical studies, including observational
studies; and adverse event reports that have been subject to sound
scientific analysis. The Government's burden of proof for ``significant
or unreasonable risk'' can be met with any science-based evidence of
risk, without the need to prove that the substance has actually caused
harm in particular cases.
Thus, a dietary supplement that caused a sustained rise in blood
pressure across the population would increase the risk of
cardiovascular events including stroke, heart attack, or death to that
population. Even risks that may not be detectable in small studies or
studies of short duration could, over time, and on a population-wide
basis, result in hundreds or thousands of adverse events. The
Government's burden of proof for ``unreasonable risk'' is met when a
product's risks outweigh its benefits in light of the claims and
directions for use in the product's labeling or, if the labeling is
silent, under ordinary conditions of use.
(Comment 59) Most comments that articulated a view agreed with the
general notion that we must consider a risk-benefit calculus to
determine whether dietary supplements containing ephedrine alkaloids
present an unreasonable risk, although the comments differed as to how
to perform such a calculus and as to the conclusion about whether the
risks of these products outweigh their benefits. Several comments
agreed with our interpretation, as published in (Ref. 132), that a
``significant or unreasonable risk'' exists when a product's risks
outweigh its benefits, based on the available scientific evidence, in
light of the claims the product makes and in light of the products
being directly sold to consumers without medical supervision. One
comment from a public interest group stated that this interpretation
represents a reasonable and practical interpretation of the act that
offers some protection to consumers. One comment argued that this
interpretation is not permissible under Chevron U.S.A., Inc. because we
have never adopted a risk-benefit calculus in assessing the safety of
foods and because the legislative history of DSHEA does not indicate
any Congressional intent to establish a risk-benefit analysis for
dietary supplements. The comment stated that we should determine
whether risks are ``unreasonable'' without resorting to an assessment
of the benefits of the product.
(Response) We agree with the comments stating that a risk-benefit
calculus is appropriate to determine whether dietary supplements
containing ephedrine alkaloids present an unreasonable risk of illness
or injury under conditions of use recommended or suggested in the
labeling, or if no conditions of use are suggested or recommended in
the labeling, under ordinary conditions of use. The relevant analysis
for evaluating an agency's interpretation of a statute is set forth in
Chevron U.S.A., Inc. v. Natural Resources Defense Council, 467 U.S. 837
(1984). Under Chevron, the first question is whether Congress has
directly spoken to the precise question at issue (Step 1). If so, the
agency must implement the unambiguous intent of Congress Id. at 842-
843. If Congress has
[[Page 6823]]
not directly spoken to the precise question at issue, our
interpretation will be upheld as long as it is based on a ``permissible
construction'' of the statute (Step 2) Id. at 843-844.
In determining whether Congress has specifically addressed the
question at issue, ``courts must exhaust the traditional tools of
statutory construction, including looking at the statute's text,
structure, and legislative history.'' Chevron v. Federal Energy
Regulatory Commission, 193 F.Supp.2d 54, 67 (D.D.C. Cir. 2002). Section
402(f)(1)(A) of the act states that a dietary supplement is adulterated
if it presents a significant or unreasonable risk of illness or injury
under the conditions of use recommended or suggested in labeling, or,
if the labeling is silent, under ordinary conditions of use. The plain
meaning of the statute is the starting point of statutory
interpretation. (See 2A SUTHERLAND STATUTORY CONSTRUCTION 81 (5th ed.
1992).) The words ``significant'' and ``unreasonable'' have two
different meanings. ``Significant'' involves an evaluation of risk
alone. The plain meaning of ``unreasonable,'' on the other hand,
connotes comparison of the risks and benefits of the product. A risk
could be significant but reasonable if the benefits were great enough
to outweigh the risks. That ``unreasonable risk'' entails a balancing
test in which the benefits of the product or activity are weighed
against its dangers is well-established in tort law (See PROSSER AND
KEETON ON THE LAW OF TORTS, Sec. 31, at 173 (5th ed. 1984).)
In assessing whether Congress has clearly spoken to the question at
issue, a court ``should not confine itself to examining a particular
statutory provision in isolation. Rather, it must place the provision
in context, interpreting the statute to create a symmetrical and
coherent regulatory scheme'' (FDA v. Brown and Williamson Tobacco
Corp., 529 U.S. 120, 121 (2000)). The term ``unreasonable risk'' is
used in other provisions of the act, e.g., in the provisions related to
medical devices. In the medical device classification provisions, Class
III devices are distinguished from Class I and Class II devices in part
because they present a ``potential unreasonable risk of injury or
illness.'' The legislative history of the device provisions provides
some indication of how Congress intended FDA to interpret the term
``unreasonable risk in this context. The House Committee Report states:
``the requirement that a risk be unreasonable contemplates a balancing
of the possibility that illness or injury will occur against the
benefits of use'' (H. Rept. 853, 94th Cong., 2d Sess. 19 (1976)).
Therefore, ``unreasonable risk'' in the context of classification of
medical devices is properly interpreted to require a risk-benefit
calculus. There is nothing in the provisions of the act dealing with
dietary with dietary supplements, or the legislative history thereof,
that would suggest that FDA should interpret the term ``unreasonable
risk'' in the context of dietary supplements differently than it does
in the context of medical devices.
An interpretation of unreasonable risk as entailing a balancing of
the risks and benefits of the product is also consistent with the
interpretation of other similar statutory provisions outside the act.
The Toxic Substances Control Act contains an ``unreasonable risk''
standard, and legislative history indicates that Congress intended that
this standard be evaluated through a balancing test (e.g., H. Rept. 94-
1341, 94th Cong., 2d Sess. 32 (1976)). Indeed, it is difficult to
construct an alternative formulation for the phrase ``unreasonable
risk.''
Based upon the plain meaning of ``unreasonable risk,'' the judicial
interpretation of that phrase, and legislative history interpreting
``unreasonable risk'' in other contexts, including the device
provisions of the act and other statutes, we conclude that Congress
unambiguously intended that an assessment of ``unreasonable risk'' in
the dietary supplement context should entail a risk-benefit analysis.
In the alternative, if a court were to find that Congress has not
directly spoken to the issue of whether ``unreasonable risk'' in the
dietary supplement context is demonstrated by balancing risks and
benefits, our interpretation of an ambiguous provision should receive
deference so long as it is ``permissible'' (Chevron Step 2). In
interpreting ambiguous statutory language, we are guided by the same
criteria we evaluated in Step 1 of the Chevron analysis, i.e., the
statute's text, structure, history, and purpose (See Bell Atlantic
Telephone Cos. v. FCC, 131 F.3d 1044, 1049 (D.C. Cir. 1997); Chevron
U.S.A., Inc. v. FERC, 193 F. Supp. 2d at 68). Our interpretation of the
``unreasonable risk'' standard for dietary supplements as requiring a
comparison of the risks and benefits of use is consistent with the
purpose of the act, as amended by DSHEA, to promote public health and
safety. This interpretation is also consistent with the legislative
history of the medical device classification provisions. Therefore, our
interpretation that ``unreasonable risk'' implies a weighing of the
risks and benefits of use is, at a minimum, a ``permissible
construction.''
In the absence of explicit standards for the evaluation of
``unreasonable risk,'' one comment urged us to be guided by precedent
from other agencies. The comment highlighted the Consumer Product
Safety Act (CPSA), its implementing regulations, and related case law.
The comment stated that any assessment of ``unreasonable risk'' must
include a balancing of risks and benefits, a stringent burden on us to
demonstrate that the product poses an unreasonable risk of injury,
evidence other than consumer complaints, and valid scientific data
sufficient to predict how likely an injury is to occur. (Citing Gulf
South Insulation v. CPSC, 701 F.2d 1137, 1143 (5th Cir. 1983)), (citing
Aqua Slide `N' Dive v. CPSC, 569 F.2d 831, 838 (5th Cir. 1978)), the
comment stated, ``[T]he ultimate question in assessing unreasonable
risk is whether the record contains `such relevant evidence as a
reasonable mind might accept as adequate to support a conclusion.'''
The comment acknowledged differences in the statutes, including the
explicit statutory requirement in CPSA that the regulation impose the
least burdensome requirement that prevents or adequately reduces the
risk injury for which the rule is being issued (15 U.S.C.
2058(f)(3)(F)). The comment also cited Consumer Product Safety
Commission (CPSC) case law stating that reliable evidence of the likely
number of injuries is necessary to determine whether a risk is
unreasonable (Southland Mowor Co. v. CPSC, 619 F.2d 499, 510 (5th Cir.
1980)).
(Response) We do not agree that our interpretation of
``unreasonable risk'' must be confined to the view reflected in the
CPSC case law cited by the comment. We have concluded, based on a
Chevron analysis, that Congress expressly intended ``unreasonable
risk'' to entail a risk-benefit analysis (see the response to comment
59 of this document). In the alternative, if the term ``unreasonable
risk'' is ambiguous, we may interpret its meaning under Chevron. As the
comment noted, CPSA contains an extensive list of findings that the
CPSC must make, based on substantial evidence, before concluding that a
consumer product poses an unreasonable risk, including, for example:
(1) The degree and nature of the risk of injury the rule is designed to
eliminate or reduce; (2) the approximate number of consumer products,
or types or classes thereof, subject to such rule; and (3) any means of
achieving the objective of the order while minimizing adverse effects
on competition or disruption or dislocation of
[[Page 6824]]
manufacturing and other commercial practices (15 U.S.C. 2058(f)(1) and
(f)(3)). The requirements imposed on CPSC in the cases that the comment
cited are based on the explicit requirements of CPSA. In contrast, the
adulteration provision in section 402(f)(1)(A) of the act does not
require that we make any such findings. Like section 402(f)(1)(A) of
the act, other parts of the act that require an evaluation of
unreasonable risk, such as the device classification and banning
provisions, also do not require that we make the findings set forth in
CPSA. Had Congress intended that FDA make specific findings such as the
degree of risk of injury, it could have so directed in the act;
however, it did not. Our conclusion that dietary supplements containing
ephedrine alkaloids present an unreasonable risk is based upon our
finding that the risks of heart attack, stroke, and death outweigh the
minimal benefits conferred by the supplements. Our conclusion is
consistent with Congress's express intent in section 402(f)(1)(A) of
the act.
(Comment 60) One comment by a health professional group stated that
unreasonable risk likely exists when there is no information that
substantiates a clear therapeutic benefit or describes a predictable
relationship between exposure (dose) and response, and when the
appropriate product dose is not known or achievable.
(Response) We agree that unreasonable risk exists when a dietary
supplement presents a risk to health, and there is no information
substantiating a benefit sufficient to outweigh that risk. In this
rulemaking, we base our determination that dietary supplements
containing ephedrine alkaloids present an unreasonable risk under
section 402(f)(1)(A) of the act on a risk-benefit analysis, finding
that the risks of heart attack, stroke, and death outweigh the benefits
that may result from such products. In the absence of a use that
results in a benefit that outweighs the risks of these products, we
conclude that all such products pose an unreasonable risk. We therefore
need not determine whether an unreasonable risk exists when the precise
relationship between exposure and response is not predictable or when
the appropriate product dose is not known or achievable.
(Comment 61) Several comments stated that proof of causation is
required to establish unreasonable risk.
(Response) We do not agree that proof of causation is required to
establish unreasonable risk under section 402(f)(1)(A) of the act, and
conclude that the plain meaning of the standard precludes such an
interpretation. In determining whether Congress has specifically
addressed the question at issue, ``courts must exhaust the traditional
tools of statutory construction, including looking at the statute's
text, structure, and legislative history'' (Chevron U.S.A., Inc. v.
FERC, 193 F.Supp. 2d at 67). The plain meaning of the statute is the
starting point for an analysis of legislative intent. The most
applicable definition of the word ``risk'' in Merriam Webster's
Collegiate Dictionary is ``possibility of loss or injury'' (Merriam
Webster's Collegiate Dictionary, 10th ed. 1008 (2002)) (emphasis
added). Black's Law Dictionary defines ``risk,'' in part, as follows:
``In general, the element of uncertainty in an undertaking; the
possibility that actual future returns will deviate from expected
returns. Risk may be moral, physical, or economic.'' Black's Law
Dictionary, 6th ed. 1328 (1990) (emphasis added). The words
``possibility'' and ``uncertainty'' in these definitions indicates that
proof of a definitive causal relationship between the product and
illness or injury is not required under section 402(f)(1)(A) of the
act. If Congress had intended that definitive proof that a dietary
supplement causes harm be a requirement for a showing of adulteration,
it would not have used the word ``risk'' in the statute, and would have
instead provided that a dietary supplement is adulterated if it
``causes'' illness or injury. This interpretation is consistent with
other parts of the act, as interpreted in legislative history and case
law. For instance, the legislative history of the medical device
banning provisions, which require a showing of ``substantial deception
or an unreasonable and substantial risk of illness or injury'' states
that ``[A]ctual proof of deception or injury to an individual is [not]
required'' (Section 516 of the act (21 U.S.C. 360f), H. Rept. 853, 94th
Cong., 2d Sess. 19 (1976)). Case law on medical device classification
also supports that we need not have causal evidence of harm (See Lake
v. FDA, 1989 WL 71554 (E.D. Pa.)) (upholding FDA's finding of
unreasonable risk where the risks were unknown and the benefits
unproven)). Therefore, we conclude that Congress has spoken clearly and
unambiguously that proof of causation is not required to show that a
dietary supplement presents an ``unreasonable risk'' under section
402(f)(1)(A) of the act.
Our interpretation is also consistent with other statutes that
regulate public health risks, most notably TSCA (15 U.S.C. 2601 et seq.
(1976)). TSCA authorizes the EPA to place restrictions on chemical
substances if it finds that ``* * * there is a reasonable basis to
conclude that the [chemical substance] presents or will present an
unreasonable risk of injury to health or the environment'' (Id. Sec.
2605(a)). The legislative history of this provision states:
This standard for taking action recognizes that factual
certainty respecting the existence of an unreasonable risk of a
particular harm may not be possible and the bill does not require
it. Further, regulatory action may be taken even though there are
uncertainties as to the threshold levels of causation.
(H. Rept. 94-1341, 94th Cong., 2d Sess. 25 (1976)).
(Comment 62) Several comments stated that any FDA regulatory
approach to dietary supplements containing ephedrine alkaloids must
consider both risks and benefits, and moreover, that we should
determine, based on scientific evidence, a risk-benefit ratio for
assessing their safety. These comments suggested that, if we were to
set a break-even point, a decision matrix should be established along
the following lines: (1) A benefit-to-risk ratio below the break-even
point would mean that the risks outweigh the benefits and this would
justify either a decision to (a) ban dietary supplement products
containing ephedrine alkaloids or (b) restrict access to a case-by-
case-basis, i.e., prescription; (2) a benefit-to-risk ratio in excess
of the break-even point would mean that the benefits outweigh the risks
and this would justify continued availability, with appropriate warning
labels, dosage instructions, etc.; and (3) a benefit-to-risk ratio
equal to the break-even point would mean that the risks equaled the
benefits and this would justify either (a) continued availability under
the present regulatory framework with appropriate labeling or (b)
prescription-only access, whereby a medical professional would make the
decision as to whether or not the product was appropriate for an
individual consumer on a case-by-case basis.
One comment by a medical association stated that, because dietary
supplements are classified as foods, and therefore are assumed to be
safe, it is imperative that such products have no risks and provide
some benefit to consumers. More specifically, the comment stated that
dietary supplements containing ephedrine alkaloids should be safer than
drugs and should have a much higher overall benefit/risk ratio when
compared to drugs.
(Response) We agree that in regulating dietary supplements, we
should
[[Page 6825]]
consider both risks and benefits. As discussed previously in this
document, we also agree that we should weigh risks and benefits when
evaluating the safety of dietary supplements under the adulteration
standard in section 402(f)(1)(A) of the act. With regard to the comment
from the medical association, we agree in part and disagree in part.
Although the comment is correct that dietary supplements are classified
as foods, we do not agree that they are required to have no risks at
all. Section 402(f)(1)(A) of the act provides that a dietary supplement
is adulterated if it ``presents a significant or unreasonable risk of
illness or injury'' (emphasis added) as labeled, not if it presents any
risk at all. Accordingly, risks that are insignificant and reasonable
in light of the benefits from the supplement would not render a dietary
supplement adulterated. Further, we note that conventional foods are
not always risk-free. With regard to the comment's statements that
dietary supplements should be safer than drugs and have a higher
overall benefit/risk ratio than drugs, we do not believe it is
necessary to reach these issues. For purposes of this rulemaking, we
are considering whether the known and reasonably likely risks of
dietary supplements containing ephedrine alkaloids outweigh their known
and reasonably likely benefits. It is not necessary to determine
generally how the risk/benefit ratio of dietary supplements should
compare to that of drugs.
2. Do Dietary Supplements Containing Ephedrine Alkaloids Present an
Unreasonable Risk Under Labeled or Ordinary Conditions of Use?
(Comment 63) Several comments stated there is enough evidence, both
scientific and anecdotal, to conclude that the risks of taking dietary
supplements containing ephedrine alkaloids are so severe and reported
adverse events sufficiently numerous to conclude that the risks clearly
exceed the benefits because either there are no benefits or the
benefits are unsubstantiated or modest for both efficacy and duration.
These comments included references to support their conclusions. Some
cited the RAND report's conclusions regarding the very modest benefit
for short-term weight loss and the questionable benefit for other uses;
according to the comments, these limited or questionable benefits are
far outweighed by adverse events observed in clinical trials. Other
references submitted by these comments included (Refs. 19, 34, 42, and
133 through 136).
Several comments argued that the harm caused by certain medical
conditions--for example, obesity--is so severe as to render the
unsubstantiated (in the commenter's view) risks of taking dietary
supplements containing ephedrine alkaloids insignificant relative to
the benefits that would accrue from use of these products. In this
view, the weight loss benefit would exceed any potential risk from
taking the product and the risk is not unreasonable when compared to
the harm caused by obesity. Several comments cited the prevalence of
obesity and an increase in obesity over time, and urged us not to take
away one important tool for consumers to address the problem. Two
comments cited statistics showing that 54 percent of adults are obese
in the United States, that the prevalence of obesity increased by 30
percent from 1980 to 1994, and that in 1997 the Centers for Disease
Control and Prevention (CDC) attributed 42 percent of deaths to
conditions that typically result from obesity. One comment stated that
the risks due to obesity are a greater danger than the rare incidences
of stroke or heart attacks attributed to dietary supplements containing
ephedrine alkaloids.
Other comments concluded that dietary supplements containing
ephedrine alkaloids do not present an unreasonable risk because the
risks do not outweigh the benefits. They argued that while the benefits
of dietary supplements containing ephedrine alkaloids are
substantiated, the adverse events reported are either mild, anecdotal,
or unsubstantiated and not scientifically valid. Some comments cited
the RAND report to support the benefit of ephedrine alkaloids for
short-term weight loss and the lack of adverse effects in clinical
trials. The comments assert that only a speculative risk for serious
adverse events exists and that RAND concluded that an assessment of
case reports is insufficient to reach conclusions regarding causality.
(Response) We have carefully reviewed the preceding comments, and
note that many of these issues have been addressed in more detail in
the scientific evaluation sections V.B and C of this document. Based on
the scientific data and information discussed in those sections, we
have concluded that dietary supplements containing ephedrine alkaloids
present an unreasonable risk of illness or injury under conditions of
use recommended or suggested in their labeling, or, if no conditions of
use are suggested or recommended in the labeling, under ordinary
conditions of use. As discussed in the responses to comments 34 and 35
of this document, even if we were to extrapolate from data
demonstrating effectiveness of certain ephedrine drug products when
considering the reasonably likely benefits of dietary supplements
containing ephedrine alkaloids, we conclude that the known and
reasonably likely risks would outweigh even such extrapolated benefits.
A summary of our rationale for reaching this conclusion is presented in
our analysis below.
a. Summary of risks for dietary supplements with ephedrine
alkaloids. People who use dietary supplements containing ephedrine
alkaloids are at increased risk for serious adverse events, including
heart attack, stroke, and death. Susceptible individuals (e.g., those
with coronary artery disease or heart failure), many of whom may not
know they have underlying illnesses, are at increased risk for adverse
events because these products can cause abnormal heart rhythms (pro-
arrhythmic effect), even when the product is ingested at recommended
doses over a short course (one or a few doses). Over longer periods of
use, the risk for adverse health effects to the general population,
including susceptible individuals, increases further due to a sustained
elevation in blood pressure. This is a characteristic effect of the
sympathomimetic class of pharmacological compounds. Moreover, the
results of Boozer, et al. (2002) demonstrate that weight loss achieved
with botanical ephedrine alkaloids does not produce the expected
decrease in blood pressure (Ref. 49). The risk of experiencing harmful
effects from elevated blood pressure increases the longer the blood
pressure remains high, and such adverse effects are likely to occur
sooner in individuals with hypertension, many of whom are unaware of
their illness.
b. Summary of known and reasonably likely benefits for dietary
supplements containing ephedrine alkaloids. As discussed in the
following paragraphs, we conclude, based on all available information
and data reviewed in this rulemaking, that these products do not
provide a meaningful health benefit. The best clinical evidence for a
benefit is for weight loss, but even there the evidence supports only a
modest short-term weight loss insufficient to positively affect
cardiovascular risk factors or health conditions associated with being
overweight or obese. Other possible benefits, such as enhanced athletic
performance, enhanced energy, or a feeling of alertness, lack
scientific support and/or they would provide only temporary benefits
that are trivial in comparison to the risks of serious long-
[[Page 6826]]
term or permanent consequences like heart attack, stroke, and death.
i. Weight loss. As discussed previously, the RAND report provides
the most comprehensive review of efficacy studies for ephedrine
alkaloid containing products. The RAND report found evidence that
supported an association between short-term use of ephedrine, ephedrine
plus caffeine, or dietary supplements that contain ephedrine alkaloids
with or without herbs containing caffeine, and a statistically
significant increase in short-term weight loss compared to placebo. The
RAND report concluded that products containing ephedrine alkaloids in
combination with caffeine resulted in a modest weight loss of
approximately 2 pounds per month more than placebo over a period of 4
to 6 months. RAND concluded that the use of ephedrine without caffeine
was associated with a statistically significant increase in weight loss
(1.3 pounds of weight loss per month) compared with that of placebo for
up to 4 months of use. RAND identified a single trial of 3 months
duration that assessed the effect of herbal ephedra versus placebo.
Those in the ephedra arm lost 1.8 pounds more per month than did those
in the placebo arm. We are unaware of any appropriate, well-designed
studies showing an effect of dietary supplements containing ephedrine
alkaloids on weight loss for more than 6 months. Such a long-term
effect would be necessary to translate into health outcome
improvements.
Even if there were adequate substantiation that dietary supplements
containing ephedrine alkaloids produce long-term, sustained weight loss
in the overweight or obese population, the long-term risks posed by
these products, particularly in obese patients who may already have
underlying illnesses that can be aggravated by these products (such as
hypertension), remain a serious concern. We believe that physician
supervision is necessary to mitigate the risks associated with the use
of sympathomimetic products in the long term for weight loss and the
treatment of obesity, or for any other long-term use. This is achieved
in part by monitoring patients who use these products and discontinuing
product use if the patient develops hypertension, experiences other
adverse health effects, or fails to achieve weight loss that would
justify continued exposure to the risks associated with use of the
product.
People might choose to use a dietary supplement containing
ephedrine alkaloids to lose weight for purposes other than to improve
health (e.g., to look slimmer or fit into an outfit for a special
occasion), and we do not dismiss this use as without value to the
individual. To achieve the result of modest weight loss, however, these
products must be used over a period of months. Individuals who use
these dietary supplements over a period of months for weight loss are
at risk for the adverse events that can occur with both short- and
long-term use of these products. These risks are greater than the
modest benefits described in the RAND report.
In the case of both short-term and long-term use, any benefits of
dietary supplements containing ephedrine alkaloids for weight loss are
outweighed by their risks. Therefore, we conclude that dietary
supplements containing ephedrine alkaloids labeled or used for weight
loss present an unreasonable risk.
ii. Enhancement of athletic performance. The effects of synthetic
ephedrine on athletic performance were assessed in seven studies that
were reviewed in the RAND report. Despite the widespread marketing of
products containing ephedrine alkaloids as performance-enhancers, the
RAND report found no studies involving botanical ephedrine alkaloids,
and very limited evidence involving synthetic ephedrine, to support the
claims. Furthermore, the RAND report concluded that, ``to show even a
short-term effect of ephedrine, combination with caffeine was
required.'' Therefore, there is no evidence to indicate that ephedrine
alone enhances athletic performance. People who use dietary supplements
containing ephedrine alkaloids for athletic performance are at risk for
the same serious adverse events as individuals who use these products
for other indications. As discussed previously in section V.C.2, the
available evidence regarding a possible benefit from these products for
enhancing athletic performance is further limited: the supporting
evidence all comes from studies in which synthetic ephedrine and
caffeine in combination were administered to healthy males, and the
modest effects shown were in the very short term only. Even if one
could disregard all the gaps in the scientific evidence and assume that
ephedra has the same effect on athletic performance as synthetic
ephedrine in combination with caffeine, we do not consider a modest,
temporary enhancement of certain aspects of athletic performance to be
a benefit sufficient to outweigh the risks of dietary supplements
containing ephedrine alkaloids. Therefore, we conclude that the use of
dietary supplements containing ephedrine alkaloids to enhance athletic
performance for any duration of use present an unreasonable risk.
iii. Eased breathing and other uses. We have long recognized the
legitimate short-term oral use of sympathomimetics, such as ephedrine,
in OTC bronchodilator drug products. These products are marketed for
those who have been diagnosed with asthma by a physician. The products
are GRASE when formulated and labeled in accordance with the
requirements of the final monograph for OTC bronchodilators (21 CFR
part 341). Mandatory warnings include advising the consumer not to use
the product unless diagnosed as having asthma by a doctor and not to
use the product if suffering from heart disease or high blood pressure.
We are aware that there are dietary supplements containing
ephedrine alkaloids that are marketed for uses other than weight loss
or athletic performance enhancement, such as ``eased breathing,''
``better breathing,'' ``feel better,'' ``feel more alert,''
``energized.'' By contrast to the monograph-compliant OTC
bronchodilators, and as discussed in section V.B.3 of this document, we
have seen no data that support any benefit relating to eased breathing
in healthy people from dietary supplements containing ephedrine
alkaloids. Moreover, as also discussed in that section, because healthy
people are able to breathe without difficulty, we do not believe there
is any respiratory benefit in the absence of a disease state, such as
asthma or a respiratory infection. At the same time, however, there are
data that establish the risks of these products. We note that claims to
treat or mitigate the effects of a disease subject a product to
regulation as a drug under the act.
With regard to other claims such as ``feel better,'' ``feel more
alert,'' and ``energized,'' effects of this nature may be of modest
benefit to the individual (if they occur), but they are temporary and
do not improve health. Therefore, such effects would not be sufficient
to outweigh the risks of dietary supplements containing ephedrine
alkaloids.
There are also dietary supplements containing ephedrine alkaloids
that do not make any specific claims or otherwise suggest or recommend
conditions of use in their labeling. The use of such products presents
the same risks and can lead to the same serious adverse events as
discussed previously for weight loss and athletic performance, even if
the product is
[[Page 6827]]
taken under ordinary conditions of use (i.e., not abused).
A dietary supplement labeled for a very temporary, episodic use
might not present an unreasonable risk if there were adequate evidence
that the use resulted in a health benefit sufficient to outweigh the
health risks. Any new indication would still be subject to our post-
market risk evaluation as to whether it could be legally marketed.
Conclusions regarding the benefit of dietary supplements containing
ephedrine alkaloids for nondisease claims cannot be drawn solely from
studies using synthetic ephedrine for specific diseases. Although we
could require labeling for dietary supplements containing ephedrine
alkaloids to limit the duration of use, among other things, currently
there are no data that demonstrate that dietary supplements containing
ephedrine alkaloids provide a benefit to a particular population when
used temporarily or episodically (in contrast to OTC ephedrine and
pseudoephedrine products for disease uses).
3. Conclusion
Multiple studies demonstrate that dietary supplements containing
ephedrine alkaloids, like other sympathomimetics, raise blood pressure
and increase heart rate. These products expose users to several risks,
including the consequences of a sustained increase in blood pressure
(e.g. serious illnesses or injuries that include stroke and heart
attack that can result in death) and increased morbidity and mortality
from worsened heart failure and pro-arrhythmic effects. Although the
pro-arrhythmic effects of these products typically occur only in
susceptible individuals, the long-term risks from elevated blood
pressure can occur even in nonsusceptible, healthy individuals. These
risks are neither outweighed by any known or reasonably likely benefits
when dietary supplements containing ephedrine alkaloids are used under
conditions suggested or recommended in their labeling, such as for
weight loss, athletic performance, increased energy or alertness, or
eased breathing. Nor do the benefits outweigh the risks under ordinary
conditions of use, in the absence of suggested or recommended
conditions of use in product labeling. As discussed above in section
V.C of this document, the best scientific evidence of benefit is for
modest short-term weight loss; however, such benefit would be
insufficient to bring about an improvement in health that would
outweigh the concomitant health risks. The other possible benefits
discussed in section V.C if this document, have less scientific
support. Even assuming that these possible benefits in fact occur, such
temporary benefits are also insufficient to outweigh health risks that
can lead to serious long-term or permanent consequences like heart
attack, stroke, and death. On the other hand, we have determined that
there are benefits from the use of OTC and prescription drug products
containing ephedrine alkaloids in certain populations for certain
disease indications that outweigh their risks.
As with other sympathomimetics, the risks posed by dietary
supplements containing ephedrine alkaloids for continuous, long-term
use cannot be adequately mitigated without physician supervision.
Temporary, episodic use can be justified only if a known or reasonably
likely benefit outweighs the known and reasonably likely risks. Similar
to OTC single ingredient ephedrine products, dietary supplements
containing ephedrine alkaloids could theoretically be marketed without
physician supervision for a very temporary, episodic use if there were
adequate evidence that the use resulted in a benefit sufficient to
outweigh the risks of these products. However, we are currently unaware
of any such use, and our experience with ephedrine and pseudoephedrine
OTC drug products suggests that such benefits will be demonstrable only
for disease uses. Therefore, we conclude that dietary supplements
containing ephedrine alkaloids present an unreasonable risk of illness
or injury under conditions of use recommended or suggested in labeling
or under ordinary conditions of use, if the labeling does not suggest
or recommend conditions of use.
VI. Why We Conclude that Other Restrictions Would Not Adequately
Protect Consumers from the Risks Presented by Dietary Supplements
Containing Ephedrine Alkaloids
We considered several regulatory alternatives to this final rule.
As discussed in section I.C of this document, we issued a proposed rule
in 1997 that would have placed various restrictions on dietary
supplements containing ephedrine alkaloids. Most of the proposed
restrictions were withdrawn in 2000; only the proposed prohibition on
combining ephedrine alkaloids with other stimulant ingredients and the
proposed warning statement (as modified in FDA's March 2003 notice)
remain. As discussed in the following paragraphs, we have reached the
conclusion that those restrictions are inadequate to protect public
health. In addition, we considered other regulatory alternatives
presented in the comments received.
A. Warning Statement Alone
We first proposed a warning statement in the June 1997 proposal. At
that time, we tentatively concluded that a warning statement was
necessary to disclose material facts about the consequences of using
these products, and that it would help to reduce the risk of an adverse
event after use of dietary supplements containing ephedrine alkaloids
(62 FR 30670 at 30703). In our March 2003 notice, we reopened the
comment period to seek, among other things, comments on a revised
warning statement that we were considering at that time for dietary
supplements containing ephedrine alkaloids.
We received a number of comments on the proposed labeling
requirements in the June 1997 proposal and on the revised warning
statement in our March 2003 notice. Because we have decided to proceed
under the adulteration provision in section 402(f)(1)(A) of the act
rather than to require labeling for dietary supplements containing
ephedrine alkaloids, these comments are moot to the extent that they
discuss the substance or format of the warning statement. Nevertheless,
comments regarding the sufficiency of a warning are relevant to this
rulemaking.
(Comment 64) Many comments supported the use of a warning label as
an effective way to protect public health, although they differed on
the specific language and format of the warning. Many comments urged us
to mandate strict warning labels to inform users about the potential
health risks that have been reported to be associated with the use of
dietary supplements containing ephedrine alkaloids. One comment stated
that product labeling does influence user behavior and strongly urged
us to take action in the form of issuing a mandatory warning label for
all dietary supplements containing ephedrine alkaloids. Several
comments stated that there was a significant decrease in the number of
AERs in certain States after their respective departments of health
mandated label restrictions and strong cautionary statements. A number
of comments stated that the warning labels voluntarily adopted and
already used by industry are sufficient to protect the public from any
risks. A number of comments proposed different labels to be adopted by
the entire industry.
In contrast, many comments maintained that warnings are
insufficient and recommended a ban of these products. Several comments
pointed out that serious adverse events
[[Page 6828]]
continue to occur even though most dietary supplements containing
ephedrine alkaloids already carry warning statements, such as those
recommended by industry trade groups. For several years, warning labels
have also been mandated in several states by law or regulation. Many
comments noted that, in at least 90 percent of the adverse event
reports submitted to us, consumers reported taking dietary supplements
containing ephedrine alkaloids as directed on the label.
A few other comments asserted that warning labels are ineffective
because serious adverse events have occurred after the initial use or
after very short-term use of dietary supplements containing ephedrine
alkaloids. As pointed out in the June 1997 proposal, about 40 percent
of the 600 AERs reported between 1993 and 1996 occurred with the first
use or within 1 week of first use, providing little or no warning to
consumers of risk. Many of the adverse events occurred in individuals
who had no apparent risk factors, or who were unaware that they were at
risk.
Several comments stated that warning labels on ephedrine alkaloid-
containing dietary supplements are not sufficient to protect the public
health because many people are not aware they have medical conditions
or individual sensitivities that put them at greater risk for
experiencing serious adverse effects.
(Response) We agree that warning statements cannot adequately
protect consumers from the risks associated with dietary supplements
containing ephedrine alkaloids. Even if all consumers read the warnings
and the warnings thoroughly describe the risks, many using these
products may not be aware they have medical conditions or individual
sensitivities that put them at greater risk for experiencing serious
adverse effects. A full discussion of the risks to sensitive
populations appears previously in the response to comment 22 of this
document.
Warning labels may be beneficial when people are able themselves to
identify the risk factors they have, or when evaluation by a physician
prior to use can identify whether they have the risk factors and
further supervision by a physician is not necessary for safe use of the
product. The purpose of the physician's evaluation is to identify
individuals with underlying conditions (such as heart failure or
coronary artery disease) that place them at risk for serious adverse
events (such as death) due to pro-arrhythmic effects. Such warnings can
reduce but not eliminate the risks from episodic use of dietary
supplements containing ephedrine alkaloids because not all susceptible
individuals can be identified by a physician's evaluation. For example,
people can have asymptomatic coronary artery disease or early heart
failure that a physician would not recognize without performing tests
that would usually be reserved for patients with signs or symptoms of a
disease. We are not aware of a nondisease claim for which the known and
reasonably likely benefits of dietary supplements containing ephedrine
alkaloids would outweigh their known and reasonably likely risks when
used episodically.
A warning to consult your physician before use provides even less
risk mitigation for dietary supplements containing ephedrine alkaloids
that are used continuously because even healthy people would experience
a rise in blood pressure and, therefore, be at increased risk for heart
attack, stroke, and death. At a minimum, continued physician
supervision would be a necessary risk management tool. Thus, even if
consumers were to heed warning labels and consult their physician, the
known and reasonably likely risks of dietary supplements containing
ephedrine alkaloids when used episodically or continuously would still
outweigh their known and reasonably likely benefits.
The conclusion that warning statements are not adequate to protect
public health is consistent with the fact that, since 1993, we have
received more than 18,000 AERs (including both adverse events reported
directly to FDA and the Metabolife call records). The majority of the
products associated with these AERs contained directions for use and
warning statements. The warning statements varied from general
precautions, suggesting that consumers check with a health care
professional before beginning any diet or exercise program, to more
specific warning statements, including cautions that consumers not use
the product if they have certain diseases or health conditions or are
using certain drugs, and to stop the use of the product if they develop
certain symptoms. Despite these warning statements in the product
labeling of dietary supplements containing ephedrine alkaloids, we
continue to receive reports of serious adverse events.
(Comment 65) Several comments compared sensitivity to ephedrine
alkaloids in dietary supplements to sensitivity to food allergens. One
comment expressed the opinion that the number of individuals sensitive
to ephedrine alkaloids in dietary supplements is either less than, or
comparable with, those individuals who suffer from food allergies. One
comment argued that warning statements are effective for people who
know they are sensitive to a substance, such as peanuts. The comment
suggested that if warning labels are considered sufficient in this
context, they should also be considered sufficient in the context of
dietary supplements containing ephedrine alkaloids. Another comment
stated that, with respect to those individuals who are unaware that
they may have one of the conditions that is contraindicated on the
label, some misuse due to ignorance is unavoidable and occurs no matter
what regulations are put in place.
(Response) We do not agree that individuals sensitive to ephedrine
alkaloids in dietary supplements are comparable to individuals who
suffer from food allergies. In the case of food allergies, individuals
learn that they are allergic to certain foods (e.g., shellfish and
nuts) and, because we require that the presence of the food ingredients
be declared on the food label (see 21 CFR 101.4), these individuals can
then avoid the problem ingredient by reading the food label. The
physical manifestations of the allergic reaction are usually readily
recognized by the consumer. In the case of the ephedrine alkaloids, as
discussed previously in the responses to comments 22 and 27 of this
document, many individuals are not aware that they are sensitive to
sympathomimetic agents, such as the ephedrine alkaloids, and may not
recognize early signs of risk, such as elevated blood pressure or the
adverse cardiovascular and nervous system effects related to the use of
ephedrine alkaloids. In most instances, patients with nascent food
allergies experience classic allergy symptoms, such as tingling lips,
scratchy throat, wheezing, and shortness of breath, that alert them to
the development of a particular food allergy, whereas with ephedrine
alkaloids, severe, life-threatening reactions, may occur at any time,
even with the first exposure. Therefore, an ingredient declaration or a
warning label statement cannot assist these consumers in adequately
reducing their risk of adverse events.
B. Multiple Restrictions
(Comment 66) Addressing the inadequacy of a warning statement
alone, many comments supported multiple restrictions (e.g., dosage
limits, ingredient combination restrictions, duration of use
restrictions, label claim restrictions, good manufacturing practices
(GMP) requirements, and warning label statements) to reduce the risk of
adverse events. One comment pointed out that the frequency, severity,
and the broad cross section of the
[[Page 6829]]
population for which there are documented adverse events support at
least this level of regulation. Some comments contended that we should
establish more stringent regulations. Several of these comments
recommended that we ban the use of ephedrine alkaloids in dietary
supplements because of the serious health hazards associated with their
use and the potential for abuse and misuse of these products.
(Response) We do not agree that the restrictions recommended in
these comments will eliminate the risks imposed by dietary supplements
containing ephedrine alkaloids. As discussed in the response to comment
26 of this document, we are not aware of any evidence that establishes
a safe dose of ephedrine alkaloids in dietary supplements. Therefore,
dose limitations cannot change the unfavorable risk-benefit ratio of
these products. Similarly, a requirement for a label statement
recommending that consumers limit the duration of product use will not
provide adequate protection because adverse events sometimes occur
after the first use or in the first few days. We also do not agree that
dietary ingredient restrictions, such as limiting the presence of other
stimulant ingredients, will eliminate the unreasonable risk associated
with the use of dietary supplements containing ephedrine alkaloids. As
explained in section V.B.1 of this document, ephedrine alkaloids given
alone can be expected to cause significant increases in blood pressure,
although the presence of other stimulants combined with ephedrine
alkaloids may increase the risks associated with use of these products.
Finally, while GMP requirements may ensure consistent quality across
dietary supplement products containing ephedrine alkaloids, the risks
attributed to ephedrine alkaloids are due to their inherent
pharmacological and physiological effects rather than the quality of
their manufacture, although poor manufacturing could lead to additional
risks, such as from the introduction of toxic impurities into the
product.
C. Self-Regulation
(Comment 67) Other comments objected to the June 1997 proposal,
arguing that no FDA action is necessary. Several of these comments
recommended that we take no action but instead continue to monitor
adverse events. A number of comments stated that the dietary supplement
industry will self-regulate. These comments argued that several dietary
supplement trade associations have reacted responsibly to the public
concerns about the AERs by setting standards for the use of ephedrine
alkaloids in dietary supplements for their members (Ref. 101).
(Response) We disagree with the comments that state that no FDA
action is necessary because the industry will self-regulate. It is
incumbent upon us to respond to the serious adverse events associated
with the use of dietary supplements containing ephedrine alkaloids and
other information about the risks of these products. We have been aware
for several years that a number of trade associations have policies
concerning the formulation and labeling of dietary supplements
containing ephedrine alkaloids. These voluntary industry standards are
insufficient to alter the risk-benefit ratio for these products.
Despite the fact that these industry standards are in place, we
continue to receive reports of clinically significant adverse events
following the consumption of dietary supplements containing ephedrine
alkaloids. Some of these adverse events may be due to noncompliance
with those voluntary standards; however, for the reasons stated in the
response to comment 39 of this document, these types of standards, even
if adhered to, would be insufficient to protect consumers from the
risks posed by dietary supplements containing ephedrine alkaloids.
D. More Education
(Comment 68) One comment recommended that we provide better
education to the public on the public health concerns about dietary
supplements containing ephedrine alkaloids.
(Response) We do not agree that educating consumers about the
public health concerns related to the use of dietary supplements
containing ephedrine alkaloids is an appropriate substitute for this
regulation. Although we have been active in, and support, consumer
education activities about these supplements, consumer education will
not adequately address the risks they present. For example, many
individuals who are sensitive to sympathomimetic agents, such as the
ephedrine alkaloids, and are therefore at an increased risk of
experiencing an adverse event, are not aware that they are at risk.
Therefore, consumer education would not be expected to greatly reduce
the risk of adverse events.
E. Nonbinding Guidance
(Comment 69) Several other comments recommended the issuance of
nonbinding guidance providing notice to marketers as to which dietary
supplements containing ephedrine alkaloids would most likely be the
subject of FDA enforcement. One comment argued that a guidance document
would conform to our good guidance practices (21 CFR 10.115) and
provide guidance to the dietary supplement industry as to a level of
ephedrine alkaloids that can be used in their products with some
confidence that such products will not be subject to regulatory action.
In arguing for a guidance document and against a regulation, the
comment said that a Federal regulation is only appropriate and
necessary to protect the public health when safe use of a product
cannot be ensured absent such a regulation; the comment maintained that
we have not made this showing. One comment stated that the major
dietary supplement industry trade associations could exhort industry
compliance to guidelines issued by us or by the trade associations.
(Response) We disagree that nonbinding guidance would be an
effective substitute for this rulemaking. As stated previously in this
document, several industry trade associations have established policies
concerning the formulation and labeling of dietary supplements
containing ephedrine alkaloids. These policies are non-binding and
manufacturers and distributors are under no obligation to comply.
Moreover, as discussed previously in the responses to comments 39 and
67 of this document, guidance on labeling or product formulation, even
if adhered to, would be insufficient to protect consumers from the
risks posed by dietary supplements containing ephedrine alkaloids.
F. Targeted Enforcement Actions
(Comment 70) Other comments stated that enforcement actions against
products containing extremely high levels of ephedrine alkaloids should
be sufficient to address the problem.
(Response) We find that individual enforcement actions against
products containing high levels of ephedrine alkaloids are inadequate
to protect the public health. Data from the scientific literature and
AERs indicate that clinically significant adverse effects are not
limited to the use of products containing high levels of ephedrine
alkaloids (Refs. 109 and 134). Therefore, enforcement actions against
products containing only high levels of ephedrine alkaloids would not
be expected to eliminate the unreasonable risk presented by these
products. We also
[[Page 6830]]
note that rulemaking is a more efficient regulatory mechanism than
individual enforcement actions in cases where hundreds of different
products on the market contain the same ingredient that presents a risk
to the public health, as is the case here. Without a regulation, we
would be required to establish our case de novo with witnesses in every
enforcement proceeding. Multiple proceedings would require multiple
witnesses and extensive discovery, and would be extremely time-
consuming and burdensome for both the courts and us. However, we point
out that a regulation is not necessary to find that a dietary
ingredient or a dietary supplement presents an unreasonable risk.
VII. Miscellaneous Issues
A. Freedom of Choice/FDA Bias
(Comment 71) Many comments stated that our attempt to regulate
dietary supplements containing ephedrine alkaloids would erode personal
freedom and the public's freedom of choice, values that the comments
maintained were established through the passage of DSHEA. Several
comments stated that DSHEA gives the public a right to access
affordable, natural, and effective dietary supplements. A number of
comments alleged that we issued the June 1997 proposal because we are
biased against dietary supplements. One industry comment accused us of
selectively including information in the docket. Several of these
comments alleged that our purpose for issuing the June 1997 proposal
was to protect the business interests of the pharmaceutical industry.
Several comments explained that, if access to dietary supplements for
weight loss is restricted, consumers will have little choice but to use
prescription drugs. Many comments from consumers stated that use of
prescription drugs for weight loss is both more costly and associated
with more adverse effects than use of products containing natural
herbs. Many of these comments stated that dietary supplements
containing ephedrine alkaloids from natural sources are safe and have
no side effects. Conversely, several comments stated that the
perception that supplements are natural and, therefore, safe and
acceptable alternatives to prescribed medications is erroneous and that
there are serious concerns about the safety and efficacy of these
products.
(Response) We deny these allegations of bias against the marketing
and use of dietary supplements and any allegations of protecting or
favoring the pharmaceutical industry. We support access to dietary
supplements that are safe, properly labeled, and in compliance with
Federal law. However, we are also obligated under DSHEA to protect the
public against dietary supplements that are unsafe or otherwise
adulterated. Contrary to one comment's assertion, we did not base our
decision on selectively chosen information; instead, we considered all
information that was submitted to the relevant dockets, including more
than 48,000 comments and hundreds of studies submitted by the dietary
supplement industry, trade associations, academics, health
professionals, scientists, public health groups, and consumer groups.
Given the scientific information about the pharmacology of ephedrine
alkaloids, clinical studies examining their effects, and AERs, we found
that there are serious and well-documented public health risks
associated with the use of dietary supplements containing ephedrine
alkaloids. Therefore, our obligation under DSHEA is to take action to
address such risks, particularly in light of the products' lack of
health benefits.
Additionally, comments concerning the pharmaceutical industry's
business interests and possible consumer use of prescription drugs are
not relevant to our determination as to whether dietary supplements
containing ephedrine alkaloids are adulterated under section
402(f)(1)(A) of the act. Section 402(f)(1)(A) of the act focuses
exclusively on whether the dietary supplement or dietary ingredient
presents a significant or unreasonable risk; consequently, arguments
pertaining to other industries or other products have no bearing on
whether dietary supplements containing ephedrine alkaloids are
adulterated under the act.
B. Conduct of the Advisory Committee Meetings
(Comment 72) Several comments stated that we conducted the October
1995 meeting of the Working Group and the 1996 meeting of the Food
Advisory Committee (the Committees) in a manner that improperly
influenced their deliberations and recommendations. These comments
argued that we instructed the Committee members not to consider certain
data (e.g., data concerning the use of ephedrine-containing OTC drug
products for the treatment of asthma); misrepresented certain data
(e.g., data concerning the AERs and data from clinical trials on the
use of ephedrine in the treatment of obesity); failed to present data
that industry believed to be relevant to the evaluation (e.g., number
of units of products sold during the period of time the AERs were
received, data regarding whether a cause and effect relationship
existed between dietary supplements containing ephedrine alkaloids and
the adverse events reported to us); instructed the Committee to
evaluate safety using an interpretation of ``significant harm'' (i.e.,
either a large number of adverse events or a serious adverse event in
one individual) that is not specified in DSHEA; and improperly asked
the Committee to recommend action to reduce the risks associated with
the use of these products.
Other comments argued that the procedures we followed at the
Committees' meetings were unfair. The comments cited several reasons,
including the following: FDA materials were not made available to
dietary supplement industry groups and other interested persons prior
to the meetings; we were given unlimited time to ``influence'' the
Committee, and the time others were given to present comments was
limited; and interested persons were not allowed to question FDA
officials. For these reasons, several of these comments stated that we
must reconvene the Committee.
(Response) We disagree with the comments. The comments concerning
the data and information we presented or did not present during the
meetings are without merit because the essence of these comments is
that they disagreed with our interpretation of the data or preliminary
conclusions. Presenting our interpretation of the data and our
preliminary conclusions is entirely appropriate and does not constitute
undue influence over the Committees (Ref. 137). Interested persons,
including the dietary supplement industry, were provided with ample
opportunity to express their views and present data they believed
relevant to the evaluation during the public hearing portions of the
meetings or in written comments to the Committees. To the extent that
specific comments on the data, our interpretation of the data, and our
preliminary conclusions are relevant to this rulemaking, they are
addressed in other sections of this document.
Regarding the conduct of the Committees' meetings, those meetings
were conducted in accordance with the Federal Advisory Committee Act (5
U.S.C. App. 2), FDA's implementing regulations (21 CFR part 14), and
FDA guidance entitled ``Policy and Guidance Handbook for FDA Advisory
Committees'' (1994) (Ref. 137). We also note that the procedures
followed during these meetings were no different from the procedures
used in conducting the numerous advisory committee
[[Page 6831]]
meetings we have held on a variety of other issues.
We convened the Committees as a means to acquire independent
scientific and technical advice on the public health concerns
surrounding the use of dietary supplements containing ephedrine
alkaloids and on specific ways to address these public health concerns.
During the meetings, we implemented several safeguards to ensure the
Committees' independence and fairness to all interested parties.
First, it was made entirely clear during the meetings that the
Committees' members were invited to express a view different than ours,
so that our tentative conclusions could be revised, if necessary.
During these meetings, we presented a critical and fair evaluation and
interpretation of the available data. We also expressed our tentative
conclusions and our concern for the public health. Again, it is
entirely appropriate for us to state our views and interpretation of
the data. Furthermore, individual members of the Committees took
advantage of the many opportunities during the meetings to discuss
their views and to question FDA officials about the available data, our
interpretation of the data, and our tentative position.
Second, the Committees included consumer and industry
representatives, including two representatives from associations
representing the dietary supplement industry. The consumer and industry
representatives represented the views of consumers and industry
throughout the meeting and made recommendations to us. All FDA-prepared
materials to be considered by the Committees were sent to all members
of the Committees, including the dietary supplement industry
representatives, prior to the meeting.
Third, the Committees' meetings provided a forum for public
discussion. Interested persons, including the dietary supplement
industry, were provided with ample opportunity to express their views
and present data they believed relevant to the evaluation during the
public hearing portions of the meetings or in written comments to the
Committees. During the Committees' meetings, we provided over 2 hours
of public hearing time, which is twice the time required by our
regulations (21 CFR 14.29(a)).
Thus, contrary to the comments' assertions, we provided ample
opportunity for public participation in the meetings. The public
hearings were conducted prior to the Committees' deliberations so that
comments made by interested parties could be considered by the
Committees in making their recommendations.
VIII. Analysis of Impacts
A. Benefit-Cost Analysis
1. Introduction
We have examined the economic implications of this final rule as
required by Executive Order 12866. Executive Order 12866 directs
agencies to assess all costs and benefits of available regulatory
alternatives and, when regulation is necessary, to select regulatory
approaches that maximize net benefits (including potential economic,
environmental, public health and safety, and other advantages;
distributive impacts; and equity). Executive Order 12866 classifies a
regulatory action as a significant regulatory action if it meets any
one of a number of specified conditions, including having an annual
effect on the economy of $100 million or more, adversely affecting a
sector of the economy in a material way, adversely affecting
competition, or adversely affecting jobs. Executive Order 12866 also
classifies a regulatory action as significant if it raises novel legal
or policy issues. We have determined that this final rule is a
significant regulatory action as defined by Executive Order 12866
because the benefits of the rule could exceed $100 million per year and
because the rule raises novel legal and policy issues.
The Small Business Regulatory Enforcement Fairness Act of 1996
(Public Law 104-121) defines a major rule for the purpose of
congressional review as having caused or being likely to cause one or
more of the following: An annual effect on the economy of $100 million;
a major increase in costs or prices; significant adverse effects on
competition, employment, productivity, or innovation; or significant
adverse effects on the ability of U.S.-based enterprises to compete
with foreign-based enterprises in domestic or export markets. In
accordance with the Small Business Regulatory Enforcement Fairness Act,
the OMB has determined that this final rule will be a major rule for
the purpose of congressional review because the benefits may exceed
$100 million annually.
Title II of the Unfunded Mandates Reform Act of 1995 (Public Law
104-4) requires cost-benefit and other analyses before any rule making
if the rule would include a ``Federal mandate that may result in the
expenditure by State, local, and tribal governments, in the aggregate,
or by the private sector, of $100,000,000 or more (adjusted annually
for inflation) in any 1 year.'' The current inflation-adjusted
statutory threshold is $113 million per year. We have estimated that
the total cost of this final rule would be no more than $90 million per
year. Therefore, we have determined that this final rule does not
constitute a significant rule under the Unfunded Mandates Reform Act.
2. Regulatory Options
We discussed the following seven regulatory options in the benefit-
cost analysis of the June 1997 proposal: (1) Take no action; (2) take
no new regulatory action, but generate additional information on which
to base a future regulatory action; (3) take the actions in the June
1997 proposal; (4) take the proposed action, but with a higher potency
limit; (5) remove dietary supplements that contain ephedrine alkaloids
from the market; (6) take the proposed action, but do not require a
warning statement; and 7) require a warning statement only (62 FR 30678
at 30705). We later withdrew all elements of the proposed action except
the warning statement and prohibition of dietary supplements that
combine ephedrine alkaloids with other stimulants (65 FR 17474). In
2003, we issued a March 2003 notice seeking comment on, among other
things, a revised warning statement consisting of a short warning on
the PDP and a more detailed warning elsewhere in the product labeling.
We did not perform any economic evaluation of the revised warning
statement at that time. We received additional comments on the revised
warning statement. In addition, the comments on the June 1997 proposal
suggested some additional options. Considering the options from these
sources, we address the following options in this analysis: (1) Take no
new regulatory action; (2) remove dietary supplements containing
ephedrine alkaloids from the market; (3) require the proposed warning
statement, as revised in 2003; (4) require a warning statement, but
modify it or require it only on certain products; and (5) generate
additional information or take some action other than removing dietary
supplements containing ephedrine alkaloids from the market or requiring
warning statements. Executive Order 12866 requires us to analyze
regulatory options but recognizes that there are practical limits to
the number of options that we can analyze. The options listed above
encompass all or most of the significant suggestions raised in the
comments.
3. Summary of Conclusions
We have decided to remove dietary supplements containing ephedrine
[[Page 6832]]
alkaloids from the market, identified as option 2 in the previous
paragraph. We estimate net effects would be between -$47 million and
$125 million per year from this option, if consumer behavior does not
already incorporate the health risks posed by these products, and
between -$90 million and -$7 million per year, if consumer behavior
already incorporates the health risks. A detailed discussion of all the
options is provided in the following paragraphs.
4. Option One--Take No New Regulatory Action
We use this option as the baseline for determining the costs and
benefits of the other options. Therefore, we do not associate costs or
benefits with this option. Instead, we discuss the costs and benefits
of taking no action in the context of the costs and benefits of the
other options. As we discuss more fully under the other options, the
expected number of adverse events from these products will probably
decline, over time, even if we take no regulatory action, for two
reasons. First, many firms are moving away from the use of ephedrine
alkaloids because of media coverage of adverse events associated with
these products, the high cost of liability insurance, and the potential
for legal actions by consumers. Second, some State and local
governments have either banned the sale of these products or placed
various requirements or restrictions on sales of these products.
5. Option Two--Remove Dietary Supplements Containing Ephedrine
Alkaloids from the Market
a. Benefits of removing dietary supplements containing ephedrine
alkaloids from the market. The benefits of this final rule stem from
the reduction of risks brought about by removing dietary supplements
containing ephedrine alkaloids from the market. We measure the risk
reduction, for the purpose of estimating benefits, as the number of
illnesses and deaths averted. Because OMB's guidance to Executive Order
12866 calls for quantification of risk reduction, we place special
emphasis in this part of the document on those AERs that lend
themselves more readily to quantification.
As shown earlier in this document, dietary supplements containing
ephedrine alkaloids would be expected to increase heart rate/rhythm and
blood pressure. Increasing blood pressure in any population is
associated with increased probabilities of heart attack, stroke, and
death, which are the serious adverse events most commonly associated
with ephedrine alkaloids. The known pharmacological effects of
ephedrine alkaloids lead us to conclude that removing these dietary
supplements from the market will reduce the incidence of these adverse
events. Estimating the likely reduction, however, presents challenges.
One method used in similar situations is to combine data on exposure
with a dose-response function to generate estimates of adverse events
prevented as exposure declines. We cannot use that method here,
however, because we do not have sufficient data on exposure to
ephedrine alkaloids from dietary supplements, and we do not know the
associated dose-response function. Therefore, the best available
approach, and the method we apply here, is to use AERs to generate
estimates of the number of adverse events associated with dietary
supplements containing ephedrine alkaloids.
It is important to note that the AERs are not the principal
scientific basis for the regulatory action we selected. Instead, the
AERs are consistent with the known pharmacological and physiological
effects of ephedrine alkaloids, as well as the results of clinical
studies and, therefore, support our finding of unreasonable risk. As we
explain in more detail later in this document, we use a high barrier
before admitting an AER as evidence of adverse events associated with
ephedrine alkaloids. We also use conservative methods to infer the
total number of adverse events from the reports.
i. Use of AERs in estimating benefits and baseline number of AERs.
In the analysis of the June 1997 proposal, we based our estimate of the
impact of removing dietary supplements containing ephedrine alkaloids
from the market on the estimated annual number of adverse events caused
by dietary supplements containing ephedrine alkaloids (62 FR 30678 at
30705). We based the latter estimate on the average annual number of
AERs that we received between January 1993 and June 1996, that we
suspected of having been caused by these supplements, which we
characterized as the ``baseline number of AERs.'' We then adjusted this
number of AERs by a series of assumptions designed to reflect various
sources of uncertainty over whether these supplements actually caused
those AERs and the uncertainty over the relationship between the AERs
and the actual number of adverse events associated with the use of
dietary supplements containing ephedrine alkaloids (including both
reported and unreported adverse events).
(Comment 73) A number of comments on the June 1997 proposal
addressed the issue of the baseline number of AERs. Some comments
objected to adjusting the number of AERs with assumptions designed to
reflect uncertainty over the relevance of those AERs. One comment said
we should have used only those AERs that we were certain had been
caused by dietary supplements containing ephedrine alkaloids. Other
comments simply pointed out that some adverse events might not have
been caused by dietary supplements containing ephedrine alkaloids.
Some comments suggested that our estimate of the number of adverse
events based on the number of AERs was inconsistent with the results of
various studies on the safety of ephedrine alkaloids, herbal ephedra,
or particular dietary supplements containing ephedrine alkaloids. One
comment noted that the estimated number of adverse events, particularly
the estimated number of deaths, was inconsistent with data collected by
the Drug Abuse Warning Network program, which is administered by the
Office of Applied Studies in the Substance Abuse and Mental Health
Services Administration of HHS. Some comments made similar points with
respect to the inconsistency of our estimated adverse events with the
lower number of adverse events reported for ephedrine alkaloid-
containing products marketed in foreign countries.
Several comments suggested that our estimate of the number of
adverse events was inconsistent with their personal experience. Many
comments included information on the amount of the product sold or
estimates of the number of people who consumed the relevant product.
A number of comments discussed adverse events that purportedly
would have occurred without consumption of dietary supplements
containing ephedrine alkaloids. These comments argued that we probably
generated a large number of irrelevant AERs by asking consumers to
report ubiquitous symptoms as adverse events that may have been caused
by these products.
Some comments criticized the report that RAND prepared for HHS on
the safety and effectiveness of dietary supplements containing
ephedrine alkaloids because of its attention to AERs (Ref. 21). One
comment argued that RAND's approach was inappropriate because GAO had
previously criticized our use of the AERs in the analysis of the June
1997 proposal. Other comments supported RAND's attention to AERs. One
comment argued that RAND did not
[[Page 6833]]
adequately account for preexisting health conditions when classifying
events in the AERs as ``sentinel'' or ``possibly sentinel'' events.
Other comments criticized RAND's review of the clinical studies
involving ephedrine alkaloids. One comment argued that the method RAND
used to determine which clinical studies to review was biased. Some
comments argued that the results of RAND's review of the AERs were
inconsistent with the results of RAND's review of the clinical studies
because the clinical studies enrolled enough patients to uncover the
types of adverse events that appear in the AERs, if ephedrine alkaloids
could cause those types of events. Other comments suggested that
sources other than the RAND report provide better assessments of the
risks associated with dietary supplements containing ephedrine
alkaloids.
Other comments addressed one or more of the other articles that we
listed in the March 2003 reopening of the comment period. Many comments
criticized one or more of those studies on various bases. Other
comments supported one or more of those studies. One comment argued
that we presented a biased list of studies because we ignored four
other articles that were published at about the same time as the
articles that we listed. Some comments noted that RAND said that
clinical trials that they reviewed had enrolled enough patients to
detect serious adverse events at rates of 1 per 1,000 or higher.
Finally, some comments addressed trends that might affect the
estimated number of adverse events. Some comments addressed the
apparent upward trend in the rate at which we received AERs as of 1997,
which we mentioned in the proposed rule. Some comments suggested that
the perceived upward trend in AERs at that time may have been caused by
changes in publicity or in the methods we used to collect adverse
events, rather than by changes in the number of adverse events. One
comment noted that many firms had stopped making dietary supplements
containing ephedrine alkaloids.
(Response) Although uncertainty remains over the exact number of
adverse events that are caused by dietary supplements containing
ephedrine alkaloids, we disagree that, when estimating the number of
adverse events, we should use only those AERs that we or others have
proven to have been caused by dietary supplements containing ephedrine
alkaloids. The comments appear to suggest that we should adopt a
standard of absolute proof that a dietary supplement caused an
individual adverse event. However, establishing absolute proof for
individual cases is very difficult for dietary supplements or most
other substances other than direct poisons. It is appropriate in the
case of dietary supplements containing ephedrine alkaloids to estimate
the number of adverse events prevented by this rule based upon
scientifically established pharmacological effects of ephedrine
alkaloids and the clinical and epidemiological evidence. The RAND
report used the term ``sentinel events'' to describe adverse events
that involved ephedrine alkaloids and for which RAND could exclude
alternative explanations for the event with ``reasonable certainty.''
If other possible causes could not be excluded, then the report
classified the cases as possible sentinel events. This level of
certainty is unusually high in the context of identifying a public
health risk.
We also disagree that we should use only clinical studies when
estimating the number of adverse events. In addition, we disagree with
the comments that stated that because clinical studies find baseline
rates for stroke and major cardiac events in excess of 1 per 1,000, the
existing clinical evidence is sufficient to detect adverse events
associated with ephedrine alkaloids. The clinical studies reviewed by
RAND were not large enough to distinguish between effects of ephedrine
alkaloids and the ordinary variance around the baseline. We, therefore,
do not agree that existing clinical studies are sufficiently large to
detect additional adverse events associated with ephedra or ephedrine.
As discussed in section V.B of this document, the scientific evidence
identifies the risks presented by dietary supplements containing
ephedrine alkaloids. For example, a 6-month clinical study examining
the efficacy and safety of ephedrine alkaloids for the treatment of
obesity found a statistically significant association between treatment
with ephedrine alkaloids and higher blood pressure compared to placebo
(Ref. 49). Higher blood pressure tends to increase the likelihood of
cardiovascular disease. Thus, the clinical evidence establishes a
potential mechanism leading from the use of dietary supplements
containing ephedrine alkaloids to the occurrence of serious adverse
effects.
We link the findings from this clinical study and the well-known
pharmacological effects of ephedrine alkaloids to adverse events to
establish the likelihood that at least some adverse events reported to
be associated with the use of dietary supplements containing ephedrine
alkaloids were in fact caused by these products. Although not as
rigorous as an epidemiological case control study, this evidence is the
best available to estimate the benefits of this rule.
We agree that we should reduce the uncertainty associated with the
AERs as much as possible and accurately express any remaining
uncertainty. Therefore, we have replaced the baseline number of AERs
that we used in the analysis of the proposed rule with the number of
AERs that RAND identified as sentinel and possibly sentinel events
involving herbal ephedra. RAND identified 20 sentinel events over a
period of approximately 9 years from 1992 to 2001, which corresponds to
an average of about 2 such events per year. RAND also identified 42
possible sentinel events in this time period, which corresponds to an
average of about five such events per year.
We have based our revised estimate on the RAND report because it is
the most comprehensive review of the information that is currently
available on the safety and efficacy of dietary supplements containing
ephedrine alkaloids. However, we acknowledge that considerable
uncertainty continues to exist with respect to the number of adverse
events that have been caused by ephedrine alkaloids. We have attempted
to reflect the continuing uncertainty by updating the assumptions we
used in the analysis of the June 1997 proposal, as we discuss in the
following paragraphs.
We did not attempt to forecast trends in the number of adverse
events in the analysis of the June 1997 proposal, and we have not done
so in this analysis. Forecasting trends in the number of adverse events
would be difficult, and any such forecasts would be associated with
large uncertainty ranges. Although we recognize that some firms may
have recently discontinued the use of ephedrine alkaloids in some or
all of their products, we have insufficient information to revise the
results of the RAND report on that basis.
Assumptions used in analysis of the final rule
First assumption
Ninety percent to 100 percent of the sentinel events and 50 percent
to 100 percent of the possible sentinel events identified in the RAND
report were caused by dietary supplements that we suspect contained
ephedrine alkaloids.
(Comment 74) A number of comments addressed the first assumption.
One comment suggested that we should have set the lower bound of the
first assumption to zero because it was possible that none of the AERs
had been
[[Page 6834]]
caused by dietary supplements containing ephedrine alkaloids. Some
comments provided their own estimates of the number of AERs that had
been caused by those supplements.
(Response) We have revised our estimate of the baseline number of
AERs using the number of sentinel and possible sentinel cases
identified in the RAND report in order to address the concerns that
these comments raised about causation and the presence of ephedrine
alkaloids with respect to some of the AERs that we used as a basis for
our benefit estimates in the analysis of the June 1997 proposed rule.
Although RAND stressed that it could not conclude that these events
were definitely caused by ephedrine alkaloids and declined to make any
probabilistic statements about causality, the definitions that it used
for sentinel and possible sentinel events suggest that those AERs have
a relatively high probability of having been caused by ephedrine
alkaloids. Therefore, we have revised the assumption concerning the
proportion of the AERs that were caused by dietary supplements from 80
percent to a range of 90 percent to 100 percent for sentinel events and
50 percent to 100 percent for possible sentinel events.
Second assumption
One hundred percent of the sentinel and possible sentinel events
that were caused by dietary supplements that we suspect contained
ephedrine alkaloids involved dietary supplements that did, in fact,
contain ephedrine alkaloids.
(Comment 75) Other comments addressed the second assumption. One
comment reported that an industry review of the 920 AERs in the docket
found that more than 123, or 13 percent, involved products for which
there was no indication that the product contained ephedrine alkaloids.
One comment was from a firm that claimed it had informed us during FAC
meetings that nearly 25 percent of the AERs that involved their
products involved products that did not, in fact, contain ephedrine
alkaloids.
(Response) One of the criteria that RAND used to identify sentinel
and possible sentinel events was documentation that the person that
suffered the adverse event had consumed a dietary supplement containing
ephedra within 24 hours prior to the adverse event. The assumption in
the proposed rule that 80 percent of the AERs involved products that
contained ephedrine alkaloids applied to the set of AERs used in that
analysis. RAND has documented that all of the sentinel and possible
sentinel events it reviewed involved products containing ephedrine
alkaloids. Documentation of the presence of ephedrine alkaloids varied
from case to case, and included blood tests of the person who suffered
the adverse event, chemical analysis of capsules, and labeling of the
products consumed. RAND did not consider self-reports alone to be
sufficient documentation for sentinel and possible sentinel events.
Because we use the RAND study as the basis for the analysis of this
final rule, the 80 percent assumption is no longer relevant. In the
analysis of this final rule, we assume that 100 percent of the AERs
involved products that contained ephedrine alkaloids.
Third assumption
AERs represented 10 percent of the actual number of adverse events.
(Comment 76) Some comments argued that our assumption of a 10
percent reporting rate was too low. Some comments argued that people
are more likely to overreport than underreport adverse events involving
dietary supplements containing ephedrine alkaloids for various reasons,
including FDA's public statements and media coverage of this issue. One
comment argued that people are more likely to overreport than
underreport serious adverse events such as heart attack, stroke,
seizure, psychotic events, and death, because people tend to consider
any temporal connection equivalent to a causal connection. However,
this comment suggested that people probably underreport minor adverse
events. Some comments noted that the AERs that we discussed in the June
1997 proposal appeared to arrive in discrete groups as though in
response to inciting events, such as FDA press releases. One comment
noted that, of the 22 AERs in the docket that involved their products,
we received two-thirds of those AERs within 1 week of our April 1996
press release, and we received the other one-third over a much longer
period of 30 months. Some comments suggested that the 10 percent
assumption might be appropriate for passive reporting systems, but
argued that the reporting system that we used to generate the AERs was
not passive because both the Texas Department of Health and FDA took
various steps to solicit AERs. Two comments discussed estimates of
reporting rates for a passive adverse event reporting system in
Britain. One comment estimated the reporting rate for serious adverse
events at 50 percent. Another comment estimated the same rate at 10
percent. Both comments estimated that the system had a much smaller
reporting rate of 2 percent to 4 percent for nonserious adverse events.
Some comments noted that we assumed a 50 percent reporting rate in our
report on Eosinophilia-Myalgia Syndrome, which was an outbreak level
event (Ref. 138). These comments noted that this report referred to
adverse events related to a dietary supplement, L-tryptophan, which had
also received significant media publicity. These comments argued that
it was, therefore, a reasonable model to use for the ephedrine alkaloid
situation. Some comments suggested that we revise our reporting rate
assumption from 10 percent to a range of 10 percent to 50 percent.
Other comments argued that our assumption of a 10 percent reporting
rate was too high. Some comments argued that people are more likely to
underreport than overreport adverse events involving dietary
supplements containing ephedrine alkaloids for various reasons, such as
not wanting to acknowledge using the product. One comment noted that a
2001 report from the Office of the Inspector General of HHS concluded
that current surveillance systems for identifying adverse reactions
from dietary supplements probably detect less than 1 percent of adverse
reactions (Ref. 20). However, another comment claimed that most
researchers consider a reporting rate of less than 1 percent to reflect
a worst-case scenario. One comment noted that the report that suggested
a reporting rate of less than 1 percent did not differentiate between
serious and nonserious adverse events. This comment argued that the
reporting rate for serious adverse events is probably higher than for
nonserious adverse events.
(Response) In order to express the continuing uncertainty over the
reporting rate, we have calculated benefits based on reporting rates of
10 percent, 50 percent, and 100 percent of sentinel and possible
sentinel events. Although the reporting rate could be lower than 10
percent, the severity of the adverse events under consideration and the
level of media coverage suggest that the reporting rate may be 10
percent or higher. The assumed 100 percent reporting rate generates a
lower bound number of adverse events. We selected 50 percent as an
intermediate number. We used a 10 percent reporting rate in our summary
statements to simplify the presentation of the results and because 10
percent reporting appears to be a reasonable point estimate, taking
into account the seriousness and media coverage of these adverse events
and the estimated reporting rates of 1 percent or lower for adverse
events involving drugs (Refs. 32 and 139). The 10 percent reporting
rate applies to serious events only, and incorporates the fact that a
[[Page 6835]]
report of a serious adverse event had to fulfill the RAND criteria in
order to be included as a sentinel or possible sentinel event. We did
not consider nonsentinel events in the analysis, as explained in the
following paragraphs.
ii. Valuing reductions in adverse events.
(Comment 77) Some comments addressed the values that we placed on
eliminating various types of adverse events in the analysis of the
proposed rule. One comment objected to the value of $5 million that we
placed on one fewer fatality per year across the affected population,
which is sometimes called the value of a statistical life. This comment
described this value as the value of an average life and argued that
this figure is unrealistic because the average person does not have $5
million.
(Response) In its guidelines on performing economic analysis of
federal regulations under Executive Order 12866, OMB noted that the
term ``statistical life'' can lead to some confusion. It pointed out
that this term refers to the sum of risk reductions expected in a
population, as expressed in the following example: If the annual risk
of death is reduced by one in a million for each of two million people,
that represents two ``statistical lives'' saved per year (two million x
one in one million = two). If the annual risk of death is reduced by
one in 10 million for each of 20 million people, that also represents
two statistical lives saved (Ref. 140). Similarly, the estimated value
of a statistical life (VSL) is based on the willingness to pay for
relatively small reductions in the risk of premature death for many
people summed across a population. The individual risk management
decisions on which we base estimates of the VSL must reflect the budget
constraints of those individuals making those decisions. However, the
resulting VSL need not reflect the budget constraints of the average
person. We have revised the VSL in this analysis to a range of $5
million to $6.5 million to reflect the latest estimates of this figure
(68 FR 41433 through 41506, July 11, 2003).
In addition, we have revised our method of estimating the values of
avoiding the other health endpoints. For nonfatal myocardial infarction
(MI), we used the same procedure that we used in our analysis of the
proposed rule on trans fatty acids (64 FR 62772, November 17, 1999).
That method was based on estimating the sum of the medical costs, the
cost of functional disability, and the cost of pain and suffering. This
method assumes that someone suffering a nonfatal MI will have
functional disability or pain and suffering or both in every year after
the year following the MI. We estimated the loss per year to be 0.2
quality adjusted life years (QALYs) every year of life following the
MI. We did not include any reduction in life expectancy due to the MI.
For this rule, we based the years of disability or pain and suffering
on the ages of those suffering nonfatal myocardial infarction in the
RAND report (Ref. 141). RAND reported summary information on age by
type of adverse event using three age categories (13 to 30, 31 to 50,
and 51 to 70). We took the midpoints of the three age categories and
constructed a weighted average based on the proportion of people
suffering that adverse event in those categories. We then compared that
age to an average life expectancy in the United States in 2001 of 77.2
years to determine the years of disability or pain and suffering or
both (Ref. 142).
We used a similar procedure to estimate new values for strokes. To
estimate combined functional disability and pain and suffering we used
a 0.2 quality adjusted life year (QALY) loss per year after a stroke
(Ref. 143). We used the same QALY losses for ``other cardiovascular''
events that we used for nonfatal MI. We were unable to find information
on chronic QALY losses for acute cases of ``other neurological,''
``seizure,'' or ``psychiatric'' adverse events. For medical costs, we
used 2001 National Statistics from HCUPnet (Ref. 144). We provide
summary information on these values in table 1 of this document.
(Comment 78) Some comments that discussed the background rates of
expected but unexplained adverse events argued that many AERs involved
people with underlying health conditions and that dietary supplements
containing ephedrine alkaloids might have simply precipitated adverse
events that would have occurred within a short time anyway.
(Response) As we indicated previously in this document, we have
revised our estimate of the number of relevant AERs to reflect the RAND
report. The definition that RAND used for sentinel events involved
investigating alternative explanations and excluding them with
reasonable certainty. However, the definition that RAND used for
possible sentinel events included cases where another condition by
itself could have caused the adverse event, but for which the known
pharmacology of ephedrine made it possible that ephedra or ephedrine
may have helped precipitate the event. We have reflected the
uncertainty over causality in the first of the three assumptions that
we discussed above. We assume that dietary supplements containing
ephedrine alkaloids caused 90 percent to 100 percent of sentinel events
and 50 percent to 100 percent of possible sentinel events.
iii. Serious versus minor adverse events.
(Comment 79) Some comments suggested that some AERs that we used in
the analysis of the June 1997 proposal involved events that we should
not have classified as adverse events. These comments argued that these
events involved expected side effects of ephedrine alkaloids that are
both minor and transient.
(Response) We discussed adverse events that we classified as ``less
serious'' in the analysis of the proposed rule (62 FR 30678 at 30708).
However, we indicated that the value of eliminating those adverse
events contributed very little to total estimated benefits. RAND did
not include these types of more minor adverse events in its sentinel
and possible sentinel event cases. Although it did find evidence that
products that contained both ephedrine alkaloids and caffeine increased
the risk of certain minor adverse events, it noted that it was unable
to distinguish the effects of the ephedrine alkaloids and the caffeine.
Based on these considerations, we have not attempted to address adverse
events beyond those that RAND identified as sentinel and possible
sentinel events.
iv. Risks of substitutes and weight regain.
(Comment 80) Some comments argued that consumers would face similar
or greater health risks if they switched from dietary supplements
containing ephedrine alkaloids to alternative weight loss solutions,
such as prescription weight-loss drugs, other dietary supplements, or
weight loss surgery.
Some comments discussed what would happen if consumers stopped
using dietary supplements containing ephedrine alkaloids and did not
switch to equally effective alternative weight loss methods. Some
comments discussed the extent and rising trend of obesity in the United
States. Some comments noted that obesity increases the risk for heart
attack, stroke, diabetes, and cancer. However, other comments argued
that any countervailing health costs that would result if people
stopped using dietary supplements containing ephedrine alkaloids to
lose weight would be small or nonexistent. Some comments suggested
there were no clear health benefits from the amount of weight loss that
the RAND report attributed to dietary supplements
[[Page 6836]]
containing ephedrine alkaloids. Other comments disagreed and argued
that there were clear health benefits from the amount of weight loss
that the RAND report attributed to dietary supplements containing
ephedrine alkaloids. One comment argued that, although people often
regain weight that they lose during a diet program, people who have
participated in diet programs nevertheless generally maintain lower
weights than those who have not.
(Response) Subtracting the value of countervailing health effects
posed by substitute products and activities from the value of the
health benefits from removing dietary supplements containing ephedrine
alkaloids from the market to obtain the net health benefits is
consistent with our approach for estimating benefits. (For purposes of
this economic impact analysis, ``health benefits'' refers to an
improvement to health and is not synonymous to the ``benefits'' that we
mention in our risk-benefit analysis for purposes of determining that
these products present an unreasonable risk of illness or injury;
``health benefits'' are a type of ``benefit'' we consider when making
an unreasonable risk determination.) Our full conceptual model of
benefits is as follows: (net change in risk from the reduction in
intake of ephedrine alkaloids x value per unit change in risk) + (net
change in risk from substitute products and activity x value per unit
change in risk) + (net change in risk from weight gain x value per unit
change in risk) + (any net change in risk from the small impact on
wealth from the cost of substitute products or activity x value per
unit change in risk).
However, we do not have sufficient information to estimate all
elements of this model. In the analysis of the June 1997 proposal , we
noted one article that found that a product a firm had reformulated to
remove ephedrine alkaloids had lost approximately 33 percent of its
previous sales (Ref. 145). Since that time, a media report discussed
another reformulated product that had greater sales than the original
product (Ref. 146). Therefore, we estimate that from two-thirds to all
of the consumers of these supplements would probably switch to other
dietary supplements that firms market for the same purposes as dietary
supplements containing ephedrine alkaloids. This implies that between
one-third and none of the consumers of these products would switch to
entirely different types of weight loss or performance enhancing
substitutes.
Some manufacturers have already reformulated dietary supplements so
that products that had contained ephedrine alkaloids now contain
alternative ingredients. Some of these reformulated products contain
Citrus aurantium L., which is a source of synephrine, and caffeine,
sometimes in the form of green tea extract. Synephrine is a
sympathomimetic agent, and these agents are a class of compounds that
also includes ephedrine alkaloids. A number of other potential herbal
sources of sympathomimetics probably exist. These ingredients may pose
risks that are similar to those of ephedra. If consumers switched to
substitute products containing these ingredients, similar health risks
might be expected as those with products containing ephedrine
alkaloids. Some other ingredients that have been reported in
reformulated products include cocoa beans, yerba mate, cinnamon twig,
and galangal.
The estimated none to one-third of the consumers of dietary
supplements containing ephedrine alkaloids who would switch to products
other than other dietary supplements might switch to alternatives that
carry either health risks or benefits. Some of those who consumed these
supplements for weight loss may seek medical care to obtain
prescription weight loss medications or for weight loss surgery.
However, only some of these consumers would qualify for these medical
treatments. These treatments would carry health risks that might be
equal to, or greater than, the risks of ephedrine alkaloids. Only the
risks that remain after accounting for the management of risk under
physician supervision would be relevant in this context. In addition,
these treatments may be more expensive than dietary supplements. The
resulting relatively small reductions in the overall wealth of those
who switch to more expensive alternatives could also generate small
countervailing health risks because they have less disposable income to
spend on other risk-reducing activities.
Other consumers interested in weight loss may switch to meal
replacements or other diet products rather than seek medical treatment.
Other consumers might choose to do nothing and simply forego the weight
loss they may have obtained with ephedra products. This foregone weight
loss could, in theory, generate health costs. The lack of health
benefits from the weight loss associated with the use of these
products, however, implies that these health costs, if any, would be
negligible. Finally, some consumers might choose to reduce their
caloric intake or increase their caloric output through additional
exercise. These consumers would obtain additional health benefits
beyond eliminating the risk of adverse events associated with dietary
supplements containing ephedrine alkaloids. Those who consume
supplements containing ephedrine alkaloids to enhance their athletic
performance and who do not switch to other dietary supplements marketed
for that purpose might switch to other stimulants, including black
market products containing ephedrine alkaloids or methamphetamines.
These products would pose health risks equal to or greater than those
of currently marketed dietary supplements containing ephedrine
alkaloids.
We have insufficient information to quantify the effects of
switching to alternative weight loss or athletic performance enhancing
products or activities, or to quantify the health costs associated with
the absence of weight loss that might be achieved using dietary
supplements containing ephedrine alkaloids.
v. Risks of certain dietary supplements containing ephedrine
alkaloids.
(Comment 81) A number of comments suggested that certain dietary
supplements containing ephedrine alkaloids do not pose any health
risks. These comments addressed this point in the context of exempting
certain products from the proposed warning statement. However, these
comments are also relevant to the issue of exempting certain products
from a regulation removing dietary supplements containing ephedrine
alkaloids from the market. Therefore, we discuss these comments under
this option.
Several comments argued that we should not treat ephedrine
alkaloids in Chinese herbal formulas that are used in Chinese medicine
treatment protocols the same as dietary supplement products containing
ephedrine alkaloids that consumers use to lose weight or enhance
athletic performance. One comment suggested that warning statements are
unnecessary for herbal products that firms distribute to ``healthcare
professionals,'' including members of the American Herbalists Guild.
Some comments suggested that we should set different regulatory
requirements for different products or product types because risks vary
by product or product type.
(Response) The RAND report found little scientific agreement on the
dose-response relationship for ephedrine alkaloids (Refs. 21 and 22).
Therefore, we are unable to estimate the impact of exempting products
from this rule based on the level of ephedrine alkaloids that they
contain. As we discussed earlier in the preamble, we have determined
that botanical sources of ephedrine alkaloids
[[Page 6837]]
in traditional Asian herbal therapies are not covered by this rule. We
do not have sufficient information to estimate the impact of exempting
products based on the other considerations suggested in the comments,
including type of product, label warnings, or directions for use.
b. Revised benefit estimates. Based on the preceding discussion, we
have revised our estimate of the benefits of removing dietary
supplements containing ephedrine alkaloids from the market. The social
benefits of removing dietary supplements containing ephedrine alkaloids
from the market consist of the increase in consumer utility that would
be generated by any net health benefits resulting from removing dietary
supplements containing ephedrine alkaloids from the market. The
following table 1 of this document provides an estimate of the number
of the various types of serious adverse events that we might eliminate
by removing dietary supplements containing ephedrine alkaloids from the
market, along with an estimate of the utility loss prevented by that
reduction. As we discussed previously, benefits could be much lower and
potentially zero if the health risks posed by substitute weight loss or
sports performance products, such as other dietary supplements
containing sources of sympathomimetics, were comparable to the health
risks posed by ephedrine alkaloids.
We convert the number of deaths prevented into a monetary estimate
by multiplying by the number of deaths by the VSL. We convert the
number of nonfatal events prevented into a monetary estimate by
multiplying the number of nonfatal events by the value of the
appropriate change in quality QALYs. Acute events that do not have
clear chronic effects will generate only minimal losses in terms of
QALYs. We calculated the total benefits for each class of adverse
events as: (Number of deaths prevented) x ($ per fatal case); and
(number of nonfatal cases prevented) x (($ per QALY x QALY loss) +
medical costs per case)). The benefits for the first year would be
slightly different from the benefits in every subsequent year because
the effective date is 60 days after the publication date of the final
rule. By convention, we calculate benefits starting from the
publication date of the final rule. Therefore, the benefits in the
first year will be 5/6 (or 83 percent) of the benefits of every
subsequent year. To simplify the discussion, we use the benefits for
every year after the first year in all summary discussions.
Table 1.--Annual Number of Sentinel and Possible Sentinel Events
Prevented Under Option Two (Removing Dietary Supplements Containing
Ephedrine Alkaloids from the Market), with QALY and Medical Cost per
Case
------------------------------------------------------------------------
Annual Number QALY Loss Per Medical Costs per
Type Prevented Case Case
------------------------------------------------------------------------
Death 0.7 to 1.2 NA (used VSL) $25,742
MI (heart 0.6 to 1.0 0.29 $30,586
attack)
CVA (stroke) 1.5 to 2.1 0.2 $20,898
Other 0.1 to 0.2 0.29 $30,586
Cardiovascular
(e.g.
Cardiomyopathy,
Ventricular
Tachycardia)
Other 0.1 minimal $13,212
Neurological
(e.g. Transient
Ischemic
Attack)
Seizure 0.5 to 0.9 minimal $11,812
Psychiatric 0.9 to 1.3 minimal $6,927
------------------------------------------------------------------------
Note. All dollar values in this document represent 2003 prices.
Table 2.--Annual Benefits of Option Two (Removing Dietary Supplement
Containing Ephedrine Alkaloids from the Market) Based on Alternative
Assumptions of Reporting Rates and Values of Preventing Adverse Events,
Rounded to $ Millions
------------------------------------------------------------------------
Value of Avoiding Adverse Event Reporting Rate ($ in millions)
Fatal Cases and ----------------------------------------------------
QALY Losses 10 percent 50 percent 100 percent
------------------------------------------------------------------------
$ per fatal case = $43 to $73 $9 to $15 $4 to $7
$5 million $ per
QALY = $100,000
$ per fatal case = $53 to $91 $11 to $18 $5 to $9
$6.5 million $ per
QALY = $100,000
$ per fatal case = $56 to $93 $11 to $19 $6 to $9
$5 million $ per
QALY = $300,000
$ per fatal case = $66 to $112 $13 to $22 $7 to $11
$6.5 million $ per
QALY = $300,000
$ per fatal case = $80 to $132 $16 to $26 $8 to $13
$6.5 million $ per
QALY = $500,000
------------------------------------------------------------------------
c. Costs of removing dietary supplements containing ephedrine
alkaloids from the market. In the analysis of the proposed rule, we
identified the costs that would be generated by removing dietary
supplements containing ephedrine alkaloids from the market as the one-
time cost of reformulating and relabeling products that currently
contain ephedrine alkaloids, plus the utility loss for those consumers
who would need to switch from their preferred option (consuming these
products) to their next most preferred option (consuming an alternative
product or taking some other type of action) (62 FR 30678 at 30709). In
that analysis we did not estimate utility losses for consumers. A
number of comments stressed this cost but did not provide estimates of
it. Nevertheless, we have revised the analysis by attempting to
quantify this cost.
Theoretically, we could measure the utility loss for consumers by
looking at the difference between their willingness to pay for products
containing ephedrine alkaloids and their willingness to pay for
alternative supplements or other substitute products or activities.
However, we do not have sufficient information to implement this
approach, and may never have a direct measure of the utility loss in
this market. Instead, we attempt to measure indirectly the utility loss
for consumers of these products. We assume that the premium that these
consumers are willing to pay to consume dietary supplements containing
ephedrine alkaloids rather than whatever they perceive to be the next
closest alternative is between 1
[[Page 6838]]
percent and 10 percent of the sales price of the dietary supplements
containing ephedrine alkaloids. This range is based on the fact that
some premium must exist if consumers prefer these products to
alternatives. We selected 1 percent as a lower bound because we did not
find any large price differences between products containing ephedrine
alkaloids and those that did not contain ephedrine alkaloids. Of
course, it is possible that current consumers place a much higher
premium on products containing ephedrine alkaloids than consumers who
have already switched to alternatives. To allow for that possibility,
we selected 10 percent (a substantial premium) as the upper bound of
the range. Current market prices do not provide sufficient information
for a more precise estimate. This estimate of the utility loss assumes
that consumers do not incorporate the expected utility losses from
potential adverse events in their willingness to pay for dietary
supplements containing ephedrine alkaloids. If consumers already
incorporate this information in their purchasing decisions, then it
would be inappropriate to compare the value of the health benefits to
the estimated utility losses for consumers using willingness to pay
because the willingness to pay would already account for any adverse
health effects. In that case, the estimated utility loss from the
removal of these products from the market would represent the full net
loss of utility.
A recent article estimated that the sales of ``herbal products''
containing ephedra accounted for between 4.3 percent and 13.5 percent
of the sales for all herbal products (Ref. 135). The article did not
define ``herbal products,'' but it noted that their use of the phrase
``herbal products'' included products that a natural products
information company had classified as ``vitamins/supplements'' and
``grocery'' items rather than as ``herbal products'' (Ref. 147).
Therefore, these estimates may have included products other than
dietary supplements. Another source argued that the estimates presented
in the article that we discussed previously in this paragraph did not
include all relevant products. The source claimed that more
comprehensive data from the Nutrition Business Journal showed that the
sales of products containing herbal ephedra accounted for 33 percent of
the total sales of all herbal products and 7.5 percent of the total
sales of dietary supplements (Ref. 148). Both of these articles
apparently dealt only with products that contained herbal ephedra.
Ephedrine alkaloids are also contained in a number of different plants,
including Sida cordifolia L., and Pinellia ternata (Thunb.) Makino.
Therefore, these articles may have underestimated the number of
products that contained ephedrine alkaloids. These articles did not
present actual sales figures for herbal products, dietary supplements,
or products containing ephedra. However, the Nutritional Business
Journal estimated that the sales of all dietary supplements and all
herbal dietary supplements in 2002 were $18 billion and $4.3 billion,
respectively (Ref. 149). If one assumes that ``herbal dietary
supplements'' corresponds to ``herbal products,'' then total sales of
dietary supplements containing ephedrine alkaloids would be $185
million to $1,419 million.
In an effort to reduce this range, we estimated the sales of these
products based on a recent survey that showed that 2 million consumers
used these products at some point during a given week (Ref. 150). We
assumed that consumers who used these products at some point during a
given week probably used the products every day during that week,
because most of the labels we have examined say that the product should
be taken daily, or several times per day. We also assumed that the
particular week under study was comparable to any other week.
Therefore, we assumed that 2 million consumers use these supplements
per day. We then multiplied this number of consumers by the average
daily cost of these supplements, which we estimated from a sample of 30
dietary supplements containing ephedrine-alkaloids that we found on the
Internet. Based on the recommended intake levels appearing on the
labels of these products, the corresponding estimated total sales per
year is $559 million to $806 million. The costs in the first year after
publication of the rule would be slightly different from the cost in
every subsequent year because the effective date is 60 days after the
publication date of the final rule. Therefore, the utility losses in
the first year will be 5/6 (or 83 percent) of the losses of every
subsequent year. To simplify the discussion, we use the benefits for
every year after the first year in all summary discussions.
Earlier, we assumed that the consumer utility loss from switching
from an ephedra-based product to the next closest substitute would be
from 1 percent to 10 percent of the sales price at the current level of
consumption. Under this assumption and our estimate of total sales, the
consumer utility loss associated with removing dietary supplements
containing ephedrine alkaloids from the market would be $6 million to
$81 million per year. The loss of consumer utility would probably
decline over time as consumers find more substitute products and as
producers develop new, more acceptable substitute products. Eventually,
consumer substitutions and product development could drive this cost to
zero. We have insufficient information to estimate the rate at which
this cost would decline over time.
In the analysis of the June 1997 proposal, we estimated relabeling
costs of $3 million to $60 million and product reformulation costs of
$0 million to $25 million, for a total cost for these two activities of
$3 million to $85 million (62 FR 30678 at 30709). We did not receive
any comments on these estimates. We have, however, revised the analysis
to incorporate a new model for estimating reformulation costs that we
developed after publication of the proposed rule (Ref. 151). According
to that model, reformulation costs with a 12-month reformulation period
would be $7 million to $78 million. In deriving that figure, we assume
that reformulating dietary supplements would not be as complicated as
reformulating most other types of food and cosmetics. In particular, we
assume that reformulating dietary supplements would include the
following cost generating activities: Idea generation, product
research, analytic testing, packaging development, plant trials,
startup, and lost inventory. We assume that reformulating dietary
supplements would not include the following types of cost generating
activities: Process development, coordinating activities, consumer
tests, shelf life studies, any type of safety studies, and market
tests. If all of these other steps were involved, then estimated
reformulation costs for a 12-month reformulation period would be $22
million to $142 million. We assume that 6 months is the most likely
time period for reformulation if dietary supplements containing
ephedrine alkaloids are removed from the market. Although the effective
date of this rule is 60 days after the publication date, we do not
expect that many firms will try to condense the reformulation process
into a 60-day period. Some firms may have already done some of the
preliminary work for reformulation. Other firms might need to withdraw
their product from the market in the period between the effective date
and the date at which they complete their reformulation. FDA's
reformulation cost model does not address costs for a reformulation
time of 6 months, so we
[[Page 6839]]
extrapolated the costs based on the proportionate change in cost that
would result from halving the reformulation time from 24 months to 12
months. Under that extrapolation, we estimate that reformulation costs
for a 6-month reformulation period would be $10 million to $100
million. We annualize these estimated costs over 20 years at an
interest rate of 3 percent to convert these one-time costs to a yearly
cost of $1 million to $7 million. Annualizing these costs over 20 years
at an interest rate of 7 percent gives an annual cost of $1 million to
$9 million.
We summarize the annual costs of this option in table 3 of this
document. We compare the benefits and costs of this option in table 4
of this document. To obtain the higher bound estimate of net benefits,
we start with the higher bound estimate of benefits and subtract the
lower bound estimates of costs. To obtain the lower bound estimate of
net benefits, we start with the lower bound estimate of costs and
subtract the higher bound estimate of costs. If consumer behavior
already incorporates health risks, then utility costs would already be
net of health benefits. In that case, the net impact of this rule is
simply the total costs.
Table 3.--Annual Costs of Option Two (Removing Dietary Supplement Containing Ephedrine Alkaloids from the
Market) Rounded to $ Millions
----------------------------------------------------------------------------------------------------------------
Type of Cost Cost (rounded to $ millions)
----------------------------------------------------------------------------------------------------------------
Utility Losses for $6 to $81
Consumers
Product Reformulation $1 to $9
----------------------------------------------------------------------------------------------------------------
Table 4.--Annual Social Benefits and Costs of Option Two (Removing
Dietary Supplement Containing Ephedrine Alkaloids from the Market)
Rounded to $ Millions
------------------------------------------------------------------------
Benefit or Cost (rounded to $
Type of Benefit or Cost millions)
------------------------------------------------------------------------
Health Benefits (for 10 percent $43 to $132
reporting rate)
Cost of Utility Losses for $6 to $81
Consumers
Cost of Product Reformulation $1 to $9
Net Effect (if consumer behavior -$47 to $125
does not already incorporate
health risks)
Net Effect (if consumer behavior -$90 to -$7
already incorporates health risks)
------------------------------------------------------------------------
d. Distributional issues and impact on industry. In the analysis of
the June 1997 proposal, we estimated that removing dietary supplements
containing ephedrine alkaloids from the market would reduce the sales
of dietary supplements containing ephedrine alkaloids by between $200
million and $230 million per year (62 FR 30678 at 30710). We discussed
reduced sales because, in that analysis, we characterized a
reformulated product as the same product as before reformulation for
purposes of describing the impact of the proposed action (although the
reformulated products would obviously not be the same as the products
they replaced). We did not receive comments that would require us to
change those estimates. However, we have revised the analysis to
reflect the fact that the effect on accounting profit is a more
appropriate way to conceptualize the potential distributional impact
than reduced sales. We can use the same information that we used to
estimate consumer utility losses to consider the likely effect on the
profits of firms that currently produce dietary supplements containing
ephedrine alkaloids. In 2001, the average accounting profit for all
Fortune 500 companies was about 5 percent of revenue, and some
pharmaceutical firms had profit rates as high as 19 percent of revenue
(Ref. 150). Profit rates for firms in the dietary supplement industry
are probably toward the low end of this scale because of the low
barriers to entry for this industry. Therefore, we assume that the
profit rate for dietary supplement manufacturers is about 5 percent of
total sales. As we discussed previously, press accounts suggest that
manufacturers that have reformulated their dietary supplements to
eliminate ephedrine alkaloids have experienced declines in sales
ranging from about one-third to no decline in sales. We previously
estimated total sales to be $559 million to $806 million. Therefore, we
estimate that sales may decrease by $0 to $269 million per year.
Assuming that the profit rate is 5 percent of sales, removing dietary
supplements containing ephedrine alkaloids from the market would
generate accounting profit losses of $0 to $13 million per year. We
classify this impact as a transfer and not a social cost because
removing dietary supplements containing ephedrine alkaloids from the
market would increase the profits of firms that produce and distribute
substitute products. If these other firms also have an average profit
rate of 5 percent of sales, then the profit gained by these companies
would also equal $0 to $13 million per year.
In addition to causing a potential reduction in profits for firms
currently producing dietary supplements containing ephedrine alkaloids,
removing dietary supplements containing ephedrine alkaloids from the
market might also generate some countervailing transfers through the
elimination of insurance costs and lawsuits associated with products
containing ephedrine alkaloids. Eliminating legal fees and court costs
would also generate social benefits. Of course, if reformulated
products were eventually found to pose health risks comparable to those
found for ephedra-based products, then these effects (i.e., the
elimination of insurance and legal costs) would eventually decrease to
zero. A recent press report found that ephedra manufacturers or
distributors have settled 33 cases since 1994 and that an additional 42
cases were pending (Ref. 152). This represents 75 cases over 9 years,
or about 8 cases per year. Recent awards for cases that have gone to
court have ranged from $2.5 million to $13 million (Refs. 152 and 153).
The figures reported in the media for cases that were settled out of
court were considerably lower. One such case was settled out of court
for $25,000 (Ref. 152). If removing dietary supplements containing
ephedrine alkaloids from the market eliminated 8 cases per year, then
it would decrease transfer payments from firms to consumers by between
$0.2 million per year, if all cases were settled out of court, and $104
million per year, if all cases were lost in court at the high end of
the range of legal penalties.
One company noted in 2002 that its product-liability insurance
increased by $2.1 million from 2001 to 2002 (Ref. 146). If all 30
manufacturers saw this increase in insurance premiums, then the total
increase in insurance premiums would be $60 million. Some of the
independent distributors might also face higher insurance rates, but we
have insufficient information to estimate those costs. Insurance rates
[[Page 6840]]
would not necessarily increase at this same rate in the future, and
they could decrease. Therefore, we will assume that this adjustment in
insurance rates reflects a one-time adjustment in the perceived
liability risks associated with these products. If these higher
premiums were unnecessary for reformulated products, then removing
dietary supplements containing ephedrine alkaloids from the market
would generate a one-time reduction in private costs of $60 million.
However, if reformulated products were eventually shown to pose risks
comparable to those for ephedra-based products, then insurance rates
might increase to a comparable level for these products.
The uncertainty ranges associated with the potential transfers of
accounting profits make it impossible to estimate the impact of
removing dietary supplements containing ephedrine alkaloids from the
market on the firms that currently produce and distribute dietary
supplements containing ephedrine alkaloids. Firms that are unable or
unwilling to produce or sell substitute products would suffer losses,
and firms that are able and willing to produce or sell substitutes
might not suffer decreases in profits. Indeed, media reports suggest
that many firms have already voluntarily withdrawn their ephedra-based
products and replaced them with reformulated products to avoid the high
legal and insurance costs associated with dietary supplements
containing ephedrine alkaloids (Ref. 146).
6. Option Three--Require the 2003 Proposed Warning Statement
a. Benefits of requiring the 2003 proposed warning statement
comparison to removing dietary supplements.
i. Containing ephedrine alkaloids from the market. In the analysis
of the June 1997 proposal, we noted that estimating the benefit of
limiting our regulatory action to requiring the 1997 proposed warning
statement involved a potentially controversial value judgment about how
one evaluates risks that consumers voluntarily accept in the presence
of adequate warning statements (62 FR 30678 at 30711). Our analysis of
a mandatory warning statement is further complicated by the fact that
the labels of most dietary supplements containing ephedrine alkaloids
already bear warning statements.
(Comment 82) One perspective that we discussed in the analysis of
the June 1997 proposal was that adverse events that occur despite the
presence of adequate warning statements are not social costs but are
instead private costs that reflect informed decisions about the private
benefits and costs of using these products. A number of comments agreed
with this perspective. One comment argued that consumers have a
responsibility to read and follow warnings and instructions for use on
products that they consume. Some comments suggested that we should
expect consumers to read and follow warning statements, and we should
not hold manufacturers liable if consumers fail to do so. One comment
argued that we have adopted that viewpoint in other cases involving
products that can produce severe adverse effects. Some comments from
consumers argued that we should take no regulatory action other than
requiring a warning statement because that approach would allow
consumers to decide whether or not to assume the risks associated with
these products. One comment pointed out that a recent report on the
safety of ephedrine alkaloids that was sponsored by industry endorsed
this perspective, as expressed in the following quote: ``As the law
appropriately suggests, the FDA cannot assume responsibility for
protecting the public from themselves, if they choose to use this or
any other product at higher than recommended levels or otherwise misuse
properly labeled products.''
The other perspective on warning statements that we discussed in
the analysis of the June 1997 proposal was that adverse events that
occur despite the presence of adequate warning statements represent
social costs. Under this perspective, requiring a warning statement
would not be a sufficient regulatory action unless it actually caused
consumers to change their behavior so as to eliminate any adverse
events associated with these products. Some comments supported this
perspective by arguing that warning statements are inappropriate or
inadequate because they probably would not significantly reduce the
number of adverse events among all or some subset of consumers.
(Response) In the analysis of removing dietary supplements
containing ephedrine alkaloids from the market, we concluded that
removing dietary supplements containing ephedrine alkaloids from the
market would generate net social benefits if consumers fail to
incorporate the probability of adverse events into their demand for
those products. Our assessment of the effects of a warning statement
hinges on the same uncertainty. If consumers do not fully incorporate
the risk of adverse events into their demand for products containing
ephedrine alkaloids, and if the proposed warning label would cause at
least some consumers to change their demand so as to incorporate the
risk, then the warning label could reduce adverse events and generate
net social benefits. The likelihood of that outcome depends on the
effectiveness of current warning statements and of warning statements
in general. One consideration that suggests that consumers fail to
incorporate, at least in part, the probability of adverse events into
their market behavior is that some consumers do not know they have the
underlying conditions discussed in warning statements.
ii. Comparison to existing warning statements. In economic terms,
the benefit of changing a warning statement is the value that consumers
place on the change in the information available on product labels. If
we had information on how consumers value different warning statements,
then we would not need to consider the impact of changing the warning
statements on adverse events. Without that information, we must infer
the value from the adverse health effects that changing the warning
statement would eliminate. This value represents the minimum value of
changing the warning statements: Consumers who change their behavior in
response to the change in warning statements would presumably be
willing to pay the amount that they saved in health costs and lost
utility because of that change in warning statements, but some
consumers might value the information even though they do not change
their behavior. Because the information value for consumers who do not
change their behavior is likely to be small, the value of the
eliminated adverse events is probably a close approximation to the
value of changing the warning statements. Therefore, we have based our
analysis on estimating the impact on adverse events of changing the
warning statements from the existing voluntary industry warning
statements to the proposed mandatory warning statement.
iii. Effectiveness of warning statements in eliminating adverse
events. In the analysis of the June 1997 proposal, we estimated that
the warning statement that we proposed in 1997 would reduce the
estimated number of annual adverse events caused by dietary supplements
containing ephedrine alkaloids by 0 to 15 percent (62 FR 30678 at
30712).
(Comment 83) A number of comments addressed this estimate. One
comment suggested that the estimated impact was too low and noted that
a recent study showed that almost 70 percent of adults read product
labels every time they use
[[Page 6841]]
a product. However, another comment argued that warning statements
would probably be ineffective because most consumers do not read
product labels. This comment noted that there is no evidence that
warning labels on alcohol and tobacco products reduced consumption of
those products. Other comments simply pointed out that warning
statements might not eliminate all adverse events, because some
consumers might not read or follow them. One comment provided a number
of reasons why warning statements might be ineffective at reducing
adverse events (e.g. many consumers do not read labels for OTC drugs
and would be even less likely to do so for dietary supplements, many
consumers base their usage patterns on suggestions read in magazines
rather than on label information, many consumers believe consuming more
of a dietary supplement makes it more effective). Another comment noted
that we appeared to infer the ostensible benefit of warning statements
rather than demonstrating their effectiveness through carefully
conducted clinical trials. This comment also argued that warning
statements would not be useful for consumers with unrecognized medical
conditions that might predispose them to adverse reactions caused by
ephedrine alkaloids, such as hypertension, hyperthyroidism, vascular
malformations of the brain, and subclinical cardiac arrhythmias. One
comment suggested that the proposed warning statement was too long to
be effective. This comment claimed that the necessary print size and
spacing would discourage some consumers from reading the warning
statement.
(Response) These comments did not provide sufficient information to
allow us to change our estimate of the effectiveness of the warning
statement that we originally proposed in 1997 and revised in 2003. The
comments that noted that warning statements might not eliminate all
adverse events are consistent with the assumption that warning
statements would eliminate 0 to 15 percent of the adverse events. The
comment that noted a study that showed 70 percent of consumers read
product labels every time they purchase a product did not provide a
reference for that study, but the reported results are consistent with
other studies. The FDA 2002 Health and Diet Survey found that 80
percent of nonvitamin/mineral supplement users reported that they used
product labels to find out if there were side effects or drug
interactions associated with a product or if anyone should avoid the
product. A survey of consumer use of dietary supplements by Prevention
Magazine found that the following percentages of herbal remedy shoppers
reported looking for the following types of information: 72 percent for
possible side effects; 70 percent for warnings for people not to take
the supplement, e.g. pregnant women; 65 percent for warnings about
possible interactions with prescription medicines; and 59 percent for
warnings about possible interactions with OTC products (Ref. 154).
However, consumers who read warning statements will not necessarily
change their behavior. A 2002 recent survey of consumers who have
recently taken OTC pain medications found that 84 percent read at least
some of the label the first time they took a product but that 44
percent said they took more than the recommended dosage, despite the
warnings on the label (Ref. 155). In general, most of the literature on
warning statements has not focused on product purchase or use pattern
decisions but on issues such as comprehension, awareness, and
believability (Ref. 156). Some articles have found that alcohol warning
statements have had little or no impact on behavior (Ref. 157).
However, these results do not necessarily hold for the proposed warning
statement because the effectiveness of warning statements varies with a
number of considerations, including the content and format of the
warning and the characteristics of the consumers reading the warning.
Thus, this literature does not provide a basis for revising our
assumption that the proposed warning statement will reduce adverse
events by 0 to 15 percent. However, the fact that most dietary
supplements already bear extensive warning statements suggests that 15
percent is probably an upper bound and that a value closer to 0 percent
is probably more likely.
(Comment 84) Some comments argued that the proposed warning
statement would probably have little effect on the number of adverse
events because many dietary supplements that contain ephedrine
alkaloids already bear warning statements. One comment argued that some
existing warning statements fully and accurately describe the potential
for adverse effects and thereby satisfy the objectives of the proposed
warning statement. One comment argued that some existing warning
statements are more complete than the proposed warning statement.
However, one comment said that the proposed warning statement would
probably be more effective than existing warning statements because
existing warnings do not alert consumers to avoid taking multiple
products containing ephedrine alkaloids at the same time.
(Response) To address these comments, we reviewed and compared the
labels of forty dietary supplements containing ephedrine alkaloids that
we collected between March 20 and May 30, 2001, and also compared the
number of adverse reports received during the period January 2000 to
January 2004 as warning labels appeared on certain dietary supplements.
(Ref. 158) All of the product labels bore some sort of warning
statement. Most warning statements had many of the same basic elements
as the proposed warning statement. For example, most existing warnings
listed various conditions under which consumers should not take the
product, various conditions under which consumers should see a health
care provider before taking the product, and side effects or symptoms
that should lead consumers to consult with a health care provider.
However, the specific content of the various elements varied quite a
bit both among existing warning statements and between existing warning
statements and the proposed warning statement. Some elements of the
proposed warning statement were common in existing warning statements;
other elements were less common. For example, none of the existing
product labels carried a PDP warning statement. In contrast, most
product labels carried some sort of warning for people who had
previously experienced heart problems. In addition, parts of some
existing warnings were more strongly worded than the corresponding
parts of the proposed warning. In other cases, parts of the proposed
warning were more strongly worded than the corresponding parts of
existing labels. Our label comparison did not support the notion that
the proposed warning statement would have no effect because it was
identical to existing warning statements. The comparison did suggest
that the proposed warning statement is similar in many respects to
existing warning statements, and that the proposed warning statement
might not reduce adverse events very much. This result is consistent
with the assumption that the proposed warning statement might eliminate
between 0 and 15 percent of adverse events.
(Comment 85) Some comments argued that the proposed warning
statement would be ineffective because some States already require
warning statements, and the presence of multiple warning statements
would confuse consumers.
[[Page 6842]]
(Response) Multiple warning statements might reduce the impact of
the proposed warning statement. However, many different warning
statements might be more effective than one or a few. The comments did
not provide sufficient information to enable us to revise our estimate
of the effectiveness of the proposed warning statement based on the
possibility that some products might face multiple labeling
requirements.
b. Revised benefit estimates. When we revise the analysis as
described previously, we obtain the estimated benefits shown in table 5
of this document. The assumption underlying the table is that the
proposed warning statement would cause some proportion of consumers to
incorporate the risks from dietary supplements containing ephedrine
alkaloids into their demand for these products. Some proportion of
those consumers (0 to 15 percent) would cease using those products,
which would reduce the number of adverse events by a like percentage.
The benefits would therefore be some percentage (between 0 and 15
percent) of the benefits of removing dietary supplements containing
ephedrine alkaloids from the market. The results presented in table 5
of this document apply to every year after the first year. Benefits for
the first year would be lower because our proposed rule would have
allowed firms up to 6 months to comply with the warning statement
requirements. We do not know the actual rate at which firms would come
into compliance during the initial 6 months after publication of a rule
finalizing the proposed warning statement requirements. To simplify the
analysis, we assume that it would take all firms 6 months to comply
with such a rule. Under this assumption, the benefits in the first year
would be half those of every year after the first year. In the summary
of regulating options and table 8 of this document, we use the range $0
to $20 million for annual benefits (excluding the first year) because
it is inconsistent with the presentation of the other options.
Table 5.--Annual Benefits of Option Three (Require the 2003 Proposed
Warning Statement) Based on Eliminating 0 to 15 Percent of the Sentinel
and Possible Sentinel Events
------------------------------------------------------------------------
QALY Loss Per Medical Costs
Type Number Case Per Case
------------------------------------------------------------------------
Death 0.0 to 0.2 NA (used VSL) $25,742
MI (heart 0.0 to 0.2 0.29 $30,586
attack)
CVA (stroke) 0.0 to 0.3 0.2 $20,898
Other 0.0 0.29 $30,586
Cardiovascular
(e.g.
Cardiomyopathy
, Ventricular
Tachycardia)
Other 0.0 minimal $13,212
Neurological
(e.g.
Transient
Ischemic
Attack)
Seizure 0.0 to 0.1 minimal $11,812
Psychiatric 0.0 to 0.2 minimal $6,927
------------------------------------------------------------------------
Table 6.--Annual Benefits of Option Three (Require the 2003 Proposed
Warning Statement) Based on Alternative Assumptions of Reporting Rates,
Rounded to $ Millions
------------------------------------------------------------------------
Adverse Event Reporting Rate
Value of Avoiding Fatal -----------------------------------------------
Cases and QALY Losses 10 percent 50 percent 100 percent
------------------------------------------------------------------------
$ per fatal case = $5 $0 to $11 $0 to $2 $0 to $1
million $ per QALY =
$100,000
$ per fatal case = $6.5 $0 to $14 $0 to $3 $0 to $1
million $ per QALY =
$100,000
$ per fatal case = $5 $0 to $14 $0 to $3 $0 to $1
million $ per QALY =
$300,000
$ per fatal case = $6.5 $0 to $17 $0 to $3 $0 to $2
million $ per QALY =
$300,000
$ per fatal case = $6.5 $0 to $20 $0 to $4 $0 to $2
million $ per QALY =
$500,000
------------------------------------------------------------------------
c. Costs of requiring the 2003 proposed warning statement.
i. Label Costs.
(Comment 86) Some comments said that the proposed PDP or nonPDP
warning statements are too long to fit on the labels of most dietary
supplement products. One comment noted that firms package many
``traditional style extracts'' in containers that have a maximum label
size of 1.75 x 3.75 inches, or about 6.6 square inches. The comment
argued that the proposed warning statements cannot fit on a label of
this size. One comment argued that the proposed warning statement would
take up so much space on the label that firms would be able to provide
very little other information on the label. One comment argued that
there is not enough room on package labels for multiple warning
statements and suggested that we clarify that our proposed warning
statement would preempt any state labeling requirements.
(Response) We reviewed the labels of the 40 dietary supplements
containing ephedrine alkaloids that we collected between March 20 and
May 30, 2001, to investigate label size. Most labels were wrap-around
adhesive labels with a minimum label size of about 7.5 square inches
and an average of about 22.8 inches. Nearly all labels already bore
extensive warning statements, and most of the content of the existing
warning statements was distinct from the additional warning material
required by some States. Therefore, we conclude that the proposed
warning statements would probably have fit on most product labels.
However, some dietary supplements containing ephedrine alkaloids,
possibly including traditional style extracts, might have significantly
smaller labels than the products that we collected. If we had adopted
this option, we would have addressed this possibility in a number of
ways. Firms that cannot fit the proposed PDP warning statement on the
PDP if they use the normal font size would be able to use a smaller
font size. Firms that cannot fit the nonPDP warning statement on the
product labels could place the warning statement on any product
labeling that is an integral part of the outer product packaging such
that consumers may read the warning statement at the point of purchase,
including the rise backing, panel extension, and outsert. In some
cases, firms may already use these packaging features. These firms
would simply need to revise the content of existing
[[Page 6843]]
labeling. In other cases, firms might need to change the style of their
packaging to utilize these types of labels. Rather than changing the
style of their packaging, firms could also change the size of the
package to increase the label space available for the warning
statement. Changing the product packaging in one of these ways might
require some firms to purchase new packaging machinery, which would be
an additional cost beyond the cost of the label changes that we
discussed in the analysis of the June 1997 proposal. We have
insufficient information to estimate the number of products that might
need to take these steps. Based on our review of existing product
labels, we estimate that the number of such products is probably very
small.
We have reestimated labeling costs because we have new information
on the number of dietary supplements containing ephedrine alkaloids and
we have updated the labeling cost model that we used to estimate
labeling costs in the analysis of the June 1997 proposed rule. The cost
of changing labels varies with the amount of time that we give firms to
change the labels. We previously proposed setting the effective date
for this option to be 180 days after the publication of the final rule.
According to the revised label cost model, the one-time cost of adding
or revising a PDP and a nonPDP warning statement to the labels of all
dietary supplements under a 6-month compliance period would be
approximately $140 million to $319 million. The labeling cost model
does not differentiate dietary supplements that contain ephedrine
alkaloids from other dietary supplements. However, a database of
dietary supplements compiled by Research Triangle Institute (RTI) under
contract to FDA listed a total of 3,000 dietary supplement products in
1999, and 49 of those products, or about 2 percent, listed ephedrine or
one of the following sources of ephedrine alkaloids in their ingredient
lists: Ephedra, ephedra extract, ephedra herb, Ephedra sinica Stapf.,
ma huang, ma huang extract, ma huang herb, ma huang concentrate, or ma
huang herb extract (Ref. 159). In the absence of other information, we
assume that the cost of changing the labels of these products would be
about 2 percent of the cost of changing all dietary supplement product
labels. Therefore, we estimate that the one-time cost of changing the
labels of dietary supplements containing ephedrine alkaloids is $3
million to $6 million. Annualizing this cost over 20 years at 3 percent
gives an annual cost that rounds to $0 million per year; that is, less
than $500,000 per year. Annualizing this cost over 20 years at 7
percent gives an annual cost of $0 million to $1 million.
ii. Risks of substitutes/absence of weight loss.
(Comment 87) One comment noted that the proposed warning statement
would instruct consumers not to take dietary supplements containing
ephedrine alkaloids before or during strenuous exercise. This comment
argued that this element of the warning statement could harm consumers
by inhibiting weight loss because exercise is an essential component of
a weight loss program.
(Response) As we discussed under Option Two of this section, we
have insufficient information to estimate countervailing health effects
such as the health risks generated by the use of substitute products or
by the reduction or elimination of weight loss benefits. However, for
this option, we have calculated benefits as a range of $0 to $20
million. This range is consistent with the existence of countervailing
health risks from the source suggested by this comment.
d. Effective date.
(Comment 88) Some comments recommended that we revise the proposed
effective date for the warning statement that we proposed in 1997 and
revised in 2003. One comment suggested that we set the effective date
to 12 months after publication of the final rule, rather than the
proposed 180 days after publication of the final rule, to give industry
more time to comply with the labeling requirements. Another comment
suggested that we set the effective date to 60 days after publication
of the final rule. Some comments suggested that we base the effective
date on labeling at the manufacturing site. Under this approach, we
would require products leaving the manufacturing site after the
effective date to bear the warning statements, but firms could continue
to sell existing inventory without the warning statement after that
date.
(Response) Setting the effective date to 12 months after
publication of a final rule requiring the warning statement would lead
to one time labeling costs of between $2 million and $5 million.
Annualizing this cost over 20 years at 3 percent and 7 percent gives an
annual cost that rounds to $0 million per year (i.e., less than
$500,000 per year). This would also reduce benefits in the first year
to $0 under the simplifying assumption that all firms would take 12
months to comply with the required warning statement.
Eliminating all costs associated with unusable label or package
inventory by allowing firms to continue to sell product without the
warning statement after the effective date would lead to compliance
costs of $2 million to $6 million under the proposed 180 day compliance
period. Annualizing this cost over 20 years at 3 percent gives an
annual cost that rounds to $0 million per year (i.e., less than
$500,000 per year). Annualizing this cost over 20 years at 7 percent
gives an annual cost of $0 million to $1 million per year. In our
summary statements, we present the cost estimates under the 7 percent
discount rate because that range includes the range of costs that we
estimated under a 3 percent discount rate. However, this option would
also generate additional enforcement costs because we would need some
way of determining that the products that firms sell without the
warning statement were actually labeled before the effective date. In
addition, this revision would reduce benefits over a number of years
according to the proportion of products sold during that time that did
not bear warning statements. The period over which benefits would be
reduced could be quite large because firms might produce as much
product as possible prior to the effective date to avoid having to meet
the labeling requirements. The comments did not provide information on
this issue, and we are unable to estimate this reduction in benefits.
We compare costs of different effective dates for the proposed
labeling option in table 7 of this document. We only consider first
year net benefits because changing the effective date from 180 days to
365 days only affects benefits in the first year. After the first year,
annual benefits would be the same for either effective date. To obtain
the higher bound estimate of net benefits, we start with the higher
bound estimate of benefits and subtract the lower bound estimates of
costs. To obtain the lower bound estimate of net benefits, we start
with the lower bound estimate of costs and subtract the higher bound
estimate of costs. We do not have information suggesting that any of
these options would lead to greater net benefits than the proposed
enforcement period of 180 days.
[[Page 6844]]
Table 7.--Comparison of Effective Date Options for Option Three (Require the Proposed Warning Statement),
Rounded to $ Millions
----------------------------------------------------------------------------------------------------------------
Annualized Cost First Year Benefits First Year Net Benefits
Effective Date (millions) (millions) (millions)
----------------------------------------------------------------------------------------------------------------
180 days $0 to $1 $0 to $10 -$1 to $10
365 days $0 $0 $0
180 days at manufacturing site $0 plus additional NA NA
enforcement costs
----------------------------------------------------------------------------------------------------------------
e. Conclusions on the benefits and costs of 2003 proposed warning
statement. We estimate costs to include the one-time cost of changing
the labels of dietary supplements containing ephedrine alkaloids to be
$3 million to $6 million, which rounds to approximately $0 million per
year (i.e. less than $500,000 per year) when annualized over 20 years
at 3 percent and approximately $0 million to $1 million per year when
annualized over 20 years at 7 percent. We are unable to quantify
potential recurring countervailing health costs. We estimate the
recurring annual benefit to be $0 to $20 million, depending on the
reporting rate for adverse events, and the method used to value those
events. Therefore, we estimate the annual net benefit of this option to
be -$1 million to $20 million. In the long run, this option would
probably generate net benefits, for two reasons: First, the benefits
recur annually and any non-zero level of benefits will eventually
surpass the one-time labeling cost. Second, as we discussed above, the
recurring countervailing health costs are unlikely to exceed the
recurring health benefits.
7. Option Four--Require the Proposed Warning Statement, But Modify it
or Require it Only on Certain Products.
a. Require warning only for certain products. We discussed a number
of comments under Option Two that claimed that certain dietary
supplements containing ephedrine alkaloids do not pose any health
risks. That discussion is also relevant in the context of exempting
certain products from the proposed warning statement. The summary of
those comments and our response is the same as under Option Two in
section VIII.A.5 of this document. For example, one comment suggested
that warning statements are unnecessary for herbal products that firms
distribute to ``healthcare professionals,'' including members of the
American Herbalists Guild. We do not have sufficient information to
estimate the impact of exempting products based on patterns of
distribution or other product characteristics.
b. Placement and format of warning statement.
(Comment 89) Some comments addressed the placement of the proposed
warning statement on product packages. Some comments suggested that we
allow firms to use inserts, stickers, or ``peel away'' labels. One
comment said that we should allow firms to use alternative methods of
disseminating warning information if they dispense products that are
part of a bulk decoction formula that lacks standard labeling, such as
products compounded and dispensed in Chinese herbal medicine pharmacies
or by ``qualified health professionals.''
(Response) According to the March 2003 notice, we proposed to allow
firms to use special labeling for the nonPDP warning statement as long
as consumers could read the warning statement at the point of purchase.
(Comment 90) Some comments objected to the PDP warning statement
that was part of the revised warning statement that we proposed in
2003. Other comments supported the 2003 proposed PDP warning statement.
Some comments suggested that we require firms to use the PDP warning
statement on both the product container and the outside container or
wrapper of the retail package. One comment suggested that we require
firms to include the PDP warning statement in any promotional
literature and advertising.
(Response) Eliminating the PDP warning statement but retaining the
nonPDP warning statement would probably significantly reduce the impact
of the proposed warning statement. The PDP warning statement was one of
the main elements of the proposed warning statement that differed from
most existing warning statements. Reducing the impact of the warning
statement by eliminating the proposed PDP warning statement would
reduce both the benefits and the distributive impacts of the warning
label option. However, eliminating the PDP warning statement would have
little impact on the overall cost of changing labels to comply with the
proposed warning statement because firms would still need to change
labels even if we did not require a PDP warning statement. Requiring
firms to place the warning statement on both the product container and
the outside container or wrapper and requiring firms to include it in
any promotional literature and advertising might increase the impact of
the warning statement, but would also increase the costs. The comments
did not provide sufficient information to establish that the benefits
from these revisions would outweigh the costs.
(Comment 91) One comment argued that the PDP for mail order dietary
supplements corresponds to the front page of any product literature
that a firm uses to advertise its product. This comment said that the
proposed regulation would, therefore, require some firms to change
their pamphlets and other material. The comment argued that such a
requirement would put mail order businesses at a competitive
disadvantage relative to retail businesses. The comment suggested that
we allow the warning statement to appear either above the mail order
form that consumers use to order the product or above the toll free
telephone number that consumers call to order the product. The comment
argued that these locations would be more similar to the labeling
requirements for OTC drugs.
(Response) The PDP for mail order dietary supplements is defined in
the same way as the PDP for supplements sold in other ways: The label
that appears on the front of the product package. It does not
correspond to the front page of any product literature that a firm uses
to advertise its product.
(Comment 92) Some comments objected to the requirement that firms
set off the warning statement in a box graphic. One comment argued that
the RAND report did not support the need for a black box type of
warning statement. Some comments suggested that we give manufacturers
greater leeway with respect to the format of the warning statement.
Other comments supported the requirement that firms set off the warning
statement in a box graphic. One comment suggested that we require firms
to set off the warning
[[Page 6845]]
statement in a brightly colored or neon box instead of in a black box.
(Response) The proposed warning statement is consistent with
current research on effective warning statements. Eliminating the box
graphic would probably not significantly reduce relabeling costs.
However, it might reduce the visibility of the warning statement, which
would reduce the distributive impacts of the rule as well as the rule's
potential health benefits. We have no information establishing that
colored boxes are more effective than black boxes. Depending on the
background color of the label, colored boxes may reduce the color
contrast between the border and the background, which would decrease
visibility of the warning statement. In addition, requiring colored
boxes would increase labeling costs because some existing labels are
not printed in colors.
c. Content of PDP warning.
(Comment 93) Some comments suggested that we revise the proposed
PDP warning statement in various other ways. One comment argued that
there was no evidence that ``whole-herb products'' containing ephedrine
alkaloids have been associated with heart attack, stroke, seizure, or
death, so that the proposed PDP warning statement would be
inappropriate for those products. This comment suggested that we revise
the PDP statement so that it simply informs consumers that a product
contains ephedrine alkaloids and directs them to a warning statement
elsewhere on the label. A number of comments argued that shortening the
proposed PDP warning statement would make it more effective. One
comment noted that the proposed approach is inconsistent with the
``signal/refer/explain'' format used for the labeling of other
hazardous products. However, one comment suggested that we add material
to the PDP warning statement, rather than shortening it.
(Response) Revising the PDP warning statement for some or all
dietary supplements that contain ephedrine alkaloids would have little
effect on labeling costs because firms would still need to revise their
labels even if we did not require a PDP warning statement. The comments
did not provide sufficient information to establish that revising the
PDP warning statement would increase net benefits.
(Comment 94) A number of comments raised the issue of whom we
instruct consumers to contact under various conditions. The proposed
PDP and nonPDP warning statements suggest that consumers contact a
``doctor'' under various conditions. Some comments suggested we use a
more general phrase such as ``health care provider'' in order to
include nurse practitioners and pharmacists. One comment suggested that
we change ``doctor'' to ``licensed health care provider'' to include
acupuncturists who are trained in traditional Chinese medicine. The
comment noted that at least half of the states that regulate the
practice of acupuncture include the use of herbs in the authorized
scope of practice of acupuncturists. The comment also noted that herbal
ephedra is used by health care providers in other disciplines, such as
naturopathy and herbalism. This comment argued that it was important to
protect the ability of these groups to dispense dietary supplements
containing ephedrine alkaloids.
(Response) Changing the specification of the person that the
proposed warning label directs consumers to contact under various
conditions would have little impact on labeling costs but would affect
the benefits and distributional effects of this rule. Medical doctors
are probably in the best position to advise consumers on the health
implications of consuming ephedrine alkaloids under various conditions,
but consumers might be able to get comparable advice from some other
sources, including pharmacists and other health care providers, as well
as some practitioners of acupuncture, herbalism, and naturopathy. On
the other hand, obtaining advice from a medical doctor is probably more
costly for many consumers than obtaining advice from other potential
sources. In addition, some consumers may be unwilling to seek advice
from medical doctors on the use of dietary supplements for reasons
other than cost. These consumers may be less likely to follow
directions to contact a medical doctor than they are to follow
directions to contact a broader variety of health care providers. This
component of the warning statement could also have distributional
effects because directing consumers to contact a medical doctor
increases the demand for the services of medical doctors and reduces
the demand for the services of competing health care providers. The
comments did not provide sufficient information to allow us to
determine that changing the specification of the person that the label
directs consumers to contact would increase net benefits. The comments
also did not provide enough information for us to quantify the
potential distributional impact of revising this component of the
label.
(Comment 95) Some comments noted that the PDP warning statement
implied that ephedrine alkaloids cause heart attack, stroke, seizure,
and death. These comments argued that this is misleading because no one
has proven that ephedrine alkaloids cause these types of adverse
events. One comment suggested that if we refer to these types of
adverse events in the warning statement, then we should include a
qualifying statement explaining that no one has established a causal
link between these types of adverse events and ephedrine alkaloids.
This comment also suggested that we indicate in the warning statement
that reports of serious adverse events are extremely rare.
(Response) Although the information in the proposed warning
statement is factually correct because some people have reported the
specified adverse events after consuming ephedrine alkaloids, some
consumers might interpret the phrase ``have been reported'' to mean
that a proven causal relationship exists between the consumption of the
ephedrine alkaloids and the reported adverse events. This perception
could generate additional costs in terms of lost consumer utility
because some consumers who would choose not to consume these products
if a proven causal relationship existed might choose to continue to
consume these products if a causal relationship were only possible or
even likely. One way to reduce potential misperceptions would be to add
a disclaimer to the label, explaining that the causal relationship
between ephedrine alkaloids and these adverse events may be uncertain.
This additional material might either decrease or increase the demand
for these products, and consumers are generally less likely to respond
to a longer, qualified warning statement, than to a shorter, non-
qualified warning statement. The comments did not provide sufficient
information to establish that adding this type of clarification to the
warning would increase the benefits of the warning statement.
d. Content of nonPDP warning statement.
(Comment 96) A number of comments suggested that we revise the
proposed nonPDP warning statement. Some comments suggested that we use
the same warning statement that appears on OTC drug products containing
ephedrine alkaloids. One comment suggested that we allow firms to use
the OTC warning statement for dietary supplements that they sell
directly to health professionals for subsequent sale to consumers. One
comment argued that the warning statement should not instruct consumers
to contact a doctor if they experience nausea because nausea is not
likely to be a precursor symptom
[[Page 6846]]
of a potentially serious or life-threatening condition.
Some comments objected to the warning that the risk of serious side
effects increases with duration of use. One comment suggested that the
scientific data showed that adverse effects dramatically decline with
continued use. Some comments argued that there was no persuasive
evidence that ephedrine alkaloids had any cumulative effect on the
cardiovascular or central nervous systems.
One comment suggested that we allow manufacturers to add
contraindications beyond those listed on the required warning label.
One comment suggested that we require a statement clarifying that we
have not reviewed the product for safety or efficacy. Some comments
argued that we should require warning statements to include the toll
free telephone number and Web site address for our MedWatch program.
Some comments recommended that we require firms to indicate the amount
of ephedrine alkaloids present in a product on the product label.
(Response) These comments did not provide sufficient information to
analyze the costs and benefits of revising the proposed nonPDP warning
statement according to their recommendation.
e. Conclusions on benefits and costs of modifying the proposed
warning statement or requiring it only for certain products. Requiring
a warning statement for certain products only would reduce costs and
distributional effects and might reduce benefits compared with Option 3
(all comparisons in this section are with Option 3). Eliminating the
PDP warning statement or eliminating the box graphic would have little
effect on costs but would reduce distributional effects and probably
also reduce benefits. Requiring a colored box graphic instead of a
black and white box graphic would increase costs and possibly increase
distributional effects and benefits. Revising the content of the
warning statements would have little effect on costs but might increase
or decrease distributional effects and benefits, depending on the
revision. We have insufficient information to quantify these possible
impacts, so we are unable to provide a summary estimate of the costs
and benefits of this option.
8. Option Five--Generate Additional Information or Take Some Action
Other Than Removing Dietary Supplements Containing Ephedrine Alkaloids
From the Market or Requiring Warning Statements
(Comment 97) One comment argued that we have no controlled
epidemiological studies that support an association between ephedrine
alkaloids and stroke, seizure, or myocardial infarction. Other comments
noted that RAND said in its report that it was unable to establish that
ephedrine alkaloids caused adverse events and that RAND recommended
that someone perform a controlled clinical study to address the issue.
Another comment noted that Haller and Benowitz (2000) said that their
approach did not establish that ephedrine alkaloids caused adverse
events and suggested that someone do a large scale case control study
to quantitatively determine the risks associated with ephedrine
alkaloids (Ref. 34). One comment noted that the NIH National Advisory
Council for Complementary and Alternative Medicine Working Group on
Ephedra suggested that someone perform a multi-site prospective case-
control study to assess the risks associated with taking ephedra. This
comment suggested that such a study would require 4 to 8 years to
complete and cost $2 million to $4 million per year. Another comment
argued that even if someone were to establish that ephedrine alkaloids
increased cardiovascular risk by raising blood pressure, someone would
still need to do a controlled research study to determine whether that
effect outweighed the reduction in cardiovascular risk resulting from
any weight loss generated by these products. One comment argued that a
retrospective case control study is the correct study design for rare
events. This comment argued that someone could do multiple studies of
this type because they are quick, relatively inexpensive, and because
the population exposure level is relatively high at 1 percent,
according to a multistate survey on reported use of ephedra products
from 1996 to 1998. Some comments suggested that we not take regulatory
action until we determine that the adverse events that we suspect are
caused by these supplements are due to ephedrine alkaloids rather than
due to inconsistent and inaccurate formulations.
Some comments argued that we do not need to generate additional
information because we already have sufficient information to remove
dietary supplements containing ephedrine alkaloids from the market or
require warning statements. Other comments argued that we do not need
to generate additional information because we already have sufficient
information to establish that these restrictions are unnecessary. Some
of these comments argued that Morgenstern et al., which was published
after the RAND report, was just the type of case control study that the
RAND report recommended (Ref. 136). These comments noted that this
study found that ephedra did not raise the risk for hemorrhagic stroke.
However, other comments argued that this study found that ephedra did
raise the risk for hemorrhagic stroke. Some comments criticized various
aspects of that study. A number of comments argued that the only
additional studies that would be worthwhile to perform at this point
would be unethical. These comments suggested that a human subjects
committee would not allow a prospective study of the safety of
ephedrine alkaloids without medical screening. They also suggested that
a cohort study would be difficult because ephedrine alkaloids do not
generate significant health benefits and also because of the ethical
requirements to effectively inform participants of the risks.
(Response) Generating additional information might reduce the
remaining uncertainty associated with the benefits of this rule or it
might not. Generating additional information may be difficult, time
consuming, and expensive. In addition, it is not clear that reducing
the remaining uncertainty would change the outcome of this rulemaking.
The comments did not provide sufficient information to allow us to
estimate the costs and benefits of delaying rulemaking until we
generate additional information.
(Comment 98) Other comments suggested that we should take some type
of action other than issuing a regulation or generating additional
information. A number of comments suggested that we address any
problems with dietary supplements containing ephedrine alkaloids by
using our existing authority to seize unsafe or adulterated dietary
supplements. Other comments suggested that we address any problems by
using our existing authority to investigate and prosecute unscrupulous
multilevel marketing (MLM) distributors. Another comment suggested that
we develop a level 1 guidance document rather than taking regulatory
action.
(Response) The comments did not provide sufficient information to
establish that spending additional
[[Page 6847]]
resources on enforcement of existing regulations or on promulgating a
level 1 guidance document would generate greater net benefits than
issuing this final rule. Following guidance documents is strictly
voluntary. The fact that some manufacturers continue to produce dietary
supplements containing ephedrine alkaloids despite ongoing and well-
publicized concerns about the safety of such products suggests that
voluntary guidance documents are unlikely to have a significant effect.
9. Benefit-Cost Analysis: Summary
Removing dietary supplements containing ephedrine alkaloids from
the market (i.e. taking this final action) will generate estimated
benefits of between $43 million and $132 million per year. We used the
following assumptions to calculate this range of benefits: A 10 percent
reporting rate for adverse events, no potentially countervailing health
effects from the use of substitute products and other weight loss
alternatives, no countervailing health effects from potentially
foregone weight loss, and the fact that consumers do not already
understand and incorporate the risks posed by these products in their
consumption decisions. Including the impact of substitute products and
activities could reduce the rule's health benefit considerably,
possibly to $0 per year, although that is unlikely. These
countervailing effects may occur because this rule will not affect the
underlying demand for products having functional characteristics
similar to ephedrine alkaloids, and it is likely that products having
similar functional characteristics may contain similar types of
ingredients that may pose similar types of health risks. The range of
benefits includes alternative assumptions about the value of a
statistical life ($5 million and $6.5 million) and the value of a
statistical life year ($0.1 million, $0.3 million, and $0.5 million).
We also considered a reporting rate of 50 percent, which leads to
estimated annual benefits of $9 million to $26 million, and 100
percent, which leads to estimated annual benefits of $4 million to $13
million. More precise estimates of the health benefits would depend on
choosing a particular combination of assumptions.
Removing these products from the market will generate one-time
product reformulation costs of $10 million to $100 million, which
amounts to a yearly cost of $1 million to $7 million when annualized
over 20 years at an interest rate of 3 percent, and $1 million to $9
million at an interest rate of seven percent. These costs could be
partly offset by reductions in fees associated with legal actions
involving these products. In addition to the social costs, removing
dietary supplements containing ephedrine alkaloids from the market
could also generate distributional effects under which some firms
manufacturing or distributing dietary supplements containing ephedrine
alkaloids may experience reduced profits, while firms manufacturing or
distributing other dietary supplements or other weight loss
alternatives may experience increased profits. In addition, removing
dietary supplements containing ephedrine alkaloids from the market
would also generate costs in the form of lost consumer utility or
satisfaction because of the removal of a product from the market. We
estimated lost utility to be $6 million to $81 million per year.
Based on these estimates, the potential economic effects of this
rule range from a net annual social cost of $90 million per year, if
the rule's net health benefits are zero because of countervailing
health effects or because consumers already understand and voluntarily
accept the risks posed be these products, to an annual net social
benefit of $125 million, if there are no countervailing health risks
and consumers do not already understand and accept the known and
potential risks.
Table 8.--Summary of Options, Rounded to $ Millions
------------------------------------------------------------------------
Option Annual Cost Annual Benefit Net
------------------------------------------------------------------------
1. Take no new $0 $0 $0
regulatory action
(baseline)
------------------------------------------------------------------------
2a. Remove dietary $7 to $90 $43 to $132 - $47 to $125
supplements
containing
ephedrine
alkaloids from
the market (if
consumer behavior
does not already
incorporate risk)
------------------------------------------------------------------------
2b. Remove dietary $7 to $90 $0 - $90 to - $7
supplements
containing
ephedrine
alkaloids from
the market (if
consumer behavior
already
incorporates
risk)
------------------------------------------------------------------------
3. Require 2003 $0 to $1 $0 to $20 - $1 to $20
warning atatement
------------------------------------------------------------------------
4. Require warning NA NA NA
statement, but
modify it or
require only on
certain products
------------------------------------------------------------------------
5. Generate unknown unknown unknown
additional
information or
take some action
other than
removal or
warning
statements
------------------------------------------------------------------------
B. Small Entity Analysis
We have examined the economic implications of this final rule as
required by the Regulatory Flexibility Act (5 U.S.C. 601-612) and in
accordance with Executive Order 13272 (August 13, 2002). If a rule has
a significant economic impact on a substantial number of small
entities, the Regulatory Flexibility Act requires us to analyze
regulatory options that would lessen the economic effect of the rule on
small entities. We find that this final rule would have a significant
economic impact on a substantial number of small entities.
(Comment 99) Some comments addressed our estimate of the number of
small firms in the analysis of the proposed rule. Some comments argued
that we had ignored a large number of independent small distributors in
the analysis of the proposed rule. One comment suggested we revisit our
analysis of the impact of the rule on small businesses. One comment
[[Page 6848]]
suggested we obtain information on the impact of the rule on small
entities by opening a dialogue with industry associations.
(Response) We have revisited and revised our estimate of the number
of firms based on a database of dietary supplement products that the
Research Triangle Institute compiled under contract to FDA after
publication of the proposed rule. This database listed 30 firms
associated with 48 dietary supplement products containing ephedrine
alkaloids (Ref. 159). To estimate the number of these firms that are
small, we used a database of dietary supplement manufacturing practices
that was also compiled by RTI under contract to FDA (Ref. 160). This
database had size information for only a few of the 30 firms that we
identified as relevant from the first database. Therefore, we estimated
the number of small firms based on the percentage of all dietary
supplement firms in the database that would qualify as small firms. The
Small Business Administration (SBA) publishes definitions of small
businesses by the North American Industry Classification System (NAICS)
code. The firms in the database fell into the following NAICS codes:
(1) 311222 Soybean Processing, (2) 311920 Coffee and Tea Manufacturing,
(3) 325188 All Other Basic Inorganic Chemical Manufacturing, (4) 325199
All Other Basic Organic Chemical Manufacturing, (5) 325411 Medicinal
and Botanical Manufacturing, and (6) 325412 Pharmaceutical Preparation
Manufacturing. SBA defines small businesses in these NAICS codes based
on a maximum number of employees, as follows: 311222 and 311920--no
more than 500 employees; 325411 and 325412--no more than 750 employees;
and 325188 and 325199--no more than 1000 employees. The database of
firms listed 1,566 individual plants and 146 parent companies.
Essentially all individual plants qualified as small businesses (98
percent under a maximum of 500 employees and 100 percent under a
maximum of 1,000 employees). However, approximately 12 percent of the
individual plants were associated with parent companies, and only about
half of the parent companies qualified as small businesses (53 percent
under a maximum of 500 employees and 58 percent under a maximum of
1,000 employees). Based on this information, we estimated that about 94
percent of the 30 firms associated with dietary supplement containing
ephedrine alkaloids, or about 28 firms, would qualify as small
businesses.
There may also be a number of independent distributors that are not
captured in our database of dietary supplement firms. All or most of
these firms would probably qualify as small businesses. However, we do
not have sufficient information to estimate the number of distributors
or to compare their characteristics to the SBA definition of a small
business for that industry. As we noted in the previous paragraphs,
this final rule will generate shifts in demand that might adversely
affect these firms. However, the most likely substitutes for dietary
supplements containing ephedrine alkaloids are other dietary
supplements, and the same distributors that handle dietary supplements
containing ephedrine alkaloids might also handle these other dietary
supplements. Therefore, the net distributive impact on small
distributors may be small or nonexistent. Although demand shifts
generated by this final rule might also increase business for other
small businesses, we do not consider countervailing positive effects on
other small entities when assessing the impact of our rules on small
entities.
In response to the request that we open a dialogue with industry
associations, we note that small entities, and trade associations (with
member small entities) submitted a number of comments regarding small
business impact during the various comment periods for this rulemaking.
In the preceding cost-benefit analysis, we estimated that removing
dietary supplements containing ephedrine alkaloids from the market
would generate annualized cost of $1 million to $9 million over 20
years because of the need to reformulate products. This would
correspond to a cost per firm across 30 firms of between $30,000 and
$300,000 per year. In addition, we estimated that profits might be
reduced by $0 to $13 million per year due to decreased sales. Profits
may accrue to either manufacturers or distributors. If all profit
losses affected manufacturers only, then the annual profit loss per
firm across 30 firms would be between $0 and $ 430,000, which would
give a total cost per firm of $30,000 to $730,000. Most of these firms
are small, so even $30,000 per year (the lower bound) would be a
significant additional burden. We previously estimated total sales to
be $559 million to $806 million. If we assume that profits correspond
to approximately 5 percent of sales, then annual profits would be $28
million to $40 million. If we assume that all profits accrue to
manufacturers, then profits would be $0.9 million to $1.3 million per
year per firm across 30 firms. In that case, reformulation costs would
represent 2 percent to 33 percent of total profits, while total costs
would represent 2 percent to 81 percent of total profits. The
Regulatory Flexibility Act does not specify a threshold for costs to
have a significant economic impact, but the 2 ranges we have calculated
reach a high fraction of total profit; for some individual small firms
the fraction of profit would be higher. If some of the profit losses
accrued to distributors rather than manufacturers, then the potential
cost per firm across all firms would be lower. However, we have
insufficient information to estimate the number of distributors or the
sales or profits per distributor.
(Comment 100) One comment argued that the PDP warning statement
would have a significant economic impact on small businesses. This
comment argued that the nonPDP warning statement would be adequate to
protect consumers. This comment recommended that we eliminate the PDP
warning statement.
(Response) A PDP warning statement might have a significant impact
on small businesses. We have analyzed the costs of the proposed warning
statement as a whole (including both PDP and nonPDP components) in our
analysis of impacts under Executive Order 12866. However, the comment
did not provide sufficient information to differentiate the impact on
small businesses from the impact on other regulated entities, or to
differentiate the impact of the PDP warning from the impact of the
nonPDP warning.
(Comment 101) One comment recommended that we consider reasonable
alternatives to the rule in order to reduce the burden on small
businesses.
(Response) The discussion of regulatory options in the preceding
benefit-cost analysis pertains primarily to small businesses because
nearly all affected firms are small businesses under SBA size
definitions. We could develop a definition of a very small business
(different from the SBA definition of a small business) and develop
additional regulatory options to reduce the burden on those firms, but
those options would also be similar to those in the benefit-cost
analysis. As we stated elsewhere in this analysis, any option that
would reduce the regulatory burden on very small firms would also
reduce benefits by increasing the risk to public health. We do not have
sufficient information to compare the value of the
[[Page 6849]]
regulatory relief for very small firms to the associated reduction in
benefits.
IX. Environmental Impact
Removing dietary supplements containing ephedrine alkaloids from
the market will not have a significant impact on the human environment.
Therefore, an environmental impact statement is not required.
X. Paperwork Reduction Act
This final rule contains no collections of information. Therefore,
clearance by OMB under the Paperwork Reduction Act of 1995 is not
required.
XI. Federalism
We have analyzed this final rule in accordance with the principles
set forth in Executive Order 13132. FDA has determined that the rule
has a preemptive effect on State law. Section 4(a) of the Executive
order requires agencies to ``construe * * * a Federal statute to
preempt State law only where the statute contains an express preemption
provision or there is some other clear evidence that the Congress
intended preemption of State law, or where the exercise of State
authority conflicts with the exercise of Federal authority under the
Federal statute.'' Section 402(f)(1)(A) of the act states that a
dietary supplement or dietary ingredient shall be considered
adulterated if it presents a significant or unreasonable risk of
illness or injury under conditions of use recommended or suggested in
the product's labeling. If no conditions of use are suggested or
recommended in the product's labeling, the dietary supplement or
dietary ingredient is considered to be adulterated if it presents a
significant or unreasonable risk of illness or injury under ordinary
conditions of use. We have concluded that dietary supplements
containing ephedrine alkaloids present an unreasonable risk and are
therefore adulterated under section 402(f)(1)(A) of the act.
Section 402(f)(1)(A) of the act does not expressly preempt State or
local laws. Therefore, under section 4(b) of Executive Order 13132, we
are to construe our rulemaking authority as authorizing preemption of
State law by rulemaking ``only when the exercise of State authority
directly conflicts with the exercise of Federal authority under the
Federal statute or there is clear evidence to conclude that Congress
intended the agency to have the authority to preempt State law.''
We are aware that several States have laws concerning dietary
supplements containing ephedrine alkaloids, such as required label
statements, which clearly contemplate the continued marketing of such
products. Section 301(a) of the act (in relevant part) prohibits the
introduction or delivery for introduction into interstate commerce of
any adulterated food. In this rule, the agency has declared dietary
supplements containing ephedrine alkaloids to be adulterated. As a
result, State laws establishing label requirements or other
requirements that contemplate the continued marketing of these products
conflict with this final rule and, consequently, are preempted.
Section 4(c) of Executive Order 13132 instructs us to restrict any
Federal preemption of State law to the ``minimum level necessary to
achieve the objectives of the statute pursuant to which the regulations
are promulgated.'' This action meets the preceding requirement because
it only applies to State laws that contemplate the continued marketing
of this class of products.
Section 4(d) of Executive Order 13132 states that when an agency
foresees the possibility of a conflict between State law and federally
protected interests within the agency's area of regulatory
responsibility, the agency ``shall consult, to the extent practicable,
with appropriate State and local officials in an effort to avoid such a
conflict.'' Section 4(e) of Executive Order 13132 adds that, when an
agency proposes to act through adjudication or rulemaking to preempt
State law, the agency ``shall provide all affected State and local
officials notice and an opportunity for appropriate participation in
the proceedings.''
In the present rulemaking, consultation with and notice to State
officials under section 4(d) and (e) of Executive Order 13132 did not
occur before we published the June 1997 proposal. Such consultation and
notice was not possible because we published the proposed rule in the
Federal Register of June 4, 1997, and Executive Order 13132 was not
signed until August 4, 1999. OMB's guidance for implementing Executive
Order 13132 states that, when a final rule may have been issued as a
proposed rule before August 4, 1999, such that the intergovernmental
consultation process had not occurred as called for by Executive Order
13132, the agency's certification ``should so state'' (see Memorandum
for Heads of Executive Departments and Agencies, and Independent
Regulatory Agencies, dated October 28, 1999) (Ref. 161). Thus, we
certify that the intergovernmental consultation process described in
section 4(d) of Executive Order 13132 did not occur for the proposed
rule, but we also believe that State and local governments had
sufficient notice and an opportunity to participate in this rulemaking
process. We note that the proposed rule was subject to a previous
Executive Order, Executive Order 12612, which was also entitled,
``Federalism,'' and had a similar consultation and notification
obligation for federal agencies. When we issued the proposed rule, we
notified the States, and State and local health departments, among
others, submitted comments to the proposal (65 FR 17474, April 3, 2000)
(stating that State and local health departments and government
agencies had commented on the proposed rule)). Furthermore, a
subsequent notice, published on March 5, 2003, expressly asked whether
we should determine that dietary supplements containing ephedrine
alkaloids present a ``significant or unreasonable risk of illness or
injury'' under section 402(f)(1)(A) of the act (68 FR at 10417, 10419,
and 10420). Although the March 2003 notice did not contain a separate
Federalism analysis, we believe that States were aware of the March
2003 notice because at least five State or local governments or
legislators submitted comments in response to the March 2003 notice,
and most of these comments urged us to ban the sale of such products.
XII. References
The following references have been placed on display in the
Division of Dockets Management (see ADDRESSES) and may be seen by
interested persons between 9 a.m. and 4 p.m., Monday through Friday.
(FDA has verified the Web site addresses, but FDA is not responsible
for any subsequent changes to the nonFDA Web sites after this document
publishes in the Federal Register.)
1. Ephedra Monograph, Review of Natural Products, Der
Marderosian, A., ed., DrugFacts.com (http://www.factsandcomparisons.com), 2003.
2. Chen, K. K. and C. F. Schmidt, ``Ephedrine and Related
Substances,'' Medicine vol. 9, pp. 1-117, 1930.
3. Mahuang (Appendix: Mahuanggen)., Chapter in Chang, H-M. and
P. P-H. But, eds., Pharmacology and Applications of Chinese Materia
Medica, pp. 1119-1124, Singapore: World Scientific Publishing Co.
Pte. Ltd., pp. 1119-1124, 1987.
4. Karch, S. B., ``Other Naturally Occurring Stimulants,''
Chapter in Karch, S. B., The Pathology of Drug Abuse, pp. 177-198,
Boca Raton: CRC Press, 1996.
5. ``Phenethylamines,'' Chapter in Bruneton, J., ed.,
Pharmacognosy, Phytochemistry, Medicinal Plants, pp. 711-715, New
York: Laviosier Publishing, 1995.
6. Betz, J. M., Tab F, ``Review of Plant Chemistry: Alkaloids of
Ma Huang (ephedra
[[Page 6850]]
spp.),'' Food and Drug Administration, Briefing Materials for Food
Advisory Committee Special Working Group on Foods Containing
Ephedrine Alkaloids, Center for Food Safety and Applied Nutrition,
pp. 1-14, 1995.
7. World Health Organization (WHO) Monographs on Selected
Medicinal Plants, Herba Ephedrae, pp. 145-153, 1999.
8. Oshio, H., M. Tsukui, and T. Matsuoka, ``Isolation of l-
Ephedrine From `Pinelliae Tuber','' Chemical and Pharmaceutical
Bulletin (Tokyo), vol. 26, pp. 2096-2097, 1978.
9. Ghosal, S., R. B. Chauhan, and R. Mehta, ``Alkaloids of Sida
Cordifolia,'' Phytochemical Reports, vol. 14, pp. 830-832, 1975.
10. Cui, J., T. Zhou, J. Zhang, and Z. Lou, ``Analysis of
Alkaloids in Chinese Ephedra Species by Gas Chromatographic
Methods,'' Phytochemical Analysis, vol. 2, pp. 116-119, 1991.
11. Food and Drug Administration, Tab E: Additional Market
Review Information. Briefing Materials for Food Advisory Committee
on Dietary Supplements Containing Ephedrine Alkaloids, Center for
Food Safety and Applied Nutrition, pp. 1-35, 1996.
12. Brevoort, P., ``The U.S. Botanical Market--An Overview,''
HerbalGram, vol. 36, pp. 49-57, 1996.
13. Food and Drug Administration, Hardy, C., ``FDA Market Review
Update Ephedrine Alkaloid-Containing Dietary Supplements,'' 2000.
14. Food and Drug Administration, Food Advisory Committee on
Dietary Supplements Containing Ephedrine Alkaloids, Meeting
Transcript, Docket No. 1995N-0304, TR2, vol. 72-73, August 1996.
15. Food and Drug Administration, Food Advisory Committee,
Special Working Group on Foods Containing Ephedrine Alkaloids,
Meeting Transcript, Docket No. 1995N-0304, TR1, vol. 40-41, October
1995.
16. Commission on Dietary Supplement Labels: Report of the
Commission on Dietary Supplement Labels, 1997.
17. U.S. General Accounting Office (GAO), ``Dietary Supplements,
Uncertainties in Analyses Underlying FDA's Proposed Rule on
Ephedrine Alkaloids,'' GAO/Health, Education, and Human Services
Division (HEHS)/General Government Division (GGD), pp. 99-90, 1999.
18. Food and Drug Administration, ``Assessment of Public Health
Risks Associated with the Use of Ephedrine Alkaloid-containing
Dietary Supplements,'' 2000, Docket No. 2000N-1200, vol. 29-30, pp.
39-41, 2000.
19. Kernan, W. N., C. M. Viscoli, L. M. Brass, et al.,
``Phenylpropanolamine and the Risk of Hemorrhagic Stroke,'' New
England Journal of Medicine, vol. 343, pp. 1826-1832, 2000.
20. Department of Health and Human Services, Office of Inspector
General, ``Adverse Event Reporting for Dietary Supplements, An
Inadequate Safety Valve,'' OEI-01-00-00180, 2001.
21. Shekelle P., S. Morton, M. Maglione, et al., ``Ephedra and
Ephedrine for Weight Loss and Athletic Performance Enhancement:
Clinical Efficacy and Side Effects. Evidence Report/Technology
Assessment,'' No. 76 (Prepared by Southern California Evidence-based
Practice Center, RAND, under Contract No. 290-97-0001, Task Order
No. 9), AHRQ Publication No. 03-E022, Rockville, MD, Agency for
Healthcare Research and Quality, Docket No. 1995N-0304, BKG3, vol.
300 (http://www.fda.gov/OHRMS/DOCKETS/98fr/95n-0304-bkg0003-ref-07-01-index.htm), 2003.
22. Shekelle, P. G., M. L. Hardy, S. C. Morton, et al.,
``Efficacy and Safety of Ephedra and Ephedrine for Weight Loss and
Athletic Performance: A Meta-Analysis,'' Journal of the American
Medical Association, vol. 289, pp. 1537-1545, 2003.
23. U.S. General Accounting Office (GAO), ``Dietary Supplements
Containing Ephedra, Health Risks and FDA's Oversight,'' Testimony:
Before the Subcommittee on Oversight and Investigations, Committee
on Energy and Commerce, U.S. House of Representatives, GAO-03-1042T,
2003.
24. U.S. General Accounting Office (GAO): ``Dietary Supplements:
Review of Health-Related Call Records for Users of Metabolife 356,''
GAO-03-494, 2003.
25. Caveney, S. and A. Starratt, ``Glutamatergic Signals in
Ephedra (Scientific Correspondence),'' Lancet, vol. 372, p. 509,
1994.
26. Caveney, S., D. A. Charlet, H. Freitag, et al., ``New
Observations on the Secondary Chemistry of World Ephedra
(Ephedraceae),'' American Journal of Botany, vol. 88, pp. 1199-1208,
2001.
27. Food and Drug Administration, L. A. Love, Tab E,
``Evaluation of the Safety of Food Products Containing Sources of
Ephedrine Alkaloids,'' Briefing Materials for Food Advisory
Committee Special Working Group on Foods Containing Ephedrine
Alkaloids, Center for Food Safety and Applied Nutrition, pp. 1-51,
1995.
28. Food and Nutrition Board, Institute of Medicine, Dietary
Reference Intakes: A Risk Assessment Model for Establishing Upper
Intake Levels for Nutrients, Washington, DC, National Academy Press,
pp. 1-71, 1999.
29. Vasan, R. S., M. G. Larson, E. P. Leip, et al., ``Impact of
High-Normal Blood Pressure on the Risk of Cardiovascular Disease,''
New England Journal of Medicine, vol. 345, pp. 1291-1297, 2001.
29a. Vasan, R. S., J. M. Massaro, P. W. Wilson, et al.,
``Antecedent Blood Pressure and Risk of Cardiovascular Disease: The
Framingham Heart Study,'' Circulation, vol. 105, pp. 48-53, 2002.
30. Neaton, J. D., L. Kuller, J. Stamler, and D. N. Wentworth,
Impact of Systolic and Diastolic Blood Pressure on Cardiovascular
Mortality, Chapter in Laragh, J. H. and B. M. and Brenner, eds.,
Hypertension: Pathophysiology, Diagnosis, and Management, New York:
Raven Press Ltd., pp. 127-144, 1995.
31. National Institutes of Health (NIH), Report of Ephedra
Working Group to the National Advisory Council for Complementary and
Alternative Medicine (http://nccam.nih.gov/health/alerts/ephedra/working-group.htm), 2003.
32. Goldman, S. A., D. L. Kennedy, D. J. Graham, et al., ``The
Clinical Impact of Adverse Event Reporting,'' MedWatch Continuing
Education Article, pp. 1-11, 1996.
33. Middleton, W. S. and K. K. Chen, ``Ephedrine,'' Archives of
Internal Medicine, vol. 39, pp. 385-403, 1927.
34. Haller, C. A. and N. L. Benowitz, ``Adverse Cardiovascular
and Central Nervous System Events Associated With Dietary
Supplements Containing Ephedra Alkaloids,'' New England Journal of
Medicine, vol. 343, pp. 1833-1838, 2000.
35. Hoffman, B. B. and R. J. Lefkowitz, ``Catecholamines,
Sympathomimetic Drugs, and Adrenergic Receptor Antagonists,''
Chapter in Hardman, J. G., A. G. Gilman, and L. E. Limbird, eds.,
Goodman & Gilman's The Pharmacological Basis of Therapeutics, New
York, St. Louis, McGraw-Hill Chapter, pp. 199-248, 1996.
36. Astrup, A., S. Toubro, S. Cannon, et al., ``Thermogenic
Synergism Between Ephedrine and Caffeine in Healthy Volunteers: A
Double-Blind, Placebo-Controlled Study,'' Metabolism, vol. 40, pp.
323-329, 1991.
37. Taguchi, N.,T. Nishikawa, S. Inomata, et al., ``Hemodynamic
Effects of Intravenous Ephedrine in Infants and Children
Anesthetized With Halothane and Nitrous Oxide,'' Anesthesia and
Analgesia, vol. 82, pp. 568-573, 1996.
38. The Xamoterol in Severe Heart Failure Study Group,
``Xamoterol in Severe Heart Failure,'' Lancet, vol. 336, pp. 1-6,
1990.
39. Packer, M., J. R. Carver, R. J. Rodeheffer, et al., ``Effect
of Oral Milrinone on Mortality in Severe Chronic Heart Failure, The
PROMISE Study Research Group,'' New England Journal of Medicine,
vol. 325, pp. 1468-1475, 1991.
40. Cowley, A. J. and A. M. Skene, ``Treatment of Severe Heart
Failure: Quantity or Quality of Life? A Trial of Enoximone,''
Enoximone Investigators, British Heart Journal, vol. 72, pp. 226-
230, 1994.
41. Brown, O. M., Adrenergic Drugs, Chapter in Smith, C. M. and
A. M. Reynard, eds., Essentials of Pharmacology, Philadelphia, W. B.
Saunders Co., pp. 75-85, 1995.
42. Haller, C. A., P. Jacob, III and N. L. Benowitz,
``Pharmacology of Ephedra Alkaloids and Caffeine After Single-Dose
Dietary Supplement Use,'' Clinical Pharmacology and Therapeutics,
vol. 71, pp. 421-432, 2002.
43. White, L. M., S. F. Gardner, B. J. Gurley, et al.,
``Pharmacokinetics and Cardiovascular Effects of Ma-huang (Ephedra
Sinica) in Normotensive Adults,'' Journal Clinical Pharmacology,
vol. 37, pp. 116-122, 1997.
44. Gurley, B. J., S. F. Gardner, L. M. White, et al.,
``Ephedrine Pharmacokinetics After the Ingestion of Nutritional
Supplements Containing Ephedra Sinica (Ma Huang),'' Therapeutic Drug
Monitoring, vol. 20, pp. 439-445, 1998.
45. Eggertsen, R., A. Andreasson, T. Hedner, et al., ``Effect of
Coffee on Ambulatory Blood Pressure in Patients With Treated
Hypertension,'' Journal of Internal Medicine, vol. 233, pp. 351-355,
1993.
46. Myers, M. G. and R. A. Reeves, ``The Effect of Caffeine on
Daytime Ambulatory Blood Pressure,'' American Journal of
Hypertension, vol. 4, pp. 427-431, 1991.
[[Page 6851]]
47. Jacobs, I., H. Pasternak, and D. G. Bell, ``Effects of
Ephedrine, Caffeine, and Their Combination on Muscular Endurance,''
Medicine and Science in Sports and Exercise, vol. 35, pp. 987-994,
2003.
48. Berlin, I., D. Warot, G. Aymard, et al., ``Pharmacodynamics
and Pharmacokinetics of Single Nasal (5 mg and 10 mg) and Oral (50
mg) Doses of Ephedrine in Healthy Subjects,'' European Journal of
Clinical Pharmacology, vol. 57, pp. 447-455, 2001.
49. Boozer, C. N., P. A. Daly, P. Homel, et al., ``Herbal
Ephedra/Caffeine for Weight Loss: A 6-Month Randomized Safety and
Efficacy Trial,'' International Journal of Obesity and Related
Metabolic Disorders, vol. 26, pp. 593-604, 2002.
50. Kaplan, N., Expert Opinion: A Review of Boozer, CN, et al.,
Herbal Ephedra/Caffeine for Weight Loss: A 6-Month Randomized Safety
and Efficacy Trial, Review Prepared for the Food and Drug
Administration, 2003.
51. Atkinson, R., Expert Opinion: A Review of Boozer, CN, et
al., Herbal Ephedra/Caffeine for Weight Loss: A 6-Month Randomized
Safety and Efficacy Trial, Review Prepared for the Food and Drug
Administration, 2003.
52. Espeland, M., Expert Opinion: A Review of Boozer, CN, et
al., Herbal Ephedra/Caffeine for Weight Loss: A 6-Month Randomized
Safety and Efficacy Trial, Review Prepared for the Food and Drug
Administration, 2003.
53. Hajjar, I. and T. A. Kotchen, ``Trends in Prevalence,
Awareness, Treatment, and Control of Hypertension in the United
States, 1988-2000,'' Journal of the American Medical Association,
vol. 290, pp. 199-206, 2003.
54. Lewington, S., R. Clarke, N. Qizilbash, et al., ``Age-
Specific Relevance of Usual Blood Pressure to Vascular Mortality: A
Meta-Analysis of Individual Data for One Million Adults in 61
Prospective Studies,'' Lancet, vol. 360, pp. 1903-1913, 2002.
55. Collins, R., R. Peto, S. MacMahon, et al., ``Blood Pressure,
Stroke, and Coronary Heart Disease, Part 2, Short-term Reductions in
Blood Pressure: Overview of Randomised Drug Trials in Their
Epidemiological Context,'' Lancet, vol. 335, pp. 827-838, 1990.
56. Staessen, J. A., J. G. Wang, and L. Thijs, ``Cardiovascular
Prevention and Blood Pressure Reduction: A Quantitative Overview
Updated Until 1 March 2003,'' Journal of Hypertension, vol. 21, pp.
1055-1076, 2003.
57. Neal, B., S. MacMahon, and N. Chapman, ``Effects of ACE
Inhibitors, Calcium Antagonists, and Other Blood-Pressure-Lowering
Drugs: Results of Prospectively Designed Overviews of Randomised
Trials,'' Blood Pressure Lowering Treatment Trialists'
Collaboration, Lancet, vol. 356, pp. 1955-1964, 2000.
58. MacMahon, S., R. Peto, J. Cutler, et al., ``Blood Pressure,
Stroke, and Coronary Heart Disease. Part 1, Prolonged Differences in
Blood Pressure: Prospective Observational Studies Corrected for the
Regression Dilution Bias,'' Lancet, vol. 335, pp. 765-774, 1990.
59. Cohn, J. N., S. O. Goldstein, B. H. Greenberg, et al., ``A
Dose-Dependent Increase in Mortality With Vesnarinone Among Patients
With Severe Heart Failure,'' Vesnarinone Trial Investigators, New
England Journal of Medicine, vol. 339, pp. 1810-1816, 1998.
60. Hampton, J. R., D. J. van Veldhuisen, F. X. Kleber, et al.,
``Randomised Study of Effect of Ibopamine on Survival in Patients
With Advanced Severe Heart Failure,'' Second Prospective Randomised
Study of Ibopamine on Mortality and Efficacy (PRIME II)
Investigators, Lancet, vol. 349. pp. 971-977, 1997.
61. Cuffe, M. S., R. M. Califf, K. F. Adams, Jr., et al.,
``Short-Term Intravenous Milrinone for Acute Exacerbation of Chronic
Heart Failure: A Randomized Controlled Trial,'' Journal of the
American Medical Association, vol. 287, pp. 1541-1547, 2002.
62. Sears, M. R., ``Adverse Effects of Aeta-Agonists,'' Journal
of Allergy and Clinical Immunology, vol. 110, pp. S322-S328, 2002.
63. Wit, A. L., B. F. Hoffman, and M. R. Rosen,
``Electrophysiology and Pharmacology of Cardiac Arrhythmias, IX.
Cardiac Electrophysiologic Effects of Beta Adrenergic Receptor
Stimulation and Blockade. Part C,'' American Heart Journal, vol. 90,
pp. 795-803, 1975.
64. Mahaffey, K. W., J. A. Puma, N. A. Barbagelata, et al.,
``Adenosine as an Adjunct to Thrombolytic Therapy for Acute
Myocardial Infarction: Results of a Multicenter, Randomized,
Placebo-Controlled Trial: The Acute Myocardial Infarction Study of
Adenosine (AMISTAD) Trial,'' Journal of the American College of
Cardiology, vol. 34, pp. 1711-1720, 1999.
65. Kenchaiah, S., J. C. Evans, D. Levy, et al., ``Obesity and
the Risk of Heart Failure,'' New England Journal of Medicine, vol.
347, pp. 305-313, 2002.
66. National Institutes of Health, National Heart, Lung, and
Blood Institute, ``Clinical Guidelines on the Identification,
Evaluation, and Treatment of Overweight and Obesity in Adults: The
Evidence Report,'' NIH Publication No. 98-4083, pp. 1-262 (http://www.nhlbi.nih.gov/guidelines/obesity/ob_gdlns.htm), 1998.
67. Astrup, A., L.Breum, S. Toubro, et al., ``The Effect and
Safety of an Ephedrine/Caffeine Compound Compared to Ephedrine,
Caffeine and Placebo in Obese Subjects on an Energy Restricted Diet,
A Double Blind Trial,'' International Journal of Obesity and Related
Metabolic Disorders, vol. 16, pp. 269-277, 1992.
68. Toubro, S., A. V. Astrup, L. Breu, F. Quaade, ``Safety and
Efficacy of Long-Term Treatment With Ephedrine, Caffeine and an
Ephedrine/Caffeine Mixture,'' International Journal of Obesity and
Related Metabolic Disorders, vol. 17, suppl. 1, pp. S69-S72, 1993.
69. Quaade, F., A. Astrup, L. Breum, et al., ``The effect of an
Ephedrine/Caffeine Combination As a Supplement to a Weight-Reducing
Diet. A Randomized, Placebo-Controlled, Double-Blind Trial,'' Ugesk
Laeger, vol. 154, pp. 1258-1263, 1992.
70. Toubro, S., A. Astrup, L. Breum, et al., ``The Acute and
Chronic Effects of Ephedrine/Caffeine Mixtures on Energy Expenditure
and Glucose Metabolism in Humans,'' International Journal of Obesity
and Related Metabolic Disorders, vol. 17, suppl. 3, pp. S73-S77,
1993.
71. Astrup, A., C. Lundsgaard, J. Madsen, et al., ``Enhanced
Thermogenic Responsiveness During Chronic Ephedrine Treatment in
Man,'' American Journal Clinical Nutrition, vol. 42, pp. 83-94,
1985.
72. Astrup, A. and S. Toubro, ``Thermogenic, Metabolic, and
Cardiovascular Responses to Ephedrine and Caffeine in Man,''
International Journal of Obesity and Related Metabolic Disorders,
vol. 17, suppl. 1, pp. S41-S43, 1993.
73. Astrup, A., S. Toubro, S. Cannon, P. Hein, et al.,
``Caffeine: A Double-Blind, Placebo-Controlled Study of its
Thermogenic, Metabolic, and Cardiovascular Effects in Healthy
Volunteers,'' American Journal Clinical Nutrition, vol. 51, pp. 759-
767, 1990.
74. Breum, L., J. K. Pedersen, F. Ahlstrom, et al., ``Comparison
of an Ephedrine/Caffeine Combination and Dexfenfluramine in the
Treatment of Obesity. A Double-Blind Multi-Centre Trial in Ggeneral
Practice,'' International Journal of Obesity and Related Metabolic
Disorders, vol. 18, pp. 99-103, 1994.
75. Pasquali, R., G. Baraldi, M. P. Cesari, et al., ``A
Controlled Trial Using Ephedrine in the Treatment of Obesity,''
International Journal of Obesity and Related Metabolic Disorders,
vol. 9, pp. 93-98, 1985.
76. Pasquali, R., M. P. Cesari, N. Melchionda, et al., ``Does
Ephedrine Promote Weight Loss in Low-Energy-Adapted Obese Women?,''
International Journal of Obesity and Related Metabolic Disorders,
vol. 11, pp. 163-168, 1987.
77. Pasquali, R., F. Casimirri, N. Melchionda, et al., ``Effects
of Chronic Administration of Ephedrine During Very-Low-Calorie Diets
on Energy Expenditure, Protein Metabolism and Hormone Levels in
Obese Subjects,'' Clinical Science (London), vol. 82, pp. 85-92,
1992.
78. Krieger, D. R., Daly, P. A., Dulloo, A. G., et al.,
``Ephedrine, Caffeine and Aspirin Promote Weight Loss in Obese
Subjects,'' Transactions of the Association of American Physicians,
vol. 103, pp. 307-312, 1990.
79. Daly, P. A., D. R. Krieger, A. G. Dulloo, et al.,
``Ephedrine, Caffeine and Aspirin: Safety and Efficacy for Treatment
of Human Obesity,'' International Journal of Obesity and Related
Metabolic Disorders, vol. 17, suppl. 1, pp. S73-S78, 1993.
80. Horton, T. J. and C. A. Geissler, ``Aspirin Potentiates the
Eeffect of Ephedrine on the Thermogenic Response to a Meal in Obese
But Not Lean Women,'' International Journal of Obesity and Related
Metabolic Disorders, vol. 15, pp. 359-366, 1991.
80a. Food and Drug Administration, Memorandum, Beaston, P.,
M.D., Ph.D., ``Review of Boozer-Daly Article for Ephedrine and
Caffeine,'' January 10, 2003, amended February 2, 2004.
81. Food and Drug Administration, Memorandum: Evaluation of
Scientific References Considered by RAND, February 2, 2004.
81a. Friedman, L. M., C. D. Furberg, and D. L. DeMets,
Fundamentals of Clinical Trials, 3rd ed., Springer, New York, 1998.
81b. Ingelfinger, J.A., F. Mosteller, L. A. Thibodeau, et al.,
3rd ed., Biostatistics in Clinical Medicine, McGraw-Hill, Inc., New
York, 1994.
[[Page 6852]]
81c. Dawson, B. and R.G. Trapp, Basic and Clinical
Biostatistics, 3rd ed., Lange Medical Books/McGraw-Hill, New York,
2001.
82. Niemann R.A. and M.L. Gay, ``Determination of Ephedrine
Alkaloids and Synephrine in Dietary Supplements by Column-Switching
Cation Exchange High-Performance Liquid Chromatography with
Scanning-Wavelength Ultraviolet and Fluorescence Detection,''
Journal of Agricultural and Food Chemistry, vol. 51, pp. 5630-5638,
2003.
83. Chua, S. S. and S. I. Benrimoj, ``Non-Prescription
Sympathomimetic Agents and Hypertension,'' Medical Toxicology and
Adverse Drug Experience, vol. 3, pp. 387-417, 1988.
84. Inchiosa, M. A., ``Dose-Related Cardiovascular Effects of
Ephedrine in Humans,'' Report prepared for the Food and Drug
Administration, July 6, 1999.
85. Inchiosa, M. A., ``Bioavailability of Ephedrine Alkaloids,''
Report prepared for the Food and Drug Administration, June 14, 1999.
86. Food and Drug Administration, Food Advisory Committee on
Dietary Supplements Containing Ephedrine Alkaloids, Meeting
Transcript, II, pp. 136, 137, 203, 205, 221, 225, 227, 229-230, 236-
237, 239-240, August 27-28, 1996.
87. Food and Drug Administration, Food Advisory Committee on
Dietary Supplements Containing Ephedrine Alkaloids, Meeting
Transcript, II, pp. 137, 233, 237, 240, 272, and 277, August 27-28,
1996.
88. Kerin, K. J., Dietary Supplements Containing Ephedrine
Alkaloids, Comments from Circadian Technologies, Inc., FDA Docket
No. 1995N-0304, c3923:1-3, 2003.
89. Lee, M. K., B. W. Cheng, C. T. Che, et al., ``Cytotoxicity
Assessment of Ma-huang (Ephedra) Under Different Conditions of
Preparation,'' Toxicological Sciences, vol. 56, pp. 424-430, 2000.
90. Hikino, H., K. Ogata, C. Konno, et al., ``Hypotensive
Actions of Ephedradines, Macrocyclic Spermine Alkaloids of Ephedra
Roots,'' Planta Medica, vol. 48, pp. 290-293, 1983.
91. Minematsu, S., Y. Kobayashi, N. Kobayashi, et al., ``Acute
Ephedrae Herba and ephedrine poisoning in mice,'' Japanese Journal
of Toxicology, vol. 4, pp. 143-149, 1991.
92. Lee, M. K., Y. H. Wong, C. T. Che, et al., ``Adrenergic
Agonistic Effects and Cyto-Toxicity of Chinese Ephedra (Ma-Huang)
Used for Weight Reduction,'' Toxicological Sciences, The
Toxicologist, vol. 48, p. 58, abstract no. 272, 1999.
93. CANTOX Health Sciences International, ``Safety Assessment
and Determination of a Tolerable Upper Limit for Ephedra,'' pp. 1-
273, Docket No. 2000N-1200, Sup2 (http://www.crnusa.org/cantoxreportindex.html), 2000.
94. Ad Hoc Committee Reports, On the Safety of Ma Huang (1995,
1996), On the Safety of Dietary Supplements (1997), FDA Docket Nos.
1995N-0304, RPT 1, vols. 36-38; 1995N-0304, C57, vol. 76; 1995N-
0304, C3391, vol. 280.
95. Ephedra Education Council Expert Panel Report, Docket No.
2000N-1200, C30, vol. 70, 2000.
96. Boozer, C. N., P. A. Daly, D. Blanchard, et al., ``Herbal
Ephedra/Caffeine for Weight Loss: A 6-Month Safety and Efficacy
Trial,'' North American Association for the Study of Obesity, 2000.
97. Dulfano, M. J. and P. Glass, ``Evaluation of a New
B2 Adrenergic Receptor Stimulant, Terbutaline, in
Bronchial Asthma. II. Oral Comparison With Ephedrine,'' Current
Therapeutic Research, vol. 15, pp. 150-157, 1973.
98. Tashkin, D. P., R. Meth, D. H. Simmons, et al., ``Double-
Blind Comparison of Acute Bronchial and Cardiovascular Effects of
Oral Terbutaline and Ephedrine,'' Chest, vol. 68, pp. 155-161, 1975.
99. Brown, S. D., Jr., and F. J. Landry, ``Recognizing,
Reporting, and Reducing Adverse Drug Reactions,'' Southern Medical
Journal, vol. 94, pp. 370-373, 2001.
100. Lazarou, J., B. H. Pomeranz, and P. N. Corey, ``Incidence
of Adverse Drug Reactions in Hospitalized Patients: A Meta-Analysis
of Prospective Studies,'' Journal of the American Medical
Association, vol. 279, pp. 1200-1205, 1998.
101. American Herbal Products Association, Code of Ethics and
Business Conduct, pp. 1-9, July 2002.
102. Lake, C. R. and R. S. Quirk, ``CNS Stimulants and the Look-
Alike Drugs,'' Psychiatric Clinics of North America, vol. 7, pp.
689-701, 1984.
103. Furuya, I. and S. Watanabe, ``Discriminative Stimulus
Properties of Ephedra Herb (Ephedra Sinica) in Rats,'' Yakubutsu
Seishin Kodo (Japanese Journal of Psychopharmacology), vol. 13, pp.
33-38, 1993.
104. Tinsley, J. A. and D. D. Watkins, ``Over-the-Counter
Stimulants: Abuse and Addiction,'' Mayo Clinic Proceedings, vol. 73,
pp. 977-982, 1998.
105. Ephedra Sinica, In Gruenwald, J., T. Brendler, and C.
Jaenicke, eds., PDR for Herbal Medicines, 1st ed., Montvale: Medical
Economics Co., Inc., pp. 826-827, 1998.
106. The Complete German Commission E Monographs, Therapeutic
Guide to Herbal Medicines (English Translation), Blumenthal, M., W.
R. Busse, A. Goldberg, et al., pp. 125-126, 1998.
107. Huang, K. C., Antiasthmatic Herbs, Chapter in The
Pharmacology of Chinese Herbs, pp. 229-232, Boca Raton, CRC Press,
1993.
108. Hsu, H. Y., Y. P. Chen, S. J. Shen, et. al, Ephedrae Herba
(Ephedra, Ma-Huang), Oriental Materica Medica: A Concise Guide, pp.
52-53, Long Beach, CA, Oriental Healing Arts Institute, 1986.
109. Food and Drug Administration, Clinical Research and Review
Staff: Evaluation of Adverse Events Reported With the Use of
Ephedrine Alkaloid-containing Dietary Supplements (June 1, 1997,
through March 31, 1999), 2000.
110. Food and Drug Administration, Chen, M. and C. B.Karwoski,
Office of Post Market Drug Risk Assessment Postmarketing Safety
Review, ``Evaluation of Adverse Event Reports Associated With
Ephedrine Alkaloid-Containing Dietary Supplements,'' 2000.
111. Woosley, R. L., ``Summary of Analysis of Adverse Event
Reports for Dietary Supplements Containing Ephedrine Alkaloids,''
Report Prepared for the Food and Drug Administration, 1999.
112. Benowitz, N. L., ``Review of Adverse Reaction Reports
Involving Ephedrine-Containing Herbal Products,'' Report Prepared
for the Food and Drug Administration, 2000.
113. Ricaurte, G. A., ``Review of FDA Neuropsychiatric/central
nervous system adverse events related to ephedrine-containing
dietary supplements,'' Report Prepared for the Food and Drug
Administration, 2000.
114. Stoll, A. L., ``FDA Ephedrine Adverse Event Case Review:
Summary,'' 1999.
115. Woosley, R. L., ``FDA Addendum: Summary of Analysis of
Adverse Event Reports for Dietary Supplements Containing Ephedrine
Alkaloids,'' Report Prepared for the Food and Drug Administration,
2000.
116. Food and Drug Administration, Love, L. A., ``FDA Review of
the Published Literature on the Physiological, Pharmacological and
Toxic Effects of Ephedrine Alkaloids,'' 2000.
117. U.S. House of Representatives, Committee on Government
Reform, Minority Staff Report, ``Adverse Event Reports from
Metabolife,'' 2002.
118. Shekelle, P. G., E-Mail Response to Questions on the RAND
Report on Ephedra, April 7, 2003.
119. Shekelle, P. G., M. Maglione, and S. C. Morton,
``Preponderance of Evidence. Judging What to Do About Ephedra,''
RAND Review, vol. 27, pp. 16-21, (http://www.rand.org/publications/randreview/issues/spring2003/evidence.html), 2003, accessed in
January 2004.
120. ``Danish Medicines Agency: Letigen[reg] has been
suspended,'' Danish Medicines Agency 2002 (http://www.laegemiddelstyrelsen.dk/en/news/letigen_6_en.asp), accessed as
of March 3, 2003. (To obtain a paper copy see the FOR FURTHER
INFORMATION CONTACT section of this document.)
121. ``Adverse Reactions Letigen,'' Danish Medicines Agency,
2002 Company Report, 18, 2002.
122. Walker, A. M., ``The Relation Between Voluntary
Notification and Material Risk in Dietary Supplement Safety,''
Report prepared for the Food and Drug Administration, 2000.
123. Food and Drug Administration, Letter from Melinda Plaisier
to Congressman Henry Waxman (CA) dated July 11, 2001 and Tab C
(preliminary evaluation of data from American Association of Poison
Control Centers) enclosed with the letter, 2001.
124. U.S. General Accounting Office (GAO), GAO Report, ``Dietary
Supplements: Uncertainties in Analyses Underlying FDA's Proposed
Rule on Ephedrine Alkaloids,'' GAO, 1999, submitted to Docket No.
2000N-1200, BKG 1, vol. 32.
125. Boozer, C. N., J. A. Nasser, S. B. Heymsfield, et al., ``An
Herbal Supplement Containing Ma Huang-Guarana For Weight Loss: A
Randomized, Double-Blind Ttrial,'' International Journal of Obesity
and Related Metabolic Disorders, vol. 25, pp. 316-324, 2001.
126. National Institutes of Health, National Heart, Lung, and
Blood Institute, Third Report of the Expert Panel on Detection,
[[Page 6853]]
Evaluation, and Treatment of High Blood Cholesterol in Adults (Adult
Treatment Panel III) Final Report, NIH Publication No. 02-5215
(http://www.nhlbi.nih.gov/guidelines/cholesterol), 2002.
127. Bray, G. A. and F. L. Greenway, ``Current and Potential
Drugs for Treatment of Obesity,'' Endocrine Reviews, vol. 20, pp.
805-875, 1999.
128. Yanovski, S. Z. and J. A. Yanovski, ``Obesity,'' New
England Journal of Medicine, vol. 346, pp. 591-602, 2002.
129. Food and Drug Administration, Division of Metabolic and
Endocrine Drug Products, FDA Draft Guidance for the Clinical
Evaluation of Weight-Control Drugs, 1996.
130. International Committee on Harmonization (ICH): Guidance
for Industry, S7A Safety Pharmacology Studies for Human
Pharmaceuticals, ICH, pp. 1-11 (http://www.fda.gov/cder/guidance/4461fnl.pdf), 2001.
131. Filozof, C., C. Gonzalez, and E. Hofman, ``The Effect
Ephedrine Plus Caffeine After a 4-week Portion-Controlled Diet,''
Ninth International Congress on Obesity, Abstract 591, Ninth
International Congress on Obesity, vol. 26, suppl. 1, p. S156, 2002,
August 2002.
132. Food and Drug Administration, ``Evidence On The Safety And
Effectiveness Of Ephedra: Implications For Regulation,'' (http://www.fda.gov/bbs/topics/NEWS/ephedra/whitepaper.html), 2003.
133. Boerth, J. M., C. F. Caley, ``Possible Case of Mania
Associated With Ma-huang,'' Pharmacotherapy, vol. 23, pp. 380-383,
2003.
134. Samenuk, D., M. S. Link, M. K. Homoud, et al., ``Adverse
Cardiovascular Events Temporally Associated With Ma-huang, an Herbal
Source of Ephedrine,'' Mayo Clinic Proceedings, vol. 77, pp. 12-16,
2002.
135. Bent, S., T. N. Tiedt, M. C. Odden, et al., ``The Relative
Safety of Ephedra Compared With Other Herbal Products,'' Annals of
Internal Medicine, vol. 138, pp. 468-471, 2003.
136. Morgenstern, L. B., C. M. Viscoli, W. N. Kernan, et al.,
``Use of Ephedra-Containing Products and Risk for Hemorrhagic
Stroke,'' Neurology, vol. 60, pp. 132-135, 2003.
137. Food and Drug Administration, Policy and Guidance Handbook
For FDA Advisory Committees, pp. 113-114, 1994.
138. Food and Drug Administration, Nightingale, S. L., FDA
``Dear Colleague'' Letter Regarding the Research on Eosinophilia-
Myalgia Syndrome and Current Regulatory Status of L-Tryptophan, 1992
(http://www.cfsan.fda.gov/dms/ds-ltr3.html).
139. Chyka, P. A., McCommon, S. W., ``Reporting of adverse drug
reactions by poison control centres in the U.S.,'' Drug Safety, vol.
23, pp. 87-93, 2000.
140. Office of Management and Budget: Regulatory Analysis,
Circular A-4 (http://www.whitehouse.gov/omb/circulars/a004/a-4.html), 2003.
141. Shekelle P, S. Morton, M. Maglione, et al., ``Ephedra and
Ephedrine for Weight Loss and Athletic Performance Enhancement:
Clinical Efficacy and Side Effects. Evidence Report/Technology
Assessment,'' No. 76 (Prepared by Southern California Evidence-Based
Practice Center, RAND, under Contract No. 290-97-0001, Task Order
No. 9), AHRQ Publication No. 03-E022, Rockville, MD, Agency for
Healthcare Research and Quality, Table 23, 2003.
142. Centers for Disease Control and Prevention, ``Life
Expectancy'' (Data are for U.S. in 2001). National Center for Health
Statistics, Centers for Disease Control and Prevention, U. S.
Department of Health and Human Services (http://www.cdc.gov/nchs/fastats/lifexpec.htm), accessed on December 9, 2003.
143. Derdeyn, C. P., Powers, and W. J., ``Cost-Effectiveness of
Screening for Asymptomatic Carotid Atherosclerotic Disease,''
Stroke, vol. 27, pp. 1944-1950, 1996.
144. Agency for Healthcare Research and Quality (AHRQ),
Healthcare Cost and Utilization Project, 2001 National Statistics,
The ``National Bill'' for Conditions Treated in Hospitals (Top 100
Clinical Classifications Software (CCS) Diagnoses), (http://www.hcup.ahrq.gov/HCUPnet.asp), 2001 .
145. GNC Plans to Drop Ephedra Private-Label Supplements, FDC
Reports--Tan Sheet, pp. 11-12, 1996.
146. Redfearn, S., ``Ephedra Products Thin Out: Braced for the
Worst, Makers Sell New Herbs,'' The Washington Post, HE01, 2003
(http://www.washingtonpost.com/wp-dyn/articles/A52270-2003Jan13.html).
147. Letter Correction: The Relative Safety of Ephedra Compared
with Other Herbal Products, Annals of Internal Medicine, vol. 138,
pp. 1012, 2003 (http://www.annals.org/cgi/content/full/138/12/1012),
accessed on December 11, 2003.
148. Dickinson, A., Letter, ``The Relative Safety of Ephedra
Compared With Other Herbal Products,'' Annals of Internal Medicine,
vol. 139, pp. 385-386 (http://www.annals.org/cgi/content/full/139/5_Part_1/385-b) 2003, accessed on September 2, 2003.
149. Nutrition Business Journal, 2002 Facts and Stats about
Dietary Supplements (obtained from http://www.nnfa.org/facts), 2002.
150. Kaufman, D. W., Kelly, J. P., Mitchell, A. A., ``Use of
Ephedra-Containing Products in the U.S. Population,'' Data from the
Slone Survey, FDA Docket No. 1995N-0304, emc126, vol. 297, 2003.
151. Research Triangle Institute, ``Cost of Reformulating Foods
and Cosmetics,'' Final Report, Report Prepared for the Food and Drug
Administration, RTI Project Number 08184.003, 2002.
152. Gugliotta, G., ``Ephedra Lawsuits Show Big Increase,'' The
Washington Post, July 23, 2000, (http://www.washingtonpost.com/ac2/wp-dyn?
pagename=article&node=&contentId=
A25415-2000Jul21¬Found=true), accessed on February 3, 2004.
153. Gugliotta, G., ``Woman Wins $13.3 Million Against Dietary
Company,'' The Washington Post, February 2, 2001 (http://www.washingtonpost.com/ac2/wp-dyn?
pagename=article&node=&contentId=
A40923-2001Feb7¬Found=true), accessed on February 3, 2004.
154. Prevention Magazine, ``Consumer Use of Dietary
Supplements,'' 1999.
155. National Consumers League, National Consumers League's
Survey of Consumers Using Over-the-Counter (OTC) Medications, 2003.
156. Geiger, C. J., ``Health Claims: History, Current Regulatory
Status, and Consumer Research,'' Journal of the American Dietetic
Association, vol. 98, pp. 1312-1322, 1998.
157. MacKinnon, D. P., L. Nohre, J. Cheong, et al.,
``Longitudinal Relationship Between the Alcohol Warning Label and
Alcohol Consumption,'' Journal of Studies on Alcohol, vol. 62, pp.
221-227, 2001.
158. Food and Drug Administration, Memorandum: Label Analysis
for Dietary Supplements Containing Ephedrine Alkaloids, January 28,
2004.
159. Research Triangle Institute, Dietary Supplement Sales
Information, Supplement to the Final Report, Report Prepared for the
Food and Drug Administration, Contract No. 223-96-2290 Task Order
4:1999 (http://www.foodriskclearinghouse.umd.edu/dspd.htm), accessed
on October 1999.
160. Research Triangle Institute, Survey of Manufacturing
Practices in the Dietary Supplement Industry. Final Report, Report
prepared for the Food and Drug Administration, RTI Project Number
6673-6:2000 (http://www.foodriskclearinghouse.umd.edu/smpds.htm),
accessed on May 17, 2000.
161. Office of Management and Budget, Guidance for Implementing
E.O. 13132, ``Federalism,'' October 28, 1999.
List of Subjects in 21 CFR Part 119
Dietary ingredients, Dietary supplements, Foods.
0
Therefore, under the Federal Food, Drug, and Cosmetic Act and under
authority delegated to the Commissioner of Food and Drugs, 21 CFR part
119 is added as follows:
0
1. Part 119 consisting of Sec. 119.1 is added to read as follows:
PART 119--DIETARY SUPPLEMENTS THAT PRESENT A SIGNIFICANT OR
UNREASONABLE RISK
Sec. 119.1 Dietary supplements containing ephedrine alkaloids.
Dietary supplements containing ephedrine alkaloids present an
unreasonable risk of illness or injury under conditions of use
recommended or suggested in the labeling, or if no conditions of use
are recommended or suggested in the labeling, under ordinary conditions
of use. Therefore, dietary supplements containing ephedrine alkaloids
are adulterated under section 402(f)(1)(A) of the Federal Food, Drug,
and Cosmetic Act.
Authority: 21 U.S.C. 321, 342, 343, 371.
[[Page 6854]]
Dated: January 28, 2004.
Mark B. McClellan,
Commissioner of Food and Drugs.
Dated: February 4, 2004.
Tommy G. Thompson,
Secretary of Health and Human Services.
[FR Doc. 04-2912 Filed 2-6-04; 2:00 pm]
BILLING CODE 4160-01-S