[House Hearing, 106 Congress]
[From the U.S. Government Publishing Office]
SECURING THE HEALTH OF THE AMERICAN PEOPLE
=======================================================================
HEARING
before the
SUBCOMMITTEE ON
HEALTH AND ENVIRONMENT
of the
COMMITTEE ON COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED SIXTH CONGRESS
SECOND SESSION
__________
SEPTEMBER 13, 2000
__________
Serial No. 106-163
__________
Printed for the use of the Committee on Commerce
U.S. GOVERNMENT PRINTING OFFICE
67-114CC WASHINGTON : 2000
------------------------------------------------------------------------------
For sale by the Superintendent of Documents, Congressional Sales Office
U.S. Government Printing Office, Washington, DC 20402
COMMITTEE ON COMMERCE
TOM BLILEY, Virginia, Chairman
W.J. ``BILLY'' TAUZIN, Louisiana JOHN D. DINGELL, Michigan
MICHAEL G. OXLEY, Ohio HENRY A. WAXMAN, California
MICHAEL BILIRAKIS, Florida EDWARD J. MARKEY, Massachusetts
JOE BARTON, Texas RALPH M. HALL, Texas
FRED UPTON, Michigan RICK BOUCHER, Virginia
CLIFF STEARNS, Florida EDOLPHUS TOWNS, New York
PAUL E. GILLMOR, Ohio FRANK PALLONE, Jr., New Jersey
Vice Chairman SHERROD BROWN, Ohio
JAMES C. GREENWOOD, Pennsylvania BART GORDON, Tennessee
CHRISTOPHER COX, California PETER DEUTSCH, Florida
NATHAN DEAL, Georgia BOBBY L. RUSH, Illinois
STEVE LARGENT, Oklahoma ANNA G. ESHOO, California
RICHARD BURR, North Carolina RON KLINK, Pennsylvania
BRIAN P. BILBRAY, California BART STUPAK, Michigan
ED WHITFIELD, Kentucky ELIOT L. ENGEL, New York
GREG GANSKE, Iowa TOM SAWYER, Ohio
CHARLIE NORWOOD, Georgia ALBERT R. WYNN, Maryland
TOM A. COBURN, Oklahoma GENE GREEN, Texas
RICK LAZIO, New York KAREN McCARTHY, Missouri
BARBARA CUBIN, Wyoming TED STRICKLAND, Ohio
JAMES E. ROGAN, California DIANA DeGETTE, Colorado
JOHN SHIMKUS, Illinois THOMAS M. BARRETT, Wisconsin
HEATHER WILSON, New Mexico BILL LUTHER, Minnesota
JOHN B. SHADEGG, Arizona LOIS CAPPS, California
CHARLES W. ``CHIP'' PICKERING,
Mississippi
VITO FOSSELLA, New York
ROY BLUNT, Missouri
ED BRYANT, Tennessee
ROBERT L. EHRLICH, Jr., Maryland
James E. Derderian, Chief of Staff
James D. Barnette, General Counsel
Reid P.F. Stuntz, Minority Staff Director and Chief Counsel
______
Subcommittee on Health and Environment
MICHAEL BILIRAKIS, Florida, Chairman
FRED UPTON, Michigan SHERROD BROWN, Ohio
CLIFF STEARNS, Florida HENRY A. WAXMAN, California
JAMES C. GREENWOOD, Pennsylvania FRANK PALLONE, Jr., New Jersey
NATHAN DEAL, Georgia PETER DEUTSCH, Florida
RICHARD BURR, North Carolina BART STUPAK, Michigan
BRIAN P. BILBRAY, California GENE GREEN, Texas
ED WHITFIELD, Kentucky TED STRICKLAND, Ohio
GREG GANSKE, Iowa DIANA DeGETTE, Colorado
CHARLIE NORWOOD, Georgia THOMAS M. BARRETT, Wisconsin
TOM A. COBURN, Oklahoma LOIS CAPPS, California
Vice Chairman RALPH M. HALL, Texas
RICK LAZIO, New York EDOLPHUS TOWNS, New York
BARBARA CUBIN, Wyoming ANNA G. ESHOO, California
JOHN B. SHADEGG, Arizona JOHN D. DINGELL, Michigan,
CHARLES W. ``CHIP'' PICKERING, (Ex Officio)
Mississippi
ED BRYANT, Tennessee
TOM BLILEY, Virginia,
(Ex Officio)
(ii)
C O N T E N T S
__________
Page
Testimony of:
Bennett, Catherine P., Chair, Board of Directors, Cancer
Research Foundation of America............................. 117
Brady, Robert, Partner, Hogan & Hartson, on behalf of Biogen,
Inc........................................................ 106
Bryan, R. Nick, Professor and Chairman of Radiology, Hospital
of University of Pennsylvania.............................. 87
Burton, Hon. Dan, a Representative in Congress from the State
of Indiana................................................. 101
Dunnick, N. Reed, Professor and Chair, Department of
Radiology, University of Michigan Health System............ 77
Fraser, Tomiko, National Spokesperson, Lupus Foundation of
America, Inc............................................... 84
Gekas, Hon. George W., a Representative in Congress from the
State of Pennsylvania...................................... 70
Hillman, Bruce J., Professor and Chair, Department of
Radiology, University of Virginia.......................... 80
Lang, Thomas A., Senior Vice President, Strategic Product
Development, Serono Laboratories, Inc...................... 112
Meek, Hon. Carrie P., a Representative in Congress from the
State of Florida........................................... 72
Meyers, Abbey, President, National Organization for Rare
Disorders.................................................. 110
Navarro, James, father of Thomas Navarro..................... 103
Wirth, Dyann, Professor, Department of Immunology and
Infectious Diseases, Harvard School of Public Health....... 73
Material submitted for the record by:
Brady, Robert, Partner, Hogan & Hartson, Counsel to Biogen,
Inc., letter dated September 21, 2000, enclosing material
for the record............................................. 138
Bryan, R. Nick, Professor and Chairman of Radiology, Hospital
of University of Pennsylvania, letter dated November 20,
2000, enclosing response for the record.................... 136
Department of Health and Human Services, additional testimony
for the record............................................. 125
Fraser, Tomiko, National Spokesperson, Lupus Foundation of
America, Inc., responses for the record.................... 127
Hillman, Bruce J., Professor and Chair, Department of
Radiology, University of Virginia, responses for the record 134
Meyers, Abbey, President, National Organization for Rare
Disorders, responses for the record........................ 131
Navarro, James, responses for the record..................... 128
(iii)
SECURING THE HEALTH OF THE AMERICAN PEOPLE
----------
WEDNESDAY, SEPTEMBER 13, 2000
House of Representatives,
Committee on Commerce,
Subcommittee on Health and Environment,
Washington, DC.
The subcommittee met, pursuant to notice, at 10:10 a.m., in
room 2123, Rayburn House Office Building, Hon. Michael
Bilirakis (chairman) presiding.
Members present: Representatives Bilirakis, Upton, Stearns,
Burr, Ganske, Cubin, Bryant, Brown, Waxman, Pallone, Stupak,
Green, Barrett and Capps.
Staff present: Marc Wheat, majority counsel; Nandan
Kenkeremath, majority counsel; Kristi Gillis, legislative
clerk; and John Ford, minority counsel.
Mr. Bilirakis. The hearing will come to order. My thanks to
all of the witnesses who have taken the time to testify before
this subcommittee. This hearing will address, as you know,
several pieces of legislation designed to improve the quality
of health care.
Today we will hear about H.R. 2399, the National Commission
for the New National Goal: The Advancement of Global Health
Act. This legislation introduced by my friend, Representative
George Gekas of Pennsylvania, would establish a commission to
recommend a national strategy to coordinate public and private
sector efforts toward the global eradication of disease. The
Commission would specifically address how the United States may
assist in the global control of infectious diseases through the
development of vaccines and the sharing of health research
information on the Internet.
Also on the first panel we will hear testimony on H.R.
1795, the National Institute of Biomedical Imaging and
Engineering Establishment Act. This legislation introduced by
Representatives Richard Burr and Anna Eshoo, members of this
panel, would establish a National Institute of Biomedical
Imaging and Engineering at the National Institutes of Health.
Finally, our first panel will address legislation
introduced my Representative Carrie Meek, H.R. 762, the lupus
research and care amendments of 1999, which expands immediate
lupus research activities and authorizes the Secretary of
Health and Human Services to make grants for the delivery of
essential services to individuals with lupus and their
families. Congresswoman Meek has been a tireless proponent of
this legislation, and I would also be remiss if I failed to
mention the advocacy efforts of Sandy Freer from my area of
Florida.
I discussed this legislation at the full committee markup
of the minority health disparities bill, and I look forward not
only to the testimony today, but to advancing this very
important legislation.
Our second panel will include testimony on H.R. 4242, the
Orphan Drug Innovation Act. This bill amends the Federal Food,
Drug and Cosmetic Act to allow sponsors for a drug for a rare
disease or condition, so-called orphan drugs, to ask the
Secretary of Health and Human Services to provide written
recommendations for the nonclinical and clinical investigations
which must be conducted with a drug before it may be approved
as a new drug or licensed as a biological product. It also
authorizes the Secretary to provide recommendations on whether
such a drug is for a disease or condition which is rare in the
United States.
I would also like to welcome Mr. Jim Navarro, a concerned
parent, to our second panel. He will be discussing H.R. 3677,
the Thomas Navarro FDA Patient Rights Act. This bill is named
after his son, who was 4 years old when he was diagnosed with a
form of cancer known as medulloblastoma. After researching
their options, the family decided that the best course of
action was through a nontoxic FDA-approved clinical trial. The
FDA denied Thomas access to this clinical trial because he had
not first undergone and failed treatment by chemotherapy and
radiation, which can have, as we all realize, I think, serious
side effects for children of that age.
H.R. 3677 precludes the FDA from establishing a clinical
hold on the basis that there is a comparable or satisfactory
alternative therapy available if a patient is aware of the
other therapy and aware of the risk associated with the
investigational drug, yet still chooses to receive the
treatment.
Finally, in honor of Childhood Cancer Month, we will hear
testimony in support of a resolution sponsored by
Representative Deborah Pryce on the importance of researching
childhood cancer. I think all of us remember that
Representative Pryce lost their little daughter a few months
ago. The testimony and the resolution focus on the importance
of promoting awareness of and expanding research on childhood
cancers. The resolution would encourage medical trainees to
enter the field of pediatric oncology, encourage the
development of drugs and biologicals to treat pediatric
cancers, and promote medical curricula to improve pain
management. The resolution would also support policies that
reduce barriers to participation in clinical trials.
I welcome all of our witnesses, including our colleagues,
to this hearing. And to cover as much ground as possible, I
would ask members to limit their opening statements. Under the
rules, I can limit opening statements other than chairman and
ranking member to 3 minutes, and I would appreciate it if you
would hold them to within that period of time.
And I would also note that some Members of Congress who are
not members of the subcommittee will also give brief
introductory remarks regarding their legislation and introduce
their witnesses. And I just hope that this hearing will shed
light on a number of important public health issues and that we
can devote most of the time to our witnesses.
With that I yield to the ranking member, Mr. Brown.
Mr. Brown. Thank you, Mr. Chairman. I would like to welcome
our witnesses also. Thank you for joining us. We have an
ambitious agenda this morning. Among the six bills, we will
consider two that would affect access to medications, H.R. 4242
and H.R. 3677.
In the case of both bills, it is likely that today's
hearing will not produce definitive answers. The issues
involved are simply too complex and the implications of any
actions we take too significant. However the questions these
bills seek to answer, the concerns they seek to address are
important, and it is valuable for the subcommittee to learn
about them.
I am also glad we will have an opportunity to review
legislation focussing on lupus and childhood cancers. Both
types of illnesses devastate and too often take young lives,
and neither has received the attention that they both deserve.
But in the interest of time, I want to focus my comments on
two of the other bills we will consider this morning, H.R. 2399
and H.R. 1795. I fully support the efforts of my colleague Mr.
Gekas to establish the improvement of global health as a
national priority, because global health should be a national
priority for several reasons.
Global health and the health of Americans are linked.
Americans travel abroad, the world travels here. Lethal
infectious diseases cross borders. The reemergence of
tuberculosis in the United States now in drug-resistant strains
that are difficult to treat is a grim reminder that when a
disease affects other nations, it is bound to affect us.
Tuberculosis last year killed more people than in any year in
history; 1,100 Indians die every day from tuberculosis.
A second reason that global health should be a national
priority is because the United States is a world leader. We are
the wealthiest Nation in the world, we are the most influential
force in the world. Our action sets a precedent; our inaction
sets a precedent. The United States is in a unique position to
save lives, to save families, to save children all over the
world.
An investment that is modest by U.S. standards literally
can save millions of lives, prevent millions of children from
being orphaned, prevent the social, economic and political
turmoil these killer diseases too often engender. It is an
opportunity and it is a privilege that our Nation should
embrace.
The other bill I want to mention briefly is H.R. 1795,
which would establish an Institute for Biomedical Imaging and
Engineering within the National Institutes of Health.
Unfortunately, my colleague Mrs. Eshoo couldn't be here this
morning, but I wanted to acknowledge her outstanding leadership
and the leadership of Mr. Burr on this measure. I extend a
special welcome to Dr. Dunnick from the University of Michigan
who is joining us to discuss 1794 at Mrs. Eshoo's request.
Adding an institute to NIH is a major step, but Mrs. Eshoo and
Mr. Burr make a compelling case for it.
Advancements in medical imaging technology have led to
stunning breakthroughs in the early detection and the treatment
of many diseases. By identifying these diseases early, and
without invasive procedures, patients are often able to receive
less painful, more therapeutic treatments that greatly improve
the likelihood that they will live longer and healthier lives.
Additionally, when treatment is initiated at the early stage of
a disease, doctors are able to rely on less expensive treatment
options that reduce overall health care costs.
I am glad we are taking time to access the benefit of
establishing an institute dedicated to equipment and techniques
that are indispensable to modern health care. I thank the
chairman.
Mr. Bilirakis. I thank the gentleman.
Mr. Upton.
Mr. Upton. Thank you, Mr. Chairman. I ask unanimous consent
to put my full statement in the record.
Mr. Bilirakis. Without objection, the opening statement of
all members of the subcommittee will be made a part of the
record.
Mr. Upton. I would just like to say one thing verbally
here. I am very glad that we are having these hearings, and I
am particularly happy that we are focusing on H.R. 672, the
Lupus Research and Care Amendments Act. Sadly, my sister-in-law
suffered from this disease and died last year from this
illness, so I know how important that it is to commit ourselves
to finding a cure for this devastating disease.
I commend my colleague from Florida for offering her bill,
and I am delighted to be a cosponsor, and I yield back.
Mr. Bilirakis. Mr. Waxman for an opening statement.
Mr. Waxman. Thank you, Mr. Chairman.
Let me begin by saying how pleased I am that H.R. 762, the
Lupus Research and Care Act, is receiving our attention. It is
an excellent bill and deserves our support. Lupus is also one
of the dozens of autoimmune diseases which are the subject of
my own bill to establish an Office of Autoimmune Diseases at
NIH, which has already passed this committee and the House.
But today I am principally concerned about H.R. 4242, the
Orphan Drug Innovation Act. As the author with Senator
Metzenbaum of the Orphan Drug Act, I care deeply about the
issues raised by this legislation.
For many years I have been very gratified by the success of
the Orphan Drug Act in stimulating the development of new
treatments for rare diseases. I am pleased that we will have
Abbey Meyers testify again before our subcommittee on this, and
that she is willing to come.
Market exclusivity is the foundation of the Orphan Drug
Act. But we created exceptions to that exclusivity in the law.
The bill before us would limit the scope of exclusivity granted
to drugs proven, ``clinically superior,'' to an existing orphan
drug; that is, drugs which are safer, more effective or provide
a major contribution to patient care. It has been alleged that
the bill is intended to anoint a winner in a commercial
dispute, but the bill raises an important and legitimate
question: What is the right balance between preserving
exclusivity, encouraging competition, and encouraging
affordable access to these lifesaving drugs?
As a first step to the best answer, I was looking forward
with great interest to the FDA's public clarification of its
policy toward clinical superiority. There are questions about
its consistency and its relationship to a generic approval
process for biotech drugs. This subcommittee requires some
clear answers. They have significant implications for patient
health and for access to reasonably priced breakthrough drugs.
But this morning I learned that the FDA has withdrawn its
witness and testimony. While this may be the result of late
notice to the administration, it nevertheless ensures today's
hearing will be of less assistance in guiding our
deliberations.
I would add that orphan drug policy deserves a hearing on
its own. There are 25 million Americans suffering from over
6,000 rare diseases. There is a great deal of unfinished
business for Congress. There is the question of how high a bar
clinical superiority should be. There are some multimillion-
dollar orphan drugs, drugs for which 7 years of exclusivity is
unjustified and serves only to boost prices and profits,
putting those lifesaving therapies out of the reach of many
patients. And just as important, there is the urgent need for
more orphan disease research at NIH and FDA.
I sincerely hope that we will have an opportunity early
next year to examine these issues in greater detail than will
be possible today.
Finally, Mr. Chairman, I would like to submit for the
record a series of articles and scientific reviews relating to
the alternative cancer treatment offered by Dr. Stanislav
Burzynski, the intended beneficiary of H.R. 3677, the Thomas
Navarro Patient Rights Act.
Mr. Bilirakis. Without objection, that will be the case.
[The information referred to follows:]
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Mr. Waxman. As ranking member of the Committee on
Government Reform, I have attended that committee's many
hearings to defend and endorse alternative medicine and dietary
supplements. But I am pleased that this subcommittee, which has
jurisdiction over these issues, has finally turned its
attention to them.
Developing new forms of cancer prevention, detection, and
treatment never ends. Ensuring patients have access and
accurate information about their treatments is also vital. So
we must keep an open mind about innovative or unconventional
approaches to cancer treatment and prevention.
But our first priority must be ensuring access to
treatments which are proven to be the best chances of curing
patients. And second, our priority must be rigorous testing of
new therapies, including complementary and alternative
therapies, to determine their safety and efficacy.
The problem with H.R. 3677 is that it undercuts these
goals. If research is under a clinical hold, you can be sure
that there are unresolved questions about the conduct of that
research. But this bill would shield such research from
scrutiny, discourage practitioners from cooperating in rigorous
research, and lessen our chances of ever knowing for sure
whether an alternative treatment actually works or not.
And at a time when research and patients alike complain
that IRBs are overburdened and informed consent is not always
truly informed, this bill would increase the chances that
patients are put at inappropriate risk, not lessen them.
I join my colleagues in welcoming our witnesses, and I look
forward to their testimony.
Mr. Bilirakis. I thank the gentleman.
Mr. Burr to give an opening statement.
Mr. Burr. I thank the chairman, and I thank the chairman
for this hearing. Mr. Chairman, we have a lot on our plate this
morning, and it is all extremely important. I will focus just
briefly on the NIBIEE bill which Ms. Eshoo and I have
introduced, which currently has 169 cosponsors. It is
unfortunate today as we meet this morning that Ms. Eshoo is in
California under the weather, but I am sure if she were here,
she would speak out very loudly in support of this legislation
that she and I and others on the Hill and throughout the
country have worked on.
I don't think I can sum it up any better than the committee
brief for this hearing. In their description of the NIBIEE bill
it said: Breakthroughs in imaging such as magnetic resonance
imaging and computer tomography have revolutionized the
practice of medicine in the past quarter century. But those
technologies are inadequate in diagnosing some diseases.
What that statement says is that we have made tremendous
progress, despite a lack of a focused effort, on our ability to
detect at the early possible point. What we have heard, Mr.
Chairman, from people around the country is that we can do
better. If you give us the type of focus that it takes in
resources, we can come through with an earlier detection of
disease, and we can give physicians who are treating disease
many more options because of that early detection.
What NIBIEE does is create an institute of health for
biomedical imaging at the NIH, the same NIH that every member
of this panel and most Members of this Congress are committed
to putting new resources into. What we want to make sure when
we make that commitment to the American people for additional
resources to chase the disease that affects every family in
this country, is that biomedical imaging is one of the
concentrated focuses of the NIH because we know that early
detection will give us more options and will give patients more
options.
Mr. Chairman, I would urge my colleagues today to ask as
many questions of the witnesses that are here to testify on
this bill, but, in the end, to also be supportive of this
legislation. This is extremely important that we get it done
and we get it done now. We spend a lot of time talking about
health care policy. This is a place where we can, in fact, make
sure that our options are greater down the road. And I applaud
the chairman. And I yield back.
Mr. Bilirakis. I thank the gentleman.
Mr. Pallone for an opening statement, going on basis of
seniority.
Mr. Pallone. Thank you, Mr. Chairman. I will limit my
comments to the two bills which have I cosponsored, H.R. 3677,
the Thomas Navarro FDA Patient Rights Act, and the H.R. 1795,
National Institute of Biomedical Imaging and Engineering
Establishment Act.
The first of these, the Thomas Navarro FDA Patient Rights
Act, deals with the rights of patients and parents to make
informed choices about medical treatment. The bill's namesake,
young Thomas Navarro, has unfortunately been suffering from the
effects of a brain tumor. As any parent with a child in
Thomas's situation would, the Navarros researched the treatment
options available and found the treatment of radiation and
chemotherapy would have extremely debilitating side effects
which they did not want to risk. Rather, the Navarros preferred
to have Thomas treated with antineoplaston therapy, a therapy
surrounded by some controversy that is under clinical trial.
The Food and Drug Administration has, however, refused to allow
this to happen on the basis that his parents have not yet tried
the radiation and chemotherapy path.
I cosponsored the bill because Thomas Navarro's parents
should be allowed to decide for themselves whether or not the
antineoplaston treatment for Thomas in the setting of a
clinical trial is an appropriate path to follow. They
researched the issue, and they understand the issue, and I do
not believe in light of the circumstances surrounding the case
that the Navarros should be denied their right to choose.
The issue is important not just for Thomas, t for other
patients in similar circumstances. We shouldn't be restricting
the rational choices and measured choices of individuals who
choose to pursue alternative medical treatments whose possible
outcomes they fully comprehend.
The second bill I want to mention, the National Institute
of Biomedical Imaging and Engineering Establishment Act, is an
excellent piece of legislation that I hope all of my colleagues
on this subcommittee will support. I have discussed the
importance of this legislation on a number of occasions over
the last 2 years with medical professionals from the radiology
department of the University of Medicine and Dentistry of New
Jersey and other medical professionals from my home State. All
of them have stressed the importance an institute for imaging
research within NIH can play in promoting further breakthroughs
in a field that has already vastly changed the practice of
medicine for the better.
And I want to thank the chairman for having the hearing on
these two bills and the other that we have today. I yield back.
Mr. Bilirakis. Mr. Stupak for an opening statement. I am
going to try to continue on rather than break, as long as
someone gets back in time to spell me.
Mr. Stupak. Mr. Chairman, I thank you for holding this
hearing, and I thank the witnesses for being here. I look
forward to their testimony.
I am a cosponsor of H.R. 762 and H.R. 1795, and for the
sake of time, and I want to hear the witnesses, I yield back
the balance of my time.
Mr. Bilirakis. I thank the gentleman so very much.
Mrs. Capps for an opening statement.
Mrs. Capps. Mr. Chairman, I thank you for holding this
hearing, and I want to thank--extend a welcome to our
witnesses. Today we are going to be discussing several worthy
pieces of legislation all focused on securing the health of the
American people.
Because of the nature of our hearing today, I want to
remind you and other members of the bill H.R. 353, the ALS
Treatment and Assistance Act. This is a bill that I am
offering, enjoying bipartisan support; currently has 280
cosponsors, many of whom serve on this committee. I am so glad
that we are having a hearing today on so many important pieces
of legislation. I commend you for making that happen, and my
hope is that this committee can address H.R. 353 before the
106th Congress is over.
I want to state my strong support in this setting for H.R.
762, the lupus research and care amendments of 1999. I am so
pleased that our colleague Carrie Meek is here, the author of
this bill, which would authorize the Secretary of Health and
Human Services to make grants for the delivery of essential
services to individuals with lupus and their families. As a
nurse, I know what an insidious disease this is. For many
people lupus is a mild disease affecting only a few organs; for
others can cause serious, even life-threatening problems.
My district is home to the Scleroderma Research Foundation.
Scleroderma is a condition that is closely linked to lupus, and
I have seen the work of women like Sharon Monsky in Santa
Barbara in my district, who has fought so long to raise
awareness of scleroderma and lupus, diseases which
disproportionately affect women and can have life-and-death
consequences. So I applaud the subcommittee for recognizing
this legislation.
I also want to acknowledge the legislation sponsored by my
colleague Anna Eshoo which was described by our colleague
Richard Burr, H.R. 1795. This legislation will fill a critical
void at the NIH by creating an independent institute on this
topic of bioengineering. These disciplines have made such
contributions to the improvement, as our colleague has said,
and have no research home in the current structure of NIH. And
I support this legislation, again commending our colleagues for
their leadership in this area.
Finally, I know my statement submitted will be longer than
this, but I want to say one word about my good friend and
colleague Deborah Pryce and offer my strongest words of support
for her resolution. Her resolution focuses on the importance of
promoting awareness and expanding research on childhood cancer.
This is Childhood Cancer Month, September. It is the second
leading cause of death in children past infancy. Many childhood
cancers can be cured, but, sadly, dozens still cannot. And I
applaud Congresswoman Pryce's efforts in this difficult area
and pledge any support that I can give to help get this
legislation passed into law.
Mr. Chairman, this is an important topic. Thank you for
holding this hearing, and I yield back.
Mr. Bilirakis. I thank the gentlewoman.
[Additional statements submitted for the record follow:]
Prepared Statement of Hon. Barbara Cubin, a Representative in Congress
from the State of Wyoming
Our individual health care needs have changed over the years, but
the bottom line is we all need care--and we want it to be the best.
That's all the reason we need to make sure we keep moving forward
with innovative technologies, new drug therapies, and better standards
of care.
We must continue to protect and nurture the different fields of
research and development in health care because we live in an
unpredictable and sometimes hostile bacterial environment that is
constantly reinventing itself.
That means that clinical trials will take on an even greater role
in the future as we try to develop new drugs to counteract new
diseases.
Giving patients the proper access to these trials is therefore
paramount to our success.
Of course, these trials are not possible without the new drug
therapies and biologicals that sustain them so supporting drug research
and innovation is critical.
By the same token, we're making technological advances that we
never thought possible--such as MRIs, Cat Scans, laser devices,
telemedicine, and the like.
We must continue to push the envelope in the field of health care
technology because it has already proven to enhance the quality of
patient care.
There are, no doubt, countless things we have yet to discover about
biomedical imaging and we should continue to explore its capabilities.
Today we're discussing specific legislative proposals that are
designed to secure the future health of this country. I look forward to
the discussion and stand ready to work with all of you.
______
Prepared Statement of Hon. Tom Bliley, Chairman, Committee on Commerce
Thank you Mr. Chairman.
I am pleased that you are having this hearing today which continues
the extraordinary public health work that Members of the Commerce
Committee have done over the past six years.
I would like to take a moment of personal privilege to state how
proud the Members of the Committee should be. In the prior two
Congresses, this Committee has empowered states and localities to meet
the health care and nutritional needs of low-income residents; and
provided relief to those hardest hit by the AIDS epidemic.
The Health Insurance Portability and Accountability Act allows
working Americans to change jobs without risking the loss of their
health care insurance due to a preexisting condition. The law also
attacked health care fraud and eliminated tax code discrimination
against millions of small businesses and the self-employed. It also
provided tax relief for long-term health care needs and terminally ill
patients and their families.
The Food Quality Protection Act and the Safe Drinking Water Act
Amendments of 1996 modernized programs and enhanced Americans' access
to safe, abundant, and affordable food and water.
We established Medicare+Choice and the State Children's Health
Insurance Program. Among other bills, we enacted the Food and Drug
Administration Modernization Act; the Birth Defects Prevention Act; the
National Bone Marrow Registry Reauthorization Act; the Mammography
Quality Standards Reauthorization Act; the Women's Health Research and
Prevention Amendments.
This Congress, we have passed prescription drug legislation and HMO
reform. We reauthorized funding to help those suffering from AIDS and
moved forward on a major children's health initiative. We also have
moved on the Breast and Cervical Cancer Treatment Act, the Health Care
Fairness Act, the Cardiac Arrest Survival Act, the Developmental
Disabilities Amendments, The Drug Addiction Treatment Act, the Date-
Rape Prevention Drug Act, the Health Research and Quality Act, the Pain
Relief Promotion Act, the Organ Procurement and Transplantation Network
Amendments, and the Nursing Home Resident Protection Amendments. We
have also provided new options under Social Security for the disabled.
It is a spectacular record.
Now for the work before us today. I look forward to hearing from
today's witnesses and to making even further progress on public health
with the Members of this Committee. Today we will discuss global health
concerns, new medical technologies, drug approval systems, expanding
access to clinical trials, Lupus, and childhood cancers. Some of these
efforts may go forward this Congress and others may not. I only ask the
Members of the Committee to maintain their workman-like commitment to
bipartisan improvements to our public health programs.
______
Prepared Statement of Hon. Gene Green, a Representative in Congress
from the State of Texas
Mr. Chairman, thank you for calling this hearing today. I
appreciate the opportunity to examine legislation that has been
introduced to improve America's health care programs.
There are several bills being discussed today, two of which I am a
cosponsor of, the Lupus Research and Care Amendments of 1999 and the
National Institute of Biomedical Imaging and Engineering Establishment
Act.
H.R. 762 by Rep. Carrie Meek is a bill that would expand and
intensify research at the NIH to diagnose, treat, and eventually cure
lupus. It also increases the funding for lupus research and education
and establishes a grant program to expand availability of lupus
services.
I commend my colleague for introducing this bill and look forward
to the testimony on this piece of legislation.
I am also a cosponsor of H.R. 1795 by Rep. Richard Burr. This
legislation would create an independent institute to support basic
research in medical imaging and bioengineering. These disciplines have
made great contributions to the improvement of medical care in the past
two decades, yet they have no research home in the current NIH
structure. The creation of the National Institute of Biomedical Imaging
and Engineering would create a research environment in which new
imaging and bioengineering technologies techniques and devices can be
developed for clinical use more rapidly than under the present system.
This would allow for continued rapid progress in fields such as
genetics and molecular biology, which utilize advanced imaging
techniques.
I represent the medical center area and I can tell you that this
piece of legislation would help advance the technology that can be used
for clinical use more rapidly than under the present system.
I look forward from hearing from our witnesses today.
______
Prepared Statement of Hon. Anna G. Eshoo, a Representative in Congress
from the State of California
Thank you, Mr. Chairman, for holding today's hearing. With six
bills before us today, we have a lot on our plates. However, I'd like
to focus my comments on the bill that my colleague Mr. Burr and I have
introduced, H.R. 1795 to establish a National Institute of Biomedical
Imaging and Engineering at the National Institutes of Health (NIH).
Mr. Chairman, dramatic advances in imaging and bioengineering have
revolutionized medical practice in recent years. Development of new,
noninvasive imaging techniques, such as Magnetic Resonance Imaging
(MRI) and Computed Tomography (CT), has allowed for earlier detection
and diagnosis of disease, dramatically improving the quality of
healthcare. But, the next generation of breakthroughs will be longer in
coming, or in some fields, may not come at all unless we modernize the
structure at NIH.
Today, these disciplines, which have made unmatched contributions
to the improvement of medical care in the past two decades, have no
research home in the current NIH structure. Research at the current
institutes at NIH is based on molecular biology, which is fundamentally
different from research in imaging and bioengineering. If we are to
ensure the continued development of new techniques and technologies,
these disciplines require an identity and research home at the NIH that
is independent of the existing institute structure.
H.R. 1795 would fill a critical void at the NIH by creating an
independent institute to support basic research in medical imaging and
bioengineering. It would create a research environment in which new
imaging and bioengineering technologies, techniques, and devices can be
developed for clinical use much more rapidly than under the present
system. By doing so, H.R. 1795 replaces disorganization with
efficiency, effective management, accountability, and an improved
scientific focus.
At a time when Congress has committed to doubling the NIH budget,
we must ensure that research dollars are expended more effectively and
efficiently and that the fields of medical science that have
contributed the most to the detection, diagnosis, and treatment of
disease in recent years receive appropriate emphasis. Establishment of
a National Institute of Biomedical Imaging and Engineering at the NIH
would accelerate the development of new technologies, improve
coordination and efficiency throughout the Federal government, reduce
duplication, lay the foundation for a new medical information age, and
provide a structure to train the young researchers who will make the
pathbreaking discoveries of the next century.
I'm proud to join Representative Burr in sponsoring H.R. 1795 and
I'm pleased that the Committee has finally turned its attention to it.
I encourage my colleagues' support at tomorrow's full committee markup
and I look forward to passage by the full House.
______
Prepared Statement of Hon. John D. Dingell, a Representative in
Congress from the State of Michigan
Mr. Chairman, we have six bills and resolutions before us today, so
I will be brief.
H.R. 2399 would establish a national commission to examine how the
United States can most effectively use our scientific and technical
expertise to tackle disease on a global basis. This is an ambitious
undertaking. A panoply of public and private sector organizations spend
considerable time and resources on projects that collectively span
virtually all global health issues. Nevertheless, the commission could
generate useful information, which in turn could optimize the use of
scarce resources and which could lead to improvements in global health.
H.R. 762, the Lupus Research and Care Amendments, is an excellent
piece of legislation that has been developed by my good friend and
colleague, Representative Carrie Meek. As we will learn, lupus is a
debilitating and sometimes fatal autoimmune disease that
disproportionately afflicts women, particularly women of color.
H.Res. 576 by Representative Pryce is another measure worthy of our
support. Children are not simply ``little adults.'' I note that our
colleague, Representative Forbes, has a similar bill, H. Con. Res. 115.
H.R. 1795 would create the Institute of Biomedical Engineering at
the National Institutes of Health (NIH). I note the fine work that our
colleagues, Representatives Eshoo and Burr, have done on this bill and
believe that it deserves our careful consideration, although NIH has
some concerns with the bill.
H.R. 3677 causes me great concern, and I can not support it as
drafted. This bill would dramatically alter the basic role of the Food
and Drug Administration (FDA) in the supervision of human subject
protection in the development of experimental drugs. FDA must play a
fundamental role in protecting the public in both the development and
approval of drugs. The FDA Modernization Act, which has provisions on
this matter, is less than three years old, but we should examine the
investigational drug process to ensure that it is serving the public in
the best possible manner. The moral, ethical, and safety issues
presented in the informed consent process are particularly important in
the case of experimental drugs and their use on persons with serious
and life threatening diseases.
Finally, I oppose H.R. 4242, the Orphan Drug Innovation Act. We
will hear testimony opposing this bill from the National Organization
for Rare Disorders (NORD). NORD represents approximately 25 million
Americans with more than 6,000 ``orphan'' diseases. The orphan drug law
has been a success. Anyone wishing to enact significant changes in this
important health statute bears a heavy burden. I do not believe the
proponents of this bill have met that test.
Thank you, Mr. Chairman.
Mr. Bilirakis. Mr. Gekas and Mrs. Meek are here to testify
on behalf of their legislation briefly and also introduce the
witnesses. What is your pleasure? Should we go to you now, or
would you like to return? I don't want to really rush you, but
we do want to make this vote. Would like to return? I will be
here.
Mrs. Meek. With your pleasure and Mr. Gekas's, I would
rather go forward, if I may.
Mr. Bilirakis. What would you prefer to do?
Mr. Gekas. I do not mind proceeding.
Mr. Bilirakis. Well, then, proceed. The only thing is I am
liable to cut you off.
You are recognized, the gentleman from Pennsylvania.
STATEMENT OF HON. GEORGE W. GEKAS, A REPRESENTATIVE IN
CONGRESS FROM THE STATE OF PENNSYLVANIA
Mr. Gekas. Thank you. The opening remarks made by the
chairman and the ranking member in describing the bill which I
have placed before you were more than adequate in describing
the purport of the legislation.
I simply want to add to that the fact that the national
goal for the 20th century is well-known to everybody. The one
that we just completed, that goal was thrust upon us. It was
the repulsion of totalitarianism and the reestablishment and
preservation of democracy across the globe. That was the
national gole thrust upon us.
Now we have an opportunity in the next century to assume
leadership in what I envision to be, and others do, the
national goal for the 21st century, namely the eradication of
disease worldwide. Why is that important? Not only for the
humanitarian and altruistic rationale that are the foundation
for such a project, but also in the enlightened self-interest
of our country, which is the leader in all of these disciplines
that are so vital to the health of the world, that enlightened
self-interest we not only protect our people in the future from
these diseases and other catastrophes that might occur, but at
the same time we create jobs, we create interests, we develop
new technologies, new pharmaceuticals and all the other
necessaries to further envision a world without disease with
our country in the forefront.
That is what the purpose of it is. That is why our witness
is so important. She has testified before our Biomedical
Research Caucus as one of the leading lecturers in her field,
and you will see from her testimony that she will be a vital
force if we implement the legislation which I have offered.
She is Professor Dyann Wirth, of the Department of
Immunology and Infectious Diseases, Harvard School of Public
Health, and has a slew of publications which she is the author.
She is renowned in her field. She impressed the Members of the
Hill, the Capitol, who engaged in the Biomedical Research
Caucus series, and I am sure she will impress you.
Unfortunately she has impressed me so much that I am leaving
right now to go vote, but I know what she will testify and
commend her to you.
[The prepared statement of Hon. George W. Gekas follows:]
Prepared Statement of Hon. George W. Gekas, a Representative in
Congress from the State of Pennsylvania
Mr. Chairman, and Members of the Committee, I would like to thank
you for giving me the opportunity to testify at this hearing on such an
important matter.
The latter part of the twentieth century saw the dismantling of the
U.S.S.R., the fall of the Berlin Wall, and a worldwide blossoming of
democracy. To many this seemed an impossibility at the time. Most of
the world rightly places the credit for these events in the hands of
our United States. Our leaders and our government put forth endless
efforts to achieve these ends successfully. While these accomplishments
are extraordinary, it is time to turn our attention to another
seemingly impossible goal.
With the dawning of the new millennium, the time has come to focus
our national energy and resources on efforts to eradicate all disease
across the globe. This is indeed an awesome goal that may not be
attainable in itself, but its purpose could lead to new treatments of
diseases, and new approaches to controlling them.
Beyond the humanitarian reasons for promoting this idea, the U.S.
has enlightened domestic goals as well. Eradicating global disease
would protect American citizens, improve the quality of life worldwide,
enhance our economy, and advance American interests across the globe.
Achieving this goal would impact every aspect of our society, not just
the field of health care.
In order to reach the objective of eradicating disease globally, I
have introduced H.R. 2399, the Advancement of Global Health Act, which
would establish a commission, the National Commission for the New
National Goal: The Advancement of Global Health. The commission's task
would be to recommend a strategy for the global eradication of disease.
The United States has the resources, through the National Institutes of
Health (NIH) and the National Science Foundation, to take the lead in
expanding health research information globally, especially with the
recent explosion of Internet technology. Additionally, the commission
would assist the Center for Vaccine Development at the NIH to achieve
global control over infectious disease. A one-time $1 million
allocation would be granted to the commission to coordinate and attract
other sources of funding both domestically and abroad for the purpose
of achieving these objectives.
The fifteen-member commission would be charged with coordinating
governmental, academic, and public and private health care entities for
the purpose of global disease eradication. The commission would then be
required to submit a final report to Congress within one year of its
establishment, with its recommendations for legislative,
administrative, and other appropriate actions. The wide-sweeping goals
of this legislation could encompass ideas like the simple act of
teaching a youngster how to properly wash his or her hands, reaching
across to the more complex, such as medical advances learned from space
exploration.
It gives me great pleasure to have Dr. Dyann F. Wirth here today to
further discuss the idea of eradicating global disease. Dr. Wirth
earned a Ph.D. in Cell Biology and Biochemistry from the Massachusetts
Institute of Technology. She currently serves as a professor at the
Harvard School of Public Health in its Department of Immunology and
Infectious Diseases. Dr. Wirth is a widely published author in numerous
esteemed scientific and medical journals. She also worked for ten years
as an NIH Study Section Member on Tropical Medicine and Parasitology,
which is Dr. Wirth's particular area of expertise. Again, I am glad to
have her with us today.
The intelligence and imagination of American researchers and
scientists did not fail us in our attempts to preserve and promote
freedom and democracy across the globe. It is now time for us to
unleash these same great minds towards the goal of improving the
quality of life for everyone. We must summon all of the resources at
our command in our efforts to eradicate disease from the face of the
Earth.
Again, Mr. Chairman, I thank you for this hearing on this very
important matter that can change the lives of countless individuals.
Mr. Upton [presiding]. I thank the gentleman from
Pennsylvania. Now I recognize Mrs. Meek, and I didn't know that
you were here when I gave my opening statement, but I commended
you for your good bill, and I am delighted to be a cosponsor
and delighted to recognize you now.
STATEMENT OF HON. CARRIE P. MEEK, A REPRESENTATIVE IN CONGRESS
FROM THE STATE OF FLORIDA
Mrs. Meek. Thank you, Mr. Chairman. I am pleased to be
before the committee again, and I want to thank you for
bringing this hearing up again. 762 is an extremely important
piece of legislation. It is time that it be passed, Mr.
Chairman, and of course I am hoping that this committee will
see fit to pass it out and send it to the floor as quickly as
possible so that no one else will have to wait for the kind of
assistance that this Lupus Research and Care Act will bring.
The Lupus Foundation has really stuck with the Congress
through this, Mr. Chairman. They have assiduously watched this
bill for many, many years, and I do hope we can get it passed.
What it will do is add additional services for lupus victims.
But I am here this morning because I am pleased and
privileged and blessed to have a young lady who is sitting at
the table, the testimony table, to testify for lupus, a very
beautiful, lovely lady, Tomiko Fraser. She is the spokesperson
for the Lupus Foundation, and I do not have to take your time
to tell you all of the demerits, I would say, or all of the
real terrible effects of lupus. I lost a sister to it. I have
lost so many friends to lupus. It is a young woman's disease,
and as a result of that, I think as this committee you have 243
cosponsors behind this bill.
And I want to have my statement placed in the record, Mr.
Chairman, and with your permission.
Mr. Upton. Without objection, your entire statement will be
put in the record, and, again, we thank you for your leadership
on this very important issue that touches so many American
families in every State.
[The prepared statement of Hon. Carrie P. Meek follows:]
Prepared Statement of Hon. Carrie P. Meek, a Representative in Congress
from the State of Florida
Good morning, Chairman Bilirakis, Ranking Member Brown and my other
colleagues. It's a pleasure to be with you. Thank you for holding this
hearing and inviting me to appear before your Subcommittee to discuss
my lupus bill, H.R. 762. My bill would authorize additional funding for
lupus research and treatment programs. Providing such additional help
for lupus victims is not a Democratic issue or a Republican issue. It's
an American issue. My bill has broad bipartisan support (243 cosponsors
from both sides of the aisle).
I won't take the Subcommittee's time to lay out all of the details
of H.R. 762. In a nutshell, this bill would authorize spending of up to
$75 million for lupus research and $75 million for lupus care and
treatment programs.
I want to recognize and introduce to the Subcommittee the beautiful
young women sitting next to me, Tomiko Fraser. Tomiko is a nationally
recognized spokesmodel and actress. She is the first African-American
women to be a spokesmodel for Maybelline. Tomiko's sister has lupus.
Tomiko has seen firsthand the devastation that lupus has caused for her
sister and she speaks eloquently about what it is like for her sister
to live with lupus. She makes a powerful case for why we need the
additional funding for lupus research and treatment that my lupus bill
authorizes.
Tomiko, I salute you and your sister for your courage and thank you
for your commitment to obtaining additional relief for lupus victims
and their families.
As many of you know, I know firsthand the heartache that lupus
causes, I lost a sister to lupus and have seen many others suffer from
this disease. We all know the debilitating pain and fatigue that lupus
often causes, pain and fatigue that makes it difficult for persons with
lupus to maintain employment and lead normal lives. We also know the
profound impact that this disease has on family members of those with
lupus.
Mr. Chairman, lupus victims and their families need more help and
they need it now. Congress and the President can provide it by passing
and signing H.R. 762. We can do it, and we must do it this year, but,
to make this happen, I need your help to persuade the leadership to
bring this bill to the floor immediately. I won't be satisfied, and
none of us should be, until we get this bill to the House floor, pass
it overwhelmingly, pass it in the Senate and have it signed by the
President.
I urge the Subcommittee and the full Commerce Committee to mark
this bill up immediately and implore the Leadership to bring this bill
to the floor on the Suspension Calendar at once so that it can pass the
House this Session. Thank you, Mr. Chairman.
Mrs. Meek. Thank you, Mr. Chairman. And I would love to
hear Tomiko, but I must go and vote.
Mr. Upton. Well, you can come back.
Mrs. Meek. All right.
Mr. Upton. At this point, as no members are coming back
from the vote that is currently ongoing, I would like to invite
the first panel to come to the table. They include Dr. Nick
Bryan, professor and chairman of radiology at the Hospital of
the University of Pennsylvania; Dr. Reed Dunnick, professor and
Chair of the Department of Radiology, University of Michigan.
Go blue. Beat UCLA this weekend. Dr. Bruce Hillman, professor
and Chair, Department of Radiology, University of Virginia; Ms.
Tomiko Fraser, who is, of course, at the table already, and
obviously the national spokesperson for the National Lupus
Association; and Dr. Dyann Wirth, professor at the Department
of Immunology and Infectious Diseases at Harvard.
I just want to say before we start that there are a number
of things ongoing this morning, and a number of committees and
subcommittees that are meeting. We have a very important issue
on the House floor, that being the marriage penalty tax as
well, where I am going to have to return to engage myself in
that debate a little bit later this morning.
Your statements are made part of the record in their
entirety. We would like to keep this to 5 minutes each or less.
And Dr. Wirth, we will start with you. Thank you for being here
with us this morning.
STATEMENTS OF DYANN WIRTH, PROFESSOR, DEPARTMENT OF IMMUNOLOGY
AND INFECTIOUS DISEASES, HARVARD SCHOOL OF PUBLIC HEALTH; N.
REED DUNNICK, PROFESSOR AND CHAIR, DEPARTMENT OF RADIOLOGY,
UNIVERSITY OF MICHIGAN HEALTH SYSTEM; BRUCE J. HILLMAN,
PROFESSOR AND CHAIR, DEPARTMENT OF RADIOLOGY, UNIVERSITY OF
VIRGINIA; TOMIKO FRASER, NATIONAL SPOKESPERSON, LUPUS
FOUNDATION OF AMERICA, INC.; AND R. NICK BRYAN, PROFESSOR AND
CHAIRMAN OF RADIOLOGY, HOSPITAL OF UNIVERSITY OF PENNSYLVANIA
Ms. Wirth. Mr. Chairman and members of the subcommittee,
thank you for the opportunity to testify today. My name, as you
already know, is Dyann Wirth. I am a professor at Harvard
University School of Public Health and the Department of
Immunology and Infectious Diseases, and I am here today on
behalf of the Joint Steering Committee for Public Policy, which
has worked closely with Representative Gekas in his outstanding
efforts in support of biomedical research.
The Joint Steering Committee for Public Policy is a
coalition of four life sciences societies representing more
than 25,000 researchers. I am here today to support--to express
the support of the joint steering committee for Congressman
Gekas's bill which we have just heard about on the advancement
of global health, H.R. 2399. This bill, if enacted into law,
would create a Presidential/congressional commission to
investigate how we as a Nation can most effectively seize the
scientific opportunities presented by modern advances in
research to eradicate many of the diseases that are plaguing
millions of the world's people.
We support this bill because we believe that in this next
millennium it is within the grasp of human capacity to
accelerate the role of basic biomedical research and the
translation of that research to the benefit of the world's
least fortunate people.
Now is the time. This is an attempt to focus all of the
tremendous scientific energy in the United States on fighting
diseases throughout the world. This is a noble endeavor for the
United States. We have the means to do this, and I believe we
should make it a priority.
My particular experience is in malaria, but as devastating
as malaria is, it is just one of the several infectious
diseases that are not only killing millions, but costing
billions. According to the World Health Organization,
infectious diseases account for more than 13 million deaths a
year. That is 35 percent of the deaths in the world today. That
means during the duration of this hearing, 1,500 people will
die from infectious disease. Malaria alone kills 2.7 million
people each year. Tragically, every 30 seconds a child
somewhere in the world, probably in Africa, dies of malaria.
The enormous volume of travel and trade have made
infectious diseases blind to national borders, and this has
been recognized. The January 2000 unclassified report from the
CIA's National Intelligence Council entitled ``The Global
Infectious Disease Threat and Its Implications for the United
States'' suggests that in modern warfare infectious diseases
are likely to account for more military hospital admissions
than battlefield injuries. The report claims, and I concur, New
and reemerging infectious diseases will pose a rising global
health threat and will complicate U.S. and global security over
the next two decades. These diseases will endanger U.S.
citizens at home and abroad, threaten U.S. Armed Forces
deployed overseas, and exacerbate social and political
instability in key countries and regions where the United
States has an interest.
Research into prevention, treatment and control of tropical
and infectious diseases is now more important than ever. I will
talk just briefly about malaria because that is where my
interest and passion is. Among adults living in high-
transmission areas, malaria is considered a chronic disease. A
single bout can incapacitate someone for weeks, and despite
massive efforts to eradicate this disease in the 1950's, there
is more malaria in the world today than there ever has been in
history. One-fourth of the world's population is at risk of
infection.
Clearly, we need better implementation of the tools that we
have in the short term, but our tools are not adequate. New
research interventions are desperately needed. Cutting-edge
technology has led to the development of new paradigms. The
genome of the most important parasite is being sequenced. We
have DNA vaccines, new technologies. And it is important to
seize the opportunities of these scientific advances to
accelerate and defeat malaria worldwide.
But equally important as progress in research and public
support and awareness of these major health threats, in order
to conquer malaria, AIDS, malaria, other infectious diseases,
we need a global strategy that includes American leadership and
resources to invest in continued research into prevention and
treatment.
As we begin the 21st century, we are blessed with
unimaginable opportunities to build on breakthrough research to
control and prevent global infectious diseases. This is not
just altruism to reduce suffering of the world's most needy;
this is also a question of national security and health for the
United States and its citizens. Renewed investment in the
treatment and prevention of global infectious diseases is a
win-win for the country. By helping others across the world, we
are launching the best defense to protect the health of our
Nation's people.
We hope that you will seriously consider passage of H.R.
2399 and thank you very much for the opportunity to present.
[The prepared statement of Dyann Wirth follows:]
Prepared Statement of Dyann Wirth, Professor, Harvard University School
of Public Health
Mr. Chairman and members of the Subcommittee, thank you for the
opportunity to testify today. My name is Dyann Wirth. I am a Professor
at the Harvard University School of Public Health Department of
Immunology and Infectious Disease. I am here today on behalf of the
Joint Steering Committee for Public Policy, which has worked closely
with Representative Gekas in his outstanding efforts in support of
biomedical research.
The Joint Steering Committee for Public Policy (JSC) is a coalition
of four life-science societies representing more than 25,000
researchers in the fields of genetics, cell biology, biophysics,
biochemistry and molecular biology. The JSC was formed in late 1980s to
bring scientists together to advocate for federal funding for basic
biomedical research. Eric Lander of MIT chairs JSC, and among its 20
board members are Nobelists J. Michael Bishop, Paul Berg and Harold
Varmus.
Founders of the JSC realized that there was a great need for the
United States to invest in biomedical research and called for the
doubling of the NIH budget. Since that time, Mr. Chairman, the
Congress' visionary support of the NIH has lead us to the dawn of a new
age in science. I have no doubt that the coming decade will be
remembered for major discoveries enabled by the mapping of the human
genome which will lead to the prevention and cure of diseases. The JSC
also worked with Congressman Gekas to introduce the Congressional
Biomedical Research Caucus to the Halls of Congress. Today, the
Biomedical Research Caucus has been called by Chairman John Porter and
former Speaker New Gingrich among others, ``the most influential Caucus
in Congress.''
I am here today to express the support of the Joint Steering
Committee for Congressman Gekas' bill the National Commission for the
New National Goal: The Advancement of Global Health Act, H.R. 2399.
this bill, if enacted into law would create a Presidential/
Congressional commission to investigate how we as a Nation can most
effectively seize the myriad scientific opportunities presented by
modern advances in genomic to eradicate many of the diseases that are
plaguing millions of the world's people. We support this bill because
we believe that in this third Millenium it is within the grasp of human
capability to accelerate the role of basic biomedical research and the
translation of that research to the benefit of the world's least
fortunate people. Now is the time: scientific potential is there; it
requires only political will to make it reality.
My particular experience is malaria, but as devastating as malaria
is, it is just one of several infectious diseases that are not only
killing millions but costing billions. According to the World Health
Organization, infectious diseases account for more than 13 million
deaths a year. That means that over the duration of this hearing 1,500
people will die from an infectious disease--over half of them children
under five.
According to the WHO the seven infectious diseases that caused the
highest number of deaths in 1998 are AIDS, TB, malaria, hepatitis B and
hepatitis C, diarrheal diseases, and measles. Of these, TB and
hepatitis are renewed threats because they are becoming increasingly
resistant drug resistant. But, malaria alone is estimated to cause up
to 500 million clinical cases and 2.7 million deaths each year,
representing 4 percent to 5 percent of all fatalities globally.
Tragically, every 30 seconds a child somewhere in the world dies of
malaria. As you know, most of these deaths occur in developing
countries where extreme poverty and lack of access to basic health
care, adequate sanitary and essential drugs can seal the fate of
children before they're born. However, the enormous volume of travel
and trade today have made, infectious diseases blind to national
borders.
A January, 2000, unclassified report from the CIA's National
Intelligence Council, entitled ``The Global Infectious Disease Threat
and Its Implications for the United States,'' suggests that in modern
warfare infectious diseases are likely to account for more military
hospital admissions than battlefield injuries. The report assesses
claims, and I concur, that ``New and reemerging infectious diseases
will pose a rising global health threat and will complicate US and
global security over the next 20 years. These diseases will endanger US
citizens at home and abroad, threaten US armed forces deployed
overseas, and exacerbate social and political instability in key
countries and regions in which the United States has significant
interests.''
Research into the prevention, treatment and control of tropical and
infectious disease are now more important than ever to the US and the
world. I will address malaria as an example because I know more about
malaria than about other global health threats.
Among adults living in areas of high transmission, malaria is best
thought of as a chronic, debilitating illness that robs its victims of
years of productivity. A single mosquito bite can transmit one of the
four parasites that cause malaria, setting in motion bouts of fever,
chills, and nausea that can recur for weeks. According to a 1993 World
Bank Report, malaria represents a global public health burden second
only to tuberculosis among infectious diseases of the 2-3 million
children who die of malaria each year, most of them live in Africa,
continent already overwhelmed by poverty and internal conflict. Those
who survive can suffer chronic anemia and/or immune suppression that
leave victims vulnerable to other fetal diseases.
Despite massive efforts to eradicate in the 1950s, today than at
any other time in history. More than 500 million people are infected
with malaria worldwide; one-fourth of the world's population is at risk
for infection. Better implementation of currently available control
measures, including the use of insecticide, and better and more
rational use of existing drugs, should be the goal in the short term;
in the long-term, new research interventions are desperately needed.
Cutting-edge technology has led to the development of new paradigms--
the genome of the most important malaria parasites is being sequenced,
DNA vaccines are being developed and tests of methods to prevent
transmission by the mosquito are being explored. We must seize the
opportunity presented by these scientific adversities to accelerate the
defeat of malaria worldwide. But equally important as progress in
research is public support and awareness of this major health threat.
In order to conquer malaria, we need a global strategy that includes
American leadership and resources to invest into continued research
into prevention and treatment. I must also point out the great need for
action with regard to HIV/AIDS in Africa. Africa remains the epicenter
of the pandemic, bearing the largest disease burden, with 70 percent of
people living with AIDS worldwide, 83 percent of global AIDS deaths,
and 95 percent of the world's AIDS orphans. AIDS is reversing decades
of progress from important public health efforts, lowering life
expectancy, and significantly affecting daily life form millions of
Africans. This situation cries out for leadership.
The JSC supports efforts to encourage research and development on
vaccines to combat malaria, tuberculosis, AIDS and other infectious
diseases causing and to ensure that existing vaccines are accessible to
populations in developing countries most impacted by these diseases.
These efforts will require partnerships among federal agencies,
industry, non-profit foundations and other NGOs, the World Bank, and
international organizations to combat the scourge of infectious
diseases. This Commission could vastly accelerate the pace of these
efforts.
Specific mechanisms might include, enhanced R&D tax credits and new
tax credits for sales of vaccines, contributions to international
organizations such as the Global Alliance for Vaccines and
Immunizations (GAVI) for the purchase and distribution of vaccines in
developing countries, and measures that will improve the public health
infrastructure in developing countries in order to expand
immunizations, prevent and treat infectious diseases, and build
effective delivery systems for basic health services.
As we begin the 21st century, we are blessed with unimaginable
opportunities to build on breakthrough research to control and prevent
global infectious disease. This is not just altruism to reduce the
suffering of the world's most needy: this is also a question of
national security and health for the United States and its citizens.
Renewed investment in the treatment and prevention of global infectious
disease is a win-win for the country: by helping others across the
world we are also launching the best defense to protect the health of
our Nation's people. We hope you will seriously consider passage of
H.R. 2399.
Thank you for the opportunity to present the views of the Joint
Steering Committee for Public Policy. I would be happy to answer your
questions.
Mr. Upton. Thank you, Dr. Wirth.
Dr. Dunnick, welcome to Washington's version of the ``Big
House.''
STATEMENT OF N. REED DUNNICK
Mr. Dunnick. Thank you. Good morning. Thank you for this
opportunity to share my support for H.R. 1795 with you. I also
appreciate the leadership shown by Mr. Burr and Ms. Eshoo in
support of this legislation.
I am Reed Dunnick. I currently serve as the Chair of the
Department of Radiology at the University of Michigan. Prior to
coming to Michigan in 1992, I served on the faculties at
Stanford and at Duke. In addition, I spent 4 years as a staff
radiologist in diagnostic radiology at the National Institutes
of Health.
The Congress has recognized the structural impediments to
imaging research that exist at the NIH in the conference report
on H.R. 3194, the Consolidated Appropriations Act for Fiscal
Year 2000. The language in that conference report is a good
summary of the current situation at the NIH.
Continued advances in biomedical imaging and engineering,
including the development of new techniques and technologies
for both clinical applications and medical research, and the
transfer of new technologies from research projects to the
public health sector are important. The disciplines of
biomedical imaging and engineering have broad applications to a
range of disease processes and organ systems, and research in
these fields does not fit into the current disease and organ
system organizational structure of the NIH.
The present organization of the NIH does not accommodate
basic scientific research in these fields and encourages
unproductive diffusion of imaging and engineering research.
Several efforts have been made in the past to fit imaging into
the NIH structure, but these have proved to be inadequate.
This congressional report is correct. The current structure
of the NIH does not promote basic research in medical imaging
and bioengineering, two disciplines that have become critical
to improving health care. The next logical step suggested by
this congressional finding is the creation of a National
Institute of Biomedical Imaging and Bioengineering as proposed
in H.R. 1795.
The imaging science community has worked with the NIH
leadership for three decades to fit imaging into the existing
NIH organizational structure of individual institutes dedicated
to specific disease processes and organ systems. However,
nothing short of an institute will be effective in stimulating
and coordinating biomedical research to the extent that it is
needed.
The imaging community has not proposed the establishment of
a new institute lightly. We recognize and agree that the bar
for structural change should be set high. For that reason we
looked to the National Academy of Science's Institute of
Medicine in their 1984 report titled Responding to Health Care
Needs and Scientific Opportunity: The Organizational Structure
of the National Institutes of Health.
They recommend a new institute when each of the following
five criteria are met: One, the activity is compatible with the
mission of the NIH; two, the research area in question is not
receiving adequate attention; three, there are reasonable
prospects for scientific growth; four, there are reasonable
prospects of sufficient funding; and five, the proposed
structural change will improve communication, management,
priority setting and accountability.
The proposed National Institute of Biomedical Imaging and
Bioengineering is consistent with these criteria. In
identifying imaging as one of its top research priorities, the
NCI has stated that this field is compatible with the mission
of the NIH. The NCI has indeed increased its level of support
for biomedical imaging in recent years. However, even with this
increase in resources, the amount of moneys carried out by
radiology departments throughout the country account for less
than 1 percent of the NIH budget.
Finally, the proposed institute, which would include a
division to coordinate imaging research throughout the Federal
Government, would certainly improve communication and
management in a field in which these qualities are sorely
lacking.
Mr. Chairman, breakthroughs in medical imaging have
revolutionized the way in which physicians detect, diagnose and
treat disease. Imaging holds the promise of further advances
that will move us into an era of noninvasive medicine. To reach
that goal, however, we need to create a climate that promotes
discovery and innovation in imaging just as the NIH provides
such a climate for other fields.
For that reason I urge the committee to support the
establishment of the National Institute of Biomedical Imaging
and Engineering. Thank you.
[The prepared statement of N. Reed Dunnick follows:]
Prepared Statement of N. Reed Dunnick, Professor and Chair, Department
of Radiology, University of Michigan Hospitals
Good morning. Thank you for this opportunity to share my support
for H.R. 1795 with you. My name is Reed Dunnick, and I chair the
Radiology Department at the University of Michigan. Prior to coming to
Michigan in 1992, I served on the faculties at Stanford and Duke. In
addition, I spent four years as a staff radiologist in the Diagnostic
Radiology Department at the National Institutes of Health.
Like everyone in my discipline and throughout the imaging sciences,
I was extremely pleased that the Congress recognized the structural
impediments to imaging research that exist at the NIH in the Conference
Report on H.R. 3194, the Consolidated Appropriations Act for Fiscal
Year 2000. The language in that Conference Report is a good summary of
the current situation at the NIH:
``Continued advances in biomedical imaging and engineering,
including the development of new techniques and technologies for both
clinical applications and medical research and the transfer of new
technologies from research projects to the public health sector are
important. The disciplines of biomedical imaging and engineering have
broad applications to a range of disease processes and organ systems
and research in these fields does not fit into the current disease and
organ system organizational structure of the NIH. The present
organization of the NIH does not accommodate basic scientific research
in these fields and encourages unproductive diffusion of imaging and
engineering research. Several efforts have been made in the past to fit
imaging into the NIH structure, but these have proved to be
inadequate.''
This Congressional report is correct. The current structure of the
NIH does not promote basic research in medical imaging and
bioengineering, two disciplines that have become critical to improving
health care. The logical next step suggested by this Congressional
finding is the creation of a National Institute of Biomedical Imaging
and Bioengineering as proposed in H.R. 1795. Like my colleagues Dr.
Hillman and Dr. Bryan, I have been involved in imaging research at the
NIH for many years. The imaging science community has worked with the
NIH leadership for three decades to fit imaging into the existing NIH
organizational structure of individual institutes dedicated to specific
disease processes and organ systems.
During that period, numerous plans have been tried. In 1982, for
example, most extramural research in imaging was transferred from the
National Institute of General Medical Sciences to the National Cancer
Institute with the understanding that NCI would support imaging
research beyond that related to cancer. However, the NCI has focused
almost exclusively on cancer imaging.
On the intramural side, the Laboratory of Diagnostic Radiology
Research (LDRR) was transferred from the Office of the Director of the
NIH to the Clinical Center a couple of years ago. This move was an
improvement but far from a solution because the Clinical Center lacks
the research mandate and resources to allow LDRR to achieve its full
promise.
Despite good will on both sides, these initiatives did not solve
the problems faced by either extramural or intramural imaging
investigators. Only after such repeated efforts failed did the imaging
community conclude that the NIH structure is not compatible with an
effective imaging research program and that creation of a new institute
is justified and necessary. Nothing short of an Institute will be
effective in stimulating and coordinating biomedical research to the
extent that is needed.
The imaging community has not proposed the establishment of a new
institute lightly. We recognize and agree that the bar to structural
change at the NIH should be set high. For that reason, we looked to
neutral, expert guidance, which we found in the National Academy of
Sciences' Institute of Medicine. In a 1984 report titled Responding to
Health Needs and Scientific Opportunity: The Organizational Structure
of the National Institutes of Health, issued in response to a
Congressional directive, the IOM concluded that new institutes should
be created only in unique circumstances when the following five
criteria are met:
(1) the activity is compatible with the mission of the NIH;
(2) it can be demonstrated that the research area in question (defined
either as a disease or health problem or as a biomedical or
behavioral process related to a health problem) is not
receiving adequate attention;
(3) there are reasonable prospects for scientific growth in the
research area;
(4) there are reasonable prospects of sufficient funding for the new
organization;
(5) the proposed structural change will improve communication,
management, priority setting, and accountability.
The proposed National Institute of Biomedical Imaging and
Bioengineering is consistent with these criteria. In identifying
imaging as one of its top research priorities, the NCI has stated
clearly that this field is compatible with the mission of the NIH and
that there are reasonable, even extraordinary, prospects for scientific
growth. The NCI has increased its level of support for the Biomedical
Imaging Program in recent years. Even with this increased commitment of
resources, however, extramural support for imaging research carried out
by Radiology Departments continues to account for substantially less
than one percent of the NIH budget--a level that is inadequate for an
``Extraordinary Opportunity for Investment.'' Finally, the proposed
institute, which would include a division to coordinate imaging
research throughout the federal government, would certainly improve
communication and management in a field in which these qualities are
sorely lacking.
Two years ago, again in response to a Congressional mandate, the
IOM issued another report that addressed the question of structural
change. In its 1998 report titled Scientific Opportunities and Public
Needs: Improving Priority Setting and Public Input at NIH, the IOM
recommended that ``The U.S. Congress should use its authority to
mandate specific research programs, establish levels of funding for
them, and implement new organizational entities only when other
approaches have proven inadequate.'' The imaging community agrees with
this recommended limitation on Congressional action and believes firmly
that the proposed National Institute of Biomedical Imaging and
Bioengineering is consistent with it.
In addition, and just as important, the proposed institute is also
completely consistent with the NIH's own rationale for the elevation of
the National Center for Human Genome Research to institute status in
1997. According to the statement issued by the NIH to announce the
creation of the National Human Genome Research Institute, ``As an
institute, NHGRI can more appropriately interact with other Federal
agencies, and develop collaborations with industry, academia, and
international organizations.''
The same is true for imaging. The proposed institute would
coordinate imaging research throughout the federal government, a
mission that cannot be accomplished successfully by a lower level
office or organization lacking sufficient institutional stature and
authority to deal effectively with cabinet-level departments.
Similarly, industry-government collaborations are essential to the
development of new imaging technologies. The proposed institute would
assist industry in setting research priorities and assessing potential
fields for development. The National Electrical Manufacturers
Association, which represents the companies that produce imaging
devices, supports this proposal.
This matter of coordinating imaging outside the NIH is important.
Other federal agencies such as NSF, NASA, the intelligence agencies,
and the Departments of Defense, Commerce, and Energy all support
imaging research. There is little or no coordination of these efforts.
In one key area, telemedicine, the General Accounting Office found a
couple of years ago that more than 35 separate federal organizations in
nine different departments have sponsored telemedicine initiatives.
Despite a major federal investment of $646 million over three years,
the GAO found that ``no formal mechanism or overall strategy exists to
ensure that telemedicine development is fully coordinated among federal
agencies to serve a common purpose.'' According to the GAO, the Joint
Working Group on Telemedicine, created in response to a directive from
the Vice President, encountered serious difficulties even in developing
a federal inventory of telemedicine programs.
This lack of coordination not only encourages duplication and
inefficiency, it impedes the transfer of imaging technologies from
research projects to the public health care sector. The proposed
institute could do much to ensure that federal research dollars are
expended more efficiently and productively.
The National Institute of Biomedical Imaging and Bioengineering
would also provide a focused effort to meet the medical needs of the
information age. It has been estimated that computerization
requirements in the coming decade will double just to keep pace with
developments in all segments of society. The largest component of that
need will be in the area of medical imaging information. In addition to
the enormous amount of imaging-related, unprocessed information
generated, there will be a need to process and analyze the huge date
sets produced by medical imaging to improve the medical value of those
data to the referring physicians who will access the network. An
imaging institute would support research focused on the acquisition,
transmission, processing, and optimal display of images.
Mr. Chairman, breakthroughs in medical imaging have revolutionized
the way in which physicians detect, diagnose, and treat disease in the
past two decades. Imaging holds the promise of further advances that
will move us into an era of non-invasive medicine. To reach that goal,
however, we need to create a climate that promotes discovery and
innovation in imaging just as the NIH provides such a climate for other
fields. For that reason, I urge the Committee to support the National
Institute of Biomedical Imaging and Engineering Establishment Act.
Mr. Upton. Thank you very much.
Dr. Hillman.
STATEMENT OF BRUCE J. HILLMAN
Mr. Hillman. Good morning. I am Dr. Bruce Hillman, Chair of
the Department of Radiology at the University of Virginia, and
I am also a Chancellor of the American College of Radiology. I
am grateful to the committee, especially to the bill's sponsors
Mr. Burr and Ms. Eshoo and my own Congressman Chairman Bliley,
for the invitation to testify in support of H.R. 1795, the
National Institute of Biomedical Imaging and Engineering
Establishment Act.
Medicine now stands at the threshold of a new and exciting
revolution in how we think about disease. It is the molecular
revolution wherein we will develop the tools needed to diagnose
and treat disease at its earliest stages when the chances of
success are likely to be much greater than currently.
Medical imaging must be a critical element in this new
paradigm. Medical imaging is the noninvasive biopsy that will
detect alterations in the genetic or molecular makeup of cells
that have the potential to progress to disease. Imaging
technologies will precisely determine what faction of cells are
affected. Finally, medical imaging technologies will be
integrated with new therapeutic methods to either guide or
monitor treatment so that only diseased cells are treated while
preserving normal tissue.
The basic knowledge exists to begin to implement this
vision; however, the current means by which the NIH institutes
address imaging research is as a stepchild, a part of the
research portfolio of nearly all of the institutes, but the
principal focus of none. As a result, basic research into the
development of new imaging technologies has been subject to
overlap and duplication, inefficient use of resources and lost
opportunities.
The initial invention of and basic research into new
technologies that have emerged in recent times such as CAT
scanning, MRI, and image-guided interventional methods have
most frequently occurred outside the U.S. where the logistics
and funding of basic research into medical imaging can be
handled in a more straightforward and integrated fashion.
The establishment of a new Institute of Biomedical Imaging
and Bioengineering would correct many of these structural
problems. The institute would address the needs of imaging
research directly and comprehensively. It would provide a home
and focus for the fundamental disciplines of computer sciences,
physics and engineering that are so inextricably connected to
progress in imaging research. It would foster basic imaging
research lacking in the current NIH structure that would lead
to the more efficient development of the new broad-ranging
technologies applicable to my vision of molecular medicine.
Through relationships the new institute will develop with
regulatory agencies and industry, it would facilitate
technology transfer, allowing important innovations to more
rapidly be employed in medical care. And very significantly the
new institute would foster the more rapid assessment of new
technologies as they enter practice to ensure that their use is
appropriate and cost-effective.
This last aspect of the institute's proposed functions is
critical and is yet another example of how the current
institutional structure insufficiently addresses the needs of
the American public.
Among my other responsibilities, I am Chair of the American
College of Radiology Imaging Network, or ACRIN. ACRIN is a
National Cancer Institute-funded cooperative group that through
clinical trials gathers information to extend and improve the
quality of lives of cancer patients.
ACRIN is a remarkable endeavor that evaluates the
effectiveness of imaging technologies in improving health
outcomes for individual patients and measures the balance of
benefit and costs the American public receives for its
expenditure on cancer imaging.
The results of major definitive ACRIN trials of such
technologies as digital mammography, a screen for breast
cancer, and CAT scan for the detection of early lung cancer
will guide medical practice and reimbursement in the years to
come.
The National Cancer Institute should be applauded for its
vision in establishing ACRIN less than 2 years ago. Yet again,
these same technologies that ACRIN will study and many other
current and future technologies are broadly applicable to
diseases other than cancer.
There is no counterpart to ACRIN at any of the other
institutes. Even if there were, the fragmentation of imaging
technology assessment on such arbitrary grounds would be
wasteful, inefficient and leave important gaps.
The structural inadequacies that hinder imaging research
can be rectified only through an institute. Institutes are the
standard NIH administrative units for areas of such significant
scientific research. Any entity devoted to biomedical imaging,
bioengineering, and related fields will necessarily be of the
size that will make any organizational unit short of an
institute inappropriate.
Related research occurs in numerous Federal agencies such
as the Departments of Defense and Energy, and the National
Science Foundation. A subordinate administrative unit would
lack the stature necessary to coordinate research involving
imaging outside of NIH. For these reasons and for those I have
detailed in this testimony, I hope you will vote favorably on
H.R. 1795 and pass it on to the full House of Representatives
for its consideration. Thank you.
[The prepared statement of Bruce J. Hillman follows:]
Prepared Statement of Bruce J. Hillman, Chair, Department of Radiology,
University of Virginia School of Medicine
Good morning. I am Dr. Bruce Hillman, Chair of the Department of
Radiology at the University of Virginia School of Medicine and a
Chancellor of the American College of Radiology. I greatly appreciate
your invitation so that I may testify in support of H.R. 1795, the
National Institute of Biomedical Imaging and Engineering Establishment
Act.
During the past thirty years, no specialty of medicine has advanced
technologically so much as medical imaging. And no specialty of
medicine has so drastically and positively changed the way we detect,
diagnose, stage, and treat disease. The introduction during that time
period of such computer-based technologies as CAT scanning and MRI, and
the use of imaging to guide a host of interventional procedures has
revolutionized medicine. As a result, the preferred diagnosis and
treatment methods for a broad range of diseases--cancer, heart disease,
orthopedic injuries, and infectious diseases, to name just a few--has
become less invasive, less expensive, and less risky for patients.
Medicine now stands at the threshold of a new and exciting
revolution in how we think about disease. It is the molecular
revolution, wherein we will develop the tools needed to diagnose and
treat disease at its earliest stages, when the chances of success are
likely to be much greater than currently. Virtually everyone agrees
that medical imaging must be a critical element in this new paradigm.
Medical imaging is the ``non-invasive biopsy'', the method of
disease quantitation, the guidance for new treatments still to be
developed that will form the underpinnings of molecular medicine. Under
this scenario, new medical imaging technologies will detect alterations
in the genetic or molecular makeup of cells that have the potential to
progress to disease. We then will employ imaging technologies to
precisely determine what fraction of cells are so affected. Finally,
medical imaging technologies will be integrated with new therapeutic
methods to either guide or monitor treatment, so that we can much more
precisely than ever before ensure that only diseased cells are treated
while preserving normal tissue.
The basic knowledge exists to begin to implement this vision.
However, for this optimistic and exciting prophecy to come to fruition,
we will need to invest in the development and assessment of new imaging
technologies. We are ill-equipped to do so under the current NIH
organizational structure that focuses the work of existing institutes
on specific organ systems and diseases.
Imaging technologies, with few exceptions, are multi-potential.
They are applicable to many organ systems and diseases. Their value in
detection, diagnosis, staging, and treatment of disease cuts across
traditional institute lines. The leadership of existing institutes do
not generally have training or expertise in imaging. The requests for
applications and the public announcements that are the principal
instruments used by institutes to guide research in their respective
fields view imaging more as a tool to address disease-specific
questions than as a focus for research in its own right. The critical
research training of new young imaging investigators, when it is
supported at all, is forced into unnatural pathways that address
imaging technology research obliquely rather than head-on.
Thus, the current means by which the NIH institutes address imaging
research is as a ``stepchild''--a part of the research portfolio of
nearly all of the institutes but the principal focus of none. As a
result, basic research into the development of new imaging technologies
has been subject to overlap and duplication, inefficient use of
resources, and lost opportunities. The initial invention and basic
research into new technologies that have emerged in recent times--such
as CAT scanning, MRI, and image-guided interventional methods--have
most frequently occurred outside the U.S. where the logistics and
funding of basic research into medical imaging can be handled in a more
straightforward fashion.
The establishment of a new Institute of Biomedical Imaging and
Bioengineering would correct many of these structural problems. The
Institute would:
address the needs of imaging research directly and
comprehensively;
provide a home and focus for the fundamental disciplines of
computer sciences, physics, and engineering that are so
inextricably related to progress in imaging research;
provide a home for basic imaging research--lacking in the
current NIH structure--that would lead to the more efficient
development of the new, broad-ranging technologies applicable
to the vision of molecular medicine that I have detailed
earlier;
through relationships the new institute will develop with
regulatory agencies and industry, facilitate technology
transfer, allowing important innovations to more rapidly be
employed in medical care;
and very significantly, foster the more rapid assessment of
new technologies as they enter practice, to ensure that their
use is appropriate and cost-effective.
This last aspect of the Institute's proposed functions is critical
and is yet another example of how the current institutional structure
insufficiently addresses the needs of the American public.
Among my other responsibilities, I am the chair of the American
College of Radiology Imaging Network, or ACRIN. ACRIN is a National
Cancer Institute-funded cooperative group that conducts clinical trials
of medical imaging technologies at community practices and academic
health centers across the country. The overriding goal of ACRIN is,
through clinical trials, to gather information that will extend and
improve the quality of the lives of cancer patients.
ACRIN is a remarkable endeavor that evaluates the effectiveness of
imaging technologies in improving health outcomes for individual
patients and measures the balance of benefit and costs the American
public receives for its expenditures on medical imaging. The results of
major, definitive, ACRIN trials of such technologies as digital
mammography for screening for breast cancer, CAT scanning for the
detection of early lung cancer, and the use of PET scanning for
patients' response to chemotherapy will guide medical practice and
reimbursement in the years to come.
The National Cancer Institute should be applauded for its vision in
establishing ACRIN less than two years ago. Yet again, these same
technologies that ACRIN will study, and many other current and future
technologies, are broadly applicable to diseases other than cancer.
There is no counterpart to ACRIN at the National Heart, Lung, and Blood
Institute, or the National Institute for Neural Diseases and Stroke--
institutes whose purview includes organ systems and diseases where
imaging plays a large and critical role--nor, for that matter, at any
of the other institutes. Even if there were, the fragmentation of
imaging technology assessment on such arbitrary grounds would be
wasteful, inefficient, and leave important gaps.
The unification of imaging technology assessment activities under
the aegis of the proposed Institute obviates these concerns. It ensures
that assessment activities critical to both the health and financial
well-being of the American public will be orderly, strategic, coherent,
and efficient and that they will best advise us on how to most wisely
expend our resources on imaging technologies as they are employed in
medical practice.
The structural inadequacies that hinder imaging research can be
rectified only through an institute. Institutes are the standard NIH
administrative units for areas of such significant scientific research.
An entity devoted to biomedical imaging, bioengineering, and related
fields would necessarily be of a size that would make any
organizational unit short of an institute inappropriate. Related
research occurs in numerous federal agencies, such as the Departments
of Defense and Energy and the National Science Foundation. A
subordinate administrative unit would lack the stature necessary to
coordinate research involving imaging outside of NIH.
For these reasons and those I have detailed in this testimony, I
hope that you will vote favorably on HR 1795 and pass it on to the full
House of Representatives for its consideration.
Mr. Bilirakis. Thank you, Dr. Hillman.
Ms. Tomiko Fraser is the national spokesperson for the
Lupus Foundation of America.
Thank you very much for being here today.
STATEMENT OF TOMIKO FRASER
Ms. Fraser. Good morning, Mr. Chairman and members of the
subcommittee. I appear before you today representing the Lupus
Foundation of America on behalf of the 1.4 million Americans
who have lupus erythematosus, a devastating disease that causes
the immune system to attack the body's own cells and organs.
Unfortunately, one of the victims of lupus is my younger sister
Shneequa, who has a very serious case of lupus that affects her
brain. The disease has been so devastating to Shneequa that she
must receive around-the-clock care at a skilled nursing
facility.
That is why I have agreed to serve as the national
spokesperson for the Lupus Foundation of America. I want to
help educate all Americans about the devastating impact lupus
has on its victims.
I urge Congress to pass H.R. 762, the Lupus Research and
Care Amendments Act of 1999. Congresswoman Carrie Meek, who
lost a sister to lupus, introduced this legislation. Two
hundred forty-three Members of the U.S. House of
Representatives are cosponsors of H.R. 762.
The legislation authorizes a $23 million increase to the
current funding level for lupus medical research supported
through the National Institutes of Health. It also authorizes
$75 million to fund a grant program. This program would provide
local governments, community hospitals, and other nonprofit
health care facilities with a pool of funds so they could offer
lifesaving medical care to the poor or uninsured people with
lupus.
This grant program will help local communities hardest hit
by lupus, especially in medically underserved areas including
rural and urban communities where often there is a shortage of
medical facilities to treat people with lupus.
Lupus deserves special funding consideration. Lupus is the
prototypical autoimmune disease. Research on lupus benefits all
autoimmune diseases that disproportionately affect women.
Autoimmune diseases are the fourth leading cause of disability
among women.
Lupus is an expensive disease to treat. The cost to provide
medical care for a person with lupus averages between 6- and
$10,000 annually. The Lupus Foundation of America estimates the
economic impact of lupus on the Federal Treasury to be several
billion dollars every year. These costs include disability
income payments to the tens of thousands of lupus victims
disabled every year by the disease. They also include the cost
of government-sponsored medical care provided through the
Medicare and Medicaid programs and uncollected tax revenue due
to lost wages when individuals with lupus are unable to work.
The Lupus Research and Care Amendments Act of 1999 is a
bipartisan effort to address an urgent national health care
crisis that inflicts an enormous burden on individuals,
families, the business community, the Federal Government and
society. Many scientific opportunities exist, but current
funding levels can support only one in four of the promising
studies submitted for funding that eventually lead to a cure
for lupus. By accelerating medical research for lupus now,
Congress will reduce future health care costs and save billions
of dollars for the Social Security and Medicare Trust Funds in
future years.
Lupus is a complicated and mysterious disease that needs
extensive study. Presently there is no cure for lupus, nor do
researchers fully understand what causes the disease. We do not
know why lupus alternates between periods of remission and
periods of disease activity called flares. We do not know why
the disease can remain mild in some individuals and become
life-threatening in others. What we do know, Mr. Chairman, is
that lupus has a devastating impact on its victims and their
families. We know that lupus causes debilitating health effects
including extreme joint pain and swelling, constant fevers,
overwhelming fatigue, horrible skin rashes, organ failure and a
host of other devastating symptoms.
Lupus destroys the quality of life for many of its victims.
The disease can severely damage the kidneys, heart, lungs and
other vital organs. Lupus disables one in five of its victims,
often at a very young age, and tragically every year thousands
of lupus victims die from complications of the disease.
Lupus is not an equal opportunity disease. Ninety percent
of the victims of lupus are women. Also, lupus is more common
among women of color. Lupus is two to three times more likely
to affect African Americans, Hispanics, Asians and Native
Americans than Caucasian women. Lupus also appears to be more
serious among African American women.
Approximately 20 percent of lupus cases begin in childhood.
Unfortunately, lupus is more severe in children. Nearly 70
percent of children with lupus have kidney disease as opposed
to 30 percent of adults who develop lupus. Whereas half of
those with adult onset lupus have organ-threatening disease,
nearly 80 percent of those with childhood onset lupus go on to
develop organ-threatening conditions.
Lupus strikes women in the their childbearing years between
the ages of 15 and 44. This is one of the most devastating
realities of lupus. It destroys the quality of life during a
time when young women should be enjoying their best health.
Many people with lupus suffer 3 to 5 years, visiting 5 or
more doctors before they receive a correct diagnosis. Many
medical schools do not provide family physicians with
sufficient training to diagnose lupus. By the time some lupus
patients are diagnosed, especially in poor or rural
communities, irreversible damage to vital organs may have
already occurred. This increases the need for expensive
treatments such as kidney dialysis or transplantation.
Medical researchers have made progress, and there is great
hope for new discoveries. Still, most lupus patients are
frustrated that the disease remains incurable.
As you can imagine, Mr. Chairman, lupus is not an easy
disease to live with. Over a million American families are
struggling to cope with lupus every day of their lives. I know
this personally from watching my sister suffer from the
devastating effects of the disease.
It is time for action. A majority of Members of the U.S.
House of Representatives, a total of 243, are cosponsor of H.R.
762. I urge that this legislation be brought to the floor of
the House for a vote as soon as possible.
Thank you for the opportunity to represent the victims of
lupus at today's hearing, and I will be happy to answer any of
your questions. And thank you for the extra time.
[The prepared statement of Tomiko Fraser follows:]
Prepared Statement of Tomiko Fraser, National Spokesperson, Lupus
Foundation of America
Good Morning Mr. Chairman, and Members of the Subcommittee. I
appear before you today representing the Lupus Foundation of America on
behalf of 1.4 million Americans who have lupus erythematosus, a
devastating disease that causes the immune system to attack the body's
own cells and organs.
Unfortunately, one of the victims of lupus is my younger sister,
Shneequa, who has a very serious case of lupus that affects her brain.
The disease has been so devastating to Shneequa that she must receive
around-the-clock care at a skilled-nursing facility.
That is why I have agreed to serve as a National Spokesperson for
the Lupus Foundation of America. I want to help educate all Americans
about the devastating impact lupus has on its victims.
I urge Congress to pass H.R. 762, the Lupus Research & Care
Amendments Act of 1999. Congresswoman Carrie Meek, who lost a sister to
lupus, introduced this legislation. 243 members of the U.S. House of
Representatives are cosponsors of H.R. 762. The legislation authorizes
a $23 million increase to the current funding level for lupus medical
research supported through the National Institutes of Health. It also
authorizes $75 million to fund a grant program. This program would
provide local governments, community hospitals, and other non-profit
health care facilities with a pool of funds so they could offer life-
saving medical care to poor or uninsured people with lupus.
This grant program will help local communities hardest hit by
lupus, especially in medically under-served areas, including rural and
urban communities where often there is a shortage of medical facilities
to treat people with lupus.
Lupus deserves special funding consideration. Lupus is the
prototypical autoimmune disease. Research on lupus benefits all
autoimmune diseases that disproportionately affect women. Autoimmune
diseases are the fourth leading cause of disability among women.
Lupus is an expensive disease to treat. The cost to provide medical
care for a person with lupus averages between six and ten thousand
dollars annually. The Lupus Foundation of America estimates the
economic impact of lupus on the federal treasury to be several billion
dollars every year. These costs include disability income payments to
the tens of thousands of lupus victims disabled every year by the
disease. They also include the cost of government-sponsored medical
care provided through the Medicare and Medicaid programs, and
uncollected tax revenue due to lost wages when individuals with lupus
are unable to work.
The Lupus Research and Care Amendments Act of 1999 is a bipartisan
effort to address an urgent national health care crisis that inflicts
an enormous burden on individuals, families, the business community,
the federal government, and society. Many scientific opportunities
exist, but current funding levels can support only one in four of the
promising studies submitted for funding that eventually will lead to a
cure for lupus. By accelerating medical research for lupus now,
Congress will reduce future health care costs and save billions of
dollars for the Social Security and Medicare trust funds in future
years.
Lupus is a complicated and mysterious disease that needs extensive
study. Presently there is no cure for lupus, nor do researchers fully
understand what causes the disease.
We do not know why lupus alternates between periods of remission
and periods of disease activity, called flares. We do not know why the
disease can remain mild in some individuals and become life-threatening
in others.
What we do know, Mr. Chairman, is that lupus has a devastating
impact on its victims and their families. We know that lupus causes
debilitating health effects including extreme joint pain and swelling,
constant fevers, overwhelming fatigue, horrible skin rashes, organ
failure, and a host of other devastating symptoms. Lupus destroys the
quality of life for many of its victims. The disease can severely
damage the kidneys, heart, lungs, and other vital organs. Lupus
disables one in five of its victims, often at a very young age. And
tragically, every year thousands of lupus victims die from
complications of the disease.
Lupus is not an equal opportunity disease. Ninety percent of the
victims of lupus are women. Also, lupus is more common among women of
color. Lupus is two to three times more likely to affect African-
Americans, Hispanics, Asians and Native-Americans than Caucasian women.
Lupus also appears to be more serious among African-American women.
Approximately 20% of lupus cases begin in childhood. Unfortunately,
lupus is more severe in children. Nearly 70% of children with lupus
have kidney disease, as opposed to 30% of adults who develop lupus.
Whereas half of those with adult-onset lupus have organ-threatening
disease, nearly 80% of those with childhood-onset lupus go on to
develop organ-threatening conditions.
Lupus strikes women in their child-bearing years, between the ages
of 15 and 44. This is one of the most devastating realities of lupus--
it destroys the quality of life during a time when young women should
be enjoying their best health.
Many people with lupus suffer three to five years, visiting five or
more doctors, before they receive a correct diagnosis. Many medical
schools do not provide family physicians with sufficient training to
diagnose lupus. By the time some lupus patients are diagnosed,
especially in poor or rural communities, irreversible damage to vital
organs may already have occurred. This increases the need for expensive
treatments, such as kidney dialysis or transplantation.
Medical researchers have made progress; and there is great hope for
new discoveries. Still, most lupus patients are frustrated that the
disease remains incurable.
As you can imagine, Mr. Chairman, lupus is not an easy disease to
live with. Over a million American families are struggling to cope with
lupus every day of their lives. I know this personally from watching my
sister suffer from the devastating effects of this disease.
It's time for action. A majority of members of the United States
House of Representatives--a total of 243--are cosponsors of H.R. 762. I
urge that this legislation be brought to the floor of the House for a
vote as soon as possible. Thank you for the opportunity to represent
the victims of lupus at today's hearing. Now I will be happy to answer
your questions.
Mr. Bilirakis. Thank you very much, Ms. Fraser.
Dr. Bryan.
STATEMENT OF R. NICK BRYAN
Mr. Bryan. Good morning. My name is Nick Bryan. I currently
serve as professor and Chairman of the Department of Radiology
at the University of Pennsylvania. I really appreciate this
opportunity to share some of my experiences as an imaging
researcher and a former NIH staff member with you and to
express my support for H.R. 1795.
I would like to thank you, Mr. Chairman, and the committee
leadership for holding this hearing, and I would like to thank
the sponsors of the bill, Mr. Burr and Mrs. Eshoo, for their
leadership and efforts on this issue.
You have already heard about the importance and uniqueness
of biomedical imaging and engineering, and I will not belabor
the point. I will instead focus on what I view as current
structural inadequacies to support this field in NIH.
Prior to coming to Penn, I served for 2 years as Director
of Diagnostic Radiology and Associate Director, Imaging
Sciences Program, at the Warren G. Magnuson Clinical Center at
the NIH. During my tenure, and with superb support from Dr.
John Gallen, director of the clinical center, we were able to
consolidate several disparate imaging departments into a
unified imaging sciences program, which elevated the status of
imaging research on the NIH campus and began to lay a
foundation for an advanced research program.
In the final analysis, though, I felt the imaging sciences
program could not be wholly successful mainly because the very
structure of the NIH makes such an endeavor problematic.
Research authority and resources reside in the institutes, not
in programs at the clinical center. As a result, the success of
our imaging research was ultimately dependent on the ability of
me and my colleagues to convince one or more of the institutes,
institutes whose primary missions and priorities are in areas
other than imaging, to divert funds from their main activities
and commit those funds to imaging research.
I accepted the position at the clinical center knowing that
it involved a significant challenge, but in the hope and in the
belief that an effective imaging research program could be
developed within the parameters of the NIH structure. In fact,
at that time I was skeptical about the need for a new
institute. My experience, however, gradually changed my opinion
and convinced me that the existing NIH organization will not
work optimally for imaging in bioengineering.
Ultimately, my decision to leave the NIH owed much to the
inherent obstacles to imaging research that are built into its
structure. It should be recognized that the NIH does
acknowledge the importance of imaging and has taken steps to
make imaging research a more visible part of its portfolio, as
you heard. And, for instance, the National Cancer Institute has
authorized significant expansion of the extramural biomedical
imaging program.
The NIH Biomedical Engineering Consortium, known as BECON,
sponsored a conference in 1999 entitled Biomedical Imaging
Symposium: Visualizing the Future of Biology in Medicine. This
year the NIH, in response to a congressional mandate, has begun
to organize a new Office of Bioimaging, Bioengineering and
Bioimformatics in the Office of the Director of the NIH. The
new office is to provide focus for and facilitate work in our
fields.
Unfortunately, all of these initiatives suffer from major
flaws. First, the NCI program applies real resources to
imaging, but the research is limited to cancer imaging. Cancer
imaging is clearly important and should be extremely high
priority, but imaging, as I have said, is not disease- or
organ-system-specific. It has applications far beyond cancer,
applications that are neglected when the research focus is on
cancer or any other individual disease.
Initiatives such as BECON and OB3, as it is called, the new
office, constitute a useful effort to identify research
opportunities and focus attention on imaging, but they bring
little in the way of actual research dollars to imaging
research. They represent a strong commitment by the NIH to
identify potentially fruitful areas of research, but no
commitment at all to supporting that research.
The Director of the OBBB will have to do what I did. He or
she will have to pass the hat by the current institutes for
contributions, and I am certain that the donations will be
insufficient to support a robust imaging research program.
In fact, it is unrealistic and perhaps even inappropriate
to expect existing disease and organ system institutes to
divert resources from their primary missions in order to
support basic research to advance the science of imaging. For
these reasons I believe that the creation of a National
Institute of Biomedical Imaging and Bioengineering is essential
to promote the development of new imaging techniques and
technologies.
In order to flourish and grow consistently at the NIH, a
scientific field requires organizations with the mandate, the
responsibility, the authority, and the resources to direct and
drive investigation in that field. In the NIH structure,
institutes possess those attributes.
I would like to conclude by noting that my opinions are not
alone. Nearly all of radiology and bioengineering supports this
is initiative. During the current year, I am also privileged to
serve as Chairman of the Board of Directors of the Radiological
Society of North America. The RSNA is the largest radiological
organization in the world, with a membership of more than
30,000 radiologists, physicists, and allied scientists. The
RSNA and more than 40 other professional organizations
representing physicians, radiologic technologists, bioengineers
and imaging scientists have joined coalitions that support H.R.
1795. The total individual membership of these organizations is
well over 100,000.
All of us believe that this is the time to create a
National Institute of Biomedical Imaging and Bioengineering to
support a field of inquiry that is central to continued
progress in advanced research in biomedicine as well as the
development of better systems for delivery of health care. This
institute would be good for patients, physicians, and the NIH
itself.
I urge the subcommittee to approve this bill. I would be
pleased to answer any questions.
[The prepared statement of R. Nick Bryan follows:]
Prepared Statement of R. Nick Bryan, Chairman, Department of Radiology,
University of Pennsylvania Health Systems
Good morning. My name is Nick Bryan. I have been a radiologist
specializing in neuroradiology for more than 25 years. I currently
serve as Chairman of the Department of Radiology and Eugene P.
Pendergrass Professor of Radiology at the University of Pennsylvania.
Prior to coming to Penn, I served for two years as Director of
Diagnostic Radiology and Associate Director, Radiologic and Imaging
Sciences Program, at the Warren G. Magnuson Clinical Center at the
National Institutes of Health.
During the current year I am also privileged to serve as Chairman
of the Board of Directors of the Radiological Society of North America.
The RSNA is the largest radiological organization in the world, with a
membership of more than 30,000 radiologists, physicists, and allied
scientists.
I appreciate this opportunity to share some of my experiences as an
imaging researcher and NIH staff member with you and to express my
support for H.R. 1795, the National Institute of Biomedical Imaging and
Engineering Establishment Act. I would like to thank you, Mr. Chairman,
and the Committee leadership for holding this hearing and the sponsors
of the bill, Representatives Burr and Eshoo, for their leadership and
efforts on this issue.
It is important to note that all of radiology and imaging supports
this initiative. More than 40 separate professional organizations
representing physicians, radiologic technologists, bioengineers, and
imaging scientists have joined coalitions that support H.R. 1795. The
total individual membership of these organizations is well over
100,000.
This is perhaps the most exciting time in the history of medical
imaging and, indeed, all of medicine. Breakthroughs in imaging have
allowed physicians to eliminate much surgery, including virtually all
exploratory surgery, and to diagnose disease at earlier and earlier
stages of development, when treatment is most effective. Because of
advances in imaging, patients receive more effective treatment, avoid
painful, expensive, and often dangerous surgical procedures, and live
longer.
The National Institutes of Health is the premier medical research
institution in the world and has been at the center of pathbreaking
research in most areas of medicine. In imaging, however, the NIH is
not--and under its present structure cannot be--the catalyst of imaging
innovation. The various institutes are focused on specific disease
processes or organ systems, but imaging cuts across those lines and is
broadly applicable to virtually all diseases and organ systems.
Consequently, imaging is used as a tool in all the institutes, but
there is no home at the NIH for the basic research that is essential to
develop new imaging techniques and technologies for the 21st century.
The basic science of imaging and bioengineering, it must be
remembered, is fundamentally different from that of the existing
institutes at the NIH. Imaging is based on mathematics and physics, not
the biological sciences that underly most of the research in the
current institutes. Imaging and bioengineering are unique scientific
fields at the NIH and are also critical to future advances in the
delivery of high quality health care.
While at the NIH, I directed intramural research efforts in imaging
in the Clinical Center. During my tenure, we were able to consolidate
several disparate imaging departments into the unified Imaging Sciences
Program (ISP), which elevated the status of imaging research on the NIH
campus and began to lay a foundation for an advanced research program.
In the final analysis, though, the ISP could not be wholly
successful, mainly because the very structure of the NIH makes such an
endeavor problematic. Research authority and resources reside in the
institutes, not in programs at the Clinical Center. As a result, the
success of imaging research proposals was ultimately dependent on the
ability of ISP researchers to convince one or more of the institutes--
institutes whose primary missions and priorities are in areas other
than imaging--to divert funds from their main activities and commit
those funds to imaging research. Even when imaging researchers are
successful, which sometimes requires artificially tailoring proposals
to create the appearance of disease- or organ-specific research, the
institutes are likely to assume practical control of projects and, in
all probability, recast the research to fit their own missions.
I accepted the position at the Clinical Center knowing that it
involved a significant challenge but in the hope, and in the belief,
that an effective imaging research program could be developed within
the parameters of the NIH structure. In fact, at that time I was
skeptical about the need for a new institute. My experience, however,
gradually changed my opinion and convinced me that the existing NIH
organization will not work for imaging. Ultimately, my decision to
leave the NIH owed much to the inherent obstacles to imaging research
that are built into its structure.
It should be recognized that the NIH has taken steps to make
imaging research a more visible part of its portfolio. The National
Cancer Institute, for example, has designated imaging as one of only a
few ``Extraordinary Opportunities for Investment'' and has authorized
significant expansion of the extramural Biomedical Imaging Program. The
Biomedical Imaging Program at the NCI, under the extremely able
leadership of Dr. Dan Sullivan, has benefited from growing staff
resources and new research initiatives.
In addition, the Bioengineering Consortium, known as BECON,
sponsored a conference in June 1999 titled ``Biomedical Imaging
Symposium: Visualizing the Future of Biology and Medicine.''
Participants produced an ambitious research agenda for imaging science
that calls for focused efforts in a number of fields:
--multidisciplinary research;
--imaging technology, probes, and contrast agents;
--education and training;
--clinical trials and informatics
--greater cooperation among the NIH, the Food and Drug Administration,
the Health Care Financing Administration, and private industry
to improve the speed with which new imaging technologies,
probes, and contrast agents are transferred to clinical
practice.
The 1999 BECON symposium was actually the second NIH-sponsored
conference in recent years devoted to imaging research. In 1994, the
NIH brought together more than 40 top researchers from inside and
outside government at a Conference on Developing a Long-term Plan for
Imaging Research. Conference participants developed a set of
recommended research goals in 33 separate areas of basic science, basic
and applied technology, and organ-based clinical research.
Finally, this year the NIH, in response to a Congressional mandate,
has begun to organize a new Office of Bioengineering, Bioimaging, and
Bioinformatics in the Office of the Director, NIH. According to the
Vacancy Announcement seeking candidates to direct the OBBB, the
Director of the new Office ``will provide a focus for stimulating and
coordinating the development of biomedical engineering, bioimaging and
bioinformatics activities among the 25 Institutes and Centers (ICs) at
the NIH; and will facilitate the overall planning, development, and
implementation of NIH biomedical engineering, bioimaging and
bioinformatics research programs and activities.''
Unfortunately, all of these initiatives suffer from one of two
fatal flaws. First, the NCI efforts apply real resources to imaging,
but all of the research is on cancer imaging. Cancer imaging clearly
should be an extremely high priority, but imaging, as I have said, is
not disease- or organ-system specific. It has applications far beyond
cancer--applications that are neglected when the research focus is on
cancer or any other individual disease.
ACRIN, the cooperative clinical trials group chaired by Dr.
Hillman, offers a clear example of the shortcomings of this approach.
ACRIN represents a significant and wise investment, but this
application of resources could produce so much more if imaging
technologies beyond cancer were included.
The ACRIN approach, valuable as it is, actually shortchanges cancer
research as well as the broader field of imaging. Evaluating the use of
existing techniques and technologies for the diagnosis and treatment of
breast, prostate, and other cancers will produce important knowledge
that will result in incremental improvements in patient care. But the
real breakthroughs that will produce quantum leaps forward are likely
to occur through the development of wholly new imaging modalities that
do not result from disease-specific research.
The second group of NIH initiatives--the 1994 conference, the BECON
symposium, and the creation of the OBBB--represents the second fatal
flaw. These initiatives constitute a useful effort to identify research
opportunities and focus attention on imaging, but they bring little in
the way of actual research dollars to imaging. They represent a strong
commitment by the NIH to identify potentially fruitful areas of
research but no commitment at all to supporting that research.
The Director of the OBBB will be, as I was as Director of the
Imaging Sciences Program, dependent on the goodwill and interest of the
other institutes. Again, these are institutes that do not have imaging
as primary missions and which are faced continually with competing
claims for scarce resources from within their primary research
constituencies.
The disciplines represented in the existing institutes use imaging,
but they cannot accommodate the basic science of imaging itself. It is
unrealistic, and perhaps even inappropriate, to expect the existing
disease and organ system institutes to divert resources from their
primary missions in order to support basic research to advance the
science of imaging.
Without grant-making and decision-making authority, these NIH
efforts to improve coordination can be marginally successful at best.
No one would suggest that the search for better treatments for cancer,
diabetes, or other diseases should be undertaken by organizations that
lack the capability to make research grants--or that progress in these
vital areas should be dependent on the ability of researchers to
convince other institutes to divert a portion of their resources away
from their chief responsibilities. Yet that is precisely the approach
the NIH has taken to imaging. In consequence, while the NIH might
consider the recommendations from the 1994 and 1999 imaging conferences
to be priorities, there is not much evidence that significant action
has been taken on this research agenda. The present organization of the
NIH makes such action unlikely.
For these reasons, I believe that the creation of a National
Institute of Biomedical Imaging and Bioengineering is essential to
promote the development of new imaging techniques and technologies. In
order to flourish and grow consistently at the NIH, a scientific field
requires an organization with the mandate, the responsibility, the
authority, and the resources to direct and drive investigation in that
field. In the NIH structure, only institutes possess those attributes.
Institutes, for example, can issue Requests for Applications (RFAs)
and Program Announcements (PAs) to initiate research projects and
direct resources toward investigations on specific subjects that offer
particular opportunities for scientific advances. Such institute-
initiated research projects represent a substantial portion of the
institutes' extramural research portfolios. Even smaller and mid-sized
institutes can exert considerable influence on the direction of
research. A recent analysis showed that the National Institute on Aging
(NIA), with only the 10th largest budget among the institutes and major
centers at the NIH, issued seven RFAs covering a wide variety of topics
with a total price tag of more than $16 million in Fiscal Year 1999. In
the same year, the NIA collaborated on 16 additional RFAs in which
other institutes were the primary sponsors. Without an institute,
imaging lacks this fundamental capability to guide and support the
research in this field.
Research training is another key issue. Training grants ensure the
continuity of a pool of trained researchers in the institutes' fields
of research. Under the current structure, training opportunities in
imaging are generally limited to grants that focus on the use of
imaging in connection with specific diseases and organ systems rather
than on training imaging scientists to conduct the basic research that
will produce new modalities. An analysis of NIH data on T32 grants, the
most common NIH training awards, found only three imaging awards of the
148 active T32 grants in the National Heart, Lung, and Blood Institute.
Moreover, the National Institute of Neurological Disorders and Stroke
(NINDS), which is the institute most closely related to my work in
neuroradiology, had no active imaging grants among its 44 T32 awards.
The National Institute of Biomedical Imaging and Bioengineering
would help to ensure that the brightest young radiologists and imaging
scientists have opportunities to obtain research training. Such
opportunities are largely non-existent under the present system.
For all of these reasons, I believe that this is the time to create
a National Institute of Biomedical Imaging and Bioengineering to
support a field of inquiry that is central to continued progress in
advanced research in molecular biology as well as to the development of
a better system for the delivery of health care. The proposed would be
good for patients, physicians, and the NIH itself. I urge the
Subcommittee to approve H.R. 1795, and I would be pleased to answer
questions.
Mr. Bilirakis. Thank you so much, Dr. Bryan.
I will start off the questioning.
Ms. Fraser, why does lupus seem to affect women of color
more often than Caucasian women?
Ms. Fraser. Well, this is a subject of a research project
currently under way by the NIH Lupus and Minority Studies, or
LUMINA. We believe lupus has a genetic basis, and it appears
that the gene suspected of causing lupus may be more prevalent
among women of color.
Mr. Bilirakis. So we sort of know that or have come to that
conclusion on the basis of studies that are taking place?
Ms. Fraser. Yes. That is correct.
Mr. Bilirakis. We do not know of any other reason, though,
other than the fact?
Ms. Fraser. Not yet, we are still checking.
Mr. Bilirakis. In terms of the administration's support or
nonsupport of the legislation, my understanding--and I may be
wrong, and if I am, I wish to be corrected, but I think it is
significant--that they have problems with title 2. The
administration has problems with title 2. Are you aware of
that?
Ms. Fraser. I would be happy to answer that, but I would
like to submit a written response to that question, if that is
okay with the chairman.
Mr. Bilirakis. Okay. Yes, we would like to have that from
you, by all means, because it would be very helpful in terms of
not only moving the legislation through, but we also try to
work with the Minority in most cases to work things out ahead
of time, and that would be very significant.
Dr. Wirth, can you tell us the organizations, or at least
some of the organizations, you are familiar with that are
working on the issue of global disease eradication?
Ms. Wirth. Well, there are several organizations in the
world. The World Health Organization is very active in this
area, but the World Health Organization is more of an
implementation organization rather than a research
organization. They have a very small research arm.
Really the United States is really the only--the United
States National Institutes of Health and to a certain extent
the NSF are really the only organizations that have the
knowledge base and the research base to bring that to bear on
these important tropical diseases. There is some work in Europe
funded by the European Union, but, again, I think the United
States really has the leadership role, and we need to maintain
that.
Mr. Bilirakis. I know--I am a Rotarian. Do not attend very
many meetings these days for business reasons, but in any case
they have worked on eradicating polio around the world, as you
know.
Ms. Wirth. That is correct.
Mr. Bilirakis. And that is working and has worked very
well; has not it?
Ms. Wirth. Uh-huh. Polio actually will be eradicated in
this hemisphere this year. And there have been many groups
involved in that, and certainly the Rotary has been involved
particularly in the last several years.
And, again, I think that is implementing a very important
step, implementing the sort of research in discoveries that
have been made over the years primarily here in the United
States. So I think there are many steps to eradicating global
disease. We have to get those vaccines and drugs that we have
to the people who need them, and that is very important. But we
also need for many of these diseases to develop new
interventions. The tools we have just aren't working.
Mr. Bilirakis. Okay. So the World Health Organization is
just not doing the job, and you feel that the national
commission that Mr. Gekas is a proponent of would do the job?
Ms. Wirth. I think so. And it would particularly establish
the United States in its natural leadership role in this area.
I think that we need political leadership at this point to
bring to bear on this problem. We have the skill set in the
United States to develop these interventions and to implement
them, but we need to take that leadership role in the world.
Mr. Bilirakis. Do we have now--and if not, is that the
reason maybe for the national commission--do we have the proper
coordination? For instance, I don't know, how has Rotary gone
about it all to know exactly where to go? Have they
coordinated?
Ms. Wirth. That is right. I think one of the things that
the National Commission could do is to have a focus point for
this kind of work in the United States. I think in the case of
Rotary and other nongovernmental organizations, they have sort
of gone about it themselves, having to go to different
agencies, to different interest groups to begin to find out
about it, and then to become involved with the implementation.
Mr. Bilirakis. Well, let me ask you this. If this
commission is formed, the administration feels that CDC and
NVPO, the National Vaccine Program Office, should be included
in the composition of the commission's membership. And also
that the FDA, as the agency overseeing vaccine safety and
approval of new vaccines, should also have a role in it if the
bill is enacted. Your opinion?
Ms. Wirth. I think that those, particularly the CDC and the
FDA, would be appropriate to become involved in this. They have
implementation roles. And I think the national vaccine program
is also one that certainly could be involved. They are dealing
very specifically with vaccine issues. As you know, there are
broader issues of implementation including drug development and
development of other controlled, measured environmental and
insecticides at some point.
Mr. Bilirakis. Any other opinions regarding that particular
legislation that you all may want to offer?
All right. That being the case, the Chair yields to Mr.
Brown.
Mr. Brown. Thank you, Mr. Chairman.
Dr. Wirth, I am sorry I missed your testimony. I was
voting. But I read your testimony, and you said, as we begin
the 21st century, we are blessed with unimaginable
opportunities to build on breakthrough research to control and
prevent world disease.
Dr. Gro Brundtland of the World Health Organization--I
would argue that the World Health Organization has done
phenomenal work over the last 20 years. Nonetheless, she said
talking about tuberculosis--and I know your expertise is more
malaria, and I want to get to that in a second, but she said it
is not medical eradication--dealing with tuberculosis is not a
medical problem, it is a political problem. But--and I think
back just less than a year ago in December 1999, the Government
of India, working with nongovernment organizations around the
world, including the World Health Organization, including all
kinds of groups from this country, had a national immunization
day and immunized 134 million children in 1 day, which tells me
that Mr. Gekas's bill goes in the right direction in this
country. Our country should show a great deal more leadership
in dealing with issues that surely we can.
Tuberculosis and malaria both do not get the attention from
the big drug manufacturers in their research arms that they
should. The drug companies seem much too interested, in my
mind, in ``me too'' drugs, in drugs that are more cure for
baldness than for tuberculosis, malaria, lupus, a whole host of
diseases where there simply is not the moneys, potential profit
available. There is not a lot of profit in malaria or TB
especially, diseases that hit this country not very hard and
hit the poorest countries with the poorest citizens especially
hard.
Shift gears to Walter--Walter Reed has done especially and
the Defense Department has done especially good research in
malaria. We underfund Walter Reed. We fund organizations like
NIH, a wonderful government agency. We want to double its
budget in the next 5 years, yet we do not fund CDC very well,
which its budget is about one-sixth of the NIH. And we do not
fund the Walter Reed research arm of the Defense Department
particularly well, putting it mildly.
Is the only real hope for a malaria vaccine, TB vaccine,
better treatment of those diseases--TB, as you know, you need
to take a pill every day for 6 months, which in countries with
military occupation, in places like Chiapas in southern Mexico,
people are afraid to go by the military checkpoint to get their
TB pills every day. And even though we can cure it, it is
difficult because of that. Is the only hope for a TB vaccine or
malaria vaccine better medicines to treat those two diseases?
Must there be government funding because the drug companies
won't do it? And what do we need to do? Talk about malaria.
What do we do with Walter Reed in the Defense Department, and
what do we do with NIH and CDC on malaria? Even though it is
not a great issue for me to go back to my district in Ohio and
say, I am working on malaria and TB, it does not matter much
directly today to citizens of this country, it will down the
road, and that is a whole other issue. But what do we do with
places like Walter Reed?
Ms. Wirth. I share your respect for Walter Reed and the
work they have done over the last several years in developing
antimalarial drugs. They really are the only group that has
consistently maintained a research program, even in spite of
very limited funding.
And, in fact, I think that the solution to these diseases
is going to require a very large governmental component because
the pharmaceutical industry, as you say, is driven by
developing drugs that are important for this country. These are
important drugs for this country, but the diseases of
tuberculosis and malaria and many other diseases found in
tropical countries just will never have profits like drugs for
diseases in this country. The drug companies will not develop
them. And I think we are going to have to--it is going to
require governmental intervention and governmental funding.
I recommend that Walter Reed certainly receive funding,
that the NIH receive funding for basic research and for
translational research, something I think that the NIH has
become very interested and very active in.
And in terms of CDC, CDC is our implementation arm; once we
have these tools, we have to get them out. And, in fact, for
many diseases we could certainly improve the situation today
just by better implementation of the tool we have. We still
face the challenges, but certainly better implementation
through CDC is important. So I recommend support for all of
these organizations, and let me correct myself if I misspoke. I
certainly have a great deal of respect for the World Health
Organization, but I think they need help and they need
leadership from the United States. Their budget is very small
compared to the budget of the NIH, for example, and I think
they provide a forum, but I think they need help from us, and I
think we can assume the leadership role in these diseases.
Mr. Brown. Thank you.
One last brief question. Should Walter Reed and the CDC be
included in this bill, both?
Ms. Wirth. Yes. I think that is an excellent idea.
Mr. Brown. Okay. Thanks.
Mr. Bilirakis. Mr. Burr to inquire.
Mr. Burr. Thank you, Mr. Chairman.
Dr. Wirth, I don't know that you misspoke, I don't think
you need to apologize. Sherrod and I participated in the same
hearing in International Relations on the threat of global
infections, a debate over whether it was a public health issue
or whether it is a national security issue, and I think we can
all agree it is probably (d) All of the above.
Clearly the World Health Organization and other
international organizations that are targeted toward health
issues have been effective on some things. Clearly there are
other things where health care professionals have pointed out
the deficiencies that exist; and with deficiencies in place, we
cannot be assured of successful immunization or successful
eradication of diseases that ultimately we see as a threat, not
only here but spreading throughout the globe.
And your reference to AIDS in Africa is a very good one. It
is important that we recognize that that spread, as it begins
to happen in Asia, is of a magnitude that we have never seen
before, potentially; and that every effort that we can make,
not relying on any one entity, is in fact the policy that we
should adopt.
And I appreciate your allowing me to editorialize just a
little bit.
Let me move to some of the other witnesses if I can because
I do have some real interest in another piece of legislation.
Let me turn to you, Dr. Bryan.
Who benefits? Who benefits from the creation of an
institute for biomedical imaging?
Mr. Bryan. Well, the people who benefit the most will be
the patients.
Mr. Burr. Isn't that who it is all supposed to be about?
Mr. Bryan. That is exactly right.
Mr. Burr. For any person who is on the fence about this
issue as to whether we should create this, if they stopped for
a minute and thought, who is this about; if their answer was,
the patients, then the answer is, vote for this bill.
Mr. Bryan. I would agree.
Mr. Burr. Is it safe to say--and I open this up to anyone--
as we identify breakthroughs in technology, that we can also
expect health care costs to possibly decline because if we
detect earlier, our treatments may be less intensive as it
relates to a period of time; and if you looked at the patient
from that standpoint, the quality of the care we deliver might
in fact be better because we have put them through less?
Mr. Hillman. That has been the history of the development
of imaging technologies: that, in fact, they do detect disease
earlier, they do replace more morbidity-inducing, more illness-
inducing technologies. And over time I believe that imaging
technologies have been cost-saving and also improve patient
outcomes.
Mr. Dunnick. I would like to make two comments in response
to that. First, when the DRGs were established a number of
years ago, my assumption was that the medical centers would try
to reduce the number of ancillary tests being performed. In
fact, just the opposite occurred. We went to more ancillary
testing in an effort to get to the answer faster, which in the
long run will reduce the cost of medical care.
My second comment is a reflection of my own experience.
When I was a medical student, my first research project was
with influenza, and we tried to use immunization to protect
against that disease.
We use death as the end point. Fortunately, we were using
mice as an animal model to test that. As we move along,
radiology has become very good at identifying disease processes
being able to quantify them in many cases. And so we can use
changes in imaging assessment as the end point for testing
this.
We are now in what we call the era of molecular imaging or
functional imaging, where we can actually detect changes before
they become manifest with routine testing. This allows us to
see the changes, see whether treatment is effective before the
disease has gotten out of control. I think these will make
dramatic changes in decreasing the cost of health care.
Mr. Burr. Can any of you address a specific disease where,
say, in the last decade the imaging improvements have changed
in----
Mr. Dunnick. Absolutely. Trauma would be the first response
to that. The patient comes into the emergency room, and in fact
it does not even have to be a traumatic injury. It can be a
patient with abdominal pain and the conventional way to treat
that would be first to do an operation to open the abdomen and
find where the pathology is.
We can do that noninvasively. In the trauma setting
specifically, we can now identify not only the problem, but in
many cases, quantify it, which enables more conservative
therapy.
So it has resulted in a dramatic decrease in the number of
patients that have to go to the operating room.
Mr. Burr. Let me ask one last question with the chairman's
indulgence.
One of the fears that I have is that we are successful and
that not only in imaging, but in other areas of medical
breakthroughs, we are successful. Technological improvements
have not necessarily been rewarded through the reimbursement
process in this country, specifically Medicare.
If, in fact our reimbursement system does not recognize the
cost of technology and the cost of this research, what will
that do to further development of new innovations, new
treatments, new imaging that might detect disease earlier?
Mr. Bilirakis. Important question, but brief answers,
please.
Mr. Hillman. Yes, there are two things that this new
institute will be able to do better than we are currently: One,
as I indicated, that it will have an assessment component that
will run clinical trials in a timely fashion to provide the
information to guide reimbursement. In fact that has been
problematic under the current NIH structure.
The other is that we will develop relationships directly
with the regulatory agency and payers to quickly move these
technologies into practice.
Mr. Burr. I thank the witnesses. I thank the chairman. I
yield back.
Mr. Bilirakis. Dr. Ganske.
Mr. Ganske. Thank you, Mr. Chairman, for having this
hearing. I think there are several bills that we are talking
about that have merit, and while they may not be the biggest
health care issues that Congress is facing, such as
prescription drugs or patient protection legislation or even,
for that matter, a bill that this committee will be doing
shortly on providing relief for Medicare, in particular, I
hope, relief for rural hospitals.
I just completed my series of town hall meetings back in
the district, and I get asked a lot about the high cost of
prescription drugs, and I find that there is one of these bills
that I think relates to that and that is the Orphan Drug Act
which created incentives for drug companies to develop
therapies for rare diseases by awarding a period of 7 years of
market exclusivity to a product approved for an orphan
indication.
I find the testimony of Mr. Thomas Lang to be convincing.
He says in his testimony, recently FDA has adopted a policy
position related to the scope of a clinically superior orphan
drug's exclusivity that actually undermines the incentives for
companies to continue to innovate for additional improvements
in these areas. As noted earlier, FDA's policy also raises
questions of fairness, alternate product availability and
patient and physician choice of therapy.
Now, after approval an original orphan drug, whenever a
subsequent orphan drug with a clinically superior improvement
has also been approved and awarded exclusivity, FDA totally
restarts the 7-year exclusivity clock for the drug as a whole,
and in this way the improved drug shields the original drug
from competition, even when--after the original drug's
exclusivity period is over. In these instances, companies that
have developed new competing versions of the same drug to treat
the disease in anticipation of the expiration of the original
7-year exclusivity are unfairly denied access to the market for
an additional 7-year period.
I think this has pertinence to the high cost of
prescription drugs. And Congress, even in the short time period
that we have left, should significantly look at the Thornberry
bill, H.R. 4242, because I think that additional extensions of
exclusivity will surely keep prices higher. That is why I and
others have been fighting an extension of--patent extension for
the drug Claritin.
And so, Mr. Chairman, I again thank you for holding this
hearing. I thank the people for testifying. I have another
hearing that is ongoing at this moment that I will be going to
and I will yield back.
Mr. Bilirakis. And I thank the gentleman. If he would yield
to me maybe 30 seconds of his time before me yields----
Mr. Ganske. I will.
Mr. Bilirakis. You know we have a dilemma here in terms of,
let's say, NIH funding. Let's just talk NIH funding, and the
dilemma is, should we in this so-called ivory tower determine
the amount of money for research that ought to go to specific
diseases? I mean, the experience that we have had on this
committee has been just amazing, the number of diseases that I
am sure most of us, if any of us, although some are medical
doctors, were not even aware of.
Just some terribly sad stories that we are going to hear,
and we are going to hear certainly even on the next panel, and
the plea for more funding for Parkinson's, more funding for
lupus. We can just go on and on. Mohammad Ali was here pleading
for more funding for Parkinson's.
So the thought has been that we just do not know enough of
actually what is taking place up there in terms of research and
how close they may be to a breakthrough and that sort of thing;
and should we be telling them, rather than just giving the
money or doubling the money as Mr. Brown has indicated?
Any opinions in that regard, because I consider that quite
a dilemma. We have come to maybe a conclusion.
I have not talked with Mr. Brown on his feelings on that
subject. I don't remember that we have in any case. But any
feelings in that regard? Just very quickly, please.
Mr. Bryan. Mr. Chairman, I think you do have indeed a major
challenge, and that is the responsibility you all accept as our
public representatives. I think that your directive is to
provide broad strokes of direction to institutes such as the
NIH. And I do think you have to leave some of the details to
them.
Mr. Bilirakis. Yes, sir?
Mr. Dunnick. I think in terms of H.R. 1795, what we are
really talking about is not necessarily more funding, but
reorganization to establish focus and priority setting.
Mr. Bilirakis. Which is basically what Ms. Fraser has
testified to and what Mrs. Meek's bill does, right?
Ms. Fraser. Yes. I just want to say that we just want to
level the playing field pretty much. Lupus, I did not really
know a lot about it before my sister was infected with the
disease. And as I learned more about it and I learned that
there are so many Americans, 1.4 million infected with it, I
think it is a disease that should be on the forefront right
now.
Not putting anybody else's cause down or their testimony,
but we just want to level the playing field is why we are here.
Mr. Bilirakis. I just wanted to sort of share with you the
dilemma that we have and the difficulty sometimes. Did you want
to add something quickly?
Ms. Wirth. Yes. Very quickly, I come from the sort of
training where I feel getting basic training and understanding
fundamental mechanisms is very important to understanding
disease. So, in general, I think it is very important that NIH
be given as much free rein to follow the advances as they come.
But I also think it is important that the interests of
individuals, who perhaps cannot sit at a table like this, are
represented in the area of biomedical research. And I think
without influence from the public to help direct the NIH to
areas of importance--I mean, the area of importance I clearly
consider very important is global health; and rarely is there
anyone sitting at this table with direct experience in it.
So I think it is very important that that be heard at NIH
at least in an advisory and perhaps not absolutely directive
way.
Mr. Bilirakis. Thank you. I see that Mr. Bryant, who was
here earlier and had to leave, has returned. Did you have any
questions of this panel, Ed?
Mr. Bryant. Mr. Chairman, thank you. Just some very brief
statements and perhaps a question of Dr. Bryan. I think I am
thinking more of the medical research at Pittsburgh, and you
are down the road a little bit I guess in the other direction.
But perhaps you know something about this.
I agree with Dr. Wirth in terms of that NIH ought to be
given a broad rein--range, I guess, in which to make their
decisions and less input from those of us who come into contact
with a lot of these difficult situations and have to--can't
really pick and choose. We are not knowledgeable either to make
those determinations.
But on the other hand, I think there is some need for input
from outside, as you point out, some representation, and I
guess to a degree we do that.
It seems to me--and maybe I am not using the setup of NIH
correctly, but I have heard the representation on their board
or perhaps the doctors' panels that help set these priorities.
Perhaps maybe we could have a better play in what groups are
represented there--what specialties, what doctors, what
diseases are represented there. And that would be a way of
again giving them broad powers, but yet we in Congress being
able to make sure that one disease is not given priority over
another one for the wrong reasons.
Second--and my last comment in this area, and I am going
toward something that I just mentioned earlier--I have been
working really closely with a group in Memphis in terms of a
disease that again does not address a large part of our
population, but a lot of our--a percent of our young children.
It is Duchenne's muscular dystrophy; I was at a fund-raiser for
them about a week ago in Memphis, and I am told that that is a
disease in which there have been great advancements made, and I
think a lot of that has come out of the University of
Pittsburgh or the Pittsburgh area.
I think our priorities also ought to be, in addition to all
the other priorities, trying to find cures for those diseases
regardless of the size of the population affected; those
diseases that are getting close to being solved, cured. That,
to me as a layperson, a nonmedical person, makes some sense,
that if we are getting close--because that can open the doors
to other related diseases, I would think. I would think
Duchenne's muscular dystrophy would have some very close
cousins in terms of diseases that could be affected in a
positive sense.
So, Dr. Bryan, I am asking you cold, do you know anything
about that particular disease in terms of are we making
progress there?
Mr. Bryan. I am familiar; I am not an authority on that
disease. But you are correct, it is a disease that affects a
relatively small population, but in a devastating fashion. And
remarkable advances have been made, mostly in understanding the
genetics and etiology of the disease.
I think the dilemma is one that is difficult. Your
committee has to face the public needs, define areas where you
think emphasis should be placed. But then I think, to be
honest, one has to defer to our peer review system which--the
NIH has a superb peer review system, where the experts have to
adjudicate whether, in fact, it is time, whether the knowledge
is there, the technology is there, the feasibility is there, to
actually, at that time, fund the additional research in that
area.
So I think you all have to define priority from a public
perspective, but then I think you have to take into account the
experts and the peer review system to help decide when you
actually support a particular research project area.
Mr. Bryant. Quickly, does anyone have an additional
comment? Thank you for being here and thank you, Mr. Chairman.
Mr. Bilirakis. And I thank the gentleman.
We will excuse this panel at this time. We customarily
furnish written questions, and we request written responses. We
would appreciate your assistance if you are all willing, to do
that in a timely fashion, Ms. Fraser, sooner rather than later,
particularly on the question that I raised----
Ms. Fraser. That will not be a problem.
Mr. Bilirakis. Thank you very much.
The second panel consists of--was scheduled at least--Mr.
Jack McCormick, Deputy Director of the Office of Orphan Drugs
for the Food and Drug Administration. Is Mr. McCormick here?
No? Is someone else going to be here to represent FDA on this
matter?
Mr. Doleski. I work for the FDA.
Mr. Bilirakis. Well, you do not want to testify at all?
Technical responses?
In any case, you will be here for the testimony and the
questions so that you can take those back too? I appreciate
that.
Why don't you give us your name, sir, for the record?
Mr. Doleski. Dave Doleski, D-O-L-E-S-K-I, Legislative
Analyst with the FDA.
Mr. Bilirakis. I am going to introduce Mr. Robert Brady, a
partner with Hogan & Hartson. They are here on behalf of
Biogen; Ms. Abbey Meyers, President of the National
Organization of Rare Disorders; Mr. Thomas A. Lang, Senior Vice
President, Strategic Product Development, Serono Laboratories,
Rockville, Maryland, and he is accompanied by Nick Ruggieri,
Vice President of Governmental Affairs; Ms. Catherine Bennett,
Chair, Board of Directors, Cancer Research Foundation of
America.
And I would now yield with the committee's indulgence to
Mr. Dan Burton, who is not on this committee, but who chairs of
course another very significant committee, who will introduce
Mr. Navarro and at the same time take 2 to 3 minutes to talk
about his legislation. You are recognized.
STATEMENT OF THE HON. DAN BURTON, A REPRESENTATIVE IN CONGRESS
FROM THE STATE OF INDIANA
Mr. Burton. Thank you, Mr. Chairman. And I hope you will
grant me just a minute or 2 latitude because I think some of
the things that I would like to say are very important.
To my classmate and chairman of this committee, Chairman
Bilirakis, it is nice to be with you. I think it is the first
time in the 18 years that we have been here that I have
appeared before your committee.
Mr. Bilirakis. I am sure that is true. And, Dan, I am sure
the thought has crossed your mind, there are not too many of us
left.
Mr. Burton. No, and unfortunately we just lost one of our
classmates.
Mr. Bilirakis. Yes, we just lost one of ours.
Mr. Burton. Anyhow, I appreciate your holding this hearing
and allowing us to testify on H.R. 3677, the Thomas Navarro FDA
Patient Rights Act.
The United States of America is a country based on
freedoms, and among the freedoms guaranteed through our
Constitution are freedom of speech, freedom to practice the
religion of our choice and a free press. However, we are not,
as individuals, guaranteed the freedom to make a life-and-death
decision in the area of medicine.
Imagine our own government forbidding your child access to
a nontoxic treatment, a nontoxic treatment with full human
subject protection through clinical trials that has already
saved the lives of other children. Imagine being told that you
must subject your child to treatments that may cause him to be
blind, to be deaf, to make him sterile, to stunt his growth, to
give him hormonal deficiencies, to lower his IQ and to give him
secondary cancers.
Imagine having your choices reduced to chancing no
treatment and possibly death or toxic treatment and possibly
creating a special-needs child with no guarantee of success,
all at a time when another treatment is available.
Imagine learning that the treatment that the FDA wants your
child to receive, that two of the three drugs in the, quote,
``standard protocol'' of approval drugs, clearly state on their
package inserts, ``Not proven safe or effective in the
pediatric population.''
Now, that is exactly what Donna and Jim Navarro have been
faced with. Imagine being a doctor who has treated cancer
patients successfully for over 20 years. Imagine being
repeatedly attacked by the FDA in an attempt to stop your work.
Attacked by the very agency that is supposed to encourage and
promote research.
Imagine submitting the BT-29 protocol so that a 4-year-old
boy can be treated with a nontoxic cancer therapy whose safety
has been established. A treatment which has saved the lives of
other children with the same type of cancer. Imagine this
government agency putting that protocol on hold because of
other existing treatments. That is exactly what has happened to
Dr. Burzynski down in Texas.
Many have heard the story of little Thomas Navarro. You may
have seen his story in ``People'' magazine, in the New York
Post or on CNN. His father, Jim Navarro, is here today to
testify, and I will leave the full story of Thomas' specific
condition for Mr. Navarro to talk about.
Two years ago the parents of another little boy, Dustin
Kunnari, testified before our committee, the Government Reform
Committee, about FDA's gatekeeping on clinical trials. Dustin
had the same form of cancer as little Thomas Navarro. Dustin
was the last person the FDA allowed to receive antineoplastons
without having first failed chemotherapy and radiation. He is
healthy and cancer-free today and without the devastation of
chemotherapy and radiation side effects.
Over the last 3 years, the Committee on Government Reform
has conducted five hearings looking at cancer treatments and
access to care. Unfortunately, Thomas Navarro is just one of
thousands of Americans who have been excluded from clinical
research because of the FDA. He is just one of the thousands of
children who are denied access to the parents' treatment of
choice because the government's agency has made a life-or-death
decision for the family and not allowed them the freedom to
choose.
The heart of this whole issue, Mr. Chairman, is, who
decides? Is it the role of the U.S. Government to make a
treatment decision? Or is it the right of the patient and the
family to make an informed treatment choice?
H.R. 3677, the Thomas Navarro FDA Patient Rights Act is the
first step in restoring medical freedom. It is the first step
in taking the decisionmaking out of the hands of the government
and putting it back in the hands of the individual where it
belongs, an informed decision.
Mr. Chairman and members of the committee, H.R. 3677 has 43
cosponsors from both sides of the aisle, Democrat and
Republican. I respectfully request your help in getting this
bill passed during this Congress.
I am now pleased to introduce Jim Navarro, Mr. Chairman.
And once again I want to thank you very much, my colleague, for
holding this hearing.
Jim testified at our June hearing and shared with us the
challenges that they faced as a family dealing with a cancer
diagnosis and the Federal agency that has forced them into a
corner. They have spent almost all of their money--I think they
sold their house. They completely depleted all of their
resources--in trying to solve the problem of their boy. And it
is a heart-rending story and I know Jim is going to go into it
in detail. He is here to testify about this bill.
Jim and Donna Navarro are intelligent, conscientious
parents. They love their son. They stood firm in the battle to
find the best and safest treatment for their child. And Jim is
a brave man fighting a battle on two fronts. While he is in the
battle for his son's life, he is in a battle for his own life.
Several months ago Jim was diagnosed with prostate cancer. So,
Jim, we wish you the best. And we pray for you and your boy.
And with that, Mr. Chairman, I would like to yield, if you
do not mind, to Mr. Navarro.
Mr. Bilirakis. Thank you, Dan. Thanks for your interest in
this subject, all issues in America.
The Chair will now yield to Mr. Navarro, who uses as his
address: Ronald McDonald House, Room 1101, 405 East 73rd
Street, New York, New York.
Mr. Navarro, please proceed, sir.
STATEMENTS OF JAMES NAVARRO, FATHER OF THOMAS NAVARRO; ROBERT
BRADY, PARTNER, HOGAN & HARTSON, ON BEHALF OF BIOGEN, INC.;
ABBEY MEYERS, PRESIDENT, NATIONAL ORGANIZATION FOR RARE
DISORDERS; THOMAS A. LANG, SENIOR VICE PRESIDENT, STRATEGIC
PRODUCT DEVELOPMENT, SERONO LABORATORIES, INC.; AND CATHERINE
P. BENNETT, CHAIR, BOARD OF DIRECTORS, CANCER RESEARCH
FOUNDATION OF AMERICA
Mr. Navarro. That is home, as you stated because as the
chairman stated, there is no longer a home for us.
Good morning, Mr. Chairman, my name is Jim Navarro and I am
the father of 5-year-old Thomas Navarro for whom this bill is
named.
I have been asked to speak on the benefits of this bill,
and I would like to first go on record saying that my son's
health has not stood still while the slow wheels of government
move, and thus this bill will not help my son. It is too late
to bring hope to our family, hope that the FDA would stand down
and allow my son access to our first choice of treatment.
We were forced to begin the FDA's preferred treatment this
summer. This bill will, however, help thousands of others. This
bill was conceived as a result of the FDA's unwillingness to
allow Thomas access to a treatment which had a higher rate of
success than the treatment offered through conventional means.
This bill will, however, bring hope to others, others who,
like us, have been denied access to treatments that show
promise and give a chance of survival, treatments which are
good or greater than those treatments currently available for
treating pediatric cancer.
We were faced with a decision almost a year ago, which
changed our lives forever, when our son Thomas was diagnosed
with medulloblastoma, which is a nonsurvivable type of cancer.
Thomas was rushed to surgery within hours to remove a 4 by 6
centimeter tumor from his cerebellum.
After surgery, we were faced with the decision of follow-up
therapy. We discovered in short order that the standard follow-
up therapies, radiation and chemotherapy, both had severe and
irreversible side effects. These included the possibility that
he would become blind, deaf, and sterile; that Thomas would
develop hormonal deficiencies that would have stunted growth;
that he would have had an immediate and progressive loss of IQ;
and that he would develop secondary cancers as a result of the
treatment itself.
We immediately began our search for a safer, nontoxic means
of treating our son. We found a treatment that showed a great
promise for treating medulloblastoma only to discover that our
son would be denied access to the treatment by the FDA.
The doctor was not allowed to treat my son because the FDA
did not approve his access to the treatment. Yet the FDA has
never approved radiation and chemotherapy for treating
pediatric cancers. In fact, if you read the manufacturer's
information that the drug companies put in the box, they state,
``Safety and effectiveness in pediatric patients has not been
established.''
This sentence, in and of itself, should cause concern. The
FDA has no problem forcing this therapy on my son and thousands
of others, even though the safety and efficacy has not been
established in children. In fact, if you are the parent of a
terminally ill child, your child can be taken away from you for
experimentation and, as parents, if you do not cooperate with
this madness, you can be thrown into jail for being bad
parents.
Based on your experience, Mr. Chairman, what actions must I
take today to get you and your committee to take the required
actions to save the Thomas Navarros of tomorrow? During the
course of this last year, my family has lost everything--our
home, our business, even our State of residency, which although
it is hot, it is a dry heat. It has been because of the
kindness and generosity of others, especially the support of
Citizens for Health, that Thomas has been able to receive
medical care.
H.R. 3677 introduced by Congressman Dan Burton now has 43
cosponsors, and I implore to you take this issue up and get
H.R. 3677 passed into law.
Thomas is very hard to recognize now as a result of
conventional therapy. And I would like to encourage Mr. Waxman
and others that would stand in opposition to this bill to come
see him, what he looked like before and what he looks like now.
Thomas's fight for his life now includes fighting against
the very treatment that he has been forced to take. And I can
only tell you it has been a very long and hard year.
Thank you for letting me speak.
[The prepared statement of James Navarro follows:]
Prepared Statement of James Navarro
Good morning Mr. Chairman, my name is Jim Navarro. I am the father
of 5 year old, Thomas Navarro, for whom this Bill is named. I have been
asked to speak of the benefits this Bill would present. I would first
like to go on record saying that my son's health has not stood still
for the slow wheels of Government to move, and thus this Bill may not
help my son. It is too late to bring hope to our family. Hope that the
FDA would stand down and allow my son access to our first choice in
treatment. We were forced to begin the FDA's preferred treatment this
summer. This Bill will, however; help thousands of others.
This Bill was conceived as a result of the FDA's unwillingness to
allow Thomas access to a treatment which had a higher rate of success
than the treatment offered through conventional means. This Bill will
however bring hope to others. Others, who like us, have been denied
access to treatments that show promise and give a chance of survival.
Treatments which are as good or greater than those treatments currently
available for treating pediatric cancers.
We were faced with a decision almost one year ago, which changed
our lives forever. When our son, Thomas, was diagnosed with
Medulloblastoma, Thomas was rushed to surgery within hours to remove a
4 X 6-cm tumor on his cerebellum. After the surgery we were faced with
the decision of follow up therapy. We discovered in short order that
the standard follow up therapies, radiation and chemotherapy, both had
severe and irreversible side effects. These side effects included the
possibility that he would become blind, deaf, and sterile. That Thomas
would develop hormonal deficiencies would have stunted growth, that he
would have an immediate and progressive loss of IQ. And that he could
develop secondary cancers as a result of the treatment.
We immediately began our search for a safer, non-toxic means for
treating our son. We found a treatment that showed great promise for
treating Medulloblastoma. Only to discover that our son would be denied
access to the treatment by the FDA. The doctor was not allowed to treat
my son because the FDA did not approve his access to the treatment.
Yet, the FDA has never approved radiation and chemotherapy for treating
pediatric cancers. In fact, if you read the manufacture's information
that the drug companies put in the boxes, they state, ``safety and
effectiveness in pediatric patients have not been established.'' This
sentence in and of itself should cause concern. The FDA has no problem
forcing this therapy on my son and thousands of others--even though the
safety and efficacy has not been established in children. In fact, if
you are the parent of a terminally ill child, your child can be taken
away from you for experimentation. And as parents, if you do not
cooperate with this madness, you can be thrown in jail for being ``bad
parents.''
Based on your own experience Mr. Chairman, what actions must I take
today to get you and your committee to take the required actions to
save the Thomas Navarros of tomorrow?
During the course of the last year, my family has lost everything--
our home and business in Arizona. It has been because of the kindness
and generosity of others, especially the support of Citizen's For
Health, that Thomas has been able to receive medical care. HR 3677
introduced by Congressman Dan Burton, now has 43 cosponsors. I implore
you take this issue up and get HR 3677 passed into law.
I would be pleased to entertain any questions from the Committee.
Mr. Bilirakis. Thank you very much, Mr. Navarro. I do--
well, what can one say? We want to be able to accomplish
something here. We want to be able to pass Dan's bill, or
essentially Dan's bill, but it has got to be done on a
bipartisan basis. That is your reason for imploring Mr. Waxman,
who frankly has just been very much interested in health care
all through the years. I know I have worked with him on this
committee for many, many years. And I do not really know
personally what his position is on the legislation. I guess
staff here does, but we are going to do everything we possibly
can.
Mr. Navarro. Mr. Chairman, if I might add real quick: It is
interesting, when I testified last time in Chairman Burton's
hearings and spoke to a number of the directors of the FDA
outside in the halls, they kept trying to reiterate the great
successes of conventional therapy in Thomas's case. Yet the
irony is, here before me are the consent forms to the treatment
Thomas is going through now, which state, ``Permission to for
participation of child in research.''
He is not in a protocol because they do not have a
protocol. They really do not know quite what they are going to
do with him. It is a hit and miss, and as they would say, a
crapshoot. In fact, one of the things that is sad that
disturbed me, especially after hearing Dr. Pazdur testify that
the rate of success was 70 and 80 percent--and this is coming
out of the horse's mouth--however, with standard therapy, there
is less than a 30 percent chance of curing these malignant
brain tumors in young children. Furthermore, young children
treated with radiation therapy for brain tumors may experience
serious and irreversible, long-term side effects from the
radiation.
And yet yesterday, the doctors announced to us that because
Thomas has fared the toxic side effects better than the other
children in the ward, they are anxious to start using high,
high-dose radiation and chemotherapy five to six times greater
than they have used on him so far.
And to be honest sir, he is tired of fighting the drugs. We
need to have the freedom to seek out a treatment that is
nontoxic and nonlethal. It is our right as Americans to have
that freedom.
Mr. Bilirakis. Thank you, Mr. Navarro.
Mr. Brady. Mr. Brady? Obviously, the written statement that
you all submitted is made a part of the record, and we would
prefer that you might sort of supplement it.
STATEMENT OF ROBERT BRADY
Mr. Brady. Thank you, Mr. Chairman. I will be quite brief
and just summarize my comments.
I am Bob Brady. I am here appearing this morning on behalf
of Biogen, Inc., a biotechnology company from Massachusetts. I
am a partner in the law firm of Hogan & Hartson where I have
been practicing food and drug law, focusing on pharmaceutical
matters for 25 years, including the implementation of the
Orphan Drug Act.
Let me summarize my points, and then I am just going to
focus on two or three of them.
If enacted, H.R. 4242, the Orphan Drug Innovation Act would
actually undercut the carefully crafted incentives of the
Orphan Drug Act without providing any real benefit to patients
or promoting innovation. The Orphan Drug Act has been an
unparalleled success. Any changes to the act should be made
only after careful analysis and consideration by fully informed
Members of the Congress in the context of the entire law.
The FDA and its Office of Orphan Products Development,
which has done a conscientious and successful job in
implementing the law to date, should be consulted and its views
taken into account. Moreover, Biogen knows of no patient
advocacy groups supporting this law nor do we know of any other
organization, other than one here at the table, supporting this
provision.
The Orphan Drug Act shouldn't be amended piecemeal by an
amendment hastily packaged together with noncontroversial
measures at the end of a legislative session. It would
undermine the foundation of the Orphan Drug Act so a single
company can market a product that has not been shown to be
clinically superior to orphan drug products already on the
market.
Let me speak one moment about the Biogen product, which is
a product to treat multiple sclerosis, which was approved and
is the only multiple sclerosis drug approved for two
indications to treat this terrible disease. And it has been
approved by the judgment of FDA that it is clinically superior
for safety reasons to the prior drug approved in this
marketplace. That is important, because that will be a point of
discussion here during the rest of this morning's testimony.
The Orphan Drug Act is one of the most effective laws
enacted by Congress with full bipartisan support in the last 20
years, especially in terms of the lives it has enhanced, the
pain and suffering it has diminished, and the hope it
represents to Americans with rare diseases.
I might also add parenthetically, after 25 years it has
been the least controversial piece of FDA law ever enacted in
terms of subsequent debate and litigation, suggesting that it
was well done to begin with and remains properly implemented.
However, H.R. 4242 would undercut the overwhelming success
of this act. The key incentive of the act is a 7-year period of
marketing exclusivity for the first product to be approved as
an orphan drug. H.R. 4242 would significantly narrow the scope
of this exclusivity by limiting it to particular aspects of the
orphan product subsequently approved.
Narrowing this key incentive, especially for a product
which has not shown any clinical superiority, would not only
hurt companies that make orphan drugs, but would also undercut
Congress' intent that there be new and innovative treatments
developed for millions of Americans who suffer from rare
diseases.
Orphan drug policies first passed by Congress and
implemented by FDA have been fair. Companies are rewarded when
they produce a clinically superior drug that represents an
innovation among the current marketplace.
Biogen, in fact, satisfied this standard in the law in the
mid-1990's when it was found to be clinically superior to the
existing multiple sclerosis product. Serono Laboratories is
testifying here today on behalf of this bill. They manufacture
a drug for multiple sclerosis that is the same as Biogen's
multiple sclerosis product. Serono would like to get their drug
into the American market, but they are blocked by the market
exclusivity of Biogen's product, which does not expire until
May of 2003.
The Orphan Drug Act and the FDA Act implementing
regulations currently provide a way for Serono's product to get
to market precisely the same way that Biogen's product got to
market in 1996, which is to prove clinical superiority to the
two existing products that are already available to multiple
sclerosis patients today.
This is not a situation where there are not products
available to patients. There are two Interferon products
already approved by FDA in this area. Serono or any other
company should not be held to a lesser standard than the
products that are already on the market.
Thank you very much, Mr. Chairman, for allowing me this
brief statement, and I am prepared to answer any questions you
may have.
[The prepared statement of Robert Brady follows:]
Prepared Statement of Robert P. Brady on Behalf of Biogen, Inc.
I am Robert P. Brady, and I appear here this morning on behalf of
Biogen, Inc. (Biogen). I am a partner in the law firm of Hogan &
Hartson L.L.P., in Washington, D.C. I have practiced law for 25 years,
and spend almost all my time on matters involving pharmaceutical and
biotechnology laws including the Orphan Drug Act. I would like to thank
you for the invitation to Biogen to testify before this Committee.
At the outset I would like to summarize the key points of my
testimony. If enacted H.R. 4242, the ``Orphan Drug Innovation Act,''
would actually undercut the carefully crafted incentives of the Orphan
Drug Act without providing any real benefit to patients or promoting
innovation. The Orphan Drug Act has been an unparalleled success. Any
changes to the Act should only be made after careful analysis and
consideration by fully informed members of Congress in the context of
the whole Orphan Drug Act. The FDA office of Orphan Products
Development, which has done such a conscientious and successful job in
implementing the Orphan Drug Act, should be consulted and its views
taken into account. Moreover, Biogen knows of no patient advocacy
groups supporting H.R. 4242, including the National Organization of
Rare Disorders (NORD) which is the chief consumer advocacy organization
for orphan drug research and development and was instrumental in the
development of the Act. The Orphan Drug Act should not be amended piece
meal, by an amendment hastily packaged together with non-controversial
measures at the end of a legislative session. It would undermine the
foundation of the Orphan Drug Act, so a single company can market a
product that has not been shown to be clinically superior to orphan
drug products already on the market.
Biogen is a biotechnology company based in Cambridge, MA and
manufacturer of a product for the treatment of relapsing forms of
remitting multiple sclerosis (MS) that was approved in 1996. Biogen's
product (Avonex') was approved as an orphan drug and thereby
received a grant of seven years of marketing exclusivity. Two important
medical facts about Avonex' must be kept in mind. It is the
only MS drug approved for both reducing the number of exacerbations and
slowing disease progression. Also, the FDA concluded that it was
clinically superior to the existing MS product due to greater safety.
As a result patients have benefited greatly from this approval.
Biogen is strongly opposed to H.R. 4242 for the following reasons:
The Orphan Drug Act is one of the most effective laws enacted by
Congress with full bipartisan support in the last twenty years,
especially in terms of the lives it has enhanced, the pain and
suffering it has diminished, and the hope it represents to Americans
with rare diseases. The Orphan Drug Act has been an unqualified
success. In particular, it spurred the development of breakthrough
drugs for Multiple Sclerosis, Cystic Fibrosis, Hemophilia, Leukemia, as
well as over 100 other rare diseases. During the ten years before the
law, only ten drugs were approved by the Food and Drug Administration
(FDA) for the treatment of rare diseases. Since the law was enacted,
however, about 200 orphan drugs have been approved and about 1,000 are
in the pipeline. Clearly, any modification of the Act must be the end
result of a deliberative process that increases incentives to develop
breakthrough treatments and, most importantly, benefits patients.
This is not the case with H.R. 4242--it would undercut the
overwhelming success of the Orphan Drug Act with no real benefit to
patients. The key incentive of the Act is a seven-year period of
marketing exclusivity for the first product to be approved as an orphan
drug. H.R. 4242 would significantly narrow the scope of this
exclusivity incentive by limiting it to particular aspects of the
orphan product. Narrowing this key incentive would not only hurt
companies that make orphan drugs but would also undercut Congress's
intent that there be new and innovative treatments developed for the 20
million Americans who suffer from rare diseases.
The FDA has skillfully implemented the Congressional intent of the
Orphan Drug Act in a manner that balances the seven year marketing
exclusivity incentive with the need to foster the public health goals.
One way in which FDA has balanced this issue is through the development
of a regulation defining scientific/medical criteria under which an
orphan drug which is the same as an orphan drug already approved for
marketing can be determined to be ``clinically superior'' and,
therefore, allowed to come to market in spite of any remaining
marketing exclusivity granted to the original approved product. (See 21
C.F.R. Sec. 316). FDA, the scientific and medical expert in this area,
has defined three such criteria to determine clinical superiority.
These criteria were thoughtfully and deliberately developed through
notice and comment rulemaking by the FDA. All interested parties had an
opportunity to present their views. Ultimately, the FDA crafted a well
reasoned definition of clinical superiority.
Critically important since the finalization of this rule almost a
decade ago, FDA has carefully exercised its scientific and medical
judgment in implementing this rule in a manner that is truly in the
best interests of patients. When another orphan drug truly is
clinically superior, FDA has allowed it to go to market so that
particular patients will benefit. It has done so sparingly, however,
because the FDA has correctly concluded, based on its extensive
experience, that, absent a real showing of clinical superiority,
preserving the seven year marketing exclusivity incentive is vitally
important to the development of new orphan drugs and that will help
even more patients suffering from orphan diseases.
These regulations are sound and fair. Companies are rewarded when
they produce a clinically superior drug that represents an innovation
above the current marketplace. Biogen satisfied these regulations when
the FDA found that its product Avonex was ``clinically superior'' to
another existing beta interferon product in 1996.
Serono Laboratories (Serono) is testifying on behalf of H.R. 4242
today. They manufacture a drug for multiple sclerosis that is the same
as Biogen's MS drug. Serono would like to get their drug onto the
American market, but they are blocked by the market exclusivity of
Biogen's multiple sclerosis product, which does not expire until May
17, 2003. The Orphan Drug Act and the FDA implementing regulations
currently provide a way for Serono's product to get to market: a
showing of ``clinical superiority'' based on appropriate scientific
data.
Serono, or any other company, should not be held to a lesser
standard than its competitors in the marketplace. To date Serono has
not demonstrated in head to head comparative trials that its product is
safer or more effective than the other beta interferon products on the
market for relapsing remitting multiple sclerosis. In 1999, the FDA
found, based on data submitted to the FDA by Serono, that Serono's
product was not clinically superior to the other similar multiple
sclerosis products on the market. Therefore, it is barred from the U.S.
market until the Biogen marketing exclusivity expires in May 2003.
Because Serono does not represent an innovation, patients are not being
denied a new or improved therapy. Serono is seeking to change these
requirements because Serono has not been able to satisfy them.
Serono's situation highlights one of the key problems with H.R.
4242. Under the bill, Biogen's market exclusivity for its multiple
sclerosis drug would be limited to blocking from the market only those
products that cause fewer injection site reactions and less skin
necrosis. However, a product which is less safe than Biogen's by
causing more site reactions and skin necrosis--such as Serono's
product--would be, under this bill, eligible for approval by the FDA.
Biogen does not understand how it benefits patients to allow a drug on
to the market during the exclusivity period that is neither safer nor
more effective.
The Orphan Drug Act's market exclusivity is not a barrier to
approval of a subsequent product that is not the same drug. A
subsequent drug for the same indication may be found to be not the same
drug if it is either chemically different or clinically superior.
Serono's Rebif product meets the statutory and regulatory standards for
``same drug'' because chemically it is the same drug for the same
disease as both Avonex and Betaseron. Because Rebif chemically is the
same drug as Avonex, in order for Serono to receive marketing approval
prior to 2003, Serono must demonstrate that it is clinically superior
to Avonex. The FDA's clinically superior criteria protects the drug
development incentive, while permitting the introduction of better
products to treat serious illness.
Aside from the serious policy concerns with H.R. 4242, another
fundamental flaw is that it is, in part, unconstitutional. As presently
introduced, the bill would apply to any drug designated on or after
January 1, 1990. By going back and retroactively narrowing the scope of
the market exclusivity, this would constitute an unconstitutional
taking under the Fifth Amendment. This legislation would not only
effect the property rights of Biogen but property rights of many other
companies with orphan drug designations that have made significant
economic investments based on an expectation of market exclusivity.
Biogen has attached a legal opinion on the unconstitutionality of the
bill that was previously sent to the Committee.
For Congress to make a change to a law as successful and important
to the health of the American people, as the Orphan Drug Act, one would
expect that there would be strong support from the biotechnology and
pharmaceutical industries and patient advocacy groups. Biogen knows of
no outside parties that favor the H.R. 4242. There are no patient
advocacy groups supporting this bill. The National Organization of Rare
Disorders (NORD), which is the chief consumer advocacy organization for
orphan drug research and development and was instrumental in the
development of the law, is opposed to the bill. The views of NORD and
other patient groups should not be ignored.
The stated purpose of this hearing today is to consider legislation
that would secure the health of the American people. The Orphan Drug
Act has already fostered the development of numerous breakthrough
treatments for rare disorders and helped countless persons. H.R. 4242
will threaten this continued development and risk the security of the
health of the American people with rare disorders.
Mr. Bilirakis. Thank you, Mr. Brady.
Ms. Meyers, please proceed, ma'am.
STATEMENT OF ABBEY MEYERS
Ms. Meyers. Thank you, Mr. Chairman.
For those of you who do not know us, the National
Organization for Rare Disorders is the consumer organization
that advocated for passage of the Orphan Drug Act, and we
continue to monitor its implementation.
We do not support H.R. 4242. And let me say at the outset,
we have no relationship with Biogen. Biogen has never donated
to NORD. This is totally independent.
Most orphan drugs have only one sponsor, and that is very
important to understand, because this situation comes up very
rarely when more than one sponsor is interested in the same
drug. And so we caution you not to change the Orphan Drug Act
in any way based on something that happens so rarely.
You can get the same drug, orphan drug, on the market to
compete against the innovator drug. You can do that in several
ways. You can get an orphan drug approved for a different
disease.
For example, if beta Interferon was approved for cancer or
something else, they could get it on the market and it could
compete in the marketplace. Or you could prove that it is
chemically or structurally different than the first drug to get
on the market. Or you can show that it has clinical
superiority.
Clinical superiority means that you have to prove that it
is safer or more effective or a major contribution to patient
care. In the case of Avonex and Betaseron, for example, Avonex
showed that you need less injections every week, it had fewer
side effects and did not cause one particular side effect. And
so it was a major contribution to patient care and you needed
only one injection a week.
So the current law protects the major incentive of the
Orphan Drug Act, which is 7 years of exclusive marketing
rights; and it is only through regulations that the current
definitions of ``same'' and ``different'' was created. And I
want to tell you that the Orphan Drug Act passed in 1983, but
those regulations weren't written and published until 1992. So
we waited many years and there was a lot of public input on the
development of those regulations.
The Thornberry amendment, we feel, would destroy the
backbone of the law because it would undermine the major
incentive of the Orphan Drug Act. And also be aware that the
Orphan Drug Act's success has been replicated all over the
world. The European Union just passed an orphan drug law; Japan
has one, Singapore, every country in the world admires what we
have been able to do here. So we need to keep the incentive in
place that would spur other manufacturers to develop clinically
superior orphan drugs.
In the case of multiple sclerosis, for example, people have
very poor muscular control. Giving themselves an injection is
climbing Mount Everest every day. And then they lose their
eyesight, so they can't see to fill up their syringe. It is a
major improvement to patient care when you only need one shot a
week and a nurse can come to your house to do it.
So what do we see in this situation here with this argument
over beta Interferon? If Serono--and we have the greatest
respect for Serono, but if Serono believes that its drug is
either safer or more effective or that it is clinically
superior, or even if they want to say that their drug is the
same as the original orphan drug so it would be able to get on
the market today if it could prove it is the same as Betaseron,
they should take their proof to the courts. They should not be
hiring lobbyists to come down here and ask you to change the
law. It is wrong for company after company, year after year to
come to you and ask for an amendment to the Orphan Drug Act. It
works; and if it ain't broke, don't fix it.
I will be glad to answer any of your questions that you may
have about the law. And we say again, we do not support this
amendment.
[The prepared statement of Abbey Meyers follows:]
Prepared Statement of Abbey Meyers, President, National Organization
for Rare Disorders
Mr. Chairman and members of the Committee, the National
Organization for Rare Disorders (NORD) is the consumer organization
that worked for passage of the Orphan Drug Act (ODA) of 1983, and we
continue to closely monitor its implementation today. NORD represents
approximately 25 million Americans with more than 6,000 rare ``orphan
diseases'', and we are very pleased to be here today. Thank you.
As you know, the Orphan Drug Act is one of the most successful
pieces of health legislation ever enacted by Congress. Today,
approximately 1,000 orphan drugs have been designated by the FDA, and
over 200 of them have been approved for marketing in the United States.
Recognizing the public health impact of the American law, other nations
have implemented orphan drug statutes including Japan, Singapore,
Australia and the entire European Union.
It is extremely important that Congress not undermine the intent of
the law, which is to encourage the commercial development of treatments
for small populations of patients. We do not support H.R. 4242, the
Orphan Drug Innovation Act, for many reasons, and we urge the committee
not to approve the legislation for further consideration by Congress.
Let me explain why we do not support H.R. 4242.
Early in the evolution of the American Orphan Drug Act, it became
necessary to define the words ``same'' and ``different''. In other
words, as defined by FDAs carefully crafted regulations, if a
manufacturer of a similar orphan drug can prove that their drug is
chemically different or clinically superior, even though it contains
the same active ingredients as the original orphan drug, the FDA will
approve it for marketing.
There are several ways to prove that a drug is ``clinically
superior'': either by providing FDA with scientific data proving that
it is either safer or more effective, or that it represents a ``major
contribution to patient care''. The latter is usually an obvious
improvement, such as developing an oral version of an injectable drug--
so that patients no longer have to suffer painful injections--or
developing a long-acting version of a drug that must otherwise be taken
several times each day, for example. Thus, the orphan drug regulation
defining ``same'' and ``different'' not only promotes, but encourages
development of new improved versions of marketed orphan drugs while
PRESERVING the chief incentive of the ODA.
Since most orphan drugs have no competition because companies are
generally not interested in investing the huge sums necessary for
research and development of a drug that will have a very small market,
the ODA offers an important incentive to encourage orphan drug
innovation.
Companies can receive seven years of exclusive marketing rights for
both the innovator drug and the clinically superior follow-on drug.
Mr. Chairman, from time to time there are orphan drug ``races''
when more than one company is developing the same orphan drug for the
same disease, and the law purposely creates a winner-take-all contest.
This is the very core of the success of the ODA because it prevents
competition for seven years, and ensures that a manufacturer of an FDA-
approved orphan drug will recoup its investment and make a profit. But
losers of the race sometimes ask Congress to change the law because
they want an exception for their drug. Thankfully Congress has been
wise enough not to allow this, knowing that tinkering with the Act
could destroy it.
In this case, Congressman Thornberry's bill aims to redefine FDA's
definition of ``same'' and ``different'', and to codify it into law,
based on the mistaken belief that people with rare diseases do not
already have access to clinically superior orphan drugs. However, H.R.
4242 will NOT enhance patient choice because current regulations not
only permit, but encourage competition when a therapeutic advantage can
be scientifically proven.
We believe that H.R. 4242 would disincentivize companies to develop
clinically superior orphan drugs and biologics, and it would allow
companies to seek approval for clinically inferior products. Moreover,
H.R. 4242 would reduce the exclusivity of orphan drugs and biologics
that have demonstrated they are clinically superior, because it would
limit exclusivity to the innovation that enabled a clinically superior
product to reach the market.
There are no benefits to patients if H.R. 4242 becomes law. There
are only benefits to companies that want to break the innovators
exclusivity, but that exclusivity is the very backbone of the Orphan
Drug Act which has since 1983 saved the lives of millions, and improved
the quality of life for countless others.
The current regulations, which are based on sound scientific
knowledge and common sense, were written to promote innovation and to
allow consumers access to clinically superior orphan drugs. They are
fair to consumers and fair to companies. The ODA is good public health
policy and continues to be one of the most successful pieces of health
legislation ever written.
We are profoundly grateful to Congress for enacting the Orphan Drug
Act, and for preserving its integrity since 1983. If you write the
Thornberry bill into stone, it will require an act of Congress to
change it when new medical technologies emerge in the future. If you
leave the orphan drug regulations alone, the FDA can easily fine tune
the rules, if and when that becomes necessary.
Mr. Chairman and members of the Committee, ``if it ain't broke,
don't fix it.'' We urge you NOT to enact H.R.4242.
Mr. Bilirakis. Thank you, Ms. Meyers.
Mr. Lang.
STATEMENT OF THOMAS A. LANG
Mr. Lang. Good morning. My name is Tom Lang. I am Senior
Vice President for Strategic Product Development at Serono,
Inc. I want to thank the committee for the opportunity to
testify on the issue of orphan drug evergreening, which is
addressed by H.R. 4242.
Serono believes this issue needs to be addressed
irrespective of the impact on our products. FDA's current
evergreening policy affects all drugs governed by the Orphan
Drug Act. We fully support the remedy posed by H.R. 4242,
whether or not it would apply to any of our products.
Furthermore, Serono believes orphan drug evergreening is
not a single-product issue.
Serono is a strong supporter of the Orphan Drug Act.
However, we have recently encouraged an anomalous and confusing
interpretation of the FDA orphan drug regulations which results
in orphan drug exclusivity evergreening. ``evergreening''
refers to FDA's granting of a new 7-year orphan drug
exclusivity period for the entire drug substance upon the
approval of a clinically superior version of the same drug,
rather than protecting only the innovative feature exhibited by
the second drug.
The results are to close the market to competition beyond
the initial 7 years of exclusivity intended by Congress. This
raises troubling policy issues of fairness, impediments to
price competition that would benefit consumers, and delays in
availability of alternative therapies for patients.
Mr. Chairman, Congress needs to decide exactly what the
scope of exclusivity should be for improved versions of
originator orphan drugs. We note that other areas of food and
drug law limit the scope of exclusivity for new versions of
previously approved products in a manner consistent with H.R.
4242. Like the Orphan Drug Act, the Waxman-Hatch Act seeks to
create incentives for continued research on improved drugs and
product improvements. The Waxman-Hatch Act, as one would
expect, rewards only innovative features with exclusivity,
rather than shielding the entire drug substance from
competition when its original exclusivity period has run. This
serves as evidence of Congress' intent and provides a basis for
supporting the principle in H.R. 4242.
FDA's handling of one particular product has resulted in
several very strained policy positions on the part of FDA. In a
letter to Serono, dated November 1999, FDA indicated that while
it would not allow an NDA or a BLA product to be marketed in
competition with the original drug, it would allow a generic
version of the original product to come on the market if it
were eligible for an abbreviated new application.
Mr. Chairman, there is no rationale whatsoever for
preventing competition from products that are supported by full
NDAs and BLAs. Subsequently, Serono became aware of an instance
where FDA has taken what appears to be a different position
than that previously described, a position which actually is
consistent with H.R. 4242, in a letter to Genentech.
Serono believes orphan drug exclusivity evergreening can be
resolved by FDA or Congress by simply limiting the second
clinically superior drug's scope of orphan drug exclusivity to
the superior characteristic that distinguished it as clinically
superior. This solution would probably reward the improvement
found in the clinically superior drug while still allowing
competition with the expired drug as intended by the law.
This would be consistent with other exclusivity-related
legislative initiatives, such as the Waxman-Hatch amendments
and patent law as well.
Limiting the scope of exclusivity of a clinically superior
orphan drug to its clinically superior feature still leaves the
drug sponsor with adequate incentives. A clinically superior
drug would gain three significant rewards as follows: .
First, it would achieve the benefit of being allowed on the
market immediately despite the originator drug's exclusivity;
Second, it would obtain 7 years exclusivity for the improved
feature; and Third, the company would be able to market its
product as a clinically superior product.
These are substantial awards and incentives. These
incentives make it unnecessary to keep the market closed to
other products wishing to compete with previous versions whose
exclusivity have expired.
In summary, the current evergreening policy actually
inhibits innovation, deters competition, and creates an
anomalous windfall extension of drug exclusivity.
We have attempted to work with the FDA to resolve this
issue for 2 years. Nevertheless, in Serono's opinion, FDA
continues to administer the exclusivity principle in an
inconsistent and unclear manner. The evergreening policy is now
riddled with ad hoc exceptions not found anywhere in the
statute or in the regulations. We, therefore, believe that
clarifying legislation is warranted.
Again, I would like to thank the committee for the
opportunity to testify on this important matter affecting the
incentive to develop improved drugs for rare diseases. We
appreciate the committee's attention and consideration, and I
would like to thank Dr. Ganske for his earlier comments.
[The prepared statement of Thomas A. Lang follows:]
Prepared Statement of Thomas A. Lang, Senior Vice President, Strategic
Product Development, Serono, Inc.
introduction.
Good morning, my name is Tom Lang. I am Senior Vice President for
Strategic Product Development at Serono, Inc. I would like to thank the
Chairman, and other members of the Committee, for the opportunity to
testify on the issue of orphan drug exclusivity ``evergreening,'' which
is addressed by H.R. 4242. At this time, I want to make it clear to the
Committee that Serono believes this issue needs to be addressed
irrespective of the impact on our products. FDA's current evergreening
policy affects all drugs governed by the Orphan Drug Act (ODA) and
represents a policy issue that demands your attention. We fully support
the remedy posed by H.R. 4242; however, we would be equally supportive
of an administrative remedy accomplishing the same goal.
As a global leader in biotechnology with a number of drugs and
biologic products already approved in the U.S. and many more in our
research pipeline, Serono is a strong supporter of the Orphan Drug Act.
We recognize the need to provide strong incentives to develop drugs for
rare diseases, and indeed, many of our drugs have orphan drug
designations as well as orphan drug exclusivity. However, we have
recently encountered a problem with FDA's anomalous and confusing
interpretation of the Orphan Drug regulations, which results in orphan
drug exclusivity ``evergreening.'' In this context, ``evergreening''
refers to FDA's granting of a new seven year orphan drug exclusivity
period for the entire drug substance upon the approval of a second,
clinically superior, version of the same drug, rather than protecting
only the innovation exhibited by the second drug. This results in
closing the market to competition beyond the initial seven years of
exclusivity intended by Congress.
This is an unwarranted extension of orphan drug exclusivity. It
presents troubling policy issues of fairness, impediments to price
competition that would benefit consumers, and the delay in availability
of alternative therapies for patients.
background.
The phenomenon of orphan drug exclusivity ``evergreening'' is a
result of FDA's interpretation of its regulations giving effect to the
ODA, and not a result contemplated or intended by Congress. The ODA
itself created incentives for drug companies to develop therapies for
rare diseases by awarding a period of seven years of market exclusivity
to a product approved for an orphan indication. During these seven
years, FDA may not approve other applications which are for the ``same
drug'' and the same disease or condition. Congress intended that after
this period of exclusivity, the public would benefit from increased
treatment options as well as price competition among various products
in these areas. The ODA overall has been a significant success in
driving research for rare diseases.
However, the statute is silent as to improved versions of
previously approved orphan drugs. In the regulations adopted by FDA to
implement the ODA, the agency created a mechanism by which a second
product could also be approved during the period of market exclusivity
awarded to the first drug. Such exceptions are made where a second drug
is deemed to be clinically superior to the first orphan drug. FDA's
intention in creating this mechanism was to maintain incentives for
companies to continue research on orphan drugs, and to reward
additional advancements by allowing them earlier access to an otherwise
closed market. While this objective is laudable, FDA has recently
chosen to implement it in a very problematic fashion.
fda's exclusivity policy.
Recently, FDA has adopted a policy position related to the scope
of a clinically superior orphan drug's exclusivity that actually
undermines the incentives for companies to continue to innovate for
additional improvements in these areas. As noted earlier, FDA's policy
also raises questions of fairness, alternate product availability, and
patient and physician choice of therapy.
Now, after approval of an original orphan drug, whenever a
subsequent orphan drug with a clinically superior improvement has also
been approved and awarded exclusivity, FDA totally restarts the seven-
year exclusivity clock for the drug as a whole. In this way, the
improved drug shields the original drug from competition, even after
the original drug's exclusivity period is over. In these instances,
companies that have developed new competing versions of the same drug
to treat the disease in anticipation of the expiration of the original
seven-year exclusivity are unfairly denied access to the market for an
additional seven-year period. Under FDA's current policy, this total
period of exclusivity barring the entry into the market of competing
product versions could theoretically be as long as fourteen years if
two drugs were approved; as long as twenty-one years if three drugs
were approved, and so on. This possible extension, or ``evergreening''
of the original drug's exclusivity period by restarting the clock for
the entire drug substance when a second or third clinically superior
version is approved is the problem sought to be addressed by the
Thornberry Bill (H.R. 4242).
orphan drug evergreening is not a single product issue.
FDA has designated at least five drugs as orphans, based solely on
their demonstration of superiority over a previous version.
Avonex' (recombinant beta interferon la for
relapsing remitting multiple sclerosis), exclusivity based on
clinical superiority to Betaseron.
Sandostatin LAR' Depot (generic name octreotide for
several orphan indications, primarily transplant rejection)--
long acting formulation, exclusivity based on superiority in
dosing to original drug.
Nutropin Depot' (recombinant human growth hormone
for pediatric growth hormone deficiency) dosing superiority,
long acting formulation.
Benefix TM (Coagulation Factor IX for hemophilia) -
Recombinant version judged safer than two previous versions.
Prolastin' (Alpha 1 proteinase inhibitor for
emphysema), long acting formulation superior in dosing
convenience to original orphan drug.
All five were considered to have presented a safety or dosing
improvement over the original version of the drug. The active chemical
entity in all five cases is the same as the original drug. The older
versions were all approved as safe and effective products and are still
being marketed. Thus, a new competitor in these other disease areas
would also be expected to face an evergreening problem, based on FDA's
policy.
other areas of food and drug law limit the scope of exclusivity for new
versions of previously approved products.
As with the Orphan Drug Act, the Waxman-Hatch Act seeks to create
incentives for continued research on approved drugs and product
improvements. The Waxman-Hatch Act, as one would expect, rewards only
the innovative feature with exclusivity, rather than shielding the drug
substance from generic competition when its original exclusivity period
has run. This serves as evidence of Congress' intent, and provides a
basis for supporting the principle in H.R. 4242.
fda's position of only allowing abbreviated applications to compete
with an expired orphan lacks a rationale.
FDA's handling of our product has resulted in several very strained
policy positions on the part of the agency. For example, in a letter to
Serono dated November 8, 1999 (copy attached), FDA indicated that while
it would not allow our product to be marketed in competition with the
original drug approved for this orphan drug indication, it would
approve a generic version of the original product to come on the market
if it were eligible for an abbreviated new drug application (ANDA.)
In Serono's opinion, this position indicates that FDA in fact
agrees in principle that the exclusivity that was awarded to the second
clinically superior drug should not prevent competition with the
original product whose exclusivity has lapsed. However, FDA makes an
arbitrary determination that only ANDA drugs can compete, but not drugs
that are supported by full new drug applications (NDAs and BLAs). There
is no rationale whatsoever for preventing competition from products
that are supported by full NDAs and BLAs.
fda's action in a subsequent case is actually consistent with h.r.
4242.
Recently, Serono became aware of an instance where FDA has taken
what appears to be a different position than with our product, and one
which appears to be consistent with H.R. 4242. In a letter to Genentech
dated October 28, 1999 (copy attached), FDA advised the company that
new long acting formulation of recombinant growth hormone (Nutropin
Depot) has been designated as an orphan, but that the orphan
designation ``applies only to the long acting formulation,'' rather
than to the entire drug substance. This means that the Nutropin Depot
improved formulation, once approved, would achieve orphan protection
for seven years, but its exclusivity only would cover the improvement,
and manufacturers wishing to introduce additional versions of the
conventional dosage form would not be blocked. Thus, in this instance,
FDA's position appears to be totally consistent with H.R. 4242.
solution to ``evergreening'' problem.
Fortunately, the ``evergreening'' problem is one that has an
extremely simple solution. Orphan drug exclusivity evergreening can be
resolved by FDA or Congress by simply limiting the second, ``clinically
superior'' drug's scope of orphan drug exclusivity to the superior
innovation, feature, or characteristic that distinguished it as
clinically superior. This solution would properly reward the innovation
found in the clinically superior drug, while still allowing competition
with an expired original drug, as intended by the law. Again, FDA could
remedy this problem itself, without legislation, by simply modifying
its current policy as to the scope of exclusivity associated with a
``clinically superior'' orphan. This would be consistent with other
exclusivity-related legislative initiatives, such as the Waxman-Hatch
Amendments, and patent law as well.
the solution proposed in h.r. 4242 would retain incentives for
innovation in orphan drug research.
Limiting the scope of exclusivity of a clinically superior orphan
drug to its clinically superior feature still leaves the drug sponsor
with an adequate incentive. A clinically superior drug would gain three
significant rewards. First, it achieves the benefit of being allowed
onto the market immediately despite the originator drug's exclusivity.
Second, it obtains seven years' exclusivity for the improved feature.
Third, it will be able to market its product as clinically superior.
These are substantial rewards and incentives. These substantial
incentives make it unnecessary to keep the market closed to other
products wishing to compete with previous versions whose exclusivity
may have expired.
conclusion
In summary, the current evergreening policy unnecessarily denies
patients and physicians alternative therapeutic options. In our view,
given the way it is being administered, it actually inhibits innovation
and deters competition, and creates anomalous windfall extensions of
drug exclusivity. We have attempted to work with FDA to resolve this
issue for two years. Nonetheless, in Serono's opinion FDA continues to
administer the exclusivity principle in an inconsistent and unclear
manner. The evergreen policy is now riddled with ad hoc exceptions not
found anywhere in the statute or in the regulations. This has caused
significant confusion for industry. We therefore believe that
clarifying legislation is warranted to avoid policy that we believe was
never intended by Congress. That is why we support H.R. 4242.
Again, I would like to thank the Committee for the opportunity to
testify on this important matter affecting the incentive to develop
improved drugs for rare diseases. We appreciate the Committee's
attention and consideration.
Mr. Bilirakis. Ms. Bennett, please.
STATEMENT OF CATHERINE P. BENNETT
Ms. Bennett. Thank you, Mr. Chairman and members of the
subcommittee. I appreciate this opportunity to testify on an
issue of great personal importance to me, cancer awareness
treatment and research.
I am here representing the Cancer Research Foundation of
America as chairman of its board of directors. We are a
national nonprofit health organization whose mission is cancer
prevention through scientific research and education.
Since its founding in 1985, the Foundation has funded
research by more than 200 scientists at more than 100 leading
universities and medical centers. And it is one of the only 10
non-Federal agencies whose grant review process is approved by
the National Institutes of Health.
Within the last year, CRFA has increased its focus on
childhood cancers with the establishment of Hope Street Kids, a
foundation created under the umbrella of CRFA following the
loss of Caroline Pryce Walker. The mission of Hope Street Kids
is to eliminate childhood cancer through advocacy education and
cutting-edge research and to help sustain and support children
with cancer and their families during and after treatment.
Unfortunately, most us have had a personal experience with
cancer. We have seen it attack a family member, a friend, a
coworker, or we have been diagnosed ourselves. I was diagnosed
with breast cancer in 1993. It is a dreaded and pervasive
disease that claims the lives of more than 500,000 Americans
each year, and it is a disease that knows no racial, ethnic,
economic or gender boundaries. Perhaps what is even more
disturbing is that cancer also does not discriminate based on
age. Many of us think of it as a disease of the elderly or
middle aged, but we must also recognize that cancer is the No.
1 cause of death by disease for children.
Each year, more than 12,000 children are diagnosed with
cancer and some 2,300 children will die from the disease. That
is about 100 classrooms filled with children who won't start
school next September. September is significant in that it is
recognized as National Childhood Cancer Awareness Month.
So it is appropriate that the committee has House
Resolution 576 sponsored by Congresswoman Deborah Pryce on its
agenda.
I am pleased to testify in support of in resolution which
seeks to raise awareness about the realities of childhood
cancer and make suggestions or recommendation about where
Congress could help ensure that more children live to start a
new school year. The statistics in the resolution demonstrate
the challenges we face. The incidence of cancer among children
is rising by 1 percent each year. One in every 330 Americans
develops cancer before age 20. It constitutes about 8 percent
of deaths between the ages of 1 and 19. And as I mentioned, it
is the leading cause of death by disease in children.
It is clear to me that we cannot dismiss this disease as
rare or ignore the substantial loss of life for which childhood
cancer is responsible. In my mind, even one child lost to
cancer is unacceptable. The good news is that progress has been
made. Four years ago a diagnosis of childhood cancer was a
death sentence. Today, almost 70 percent of children diagnosed
will survive. Nonetheless, that means 30 percent do, in fact,
succumb. The success rate can be attributed in part through
research through clinical trials. They have become the standard
of care for pediatric oncology patients with approximately 70
percent of the children who are diagnosed participating. This
makes sense to build on these efforts by making sure that
opportunities for childhood cancer research are funded and that
we attract the best and brightest to pediatric oncology and
that we make sure that as many children as possible have access
to the centers of excellence and clinical trials.
The resolution suggests that Congress support such
policies. Additionally, H. Res. 576 encourages support for
policies that encourage the development of new drugs in
biologics. As members of this committee know, the Food and Drug
Administration Modernization Act provides additional incentives
to encourage greater private investments and research by
providing some additional 6 months of market exclusivity to
sponsors of new or approved drugs if they conduct pediatric
studies. Despite the good intentions of this law, the policy
has not proven as effective in stimulating research or
providing additional information about drugs that may prove
useful in pediatric oncology. I believe it is worth
reevaluating the policies reflected in that statute.
While we look to the future with hope that we will see the
day when no child becomes the innocent victim of cancer, we
must also face the reality that children today are suffering
and are dying. We must focus our attention on improving the
quality of life for these patients. The horrors of cancer are
many, but it is hard to imagine anything more tortuous than a
parent witnessing their child in pain. Yet many will tell you
that they have been forced to stand helplessly by while their
children are enduring invasive and painful treatments.
The resolution points out a recent study which revealed
that 89 percent of children with cancer experienced substantial
suffering in the last month of life. Why, in this day of modern
medicine and technology, is this necessary or acceptable? In my
view it is not. The reason for inadequate pain relief for
children in cancer patients may be many, but one can be found
in the lack of training for pain management received by
physicians in their medical training.
We can begin to address this issue by expanding knowledge
among medical personnel to help them recognize the signs of
pain and treat them effectively. The resolution is supportive
of such curriculum as part of medical training.
The battle against childhood cancer is being hard fought,
but those that know the horrors of this disease, and many of
them will be in Washington this week to do what they can to
raise awareness and recruit Congress and others, whoever will
listen, in fact, to their cause. I believe Mrs. Pryce's
resolution is a good first step that indicates a congressional
understanding of the issues at hand and provides an outline for
what a successful policy aimed at defeating childhood cancer
should entail. I encourage the subcommittee to lend its support
to this legislation and again, appreciate the opportunity to
participate today.
[The prepared statement of Catherine P. Bennett follows:]
Prepared Statement of Catherine P. Bennett, Chairman, Board of
Directors, Cancer Research Foundation of America
Thank you, Mr. Chairman and members of the Subcommittee. I
appreciate this opportunity to testify on an issue that is of great
personal importance to me--cancer awareness, treatment, and research.
I am here representing the Cancer Research Foundation of America,
as Chairman of their Board of Directors. For those of you not familiar
with CRFA, we are a national, non-profit health organization whose
mission is the prevention of cancer through scientific research and
education. Founded in 1985 by Carolyn Aldige, the Foundation has funded
research by more than 200 scientists at more than 100 leading
universities and medical centers, and is one of only ten non-federal
agencies whose grant review process is approved by the National
Institutes of Health. And, I am pleased that within the last year, CRFA
has increased its focus on childhood cancers with the establishment of
Hope Street Kids, a foundation created under the umbrella of CRFA. The
mission of Hope Street Kids is to eliminate childhood cancer through
advocacy, education and cutting-edge research, and to help sustain and
support children with cancer and their families during and after
treatment
Unfortunately, most of us have had a personal experience with
cancer. We have seen it attack a family member, a friend, a coworker,
or we have been diagnosed ourselves. This dreaded and pervasive disease
claims the lives of more than 500,000 Americans each year. And, it is a
disease that knows no racial, ethnic, economic, or gender boundaries.
Perhaps what is even more disturbing is that cancer also does not
discriminate based on age. Many of us think of cancer as a disease of
the elderly or middle-aged. But, we must also recognize that cancer is
the number one cause of death by disease for children. Each year, more
than 12,000 children are diagnosed with cancer and each year, some
2,300 children will die from the disease. That's about 100 classrooms
filled with children, who won't start school next September.
September is also significant in that it is recognized as National
Childhood Cancer Awareness Month. So it is appropriate that the
Committee has house resolution ______, sponsored by Congresswoman
Deborah Pryce, on its agenda. I am pleased to testify in support of
this resolution, which seeks to raise awareness about the realities of
childhood cancer and make suggestions about where Congress can help
ensure that more children live to start a new school year.
The statistics in the resolution demonstrate the challenge we face:
The incidence of cancer among children is rising by one
percent each year.
One in every 330 Americans develops cancer before age 20.
Cancer constitutes about 8% of deaths between ages 1 and 19.
And, as I mentioned earlier, it is the leading cause of death
by disease in children.
It is clear to me that we cannot dismiss this disease as
``rare'' or ignore the substantial loss of life for which
childhood cancer is responsible. In my mind, even one child
lost to cancer is unacceptable.
The good news is that progress has been made. Forty years ago,
a diagnosis of childhood cancer was a death sentence, but today
almost 70 percent of children diagnosed with the disease will
survive. This success rate can be attributed to research
through clinical trials. In fact, clinical trials have become
the standard of care for pediatric oncology patients, and about
60 percent of children that are diagnosed with cancer
participate. That compares with only 3% of adult cancer
patients and 1.5 % of Medicare patients.
It makes sense to build on these efforts by making sure that
opportunities for childhood cancer research are funded, that we attract
the best and brightest scientists to pediatric oncology, and that as
many children as possible participate in and benefit from the
discoveries made through clinical trials. H. Res. ______ suggests that
Congress support policies consistent with these goals.
Additionally, H.Res. ______ encourages support for policies that
encourage the development of new drugs and biologics. As the Members of
this Committee know, the Food and Drug Administration Modernization Act
of 1997 provided an incentive to encourage greater private investment
in research on the use of drugs to treat pediatric diseases.
Specifically, the Act provides an additional six months of market
exclusivity to sponsors of new or approved drugs if they conduct
pediatric studies that may produce benefits for children. Despite the
good intentions of this law, the policy has not proven effective in
stimulating research or providing additional information about drugs
that may prove useful in treating pediatric cancer. It is worth re-
evaluating this policy and its implementation by the FDA so that we can
ensure that children are not left out of the tremendous advances in the
treatment of disease that new drugs and biologics can provide.
While we look to the future with hope that we will see the day when
no child becomes the innocent victim of cancer, we must face the
reality that children today are suffering and dying. We must also focus
our attention on improving the quality of care and of life for these
patients. The horrors of cancer are many, but it is hard to imagine
anything more torturous for a parent than witnessing their child in
pain. Yet, many parents will tell you that they have been forced to
stand by helplessly while their child endured invasive and painful
treatments. As H.Res. ______ points out, a recent study revealed that
89 percent of children with cancer experienced substantial suffering in
the last month of life. Why, in this day of modern medicine and
technology, is this necessary or acceptable? In my view, it is not. The
reasons for inadequate pain relief for children and cancer patients may
be many, but one can be found in the lack of training for pain
management received by physicians in their medical training. We can
begin to address this issue by expanding knowledge among medical
personnel to help them recognize the signs of pain and treat them
effectively. H.Res. ______ is supportive of such curriculum as part of
medical training.
The battle against childhood cancer is being hard fought by those
that know the horrors of this disease, and many of them will be in
Washington this week to do what they can to raise awareness and recruit
Congress and others--whoever will listen--to their cause. I believe
H.Res. ______ is a good first step that indicates a congressional
understanding of the issues at hand and provides an outline for what a
successful policy aimed at defeating childhood cancer should entail. I
encourage this subcommittee to lend its support to this legislation.
Again, I appreciate the opportunity to participate in today's
hearing and to speak to this issue during Childhood Cancer Awareness
Month.
Thank you, Mr. Chairman.
Mr. Burr [presiding]. Thank you, Cathy, and welcome. The
Chair would take this opportunity to recognize himself, as soon
as he gets his thoughts. And we apologize that there are
members trying to get back. And it is the intent of the
committee to continue with the hearing rather than take a break
for lunch because of the afternoon schedule. Here we think it
is not only more beneficial to us, but also to you to go ahead
and allow those members to make it back to ask questions.
Ms. Meyers, let me ask you some specific questions. Things
have changed significantly since we originally put together the
orphan drug legislation, haven't they?
Ms. Meyers. Yes.
Mr. Burr. Can you be a visionary for us just a minute and
look out once the human genome project is complete, once we
have mapped the genetic outlay of the human structure, how many
of what we classify as rare diseases today do you think that
researchers will be out there trying to find the key to the
cure for in the future?
Ms. Meyers. Well, it is very complicated because it is not
just a matter of everybody, for example, with muscular
dystrophy having the same genetic defect. There are many
different genetic defects that may result in Duchenne's
muscular dystrophy. And down the road in about 20 years, the
way I foresee it, the way the scientists do, is that they will
be able to personalize drugs for the particular genetic defect
that has occurred in individuals. So we will have custom-made
bio technology drugs to address the specific defect in that
gene.
Mr. Burr. Which means the population, that because they are
going to be subsets of disease, the population that they are
going to target is going to be tremendously small.
Ms. Meyers. Minuscule.
Mr. Burr. So is it safe for the members of this committee
to assume that a large share of the pharmaceutical applications
that will go in are going to be under the orphan drug
legislation, because the population is defined at 200,000
people, if I remember correctly, we will clearly be chasing a
multitude of things under that population.
Ms. Meyers. It will be a growing number of treatments for
the small populations, most of which will come from
biotechnology.
Mr. Burr. Is there any reason that we as a committee and as
an institution should, in any way, shape or form, look out at
that 200,000 person number, knowing the changes that are going
to take place through the genetic mapping, and at least debate
or possibly change that number to be more reflective of where
we think exclusivity should be in the future? I am not talking
about the debate that we are at at this table, I am trying to
think out a number of years.
Ms. Meyers. Well, looking back over the 17-year history of
the Orphan Drug Act, the problems that have arisen have not
arisen around the size of the population. The problems that
have arisen are pricing problems. And even drugs for very tiny
numbers of people, if you are going to charge $100- or $200,000
a year for that treatment, you are going to make a lot of
profit. And so, if there are any changes to the Orphan Drug Act
and one of them was introduced and actually passed the House
and Senate by Mr. Waxman in 1990, and it was vetoed by
President Bush and that was aimed at shortening the period of
exclusivity for blockbuster drugs. So I would not lower the
size of the population because 200,000 is not a huge number and
believe me, it is even hard to find companies that are willing
to make drugs for 3- or 400,000 Americans.
Mr. Burr. In today's research environment?
Ms. Meyers. Yes.
Mr. Burr. Ten years from now, in tomorrow's research
environment where you have got a map that leads to you a point
that it took you 5 years now to hopefully do research to find,
my question was not should we, it was should we at least have a
debate on it? Should we bring in individuals out of
biotechnology and pharmacological research to discuss what do
you see down the road? Are you going to be chasing diseases
because of the information you have that there is a population
of 5,000 and 7,000 and 12,000, which means that the majority of
the stuff that we do will be classified as under the Orphan
Drug.
Ms. Meyers. It is true, but, you know, the bigger these
companies are getting with their mergers and their
acquisitions, I mean, they are interested in Viagra, they are
not even going to be looking at these types of diseases. The
latest one is something about removing facial hair. I mean
these kinds of markets are so huge, the big companies don't
want to look at a drug with an estimated sales under a billion
a year.
Mr. Burr. Clearly you make a point that is probably an
accurate one today, if through these advances it is much easier
for them to design that drug of the future, as you said, a
custom-designed drug, it may be a whole different situation.
Ms. Meyers. I think it will be, yes.
Mr. Burr. Did you ever envision under the Orphan Drug law
that--let me ask it a different way: Do you think it is right
under the Orphan Drug law that a company could have an
approval, could have their exclusivity, at some point during
that period of exclusivity they made an enhancement to the
product, they reapplied, and were approved for whatever reason
at FDA and got a new year--7-year exclusivity?
Ms. Meyers. Yes.
Mr. Burr. Did you envision when the Orphan Drug law came
about, that that was something that would happen?
Ms. Meyers. First of all biotechnology was in its infancy.
We couldn't imagine what would happen with biotechnology. But
we saw early on in 1985, Genentech got approval for human
growth hormone, and a few months later Eli Lilly came on with a
different version of human growth hormone. And it created
exactly this situation. FDA approved Eli Lilly's second version
of human growth hormone saying it was economically or
structurally different.
Mr. Burr. Two different companies.
Ms. Meyers. Two different companies.
Mr. Burr. Should the same company have the ability to
reapply for whatever changes, get a new 7-year exclusivity
agreement?
Ms. Meyers. Yes. And that also happened with Genzyme's drug
for genetic disease for Gaucher's disease where they did
improve it. It had been made out of a natural blood substance
or something, and then they made a biotechnology version and
they got another 7 years. Yes, they should, because the main
incentive is to develop a better drug. And it worked.
Mr. Burr. Do you see any problem with the fact that the
company who has the current exclusivity certainly has a
tremendous advantage because they have the data? That is not--
that is not data that is shared within the community of
researchers that are out there. And if, in fact, nobody wants
to invest the money to create that data base to chase that
small population drug, you really do have an inherent ability
of one company to continue to restart the clock. I am not
saying that it happens today. I think that to some degree, in
health care we have to start getting visionary in this
institution.
We do a poor job at crisis management, but that seems to be
the only thing that we try to address now is the crisis
management of today's problem. And I think that we have got to
focus out on the future and ask ourself what do we need to do
in preparation for the changes. I would only suggest to you
that I see a potential problem there as a Member of Congress. I
see the ability for one company to continue to restart the
clock almost like FDA used to do in their application process
when they changed investigators and when they wanted to slow
down the process, they asked for a new piece of information and
the new 180 days started. And that became more the norm than
the exception. But it is a question that I raise.
Ms. Meyers. I agree with you. It could be a potential
problem, but the fact is when the exclusivity on the first drug
expires, any other company can get on the market and make that
first drug. Like a generic drug, except for one thing. Congress
has never passed a law that allows the FDA to approve generic
biologics. And so they have to do all of the research, all of
the clinical research and prove all the safety and
effectiveness of a brand new drug, and then they are allowed on
the market to compete with the drugs whose exclusivity has
expired. That is what could happen here. Betaseron, the first
beta interferon, their exclusivity has expired. If any company
can prove that their beta interferon is the same as Betaseron,
they would get on the market.
Mr. Burr. We have clearly got some work that we know we
need to do.
I want to turn to Mr. Navarro. Mr. Navarro, I don't want
you to think in any way, shape or form that members of this
committee and Members of Congress haven't struggled not just
this year, but for a number of years to try to find the right
balance of the goals standard of the FDA, their process, and
the innovative treatments that Dr. Brezinski and others have in
the marketplace today, and certainly I have been involved for 4
years in Dr. Brezinski's treatments. And hopefully we have--
this committee has contributed greatly to the process forward
of the current clinical trials that he has, the expansion of
those trials as a liaison between FDA and Dr. Brezinski on the
data that was needed for us to get expansions. But I don't want
to address Dr. Brezinski's treatment specifically, because one
thing I want you to understand is that Members of Congress are
not here to practice medicine. But we are here to try to
address the structure that is needed for everybody to receive
the quality of care that they deserve. In doing that, I have
found it to be very difficult. Because quite honestly, many of
the patients that visit me with the personal stories of their
fight don't come back the next year. That makes a very, very
big impact on every Member of Congress I can assure you, as it
does the families, of which many of us have affected in our
families.
My hope is that we can be visionary, we can look at some of
the treatments that exist out there. And that we can form a
partnership between medicine and FDA and medicine and NIH and
medicine and HCFA, and that we can get patients back to the
forefront of the health care delivery system in this country.
We spent a lot of time arguing whether it is reimbursements or
whether it is doctors or whether it is hospitals or whether it
is insurers, and really more time about the process than we do
about the outcome. I understand you are only concerned with the
outcome. That is all you should be concerned with. We have got
to deal with everything else.
But let me ask you specifically as it relates to your son,
is it your understanding from the health care professionals
that treated your son, that there was no conventional treatment
that was FDA approved, be it chemotherapy or anything else that
they had suggested that was specifically FDA approved for
pediatrics?
Mr. Navarro. You have to understand that radiation and
chemotherapy, and I am going to pick just on Thomas's disease
for a minute, has not been approved for the very reason that it
doesn't produce successful results. I have had the opportunity
in the last year to speak to more parents than I care to
remember that are the parents of dead children who presented to
me their medical records, the results of the chemotherapy the
results of the radiation. And it became very clear very early
on that this was a very, very dangerous option that I did not
want to take with Thomas. In the case of chemotherapy you have
to understand that chemotherapy is a cytotoxic poison and
Thomas' oncologist made it very clear that we are going to
basically stretch you to your limits because it is unnatural
for a parent to voluntarily poison their child in the hopes
that it will create a cure. And we have had to go through this
process with Thomas and watch him endure the poisoning with
great frustration and anxiety realizing that there was a non
toxic chemotherapy available that other children had been
allowed to use and yet Thomas had been denied access to that.
That particular therapy has been with us for almost 30
years, and for the last 18 years there have been efforts made
to bring it to the forefront where it can be approved but
again, it has been continually blocked. And the point that I
have been trying to make for the last year is if I have to
choose between two unapproved therapies, which both are, why as
a parents do I not have the right to go with the therapy that
will do the least amount of damage to my son and then if it
doesn't work, step up to a more aggressive therapy. I have
spoken to parents of children that have been destroyed by
radiation and chemo. And I am thinking in particular of the
young man from Houston, Texas who today, at 19 years old, is
deaf, dumb, blind, strapped to a wheelchair, has an arm's
length list of side effects, and yet now that his parents need
help in his maintenance and care, are denied access to that.
Because this child, who is--picture him strapped to a chair, he
can't see, speak or hear, can't move, can't function, is deemed
a danger to the other patients, therefore he can't go into a
group home. I am still, after almost a year, trying to figure
out how he becomes dangerous if he can't operate under his own
power.
And again, my frustration with the FDA is we have even
applied for a compassionate use exemption. Now the doctor that
we chose to go to in Houston has a protocol for
medulloblastoma. He has treated with the blessings of the FDA
children with medulloblastoma. But I found it odd that as more
and more children came through the process well, that all of a
sudden there was a new step put in place saying we can no
longer allow you to treat these children until they first go
through radiation and chemotherapy and fail and have recurrent
measurable tumor.
If this is a pure glass of water, and my agency is in
charge of making sure that water is available that is healthy
to all and I allow someone to come over here and pour a
substance into it and say, well, Mr. Brady, we want you to have
clean water, but before you can drink this clean water, someone
is going to be allowed to taint it. I think he would be
reluctant to drink the water.
And in Thomas' case, how can we know if the FDA is truly
and sincerely interested in progress and medical and scientific
breakthroughs? How can we ever know if the treatment we wanted
for him is successful, if we taint the baseline about other
therapies that take, to be honest, a lifetime to recover from?
Mr. Burr. I hope you will accept my answer which is, I
don't have one. I can't explain it to you. But I will assure
you that this committee has not quit, the members on this
committee have not quit to try to one, wade through the
modernization of the Food and Drug Administration, which we
completed in 1997, which people gave us no hope could be done
and had passed with unanimous bipartisan support from this
House and from the Senate and was signed into law by the
President. We are still waiting to see the full changes as they
are implemented from that legislation. It is not always a fast
process. But much of that is by design in this system. I hope
you understand that we will continue to strive to make sure
that we have the answer for you and for the other parents that
I know will be here in the future, whether it is on this
treatment or another treatment, because we will never build a
system that is perfect. But we will never be content with what
we have. We will always strive for something better.
The unfortunate thing is I have been notified that we have
a series of votes. And because I know that that will throw
further the end of the turmoil of members' schedules this
afternoon because of the knowledge of their schedules, I am
going to take this opportunity to apologize to all of you,
because I know that we will have a lack of participation if we
take 45 minutes off and try to reconvene.
I am going to leave the record open for written questions
to come to each of you from any members of the subcommittee.
And I hope you will respond to those written questions with
answers for the purposes of the record. Let me, once again, on
behalf of the chairman, thank you for your participation in
this hearing. This hearing is now adjourned.
[Whereupon, at 12:45 p.m., the subcommittee was adjourned.]
[Additional material submitted for the record follows:]
Additional Testimony for the Record of the Department of Health and
Human Services
The Food and Drug Administration (FDA or Agency) has serious
concerns about H.R. 3677, Thomas Navarro Patients Rights Act because
this bill will seriously weaken the Agency's ability to protect
individuals who volunteer to participate in clinical trials, one of its
primary statutory missions. The Agency would be precluded from
protecting a segment of the population from insufficient information,
misinformation and, at worst, fraud. The Federal Food, Drug and
Cosmetic (FD&C) Act created a process that carefully balances the need
for scientific support for new products with the overriding need to
protect human subjects. H.R. 3677 upsets this balance and could lead to
harm or possibly death for some patients.
Under the FD&C Act, FDA has been provided the authority to regulate
the use of investigational (unapproved) drugs. As an element of this
authority, FDA regulates clinical human investigation conducted by a
sponsor. Under statutory and regulatory authority, sponsors wishing to
use investigational, unapproved drugs in human patients must file an
Investigational New Drug application (IND) with FDA. This IND is either
an IND that is designed to involve a number of patients or can be filed
as a single patient IND to treat one patient.
The Food and Drug Administration Modernization Act of 1997 (FDAMA)
codified and expanded FDA's programs to provide access to unapproved
products in section 561 of the FD&C Act. This was done after extensive
review and consideration of the needs of patients. The intent was to
provide access to therapies for which there was no effective, approved
therapy and particularly for patients who had failed existing approved
therapy. The provisions are clear in requiring the individual
physician, FDA and the sponsor of the investigational drug to go
through a series of steps before allowing the drug to be made available
to the patient. These ultimately require a balancing of the risks and
benefits of the proposed administration of the investigational drug. As
part of the access program, the sponsor is still required to file
either a single patient IND or an IND to treat a small number of
patients.
FDA is obligated to evaluate an IND submission within 30 days and
make a determination if the clinical investigation should proceed or
the investigation should be put on clinical hold. The primary focus of
this decision is the safety of the patients who may receive the
unapproved drug. The evaluation includes a determination of the nature
and level of risk to the patients who may be the subject of the
clinical investigation, design of the clinical investigation, various
qualifications of the investigators, the disease being investigated and
other information about the drug. The same evaluation is done for
submissions that may be considered single patient INDs or emergency
INDs, although FDA makes the decision in a considerably shorter time
period than 30 days and in the case of an emergency IND usually within
24 hours or less.
FDA has, in rare instances, placed on clinical hold a proposal to
administer an investigational drug about which little was known to
treat a serious or life threatening disease when there was known
effective, or life saving, therapy for that condition. We did so
because those subjects would have been exposed to an unreasonable and
significant risk of illness or injury. This is also required under FDA
regulations governing clinical holds. (Title 21, Code of Federal
Regulations Sec. 312.42). The risk in these cases was not simply the
possible untoward effects of the investigational agent but also the
risk of substituting a known effective therapy with something that
might have no effect. Although these circumstances occur rarely and are
dwarfed in numbers by those clinical trials allowed to proceed, the
ramifications of being unable to put a particular investigation on hold
would be dramatic.
FDA understands the seriousness of placing any investigation on
clinical hold and does so only after following a careful process that
includes deliberation with internal experts and, if necessary,
consultation with external experts about the particulars of the disease
state and potential for available therapies.
The ability to put an investigation on clinical hold is the primary
mechanism FDA has to protect human subjects who are being asked to
participate in a clinical investigation and asked to be part of an
experiment with unapproved investigational drugs. H.R. 3677 contains
several provisions that could impair the ability of FDA to put clinical
investigations on hold even if a patient's safety is being compromised
and could prevent FDA from taking action specifically for an individual
patient who may be at a high level of risk.
Section 2 (a) of H.R. 3677 amends section 505(i)(3) of the FD&C Act
by limiting the ability of FDA to impose a clinical hold on an
investigation. The new provision would prohibit a clinical hold even if
another therapy is determined to be safer because it is a known
curative therapy. The entire basis for this prohibition on FDA is that
the patient has, in effect, waived their right to informed consent. The
patient, by declaring in writing that s/he is ``aware of the comparable
or satisfactory alternative therapy . . . aware of the risk involved in
receiving the drug in the investigation, and chooses to receive the
drug notwithstanding such risk and notwithstanding the comparable or
satisfactory alternative therapy'' could prevent the imposition of a
clinical hold even for safety reasons which may be directly related to
the individual patient's safety.
The bill creates a situation in which research subjects may be
presented with incomplete and/or biased information by investigators
who often have substantial personal, financial or professional
interests at stake in the research. FDA is concerned that the consent
obtained under these circumstances will not be truly informed consent
because it will not be based on a thorough and objective explanation of
the risks and benefits of both the unapproved investigational product
and the proven treatment that is being foregone.
H.R. 3677 does not even require that the sponsor administering the
unapproved investigational drug provide all the information to the
patient typically contained in an informed consent. The idea that a
patient could essentially sign away their rights to adequate health
care to anyone who is able to convince them to do so, without oversight
and without any assurance of proper information, is offensive and
unethical. Even absent mal-intent, the most well intentioned
investigator is unlikely to have access to all that is known about an
unapproved investigational drug very early in its development.
Furthermore, since FDA has been provided the authority to oversee
the use of unapproved investigational drugs, the Agency is the one
central location that collects data on those unapproved investigational
drugs. FDA is often the only party in possession of information from
different sponsors that could impact the decision as to whether an
unapproved drug should be used in a clinical investigation. There is no
means of ensuring that the patient actually knows all of the risks
involved. Reliance on the sponsor of the investigational drug to
provide all the known risks is misplaced since that particular sponsor
may not have access to all of the information known about a drug. Thus,
through no fault of the sponsor, complete information on risk may not
be provided to the patient considering the use of the unapproved
investigational drug.
The importance of clinical hold as an Agency tool to help ensure
the protection of human subjects in clinical trials is illustrated by
the recent example of the Jesse Gelsinger case. At age 18, Mr.
Gelsinger volunteered to participate in a clinical trial of a gene
therapy product and died shortly after the administration of the
product. The investigator who recruited Mr. Gelsinger to the trial did
not follow the terms of his protocol and did not reveal important
safety information during the informed consent process, so that Mr.
Gelsinger's consent was not fully informed. When FDA learned of this
subject's death, the Agency put this trial on clinical hold so that no
other patients would be subjected to the dangers inherent in this
trial.
Under H.R. 3677, FDA would have no ability to protect subjects even
if the Agency had information that practitioners were using dishonest
means to coerce patients into participating in trials in lieu of taking
proven therapy. If H.R. 3677 is enacted and such ethically suspect
practices are allowed to proliferate, it also could seriously undermine
the public's confidence in the process of conducting biomedical
research in this country with dangerous consequences to the public
health.
There are other technical drafting problems with H.R. 3677 which
are not included in this submission. Even if technical changes were
made, however, FDA would still have concerns with the impact of the
proposed legislation.
FDA has a long, and successful, history of expediting access to
investigational agents for those who patients with serious and life
threatening illnesses who have no satisfactory therapy available to
them. We also have a long and successful history of protecting patients
who are most vulnerable, including those with life threatening
illnesses who are desperately seeking help and hope.
______
Lupus Foundation of America
November 9, 2000
The Honorable Michael Bilirakis
Chairman, Subcommittee on Health & Environment
U.S. House of Representatives
Committee on Commerce
2125 Rayburn House Office Building
Washington, DC 20515
Dear Mr. Chairman: In response to your letter of November 2, 2000,
I am pleased to submit the attached answers to the questions prepared
by members of the Subcommittee on Health & Environment. Please contact
me if I can provide additional information.
It was a pleasure to appear before your Subcommittee on behalf of
the 1.4 million Americans with lupus. I want to express my sincere
gratitude to you and the Members and staff of the Subcommittee for your
support of the Lupus Research & Care Amendments of 2000 Act.
Sincerely,
Tomiko Fraser
National Spokesperson
Answers to Questions submitted by Subcommittee members.
Question 1. Why does lupus seem to affect women of color more often
than Caucasian women?
Response. This is the subject of a research project currently
underway by NIH. (Lupus in Minorities Study, or LUMINA) We believe
lupus has a genetic basis and it appears that the genes suspected of
causing lupus might be more prevalent among women of color.
Question 2. Are manifestations of lupus more serious among African-
Americans than among Caucasians?
Response. African Americans have more kidney involvement than
Caucasians. Researchers may have located a gene believed to cause
kidney disease in African American lupus patients.
Question 3. Can lupus be prevented, or the health impact minimized
in any way?
Response. Unfortunately, you can't prevent lupus. However, you can
take steps to minimize health effects of lupus, such as adopting a
healthy lifestyle, such as avoiding stress, getting plenty of rest,
eating well, light exercise, and following your doctor's advice.
Diagnosing lupus early, and seeing a doctor regularly can minimize
damage to vital organs. The earlier lupus is diagnosed and treated, the
more likelihood of preventing the need for more expensive treatment.
Question 4. Do we know why lupus strikes mostly young women?
Response. The exact cause of lupus is unknown, but researchers
believe lupus has a genetic basis. Hormonal influences may explain why
lupus affects mostly women in their childbearing years. But we don't
know if there is something about women that makes them more vulnerable
to developing lupus, or if there is something about men that protects
them from lupus.
Question 5. Why is lupus so expensive to treat?
Response. Lupus requires constant medical attention by a number of
specialists, requiring many tests to monitor the status of the immune
system. Because the disease usually presents multiple symptoms, people
with lupus must take many medications. It is common for people with
lupus to take over a dozen medications. In addition, monitoring lupus
activity requires many expensive tests to determine the functioning of
the immune system and many vital organs, such as the kidneys, lungs,
heart, and brain.
Question 6. What impact does lupus have on the work force or
corporate community?
Response. One in five people with lupus is disabled. Many victims
of lupus must cut back their work hours, or change jobs to reduce
stress. This results in lost productivity for corporations, and costs
millions of dollars. In addition, disability costs rise to cover people
with lupus.
Question 7. What is The financial impact of lupus on the rest of
the family?
Response. It's difficult to measure, but it has to be significant.
On average lupus costs $6,000 to $10,000 annually to treat, but some
victims incur costs of several thousand dollars a month. This level of
expense can have an enormous impact on the family budget.
Question 8. Does the environment play a role in causing lupus?
Response. Yes, while lupus has a genetic basis, we know that
certain environmental factors trigger disease activity, including UV
light, infections, certain chemicals and drugs, and stress. There is
much interest in this area of research, particularly in regard with the
role breast implants may have in causing an autoimmune reaction.
Question 9. Are we seeing more cases of lupus today than in the
past?
Response. It's hard to determine the exact number of lupus cases.
We don't know if lupus is on the rise, but doctors are getting better
at diagnosing the disease, which may be the reason we are seeing more
cases of the disease. However, some researchers do believe that lupus
is increasing among young women.
Question 10. Can lupus be treated with less expensive herbal or
complementary therapies?
Response. There is considerable interest in this area. Some
therapies, when used in combination with a doctor's care, can help
reduce symptoms, but they are no substitute for traditional medicine.
Lupus is a dangerous disease that needs constant monitoring by a
trained doctor. The FDA has given fast track designation to a new
therapy for lupus using the hormone, DHEA.
Question 11. Are there new treatments in the pipeline?
Response. Yes, there are several therapies undergoing clinical
trials that will address various manifestations of lupus, but the
disease is so complicated, no magic pill will ever cure lupus. It will
take much more research. Some therapies include biologics that can
block the immune system from producing autoantibodies.
Question 12. What is the outlook for a cure for lupus?
Response. While there has been progress, a cure is not on the
horizon. We are getting better at treating symptoms of the diseases and
patients are living longer, but there is no cure for the foreseeable
future.
______
Response of James Navarro to Questions of the Subcommittee on Health
and Environment
Question 1. Mr. Navarro, in your opinion, who should decide the
course of medical treatment for your son: you, or the FDA?
Response. This answer requires very little thought. The Food and
Drug Administration (FDA) is an agency made up of many different people
from many schools of thought. These people many times as we have
discovered, get caught up in defending a position, or a particular
discipline of medicine and often forget the ``PATIENT'S RIGHT TO
CHOOSE''. Often, the patient is ignored entirely. We as parents of a
terminally ill child, have only one agenda, and that is to see our son
survive, with dignity and quality of life. Agencies and doctors are
often driven by egos and money, as we have tragically experienced
during our journey with Thomas. Nobody and I mean nobody, will ever
have his best interest at heart, more so than his mother, Donna, and I
do.
Question 2. My understanding is that the FDA denied Thomas access
to a clinical trial because he had not first gone through and failed
chemotherapy and radiation. Why did you decide to forgo this treatment?
Response. Before I can answer this question, we must first clarify
a very important fact which is quite often ignored, that fact is that,
``RADIATION AND CHEMOTHERAPY HAVE EVER BEEN APPROVED BY THE FDA FOR USE
IN TREATING PEDIATRIC CANCERS'', it has merely become the unopposed
standard of care for treatment, because anyone who stands in opposition
to its use is either crushed as far as career advancement goes, or is
dismissed as a charlatan as history has bore out. Chemotherapy alone
represents a $107,000,000,000.00 ($107 Billion) a year industry. Our
history unfortunately has always born out that profit always comes
before people.
It is important to remember that ``STANDARDS OF CARE'' does not
mean that a treatment has been thoroughly tested and adequate evidence
reviewed by the FDA or other Federal Agency to assure safety and
efficacy. A ``STANDARD OF CARE'' is not and should not be an
endorsement by a Federal agency that has never evaluated the data.
``STANDARDS OF CARE'' are protocols that doctors in cancer research
centers have developed according to their own research as the best
treatment option according to their perspective in how to treat cancer.
Now, to be more specific, our reasons for not wanting to use these
``STANDARDS OF CARE'', on our son were much simpler. The simple fact is
these treatments are extremely dangerous to use, and more often than
not, end in permanently damaging or even killing the patient. Quite
simply, if it is all a ``crap shoot'', we as parents are compelled to
start with a treatment which will do the least amount of harm first.
The following is a list of just some of what Thomas would suffer as a
result of being treated by Radiation and Chemotherapy: FATIGUE, NAUSEA,
VOMITING, ABDOMINAL CRAMPING, HAIR LOSS, LOSS OF IQ, LOSS OF HEARING,
LOSS OF MEMORY, FLUID IN THE MIDDLE EAR, HYPOTHYROIDISM, SPINAL GROWTH
DEFICIT, HYPOPITUITARISM, SECONDARY TUMORS, LOW LEVEL HORMONES (For the
rest of his life), RADIATION NECROSIS, KIDNEY NECROSIS, AND DEATH FROM
EITHER RADIATION POISIONNG OR CYTOTOXIC POISIONING. These are just some
of the possibilities we faced with Thomas. You must also understand
that every child we have met during Thomas's journey that was treated
using the ``STANDARD OF CARE'', is now dead, the most recent we buried
just two weeks ago. The choice we made was not a difficult one. We
chose life. We knew through careful research that there were other
possibilities for Thomas, we never thought our government would stand
against us in making those choices.
Question 3. According to the administration's written testimony,
the intent of current law is to protect ``our most valuable citizens,
those who are desperately ill'' and that the FDA believes this bill
would undermine FDA's ability to help assure reasonable safety and
effectiveness of subjects in clinical trails and informed consent for
patients given access to experimental therapies.'' In that
Administration statement, with what do you agree or disagree?
Response. The opening statement of this question reminds me of the
old axiom; ``THE ROAD TO HELL IS PAVED WITH GOOD INTENTIONS.'' First,
all citizens of this great country should be considered valuable, not
just the sick, and in keeping with that thought, it is we the people
who should be allowed to decide what is best for us. Second of all, the
Agency's roll is to monitor safety and effectiveness, not to sit in
judgment, deciding who can and can't introduce new promising therapies:
as is the case with the treatment we choose for Thomas. For almost
twenty years, the FDA has stood as a roadblock to progress, instead of
helping to advance a therapy which shows great promise in treating the
specific cancer that Thomas suffers from. Third, as far as informed
consent goes, this is a process that we personally have seen abused and
the FDA and HHS does nothing to correct it. There needs to be better
accountability at the FDA and amongst the Doctors. Until or unless that
happens, no expanded authority in the decision making process should be
considered. The role of both the doctor and the FDA should be to assist
us in making decisions by providing us with all of our treatment
options and offering their best advice. It is not the role of the FDA
or the doctor to take control of our lives and forcing us to submit to
their will.
Question 4. H.R.3677 precludes the FDA from establishing a clinical
hold on the basis that there is a comparable or satisfactory
alternative therapy if the patient is 1) aware of the other therapy; 2)
aware of the risk associated with the investigational drug; and 3)
chooses to receive the drug. How would this bill affect you and others
you may have met during the ordeal you have faced in getting your son
the treatment you have decided to be best? Do you see ways in which
this bill might be abused by charlatans pushing treatments that do not
work?
Response. Again we see in the opening statement, the FDA, usurping
a patient's right to choose. Understand that ALL CANCER THERAPIES ARE
EXPERIMENTAL AT THIS TIME IN OUR SEARCH FOR THE CURE, and to deny a
patient access to their personal choice of treatment based on a
personal bias is IMMORAL. The bill does not open the door for
charlatans to abuse the law anymore than current laws do. This bill
would have allowed us access to the therapy of our choice, a therapy
that would not have destroyed our son as conventional treatment would.
Also understand that if we had been allowed access to it and it had not
worked, we would have stepped our mode of treatment to a more
aggressive treatment like chemotherapy or radiation. But to be FORCED
into a treatment from the onset is wrong. The FDA will still be allowed
to put clinical trials on hold that have safety concerns. It is the
role of the FDA to advance science monitoring the safety of trials
involving human subjects. It is not the role of the FDA to pick one
trial over another, or to stand in the way of advancing science by
excluding access to a clinical trial for a ``STANDARD OF CARE'' that
has never been through an FDA approval process for the specific
condition or age group in question.
Question 5. In a news account entitled ``PARENTS FIGHT TO SAVE
SON'', published by Wired News, the article stated that ``When the
Navarro's decided they wanted their son to be treated by Burzynski, the
FDA denied them permission, ruling that the treatment could only be
used as a last resort. FDA officials threatened to take Thomas into
protective custody if the Navarro's denied him traditional treatment.''
Do you have any documentation you can provide the Committee that the
FDA, or any other government agency, was going to place your son in
protective custody?
Response. First of all, I would like to state for the record that I
was unaware of this article until I read of it in your questions from
the subcommittee. With that said, I found a copy of the article and
read it. Now in answer to your question:
It is true that the FDA denied Thomas access to treatment at the
Burzynski Clinic, citing that there was no scientific, ethical, or
moral basis for allowing Thomas access to treatment when an existing
``STANDARD OF CARE'' was already available. It didn't matter to them
that the ``STANDARD OF CARE'' had such devastating and deadly results.
It didn't matter to them that in the ``STANDARD OF CARE'' two of the
three recommended chemotherapy drugs stated on the package insert,
``this product not proven safe or effective in the pediatric
population.'' It was their way of doing business, and to that end their
word was final.
In following their logic for a moment, if you have used the most
damaging and deadly treatment first, what is left of the patient to
treat if the patient has been rendered permanently damaged, or worse,
if the patient has died? At this point, treatments that would offer as
great or greater a hope have been rendered useless by the FDA's
decision making process. In effect, ``THEY HAVE THROWN THE BABY OUT,
WITH THE BATH WATER''.
It is incorrect that the FDA threatened to take Thomas into
protective custody. In actuality, When my wife and I made the decision
not to subject Thomas to the standard of care, Thomas's oncologist back
in Arizona swore out a complaint, alleging CHILD ABUSE, CHILD
ENDANGERMENT, AND MEDICAL NEGLECT for refusing a treatment which had
discovered would leave our son retarded, deaf, sterile, blind, with
stunted growth, and even dead. WHAT IS WRONG WITH THIS PICTURE? It is
our undaunted opinion, that the current ``STANDARD OF CARE'' is
LEGALIZED CHILD ABUSE. What loving parent would knowingly and willing
subject their child to this type of torture with no guarantee of
success, knowing that there were other treatments available, that
didn't do this type of damage that hadn't been tried first.
Question 6. Critics at the FDA state that by pursuing alternative
therapies that are ``not FDA approved'', you are placing your ``son at
greater risk of death'' than if he first pursued FDA approved therapies
that include radiation and chemotherapy, that may render your son to be
retarded. How would you respond to your critics at the agency?
Response. First, I would like to remind my critics at the FDA of an
oath they took many years ago. And I quote, ``FIRST, DO NO HARM'', it
is the beginning of the Hippocratic Oath. With that said, let me start
by saying, I agree, unapproved therapies can present risks. Some are
great and some are small. The risk is no greater than the risks taken
by using Radiation and Chemotherapy, which again I will remind you are,
`` NOT FDA APPROVED FOR TREATING PEDIATRIC CANCERS''. They are standard
treatments used, which ``IMPLIES APPROVAL'', it does not mean that have
been approved going through the ``NORMAL APPROVAL PROCESS''. LET US NOT
CONFUSE THE TWO! If under any other means, if I were to go out with
forethought and blind my son, or deafen my son or by some means, cause
him to become retarded I would be arrested, tried, and thrown into
jail, and rightfully so, but it is done everyday in the treating of
childhood cancer with NO ACCOUNTABILITY . And not allowing my son to be
subjected to these medieval treatments, makes me a bad parent in the
eyes of the ``STANDARD OF CARE COMMUNITY''. There is nothing in my life
that I love greater than my children, and they will be no doctor's
``LAB RAT'', not while I breath.
I hope this has clarified any questions you might have. Please feel
free to contact me if you have any further questions.
______
National Organization for Rare Disorders, Inc.
November 15, 2000
The Honorable Michael Bilirakis
Chairman
Subcommittee on Health and the Environment
US House of Representatives
2369 Rayburn House Office Bldg.
Washington, DC 20515
Dear Chairman Bilirakis: Thank you for your letter of November 2,
containing additional questions from the September 13, 2000 hearing on
H.R. 4242, the Orphan Drug Innovation Act. My answers to the questions
follow:
Question 1. Do you believe that shielding the original orphan drug
from competition for longer than 7 years is good policy?
Response. No. The Orphan Drug Act of 1993 provides seven years of
exclusive marketing rights to the sponsor of an orphan drug, and no
manufacturer should have more than seven years without competition.
However, there are many orphan drugs that have had no competition after
seven years on the market simply because no other companies have made
an effort to compete after the innovator's exclusivity expired.
On the other hand, the European Union enacted the Orphan Medical
Products Regulation in December 1999, providing ten years of
exclusivity to orphan drug manufacturers. Since this is a new law, we
do not yet know how successful it will be in comparison to the U.S.
law. In other words, is ten years of exclusivity a better incentive
than seven years? Will more companies develop orphan drugs in Europe
than the United States because American incentives are not as strong?
We believe it is premature for Congress to consider revising (either
contracting or expanding) orphan drug exclusivity in the United States
at this time.
Question 2. Do you believe that this policy needs clarification?
Response. Under very limited circumstances the FDA may allow the
manufacturer of a ``similar'' orphan drug to reach the American market
before an innovator's orphan drug exclusivity expires IF it can prove
that the follow-on drug is ``different'' from the original orphan drug.
A manufacturer must prove that its drug is chemically or structurally
different from the first orphan drug, or that it is superior because it
is more effective or safer, or represents a ``major contribution to
patient care.'' FDA's regulations defining these requirements were
established in 1992. We believe the regulations have worked very well
for many years, and they do not need to be revised at the current time.
Please note that the orphan drug regulations were printed nine
years after the Orphan Drug Act became law, they went through extensive
public comment periods, and they have been time tested since 1992. Mr.
Chairman we believe these regulations effectively carry out the spirit
and intent of the law, and they should NOT be written into law because
minor changes would subsequently require an act of Congress.
Regulations are easier to change when the need arises, and the public
is encouraged to comment and provide input into revised regulations.
Question 3. On February 7, 2000, you sent a letter to FDA
Commissioner Henney stating, ``As you probably know, we have been
concerned about the blockage of a new version of beta interferon form
multiple sclerosis. FDA seems to believe that the product is the same
as both Avonex and Betaseron, even though those two products have
already been determined by both FDA and a federal court to be
`different.' This puts Serono in a no-win situation, even with its full
BLA and having been initially determined to be `the same' as Betaseron
under the Orphan Drug Act.'' Why did you write that the FDA
implementation of the Orphan Drug Act puts Serono in a no-win
situation?
Response. If you read my entire letter of February 7, 2000 to FDA
Commissioner Henney, you will clearly see that the question I raised
involved FDA's refusal at the beginning of this year, to decide whether
Avonex was the ``same'' as Betaseron or the ``same'' as Avonex, but I
asserted it could not be the same as BOTH. The letter was an effort to
force the agency to decide which ONE of those two drugs Rebif is the
same as.
Let me explain. At the beginning of this year, FDA determined that
Rebif is the same as the two competing versions of the multiple
sclerosis treatments, Betaseron and Avonex. Betaseron was the first
version of beta interferon to reach the market, but FDA subsequently
decided that Avonex was a ``clinically superior'' orphan drug, and
therefore should be made available to multiple sclerosis patients
before Betaseron's exclusivity expired. The manufacturer of Betaseron
went to court to stop Avonex from reaching the market, claiming that
its orphan drug exclusivity would be violated. The court decided that
FDA's decision was correct and that Avonex is a ``different'' drug
because it was ``clearly superior,'' and thus could be approved by the
agency despite Betaseron's exclusivity.
Late last year, FDA told Serono that it could not approve its
multiple sclerosis treatment, Rebif, because it was the ``same'' as
both Betaseron and Avonex. I wrote to Commissioner Henney in February
explaining that FDA cannot claim Rebif is the ``same'' as both of these
drugs because the FDA had already determined, and a federal court had
agreed, that Betaseron and Avonex are not the same drug. Serono was in
a no-win situation because the company would have to prove that Rebif
was ``different'' than two different drugs, not one. I urged the
Commissioner to determine which of those two Multiple Sclerosis
treatments Rebif was the ``same'' as. I did not urge the Commissioner
to cast a vote toward one drug or the other; we simply wanted a
decision to be made. Later this year FDA did make the decision that
NORD was asking the FDA to make, and the agency decided that Rebif is
the ``same'' as Avonex. Apparently Serono disagrees with that decision.
Note. The answer to the following three questions is written below:
Question 4. Can you reconcile your written testimony today with
your February 7 letter to FDA Commissioner Henney where you state, in
reference to 7-year market exclusivity enjoyed by Biogen, ``However,
after seven years expire, competitors should be allowed on the market
without undue delay, and the beta interferon scenario, as a precedent,
very troubling.''
Question 5. Would you consider your February 7, 2000 letter to FDA
Commissioner Henney to be at variance with your testimony today?
Question 6. What caused you to change your mind?
Response. My testimony on September 13 conforms to the statements
made in the February 7 letter to Commissioner Henney. The beta
interferon scenario was very troubling because there was no way that
Rebif could be the ``same'' as both Avonex and Betaseron! It could only
be the same as ONE of those drugs.
I believe that FDA was wrong for not making a decision on this
matter much sooner, and for keeping their decision confidential when it
was made. Unfortunately, FDA is required under law to keep all
information confidential unless a drug sponsor agrees to make it
public. It was only after Serono gave written permission to FDA to talk
to me about Rebif that I was told the agency's scientific analysis
concluded that Rebif is the ``same'' as Avonex, and not the same as
Betaseron. This means FDA cannot approve Rebif for sale in the United
States until the exclusivity of Avonex expires. If Serono disagrees
with this decision, the onus is on the company to scientifically prove
their drug is not chemically the ``same'' as Avonex, or to prove their
drug is ``clinically superior'' to Avonex. I understand that Serono is
conducting clinical trials now in an effort to prove clinical
authority.
Question 7. The testimony of your organization was requested by
Biogen, the company that has been trying to thwart Serono's legislative
activities in this area. Have you had any contacts with Biogen or its
agents on this matter?
Response. I have had no contact with Biogen on this or any matter.
I am not surprised that Biogen recommended that we testify at the
hearing because we are usually invited to testify at all hearings
concerning the Orphan Drug Act. In fact, I was quite surprised when I
learned there would be a hearing about orphan drugs, and we were not
invited to testify. I finally received a telephone request from your
staff a few days before the hearing and was delighted to change my
plans and go to Washington, DC. I had no idea that Biogen made this
recommendation, and let me assure you NORD is not conspiring with
anyone to keep Serono off the market.
Since Biogen had no way to know what I would say in NORD's
testimony, it was certainly considerate of them to suggest that NORD be
included. However, people in Washington generally know that NORD is
always fair and unbiased, it is the quintessential patient advocacy
organization, it does not favor one drug over another, one company over
another, and NORD can always be relied on to defend the Orphan Drug Act
and its regulations.
Question 8. Do you believe that co-marketing during the original
orphan's exclusivity period, protection for that innovation, and the
opportunity to market that advantage are enough of an incentive to
foster research and development investment to improve orphan drugs? If
not why not?
Response. This is a very complicated question, but the basic answer
is: Exclusivity is the most important incentive of the Orphan Drug Act,
and any weakening of exclusive marketing rights would greatly undermine
development of future treatments for rare diseases. If you allow orphan
drug exclusivity to be violated because a manufacturer makes a minor
change to a drug that has no substantial benefit to patients, then you
will have diluted the ODA's chief incentive, and fatally weakened the
law.
Mr. Chairman, when a pharmaceutical company decides which drugs to
invest in, they prefer to focus R&D on the least risky products. This
is why so many drug companies invest billions of dollars in research
and development of ``me-too'' drugs, compounds that are very similar
and vary only slightly from other marketed drugs that are very
successful and profitable. They practically copy the original compound
and make some minor chemical or structural changes so that they do not
violate the innovator's patent, and then they get the drug on the
market to compete with the original drug. Historically this is why we
have so many beta blockers on the market for hypertension, so many
anti-inflammatories for arthritis, and soon there will be many drugs
for erectile dysfunction.
This is the way the marketplace works so well for drugs that are
used by large numbers of people. But orphan drugs affect few people,
manufacturers know there is a limited potential market to buy their
drug, and they want assurance that a competitor will not take part of
their market away. The industry did not manufacture orphan drugs before
the Orphan Drug Act was enacted (e.g., only ten orphan drugs were
brought to market in the 10 years before the Orphan Drug Act became
law); but since 1983, over 200 orphan drugs have reached the American
market.
These companies will tell you they would not have invested in
development of their orphan drug if they were not guaranteed seven
years of exclusivity.
In the very few instances when FDA has approved a ``similar''
orphan drug before the exclusivity of the first drug expired, it has
only occurred when manufacturers AGREED to SHARE exclusivity, or the
follow-on company PROVED SCIENTIFICALLY that their drug is clinically
superior.
In the case of the three versions of beta interferon the first
version (Betaseron) caused a very serious injection site reaction
necessitating surgery. The second version (Avonex) does not cause this
very serious adverse reaction, so it was allowed on the market. This is
a safety advantage, and it would have been unjust to prevent Avonex
from reaching Multiple sclerosis patients. Rebif on the other hand has
not yet proven that it is clinically superior to Avonex, FDA has
determined that Rebif is scientifically the ``same'' as Avonex, but it
also causes the same serious injection site reactions as Betaseron.
The onus is now on Serono to prove that their drug is clinically
superior to Avonex. The company is conducting a clinical trial right
now, comparing Avonex to Rebif. We believe that the scientists at FDA
will then be able to determine whether Rebif is clinically superior to
Avonex. Thus Congress should await the results of that trial and allow
FDA to make that determination. We believe that tinkering with the law
now, and diluting the exclusivity incentive of the Orphan Drug Act,
will weaken the law and remove any incentive for manufacturers to
develop clinically superior orphan drugs that patients need.
Question 9. Do you believe FDA's policy should distinguish between
drugs that are clinically superior based on improved safety or efficacy
from those based on being a major contribution to patient care?
Response. We believe FDA's current regulations defining clinical
superiority are right on target, and they should be left as they are.
If and when FDA decides to change the regulations, the agency is
required to publish the proposed revisions in the Federal Register, and
patients will then have an opportunity to express their point of view.
Today the agency will only approve a competing orphan drug if it can
prove that it is safer, more effective, or is a major contribution to
patient care. Patients want and need these therapeutic improvements.
The current regulations act as an important incentive to companies to
develop better orphan drugs.
Question 10. Do you object to Congress providing FDA with guidance
on handling clinically superior drugs, an issue not currently covered
in the statute?
Response. We do not believe it is necessary or warranted for
Congress to provide guidance to FDA on handling ``superior'' orphan
drugs. Firstly, we recommend that Congress ought to leave these
decisions up to the physicians and dentists at FDA. Secondly, although
``clinical superiority'' is not specifically mentioned in the statute,
it was absolutely proper and necessary for the agency to develop this
regulation because the terms ``same'' and ``different'' in the statute
had to be defined in regulations.
The FDA's orphan drug regulations define the terms ``same drug''
and ``different drug'' because recombinant DNA technology made this
necessary. Seventeen years ago when the law was written we were dealing
solely with chemical compounds that could easily be differentiated.
Today, however, many new treatments are developed through biotechnology
engineering and the differences between biologics is very difficult to
discern. One simply cannot define the chemical structure of a biologic
and determine if it is the same or different from a ``similar''
biologic. Most of these products are copies of proteins or enzymes that
human bodies naturally make. Moreover, Congress cannot develop a
formula that fits every clinically significant difference, and which
factors should be considered. For example, is a three-hour intravenous
infusion clinically superior to a six-hour infusion? Does a lower price
for a follow-on orphan drug represent clinical superiority? Once you
ask questions like these, you open a very big can of worms.
In conclusion Mr. Chairman, the patient community is indebted to
Congress for enacting the Orphan Drug Act in 1983. We advise that if
``it ain't broke, don't fix it,'' nor its regulations. Serono is doing
the right thing now; they are conducting clinical trials to try to
prove scientifically that its drug is superior to other competing
drugs. This process is available to them under the current Act and its
regulations. NORD's concern is not which company is right or wrong,
nor, how much profit they may lose when FDA denies them early marketing
approval, but whether patients are suffering because of lack of access.
There are three good FDA approved treatments for multiple sclerosis
available to American patients today, and at best any clinical
superiority that Serono will claim will not indicate that Rebif is a
cure for multiple sclerosis. If it were, FDA would approve it! Rebif
may be a treatment that is superior in some ways, but inferior in
others. Only science can tell us, and we await results of its clinical
trials comparing it directly to Avonex.
Please do not hesitate to contact me if you have any other
questions.
Very truly yours,
Abbey S. Meyers
President
______
University of Virginia
Department of Radiology
November 10, 2000
Congressman Michael Bilirakis
Chair, Subcommittee on Health and Environment
U.S. House of Representatives
Rayburn House Office Building, Room 2125
Washington, DC 20515-6115
Dear Mr. Bilirakis: The following are my responses to the questions
in your letter of November 2, concerning my testimony on HR 1795.
Question 1a. Why is an institute necessary to solve the problems
associated with biomedical imaging research at the NIH?
Response. That imaging research is spread over 16 institutes and
centers means that there is no single institute charged with
responsibility for support of basic research to develop new imaging
techniques and technologies with broad applications to the diseases and
organ systems that are the focus of the existing institutes.
Consequently, such research, which is critical to advances in imaging,
receives little or no support from the NIH. There is also no
coordination of imaging research at NIH. Major opportunities are lost,
as they are not apparent in the content of the institutes' disease
focus. There is duplication, as there is little coordination of imaging
opportunities. Given the declining ability of both academic departments
and industry to finance imaging research, an institute directed
specifically at imaging, with a comprehensive plan, able to prioritize
and pursue opportunities is essential.
Question 1b. Could the Bioengineering Consortium (BECON) that the
NIH has established, or a coordinating committee of some type be an
alternative?
Response. Such alternatives fall short of what is needed in both
scope and authority. An institute has the capability of addressing the
diverse needs that are essential to successfully addressing the
nation's needs with respect to medical imaging, including the authority
to fund grants, set a research priorities through the request for
applications process, and enable research training. This level of
comprehensiveness does not exist through any other mechanism.
Question 2. The National Cancer Institute has made imaging a top
research priority and has put much more money into this field. Can't we
solve the problem by further increasing the amount of money that NCI
commits to imaging?
Response. While more NCI money certainly can increase imaging
research in cancer, it does not address the need to advance imaging
research more broadly in support of the nation's health. Indeed, it is
this ``silo'' approach to imaging that is so ineffective. Imaging is
applicable across a broad range of organ systems and diseases and
needs, as such, to be addressed more directly.
Question 3. Other groups have come to the Congress requesting that
new institutes be created. How can Congress distinguish among these
proposals?
Response. The Institute of Medicine has advised Congress in a 1984
report titled Responding to Health Needs and Scientific Opportunity:
The Organizational Structure of the National Institutes of Health--on
when it should consider establishing a new institute. The proposed
Institute for Biomedical Imaging and Bioengineering fulfills all of
these criteria and is also consistent with a second IOM report, which
was written in 1998 and titled Scientific Opportunities and Public
Needs: Improving Priority Setting and Public Input at NIH.
Establishment of the proposed institute will advance research that will
have a positive impact on the public health while, at the same time,
reducing inefficiencies and duplication. Dr. Reed Dunnick discussed
both IOM reports at greater length in his testimony.
Question 4. What role can new imaging technologies play in the
advanced research in molecular biology and genetics that is conducted
by the other institutes at the NIH?
Response. Both advances in established imaging technologies and the
emergence of a host of new technologies promise spectacular
contributions to our understanding of the early phases of such
important disease processes as cancer and heart disease. Medical
imaging is, in essence, the ``noninvasive biopsy'' that can provide
insight into how subcellular structures are altered by disease in both
their morphology and function. Imaging technologies already are being
used for these purposes. Their importance in investigation, diagnosis,
and treatment will grow over the near term.
Question 5. What makes the basic scientific research involved in
imaging and bioengineering different from the scientific research at
the disease and organ-system institutes?
Response. The most fundamental difference is in the nature of the
training and expertise of the individuals involved. Medical imaging
technology development requires the skills of physicians, computer
scientists, physicists, mathematicians, information technology
specialists, and engineers. The research is interdisciplinary and cuts
across disease and organ system lines.
I appreciate the opportunity to further comment on the need for an
Institute for Biomedical Imaging and Bioengineering. Please let me know
if you wish me to address these or other issues further.
Sincerely,
Bruce J. Hillman, MD
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