[107th Congress Public Law 109]
[From the U.S. Government Printing Office]
<DOC>
[DOCID: f:publ109.107]
[[Page 1407]]
BEST PHARMACEUTICALS FOR CHILDREN ACT
[[Page 115 STAT. 1408]]
Public Law 107-109
107th Congress
An Act
To amend the Federal Food, Drug, and Cosmetic Act to improve the safety
and efficacy of pharmaceuticals for children. <<NOTE: Jan. 4,
2002 - [S. 1789]>>
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress <<NOTE: Best Pharmaceuticals for
Children Act.>> assembled,
SECTION 1. <<NOTE: 21 USC 301 note.>> SHORT TITLE.
This Act may be cited as the ``Best Pharmaceuticals for Children
Act''.
SEC. 2. PEDIATRIC STUDIES OF ALREADY-MARKETED DRUGS.
Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355a) is amended--
(1) by striking subsection (b); and
(2) in subsection (c)--
(A) by inserting after ``the Secretary'' the
following: ``determines that information relating to the
use of an approved drug in the pediatric population may
produce health benefits in that population and''; and
(B) by striking ``concerning a drug identified in
the list described in subsection (b)''.
SEC. 3. RESEARCH FUND FOR THE STUDY OF DRUGS.
Part B of title IV of the Public Health Service Act (42 U.S.C. 284
et seq.) is amended--
(1) by redesignating the second section 409C, relating to
clinical research (42 U.S.C. 284k), as section 409G;
(2) by redesignating the second section 409D, relating to
enhancement awards (42 U.S.C. 284l), as section 409H; and
(3) by adding at the end the following:
``SEC. 409I. <<NOTE: 42 USC 284m.>> PROGRAM FOR PEDIATRIC STUDIES OF
DRUGS.
``(a) List of Drugs for Which Pediatric Studies Are Needed.--
``(1) In general.--Not later than one year after the date of
enactment of this section, the Secretary, acting through the
Director of the National Institutes of Health and in
consultation with the Commissioner of Food and Drugs and experts
in pediatric research, shall develop, prioritize, and publish an
annual list of approved drugs for which--
``(A)(i) there is an approved application under
section 505(j) of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355(j));
``(ii) there is a submitted application that could
be approved under the criteria of section 505(j) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(j));
[[Page 115 STAT. 1409]]
``(iii) there is no patent protection or market
exclusivity protection under the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 301 et seq.); or
``(iv) there is a referral for inclusion on the list
under section 505A(d)(4)(C) of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 355a(d)(4)(C)); and
``(B) in the case of a drug referred to in clause
(i), (ii), or (iii) of subparagraph (A), additional
studies are needed to assess the safety and
effectiveness of the use of the drug in the pediatric
population.
``(2) Consideration of available information.--In developing
and prioritizing the list under paragraph (1), the Secretary
shall consider, for each drug on the list--
``(A) the availability of information concerning the
safe and effective use of the drug in the pediatric
population;
``(B) whether additional information is needed;
``(C) whether new pediatric studies concerning the
drug may produce health benefits in the pediatric
population; and
``(D) whether reformulation of the drug is
necessary.
``(b) Contracts for Pediatric Studies.--The Secretary shall award
contracts to entities that have the expertise to conduct pediatric
clinical trials (including qualified universities, hospitals,
laboratories, contract research organizations, federally funded programs
such as pediatric pharmacology research units, other public or private
institutions, or individuals) to enable the entities to conduct
pediatric studies concerning one or more drugs identified in the list
described in subsection (a).
``(c) Process for Contracts and Labeling Changes.--
``(1) Written request to holders of approved applications
for drugs lacking exclusivity.--The Commissioner of Food and
Drugs, in consultation with the Director of the National
Institutes of Health, may issue a written request (which shall
include a timeframe for negotiations for an agreement) for
pediatric studies concerning a drug identified in the list
described in subsection (a)(1)(A) (except clause (iv)) to all
holders of an approved application for the drug under section
505 of the Federal Food, Drug, and Cosmetic Act. Such a written
request shall be made in a manner equivalent to the manner in
which a written request is made under subsection (a) or (b) of
section 505A of the Federal Food, Drug, and Cosmetic Act,
including with respect to information provided on the pediatric
studies to be conducted pursuant to the request.
``(2) Requests <<NOTE: Deadline.>> for contract proposals.--
If the Commissioner of Food and Drugs does not receive a
response to a written request issued under paragraph (1) within
30 days of the date on which a request was issued, or if a
referral described in subsection (a)(1)(A)(iv) is made, the
Secretary, acting through the Director of the National
Institutes of Health and in consultation with the Commissioner
of Food and Drugs, shall publish a request for contract
proposals to conduct the pediatric studies described in the
written request.
``(3) Disqualification.--A holder that receives a first
right of refusal shall not be entitled to respond to a request
for contract proposals under paragraph (2).
``(4) Guidance.--Not <<NOTE: Deadline.>> later than 270 days
after the date of enactment of this section, the Commissioner of
Food and
[[Page 115 STAT. 1410]]
Drugs shall promulgate guidance to establish the process for the
submission of responses to written requests under paragraph (1).
``(5) Contracts.--A contract under this section may be
awarded only if a proposal for the contract is submitted to the
Secretary in such form and manner, and containing such
agreements, assurances, and information as the Secretary
determines to be necessary to carry out this section.
``(6) Reporting of studies.--
``(A) In general.--On completion of a pediatric
study in accordance with a contract awarded under this
section, a report concerning the study shall be
submitted to the Director of the National Institutes of
Health and the Commissioner of Food and Drugs. The
report shall include all data generated in connection
with the study.
``(B) Availability of reports.--Each report
submitted under subparagraph (A) shall be considered to
be in the public domain (subject to section
505A(d)(4)(D) of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355a(d)(4)(D)) and shall be assigned a
docket number by the Commissioner of Food and Drugs. An
interested person may submit written comments concerning
such pediatric studies to the Commissioner of Food and
Drugs, and the written comments shall become part of the
docket file with respect to each of the drugs.
``(C) Action by commissioner.--The Commissioner of
Food and Drugs shall take appropriate action in response
to the reports submitted under subparagraph (A) in
accordance with paragraph (7).
``(7) Requests for labeling change.--During the 180-day
period after the date on which a report is submitted under
paragraph (6)(A), the Commissioner of Food and Drugs shall--
``(A) review the report and such other data as are
available concerning the safe and effective use in the
pediatric population of the drug studied;
``(B) negotiate with the holders of approved
applications for the drug studied for any labeling
changes that the Commissioner of Food and Drugs
determines to be appropriate and requests the holders to
make; and
``(C)(i) place in the public docket file a copy of
the report and of any requested labeling changes; and
``(ii) publish in the Federal Register a summary of
the report and a copy of any requested labeling changes.
``(8) Dispute resolution.--
``(A) Referral to pediatric advisory subcommittee of
the anti-infective drugs advisory committee.--If, not
later than the end of the 180-day period specified in
paragraph (7), the holder of an approved application for
the drug involved does not agree to any labeling change
requested by the Commissioner of Food and Drugs under
that paragraph, the Commissioner of Food and Drugs shall
refer the request to the Pediatric Advisory Subcommittee
of the Anti-Infective Drugs Advisory Committee.
``(B) Action by the pediatric advisory
subcommittee <<NOTE: Deadline.>> of the anti-infective
drugs advisory committee.--Not later than 90 days after
receiving a referral
[[Page 115 STAT. 1411]]
under subparagraph (A), the Pediatric Advisory
Subcommittee of the Anti-Infective Drugs Advisory
Committee shall--
``(i) review the available information on the
safe and effective use of the drug in the
pediatric population, including study reports
submitted under this section; and
``(ii) make a recommendation to the
Commissioner of Food and Drugs as to appropriate
labeling changes, if any.
``(9) FDA <<NOTE: Deadline.>> determination.--Not later than
30 days after receiving a recommendation from the Pediatric
Advisory Subcommittee of the Anti-Infective Drugs Advisory
Committee under paragraph (8)(B)(ii) with respect to a drug, the
Commissioner of Food and Drugs shall consider the recommendation
and, if appropriate, make a request to the holders of approved
applications for the drug to make any labeling change that the
Commissioner of Food and Drugs determines to be appropriate.
``(10) Failure to agree.--If a holder of an approved
application for a drug, within 30 days after receiving a request
to make a labeling change under paragraph (9), does not agree to
make a requested labeling change, the Commissioner may deem the
drug to be misbranded under the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 301 et seq.).
``(11) No effect on authority.--Nothing in this subsection
limits the authority of the United States to bring an
enforcement action under the Federal Food, Drug, and Cosmetic
Act when a drug lacks appropriate pediatric labeling. Neither
course of action (the Pediatric Advisory Subcommittee of the
Anti-Infective Drugs Advisory Committee process or an
enforcement action referred to in the preceding sentence) shall
preclude, delay, or serve as the basis to stay the other course
of action.
``(12) Recommendation for formulation changes.--If a
pediatric study completed under public contract indicates that a
formulation change is necessary and the Secretary agrees, the
Secretary shall send a nonbinding letter of recommendation
regarding that change to each holder of an approved application.
``(d) Authorization of Appropriations.--
``(1) In general.--There are authorized to be appropriated
to carry out this section--
``(A) $200,000,000 for fiscal year 2002; and
``(B) such sums as are necessary for each of the
five succeeding fiscal years.
``(2) Availability.--Any amount appropriated under paragraph
(1) shall remain available to carry out this section until
expended.''.
SEC. 4. WRITTEN REQUEST TO HOLDERS OF APPROVED APPLICATIONS FOR
DRUGS THAT HAVE MARKET EXCLUSIVITY.
Section 505A(d) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 355a(d)) is amended by adding at the end the following:
``(4) Written request to holders of approved applications
for drugs that have market exclusivity.--
[[Page 115 STAT. 1412]]
``(A) Request <<NOTE: Deadline.>> and response.--If
the Secretary makes a written request for pediatric
studies (including neonates, as appropriate) under
subsection (c) to the holder of an application approved
under section 505(b)(1), the holder, not later than 180
days after receiving the written request, shall respond
to the Secretary as to the intention of the holder to
act on the request by--
``(i) indicating when the pediatric studies
will be initiated, if the holder agrees to the
request; or
``(ii) indicating that the holder does not
agree to the request.
``(B) No agreement to request.--
``(i) Referral.--If the holder does not agree
to a written request within the time period
specified in subparagraph (A), and if the
Secretary determines that there is a continuing
need for information relating to the use of the
drug in the pediatric population (including
neonates, as appropriate), the Secretary shall
refer the drug to the Foundation for the National
Institutes of Health established under section 499
of the Public Health Service Act (42 U.S.C. 290b)
(referred to in this paragraph as the
`Foundation') for the conduct of the pediatric
studies described in the written request.
``(ii) Public notice.--The Secretary shall
give public notice of the name of the drug, the
name of the manufacturer, and the indications to
be studied made in a referral under clause (i).
``(C) Lack of funds.--On referral of a drug under
subparagraph (B)(i), the Foundation shall issue a
proposal to award a grant to conduct the requested
studies unless the Foundation certifies to the
Secretary, within a timeframe that the Secretary
determines is appropriate through guidance, that the
Foundation does not have funds available under section
499(j)(9)(B)(i) to conduct the requested studies. If the
Foundation so certifies, the Secretary shall refer the
drug for inclusion on the list established under section
409I of the Public Health Service Act for the conduct of
the studies.
``(D) Effect of subsection.--Nothing in this
subsection (including with respect to referrals from the
Secretary to the Foundation) alters or amends section
301(j) of this Act or section 552 of title 5 or section
1905 of title 18, United States Code.
``(E) No requirement to refer.--Nothing in this
subsection shall be construed to require that every
declined written request shall be referred to the
Foundation.
``(F) Written requests under subsection (b).--For
drugs under subsection (b) for which written requests
have not been accepted, if the Secretary determines that
there is a continuing need for information relating to
the use of the drug in the pediatric population
(including neonates, as appropriate), the Secretary
shall issue a written request under subsection (c) after
the date of approval of the drug.''.
[[Page 115 STAT. 1413]]
SEC. 5. TIMELY LABELING CHANGES FOR DRUGS GRANTED EXCLUSIVITY;
DRUG FEES.
(a) Elimination of User Fee Waiver for Pediatric Supplements.--
Section 736(a)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
379h(a)(1)) is amended--
(1) by striking subparagraph (F); and
(2) by redesignating subparagraph (G) as subparagraph (F).
(b) Labeling Changes.--
(1) Definition of priority supplement.--Section 201 of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 321) is amended
by adding at the end the following:
``(kk) Priority supplement.--The term `priority supplement'
means a drug application referred to in section 101(4) of the
Food and Drug Administration Modernization Act of 1997 (111
Stat. 2298).''.
(2) Treatment as priority supplements.--Section 505A of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is amended
by adding at the end the following:
``(l) Labeling Supplements.--
``(1) Priority status for pediatric supplements.--Any
supplement to an application under section 505 proposing a
labeling change pursuant to a report on a pediatric study under
this section--
``(A) shall be considered to be a priority
supplement; and
``(B) shall be subject to the performance goals
established by the Commissioner for priority drugs.
``(2) Dispute resolution.--
``(A) Request <<NOTE: Deadline.>> for labeling
change and failure to agree.--If the Commissioner
determines that an application with respect to which a
pediatric study is conducted under this section is
approvable and that the only open issue for final action
on the application is the reaching of an agreement
between the sponsor of the application and the
Commissioner on appropriate changes to the labeling for
the drug that is the subject of the application, not
later than 180 days after the date of submission of the
application--
``(i) the Commissioner shall request that the
sponsor of the application make any labeling
change that the Commissioner determines to be
appropriate; and
``(ii) if the sponsor of the application does
not agree to make a labeling change requested by
the Commissioner, the Commissioner shall refer the
matter to the Pediatric Advisory Subcommittee of
the Anti-Infective Drugs Advisory Committee.
``(B) Action by the pediatric advisory subcommittee
of the anti-infective drugs advisory committee.--
Not <<NOTE: Deadline.>> later than 90 days after
receiving a referral under subparagraph (A)(ii), the
Pediatric Advisory Subcommittee of the Anti-Infective
Drugs Advisory Committee shall--
``(i) review the pediatric study reports; and
``(ii) make a recommendation to the
Commissioner concerning appropriate labeling
changes, if any.
[[Page 115 STAT. 1414]]
``(C) Consideration <<NOTE: Deadline.>> of
recommendations.--The Commissioner shall consider the
recommendations of the Pediatric Advisory Subcommittee
of the Anti-Infective Drugs Advisory Committee and, if
appropriate, not later than 30 days after receiving the
recommendation, make a request to the sponsor of the
application to make any labeling change that the
Commissioner determines to be appropriate.
``(D) Misbranding.--If the sponsor of the
application, within 30 days after receiving a request
under subparagraph (C), does not agree to make a
labeling change requested by the Commissioner, the
Commissioner may deem the drug that is the subject of
the application to be misbranded.
``(E) No effect on authority.--Nothing in this
subsection limits the authority of the United States to
bring an enforcement action under this Act when a drug
lacks appropriate pediatric labeling. Neither course of
action (the Pediatric Advisory Subcommittee of the Anti-
Infective Drugs Advisory Committee process or an
enforcement action referred to in the preceding
sentence) shall preclude, delay, or serve as the basis
to stay the other course of action.''.
SEC. 6. <<NOTE: 21 USC 393a.>> OFFICE OF PEDIATRIC THERAPEUTICS.
(a) Establishment.--The Secretary of Health and Human Services shall
establish an Office of Pediatric Therapeutics within the Food and Drug
Administration.
(b) Duties.--The Office of Pediatric Therapeutics shall be
responsible for coordination and facilitation of all activities of the
Food and Drug Administration that may have any effect on a pediatric
population or the practice of pediatrics or may in any other way involve
pediatric issues.
(c) Staff.--The staff of the Office of Pediatric Therapeutics shall
coordinate with employees of the Department of Health and Human Services
who exercise responsibilities relating to pediatric therapeutics and
shall include--
(1) one or more additional individuals with expertise
concerning ethical issues presented by the conduct of clinical
research in the pediatric population; and
(2) one or more additional individuals with expertise in
pediatrics as may be necessary to perform the activities
described in subsection (b).
SEC. 7. NEONATES.
Section 505A(g) of the Federal Food, Drug, and Cosmetic Act (21
U.S.C. 355a(g)) is amended by inserting ``(including neonates in
appropriate cases)'' after ``pediatric age groups''.
SEC. 8. SUNSET.
Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355a) is amended by striking subsection (j) and inserting the following:
``(j) Sunset.--A drug may not receive any 6-month period under
subsection (a) or (c) unless--
``(1) on or before October 1, 2007, the Secretary makes a
written request for pediatric studies of the drug;
[[Page 115 STAT. 1415]]
``(2) on or before October 1, 2007, an application for the
drug is accepted for filing under section 505(b); and
``(3) all requirements of this section are met.''.
SEC. 9. DISSEMINATION OF PEDIATRIC INFORMATION.
Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355a) (as amended by section 5(b)(2)) is amended by adding at the end
the following:
``(m) Dissemination of Pediatric Information.--
``(1) In general.--Not <<NOTE: Deadline.>> later than 180
days after the date of submission of a report on a pediatric
study under this section, the Commissioner shall make available
to the public a summary of the medical and clinical pharmacology
reviews of pediatric studies conducted for the supplement,
including by publication in the Federal Register.
``(2) Effect of subsection.--Nothing in this subsection
alters or amends section 301(j) of this Act or section 552 of
title 5 or section 1905 of title 18, United States Code.''.
SEC. 10. CLARIFICATION OF INTERACTION OF PEDIATRIC EXCLUSIVITY
UNDER SECTION 505A OF THE FEDERAL FOOD,
DRUG, AND COSMETIC ACT AND 180-DAY
EXCLUSIVITY AWARDED TO AN APPLICANT FOR
APPROVAL OF A DRUG UNDER SECTION 505(j) OF
THAT ACT.
Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355a) (as amended by section 9) is amended by adding at the end the
following:
``(n) Clarification of Interaction of Market Exclusivity Under This
Section and Market Exclusivity Awarded to an Applicant for Approval of a
Drug Under Section 505(j).--If a 180-day period under section
505(j)(5)(B)(iv) overlaps with a 6-month exclusivity period under this
section, so that the applicant for approval of a drug under section
505(j) entitled to the 180-day period under that section loses a portion
of the 180-day period to which the applicant is entitled for the drug,
the 180-day period shall be extended from--
``(1) the date on which the 180-day period would have
expired by the number of days of the overlap, if the 180-day
period would, but for the application of this subsection, expire
after the 6-month exclusivity period; or
``(2) the date on which the 6-month exclusivity period
expires, by the number of days of the overlap if the 180-day
period would, but for the application of this subsection, expire
during the six-month exclusivity period.''.
SEC. 11. PROMPT APPROVAL OF DRUGS UNDER SECTION 505(j) WHEN
PEDIATRIC INFORMATION IS ADDED TO LABELING.
(a) In General.--Section 505A of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 355a) (as amended by section 10) is amended by
adding at the end the following:
``(o) Prompt Approval of Drugs Under Section 505(j) When Pediatric
Information Is Added to Labeling.--
``(1) General rule.--A drug for which an application has
been submitted or approved under section 505(j) shall not be
considered ineligible for approval under that section or
misbranded under section 502 on the basis that the labeling of
the drug omits a pediatric indication or any other aspect of
labeling pertaining to pediatric use when the omitted indication
[[Page 115 STAT. 1416]]
or other aspect is protected by patent or by exclusivity under
clause (iii) or (iv) of section 505(j)(5)(D).
``(2) Labeling.--Notwithstanding clauses (iii) and (iv) of
section 505(j)(5)(D), the Secretary may require that the
labeling of a drug approved under section 505(j) that omits a
pediatric indication or other aspect of labeling as described in
paragraph (1) include--
``(A) a statement that, because of marketing
exclusivity for a manufacturer--
``(i) the drug is not labeled for pediatric
use; or
``(ii) in the case of a drug for which there
is an additional pediatric use not referred to in
paragraph (1), the drug is not labeled for the
pediatric use under paragraph (1); and
``(B) a statement of any appropriate pediatric
contraindications, warnings, or precautions that the
Secretary considers necessary.
``(3) Preservation of pediatric exclusivity and other
provisions.--This subsection does not affect--
``(A) the availability or scope of exclusivity under
this section;
``(B) the availability or scope of exclusivity under
section 505 for pediatric formulations;
``(C) the question of the eligibility for approval
of any application under section 505(j) that omits any
other conditions of approval entitled to exclusivity
under clause (iii) or (iv) of section 505(j)(5)(D); or
``(D) except as expressly provided in paragraphs (1)
and (2), the operation of section 505.''.
(b) Effective <<NOTE: 21 USC 355a note.>> Date.--The amendment made
by subsection (a) takes effect on the date of enactment of this Act,
including with respect to applications under section 505(j) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(j)) that are
approved or pending on that date.
SEC. 12. <<NOTE: 42 USC 289 note.>> STUDY CONCERNING RESEARCH INVOLVING
CHILDREN.
(a) Contract With Institute of Medicine.--The Secretary of Health
and Human Services shall enter into a contract with the Institute of
Medicine for--
(1) the conduct, in accordance with subsection (b), of a
review of--
(A) Federal regulations in effect on the date of the
enactment of this Act relating to research involving
children;
(B) federally prepared or supported reports relating
to research involving children; and
(C) federally supported evidence-based research
involving children; and
(2) the submission to the Committee on Health, Education,
Labor, and Pensions of the Senate and the Committee on Energy
and Commerce of the House of Representatives, not later than two
years after the date of enactment of this Act, of a report
concerning the review conducted under paragraph (1) that
includes recommendations on best practices relating to research
involving children.
(b) Areas of Review.--In conducting the review under subsection
(a)(1), the Institute of Medicine shall consider the following:
[[Page 115 STAT. 1417]]
(1) The written and oral process of obtaining and defining
``assent'', ``permission'' and ``informed consent'' with respect
to child clinical research participants and the parents,
guardians, and the individuals who may serve as the legally
authorized representatives of such children (as defined in
subpart A of part 46 of title 45, Code of Federal Regulations).
(2) The expectations and comprehension of child research
participants and the parents, guardians, or legally authorized
representatives of such children, for the direct benefits and
risks of the child's research involvement, particularly in terms
of research versus therapeutic treatment.
(3) The definition of ``minimal risk'' with respect to a
healthy child or a child with an illness.
(4) The appropriateness of the regulations applicable to
children of differing ages and maturity levels, including
regulations relating to legal status.
(5) Whether payment (financial or otherwise) may be provided
to a child or his or her parent, guardian, or legally authorized
representative for the participation of the child in research,
and if so, the amount and type of payment that may be made.
(6) Compliance with the regulations referred to in
subsection (a)(1)(A), the monitoring of such compliance
(including the role of institutional review boards), and the
enforcement actions taken for violations of such regulations.
(7) The unique roles and responsibilities of institutional
review boards in reviewing research involving children,
including composition of membership on institutional review
boards.
(c) Requirements of Expertise.--The Institute of Medicine shall
conduct the review under subsection (a)(1) and make recommendations
under subsection (a)(2) in conjunction with experts in pediatric
medicine, pediatric research, and the ethical conduct of research
involving children.
SEC. 13. FOUNDATION FOR THE NATIONAL INSTITUTES OF HEALTH.
Section 499 of the Public Health Service Act (42 U.S.C. 290b) is
amended--
(1) in subsection (b), by inserting ``(including collection
of funds for pediatric pharmacologic research)'' after
``mission'';
(2) in subsection (c)(1)--
(A) by redesignating subparagraph (C) as
subparagraph (D); and
(B) by inserting after subparagraph (B) the
following:
``(C) A program to collect funds for pediatric
pharmacologic research and studies listed by the
Secretary pursuant to section 409I(a)(1)(A) of this Act
and referred under section 505A(d)(4)(C) of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C.
355a(d)(4)(C)).'';
(3) in subsection (d)--
(A) in paragraph (1)--
(i) in subparagraph (B)--
(I) in clause (ii), by striking
``and'' at the end;
(II) in clause (iii), by striking
the period and inserting ``; and''; and
(III) by adding at the end the
following:
``(iv) the Commissioner of Food and Drugs.'';
and
[[Page 115 STAT. 1418]]
(ii) by striking subparagraph (C) and
inserting the following:
``(C) The ex officio members of the Board under
subparagraph (B) shall appoint to the Board individuals
from among a list of candidates to be provided by the
National Academy of Science. Such appointed members
shall include--
``(i) representatives of the general
biomedical field;
``(ii) representatives of experts in pediatric
medicine and research;
``(iii) representatives of the general
biobehavioral field, which may include experts in
biomedical ethics; and
``(iv) representatives of the general public,
which may include representatives of affected
industries.''; and
(B) in paragraph (2), by realigning the margin of
subparagraph (B) to align with subparagraph (A);
(4) in subsection (k)(9)--
(A) by striking ``The Foundation'' and inserting the
following:
``(A) In general.--The Foundation''; and
(B) by adding at the end the following:
``(B) Gifts, grants, and other donations.--
``(i) In general.--Gifts, grants, and other
donations to the Foundation may be designated for
pediatric research and studies on drugs, and funds
so designated shall be used solely for grants for
research and studies under subsection (c)(1)(C).
``(ii) Other gifts.--Other gifts, grants, or
donations received by the Foundation and not
described in clause (i) may also be used to
support such pediatric research and studies.
``(iii) Report.--The recipient of a grant for
research and studies shall agree to provide the
Director of the National Institutes of Health and
the Commissioner of Food and Drugs, at the
conclusion of the research and studies--
``(I) a report describing the
results of the research and studies; and
``(II) all data generated in
connection with the research and
studies.
``(iv) Action by the commissioner of food and
drugs.--The Commissioner of Food and Drugs shall
take appropriate action in response to a report
received under clause (iii) in accordance with
paragraphs (7) through (12) of section 409I(c),
including negotiating with the holders of approved
applications for the drugs studied for any
labeling changes that the Commissioner determines
to be appropriate and requests the holders to
make.
``(C) Applicability.--Subparagraph (A) does not
apply to the program described in subsection
(c)(1)(C).'';
(5) by redesignating subsections (f) through (m) as
subsections (e) through (l), respectively;
(6) in subsection (h)(11) (as so redesignated), by striking
``solicit'' and inserting ``solicit,''; and
[[Page 115 STAT. 1419]]
(7) in paragraphs (1) and (2) of subsection (j) (as so
redesignated), by striking ``(including those developed under
subsection (d)(2)(B)(i)(II))'' each place it appears.
SEC. 14. <<NOTE: 42 USC 284m note.>> PEDIATRIC PHARMACOLOGY ADVISORY
COMMITTEE.
(a) In General.--The Secretary of Health and Human Services shall,
under section 222 of the Public Health Service Act (42 U.S.C. 217a),
convene and consult an advisory committee on pediatric pharmacology
(referred to in this section as the ``advisory committee'').
(b) Purpose.--
(1) In general.--The advisory committee shall advise and
make recommendations to the Secretary, through the Commissioner
of Food and Drugs and in consultation with the Director of the
National Institutes of Health, on matters relating to pediatric
pharmacology.
(2) Matters included.--The matters referred to in paragraph
(1) include--
(A) pediatric research conducted under sections 351,
409I, and 499 of the Public Health Service Act and
sections 501, 502, 505, and 505A of the Federal Food,
Drug, and Cosmetic Act;
(B) identification of research priorities related to
pediatric pharmacology and the need for additional
treatments of specific pediatric diseases or conditions;
and
(C) the ethics, design, and analysis of clinical
trials related to pediatric pharmacology.
(c) Composition.--The advisory committee shall include
representatives of pediatric health organizations, pediatric
researchers, relevant patient and patient-family organizations, and
other experts selected by the Secretary.
SEC. 15. PEDIATRIC SUBCOMMITTEE OF THE ONCOLOGIC DRUGS ADVISORY
COMMITTEE.
(a) Clarification of Authorities.--
(1) In general.--The Pediatric Subcommittee of the Oncologic
Drugs Advisory Committee (referred to in this section as the
``Subcommittee''), in carrying out the mission of reviewing and
evaluating the data concerning the safety and effectiveness of
marketed and investigational human drug products for use in the
treatment of pediatric cancers, shall--
(A) evaluate and, to the extent practicable,
prioritize new and emerging therapeutic alternatives
available to treat pediatric cancer;
(B) provide recommendations and guidance to help
ensure that children with cancer have timely access to
the most promising new cancer therapies; and
(C) advise on ways to improve consistency in the
availability of new therapeutic agents.
(2) Membership.--
(A) In general.--The Secretary shall appoint not
more than 11 voting members to the Pediatric
Subcommittee from the membership of the Pediatric
Pharmacology Advisory Committee and the Oncologic Drugs
Advisory Committee.
(B) Request for participation.--The Subcommittee
shall request participation of the following members in
[[Page 115 STAT. 1420]]
the scientific and ethical consideration of topics of
pediatric cancer, as necessary:
(i) At least two pediatric oncology
specialists from the National Cancer Institute.
(ii) At least four pediatric oncology
specialists from--
(I) the Children's Oncology Group;
(II) other pediatric experts with an
established history of conducting
clinical trials in children; or
(III) consortia sponsored by the
National Cancer Institute, such as the
Pediatric Brain Tumor Consortium, the
New Approaches to Neuroblastoma Therapy
or other pediatric oncology consortia.
(iii) At least two representatives of the
pediatric cancer patient and patient-family
community.
(iv) One representative of the nursing
community.
(v) At least one statistician.
(vi) At least one representative of the
pharmaceutical industry.
(b) Pre-Clinical Models To Evaluate Promising Pediatric Cancer
Therapies.--Section 413 of the Public Health Service Act (42 U.S.C.
285a-2) is amended by adding at the end the following:
``(c) Pre-Clinical Models To Evaluate Promising Pediatric Cancer
Therapies.--
``(1) Expansion and coordination of activities.--The
Director of the National Cancer Institute shall expand,
intensify, and coordinate the activities of the Institute with
respect to research on the development of preclinical models to
evaluate which therapies are likely to be effective for treating
pediatric cancer.
``(2) Coordination with other institutes.--The Director of
the Institute shall coordinate the activities under paragraph
(1) with similar activities conducted by other national research
institutes and agencies of the National Institutes of Health to
the extent that those Institutes and agencies have
responsibilities that are related to pediatric cancer.''.
(c) Clarification of Availability of Investigational New Drugs for
Pediatric Study and Use.--
(1) Amendment of the federal food, drug, and cosmetic act.--
Section 505(i)(1) of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 355(i)(1)) is amended--
(A) in subparagraph (B), by striking ``and'' at the
end;
(B) in subparagraph (C), by striking the period at
the end and inserting ``; and''; and
(C) by adding at the end the following:
``(D) the submission to the Secretary by the
manufacturer or the sponsor of the investigation of a
new drug of a statement of intent regarding whether the
manufacturer or sponsor has plans for assessing
pediatric safety and efficacy.''.
(2) Amendment of the public health service act.--Section
402(j)(3)(A) of the Public Health Service Act (42 U.S.C.
282(j)(3)(A)) is amended in the first sentence--
(A) by striking ``trial sites, and'' and inserting
``trial sites,''; and
[[Page 115 STAT. 1421]]
(B) by striking ``in the trial,'' and inserting ``in
the trial, and a description of whether, and through
what procedure, the manufacturer or sponsor of the
investigation of a new drug will respond to requests for
protocol exception, with appropriate safeguards, for
single-patient and expanded protocol use of the new
drug, particularly in children,''.
(d) Report.--Not <<NOTE: Deadline. 21 USC 355 note.>> later than
January 31, 2003, the Secretary of Health and Human Services, acting
through the Commissioner of Food and Drugs and in consultation with the
Director of the National Institutes of Health, shall submit to the
Committee on Health, Education, Labor, and Pensions of the Senate and
the Committee on Energy and Commerce of the House of Representatives a
report on patient access to new therapeutic agents for pediatric cancer,
including access to single patient use of new therapeutic agents.
SEC. 16. <<NOTE: 21 USC 355a note.>> REPORT ON PEDIATRIC EXCLUSIVITY
PROGRAM.
Not <<NOTE: Deadline.>> later than October 1, 2006, the Comptroller
General of the United States, in consultation with the Secretary of
Health and Human Services, shall submit to Congress a report that
addresses the following issues, using publicly available data or data
otherwise available to the Government that may be used and disclosed
under applicable law:
(1) The effectiveness of section 505A of the Federal Food,
Drug, and Cosmetic Act and section 409I of the Public Health
Service Act (as added by this Act) in ensuring that medicines
used by children are tested and properly labeled, including--
(A) the number and importance of drugs for children
that are being tested as a result of this legislation
and the importance for children, health care providers,
parents, and others of labeling changes made as a result
of such testing;
(B) the number and importance of drugs for children
that are not being tested for their use notwithstanding
the provisions of this legislation, and possible reasons
for the lack of testing; and
(C) the number of drugs for which testing is being
done, exclusivity granted, and labeling changes
required, including the date pediatric exclusivity is
granted and the date labeling changes are made and which
labeling changes required the use of the dispute
resolution process established pursuant to the
amendments made by this Act, together with a description
of the outcomes of such process, including a description
of the disputes and the recommendations of the Pediatric
Advisory Subcommittee of the Anti-Infective Drugs
Advisory Committee.
(2) The economic impact of section 505A of the Federal Food,
Drug, and Cosmetic Act and section 409I of the Public Health
Service Act (as added by this Act), including an estimate of--
(A) the costs to taxpayers in the form of higher
expenditures by medicaid and other Government programs;
(B) sales for each drug during the 6-month period
for which exclusivity is granted, as attributable to
such exclusivity;
[[Page 115 STAT. 1422]]
(C) costs to consumers and private insurers as a
result of any delay in the availability of lower cost
generic equivalents of drugs tested and granted
exclusivity under the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 301 et seq.), and loss of revenue by the
generic drug industry and retail pharmacies as a result
of any such delay; and
(D) the benefits to the government, to private
insurers, and to consumers resulting from decreased
health care costs, including--
(i) decreased hospitalizations and fewer
medical errors, due to more appropriate and more
effective use of medications in children as a
result of testing and re-labeling because of the
amendments made by this Act;
(ii) direct and indirect benefits associated
with fewer physician visits not related to
hospitalization;
(iii) benefits to children from missing less
time at school and being less affected by chronic
illnesses, thereby allowing a better quality of
life;
(iv) benefits to consumers from lower health
insurance premiums due to lower treatment costs
and hospitalization rates; and
(v) benefits to employers from reduced need
for employees to care for family members.
(3) The nature and type of studies in children for each drug
granted exclusivity under the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 301 et seq.), including--
(A) a description of the complexity of the studies;
(B) the number of study sites necessary to obtain
appropriate data;
(C) the number of children involved in any clinical
studies; and
(D) the estimated cost of each of the studies.
(4) Any recommendations for modifications to the programs
established under section 505A of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 355a) and section 409I of the Public
Health Service Act (as added by section 3) that the Secretary
determines to be appropriate, including a detailed rationale for
each recommendation.
(5) The increased private and Government-funded pediatric
research capability associated with this Act and the amendments
made by this Act.
(6) The number of written requests and additional letters of
recommendation that the Secretary issues.
(7) The prioritized list of off-patent drugs for which the
Secretary issues written requests.
(8)(A) The efforts made by the Secretary to increase the
number of studies conducted in the neonate population; and
(B) the results of those efforts, including efforts made to
encourage the conduct of appropriate studies in neonates by
companies with products that have sufficient safety and other
information to make the conduct of studies ethical and safe.
SEC. 17. <<NOTE: 21 USC 355b.>> ADVERSE-EVENT REPORTING.
(a) Toll-Free <<NOTE: Deadline. Regulations.>> Number in Labeling.--
Not later than one year after the date of the enactment of this Act, the
Secretary of Health and Human Services shall promulgate a final rule
requiring that
[[Page 115 STAT. 1423]]
the labeling of each drug for which an application is approved under
section 505 of the Federal Food, Drug, and Cosmetic Act (regardless of
the date on which approved) include the toll-free number maintained by
the Secretary for the purpose of receiving reports of adverse events
regarding drugs and a statement that such number is to be used for
reporting purposes only, not to receive medical advice. With respect to
the final rule:
(1) The rule shall provide for the implementation of such
labeling requirement in a manner that the Secretary considers to
be most likely to reach the broadest consumer audience.
(2) In promulgating the rule, the Secretary shall seek to
minimize the cost of the rule on the pharmacy profession.
(3) The rule shall take effect not later than 60 days after
the date on which the rule is promulgated.
(b) Drugs With Pediatric Market Exclusivity.--
(1) In general.--During the one year beginning on the date
on which a drug receives a period of market exclusivity under
505A of the Federal Food, Drug, and Cosmetic Act, any report of
an adverse event regarding the drug that the Secretary of Health
and Human Services receives shall be referred to the Office of
Pediatric Therapeutics established under section 6 of this Act.
In considering the report, the Director of such Office shall
provide for the review of the report by the Pediatric Advisory
Subcommittee of the Anti-Infective Drugs Advisory Committee,
including obtaining any recommendations of such subcommittee
regarding whether the Secretary should take action under the
Federal Food, Drug, and Cosmetic Act in response to the report.
(2) Rule of construction.--Paragraph (1) may not be
construed as restricting the authority of the Secretary of
Health and Human Services to continue carrying out the
activities described in such paragraph regarding a drug after
the one-year period described in such paragraph regarding the
drug has expired.
SEC. 18. MINORITY CHILDREN AND PEDIATRIC-EXCLUSIVITY PROGRAM.
(a) Protocols for Pediatric Studies.--Section 505A of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is amended in subsection
(d)(2) by inserting after the first sentence the following: ``In
reaching an agreement regarding written protocols, the Secretary shall
take into account adequate representation of children of ethnic and
racial minorities.''.
(b) Study <<NOTE: 21 USC 355a note.>> by General Accounting
Office.--
(1) In general.--The Comptroller General of the United
States shall conduct a study for the purpose of determining the
following:
(A) The extent to which children of ethnic and
racial minorities are adequately represented in studies
under section 505A of the Federal Food, Drug, and
Cosmetic Act; and to the extent ethnic and racial
minorities are not adequately represented, the reasons
for such under representation and recommendations to
increase such representation.
[[Page 115 STAT. 1424]]
(B) Whether the Food and Drug Administration has
appropriate management systems to monitor the
representation of the children of ethnic and racial
minorities in such studies.
(C) Whether drugs used to address diseases that
disproportionately affect racial and ethnic minorities
are being studied for their safety and effectiveness
under section 505A of the Federal Food, Drug, and
Cosmetic Act.
(2) Date <<NOTE: Deadline.>> certain for completing study.--
Not later than January 10, 2003, the Comptroller General shall
complete the study required in paragraph (1) and submit to the
Congress a report describing the findings of the study.
SEC. 19. TECHNICAL AND CONFORMING AMENDMENTS.
Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
355a) (as amended by sections 2(1), 5(b)(2), 9, 10, 11, and 17) is
amended--
(1)(A) by striking ``(j)(4)(D)(ii)'' each place it appears
and inserting ``(j)(5)(D)(ii)'';
(B) by striking ``(j)(4)(D)'' each place it appears and
inserting ``(j)(5)(D)''; and
(C) by striking ``505(j)(4)(D)'' each place it appears and
inserting ``505(j)(5)(D)'';
(2) by redesignating subsections (a), (g), (h), (i), (j),
(k), (l), (m), (n), and (o) as subsections (b), (a), (g), (h),
(n), (m), (i), (j), (k), and (l) respectively;
(3) by moving the subsections so as to appear in
alphabetical order;
(4) in paragraphs (1), (2), and (3) of subsection (d),
subsection (e), and subsection (m) (as redesignated by paragraph
(2)), by striking ``subsection (a) or (c)'' and inserting
``subsection (b) or (c)''; and
(5) in subsection (g) (as redesignated by paragraph (2)), by
striking ``subsection (a) or (b)'' and inserting ``subsection
(b) or (c)''.
Approved January 4, 2002.
LEGISLATIVE HISTORY--S. 1789 (H.R. 2887):
---------------------------------------------------------------------------
HOUSE REPORTS: No. 107-277 accompanying H.R. 2887 (Comm. on Energy and
Commerce).
CONGRESSIONAL RECORD, Vol. 147 (2001):
Dec. 12, considered and passed Senate.
Dec. 18, considered and passed House.
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