Establishes a Food and Drug Administration (FDA) contract process for related labeling changes.
Amends the Federal Food, Drug, and Cosmetic Act to: (1) eliminate the user fee waiver for pediatric supplements to a human drug application; (2) provide priority status for pediatric supplements; (3) include neonates within the definition of pediatric studies; (4) provide for dissemination of pediatric supplement information; and (5) set forth requirements for the additional six-month exclusivity period for new or already-marketed pediatric drugs.
Directs the Secretary to establish an Office of Pediatric Therapeutics within the Office of the Commissioner of Food and Drugs, which shall coordinate all FDA pediatric activities.
Includes within the purposes and activities of the Foundation for the National Institutes of Health the collection of funds for pediatric pharmacological research.
Directs the Secretary to contract with the Institute of Medicine to review Federal regulations, reports, and support for research involving children, with particular attention to issues of compensation, informed consent, and risk/ benefits assessments in terms of research versus therapeutic treatment.
[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[H.R. 3452 Introduced in House (IH)]
107th CONGRESS
1st Session
H. R. 3452
To amend the Federal Food, Drug, and Cosmetic Act to improve the safety
and efficacy of pharmaceuticals for children.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
December 11, 2001
Mr. Greenwood (for himself and Ms. Eshoo) introduced the following
bill; which was referred to the Committee on Energy and Commerce
_______________________________________________________________________
A BILL
To amend the Federal Food, Drug, and Cosmetic Act to improve the safety
and efficacy of pharmaceuticals for children.
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress assembled,
SECTION 1. 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. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.
``(a) List of Drugs for Which Pediatric Studies Are Needed.--
``(1) In general.--Not later than 1 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));
``(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 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 later than 270 days after the date of
enactment of this section, the Commissioner of Food and 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
of the anti-infective drugs advisory committee.--Not
later than 90 days after receiving a referral 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 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 5
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.--
``(A) Request 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.''.
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 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
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.
``(C) Consideration 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. 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) 1 or more additional individuals with expertise
concerning ethical issues presented by the conduct of clinical
research in the pediatric population; and
(2) 1 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;
``(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 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 6 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
Cosmetics 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 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 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. 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 2
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:
(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
(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
(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. 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
the scientific and ethical consideration of topics of
pediatric cancer, as necessary:
(i) At least 2 pediatric oncology
specialists from the National Cancer Institute.
(ii) At least 4 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 2 representatives of the
pediatric cancer patient and patient-family
community.
(iv) 1 representative of the nursing
community.
(v) At least 1 statistician.
(vi) At least 1 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
(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 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. REPORT ON PEDIATRIC EXCLUSIVITY PROGRAM.
Not 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;
(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 numbers 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 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. ADVERSE-EVENT REPORTING.
(a) Toll-Free 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 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 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.
(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 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)''.
<all>
Introduced in House
Introduced in House
Referred to the House Committee on Energy and Commerce.
Referred to the Subcommittee on Health.
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