[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[S. 1789 Enrolled Bill (ENR)]
S.1789
One Hundred Seventh Congress
of the
United States of America
AT THE FIRST SESSION
Begun and held at the City of Washington on Wednesday,
the third day of January, two thousand and one
An Act
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,

a68f19c3046fbad03eb15741fba01513a27252cb11a33d681ce2bd32c856af5b

This Act may be cited as the ``Best Pharmaceuticals for Children
Act''.

6e7d7d4b8b65cfbd0627ea8f0f803e8988787ec63a7468177dfbdf713eab6d88

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)''.

adb030423bf6fdea03739bce2a50f36bbc82939711a655cfcb7d7b7c39c161c7

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 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));
``(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 five
succeeding fiscal years.
``(2) Availability.--Any amount appropriated under paragraph
(1) shall remain available to carry out this section until
expended.''.

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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.''.

822af9c54cf757b844b047a48a2c93a83df8400234e712a122adcdbbde8626bf

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.''.

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(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).

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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''.

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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.''.

d8bdc4c24ce54bccf84586e240562130511aa1d524572503316a6b04d3fb60c6

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.''.

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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.''.

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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 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.

882105acbacec27f83b510fed0815eed2675409c2c682c448e12afc00495bbd8

(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:
(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.

5339d924c9361b334f0dcb20e8398a990d8a0988612948aa96d94f3a5cc6f3cf

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.

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(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.

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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 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
(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.

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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 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.

de4decb7b49bd609ee3d29d7b5c151dc83ff4050ca51ee9164454bcad44f34bc

(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.

e94a08e3c046e067b200783215367df117e41fb4fad428875276065719015581

(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.

930311c96a57b2fec4e6e438d496802de7514db55350f1c1bdaeae41b652d0e8

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)''.
Speaker of the House of Representatives.
Vice President of the United States and
President of the Senate.

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Best Pharmaceuticals for Children Act

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