I
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 Representa-
tives 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 Cos-
metic 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 identi-
fied 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 STUD-
IES 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 Insti-
tutes of Health and in consultation with the Com-
missioner of Food and Drugs and experts in pedi-
atric 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 Cos-
metic Act (21 U.S.C. 355(j));
‘‘(iii) there is no patent protection or mar-
ket 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), ad-
ditional studies are needed to assess the safety
and effectiveness of the use of the drug in the
pediatric population.
‘‘(2) CONSIDERATION OF AVAILABLE INFORMA-
TION.—In developing and prioritizing the list under
paragraph (1), the Secretary shall consider, for each
drug on the list—
‘‘(A) the availability of information con-
cerning the safe and effective use of the drug
in the pediatric population;
‘‘(B) whether additional information is
needed;
‘‘(C) whether new pediatric studies con-
cerning 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 re-
search organizations, federally funded programs such as
pediatric pharmacology research units, other public or pri-
vate 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 AP-
PROVED APPLICATIONS FOR DRUGS LACKING EXCLU-
SIVITY.—The Commissioner of Food and Drugs, in
consultation with the Director of the National Insti-
tutes 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 ap-
proved 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 equiva-
lent 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, includ-
ing with respect to information provided on the pedi-
atric studies to be conducted pursuant to the re-
quest.
‘‘(2) REQUESTS FOR CONTRACT PROPOSALS.—
If the Commissioner of Food and Drugs does not re-
ceive 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, act-
ing through the Director of the National Institutes
of Health and in consultation with the Commissioner
of Food and Drugs, shall publish a request for con-
tract proposals to conduct the pediatric studies de-
scribed in the written request.
‘‘(3) DISQUALIFICATION.—A holder that re-
ceives 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 Com-
missioner of Food and Drugs shall promulgate guid-
ance to establish the process for the submission of
responses to written requests under paragraph (1).
‘‘(5) CONTRACTS.—A contract under this sec-
tion may be awarded only if a proposal for the con-
tract is submitted to the Secretary in such form and
manner, and containing such agreements, assur-
ances, 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 Com-
missioner 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 (sub-
ject 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 writ-
ten 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 ap-
propriate action in response to the reports sub-
mitted under subparagraph (A) in accordance
with paragraph (7).
‘‘(7) REQUESTS FOR LABELING CHANGE.—Dur-
ing the 180-day period after the date on which a re-
port is submitted under paragraph (6)(A), the Com-
missioner of Food and Drugs shall—
‘‘(A) review the report and such other data
as are available concerning the safe and effec-
tive use in the pediatric population of the drug
studied;
‘‘(B) negotiate with the holders of ap-
proved applications for the drug studied for any
labeling changes that the Commissioner of Food
and Drugs determines to be appropriate and re-
quests 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 re-
quested 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 appli-
cation for the drug involved does not agree to
any labeling change requested by the Commis-
sioner of Food and Drugs under that para-
graph, the Commissioner of Food and Drugs
shall refer the request to the Pediatric Advisory
Subcommittee of the Anti-Infective Drugs Advi-
sory 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 sub-
paragraph (A), the Pediatric Advisory Sub-
committee 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 ap-
propriate labeling changes, if any.
‘‘(9) FDA DETERMINATION.—Not later than 30
days after receiving a recommendation from the Pe-
diatric 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 recommenda-
tion and, if appropriate, make a request to the hold-
ers 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 re-
quested 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 proc-
ess or an enforcement action referred to in the pre-
ceding 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 re-
garding 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 AP-
PLICATIONS FOR DRUGS THAT HAVE MAR-
KET EXCLUSIVITY.
Section 505A(d) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355a(d)) is amended by adding at
the end the following:
‘‘(4) WRITTEN REQUEST TO HOLDERS OF AP-
PROVED
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 applica-
tion approved under section 505(b)(1), the
holder, not later than 180 days after receiving
the written request, shall respond to the Sec-
retary 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 relat-
ing to the use of the drug in the pediatric
population (including neonates, as appro-
priate), the Secretary shall refer the drug
to the Foundation for the National Insti-
tutes of Health established under section
499 of the Public Health Service Act (42
U.S.C. 290b) (referred to in this para-
graph 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 re-
ferral under clause (i).
‘‘(C) LACK OF FUNDS.—On referral of a
drug under subparagraph (B)(i), the Founda-
tion shall issue a proposal to award a grant to
conduct the requested studies unless the Foun-
dation certifies to the Secretary, within a time-
frame that the Secretary determines is appro-
priate through guidance, that the Foundation
does not have funds available under section
499(j)(9)(B)(i) to conduct the requested stud-
ies. If the Foundation so certifies, the Secretary
shall refer the drug for inclusion on the list es-
tablished under section 409I of the Public
Health Service Act for the conduct of the stud-
ies.
‘‘(D) EFFECT OF SUBSECTION.—Nothing
in this subsection (including with respect to re-
ferrals 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
SUB-
SECTION (b).—For drugs under subsection (b)
for which written requests have not been ac-
cepted, 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 Sec-
retary shall issue a written request under sub-
section (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 PEDI-
ATRIC 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 sub-
paragraph (F).
(b) LABELING CHANGES.—
(1) DEFINITION OF PRIORITY SUPPLEMENT.—
Section 201 of the Federal Food Drug, and Cos-
metic Act (21 U.S.C. 321) is amended by adding at
the end the following:
‘‘(kk) PRIORITY SUPPLEMENT.—The term ‘pri-
ority supplement’ means a drug application referred
to in section 101(4) of the Food and Drug Adminis-
tration Modernization Act of 1997 (111 Stat.
2298).’’.
(2) TREATMENT AS PRIORITY SUPPLEMENTS.—
Section 505A of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355a) is amended by adding
at the end the following:
‘‘(l) LABELING SUPPLEMENTS.—
‘‘(1) PRIORITY
STATUS
FOR
PEDIATRIC
SUP-
PLEMENTS.—Any supplement to an application
under section 505 proposing a labeling change pur-
suant 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 pri-
ority 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 ap-
propriate 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 Com-
missioner shall refer the matter to the Pe-
diatric 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 sub-
paragraph (A)(ii), the Pediatric Advisory Sub-
committee of the Anti-Infective Drugs Advisory
Committee shall—
‘‘(i) review the pediatric study reports;
and
‘‘(ii) make a recommendation to the
Commissioner concerning appropriate la-
beling changes, if any.
‘‘(C) CONSIDERATION
OF
RECOMMENDA-
TIONS.—The Commissioner shall consider the
recommendations of the Pediatric Advisory
Subcommittee of the Anti-Infective Drugs Advi-
sory Committee and, if appropriate, not later
than 30 days after receiving the recommenda-
tion, make a request to the sponsor of the ap-
plication 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 re-
quest 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.—Noth-
ing 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 pre-
ceding 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 De-
partment of Health and Human Services who exercise re-
sponsibilities relating to pediatric therapeutics and shall
include—
(1) 1 or more additional individuals with exper-
tise concerning ethical issues presented by the con-
duct of clinical research in the pediatric population;
and
(2) 1 or more additional individuals with exper-
tise 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 Cos-
metic Act (21 U.S.C. 355a(g)) is amended by inserting
‘‘(including neonates in appropriate cases)’’ after ‘‘pedi-
atric age groups’’.
SEC. 8. SUNSET.
Section 505A of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355a) is amended by striking sub-
section (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 Sec-
retary makes a written request for pediatric studies
of the drug;
‘‘(2) on or before October 1, 2007, an applica-
tion 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 Cos-
metic 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
INFORMA-
TION.—
‘‘(1) IN GENERAL.—Not later than 180 days
after the date of submission of a report on a pedi-
atric study under this section, the Commissioner
shall make available to the public a summary of the
medical and clinical pharmacology reviews of pedi-
atric 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 Cos-
metic 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 EX-
CLUSIVITY 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 ex-
clusivity 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 ap-
plication 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 exclu-
sivity 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 LA-
BELING.—
‘‘(1) GENERAL RULE.—A drug for which an ap-
plication has been submitted or approved under sec-
tion 505(j) shall not be considered ineligible for ap-
proval under that section or misbranded under sec-
tion 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 mar-
keting exclusivity for a manufacturer—
‘‘(i) the drug is not labeled for pedi-
atric use; or
‘‘(ii) in the case of a drug for which
there is an additional pediatric use not re-
ferred to in paragraph (1), the drug is not
labeled for the pediatric use under para-
graph (1); and
‘‘(B) a statement of any appropriate pedi-
atric 
contraindications, 
warnings, 
or 
pre-
cautions that the Secretary considers necessary.
‘‘(3) PRESERVATION
OF
PEDIATRIC
EXCLU-
SIVITY
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 ap-
proval of any application under section 505(j)
that omits any other conditions of approval en-
titled to exclusivity under clause (iii) or (iv) of
section 505(j)(5)(D); or
‘‘(D) except as expressly provided in para-
graphs (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 sec-
tion 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 CHIL-
DREN.
(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 re-
search involving children;
(B) federally prepared or supported re-
ports relating to research involving children;
and
(C) federally supported evidence-based re-
search 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 par-
ticipants and the parents, guardians, and the indi-
viduals who may serve as the legally authorized rep-
resentatives of such children (as defined in subpart
A of part 46 of title 45, Code of Federal Regula-
tions).
(2) The expectations and comprehension of
child research participants and the parents, guard-
ians, 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 re-
spect to a healthy child or a child with an illness.
(4) The appropriateness of the regulations ap-
plicable 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 com-
pliance (including the role of institutional review
boards), and the enforcement actions taken for viola-
tions of such regulations.
(7) The unique roles and responsibilities of in-
stitutional review boards in reviewing research in-
volving children, including composition of member-
ship 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 re-
search)’’ 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 pedi-
atric 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 can-
didates to be provided by the National Academy
of Science. Such appointed members shall
include—
‘‘(i) representatives of the general bio-
medical field;
‘‘(ii) representatives of experts in pe-
diatric medicine and research;
‘‘(iii) representatives of the general
biobehavioral field, which may include ex-
perts 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 sub-
paragraph (A);
(4) in subsection (k)(9)—
(A) by striking ‘‘The Foundation’’ and in-
serting the following:
‘‘(A) IN GENERAL.—The Foundation’’; and
(B) by adding at the end the following:
‘‘(B) GIFTS, GRANTS, AND OTHER DONA-
TIONS.—
‘‘(i) IN GENERAL.—Gifts, grants, and
other donations to the Foundation may be
designated for pediatric research and stud-
ies 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 Foun-
dation and not described in clause (i) may
also be used to support such pediatric re-
search and studies.
‘‘(iii) REPORT.—The recipient of a
grant for research and studies shall agree
to provide the Director of the National In-
stitutes of Health and the Commissioner of
Food and Drugs, at the conclusion of the
research and studies—
‘‘(I) a report describing the re-
sults of the research and studies; and
‘‘(II) all data generated in con-
nection 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 para-
graphs (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 Commis-
sioner determines to be appropriate and re-
quests the holders to make.
‘‘(C) APPLICABILITY.—Subparagraph (A)
does not apply to the program described in sub-
section (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 COM-
MITTEE.
(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 (re-
ferred to in this section as the ‘‘advisory committee’’).
(b) PURPOSE.—
(1) IN
GENERAL.—The advisory committee
shall advise and make recommendations to the Sec-
retary, 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 re-
ferred 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 Cos-
metic Act;
(B) identification of research priorities re-
lated to pediatric pharmacology and the need
for additional treatments of specific pediatric
diseases or conditions; and
(C) the ethics, design, and analysis of clin-
ical trials related to pediatric pharmacology.
(c) COMPOSITION.—The advisory committee shall in-
clude representatives of pediatric health organizations, pe-
diatric researchers, relevant patient and patient-family or-
ganizations, 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 (re-
ferred 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 alter-
natives 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 ap-
point not more than 11 voting members to the
Pediatric Subcommittee from the membership
of the Pediatric Pharmacology Advisory Com-
mittee and the Oncologic Drugs Advisory Com-
mittee.
(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 spe-
cialists from the National Cancer Institute.
(ii) At least 4 pediatric oncology spe-
cialists 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 
Neuro-
blastoma 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 PROM-
ISING 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 PROM-
ISING PEDIATRIC CANCER THERAPIES.—
‘‘(1) EXPANSION AND COORDINATION OF AC-
TIVITIES.—The Director of the National Cancer In-
stitute 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
INSTI-
TUTES.—The Director of the Institute shall coordi-
nate the activities under paragraph (1) with similar
activities conducted by other national research insti-
tutes and agencies of the National Institutes of
Health to the extent that those Institutes and agen-
cies have responsibilities that are related to pediatric
cancer.’’.
(c) CLARIFICATION OF AVAILABILITY OF INVESTIGA-
TIONAL 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 inves-
tigation of a new drug of a statement of intent
regarding whether the manufacturer or sponsor
has plans for assessing pediatric safety and effi-
cacy.’’.
(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 in-
serting ‘‘trial sites,’’; and
(B) by striking ‘‘in the trial,’’ and insert-
ing ‘‘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 Gen-
eral of the United States, in consultation with the Sec-
retary 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 la-
beling 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 legis-
lation, and possible reasons for the lack of test-
ing; 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 proc-
ess established pursuant to the amendments
made by this Act, together with a description of
the outcomes of such process, including a de-
scription of the disputes and the recommenda-
tions 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 Gov-
ernment 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 insur-
ers 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 pri-
vate insurers, and to consumers resulting from
decreased health care costs, including—
(i) 
decreased 
hospitalizations 
and
fewer medical errors, due to more appro-
priate and more effective use of medica-
tions in children as a result of testing and
re-labeling because of the amendments
made by this Act;
(ii) direct and indirect benefits associ-
ated 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 bet-
ter 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 mem-
bers.
(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 stud-
ies.
(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 deter-
mines to be appropriate, including a detailed ration-
ale 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 addi-
tional 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 in-
crease 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 stud-
ies 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 promul-
gate 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 num-
ber maintained by the Secretary for the purpose of receiv-
ing reports of adverse events regarding drugs and a state-
ment that such number is to be used for reporting pur-
poses only, not to receive medical advice. With respect to
the final rule:
(1) The rule shall provide for the implementa-
tion 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 promul-
gated.
(b) DRUGS
WITH
PEDIATRIC
MARKET
EXCLU-
SIVITY.—
(1) IN GENERAL.—During the one-year begin-
ning 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 ad-
verse event regarding the drug that the Secretary of
Health and Human Services receives shall be re-
ferred to the Office of Pediatric Therapeutics estab-
lished 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 recommenda-
tions of such Subcommittee regarding whether the
Secretary should take action under the Federal
Food, Drug, and Cosmetic Act in response to the re-
port.
(2) RULE OF CONSTRUCTION.—Paragraph (1)
may not be construed as restricting the authority of
the Secretary of Health and Human Services to con-
tinue carrying out the activities described in such
paragraph regarding a drug after the one-year pe-
riod described in such paragraph regarding the drug
has expired.
SEC. 18. MINORITY CHILDREN AND PEDIATRIC-EXCLU-
SIVITY 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 pur-
pose 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 ex-
tent ethnic and racial minorities are not ade-
quately represented, the reasons for such under
representation and recommendations to increase
such representation.
(B) Whether the Food and Drug Adminis-
tration 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 dis-
eases 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 para-
graph (1) and submit to the Congress a report de-
scribing the findings of the study.
SEC. 19. TECHNICAL AND CONFORMING AMENDMENTS.
Section 505A of the Federal Food, Drug, and Cos-
metic 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 ap-
pears and inserting ‘‘(j)(5)(D)’’; and
(C) by striking ‘‘505(j)(4)(D)’’ each place it ap-
pears 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) respec-
tively;
(3) by moving the subsections so as to appear
in alphabetical order;
(4) in paragraphs (1), (2), and (3) of sub-
section (d), subsection (e), and subsection (m) (as
redesignated by paragraph (2)), by striking ‘‘sub-
section (a) or (c)’’ and inserting ‘‘subsection (b) or
(c)’’; and
(5) in subsection (g) (as redesignated by para-
graph (2)), by striking ‘‘subsection (a) or (b)’’ and
inserting ‘‘subsection (b) or (c)’’.
Æ