IB
Union Calendar No. 167
107TH CONGRESS
1ST SESSION
H. R. 2887
[Report No. 107–277]
To amend the Federal Food, Drug, and Cosmetic Act to improve the safety
and efficacy of pharmaceuticals for children.
IN THE HOUSE OF REPRESENTATIVES
SEPTEMBER 13, 2001
Mr. GREENWOOD (for himself, Ms. ESHOO, Mr. UPTON, Mr. WYNN, Mr.
BUYER, Mr. RUSH, Mr. BRADY of Pennsylvania, Ms. ROYBAL-ALLARD,
and Ms. LOFGREN) introduced the following bill; which was referred to
the Committee on Energy and Commerce
NOVEMBER 9, 2001
Additional sponsors: Mrs. ROUKEMA, Mr. SMITH of New Jersey, Mr. RANGEL,
Mr. FRANK, Mr. FATTAH, Ms. WOOLSEY, Mr. WHITFIELD, Mr. OWENS,
Mrs. MORELLA, Mr. DOOLEY of California, Mr. MCGOVERN, Mr. LAN-
TOS, Mr. CAPUANO, and Mr. KIND
NOVEMBER 9, 2001
Reported with an amendment, committed to the Committee of the Whole
House on the State of the Union, and ordered to be printed
[Strike out all after the enacting clause and insert the part printed in italic]
[For text of introduced bill, see copy of bill as introduced on September 13, 2001]
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.
(a) IN GENERAL.—Section 505A of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355a) is amended—
(1) by striking subsection (b); and
(2) by redesignating subsections (c) through
through (k) as subsections (b) through (j), respectively.
(b) CONFORMING AMENDMENTS.—Section 505A of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a)
is amended in subsection (b) (as redesignated by subsection
(a)(2) of this section)—
(1) by inserting after ‘‘the Secretary’’ the fol-
lowing: ‘‘determines that information relating to the
use of an approved drug in the pediatric population
may produce health benefits in that population and’’;
and
(2) by striking ‘‘concerning a drug identified in
the list described in subsection (b)’’.
SEC. 3. RESEARCH FUND FOR THE STUDY OF DRUGS LACK-
ING EXCLUSIVITY.
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 (re-
lating to clinical research) as section 409G;
(2) by redesignating the second section 409D (re-
lating to enhancement awards) as section 409H; and
(3) by adding at the end the following:
‘‘SEC. 409I. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS
LACKING EXCLUSIVITY.
‘‘(a) LIST
OF DRUGS LACKING EXCLUSIVITY
FOR
WHICH PEDIATRIC STUDIES ARE NEEDED.—
‘‘(1) IN GENERAL.—Not later than 1 year after
the date of enactment of this section, the Secretary,
acting through the Director of the National Institutes
of Health and in consultation with the Commissioner
of Food and Drugs and experts in pediatric research,
shall develop, prioritize, and publish an annual list
of approved drugs for which—
‘‘(A)(i) there is an approved application
under section 505(j) of the Federal Food, Drug,
and Cosmetic Act;
‘‘(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;
‘‘(iii) there is no patent protection or mar-
ket exclusivity protection under the Federal
Food, Drug, and Cosmetic Act; or
‘‘(iv) there is, under section 505A(c)(4)(C)
of the Federal Food, Drug, and Cosmetic Act, a
referral for inclusion on such list; and
‘‘(B) additional 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 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 Sec-
retary shall award contracts to entities that have the exper-
tise to conduct pediatric clinical trials (including qualified
universities, hospitals, laboratories, contract research orga-
nizations, federally funded programs such as pediatric
pharmacology research units, other public or private insti-
tutions, or individuals) to enable the entities to conduct pe-
diatric 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.—
‘‘(A) IN GENERAL.—The Commissioner of
Food and Drugs, in consultation with the Direc-
tor of 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) 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 in-
formation provided on the pediatric studies to be
conducted pursuant to the request.
‘‘(B) PUBLICATION
OF
REQUEST.—If the
Commissioner of Food and Drugs does not re-
ceive a response to a written request issued
under subparagraph (A) within 30 days of the
date on which a request was issued, the Sec-
retary, acting through the Director of 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.
‘‘(C) DISQUALIFICATION.—A holder that re-
ceives a first right of refusal shall not be entitled
to respond to a request for contract proposals
under subparagraph (B).
‘‘(D) GUIDANCE.—Not later than 270 days
after the date of enactment of this section, the
Commissioner of Food and Drugs shall promul-
gate guidance to establish the process for the sub-
mission of responses to written requests under
subparagraph (A).
‘‘(2) 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 man-
ner, and containing such agreements, assurances, and
information as the Secretary determines to be nec-
essary to carry out this section.
‘‘(3) REPORTING OF STUDIES.—
‘‘(A) Upon 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 National Insti-
tutes of Health and the Commissioner of Food
and Drugs. The report shall include all data gen-
erated in connection with the study.
‘‘(B) AVAILABILITY OF REPORTS.—Each re-
port submitted under subparagraph (A) shall be
considered to be in the public domain, and shall
be assigned a docket number by the Commis-
sioner 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 be-
come part of the docket file with respect to each
of the drugs.
‘‘(C) ACTION BY COMMISSIONER.—The Com-
missioner of Food and Drugs shall take appro-
priate action in response to the reports submitted
under subparagraph (A) in accordance with
paragraph (4).
‘‘(4) REQUEST FOR LABELING CHANGES.—Dur-
ing the 180-day period after the date on which a re-
port is submitted under paragraph (3)(A), the Com-
missioner 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 stud-
ied;
‘‘(B) negotiate with the holders of approved
applications for the drug studied for any label-
ing 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 sum-
mary of the report and a copy of any requested
labeling changes.
‘‘(5) DISPUTE RESOLUTION.—If, not later than
the end of the 180-day period specified in paragraph
(4), 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—
‘‘(A) the Commissioner of Food and Drugs
shall immediately refer the request to the Pedi-
atric Advisory Subcommittee of the Anti-Infec-
tive Drugs Advisory Committee; and
‘‘(B) not later than 90 days after receiving
the referral, the Subcommittee 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.
‘‘(6) FDA DETERMINATION.—Not later than 30
days after receiving a recommendation from the Sub-
committee under paragraph (5)(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 applica-
tions for the drug to make any labeling change that
the Commissioner of Food and Drugs determines to be
appropriate.
‘‘(7) FAILURE TO AGREE.—If a holder of an ap-
proved application for a drug, within 30 days after
receiving a request to make a labeling change under
paragraph (6), does not agree to make a requested la-
beling change, the Commissioner may deem the drug
to be misbranded under the Federal Food, Drug, and
Cosmetic Act.
‘‘(8) 
RECOMMENDATION
FOR
FORMULATION
CHANGES.—If a pediatric study completed under pub-
lic 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) 
CONFIDENTIAL
COMMERCIAL
INFORMATION;
TRADE SECRETS.—Nothing in this section requires or au-
thorizes the use or disclosure of confidential commercial in-
formation or trade secrets.
‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—
‘‘(1) IN GENERAL.—For the purpose of carrying
out this section, there are authorized to be appro-
priated $200,000,000 for fiscal year 2002, and such
sums as may be necessary for each of the fiscal years
2003 through 2007.
‘‘(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 MARKET
EXCLUSIVITY.
Section 505A of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355a) is amended in subsection (c) (as redes-
ignated by section 2(a)(2) of this Act) by adding at the end
the following:
‘‘(4) WRITTEN REQUEST TO HOLDERS OF AP-
PROVED APPLICATIONS FOR DRUGS THAT HAVE MAR-
KET EXCLUSIVITY.—
‘‘(A) REQUEST AND RESPONSE.—If the Sec-
retary makes a written request for pediatric
studies under subsection (b) to the holder of an
application approved under section 505(b)(1),
the holder, not later than 180 days after receiv-
ing 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 relating to
the use of the drug in the pediatric popu-
lation (including neonates as appropriate),
the Secretary shall refer the drug to the
Foundation for Pediatric Research estab-
lished under section 499A of the Public
Health Service Act (referred to in this para-
graph as the ‘Foundation’) for consideration
for the conduct of the pediatric studies de-
scribed in the written request.
‘‘(ii) PUBLIC NOTICE.—The Secretary
shall give public notice of a referral under
clause (i), including notice of the name of
the drug, the name of the manufacturer,
and the indication to be studied.
‘‘(C) LACK OF FUNDS.—If, on referral of a
drug under subparagraph (B)(i), the Foundation
certifies to the Secretary that the Foundation
does not have funds available to conduct the re-
quested studies, 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) CONFIDENTIAL COMMERCIAL INFORMA-
TION; TRADE SECRETS.—Nothing in this para-
graph requires or authorizes the use or disclosure
of confidential commercial information or trade
secrets.
‘‘(E) NO REQUIREMENT TO REFER.—Noth-
ing in this subsection shall be construed to re-
quire that every declined written request shall be
referred to the Foundation.’’.
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 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 Moderniza-
tion 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), as amended by section
2(a)(2) of this Act, is amended by adding at the end
the following:
‘‘(k) LABELING SUPPLEMENTS.—
‘‘(1) PRIORITY STATUS FOR PEDIATRIC SUPPLE-
MENTS.—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 pri-
ority drugs.
‘‘(2) DISPUTE
RESOLUTION.—If the Commis-
sioner determines that an application with respect to
which a pediatric study is conducted under this sec-
tion 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 label-
ing for the drug that is the subject of the
application—
‘‘(A) 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 re-
quested by the Commissioner by that date,
the Commissioner shall immediately refer
the matter to the Pediatric Advisory Sub-
committee of the Anti-Infective Drugs Advi-
sory Committee;
‘‘(B) not later than 90 days after receiving
the 
referral, 
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 label-
ing changes, if any;
‘‘(C) the Commissioner shall consider the
recommendations of the Pediatric Advisory Sub-
committee 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 de-
termines to be appropriate; and
‘‘(D) if the sponsor of the application, with-
in 30 days after receiving a request under sub-
paragraph (C), does not agree to make a labeling
change requested by the Commissioner, the Com-
missioner may deem the drug that is the subject
of the application to be misbranded.’’.
SEC. 6. OFFICE OF PEDIATRIC THERAPEUTICS.
(a) ESTABLISHMENT.—The Secretary of Health and
Human Services shall establish an Office of Pediatric
Therapeutics within the Office of the Commissioner of Food
and Drugs.
(b) DUTIES.—The Office of Pediatric Therapeutics
shall be responsible for oversight and coordination of all ac-
tivities 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 Thera-
peutics shall include—
(1) employees of the Department of Health and
Human Services who, as of the date of enactment of
this Act, exercise responsibilities relating to pediatric
therapeutics;
(2) 1 or more additional individuals with exper-
tise concerning ethical issues presented by the conduct
of clinical research in the pediatric population; and
(3) 1 or more additional individuals with exper-
tise in pediatrics who shall consult and collaborate
with all components of the Food and Drug Adminis-
tration concerning activities described in subsection
(b).
SEC. 7. NEONATES.
Section 505A of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355a) is amended in subsection (f) (as redes-
ignated by section 2(a)(2) of this Act) by inserting ‘‘(includ-
ing neonates in appropriate cases)’’ after ‘‘pediatric age
groups’’.
SEC. 8. SUNSET.
Section 505A of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355a) is amended by striking subsection (i)
(as redesignated by section 2(a)(2) of this Act) and insert-
ing the following:
‘‘(i) SUNSET.—A drug may not receive any 6-month
period under subsection (a) or (b) 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 approvable
application for the drug is submitted under section
505(b)(1); and
‘‘(3) all requirements of this section are met.’’.
SEC. 9. DISSEMINATION OF PEDIATRIC INFORMATION.
Section 505A of the Federal Food, Drug, and Cosmetic
Act, as amended by section 5(b)(2) of this Act, is amended
by adding at the end the following:
‘‘(l) 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 publica-
tion in the Federal Register.
‘‘(2) EFFECT OF SUBSECTION.—Nothing in this
subsection alters or amends in any way section 552
of title 5 or section 1905 of title 18, United States
Code.’’.
SEC. 10. CLARIFICATION OF INTERACTION OF MARKET EX-
CLUSIVITY UNDER SECTION 505A OF THE
FEDERAL FOOD, DRUG, AND COSMETIC ACT
AND MARKET 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, as amended by section 9 of this Act, is amended by
adding at the end the following:
‘‘(m) CLARIFICATION OF INTERACTION OF MARKET
EXCLUSIVITY UNDER THIS SECTION AND MARKET EXCLU-
SIVITY AWARDED TO AN APPLICANT FOR APPROVAL OF A
DRUG UNDER SECTION 505(j).—
‘‘(1) IN GENERAL.—If a 180-day period under
section 505(j)(5)(B)(iv) overlaps with a 6-month ex-
tension 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—
‘‘(A) if the 180-day period would, but for
this subsection, expire after the 6-month exten-
sion, by the number of days of the overlap; or
‘‘(B) if the 180-day period would, but for
this subsection, expire during the 6-month exten-
sion, by 6 months.
‘‘(2) EFFECT OF SUBSECTION.—Under no cir-
cumstances shall application of this section result in
an applicant for approval of a drug under section
505(j) being enabled to commercially market the drug
to the exclusion of a subsequent applicant for ap-
proval of a drug under section 505(j) for more than
180 days.’’.
SEC. 11. PROMPT APPROVAL OF GENERIC DRUGS WHEN PE-
DIATRIC INFORMATION ADDED TO LABELING.
(a) IN GENERAL.—Section 505A of the Federal Food,
Drug, and Cosmetic Act, as amended by section 10 of this
Act, is amended by adding at the end the following sub-
section:
‘‘(n) PROMPT APPROVAL OF GENERIC DRUGS WHEN
PEDIATRIC INFORMATION ADDED TO LABELING.—
‘‘(1) IN GENERAL.—A drug for which an appli-
cation has been submitted or approved under section
505(j) and which otherwise meets all other applicable
requirements under that section shall be considered el-
igible for approval and shall not be considered mis-
branded under section 502 even when its labeling
omits a pediatric indication or other aspect of label-
ing pertaining to pediatric use that is protected by
patent or by market exclusivity pursuant to clause
(iii) or (iv) of section 505(j)(5)(D).
‘‘(2) LABELING
OF
GENERIC
DRUG.—Notwith-
standing the provisions of clause (iii) or (iv) of sec-
tion 505(j)(5)(D), the Secretary may require that the
labeling of a drug approved under section 505(j) that
omits pediatric labeling pursuant to paragraph (1)
include—
‘‘(A) a statement that the drug is not la-
beled for the protected pediatric use; and
‘‘(B) any warnings against unsafe pediatric
use that the Secretary considers necessary.
‘‘(3) RULE
OF
CONSTRUCTION.—Paragraphs 1
and 2 of this subsection do 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; or
‘‘(C) except as expressly provided in para-
graph (1) and (2), the operation of section 505.’’.
(b) EFFECTIVE DATE.—The amendments made by sub-
section (a) take effect on the date of the enactment of this
Act, including with respect to applications under section
505(j) of the Federal Food, Drug, and Cosmetic Act that
are approved or pending on that date.
SEC. 12. 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 number
maintained by the Secretary for the purpose of receiving
reports of adverse events regarding drugs. 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 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 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 sec-
tion 6 of this Act. In considering the report, the Di-
rector 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 
Sub-
committee regarding whether the Secretary should
take action under the Federal Food, Drug, and Cos-
metic 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 con-
tinue carrying out the activities described in such
paragraph regarding a drug after the one-year period
described in such paragraph regarding the drug has
expired.
SEC. 13. FOUNDATION FOR PEDIATRIC RESEARCH.
Title IV of the Public Health Service Act (42 U.S.C.
281 et seq.) is amended by adding at the end the following
part:
‘‘PART J—FOUNDATION FOR PEDIATRIC
RESEARCH
‘‘SEC. 499A. ESTABLISHMENT AND DUTIES OF FOUNDATION.
‘‘(a) IN GENERAL.—The Secretary, acting through the
Director of NIH and in consultation with the Commissioner
of Food and Drugs, shall establish a nonprofit corporation
to be known as the Foundation for Pediatric Research (here-
after in this section referred to as the ‘Foundation’). The
Foundation shall not be an agency or instrumentality of
the United States Government.
‘‘(b) PURPOSE OF FOUNDATION.—The purpose of the
Foundation shall be to collect funds and award grants for
research on drugs listed by the Secretary pursuant to sec-
tion 409I(a)(1)(A).
‘‘(c) CERTAIN ACTIVITIES OF FOUNDATION.—
‘‘(1) IN GENERAL.—In carrying out subsection
(b), the Foundation may solicit and accept gifts,
grants, and other donations, establish accounts, and
invest and expend funds in support of a program to
encourage donations for the conduct of studies of
drugs referred to in subsection (b).
‘‘(2) FEES.—The Foundation may assess fees for
the provision of professional, administrative and
management services by the Foundation in amounts
determined reasonable and appropriate by the Execu-
tive Director.
‘‘(3) AUTHORITY OF FOUNDATION.—The Founda-
tion shall be the sole entity responsible for carrying
out the activities described in this subsection.
‘‘(d) BOARD OF DIRECTORS.—
‘‘(1) COMPOSITION.—
‘‘(A) The Foundation shall have a Board of
Directors (hereafter referred to in this section as
the ‘Board’), which shall be composed of ex offi-
cio and appointed members in accordance with
this subsection. Appointed members of the Board
shall be the voting members.
‘‘(B) The ex officio members of the Board
shall be—
‘‘(i) the Chairman and ranking minor-
ity member of the Subcommittee on Health
(Committee on Energy and Commerce) or
their designees, in the case of the House of
Representatives;
‘‘(ii) the Chairman and ranking mi-
nority member of the Committee on Health,
Education, Labor and Pensions or their
designees, in the case of the Senate;
‘‘(iii) the Director of NIH; and
‘‘(iv) the Commissioner of Food and
Drugs.
‘‘(C) The ex officio members of the Board
under subparagraph (B) shall appoint to the
Board 11 individuals from among a list of can-
didates to be provided by the National Academy
of Science. Of such appointed members—
‘‘(i) 5 shall be representative of the ex-
perts in pediatric medicine and research
field;
‘‘(ii) 1 shall be a biomedical ethicist;
and
‘‘(iii) 5 shall be representatives of the
general public, which may include rep-
resentatives of affected industries.
‘‘(D)(i) Not later than 30 days after the
date of the enactment of the Best Pharma-
ceuticals for Children Act, the Director of NIH
shall convene a meeting of the ex officio members
of the Board to—
‘‘(I) incorporate the Foundation and
establish the general policies of the Founda-
tion for carrying out the purposes of sub-
section (b), including the establishment of
the bylaws of the Foundation; and
‘‘(II) appoint the members of the
Board in accordance with subparagraph
(C).
‘‘(ii) Upon the appointment of the members
of the Board under clause (i)(II), the terms of
service of the ex officio members of the Board as
members of the Board shall terminate.
‘‘(E) The agreement of not less than three-
fifths of the members of the ex officio members of
the Board shall be required for the appointment
of each member to the initial Board.
‘‘(F) No employee of the National Institutes
of Health shall be appointed as a member of the
Board.
‘‘(2) CHAIR.—
‘‘(A) The ex officio members of the Board
under paragraph (1)(B) shall designate an indi-
vidual to serve as the initial Chair of the Board.
‘‘(B) Upon the termination of the term of
service of the initial Chair of the Board, the ap-
pointed members of the Board shall elect a mem-
ber of the Board to serve as the Chair of the
Board.
‘‘(3) TERMS AND VACANCIES.—
‘‘(A) The term of office of each member of
the Board appointed under paragraph (1)(C)
shall be 5 years, except that the terms of offices
for the initial appointed members of the Board
shall expire as determined by the ex officio mem-
bers and the Chair.
‘‘(B) Any vacancy in the membership of the
Board shall be filled in the manner in which the
original position was made and shall not affect
the power of the remaining members to execute
the duties of the Board.
‘‘(C) If a member of the Board does not
serve the full term applicable under subpara-
graph (A), the individual appointed to fill the
resulting vacancy shall be appointed for the re-
mainder of the term of the predecessor of the in-
dividual.
‘‘(D) A member of the Board may continue
to serve after the expiration of the term of the
member until a successor is appointed.
‘‘(4) COMPENSATION.—Members of the Board
may not receive compensation for service on the
Board. Such members may be reimbursed for travel,
subsistence, and other necessary expenses incurred in
carrying out the duties of the Board, as set forth in
the bylaws issued by the Board.
‘‘(5) MEETINGS AND QUORUM.—A majority of
the members of the Board shall constitute a quorum
for purposes of conducting the business of the Board.
‘‘(6) CERTAIN BYLAWS.—
‘‘(A) In establishing bylaws under this sub-
section, the Board shall ensure that the following
are provided for:
‘‘(i) Policies for the selection of the offi-
cers, employees, and agents of the Founda-
tion.
‘‘(ii) Policies, including ethical stand-
ards, for the acceptance, solicitation, and
disposition of donations and grants to the
Foundation and for the disposition of the
assets of the Foundation. Policies with re-
spect to ethical standards shall ensure that
officers, employees and agents of the Foun-
dation (including members of the Board)
avoid encumbrances that would result in a
conflict of interest, including a financial
conflict of interest or a divided allegiance.
Such policies shall include requirements for
the provision of information concerning any
ownership or controlling interest in entities
related to the activities of the Foundation
by such officers, employees and agents and
their spouses and relatives.
‘‘(iii) Policies for the conduct of the
general operations of the Foundation.
‘‘(B) In establishing bylaws under this sub-
section, the Board shall ensure that such bylaws
(and activities carried out under the bylaws) do
not—
‘‘(i) reflect unfavorably upon the abil-
ity of the Foundation to carry out its re-
sponsibilities or official duties in a fair and
objective manner; or
‘‘(ii) compromise, or appear to com-
promise, the integrity of any governmental
agency or program, or any officer or em-
ployee involved in such program.
‘‘(e) INCORPORATION.—The initial members of the
Board shall serve as incorporators and shall take whatever
actions necessary to incorporate the Foundation.
‘‘(f) NONPROFIT STATUS.—The Foundation shall be
considered to be a corporation under section 501(c) of the
Internal Revenue Code of 1986, and shall be subject to the
provisions of such section.
‘‘(g) EXECUTIVE DIRECTOR.—
‘‘(1) IN GENERAL.—The Foundation shall have
an Executive Director who shall be appointed by the
Board and shall serve at the pleasure of the Board.
The Executive Director shall be responsible for the
day-to-day operations of the Foundation and shall
have such specific duties and responsibilities as the
Board shall prescribe.
‘‘(2) COMPENSATION.—The rate of compensation
of the Executive Director shall be fixed by the Board.
‘‘(h) POWERS.—In carrying out subsection (b), the
Foundation shall operate under the direction of its Board,
and may—
‘‘(1) adopt, alter, and use a corporate seal, which
shall be judicially noticed;
‘‘(2) provide for 1 or more officers, employees,
and agents, as may be necessary, define their duties,
and require surety bonds or make other provisions
against losses occasioned by acts of such persons;
‘‘(3) hire, promote, compensate, and discharge of-
ficers and employees of the Foundation, and define
the duties of the officers and employees;
‘‘(4) with the consent of any executive depart-
ment or independent agency, use the information,
services, staff, and facilities of such in carrying out
this section;
‘‘(5) sue and be sued in its corporate name, and
complain and defend in courts of competent jurisdic-
tion;
‘‘(6) modify or consent to the modification of
any contract or agreement to which it is a party or
in which it has an interest under this part;
‘‘(7) establish a process for the selection of can-
didates for positions under subsection (c);
‘‘(8) solicit, accept, hold, administer, invest, and
spend any gift, devise, or bequest of real or personal
property made to the Foundation;
‘‘(9) enter into such other contracts, leases, coop-
erative agreements, and other transactions as the Ex-
ecutive Director considers appropriate to conduct the
activities of the Foundation; and
‘‘(10) exercise other powers as set forth in this
section, and such other incidental powers as are nec-
essary to carry out its powers, duties, and functions
in accordance with this part.
‘‘(i) ADMINISTRATIVE CONTROL.—No participant in
the program established under this part shall exercise any
administrative control over any Federal employee, nor shall
the Foundation attempt to influence an executive branch
agency or employee.
‘‘(j) GENERAL PROVISIONS.—
‘‘(1) FOUNDATION INTEGRITY.—The members of
the Board shall be accountable for the integrity of the
operations of the Foundation and shall ensure such
integrity through the development and enforcement of
criteria and procedures relating to standards of con-
duct (including those developed under subsection
(d)(6)(A)(ii), financial disclosure statements, conflict
of interest rules, recusal and waiver rules, audits and
other matter determined appropriate by the Board.
‘‘(2) FINANCIAL CONFLICTS OF INTEREST.—Any
individual who is an officer, employee, or member of
the Board of the Foundation may not (in accordance
with policies and requirements developed under sub-
section (d)(6)(A)(ii) personally or substantially par-
ticipate in the consideration or determination by the
Foundation of any matter that would directly or pre-
dictably affect any financial interest of the individual
or a relative (as such term is defined in section
109(16) of the Ethics in Government Act of 1978) of
the individual, of any business organization or other
entity, or of which the individual is an officer or em-
ployee, or is negotiating for employment, or in which
the individual has any other financial interest.
‘‘(3) AUDITS; AVAILABILITY OF RECORDS.—The
Foundation shall—
‘‘(A) provide for annual audits of the finan-
cial condition of the Foundation; and
‘‘(B) make such audits, and all other
records, documents, and other papers of the
Foundation, available to the Secretary and the
Comptroller General of the United States for ex-
amination or audit.
‘‘(4) REPORTS.—
‘‘(A) Not later than 5 months following the
end of each fiscal year, the Foundation shall
publish a report describing the activities of the
Foundation during the preceding fiscal year.
Each such report shall include for the fiscal year
involved a comprehensive statement of the oper-
ations, activities, financial condition, and ac-
complishments of the Foundation.
‘‘(B) With respect to the financial condition
of the Foundation, each report under subpara-
graph (A) shall include the source, and a de-
scription of, all gifts or grants to the Foundation
of real or personal property, and the source and
amount of all gifts or grants to the Foundation
of money. Each such report shall include a speci-
fication of any restrictions on the purposes for
which gifts or grants to the Foundation may be
used.
‘‘(C) The Foundation shall make copies of
each report submitted under subparagraph (A)
available for public inspection, and shall upon
request provide a copy of the report to any indi-
vidual for a charge not exceeding the cost of pro-
viding the copy.
‘‘(D) The Board shall annually hold a pub-
lic meeting to summarize the activities of the
Foundation and distribute written reports con-
cerning such activities and the scientific results
derived from such activities.
‘‘(5) SERVICE OF FEDERAL EMPLOYEES.—Fed-
eral employees may serve on committees advisory to
the Foundation and otherwise cooperate with and as-
sist the Foundation in carrying out its function, so
long as the employees do not direct or control Foun-
dation activities.
‘‘(6) RELATIONSHIP WITH EXISTING ENTITIES.—
The Foundation may, pursuant to appropriate agree-
ments, acquire the resources of existing nonprofit pri-
vate corporations with missions similar to the pur-
poses of the Foundation.
‘‘(7) INTELLECTUAL
PROPERTY
RIGHTS.—The
Board may adopt written standards with respect to
the ownership of any intellectual property rights de-
rived from the collaborative efforts of the Foundation
prior to the commencement of such efforts.
‘‘(8) NATIONAL INSTITUTES OF HEALTH AMEND-
MENTS OF 1990.—The activities conducted in support
of the National Institutes of Health Amendments of
1990 (Public Law 101–613), and the amendments
made by such Act, shall not be nullified by the enact-
ment of this section.
‘‘(9) LIMITATION OF ACTIVITIES.—The Founda-
tion shall exist solely as an entity to collect funds and
award grants for research on drugs listed by the Sec-
retary pursuant to section 409I(a)(1)(A).
‘‘(10) TRANSFER
OF
FUNDS.—The Foundation
may transfer funds to the National Institutes of
Health. Any funds transferred under this paragraph
shall be subject to all Federal limitations relating to
federally-funded research.
‘‘(k) DUTIES OF THE DIRECTOR.—
‘‘(1) APPLICABILITY OF CERTAIN STANDARDS TO
NON-FEDERAL EMPLOYEES.—In the case of any indi-
vidual who is not an employee of the Federal Govern-
ment and who serves in association with the National
Institutes of Health, with respect to financial assist-
ance received from the Foundation, the Foundation
may not provide the assistance of, or otherwise permit
the work at the National Institutes of Health to begin
until a memorandum of understanding between the
individual and the Director of NIH, or the designee
of such Director, has been executed specifying that the
individual shall be subject to such ethical and proce-
dural standards of conduct relating to duties per-
formed at the National Institutes of Health, as the
Director of NIH determines is appropriate.
‘‘(2) SUPPORT SERVICES.—The Director of NIH
shall provide facilities, utilities and support services
to the Foundation.
‘‘(l) REPORTS OF STUDIES; LABELING CHANGES.—
‘‘(1) IN GENERAL.—Upon completion of a pedi-
atric study conducted pursuant to this section, a re-
port concerning the study shall be submitted to the
Director of National Institutes of Health and the
Commissioner of Food and Drugs. The report shall
include all data generated in connection with the
study.
‘‘(2) AVAILABILITY
OF
REPORTS; ACTION
BY
FOOD
AND
DRUG
ADMINISTRATION; 
LABELING
CHANGES.—With respect to a report submitted under
paragraph (1), the provisions of paragraphs (3)(B)
through (8) of section 409I(c) apply to such report to
the same extent and in the same manner as such pro-
vision apply to a report submitted under section
409I(c)(3)(A).
‘‘(m) FUNDING.—
‘‘(1) AUTHORIZATION OF APPROPRIATIONS.—For
the purpose of carrying out this part, there are au-
thorized to be appropriated such sums as may be nec-
essary for fiscal year 2002 and each subsequent fiscal
year.
‘‘(2) LIMITATION REGARDING OTHER FUNDS.—
Amounts appropriated under any provision of law
other than paragraph (1) may not be expended to es-
tablish or operate the Foundation.’’.
SEC. 14. 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 research
involving children;
(B) federally-prepared or supported reports
relating to research involving children; and
(C) federally-supported evidence-based re-
search involving children; and
(2) the submission to the appropriate committees
of Congress, by 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 re-
search involving children.
(b) AREAS OF REVIEW.—In conducting the review
under subsection (a)(1), the Institute of Medicine shall con-
sider 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 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 in-
volvement, 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 appli-
cable 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 insti-
tutional review boards in reviewing research involv-
ing 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 con-
junction with experts in pediatric medicine, pediatric re-
search, and the ethical conduct of research involving chil-
dren.
SEC. 15. STUDY ON EFFECTS OF THIS ACT.
Not later than October 1, 2006, the Comptroller Gen-
eral of the United States shall submit to the Congress and
the Secretary of Health and Human Services a report that
describes the following:
(1) The effectiveness of the amendments made by
this Act in ensuring that all drugs used by children
are tested and properly labeled, including—
equivalents of drugs tested and granted exclu-
sivity pursuant to such amendments, and loss of
revenue by the generic drug industry and any
other affected industry as a result of any such
delay; and
(C) benefits to the government, to private
insurers, and to consumers resulting from de-
creased health care costs, including—
(i) decreased hospitalizations, due to
more appropriate and more effective use of
medications in children as a result of test-
ing and re-labeling because of such amend-
ments;
(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 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 mem-
bers.
(3) The nature and types of studies in children
of drugs granted a period of market exclusivity pur-
suant to the amendments made by this Act, including
a description of the complexity of such studies, the
number of study sites necessary to obtain appropriate
data, and the numbers of children involved in any
clinical studies, and the cost of such studies for each
type of study identified.
(4) The increased pediatric research capability,
both private and government-funded, associated with
the amendments made by this Act.
SEC. 16. 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 (c)(2) (as redesig-
nated by section 2(a)(2) of this Act) by inserting after the
first sentence the following: ‘‘In reaching an agreement re-
garding written protocols, the Secretary shall take into ac-
count adequate representation of children of ethnic and ra-
cial 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 extent
ethnic and racial minorities are not adequately
represented, the reasons for such under represen-
tation 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 eth-
nic 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 para-
graph (1) and submit to the Congress a report de-
scribing the findings of the study.
SEC. 17. TECHNICAL AND CONFORMING AMENDMENTS.
Section 505A of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355a) is amended—
(1)(A) by striking ‘‘(j)(4)(D)(ii)’’ each place such
term appears and inserting ‘‘(j)(5)(D)(ii)’’; and
(B) by striking ‘‘(j)(4)(D)’’ each place such term
appears and inserting ‘‘(j)(5)(D)’’; and
(2)(A) in subsection (c) (as redesignated by sec-
tion 2(a)(2) of this Act), in each of paragraphs (1)
through (3), by striking ‘‘subsection (a) or (c)’’ and
inserting ‘‘subsection (a) or (b)’’; and
(B) in subsection (d) (as so redesignated), in the
last sentence, by striking ‘‘subsection (a) or (c)’’ and
inserting ‘‘subsection (a) or (b)’’.
Union Calendar No. 167
107TH CONGRESS
1ST SESSION
H. R. 2887
[Report No. 107–277]
A BILL
To amend the Federal Food, Drug, and Cosmetic
Act to improve the safety and efficacy of phar-
maceuticals for children.
NOVEMBER 9, 2001
Reported with an amendment, committed to the Com-
mittee of the Whole House on the State of the Union,
and ordered to be printed