I
107TH CONGRESS
1ST SESSION
H. R. 3047
To amend the Federal Food, Drug, and Cosmetic Act and the Public Health
Service Act with respect to pediatric studies of drugs, and for other purposes.
IN THE HOUSE OF REPRESENTATIVES
OCTOBER 4, 2001
Mr. WAXMAN (for himself, Mr. BROWN of Ohio, Mr. DINGELL, Mr. DEUTSCH,
Mr. PALLONE, Mr. GREEN of Texas, Mr. STUPAK, and Mr. BARRETT of
Wisconsin) introduced the following bill; which was referred to the Com-
mittee on Energy and Commerce
A BILL
To amend the Federal Food, Drug, and Cosmetic Act and
the Public Health Service Act with respect to pediatric
studies of drugs, and for other purposes.
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 DRUGS.
(a) IN GENERAL.—Section 505A of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is
amended to read as follows:
‘‘SEC. 505A. PEDIATRIC STUDIES OF DRUGS; COST-PLUS
PAYMENTS.
‘‘(a) REQUIRED STUDIES.—
‘‘(1) IN GENERAL.—In the case of a drug for
which a market application is pending or for which
such an application has been approved, or for which
a research exemption has been granted, if (pursuant
to regulations promulgated by the Secretary) the
Secretary requires the sponsor or holder for the drug
(as the case may be) to conduct a pediatric study of
the drug, the Secretary may enter into a contract
for payments in accordance with subsection (d) re-
garding such study.
‘‘(2) MARKET APPLICATION; RESEARCH EXEMP-
TION.—For purposes of this section, the term ‘mar-
ket application’ means an application under section
505 or under section 351 of the Public Health Serv-
ice Act, and the term ‘research exemption’ means an
exemption under section 505(i).
‘‘(3) DISPUTE
RESOLUTION.—The Secretary
shall develop and publish in the Federal Register cri-
teria describing the manner in which the process of
dispute resolution under section 562 will be applied
with respect to paragraph (1). Such criteria shall be
so published not later than 180 days after the date
of the enactment of the Best Pharmaceuticals for
Children Act. Any process of dispute resolution re-
garding a requirement described in subsection (a)
that was commenced before the publication of the
criteria, including a process commenced before the
date of the enactment of such Act, may continue in
accordance with the applicable terms in effect before
the publication of the criteria.
‘‘(b) REQUESTED STUDIES.—
‘‘(1) IN GENERAL.—In the case of a drug that
is included on the list under paragraph (2), the Sec-
retary may award contracts for payments in accord-
ance with subsection (d) for the conduct of pediatric
studies of the drug.
‘‘(2) LIST OF DRUGS.—The Secretary shall es-
tablish and maintain a list of drugs—
‘‘(A) for which an approved market appli-
cation is in effect;
‘‘(B) for which there is no patent protec-
tion, and for which no period of market exclu-
sivity is in effect under section 505 or section
527; and
‘‘(C) for which the Secretary has deter-
mined that pediatric studies are needed to as-
sess the safety and effectiveness of the use of
the drug in a pediatric population.
The initial list under this paragraph shall be estab-
lished not later than one year after the date of the
enactment of the Best Pharmaceuticals for Children
Act. The reference in subparagraph (B) to a period
of market exclusivity in effect under section 505 in-
cludes such a period in effect pursuant to this sec-
tion as this section was in effect on the day before
such date of enactment.
‘‘(3) 
PREFERENCE
IN
AWARDING
CON-
TRACTS.—In awarding a contract under paragraph
(1) regarding a drug, the Secretary shall give pref-
erence to one or more of the holders for the drug (in
the event that Secretary has elected not to use the
authority under subsection (a) regarding the drug),
to the extent that one or more holders submit appli-
cations for the contract.
‘‘(4) APPLICATION FOR CONTRACT.—A contract
may be made under subsection (a) only if an appli-
cation for the contract is submitted to the Secretary
and the application is in such form, is made in such
manner, and contains such agreements, assurances,
and information as the Secretary determines to be
necessary to carry out this subsection.
‘‘(5) COMPETITIVE PROCESS.—Awards of con-
tracts under paragraph (1) shall be made through a
competitive process, and applications for such
awards shall undergo technical and scientific peer
review. The Secretary may provide for the assistance
of the Director of the National Institutes of Health
in the administration of the requirements of the pre-
ceding sentence.
‘‘(c) CERTAIN STUDY PROCEDURES.—In providing
for pediatric studies under subsection (a) or (b), the Sec-
retary shall in writing (through publication in the Federal
Register or otherwise) specify the criteria of the Secretary
for the conduct of the studies, including with respect to
protocols and including timeframes for completion of the
studies and the submission to the Secretary of reports on
the studies.
‘‘(d) PAYMENTS.—A contract under subsection (a) or
(b)—
‘‘(1) shall provide for reimbursement by the
Secretary of the costs of conducting the pediatric
studies involved, upon the Secretary determining
that the applicable conditions under subsection (c)
are being or have been met; and
‘‘(2) shall provide for an amount additional to
such reimbursement (payable once the reimburse-
ment in full has been made), which additional
amount shall be equal to 100 percent of the costs of
conducting such pediatric studies.
‘‘(e) LABELING CHANGES FOR ALREADY-MARKETED
DRUGS.—
‘‘(1) 
SUPPLEMENTAL
APPLICATION; 
PRI-
ORITY.—Not later than 180 days after receiving a
report on pediatric studies under this section, the
Secretary shall determine whether a holder for the
drug involved should alter the labeling for the drug.
If the Secretary determines that a labeling change
should be made, the Secretary shall—
‘‘(A) promptly request the holder in writ-
ing to submit to the Secretary a supplemental
application for purposes of making the labeling
change;
‘‘(B) consider such application to be a pri-
ority application, except that the Secretary may
give greater priority to such applications re-
garding fast track products under section 506
(relating to the treatment of serious or life-
threatening conditions) as the Secretary deter-
mines to be appropriate;
‘‘(C) apply to the application the perform-
ance goals established by the Secretary for pri-
ority drugs; and
‘‘(D) upon submission of the application,
promptly offer to enter into negotiations with
the holder regarding the labeling change, and
promptly arrange for the negotiations to take
place.
‘‘(2) DISPUTE RESOLUTION.—
‘‘(A) IN GENERAL.—With respect to nego-
tiations under paragraph (1), if the Secretary
and the holder involved have not reached agree-
ment on a labeling change as of the expiration
of the 45-day period beginning on the date on
which the negotiations commenced, the Sec-
retary, upon the request of the holder, shall
commence the process of dispute resolution
under section 562 regarding the labeling
change. In providing for the review under such
section of the matter by an advisory panel, the
Secretary shall ensure that the matter is re-
viewed by the Pediatric Advisory Subcommittee
of the Anti-Infective Drugs Advisory Com-
mittee.
‘‘(B) REPORT FROM ADVISORY PANEL.—
Not later than 90 days after receiving a referral
under subparagraph (A), the subcommittee re-
ferred to in such subparagraph shall—
‘‘(i) review the available information
on the safety and effectiveness of the use
of the drug in the pediatric population, in-
cluding reports submitted under this sec-
tion; and
‘‘(ii) make a recommendation to the
Secretary regarding a labeling change.
‘‘(3) DETERMINATION
BY
SECRETARY.—Not
later than 30 days after receiving a recommendation
under paragraph (2)(B)(ii) with respect to a drug,
the Secretary shall make a determination regarding
a labeling change for the drug and inform the holder
involved in writing of the determination. Upon the
expiration of the 30-day period beginning on the
date on which the Secretary so informs the holder,
the Secretary may with respect to the labeling of the
drug take such actions in accordance with this Act
as the Secretary determines to be appropriate.
‘‘(f) DEFINITIONS.—For purposes of this section:
‘‘(1) The term ‘holder for a drug’ means the
holder of an approved market application.
‘‘(2) The term ‘market application’ has the
meaning given such term in subsection (a)(2).
‘‘(3) The term ‘pediatric studies’ means at least
one clinical investigation (that, at the Secretary’s
discretion, may include pharmacokinetic studies) in
pediatric age groups in which a drug is anticipated
to be used.
‘‘(4) The term ‘priority application’ means a
drug application referred to in section 101(4) of the
Food and Drug Administration Modernization Act of
1997 (111 Stat. 2298).
‘‘(5) The term ‘research exemption’ has the
meaning given such term in subsection (a)(2).
‘‘(6) The term ‘sponsor for a drug’ means the
sponsor of an application for the drug under section
505(b)(1) or under section 351 of the Public Health
Service Act, or the sponsor of a clinical investigation
of the drug under an exemption under section
505(i), as the case may be.
‘‘(g) FUNDING.—
‘‘(1) AUTHORIZATION
OF
APPROPRIATIONS.—
For the purpose of contracts under subsection (a)
and (b), there are authorized to be appropriated
$200,000,000 for fiscal year 2002, and such sums
as may be necessary for each of the fiscal years
2003 through 2007.
‘‘(2) LIMITATION.—The authority of the Sec-
retary to enter into contracts under subsection (a)
or (b) is subject to the extent of amounts provided
in advance in an appropriations Act.’’.
(b) FEES FOR SUPPLEMENTAL APPLICATIONS.—Sec-
tion 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).
SEC. 3. SAVINGS PROVISION; INTERACTION OF VARIOUS
MARKET-EXCLUSIVITY PROVISIONS.
(a) SAVINGS PROVISION.—The amendment made by
section 2(a) does not apply with respect to pediatric stud-
ies of drugs that, before the date of the enactment of this
Act, were requested by the Secretary of Health and
Human Services under section 505A of the Federal Food,
Drug, and Cosmetic Act, as in effect on the day before
such date of enactment, or were required by the Secretary
within the meaning of subsection (i) of such section as
so in effect. Such section 505A as so in effect continues
to apply to the pediatric studies described in the preceding
sentence.
(b) MARKET-EXCLUSIVITY INTERACTIONS.—
(1) IN GENERAL.—Paragraph (2) applies in the
case of a period of market exclusivity that is in ef-
fect pursuant to subsection (a). References in para-
graph (2) to section 505A of the Federal Food,
Drug, and Cosmetic Act are references to such sec-
tion as in effect on the day before the date of the
enactment of this Act.
(2) OVERLAP
OF
PROVISIONS.—If a 180-day
period under section 505(j)(5)(B)(iv) of the Federal
Food, Drug, and Cosmetic Act overlaps with a 6-
month extension under section 505A of such Act, so
that the applicant for approval of a drug under sec-
tion 505(j) of such Act entitled to the 180-day pe-
riod 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 ex-
tension, by 6 months.
(3) EFFECT OF SUBSECTION.—Under no cir-
cumstances shall application of this subsection result
in an applicant for approval of a drug under section
505(j) of the Federal Food, Drug, and Cosmetic Act
being enabled to commercially market the drug to
the exclusion of a subsequent applicant for approval
of a drug under section 505(j) of such Act for more
than 180 days.
SEC. 4. 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 Commis-
sioner of Food and Drugs, shall establish a nonprofit cor-
poration to be known as the Foundation for Pediatric Re-
search (hereafter in this section referred to as the ‘Foun-
dation’). The Foundation shall not be an agency or instru-
mentality of the United States Government.
‘‘(b) PURPOSE OF FOUNDATION.—The purpose of
the Foundation shall be to support the conduct of research
on drugs listed by the Secretary pursuant to section
505A(b)(2) of the Federal Food, Drug, and Cosmetic Act.
‘‘(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 Exec-
utive Director.
‘‘(3) AUTHORITY OF FOUNDATION.—The Foun-
dation shall be the sole entity responsible for car-
rying 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 sec-
tion as the ‘Board’), which shall be composed of
ex officio and appointed members in accordance
with this subsection. All appointed members of
the Board shall be 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;