146 
diatric population (including neonates, as appro-
priate), the Secretary shall— 
‘‘(A) for a drug for which listed patents 
have not expired, make a determination regard-
ing whether an assessment shall be required to 
be submitted under section 505B; or 
‘‘(B) for a drug that has no listed patents 
or has 1 or more listed patents that have ex-
pired, determine whether there are funds avail-
able under section 736 to award a grant to con-
duct the requested studies pursuant to para-
graph (2). 
‘‘(2) FUNDING OF STUDIES.—If, pursuant to 
paragraph (1), the Secretary determines that there 
are funds available under section 736 to award a 
grant to conduct the requested pediatric studies, 
then the Secretary shall issue a proposal to award 
a grant to conduct the requested studies. If the Sec-
retary determines that funds are not available under 
section 736, the Secretary shall refer the drug for 
inclusion on the list established under section 409I 
of the Public Health Service Act or the conduct of 
studies. 
‘‘(3) PUBLIC NOTICE.—The Secretary shall give 
the public notice of—
147 
‘‘(A) a decision under paragraph (1)(A) 
not to require an assessment under section 
505B and the basis for such decision; 
‘‘(B) the name of any drug, its manufac-
turer, and the indications to be studied pursu-
ant to a grant made under paragraph (2); and 
‘‘(C) any decision under paragraph (2) to 
include a drug on the list established under sec-
tion 409I of the Public Health Service Act. 
‘‘(4) 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. 
‘‘(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
148 
by exclusivity under clause (iii) or (iv) of section 
505(j)(5)(F). 
‘‘(2) LABELING.—Notwithstanding clauses (iii) 
and (iv) of section 505(j)(5)(F), 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;
149 
‘‘(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)(F); or 
‘‘(D) except as expressly provided in para-
graphs (1) and (2), the operation of section 
505. 
‘‘(p) INSTITUTE OF MEDICINE STUDY.—Not later 
than 3 years after the date of the enactment of the Best 
Pharmaceuticals for Children Act of 2007, the Secretary 
shall enter into a contract with the Institute of Medicine 
to conduct a study and report to Congress regarding the 
written requests made and the studies conducted pursuant 
to this section. The Institute of Medicine may devise an 
appropriate mechanism to review a representative sample 
of requests made and studies conducted pursuant to this 
section in order to conduct such study. Such study shall— 
‘‘(1) review such representative written requests 
issued by the Secretary since 1997 under sub-
sections (b) and (c); 
‘‘(2) review and assess such representative pedi-
atric studies conducted under subsections (b) and (c)
150 
since 1997 and labeling changes made as a result of 
such studies; 
‘‘(3) review the use of extrapolation for pedi-
atric subpopulations, the use of alternative endpoints 
for pediatric populations, neonatal assessment tools, 
and ethical issues in pediatric clinical trials; and 
‘‘(4) make recommendations regarding appro-
priate incentives for encouraging pediatric studies of 
biologics. 
‘‘(q) SUNSET.—A drug may not receive any 6-month 
period under subsection (b) or (c) unless— 
‘‘(1) on or before October 1, 2012, the Sec-
retary makes a written request for pediatric studies 
of the drug; 
‘‘(2) on or before October 1, 2012, an applica-
tion for the drug is accepted for filing under section 
505(b); and 
‘‘(3) all requirements of this section are met.’’. 
(2) EFFECTIVE DATE.—The amendment made 
by this subsection shall apply to written requests 
under section 505A of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355a) made after the date 
of the enactment of this Act.
151 
(b) PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.— 
Section 409I of the Public Health Service Act (42 U.S.C. 
284m) is amended to read as follows: 
‘‘SEC. 409I. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS. 
‘‘(a) LIST
OF
PRIORITY
ISSUES
IN
PEDIATRIC 
THERAPEUTICS.— 
‘‘(1) IN GENERAL.—Not later than one year 
after the date of the enactment of the Best Pharma-
ceuticals for Children Act of 2007, 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 and publish a priority 
list of needs in pediatric therapeutics, including 
drugs or indications that require study. The list 
shall be revised every three years. 
‘‘(2) CONSIDERATION OF AVAILABLE INFORMA-
TION.—In developing and prioritizing the list under 
paragraph (1), the Secretary shall consider— 
‘‘(A) therapeutic gaps in pediatrics that 
may 
include 
developmental 
pharmacology, 
pharmacogenetic determinants of drug re-
sponse, metabolism of drugs and biologics in 
children, and pediatric clinical trials;
152 
‘‘(B) particular pediatric diseases, dis-
orders or conditions where more complete 
knowledge and testing of therapeutics, including 
drugs and biologics, may be beneficial in pedi-
atric populations; and 
‘‘(C) the adequacy of necessary infrastruc-
ture to conduct pediatric pharmacological re-
search, including research networks and trained 
pediatric investigators. 
‘‘(b) PEDIATRIC STUDIES
AND RESEARCH.—The 
Secretary, acting through the National Institutes of 
Health, shall award funds to entities that have the exper-
tise to conduct pediatric clinical trials or other research 
(including qualified universities, hospitals, laboratories, 
contract research organizations, practice groups, federally 
funded programs such as pediatric pharmacology research 
units, other public or private institutions, or individuals) 
to enable the entities to conduct the drug studies or other 
research on the issues described in subsection (a). The 
Secretary may use contracts, grants, or other appropriate 
funding mechanisms to award funds under this subsection. 
‘‘(c) PROCESS
FOR PROPOSED PEDIATRIC STUDY 
REQUESTS AND LABELING CHANGES.— 
‘‘(1) SUBMISSION
OF
PROPOSED
PEDIATRIC 
STUDY REQUEST.—The Director of the National In-
153 
stitutes of Health shall, as appropriate, submit pro-
posed pediatric study requests for consideration by 
the Commissioner of Food and Drugs for pediatric 
studies of a specific pediatric indication identified 
under subsection (a). Such a proposed pediatric 
study request shall be made in a manner equivalent 
to a written request made under subsection (b) or 
(c) of section 505A of the Federal Food, Drug, and 
Cosmetic Act, including with respect to the informa-
tion provided on the pediatric studies to be con-
ducted pursuant to the request. The Director of the 
National Institutes of Health may submit a pro-
posed pediatric study request for a drug for which— 
‘‘(A)(i) there is an approved application 
under section 505(j) of the Federal Food, 
Drug, and Cosmetic Act; or 
‘‘(ii) there is a submitted application that 
could be approved under the criteria of such 
section; and 
‘‘(B) there is no patent protection or mar-
ket exclusivity protection for at least one form 
of the drug under the Federal Food, Drug, and 
Cosmetic Act; and
154 
‘‘(C) additional studies are needed to as-
sess the safety and effectiveness of the use of 
the drug in the pediatric population. 
‘‘(2) 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 based 
on the proposed pediatric study request for the indi-
cation or indications submitted pursuant to para-
graph (1) (which shall include a timeframe for nego-
tiations for an agreement) for pediatric studies con-
cerning a drug identified under 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 (b) or (c) 
of section 505A of such Act, including with respect 
to information provided on the pediatric studies to 
be conducted pursuant to the request and using ap-
propriate formulations for each age group for which 
the study is requested. 
‘‘(3) REQUESTS FOR PROPOSALS.—If the Com-
missioner of Food and Drugs does not receive a re-
155 
sponse to a written request issued under paragraph 
(2) not later than 30 days after the date on which 
a request was issued, the Secretary, acting through 
the Director of the National Institutes of Health and 
in consultation with the Commissioner of Food and 
Drugs, shall publish a request for proposals to con-
duct the pediatric studies described in the written 
request in accordance with subsection (b). 
‘‘(4) DISQUALIFICATION.—A holder that re-
ceives a first right of refusal shall not be entitled to 
respond to a request for proposals under paragraph 
(3). 
‘‘(5) CONTRACTS, GRANTS, OR OTHER FUNDING 
MECHANISMS.—A contract, grant, or other funding 
may be awarded under this section only if a proposal 
is submitted to the Secretary in such form and man-
ner, and containing such agreements, assurances, 
and information as the Secretary determines to be 
necessary to carry out this section. 
‘‘(6) REPORTING OF STUDIES.— 
‘‘(A) IN
GENERAL.—On completion of a 
pediatric study in accordance with an award 
under this section, a report concerning the 
study shall be submitted to the Director of the 
National Institutes of Health and the Commis-
156 
sioner of Food and Drugs. The report shall in-
clude all data generated in connection with the 
study, including a written request if issued. 
‘‘(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) of the Federal Food, 
Drug, and Cosmetic Act) and shall be assigned 
a docket number by the Commissioner of Food 
and Drugs. An interested person may submit 
written comments concerning such pediatric 
studies to the Commissioner of Food and 
Drugs, and the written comments shall become 
part of the docket file with respect to each of 
the drugs. 
‘‘(C) ACTION
BY
COMMISSIONER.—The 
Commissioner of Food and Drugs shall take 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—
157 
‘‘(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 and 
through a posting on the website of the Food 
and Drug Administration a summary of the re-
port and a copy of any requested labeling 
changes. 
‘‘(8) DISPUTE RESOLUTION.— 
‘‘(A) REFERRAL TO PEDIATRIC ADVISORY 
COMMITTEE.—If, not later than the end of the 
180-day period specified in paragraph (7), the 
holder of an approved application for the drug 
involved does not agree to any labeling change 
requested by the Commissioner of Food and 
Drugs under that paragraph, the Commissioner
158 
of Food and Drugs shall refer the request to 
the Pediatric Advisory Committee. 
‘‘(B) ACTION BY THE PEDIATRIC ADVISORY 
COMMITTEE.—Not later than 90 days after re-
ceiving a referral under subparagraph (A), the 
Pediatric 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 
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
159 
under paragraph (9), does not agree to make a re-
quested labeling change, the Commissioner of Food 
and Drugs may deem the drug to be misbranded 
under the Federal Food, Drug, and Cosmetic Act. 
‘‘(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 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. 
‘‘(d) DISSEMINATION
OF
PEDIATRIC
INFORMA-
TION.—Not later than one year after the date of the enact-
ment of the Best Pharmaceuticals for Children Act of 
2007, the Secretary, acting through the Director of the 
National Institutes of Health, shall study the feasibility 
of establishing a compilation of information on pediatric 
drug use and report the findings to Congress. 
‘‘(e) AUTHORIZATION OF APPROPRIATIONS.— 
‘‘(1) IN GENERAL.—There are authorized to be 
appropriated to carry out this section— 
‘‘(A) $200,000,000 for fiscal year 2008; 
and
160 
‘‘(B) such sums as are necessary for each 
of the four succeeding fiscal years. 
‘‘(2) AVAILABILITY.—Any amount appropriated 
under paragraph (1) shall remain available to carry 
out this section until expended.’’. 
(c) FEES RELATING TO DRUGS.—Section 735(6) of 
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
379(6)) is amended by adding at the end the following 
new subparagraph: 
‘‘(G) Activities relating to the support of 
studies of drugs on pediatric populations under 
section 505A(n)(1).’’. 
(d) FOUNDATION FOR THE NATIONAL INSTITUTES 
OF HEALTH.—Section 499(c)(1)(C) of the Public Health 
Service Act (42 U.S.C. 290b(c)(1)(C)) is amended by 
striking ‘‘and studies listed by the Secretary pursuant to 
section 409I(a)(1)(A) of this Act and referred under sec-
tion 505A(d)(4)(C) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355(a)(d)(4)(C))’’. 
(e) 
CONTINUATION
OF
OPERATION
OF
COM-
MITTEE.—Section 14 of the Best Pharmaceuticals for 
Children Act (42 U.S.C. 284m note) is amended by adding 
at the end the following new subsection: 
‘‘(d) CONTINUATION
OF
OPERATION
OF
COM-
MITTEE.—Notwithstanding section 14 of the Federal Ad-
161 
visory Committee Act, the advisory committee shall con-
tinue to operate during the five-year period beginning on 
the date of the enactment of the Best Pharmaceuticals for 
Children Act of 2007.’’. 
(f) PEDIATRIC SUBCOMMITTEE OF THE ONCOLOGIC 
DRUGS ADVISORY COMMITTEE.—Section 15 of the Best 
Pharmaceuticals for Children Act (42 U.S.C. 284m note) 
is amended— 
(1) in subsection (a)— 
(A) in paragraph (1)— 
(i) in subparagraph (B), by striking 
‘‘and’’ after the semicolon; 
(ii) in subparagraph (C), by striking 
the period at the end and inserting ‘‘; 
and’’; and 
(iii) by adding at the end the fol-
lowing new subparagraph: 
‘‘(D) provide recommendations to the in-
ternal review committee created under section 
505A(f) of the Federal Food, Drug, and Cos-
metic Act regarding the implementation of 
amendments to sections 505A and 505B of the 
Federal Food, Drug, and Cosmetic Act with re-
spect to the treatment of pediatric cancers.’’; 
and