130 
sions on all pediatric plans and assessments prior to 
approval of an application or supplement for which 
a pediatric assessment is required under this section 
and all deferral and waiver requests granted pursu-
ant to this section. 
‘‘(2) ACTIVITY BY COMMITTEE.—The committee 
referred to in paragraph (1) may operate using ap-
propriate members of such committee and need not 
convene all members of the committee. 
‘‘(3) DOCUMENTATION
OF
COMMITTEE
AC-
TION.—For each drug or biological product, the 
committee referred to in paragraph (1) shall docu-
ment, for each activity described in paragraph (4) or 
(5), which members of the committee participated in 
such activity. 
‘‘(4) REVIEW
OF
PEDIATRIC
PLANS, ASSESS-
MENTS, DEFERRALS, AND
WAIVERS.—Consultation 
on pediatric plans and assessments by the committee 
referred to in paragraph (1) pursuant to this section 
shall occur prior to approval of an application or 
supplement for which a pediatric assessment is re-
quired under this section. The committee shall re-
view all requests for deferrals and waivers from the 
requirement to submit a pediatric assessment grant-
ed under this section and shall provide recommenda-
131 
tions as needed to reviewing divisions, including with 
respect to whether such a supplement, when sub-
mitted, shall be considered for priority review. 
‘‘(5) RETROSPECTIVE
REVIEW
OF
PEDIATRIC 
ASSESSMENTS, 
DEFERRALS, 
AND
WAIVERS.—Not 
later than 1 year after the date of the enactment of 
the Pediatric Research Equity Act of 2007, the com-
mittee referred to in paragraph (1) shall conduct a 
retrospective review and analysis of a representative 
sample of assessments submitted and deferrals and 
waivers approved under this section since the enact-
ment of the Pediatric Research Equity Act of 2003. 
Such review shall include an analysis of the quality 
and consistency of pediatric information in pediatric 
assessments and the appropriateness of waivers and 
deferrals granted. Based on such review, the Sec-
retary shall issue recommendations to the review di-
visions for improvements and initiate guidance to in-
dustry related to the scope of pediatric studies re-
quired under this section. 
‘‘(6) TRACKING OF ASSESSMENTS AND LABEL-
ING CHANGES.—The Secretary, in consultation with 
the committee referred to in paragraph (1), shall 
track and make available to the public in an easily
132 
accessible manner, including through posting on the 
Web site of the Food and Drug Administration— 
‘‘(A) the number of assessments conducted 
under this section; 
‘‘(B) the specific drugs and biological prod-
ucts and their uses assessed under this section; 
‘‘(C) the types of assessments conducted 
under this section, including trial design, the 
number of pediatric patients studied, and the 
number of centers and countries involved; 
‘‘(D) the total number of deferrals re-
quested and granted under this section and, if 
granted, the reasons for such deferrals, the 
timeline for completion, and the number com-
pleted and pending by the specified date, as 
outlined in subsection (a)(3); 
‘‘(E) the number of waivers requested and 
granted under this section and, if granted, the 
reasons for the waivers; 
‘‘(F) the number of pediatric formulations 
developed and the number of pediatric formula-
tions not developed and the reasons any such 
formulation was not developed; 
‘‘(G) the labeling changes made as a result 
of assessments conducted under this section;
133 
‘‘(H) an annual summary of labeling 
changes made as a result of assessments con-
ducted under this section for distribution pursu-
ant to subsection (h)(2); 
‘‘(I) an annual summary of information 
submitted pursuant to subsection (a)(3)(B); 
and 
‘‘(J) the number of times the committee 
referred to in paragraph (1) made a rec-
ommendation to the Secretary under paragraph 
(4) regarding priority review, the number of 
times the Secretary followed or did not follow 
such a recommendation, and, if not followed, 
the reasons why such a recommendation was 
not followed. 
‘‘(g) LABELING CHANGES.— 
‘‘(1) DISPUTE RESOLUTION.— 
‘‘(A) REQUEST
FOR
LABELING
CHANGE 
AND FAILURE TO AGREE.—If, on or after the 
date of the enactment of the Pediatric Research 
Equity Act of 2007, the Commissioner deter-
mines that a sponsor and the Commissioner 
have been unable to reach agreement on appro-
priate changes to the labeling for the drug that 
is the subject of the application or supplement,
134 
not later than 180 days after the date of the 
submission of the application or supplement— 
‘‘(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 does not agree 
within 30 days after the Commissioner’s 
request to make a labeling change re-
quested by the Commissioner, the Commis-
sioner shall refer the matter to the Pedi-
atric Advisory Committee. 
‘‘(B) ACTION BY THE PEDIATRIC ADVISORY 
COMMITTEE.—Not later than 90 days after re-
ceiving a referral under subparagraph (A)(ii), 
the Pediatric 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 
Committee and, if appropriate, not later than
137 
tribute such information to physicians and other 
health care providers. 
‘‘(3) EFFECT OF SUBSECTION.—Nothing in this 
subsection shall alter or amend section 301(j) of this 
Act or section 552 of title 5 or section 1905 of title 
18, United States Code. 
‘‘(i) ADVERSE EVENT REPORTING.— 
‘‘(1) REPORTING IN YEAR ONE.—Beginning on 
the date of the enactment of the Pediatric Research 
Equity Act of 2007, during the one-year period be-
ginning on the date a labeling change is made pur-
suant to subsection (g), the Secretary shall ensure 
that all adverse event reports that have been re-
ceived for such drug (regardless of when such report 
was received) are referred to the Office of Pediatric 
Therapeutics. In considering such reports, the Direc-
tor of such Office shall provide for the review of 
such reports by the Pediatric Advisory Committee, 
including obtaining any recommendations of such 
committee regarding whether the Secretary should 
take action under this Act in response to such re-
ports. 
‘‘(2) REPORTING IN SUBSEQUENT YEARS.—Fol-
lowing the one-year period described in paragraph 
(1), the Secretary shall, as appropriate, refer to the
138 
Office of Pediatric Therapeutics all pediatric adverse 
event reports for a drug for which a pediatric study 
was conducted under this section. In considering 
such reports, the Director of such Office may pro-
vide for the review of such reports by the Pediatric 
Advisory Committee, including obtaining any rec-
ommendation of such Committee regarding whether 
the Secretary should take action in response to such 
reports. 
‘‘(3) EFFECT.—The requirements of this sub-
section shall supplement, not supplant, other review 
of such adverse event reports by the Secretary. 
‘‘(j) SCOPE OF AUTHORITY.—Nothing in this section 
provides to the Secretary any authority to require a pedi-
atric assessment of any drug or biological product, or any 
assessment regarding other populations or uses of a drug 
or biological product, other than the pediatric assessments 
described in this section. 
‘‘(k) ORPHAN DRUGS.—Unless the Secretary re-
quires otherwise by regulation, this section does not apply 
to any drug for an indication for which orphan designation 
has been granted under section 526. 
‘‘(l) INSTITUTE OF MEDICINE STUDY.— 
‘‘(1) IN GENERAL.—Not later than three years 
after the date of the enactment of the Pediatric Re-
139 
search Equity Act of 2007, the Secretary shall con-
tract with the Institute of Medicine to conduct a 
study and report to Congress regarding the pediatric 
studies conducted pursuant to this section or pre-
cursor regulations since 1997 and labeling changes 
made as a result of such studies. 
‘‘(2) CONTENT OF STUDY.—The study under 
paragraph (1) shall review and assess the use of ex-
trapolation for pediatric subpopulations, the use of 
alternative endpoints for pediatric populations, neo-
natal assessment tools, the number and type of pedi-
atric adverse events, and ethical issues in pediatric 
clinical trials. 
‘‘(3) REPRESENTATIVE SAMPLE.—The Institute 
of Medicine may devise an appropriate mechanism to 
review a representative sample of studies conducted 
pursuant to this section from each review division 
within the Center for Drug Evaluation and Research 
in order to make the requested assessment. 
‘‘(m) INTEGRATION WITH OTHER PEDIATRIC STUD-
IES.—The authority under this section shall remain in ef-
fect so long as an application subject to this section may 
be accepted for filing by the Secretary on or before the 
date specified in section 505A(q).’’. 
(b) APPLICABILITY.—
140 
(1) IN GENERAL.—Notwithstanding subsection 
(h) of section 505B of the Federal Food, Drug and 
Cosmetic Act, as in effect on the day before the date 
of the enactment of this Act, a pending assessment, 
including a deferred assessment, required under 
such section 505B shall be deemed to have been re-
quired under section 505B of the Federal Food, 
Drug and Cosmetic Act as in effect on or after the 
date of the enactment of this Act. 
(2) CERTAIN ASSESSMENTS AND WAIVER RE-
QUESTS.—An assessment pending on or after the 
date that is 1 year prior to the date of the enact-
ment of this Act shall be subject to the tracking and 
disclosure requirements established under such sec-
tion 505B, as in effect on or after such date of en-
actment, except that any such assessments sub-
mitted or waivers of such assessments requested be-
fore such date of enactment shall not be subject to 
subsections (a)(4)(C), (b)(2)(C), (f)(6)(F), and (h) 
of such section 505B. 
SEC. 403. ESTABLISHMENT OF INTERNAL COMMITTEE. 
Chapter V of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 351 et seq.) is amended by inserting after 
section 505B the following:
141 
‘‘SEC. 505C. INTERNAL COMMITTEE FOR REVIEW OF PEDI-
ATRIC PLANS, ASSESSMENTS, DEFERRALS, 
AND WAIVERS. 
‘‘The Secretary shall establish an internal committee 
within the Food and Drug Administration to carry out the 
activities as described in sections 505A(f) and 505B(f). 
Such internal committee shall include employees of the 
Food and Drug Administration, with expertise in pediat-
rics (including representation from the Office of Pediatric 
Therapeutics), biopharmacology, statistics, chemistry, 
legal issues, pediatric ethics, and the appropriate expertise 
pertaining to the pediatric product under review, such as 
expertise in child and adolescent psychiatry, and other in-
dividuals designated by the Secretary.’’. 
SEC. 404. GOVERNMENT ACCOUNTABILITY OFFICE RE-
PORT. 
Not later than January 1, 2011, the Comptroller 
General of the United States, in consultation with the Sec-
retary of Health and Human Services, shall submit to the 
Congress a report that addresses the effectiveness of sec-
tions 505A and 505B of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355a, 355c) and section 409I of the 
Public Health Service Act (42 U.S.C. 284m) in ensuring 
that medicines used by children are tested and properly 
labeled. Such report shall include—
142 
(1) the number and importance of drugs and 
biological products for children that are being tested 
as a result of the amendments made by this title and 
title V and the importance for children, health care 
providers, parents, and others of labeling changes 
made as a result of such testing; 
(2) the number and importance of drugs and 
biological products for children that are not being 
tested for their use notwithstanding the provisions of 
this title and title V and possible reasons for the 
lack of testing; 
(3) the number of drugs and biological products 
for which testing is being done and labeling changes 
required, including the date labeling changes are 
made and which labeling changes required the use of 
the dispute resolution process established pursuant 
to the amendments made by this title, together with 
a description of the outcomes of such process, in-
cluding a description of the disputes and the rec-
ommendations of the Pediatric Advisory Committee; 
(4) any recommendations for modifications to 
the programs established under sections 505A and 
505B of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a) and section 409I of the Public 
Health Service Act (42 U.S.C. 284m) that the Sec-
145 
(c)(3)(E)(ii) and (j)(5)(F)(ii) of such section to 
four years, to forty-eight months, and to seven 
and one-half years are deemed to be four and 
one-half years, fifty-four months, and eight 
years, respectively; or 
‘‘(II) the period referred to in clauses (iii) 
and (iv) of subsection (c)(3)(E) of such section, 
and in clauses (iii) and (iv) of subsection 
(j)(5)(F) of such section, is deemed to be three 
years and six months rather than three years; 
and 
‘‘(ii) if the drug is designated under sec-
tion 526 for a rare disease or condition, the pe-
riod referred to in section 527(a) is deemed to 
be seven years and six months rather than 
seven years; and 
‘‘(B)(i) if the drug is the subject of— 
‘‘(I) a listed patent for which a certifi-
cation has been submitted under sub-
section (b)(2)(A)(ii) or (j)(2)(A)(vii)(II) of 
section 505 and for which pediatric studies 
were submitted prior to the expiration of 
the patent (including any patent exten-
sions); or
146 
‘‘(II) a listed patent for which a cer-
tification has been submitted under sub-
sections (b)(2)(A)(iii) or (j)(2)(A)(vii)(III) 
of section 505, 
the period during which an application may not 
be approved under section 505(c)(3) or section 
505(j)(5)(B) shall be extended by a period of 
six months after the date the patent expires (in-
cluding any patent extensions); or 
‘‘(ii) if the drug is the subject of a listed 
patent for which a certification has been sub-
mitted 
under 
subsection 
(b)(2)(A)(iv) 
or 
(j)(2)(A)(vii)(IV) of section 505, and in the pat-
ent infringement litigation resulting from the 
certification the court determines that the pat-
ent is valid and would be infringed, the period 
during which an application may not be ap-
proved under section 505(c)(3) or section 
505(j)(5)(B) shall be extended by a period of 
six months after the date the patent expires (in-
cluding any patent extensions). 
‘‘(2) EXCEPTION.—The Secretary shall not ex-
tend the period referred to in paragraph (1)(A) or 
(1)(B) if the determination made under subsection
147 
(d)(3) is made later than 9 months prior to the expi-
ration of such period. 
‘‘(c) MARKET EXCLUSIVITY
FOR ALREADY-MAR-
KETED DRUGS.— 
‘‘(1) IN GENERAL.—Except as provided in para-
graph (2), if the Secretary determines that informa-
tion relating to the use of an approved drug in the 
pediatric population may produce health benefits in 
that population and makes a written request to the 
holder of an approved application under section 
505(b)(1) for pediatric studies (which shall include 
a timeframe for completing such studies), the holder 
agrees to the request, such studies are completed 
using appropriate formulations for each age group 
for which the study is requested within any such 
timeframe, and the reports thereof are submitted 
and accepted in accordance with subsection (d)(3)— 
‘‘(A)(i)(I) the period referred to in sub-
section (c)(3)(E)(ii) of section 505, and in sub-
section (j)(5)(F)(ii) of such section, is deemed 
to be five years and six months rather than five 
years, and the references in subsections 
(c)(3)(E)(ii) and (j)(5)(F)(ii) of such section to 
four years, to forty-eight months, and to seven 
and one-half years are deemed to be four and
148 
one-half years, fifty-four months, and eight 
years, respectively; or 
‘‘(II) the period referred to in clauses (iii) 
and (iv) of subsection (c)(3)(D) of such section, 
and in clauses (iii) and (iv) of subsection 
(j)(5)(F) of such section, is deemed to be three 
years and six months rather than three years; 
and 
‘‘(ii) if the drug is designated under sec-
tion 526 for a rare disease or condition, the pe-
riod referred to in section 527(a) is deemed to 
be seven years and six months rather than 
seven years; and 
‘‘(B)(i) if the drug is the subject of— 
‘‘(I) a listed patent for which a certifi-
cation has been submitted under sub-
section (b)(2)(A)(ii) or (j)(2)(A)(vii)(II) of 
section 505 and for which pediatric studies 
were submitted prior to the expiration of 
the patent (including any patent exten-
sions); or 
‘‘(II) a listed patent for which a cer-
tification has been submitted under sub-
section (b)(2)(A)(iii) or (j)(2)(A)(vii)(III) 
of section 505,
149 
the period during which an application may not 
be approved under section 505(c)(3) or section 
505(j)(5)(B)(ii) shall be extended by a period of 
six months after the date the patent expires (in-
cluding any patent extensions); or 
‘‘(ii) if the drug is the subject of a listed 
patent for which a certification has been sub-
mitted 
under 
subsection 
(b)(2)(A)(iv) 
or 
(j)(2)(A)(vii)(IV) of section 505, and in the pat-
ent infringement litigation resulting from the 
certification the court determines that the pat-
ent is valid and would be infringed, the period 
during which an application may not be ap-
proved under section 505(c)(3) or section 
505(j)(5)(B) shall be extended by a period of 
six months after the date the patent expires (in-
cluding any patent extensions). 
‘‘(2) EXCEPTION.—The Secretary shall not ex-
tend the period referred to in paragraph (1)(A) or 
(1)(B) if the determination made under subsection 
(d)(3) is made later than 9 months prior to the expi-
ration of such period. 
‘‘(d) CONDUCT OF PEDIATRIC STUDIES.— 
‘‘(1) REQUEST FOR STUDIES.—
150 
‘‘(A) IN
GENERAL.—The Secretary may, 
after consultation with the sponsor of an appli-
cation for an investigational new drug under 
section 505(i), the sponsor of an application for 
a new drug under section 505(b)(1), or the 
holder of an approved application for a drug 
under section 505(b)(1), issue to the sponsor or 
holder a written request for the conduct of pedi-
atric studies for such drug. In issuing such re-
quest, the Secretary shall take into account 
adequate representation of children of ethnic 
and racial minorities. Such request to conduct 
pediatric studies shall be in writing and shall 
include a timeframe for such studies and a re-
quest to the sponsor or holder to propose pedi-
atric labeling resulting from such studies. 
‘‘(B) SINGLE WRITTEN REQUEST.—A sin-
gle written request— 
‘‘(i) may relate to more than one use 
of a drug; and 
‘‘(ii) may include uses that are both 
approved and unapproved. 
‘‘(2) WRITTEN REQUEST FOR PEDIATRIC STUD-
IES.— 
‘‘(A) REQUEST AND RESPONSE.—
151 
‘‘(i) IN
GENERAL.—If the Secretary 
makes a written request for pediatric stud-
ies (including neonates, as appropriate) 
under subsection (b) or (c), the applicant 
or holder, not later than 180 days after re-
ceiving the written request, shall respond 
to the Secretary as to the intention of the 
applicant or holder to act on the request 
by— 
‘‘(I) indicating when the pediatric 
studies will be initiated, if the appli-
cant or holder agrees to the request; 
or 
‘‘(II) indicating that the appli-
cant or holder does not agree to the 
request and stating the reasons for 
declining the request. 
‘‘(ii) DISAGREE WITH REQUEST.—If, 
on or after the date of the enactment of 
the Best Pharmaceuticals for Children Act 
of 2007, the applicant or holder does not 
agree to the request on the grounds that it 
is not possible to develop the appropriate 
pediatric formulation, the applicant or 
holder shall submit to the Secretary the
152 
reasons such pediatric formulation cannot 
be developed. 
‘‘(B) ADVERSE EVENT REPORTS.—An ap-
plicant or holder that, on or after the date of 
the enactment of the Best Pharmaceuticals for 
Children Act of 2007, agrees to the request for 
such studies shall provide the Secretary, at the 
same time as the submission of the reports of 
such studies, with all postmarket adverse event 
reports regarding the drug that is the subject 
of such studies and are available prior to sub-
mission of such reports. 
‘‘(3) MEETING THE STUDIES REQUIREMENT.— 
Not later than 180 days after the submission of the 
reports of the studies, the Secretary shall accept or 
reject such reports and so notify the sponsor or 
holder. The Secretary’s only responsibility in accept-
ing or rejecting the reports shall be to determine, 
within the 180-day period, whether the studies fairly 
respond to the written request, have been conducted 
in accordance with commonly accepted scientific 
principles and protocols, and have been reported in 
accordance with the requirements of the Secretary 
for filing.
153 
‘‘(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. 
‘‘(e) NOTICE OF DETERMINATIONS ON STUDIES RE-
QUIREMENT.— 
‘‘(1) IN GENERAL.—The Secretary shall publish 
a notice of any determination, made on or after the 
date of the enactment of the Best Pharmaceuticals 
for Children Act of 2007, that the requirements of 
subsection (d) have been met and that submissions 
and approvals under subsection (b)(2) or (j) of sec-
tion 505 for a drug will be subject to the provisions 
of this section. Such notice shall be published not 
later than 30 days after the date of the Secretary’s 
determination regarding market exclusivity and shall 
include a copy of the written request made under 
subsection (b) or (c). 
‘‘(2) IDENTIFICATION
OF
CERTAIN
DRUGS.— 
The Secretary shall publish a notice identifying any 
drug for which, on or after the date of the enact-
ment of the Best Pharmaceuticals for Children Act 
of 2007, a pediatric formulation was developed, 
studied, and found to be safe and effective in the pe-
diatric population (or specified subpopulation) if the
154 
pediatric formulation for such drug is not introduced 
onto the market within one year after the date that 
the Secretary publishes the notice described in para-
graph (1). Such notice identifying such drug shall be 
published not later than 30 days after the date of 
the expiration of such one year period. 
‘‘(f) INTERNAL REVIEW
OF WRITTEN REQUESTS 
AND PEDIATRIC STUDIES.— 
‘‘(1) INTERNAL REVIEW.—The Secretary shall 
utilize the internal review committee established 
under section 505C to review all written requests 
issued on or after the date of the enactment of the 
Best Pharmaceuticals for Children Act of 2007, in 
accordance with paragraph (2). 
‘‘(2) REVIEW
OF
WRITTEN
REQUESTS.—The 
committee referred to in paragraph (1) shall review 
all written requests issued pursuant to this section 
prior to being issued. 
‘‘(3) REVIEW
OF
PEDIATRIC
STUDIES.—The 
committee referred to in paragraph (1) may review 
studies conducted pursuant to this section to make 
a recommendation to the Secretary whether to ac-
cept or reject such reports under subsection (d)(3). 
‘‘(4) ACTIVITY BY COMMITTEE.—The committee 
referred to in paragraph (1) may operate using ap-
155 
propriate members of such committee and need not 
convene all members of the committee. 
‘‘(5) DOCUMENTATION
OF
COMMITTEE
AC-
TION.—For each drug, the committee referred to in 
paragraph (1) shall document, for each activity de-
scribed in paragraph (2) or (3), which members of 
the committee participated in such activity. 
‘‘(6) TRACKING PEDIATRIC STUDIES AND LA-
BELING CHANGES.—The Secretary, in consultation 
with the committee referred to in paragraph (1), 
shall track and make available to the public, in an 
easily accessible manner, including through posting 
on the Web site of the Food and Drug Administra-
tion— 
‘‘(A) the number of studies conducted 
under this section and under section 409I of 
the Public Health Service Act; 
‘‘(B) the specific drugs and drug uses, in-
cluding labeled and off-labeled indications, stud-
ied under such sections; 
‘‘(C) the types of studies conducted under 
such sections, including trial design, the num-
ber of pediatric patients studied, and the num-
ber of centers and countries involved;
158 
Commissioner have been unable to reach agree-
ment on appropriate changes to the labeling for 
the drug that is the subject of the application, 
not later than 180 days after the date of sub-
mission 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 within 30 days after the 
Commissioner’s request to make a labeling 
change requested by the Commissioner, the 
Commissioner shall refer the matter 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)(ii), 
the Pediatric Advisory Committee shall— 
‘‘(i) review the pediatric study reports; 
and 
‘‘(ii) make a recommendation to the 
Commissioner concerning appropriate la-
beling changes, if any.
159 
‘‘(C) CONSIDERATION
OF
RECOMMENDA-
TIONS.—The Commissioner shall consider the 
recommendations of the Pediatric Advisory 
Committee and, if appropriate, not later than 
30 days after receiving the recommendation, 
make a request to the sponsor of the applica-
tion to make any labeling change that the Com-
missioner 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 Committee process or an en-
forcement action referred to in the preceding 
sentence) shall preclude, delay, or serve as the 
basis to stay the other course of action.
160 
‘‘(j) OTHER LABELING CHANGES.—If, on or after the 
date of the enactment of the Best Pharmaceuticals for 
Children Act of 2007, the Secretary determines that a pe-
diatric study conducted under this section does or does 
not demonstrate that the drug that is the subject of the 
study is safe and effective, including whether such study 
results are inconclusive, in pediatric populations or sub-
populations, the Secretary shall order the labeling of such 
product to include information about the results of the 
study and a statement of the Secretary’s determination. 
‘‘(k) DISSEMINATION
OF
PEDIATRIC
INFORMA-
TION.— 
‘‘(1) IN GENERAL.—Not later than 210 days 
after the date of submission of a report on a pedi-
atric study under this section, the Secretary shall 
make available to the public the medical, statistical, 
and clinical pharmacology reviews of pediatric stud-
ies conducted under subsection (b) or (c). 
‘‘(2) DISSEMINATION
OF
INFORMATION
RE-
GARDING
LABELING
CHANGES.—Beginning on the 
date of the enactment of the Best Pharmaceuticals 
for Children Act of 2007, the Secretary shall include 
as a requirement of a written request that the spon-
sors of the studies that result in labeling changes 
that are reflected in the annual summary developed
161 
pursuant to subsection (f)(3)(F) distribute, at least 
annually (or more frequently if the Secretary deter-
mines that it would be beneficial to the public 
health), such information to physicians and other 
health care providers. 
‘‘(3) 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. 
‘‘(l) ADVERSE EVENT REPORTING.— 
‘‘(1) REPORTING IN YEAR ONE.—Beginning on 
the date of the enactment of the Best Pharma-
ceuticals for Children Act of 2007, during the one- 
year period beginning on the date a labeling change 
is approved pursuant to subsection (i), the Secretary 
shall ensure that all adverse event reports that have 
been received for such drug (regardless of when such 
report was received) are referred to the Office of Pe-
diatric Therapeutics established under section 6 of 
the Best Pharmaceuticals for Children Act (Public 
Law 107–109). In considering the reports, the Di-
rector of such Office shall provide for the review of 
the reports by the Pediatric Advisory Committee, in-
cluding obtaining any recommendations of such 
Committee regarding whether the Secretary should
162 
take action under this Act in response to such re-
ports. 
‘‘(2) REPORTING IN SUBSEQUENT YEARS.—Fol-
lowing the one-year period described in paragraph 
(1), the Secretary shall, as appropriate, refer to the 
Office of Pediatric Therapeutics all pediatric adverse 
event reports for a drug for which a pediatric study 
was conducted under this section. In considering 
such reports, the Director of such Office may pro-
vide for the review of such reports by the Pediatric 
Advisory Committee, including obtaining any rec-
ommendation of such Committee regarding whether 
the Secretary should take action in response to such 
reports. 
‘‘(3) EFFECT.—The requirements of this sub-
section shall supplement, not supplant, other review 
of such adverse event reports by the Secretary. 
‘‘(m) 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
163 
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 six-month exclu-
sivity period. 
‘‘(n) REFERRAL IF PEDIATRIC STUDIES NOT COM-
PLETED.— 
‘‘(1) IN GENERAL.—Beginning on the date of 
the enactment of the Best Pharmaceuticals for Chil-
dren Act of 2007, if pediatric studies of a drug have 
not been completed under subsection (d) and if the 
Secretary, through the committee established under 
section 505C, determines that there is a continuing 
need for information relating to the use of the drug 
in the pediatric population (including neonates, as 
appropriate), the Secretary shall carry out the fol-
lowing:
164 
‘‘(A) For a drug for which a listed patent 
has not expired, make a determination regard-
ing whether an assessment shall be required to 
be submitted under section 505B(b). Prior to 
making such a determination, the Secretary 
may not take more than 30 days to certify 
whether the Foundation for the National Insti-
tutes of Health has sufficient funding at the 
time of such certification to initiate and fund 
all of the studies in the written request in their 
entirety within the timeframes specified within 
the written request. Only if the Secretary 
makes such certification in the affirmative, the 
Secretary shall refer all pediatric studies in the 
written request to the Foundation for the Na-
tional Institutes of Health for the conduct of 
such studies, and such Foundation shall fund 
such studies. If no certification has been made 
at the end of the 30-day period, or if the Sec-
retary certifies that funds are not sufficient to 
initiate and fund all the studies in their en-
tirety, the Secretary shall consider whether as-
sessments shall be required under section 
505B(b) for such drug.
165 
‘‘(B) For a drug that has no listed patents 
or has 1 or more listed patents that have ex-
pired, the Secretary shall refer the drug for in-
clusion on the list established under section 
409I of the Public Health Service Act for the 
conduct of studies. 
‘‘(2) PUBLIC NOTICE.—The Secretary shall give 
the public notice of a decision under paragraph 
(1)(A) not to require an assessment under section 
505B and the basis for such decision. 
‘‘(3) 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
166 
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;
167 
‘‘(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)
168 
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; 
‘‘(4) review and assess the pediatric studies of 
biological products as required under subsections (a) 
and (b) of section 505B; and 
‘‘(5) 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) APPLICABILITY.— 
(A) IN GENERAL.—The amendment made 
by this subsection shall apply to written re-
quests under section 505A of the Federal Food,
169 
Drug, and Cosmetic Act (21 U.S.C. 355a) 
issued on or after the date of the enactment of 
this Act. 
(B) CERTAIN
WRITTEN
REQUESTS.—A 
written request issued under section 505A of 
the Federal Food, Drug, and Cosmetic Act, as 
in effect on the day before the date of the en-
actment of this Act, which has been accepted 
and for which no determination under sub-
section (d)(2) of such section has been made 
before such date of enactment, shall be subject 
to such section 505A, except that such written 
requests 
shall 
be 
subject 
to 
subsections 
(d)(2)(A)(ii), (e)(1) and (2), (f), (i)(2)(A), (j), 
(k)(1), (l)(1), and (n) of section 505A of the 
Federal Food, Drug, and Cosmetic Act, as in 
effect on or after the date of the enactment of 
this Act. 
(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.—
170 
‘‘(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; 
‘‘(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-
171 
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-
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
172 
(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 
‘‘(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-
173 
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-
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).
174 
‘‘(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-
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
176 
‘‘(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 Web site 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 
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
177 
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 
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
178 
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 
‘‘(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) 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
179 
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)’ ’’ and inserting 
‘‘and studies for which the Secretary issues a certification 
in the affirmative under section 505A(n)(1)(A) of the Fed-
eral Food, Drug, and Cosmetic Act’’. 
(d) 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-
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.’’. 
(e) 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;
180 
(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 
505B(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 
(B) by adding at the end the following new 
paragraph: 
‘‘(3) CONTINUATION OF OPERATION OF SUB-
COMMITTEE.—Notwithstanding section 14 of the 
Federal Advisory Committee Act, the Subcommittee 
shall continue to operate during the five-year period 
beginning on the date of the enactment of the Best 
Pharmaceuticals for Children Act of 2007.’’; and 
(2) in subsection (d), by striking ‘‘2003’’ and 
inserting ‘‘2009’’.