Best Pharmaceuticals for Children Act
PL 107-109 (S.1789), enacted 01/04/2002

115 STAT. 1408 
PUBLIC LAW 107-109-JAN. 4, 2002 
Public Law 107-109 
107th Congress 
An Act 
Jan. 4, 2002 
To amend the Federal Food, Drug, and Cosmetic Act to improve the safety and 
[S. 1789] 
efficacy of pharmaceuticals for children. 
Be it enacted by the Senate and House of Representatives of 
Best 
the United States of America in Congress assembled, 
Z"^Sen"£t. 
SECTION 1. SHORT TITLE. 
21 use 301 note. 
This Act may be cited as the "Best Pharmaceuticals for Children 
Act". 
SEC. 2. PEDIATRIC STUDIES OF ALREADY-MARKETED DRUGS. 
Section 505A of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a) is amended— 
(1) by striking subsection (b); and 
(2) in subsection (c)— 
(A) by inserting after "the Secretary" the following: 
"determines that information relating to the use of an 
approved drug in the pediatric population may produce 
health benefits in that population and"; and 
(B) by striking "concerning a drug identified in the 
list described in subsection (b)". 
SEC. 3. RESEARCH FUND FOR THE STUDY OF DRUGS. 
Part B of title IV of the Public Health Service Act (42 U.S.C. 
284 et seq.) is amended— 
(1) by redesignating the second section 409C, relating to 
clinical research (42 U.S.C. 284k), as section 409G; 
(2) by redesignating the second section 409D, relating to 
enhancement awards (42 U.S.C. 2841), as section 409H; and 
(3) by adding at the end the following: 
42 u s e 284m. 
"SEC. 4091. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS. 
"(a) 
LIST 
OF DRUGS FOR WHICH PEDIATRIC STUDIES ARE 
NEEDED.— 
"(1) IN GENERAL.—Not later than one year after the date 
of enactment of this section, the Secretary, acting through 
the Director of the National Institutes of Health and in con-
sultation 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 (21 
U.S.C. 355CJ)); 
"(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 (21 U.S.C. 3550")); 
PUBLIC LAW 107-109^JAN. 4, 2002 
115 STAT. 1409 
"(iii) there is no patent protection or market exclusivity 
protection under the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 301 et seq.); or 
"(iv) there is a referral for inclusion on the list under 
section 505A(d)(4)(C) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 355a(d)(4)(C)); and 
"(B) in the case of a drug referred to in clause (i), 
(ii), or (iii) of subparagraph (A), additional studies are 
needed to assess the safety and effectiveness of the use 
of the drug in the pediatric population. 
"(2) CONSIDERATION OF AVAILABLE INFORMATION.—In devel-
oping and prioritizing the list under paragraph (1), the Sec-
retary shall consider, for each drug on the list— 
"(A) the availability of information concerning the safe 
and effective use of the drug in the pediatric population; 
"(B) whether additional information is needed; 
"(C) whether new pediatric studies concerning the drug 
may produce health benefits in the pediatric population; 
and 
"(D) whether reformulation of the drug is necessary. 
"(b) CONTRACTS FOR PEDIATRIC STUDIES.—The Secretary shall 
award contracts to entities that have the expertise to conduct pedi-
atric clinical trials (including qualified universities, hospitals, lab-
oratories, contract research organizations, federally funded pro-
grams such as pediatric pharmacology research units, other public 
or private institutions, or individuals) to enable the entities to 
conduct pediatric studies concerning one or more drugs identified 
in the list described in subsection (a). 
"(c) PROCESS FOR CONTRACTS AND LABELING CHANGES.— 
"(1) WRITTEN REQUEST TO HOLDERS OF APPROVED APPLICA-
TIONS FOR DRUGS LACKING EXCLUSIVITY.—^The Commissioner 
of Food and Drugs, in consultation with the Director of the 
National Institutes of Health, may issue a written request 
(which shall include a timeframe for negotiations for an agree-
ment) for pediatric studies concerning a drug identified in the 
list described in subsection (a)(1)(A) (except clause (iv)) 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 information provided 
on the pediatric studies to be conducted pursuant to the request. 
"(2) REQUESTS FOR CONTRACT PROPOSALS.—If the Commis-
sioner of Food and Drugs does not receive a response to a 
written request issued under paragraph (1) within 30 days 
of the date on which a request was issued, or if a referral 
described in subsection (a)(l)(A)(iv) is made, 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 contract proposals to conduct the 
pediatric studies described in the written request. 
"(3) DISQUALIFICATION.^A holder that receives a first right 
of refusal shall not be entitled to respond to a request for 
contract proposals under paragraph (2). 
"(4) GUIDANCE.—Not later than 270 days after the date 
of enactment of this section, the Commissioner of Food and 
Deadline. 
DeadMne. 
115 STAT. 1410 
PUBLIC LAW 107-109-^AN. 4, 2002 
Drugs shall promulgate guidance to establish the process for 
the submission of responses to written requests under para-
graph (1). 
"(5) 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 manner, 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 a contract awarded under this section, 
a report concerning the study shall be submitted to the 
Director of the National Institutes of Health and the 
Commissioner of Food and Drugs. The report shall include 
all data generated in connection with the study. 
"(B) AVAILABILITY OF REPORTS.—Each report submitted 
under subparagraph (A) shall be considered to be in the 
public domain (subject to section 505A(d)(4)(D) of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 355a(d)(4)(D)) 
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 com-
ments 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 appropriate action in response 
to the reports submitted under subparagraph (A) in accord-
ance with paragraph (7). 
"(7) REQUESTS FOR LABELING CHANGE.—During the 
180-
day period after the date on which a report is submitted under 
paragraph (6)(A), the Commissioner of Food and Drugs shall— 
"(A) review the report and such other data as are 
available concerning the safe and effective use in the pedi-
atric population of the drug studied; 
"(B) negotiate with the holders of approved applications 
for the drug studied for any labeling changes that the 
Commissioner of Food and Drugs determines to be appro-
priate 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 summary of 
the report and a copy of any requested labeling changes. 
"(8) DISPUTE RESOLUTION.— 
"(A) REFERRAL TO PEDIATRIC ADVISORY SUBCOMMITTEE 
OF THE ANTI-INFECTIVE DRUGS ADVISORY COMMITTEE.—If, 
not later than the end of the 180-day period specified 
in paragraph (7), the holder of an approved 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 Subcommittee 
of the Anti-Infective Drugs Advisory Committee. 
"(B) 
ACTION 
BY THE 
PEDIATRIC 
ADVISORY 
SUB-
COMMITTEE OF THE ANTI-INFECTIVE DRUGS ADVISORY COM-
Deadline. 
MITTEE.—Not later than 90 days after receiving a referral 
PUBLIC LAW 107-109^JAN. 4, 2002 
115 STAT. 1411 
under subparagraph (A), the Pediatric Advisory Sub-
committee of the Anti-Infective Drugs Advisory Committee 
shall— 
"(i) review the available information on the safe 
and effective use of the drug in the pediatric popu-
lation, including study reports submitted under this 
section; and 
"(ii) make a recommendation to the Commissioner 
of Food and Drugs as to appropriate labeling changes, 
if any. 
"(9) FDA DETERMINATION.—Not later than 30 days after Deadline, 
receiving a recommendation from the Pediatric Advisory Sub-
committee of the Anti-Infective Drugs Advisory Committee 
under paragraph (8)(B)(ii) with respect to a drug, the Commis-
sioner of Food and Drugs shall consider the recommendation 
and, if appropriate, make a request to the holders of approved 
applications for the drug to make any labeling change that 
the Commissioner of Food and Drugs determines to be appro-
priate. 
"(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 requested labeling change, the Commissioner may 
deem the drug to be misbranded under the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 301 et seq.). 
"(11) No EFFECT ON AUTHORITY.—Nothing in this sub-
section limits the authority of the United States to bring an 
enforcement action under the Federal Food, Drug, and Cosmetic 
Act when a drug lacks appropriate pediatric labeling. Neither 
course of action (the Pediatric Advisory Subcommittee of the 
Anti-Infective Drugs Advisory Committee process or an enforce-
ment action referred to in the preceding sentence) shall pre-
clude, delay, or serve as the basis to stay the other course 
of action. 
"(12) RECOMMENDATION FOR FORMULATION CHANGES.—If a 
pediatric study completed under public contract indicates that 
a formulation change is necessary and the Secretary agrees, 
the Secretary shall send a nonbinding letter of recommendation 
regarding that change to each holder of an approved applica-
tion. 
"(d) AUTHORIZATION OF APPROPRIATIONS.— 
"(1) IN GENERAL.—There are authorized to be appropriated 
to carry out this section— 
"(A) $200,000,000 for fiscal year 2002; and 
"(B) such sums as are necessary for each of the five 
succeeding fiscal years. 
"(2) AVAILABILITY.—^Any amount appropriated under para-
graph (1) shall remain available to carry out this section until 
expended.". 
SEC. 4. WRITTEN REQUEST TO HOLDERS OF APPROVED 
APPLICA-
TIONS FOR DRUGS THAT HAVE MARKET EXCLUSIVITY. 
Section 505A(d) of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a(d)) is amended by adding at the end the following: 
"(4) WRITTEN REQUEST TO HOLDERS OF APPROVED APPLICA-
TIONS FOR DRUGS THAT HAVE MARKET EXCLUSIVITY.— 
115 STAT. 1412 
Deadline. 
PUBLIC LAW 107-109-^AN. 4, 2002 
"(A) REQUEST AND RESPONSE.—If the Secretary makes 
a written request for pediatric studies (including neonates, 
as appropriate) under subsection (c) to the holder of an 
application approved under section 505(b)(1), the holder, 
not later than 180 days after receiving the written request, 
shall respond to the Secretary 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 population 
(including neonates, as appropriate), the Secretary 
shall refer the drug to the Foundation for the National 
Institutes of Health established under section 499 of 
the Public Health Service Act (42 U.S.C. 290b) (referred 
to in this paragraph as the 'Foundation') for the con-
duct of the pediatric studies described in the written 
request. 
"(ii) PUBLIC NOTICE.—^The Secretary shall give 
public notice of the name of the drug, the name of 
the manufacturer, and the indications to be studied 
made in a referral under clause (i). 
"(C) LACK OF FUNDS.—On referral of a drug under 
subparagraph (B)(i), the Foundation shall issue a proposal 
to award a grant to conduct the requested studies unless 
the Foundation certifies to the Secretary, within a time-
frame that the Secretary determines is appropriate through 
guidance, that the Foundation does not have funds avail-
able under section 499(j)(9)(B)(i) to conduct the requested 
studies. If the Foundation so certifies, the Secretary shall 
refer the drug for inclusion on the list established under 
section 4091 of the Public Health Service Act for the conduct 
of the studies. 
"(D) EFFECT OF SUBSECTION.—Nothing in this sub-
section (including with respect to referrals from the Sec-
retary to the Foundation) alters or amends section 301(j) 
of this Act or section 552 of title 5 or section 1905 of 
title 18, United States Code. 
"(E) No REQUIREMENT TO REFER.—Nothing in this sub-
section shall be construed to require that every declined 
written request shall be referred to the Foundation. 
"(F) WRITTEN REQUESTS UNDER SUBSECTION (b).—For 
drugs under subsection (b) for which written requests have 
not been accepted, if the Secretary determines that there 
is a continuing need for information relating to the use 
of the drug in the pediatric population (including neonates, 
as appropriate), the Secretary shall issue a written request 
under subsection (c) cifter the date of approval of the drug.". 
PUBLIC LAW 107-109^JAN. 4, 2002 
115 STAT. 1413 
SEC. 5. TIMELY LABELING CHANGES FOR DRUGS GRANTED EXCLU-
SIVITY; DRUG FEES. 
(a) ELIMINATION OF USER FEE WAIVER FOR PEDIATRIC SUPPLE-
MENTS.—Section 736(a)(1) of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 379h(a)(l)) is amended— 
(1) by striking subparagraph (F); and 
(2) by redesignating subparagraph (G) as subparagraph 
(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 supple-
ment' means a drug application referred to in section 101(4) 
of the Food and Drug Administration Modernization Act of 
1997 (111 Stat. 2298).". 
(2) TREATMENT AS PRIORITY SUPPLEMENTS.—Section 505A 
of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) 
is amended by adding at the end the following: 
"(1) LABELING SUPPLEMENTS.— 
"(1) PRIORITY STATUS FOR PEDIATRIC SUPPLEMENTS.—-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 estab-
lished by the Commissioner for priority drugs. 
"(2) DISPUTE RESOLUTION.— 
"(A) REQUEST FOR LABELING CHANGE AND FAILURE TO 
AGREE.—If the Commissioner determines that an applica-
tion with respect to which a pediatric study is conducted 
under this section is approvable and that the only open 
issue for final action on the application is the reaching 
of an agreement between the sponsor of the application 
and the Commissioner on appropriate changes to the 
labeling for the drug that is the subject of the application, 
not later than 180 days after the date of submission of 
the application— 
"(i) the Commissioner shall request that the 
sponsor of the application make any labeling change 
that the Commissioner determines to be appropriate; 
and 
"(ii) if the sponsor of the application does not agree 
to make a labeling change requested by the Commis-
sioner, the Commissioner shall refer the matter to 
the Pediatric Advisory Subcommittee of the Anti-Infec-
tive Drugs Advisory Committee. 
"(B) 
ACTION 
BY 
THE 
PEDIATRIC 
ADVISORY 
SUB-
COMMITTEE OF THE ANTI-INFECTIVE DRUGS ADVISORY COM-
MITTEE.—Not later than 90 days after receiving a referral 
under subparagraph (A)(ii), the Pediatric Advisory Sub-
committee of the Anti-Infective Drugs Advisory Committee 
shall— 
"(i) review the pediatric study reports; and 
"(ii) make a recommendation to the Commissioner 
concerning appropriate labeling changes, if any. 
Deadline. 
Deadline. 
115 STAT. 1414 
PUBLIC LAW 107-109—JAN. 4, 2002 
Deadline. 
"(C) 
CONSIDERATION 
OF 
RECOMMENDATIONS.—The 
Commissioner shall consider the recommendations of the 
Pediatric Advisory Subcommittee 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 determines to be 
appropriate. 
"(D) MISBRANDING.—If the sponsor of the application, 
within 30 days after receiving a request under subpara-
graph (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.—Nothing in this sub-
section limits the authority of the United States to bring 
an enforcement action under this Act when a drug lacks 
appropriate pediatric labeling. Neither course of action (the 
Pediatric Advisory Subcommittee of the Anti-Infective 
Drugs Advisory Committee process or an enforcement 
action referred to in the preceding sentence) shall preclude, 
delay, or serve as the basis to stay the other course of 
action.". 
21 use 393a. 
SEC. 6. OFFICE OF PEDIATRIC THERAPEUTICS. 
(a) ESTABLISHMENT.—The Secretary of Health and Human 
Services shall establish an Office of Pediatric Therapeutics within 
the Food and Drug Administration. 
(b) DUTIES.—The Office of Pediatric Therapeutics shall be 
responsible for coordination and facilitation of all activities of the 
Food and Drug Administration that may have any effect on a 
pediatric population or the practice of pediatrics or may in any 
other way involve pediatric issues. 
(c) STAFF.—The staff of the Office of Pediatric Therapeutics 
shall coordinate with employees of the Department of Health and 
Human Services who exercise responsibilities relating to pediatric 
therapeutics and shall include— 
(1) one or more additional individuals with expertise con-
cerning ethical issues presented by the conduct of clinical 
research in the pediatric population; and 
(2) one or more additional individuals with expertise in 
pediatrics as may be necessary to perform the activities 
described in subsection (b). 
SEC. 7. NEONATES. 
Section 505A(g) of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a(g)) is amended by inserting "(including 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 (j) and inserting 
the following: 
"(j) SUNSET.—^A drug may not receive any 6-month period under 
subsection (a) or (c) unless— 
"(1) on or before October 1, 2007, the Secretary makes 
a written request for pediatric studies of the drug; 
PUBLIC LAW 107-109-^AN. 4, 2002 
115 STAT. 1415 
"(2) on or before October 1, 2007, an application for the 
drug is accepted for filing under section 505(b); and 
"(3) all requirements of this section are met.". 
SEC. 9. DISSEMINATION OF PEDIATRIC INFORMATION. 
Section 505A of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a) (as amended by section 5(b)(2)) is amended by 
adding at the end the following: 
"(m) DISSEMINATION OF PEDIATRIC INFORMATION.— 
"(1) IN GENERAL.—Not later than 180 days after the date DeadHne. 
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 publication 
in the Federal Register. 
"(2) EFFECT OF SUBSECTION.—Nothing in this subsection 
alters or amends section 301(j) of this Act or section 552 of 
title 5 or section 1905 of title 18, United States Code.". 
SEC. 10. CLARIFICATION OF INTERACTION OF PEDIATRIC EXCLU-
SIVITY UNDER SECTION 505A OF THE FEDERAL FOOD, 
DRUG, AND COSMETIC ACT AND 180-DAY EXCLUSIVITY 
AWARDED TO AN APPLICANT FOR APPROVAL OF A DRUG 
UNDER SECTION 505(j) OF THAT ACT. 
Section 505A of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a) (as amended by section 9) is amended by adding 
at the end the following: 
"(n) CLARIFICATION OF INTERACTION OF MARKET EXCLUSIVITY 
UNDER THIS SECTION AND MARKET EXCLUSIVITY 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 exclusivity period under this section, so that the 
applicant for approval of a drug under section 505(j) entitled to 
the 180-day period under that section loses a portion of the 180-
day period to which the applicant is entitled for the drug, the 
180-day period shall be extended from— 
"(1) the date on which the 180-day period would have 
expired by the number of days of the overlap, if the 180-
day period would, but for the application 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 exclusivity period.". 
SEC. 11. PROMPT APPROVAL OF DRUGS UNDER SECTION 505(j) WHEN 
PEDIATRIC INFORMATION IS ADDED TO LABELING. 
(a) IN GENERAL.—Section 505A of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 355a) (as amended by section 10) 
is amended by adding at the end the following: 
"(o) PROMPT APPROVAL OF DRUGS UNDER SECTION 505(j) WHEN 
PEDL\TRIC INFORMATION IS ADDED TO LABELING.— 
"(1) GENERAL RULE.—^A drug for which an application has 
been submitted or approved under section 505(j) shall not be 
considered ineligible for approval under that section or mis-
branded under section 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 
115 STAT. 1416 
PUBLIC LAW 107-109^JAN. 4, 2002 
or other aspect is protected by patent or by exclusivity under 
clause (iii) or (iv) of section 505{j)(5)(D). 
"(2) LABELING.—^Notwithstanding clauses (iii) and (iv) of 
section 505(j)(5)(D), the Secretary may require that the labeling 
of a drug approved under section 505(j) that omits a pediatric 
indication or other aspect of labeling as described in paragraph 
(1) include— 
"(A) a statement that, because of marketing exclusivity 
for a manufacturer— 
"(i) the drug is not labeled for pediatric use; or 
"(ii) in the case of a drug for which there is an 
additional pediatric use not referred to in paragraph 
(1), the drug is not labeled for the pediatric use under 
paragraph (1); and 
"(B) a statement of any appropriate pediatric contra-
indications, warnings, or precautions that the Secretary 
considers necessary. 
"(3) PRESERVATION OF PEDIATRIC EXCLUSIVITY AND OTHER 
PROVISIONS.—^This subsection does not affect— 
"(A) the availability or scope of exclusivity under this 
section; 
"(B) the availability or scope of exclusivity under sec-
tion 505 for pediatric formulations; 
"(C) the question of the eligibility for approval of any 
application under section 505(j) that omits any other condi-
tions of approval entitled to exclusivity under clause (iii) 
or (iv) of section 505(j)(5)(D); or 
"(D) except as expressly provided in paragraphs (1) 
and (2), the operation of section 505.". 
21 use 355a 
(b) EFFECTIVE DATE.—^The amendment made by subsection (a) 
iiote. 
takes effect on the date of enactment of this Act, including with 
respect to applications under section 505(j) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355(j*)) that are approved or 
pending on that date. 
42 u s e 289 note. 
SEC. 12. STUDY CONCERNING RESEARCH INVOLVING CHILDREN. 
(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 chil-
dren; 
(B) federally prepared or supported reports relating 
to research involving children; and 
(C) 
federally 
supported 
evidence-based 
research 
involving children; and 
(2) the submission to the Committee on Health, Education, 
Labor, and Pensions of the Senate and the Committee on 
Energy and Commerce of the House of Representatives, not 
later than two years after the date of enactment of this Act, 
of a report concerning the review conducted under paragraph 
(1) that includes recommendations on best practices relating 
to research involving children. 
(b) AREAS OF REVIEW.—In conducting the review under sub-
section (a)(1), the Institute of Medicine shall consider the following: 
PUBLIC LAW 107-109-^AN. 4, 2002 
115 STAT. 1417 
(1) The written and oral process of obtaining and defining 
"assent", "permission" and "informed consent" with respect to 
child clinical research participants and the parents, guardians, 
and the individuals who may serve as the legally authorized 
representatives 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 involvement, particularly in terms 
of research versus therapeutic treatment. 
(3) The definition of "minimal risk" with respect to a 
healthy child or a child with an illness. 
(4) The appropriateness of the regulations applicable to 
children of differing ages and maturity levels, including regula-
tions relating to legal status. 
(5) Whether payment (financial or otherwise) may be pro-
vided 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 sub-
section (a)(1)(A), the monitoring of such compliance (including 
the role of institutional review boards), and the enforcement 
actions taken for violations of such regulations. 
(7) The unique roles and responsibilities of institutional 
review boards in reviewing research involving 
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 rec-
ommendations under subsection (a)(2) in conjunction with experts 
in pediatric medicine, pediatric research, and the ethical conduct 
of research involving children. 
SEC. 13. FOUNDATION FOR THE NATIONAL INSTITUTES OF HEALTH. 
Section 499 of the Public Health Service Act (42 U.S.C. 290b) 
is amended— 
(1) in subsection (b), by inserting "(including collection 
of funds for pediatric pharmacologic research)" after "mission"; 
(2) in subsection (c)(1)— 
(A) by redesignating subparagraph (C) as subpara-
graph (D); and 
(B) by inserting after subparagraph (B) the following: 
"(C) A program to collect funds for pediatric pharmaco-
logic research and studies listed by the Secretary pursuant 
to section 4091(a)(1)(A) of this Act and referred under sec-
tion 505A(d)(4)(C) of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 355a(d)(4)(C))."; 
(3) in subsection (d)— 
(A) in paragraph (1)— 
(i) in subparagraph (B)— 
(I) in clause (ii), by striking "and" at the end; 
(II) in clause (iii), by striking the period and 
inserting "; and"; and 
(III) by adding at the end the following: 
"(iv) the Commissioner of Food and Drugs."; and 
115 STAT. 1418 
PUBLIC LAW 107-109—JAN. 4, 2002 
(ii) by striking subparagraph (C) and inserting 
the following: 
"(C) The ex officio members of the Board under 
subparagraph (B) shall appoint to the Board individuals 
from among a list of candidates to be provided by the 
National Academy of Science. Such appointed members 
shall include— 
"(i) representatives of the general biomedical field; 
"(ii) representatives of experts in pediatric medi-
cine and research; 
"(iii) representatives of the general biobehavioral 
field, which may include experts in biomedical ethics; 
and 
"(iv) representatives of the general public, which 
may include representatives of affected industries."; 
and 
(B) in paragraph (2), by realigning the margin of 
subparagraph (B) to align with subparagraph (A); 
(4) in subsection (k)(9)— 
(A) by striking "The Foundation" and inserting the 
following: 
"(A) IN GENERAL.—The Foundation"; and 
(B) by adding at the end the following: 
"(B) GIFTS, GRANTS, AND OTHER DONATIONS.— 
"(i) IN GENERAL.—Gifts, grants, and other dona-
tions to the Foundation may be designated for pediatric 
research and studies on drugs, and ftmds so designated 
shall be used solely for grants for research and studies 
under subsection (c)(1)(C). 
"(ii) OTHER GIFTS.—Other gifts, grants, or dona-
tions received by the Foundation and not described 
in clause (i) may also be used to support such pediatric 
research and studies. 
"(iii) REPORT.—The recipient of a grant for 
research and studies shall agree to provide the Director 
of the National Institutes of Health and the Commis-
sioner of Food and Drugs, at the conclusion of the 
research and studies— 
"(I) a report describing the results of the 
research and studies; and 
"(II) all data generated in connection with the 
research and studies. 
"(iv) ACTION BY THE COMMISSIONER OF FOOD AND 
DRUGS.—The Commissioner of Food and Drugs shall 
take appropriate action in response to a report received 
under clause (iii) in accordance with paragraphs (7) 
through (12) of section 4091(c), including negotiating 
with the holders of approved applications for the drugs 
studied for any labeling changes that the Commis-
sioner determines to be appropriate and requests the 
holders to make. 
"(C) APPLICABILITY.—Subparagraph (A) does not apply 
to the program described in subsection (c)(lXC)."; 
(5) by redesignating subsections (f) through (m) as sub-
sections (e) through (1), respectively; 
(6) in subsection (h)(ll) (as so redesignated), by striking 
"solicit" and inserting "solicit,"; and 
PUBLIC LAW 107-109-^AN. 4, 2002 
115 STAT. 1419 
(7) in paragraphs (1) and (2) of subsection (j) (as so redesig-
nated), by striking "(including those developed under subsection 
(d)(2)(B)(i)(II))" each place it appears. 
SEC. 14. PEDIATRIC PHARMACOLOGY ADVISORY COMMITTEE. 
42 USC 284m 
(a) IN GENERAL.—The Secretary of Health and Human Services 
shall, under section 222 of the Public Health Service Act (42 U.S.C. 
217a), convene and consult an advisory committee on pediatric 
pharmacology (referred to in this section as the "advisory com-
mittee"). 
(b) PURPOSE.— 
(1) IN GENERAL,—The advisory committee shall advise and 
make recommendations to the Secretary, through the Commis-
sioner of Food and Drugs and in consultation with the Director 
of the National Institutes of Health, on matters relating to 
pediatric pharmacology. 
(2) MATTERS INCLUDED.—The matters referred to in para-
graph (1) include— 
(A) pediatric research conducted under sections 351, 
4091, and 499 of the Public Health Service Act and sections 
501, 502, 505, and 505A of the Federal Food, Drug, and 
Cosmetic Act; 
(B) identification of research priorities related to pedi-
atric pharmacology and the need for additional treatments 
of specific pediatric diseases or conditions; and 
(C) the ethics, design, and analysis of clinical trials 
related to pediatric pharmacology. 
(c) COMPOSITION.—The advisory committee shall include rep-
resentatives of pediatric health organizations, pediatric researchers, 
relevant patient and patient-family organizations, and other experts 
selected by the Secretary. 
SEC. 15. PEDIATRIC SUBCOMMITTEE OF THE ONCOLOGIC DRUGS 
ADVISORY COMMITTEE. 
(a) CLARIFICATION OF AUTHORITIES.— 
(1) IN GENERAL.—The Pediatric Subcommittee of the Onco-
logic Drugs Advisory Committee (referred to in this section 
as the "Subcommittee"), in carrying out the mission of reviewing 
and evaluating the data concerning the safety and effectiveness 
of marketed and investigational human drug products for use 
in the treatment of pediatric cancers, shall— 
(A) evaluate and, to the extent practicable, prioritize 
new and emerging therapeutic alternatives available to 
treat pediatric cancer; 
(B) provide recommendations and guidance to help 
ensure that children with cancer have timely access to 
the most promising new cancer therapies; and 
(C) advise on ways to improve consistency in the avail-
ability of new therapeutic agents. 
(2) MEMBERSHIP.— 
(A) IN GENERAL.—The Secretary shall appoint not more 
than 11 voting members to the Pediatric Subcommittee 
from the membership of the Pediatric Pharmacology 
Advisory Committee and the Oncologic Drugs Advisory 
Committee. 
(B) REQUEST FOR PARTICIPATION.—The Subcommittee 
shall request participation of the following members in 
115 STAT. 1420 
PUBLIC LAW 107-109-^AN. 4, 2002 
the scientific and ethical consideration of topics of pediatric 
cancer, as necessary: 
(i) At least two pediatric oncology specialists from 
the National Cancer Institute. 
(ii) At least four pediatric oncology specialists 
from— 
(I) the Children's Oncology Group; 
(II) other pediatric experts with an established 
history of conducting clinical trials in children; 
or 
(III) consortia sponsored by the National 
Cancer Institute, such as the Pediatric Brain 
Tumor Consortium, the New Approaches to Neuro-
blastoma Therapy or other pediatric oncology con-
sortia. 
(iii) At least two representatives of the pediatric 
cancer patient and patient-family community. 
(iv) One representative of the nursing community. 
(v) At least one statistician. 
(vi) At least one representative of the pharma-
ceutical industry. 
(b) PRE-CLINICAL MODELS TO EVALUATE PROMISING PEDIATRIC 
CANCER THERAPIES.—Section 413 of the Public Health Service Act 
(42 U.S.C. 285a-2) is amended by adding at the end the following: 
"(c) PRE-CLINICAL MODELS TO EVALUATE PROMISING PEDL\TRIC 
CANCER THERAPIES.— 
"(1) 
EXPANSION AND COORDINATION OF ACTIVITIES.—The 
Director of the National Cancer Institute shall expand, inten-
sify, and coordinate the activities of the Institute with respect 
to research on the development of preclinical models to evaluate 
which therapies are likely to be effective for treating pediatric 
cancer. 
"(2) COORDINATION WITH OTHER INSTITUTES.—^The Director 
of the Institute shall coordinate the activities under paragraph 
(1) with similar activities conducted by other national research 
institutes and agencies of the National Institutes of Health 
to the extent that those Institutes and agencies have respon-
sibilities that are related to pediatric cancer.". 
(c) CLARIFICATION OF AVAILABILITY OF INVESTIGATIONAL NEW 
DRUGS FOR PEDIATRIC STUDY AND USE.— 
(1) AMENDMENT OF THE FEDERAL FOOD, DRUG, AND COS-
METIC ACT.—Section 505(i)(l) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355(i)(l)) is amended— 
(A) in subparagraph (B), by striking "and" at the end; 
(B) in subparagraph (C), by striking the period at 
the end and inserting "; and"; and 
(C) by adding at the end the following: 
"(D) the submission to the Secretary by the manufac-
turer or the sponsor of the investigation of a new drug 
of a statement of intent regarding whether the manufac-
turer or sponsor has plans for assessing pediatric safety 
and efficacy.". 
(2) AMENDMENT OF THE PUBLIC HEALTH SERVICE ACT.— 
Section 402(j)(3)(A) of the Public Health Service Act (42 U.S.C. 
282(j)(3)(A)) is amended in the first sentence— 
(A) by striking "trial sites, and" and inserting "trial 
sites,"; and 
PUBLIC LAW 107-109^JAN. 4, 2002 
115 STAT. 1421 
(B) by striking "in the trial," and inserting "in the 
trial, and a description of whether, and through what proce-
dure, the manufacturer or sponsor of the investigation of 
a new drug will respond to requests for protocol exception, 
with 
appropriate 
safeguards, 
for 
single-patient 
and 
expanded protocol use of the new drug, particularly in 
children,". 
(d) REPORT.—Not later than January 31, 2003, the Secretary 
of Health and Human Services, acting through the Commissioner 
of Food and Drugs and in consultation with the Director of the 
National Institutes of Health, shall submit to the Committee on 
Health, Education, Labor, and Pensions of the Senate and the 
Committee on Energy and Commerce of the House of Representa-
tives a report on patient access to new therapeutic agents for 
pediatric cancer, including access to single patient use of new thera-
peutic agents. 
SEC. 16. REPORT ON PEDIATRIC EXCLUSIVITY PROGRAM. 
Not later than October 1, 2006, the Comptroller General of 
the United States, in consultation with the Secretary of Health 
and Human Services, shall submit to Congress a report that 
addresses the following issues, using publicly available data or 
data otherwise available to the Government that may be used 
and disclosed under applicable law: 
(1) The effectiveness of section 505A of the Federal Food, 
Drug, and Cosmetic Act and section 4091 of the Public Health 
Service Act (as added by this Act) in ensuring that medicines 
used by children are tested and properly labeled, including— 
(A) the number and importance of drugs for children 
that are being tested as a result of this legislation and 
the importance for children, health care providers, parents, 
and others of labeling changes made as a result of such 
testing; 
(B) the number and importance of drugs for children 
that are not being tested for their use notwithstanding 
the provisions of this legislation, and possible reasons for 
the lack of testing; and 
(C) the number of drugs for which testing is being 
done, exclusivity granted, and labeling changes required, 
including the date pediatric exclusivity is granted and the 
date labeling changes are made and which labeling changes 
required the use of the dispute resolution process estab-
lished pursuant to the amendments made by this Act, 
together with a description of the outcomes of such process, 
including a description of the disputes and the rec-
ommendations of the Pediatric Advisory Subcommittee of 
the Anti-Infective Drugs Advisory Committee. 
(2) The economic impact of section 505A of the Federal 
Food, Drug, and Cosmetic Act and section 4091 of the Public 
Health Service Act (as added by this Act), including an estimate 
of— 
(A) the costs to taxpayers in the form of higher expendi-
tures by medicaid and other Government programs; 
(B) sales for each drug during the 6-month period 
for which exclusivity is granted, as attributable to such 
exclusivity; 
Deadline. 
21 u s e 355 note. 
21 u s e 355a 
note. 
Deadline. 
115 STAT. 1422 
PUBLIC LAW 107-109—JAN. 4, 2002 
21 u s e 355b. 
Deadline. 
Regulations. 
(C) costs to consumers and private insurers as a result 
of any delay in the availability of lower cost generic equiva-
lents of drugs tested and granted exclusivity under the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et 
seq.), and loss of revenue by the generic drug industry 
and retail pharmacies as a result of any such delay; and 
(D) the benefits to the government, to private insurers, 
and to consumers resulting from decreased health care 
costs, including— 
(i) decreased hospitalizations and fewer medical 
errors, due to more appropriate and more effective 
use of medications in children as a result of testing 
and re-labeling because of the amendments made by 
this Act; 
(ii) direct and indirect benefits associated 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 insur-
ance premiums due to lower treatment costs and hos-
pitalization rates; and 
(v) benefits to employers from reduced need for 
employees to care for family members. 
(3) The nature and type of studies in children for each 
drug granted exclusivity under the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 301 et seq.), including— 
(A) a description of the complexity of the studies; 
(B) the number of study sites necessary to obtain 
appropriate data; 
(C) the number of children involved in any clinical 
studies; and 
(D) the estimated cost of each of the studies. 
(4) Any recommendations for modifications to the programs 
established under section 505A of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 355a) and section 4091 of the 
Public Health Service Act (as added by section 3) that the 
Secretary determines to be appropriate, including a detailed 
rationale for each recommendation. 
(5) The increased private and Government-funded pediatric 
research capability associated with this Act and the amend-
ments made by this Act. 
(6) The number of written requests and additional letters 
of recommendation that the Secretary issues. 
(7) The prioritized list of off-patent drugs for which the 
Secretary issues written requests. 
(8)(A) The efforts made by the Secretary to increase the 
number of studies conducted in the neonate population; and 
(B) the results of those efforts, including efforts made to 
encourage the conduct of appropriate studies in neonates by 
companies with products that have sufficient safety and other 
information to make the conduct of studies ethical and safe. 
SEC. 17. ADVERSE-EVENT REPORTING. 
(a) TOLL-FREE NUMBER IN LABELING.—Not later than one year 
after the date of the enactment of this Act, the Secretary of Health 
and Human Services shall promulgate a final rule requiring that 
PUBLIC LAW 107-109-^AN. 4, 2002 
115 STAT. 1423 
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 and a statement that 
such number is to be used for reporting purposes only, not to 
receive medical advice. 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 beginning 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 section 6 of this Act. In considering the report, the 
Director of such Office shall provide for the review of the 
report by the Pediatric Advisory Subcommittee of the Anti-
Infective Drugs Advisory Committee, including obtaining any 
recommendations of such subcommittee regarding whether the 
Secretary should take action under the Federal Food, Drug, 
and Cosmetic 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 continue 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. 18. MINORITY CHILDREN AND PEDIATRIC-EXCLUSIVITY PRO-
GRAM. 
(a) PROTOCOLS FOR PEDIATRIC STUDIES.—Section 505A of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is amended 
in subsection (d)(2) by inserting after the first sentence the fol-
lowing: "In reaching an agreement regarding written protocols, 
the Secretary shall take into account adequate representation of 
children of ethnic and racial minorities.". 
(b) STUDY BY GENERAL ACCOUNTING OFFICE.— 
21 use 355a 
(1) IN GENERAL.—The Comptroller General of the United note. 
States shall conduct a study for the purpose of determining 
the following: 
(A) The extent to which children of ethnic and racial 
minorities are adequately represented in studies under sec-
tion 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 rep-
resentation and recommendations to increase such rep-
resentation. 
115 STAT. 1424 
PUBLIC LAW 107-109—JAN. 4, 2002 
(B) Whether the Food and Drug Administration has 
appropriate management systems to monitor the represen-
tation of the children of ethnic and racial minorities in 
such studies. 
(C) Whether drugs used to address diseases that dis-
proportionately affect racial and ethnic minorities are being 
studied for their safety and effectiveness under section 
505A of the Federal Food, Drug, and Cosmetic Act. 
Deadline. 
(2) DATE CERTAIN FOR COMPLETING STUDY.—Not l a t e r t h a n 
January 10, 2003, the Comptroller General shall complete the 
study required in paragraph (1) and submit to the Congress 
a report describing the findings of the study. 
SEC. 19. TECHNICAL AND CONFORMING AMENDMENTS. 
Section 505A of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a) (as amended by sections 2(1), 5(b)(2), 9, 10, 11, 
and 17) is amended— 
(1)(A) by striking "(j)(4)(D)(ii)" each place it appears and 
inserting "(j)(5)(D)(ii)"; 
(B) by striking "(j)(4)(D)" each place it appears and 
inserting "(j)(5)(D)"; and 
(C) by striking "505(j)(4)(D)" each place it appears and 
inserting "505(j)(5)(D)"; 
(2) by redesignating subsections (a), (g), (h), (i), (j), (k), 
(1), (m), (n), and (o) as subsections (b), (a), (g), (h), (n), (m), 
(i), (j), (k), and (1) respectively; 
(3) by moving the subsections so as to appear in alphabet-
ical order; 
(4) in paragraphs (1), (2), and (3) of subsection (d), sub-
section (e), and subsection (m) (as redesignated by paragraph 
(2)), by striking "subsection (a) or (c)" and inserting "subsection 
(b) or (c)"; and 
(5) in subsection (g) (as redesignated by paragraph (2)), 
by striking "subsection (a) or (b)" and inserting "subsection 
(b)or(c)". 
Approved January 4, 2002. 
LEGISLATIVE HISTORY—S. 1789 (H.R. 2887): 
HOUSE REPORTS: No. 107-277 accompanying H.R. 2887 (Comm. on Energy and 
Commerce). 
CONGRESSIONAL RECORD, Vol. 147 (2001): 
Dec. 12, considered and passed Senate. 
Dec. 18, considered and passed House.