Pediatric Research Equity Act of 2003
PL 108-155 (S.650), enacted 12/03/2003

117 STAT. 1936
PUBLIC LAW 108–155—DEC. 3, 2003
Public Law 108–155
108th Congress
An Act
To amend the Federal Food, Drug, and Cosmetic Act to authorize the Food and
Drug Administration to require certain research into drugs used in pediatric patients.
Be it enacted by the Senate and House of Representatives of
the United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the ‘‘Pediatric Research Equity Act
of 2003’’.
SEC. 2. RESEARCH 
INTO 
PEDIATRIC 
USES 
FOR 
DRUGS 
AND
BIOLOGICAL PRODUCTS.
(a) IN GENERAL.—Subchapter A of chapter V of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 351 et seq.) is amended
by inserting after section 505A the following:
‘‘SEC. 505B. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND
BIOLOGICAL PRODUCTS.
‘‘(a) NEW DRUGS AND BIOLOGICAL PRODUCTS.—
‘‘(1) IN GENERAL.—A person that submits an application
(or supplement to an application)—
‘‘(A) under section 505 for a new active ingredient,
new indication, new dosage form, new dosing regimen,
or new route of administration; or
‘‘(B) under section 351 of the Public Health Service
Act (42 U.S.C. 262) for a new active ingredient, new indica-
tion, new dosage form, new dosing regimen, or new route
of administration;
shall submit with the application the assessments described
in paragraph (2).
‘‘(2) ASSESSMENTS.—
‘‘(A) IN GENERAL.—The assessments referred to in para-
graph (1) shall contain data, gathered using appropriate
formulations for each age group for which the assessment
is required, that are adequate—
‘‘(i) to assess the safety and effectiveness of the
drug or the biological product for the claimed indica-
tions in all relevant pediatric subpopulations; and
‘‘(ii) to support dosing and administration for each
pediatric subpopulation for which the drug or the
biological product is safe and effective.
‘‘(B) SIMILAR COURSE OF DISEASE OR SIMILAR EFFECT
OF DRUG OR BIOLOGICAL PRODUCT.—
‘‘(i) IN GENERAL.—If the course of the disease and
the effects of the drug are sufficiently similar in adults
21 USC 355c.
21 USC 301 note.
Pediatric
Research Equity
Act of 2003.
Dec. 3, 2003
[S. 650]
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117 STAT. 1937
PUBLIC LAW 108–155—DEC. 3, 2003
and pediatric patients, the Secretary may conclude
that pediatric effectiveness can be extrapolated from
adequate and well-controlled studies in adults, usually
supplemented with other information obtained in pedi-
atric patients, such as pharmacokinetic studies.
‘‘(ii) EXTRAPOLATION
BETWEEN
AGE
GROUPS.—A
study may not be needed in each pediatric age group
if data from one age group can be extrapolated to
another age group.
‘‘(3) DEFERRAL.—On the initiative of the Secretary or at
the request of the applicant, the Secretary may defer submis-
sion of some or all assessments required under paragraph (1)
until a specified date after approval of the drug or issuance
of the license for a biological product if—
‘‘(A) the Secretary finds that—
‘‘(i) the drug or biological product is ready for
approval for use in adults before pediatric studies are
complete;
‘‘(ii) pediatric studies should be delayed until addi-
tional safety or effectiveness data have been collected;
or
‘‘(iii) there is another appropriate reason for
deferral; and
‘‘(B) the applicant submits to the Secretary—
‘‘(i) certification of the grounds for deferring the
assessments;
‘‘(ii) a description of the planned or ongoing studies;
and
‘‘(iii) evidence that the studies are being conducted
or will be conducted with due diligence and at the
earliest possible time.
‘‘(4) WAIVERS.—
‘‘(A) FULL WAIVER.—On the initiative of the Secretary
or at the request of an applicant, the Secretary shall grant
a full waiver, as appropriate, of the requirement to submit
assessments for a drug or biological product under this
subsection if the applicant certifies and the Secretary finds
that—
‘‘(i) necessary studies are impossible or highly
impracticable (because, for example, the number of
patients is so small or the patients are geographically
dispersed);
‘‘(ii) there is evidence strongly suggesting that the
drug or biological product would be ineffective or
unsafe in all pediatric age groups; or
‘‘(iii) the drug or biological product—
‘‘(I) does not represent a meaningful thera-
peutic benefit over existing therapies for pediatric
patients; and
‘‘(II) is not likely to be used in a substantial
number of pediatric patients.
‘‘(B) PARTIAL WAIVER.—On the initiative of the Sec-
retary or at the request of an applicant, the Secretary
shall grant a partial waiver, as appropriate, of the require-
ment to submit assessments for a drug or biological product
under this subsection with respect to a specific pediatric
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117 STAT. 1938
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age group if the applicant certifies and the Secretary finds
that—
‘‘(i) necessary studies are impossible or highly
impracticable (because, for example, the number of
patients in that age group is so small or patients
in that age group are geographically dispersed);
‘‘(ii) there is evidence strongly suggesting that the
drug or biological product would be ineffective or
unsafe in that age group;
‘‘(iii) the drug or biological product—
‘‘(I) does not represent a meaningful thera-
peutic benefit over existing therapies for pediatric
patients in that age group; and
‘‘(II) is not likely to be used by a substantial
number of pediatric patients in that age group;
or
‘‘(iv) the applicant can demonstrate that reasonable
attempts to produce a pediatric formulation necessary
for that age group have failed.
‘‘(C) PEDIATRIC
FORMULATION
NOT
POSSIBLE.—If a
waiver is granted on the ground that it is not possible
to develop a pediatric formulation, the waiver shall cover
only the pediatric groups requiring that formulation.
‘‘(D) LABELING REQUIREMENT.—If the Secretary grants
a full or partial waiver because there is evidence that
a drug or biological product would be ineffective or unsafe
in pediatric populations, the information shall be included
in the labeling for the drug or biological product.
‘‘(b) MARKETED DRUGS AND BIOLOGICAL PRODUCTS.—
‘‘(1) IN GENERAL.—After providing notice in the form of
a letter and an opportunity for written response and a meeting,
which may include an advisory committee meeting, the Sec-
retary may (by order in the form of a letter) require the holder
of an approved application for a drug under section 505 or
the holder of a license for a biological product under section
351 of the Public Health Service Act (42 U.S.C. 262) to submit
by a specified date the assessments described in subsection
(a)(2) if the Secretary finds that—
‘‘(A)(i) the drug or biological product is used for a
substantial number of pediatric patients for the labeled
indications; and
‘‘(ii) the absence of adequate labeling could pose signifi-
cant risks to pediatric patients; or
‘‘(B)(i) there is reason to believe that the drug or
biological product would represent a meaningful thera-
peutic benefit over existing therapies for pediatric patients
for one or more of the claimed indications; and
‘‘(ii) the absence of adequate labeling could pose signifi-
cant risks to pediatric patients.
‘‘(2) WAIVERS.—
‘‘(A) FULL WAIVER.—At the request of an applicant,
the Secretary shall grant a full waiver, as appropriate,
of the requirement to submit assessments under this sub-
section if the applicant certifies and the Secretary finds
that—
‘‘(i) necessary studies are impossible or highly
impracticable (because, for example, the number of
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117 STAT. 1939
PUBLIC LAW 108–155—DEC. 3, 2003
patients in that age group is so small or patients
in that age group are geographically dispersed); or
‘‘(ii) there is evidence strongly suggesting that the
drug or biological product would be ineffective or
unsafe in all pediatric age groups.
‘‘(B) PARTIAL WAIVER.—At the request of an applicant,
the Secretary shall grant a partial waiver, as appropriate,
of the requirement to submit assessments under this sub-
section with respect to a specific pediatric age group if
the applicant certifies and the Secretary finds that—
‘‘(i) necessary studies are impossible or highly
impracticable (because, for example, the number of
patients in that age group is so small or patients
in that age group are geographically dispersed);
‘‘(ii) there is evidence strongly suggesting that the
drug or biological product would be ineffective or
unsafe in that age group;
‘‘(iii)(I) the drug or biological product—
‘‘(aa) does not represent a meaningful thera-
peutic benefit over existing therapies for pediatric
patients in that age group; and
‘‘(bb) is not likely to be used in a substantial
number of pediatric patients in that age group;
and
‘‘(II) the absence of adequate labeling could not
pose significant risks to pediatric patients; or
‘‘(iv) the applicant can demonstrate that reasonable
attempts to produce a pediatric formulation necessary
for that age group have failed.
‘‘(C) PEDIATRIC
FORMULATION
NOT
POSSIBLE.—If a
waiver is granted on the ground that it is not possible
to develop a pediatric formulation, the waiver shall cover
only the pediatric groups requiring that formulation.
‘‘(D) LABELING REQUIREMENT.—If the Secretary grants
a full or partial waiver because there is evidence that
a drug or biological product would be ineffective or unsafe
in pediatric populations, the information shall be included
in the labeling for the drug or biological product.
‘‘(3) RELATIONSHIP TO OTHER PEDIATRIC PROVISIONS.—
‘‘(A) NO ASSESSMENT WITHOUT WRITTEN REQUEST.—No
assessment may be required under paragraph (1) for a
drug subject to an approved application under section 505
unless—
‘‘(i) the Secretary has issued a written request
for a related pediatric study under section 505A(c)
of this Act or section 409I of the Public Health Service
Act (42 U.S.C. 284m);
‘‘(ii)(I) if the request was made under section
505A(c)—
‘‘(aa) the recipient of the written request does
not agree to the request; or
‘‘(bb) the Secretary does not receive a response
as specified under section 505A(d)(4)(A); or
‘‘(II) if the request was made under section 409I
of the Public Health Service Act (42 U.S.C. 284m)—
‘‘(aa) the recipient of the written request does
not agree to the request; or
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117 STAT. 1940
PUBLIC LAW 108–155—DEC. 3, 2003
‘‘(bb) the Secretary does not receive a response
as specified under section 409I(c)(2) of that Act;
and
‘‘(iii)(I) the Secretary certifies under subparagraph
(B) that there are insufficient funds under sections
409I and 499 of the Public Health Service Act (42
U.S.C. 284m, 290b) to conduct the study; or
‘‘(II) the Secretary publishes in the Federal Reg-
ister a certification that certifies that—
‘‘(aa) no contract or grant has been awarded
under section 409I or 499 of the Public Health
Service Act (42 U.S.C. 284m, 290b); and
‘‘(bb) not less than 270 days have passed since
the date of a certification under subparagraph (B)
that there are sufficient funds to conduct the study.
‘‘(B) NO AGREEMENT TO REQUEST.—Not later than 60
days after determining that no holder will agree to the
written request (including a determination that the Sec-
retary has not received a response specified under section
505A(d) of this Act or section 409I of the Public Health
Service Act (42 U.S.C. 284m), the Secretary shall certify
whether the Secretary has sufficient funds to conduct the
study under section 409I or 499 of the Public Health Service
Act (42 U.S.C. 284m, 290b), taking into account the
prioritization under section 409I.
‘‘(c) MEANINGFUL THERAPEUTIC BENEFIT.—For the purposes of
paragraph (4)(A)(iii)(I) and (4)(B)(iii)(I) of subsection (a) and para-
graphs (1)(B)(i) and (2)(B)(iii)(I)(aa) of subsection (b), a drug or
biological product shall be considered to represent a meaningful
therapeutic benefit over existing therapies if the Secretary estimates
that—
‘‘(1) if approved, the drug or biological product would rep-
resent a significant improvement in the treatment, diagnosis,
or prevention of a disease, compared with marketed products
adequately labeled for that use in the relevant pediatric popu-
lation; or
‘‘(2) the drug or biological product is in a class of products
or for an indication for which there is a need for additional
options.
‘‘(d) SUBMISSION OF ASSESSMENTS.—If a person fails to submit
an assessment described in subsection (a)(2), or a request for
approval of a pediatric formulation described in subsection (a) or
(b), in accordance with applicable provisions of subsections (a) and
(b)—
‘‘(1) the drug or biological product that is the subject of
the assessment or request may be considered misbranded solely
because of that failure and subject to relevant enforcement
action (except that the drug or biological product shall not
be subject to action under section 303); but
‘‘(2) the failure to submit the assessment or request shall
not be the basis for a proceeding—
‘‘(A) to withdraw approval for a drug under section
505(e); or
‘‘(B) to revoke the license for a biological product under
section 351 of the Public Health Service Act (42 U.S.C.
262).
Deadline.
Certification.
Certification.
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117 STAT. 1941
PUBLIC LAW 108–155—DEC. 3, 2003
‘‘(e) MEETINGS.—Before and during the investigational process
for a new drug or biological product, the Secretary shall meet
at appropriate times with the sponsor of the new drug or biological
product to discuss—
‘‘(1) information that the sponsor submits on plans and
timelines for pediatric studies; or
‘‘(2) any planned request by the sponsor for waiver or
deferral of pediatric studies.
‘‘(f) SCOPE OF AUTHORITY.—Nothing in this section provides
to the Secretary any authority to require a pediatric 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.
‘‘(g) ORPHAN DRUGS.—Unless the Secretary requires otherwise
by regulation, this section does not apply to any drug for an indica-
tion for which orphan designation has been granted under section
526.
‘‘(h) INTEGRATION
WITH
OTHER
PEDIATRIC
STUDIES.—The
authority under this section shall remain in effect 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(n).’’.
(b) CONFORMING AMENDMENTS.—(1) Section 505(b)(1) of the
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(b)(1)) is
amended in the second sentence—
(A) by striking ‘‘and (F)’’ and inserting ‘‘(F)’’; and
(B) by striking the period at the end and inserting
‘‘, and (G) any assessments required under section 505B.’’.
(2) Section 505A(h) of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355a(h)) is amended—
(A) in the subsection heading, by striking ‘‘REGULATIONS’’
and inserting ‘‘PEDIATRIC RESEARCH REQUIREMENTS’’; and
(B) by striking ‘‘pursuant to regulations promulgated by
the Secretary’’ and inserting ‘‘by a provision of law (including
a regulation) other than this section’’.
(3) Section 351(a)(2) of the Public Health Service Act (42 U.S.C.
262(a)(2)) is amended—
(A) by redesignating subparagraph (B) as subparagraph
(C); and
(B) by inserting after subparagraph (A) the following:
‘‘(B) PEDIATRIC STUDIES.—A person that submits an
application for a license under this paragraph shall submit
to the Secretary as part of the application any assessments
required under section 505B of the Federal Food, Drug,
and Cosmetic Act.’’.
SEC. 3. TECHNICAL AND CONFORMING AMENDMENTS.
(a) ABBREVIATED NEW DRUG APPLICATION.—Section 505A of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is
amended in subparagraphs (A) and (B) of subsection (b)(2) and
subparagraphs (A) and (B) of subsection (c)(2) by striking
‘‘505(j)(4)(B)’’ and inserting ‘‘505(j)(5)(B)’’.
(b) PEDIATRIC ADVISORY COMMITTEE.—(1) Section 505A(i)(2) of
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a(i)(2))
is amended by striking ‘‘Advisory Subcommittee of the Anti-Infective
Drugs’’ each place it appears.
(2) Section 14 of the Best Pharmaceuticals for Children Act
(42 U.S.C. 284m note; Public Law 107–109) is amended—
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PUBLIC LAW 108–155—DEC. 3, 2003
(A) in the section heading, by striking ‘‘PHARMACOLOGY’’;
(B) in subsection (a), by striking ‘‘(42 U.S.C. 217a),’’ and
inserting (42 U.S.C. 217a) or other appropriate authority,’’;
(C) in subsection (b)—
(i) in paragraph (1), by striking ‘‘and in consultation
with the Director of the National Institutes of Health’’;
and
(ii) in paragraph (2), by striking ‘‘and 505A’’ and
inserting ‘‘505A, and 505B’’; and
(D) by striking ‘‘pharmacology’’ each place it appears and
inserting ‘‘therapeutics’’.
(3) Section 15(a)(2)(A) of the Best Pharmaceuticals for Children
Act (115 Stat. 1419) is amended by striking ‘‘Pharmacology’’.
(4) Section 16(1)(C) of the Best Pharmaceuticals for Children
Act (21 U.S.C. 355a note; Public Law 107–109) is amended by
striking ‘‘Advisory Subcommittee of the Anti-Infective Drugs’’.
(5) Section 17(b)(1) of the Best Pharmaceuticals for Children
Act (21 U.S.C. 355b(b)(1)) is amended in the second sentence by
striking ‘‘Advisory Subcommittee of the Anti-Infective Drugs’’.
(6) Paragraphs (8), (9), and (11) of section 409I(c) of the Public
Health Service Act (42 U.S.C. 284m(c)) are amended by striking
‘‘Advisory Subcommittee of the Anti-Infective Drugs’’ each place
it appears.
SEC. 4. EFFECTIVE DATE.
(a) IN GENERAL.—Subject to subsection (b), this Act and the
amendments made by this Act take effect on the date of enactment
of this Act.
(b) APPLICABILITY TO NEW DRUGS AND BIOLOGICAL PRODUCTS.—
(1) IN
GENERAL.—Subsection (a) of section 505B of the
Federal Food, Drug, and Cosmetic Act (as added by section
2) shall apply to an application described in paragraph (1)
of that subsection submitted to the Secretary of Health and
Human Services on or after April 1, 1999.
(2) WAIVERS AND DEFERRALS.—
(A) WAIVER OR DEFERRAL GRANTED.—If, with respect
to an application submitted to the Secretary of Health
and Human Services between April 1, 1999, and the date
of enactment of this Act, a waiver or deferral of pediatric
assessments was granted under regulations of the Sec-
retary then in effect, the waiver or deferral shall be a
waiver or deferral under subsection (a) of section 505B
of the Federal Food, Drug, and Cosmetic Act, except that
any date specified in such a deferral shall be extended
by the number of days that is equal to the number of
days between October 17, 2002, and the date of enactment
of this Act.
(B) WAIVER
AND
DEFERRAL
NOT
GRANTED.—If, with
respect to an application submitted to the Secretary of
Health and Human Services between April 1, 1999, and
the date of enactment of this Act, neither a waiver nor
deferral of pediatric assessments was granted under regula-
tions of the Secretary then in effect, the person that sub-
mitted the application shall be required to submit assess-
ments under subsection (a)(2) of section 505B of the Federal
Food, Drug, and Cosmetic Act on the date that is the
later of—
21 USC 355c
note.
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PUBLIC LAW 108–155—DEC. 3, 2003
LEGISLATIVE HISTORY—S. 650:
SENATE REPORTS: No. 108–84 (Comm. on Health, Education, Labor, and Pen-
sions).
CONGRESSIONAL RECORD, Vol. 149 (2003):
July 21, considered and passed Senate.
Nov. 19, considered and passed House.
(i) the date that is 1 year after the date of enact-
ment of this Act; or
(ii) such date as the Secretary may specify under
subsection (a)(3) of that section;
unless the Secretary grants a waiver under subsection
(a)(4) of that section.
(c) NO LIMITATION OF AUTHORITY.—Neither the lack of guidance
or regulations to implement this Act or the amendments made
by this Act nor the pendency of the process for issuing guidance
or regulations shall limit the authority of the Secretary of Health
and Human Services under, or defer any requirement under, this
Act or those amendments.
Approved December 3, 2003.
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