I
107TH CONGRESS
2D SESSION
H. R. 4730
To amend the Federal Food, Drug, and Cosmetic Act to require labeling
containing information applicable to pediatric patients.
IN THE HOUSE OF REPRESENTATIVES
MAY 14, 2002
Mr. WAXMAN (for himself, Ms. DELAURO, Ms. ESHOO, Mr. DINGELL, and
Mr. BROWN of Ohio) introduced the following bill; which was referred to
the Committee on Energy and Commerce
A BILL
To amend the Federal Food, Drug, and Cosmetic Act to
require labeling containing information applicable to pe-
diatric patients.
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled,
SECTION 1. PEDIATRIC LABELING OF DRUGS AND BIOLOGI-
CAL 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. PEDIATRIC LABELING OF DRUGS AND BIOLOGI-
CAL 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 in-
gredient, 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 bio-
logical product license;
shall submit with the application the assessments de-
scribed in paragraph (2).
‘‘(2) ASSESSMENTS.—
‘‘(A) IN GENERAL.—The assessments re-
ferred to in paragraph (1) shall contain data,
gathered using appropriate formulations, that
are adequate—
‘‘(i) to assess the safety and effective-
ness of the drug, or the biological product
licensed under section 351 of the Public
Health Service Act (42 U.S.C. 262), for
the claimed indications in all relevant pedi-
atric subpopulations; and
‘‘(ii) to support dosing and adminis-
tration for each pediatric subpopulation for
which the drug, or the biological product li-
censed under section 351 of the Public
Health Service Act (42 U.S.C. 262), is
safe and effective.
‘‘(B) SIMILAR
COURSE
OF
DISEASE
OR
SIMILAR
EFFECT
OF
DRUG
OR
BIOLOGICAL
PRODUCT.—If the course of the disease and the
effects of the drug are sufficiently similar in
adults and pediatric patients, the Secretary may
conclude that pediatric effectiveness can be ex-
trapolated from adequate and well-controlled
studies in adults, usually supplemented with
other information obtained in pediatric patients,
such as pharmacokinetic studies.
‘‘(3) DEFERRAL.—On the initiative of the Sec-
retary or at the request of the applicant, the Sec-
retary may defer submission of some or all assess-
ments required under paragraph (1) until a specified
date after approval of the drug or issuance of the li-
cense for a biological product if—
‘‘(A) the Secretary finds that—
product under section 351 of the Public Health
Service Act (42 U.S.C. 262).
‘‘(d) WAIVERS.—
‘‘(1) FULL WAIVER.—At the request of an ap-
plicant, the Secretary shall grant a full waiver, as
appropriate, of the requirement to submit assess-
ments under subsection (a) or (b) if—
‘‘(A) necessary studies are impossible or
highly impracticable;
‘‘(B) there is evidence strongly suggesting
that the drug or biological product would be in-
effective or unsafe in all pediatric age groups;
or
‘‘(C)(i) the drug or biological product—
‘‘(I) does not represent a meaningful
therapeutic benefit over existing therapies
for pediatric patients; and
‘‘(II) is not likely to be used for a
substantial number of pediatric patients;
and
‘‘(ii) the absence of adequate labeling
would not pose significant risks to pediatric pa-
tients.
‘‘(2) PARTIAL WAIVER.—At the request of an
applicant, the Secretary shall grant a partial waiver,
as appropriate, of the requirement to submit assess-
ments under subsection (a) with respect to a specific
pediatric subpopulation if—
‘‘(A) any of the grounds stated in para-
graph (1) applies to that subpopulation; or
‘‘(B) the applicant demonstrates that rea-
sonable attempts to produce a pediatric formu-
lation necessary for that subpopulation have
failed.
‘‘(3) LABELING
REQUIREMENT.—If the Sec-
retary 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.
‘‘(e) MEETINGS.—The Secretary shall meet at appro-
priate times in the investigational new drug process with
the sponsor to discuss background information that the
sponsor shall submit on plans and timelines for pediatric
studies, or any planned request for waiver or deferral of
pediatric studies.’’.
(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) o 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
STUDY 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.’’.
(c) FINAL RULE.—Except to the extent that the final
rule is inconsistent with the amendment made by sub-
section (a), the final rule promulgating regulations requir-
ing manufacturers to assess the safety and effectiveness
of new drugs and biological products in pediatric patients
(63 Fed. Reg. 66632 (December 2, 1998)), shall be con-
sidered to implement the amendment made by subsection
(a).
(d) NO EFFECT ON AUTHORITY.—Section 505B of
the Federal Food, Drug, and Cosmetic Act (as added by
subsection (a)) does not affect whatever existing authority
the Secretary of Health and Human Services has to re-
quire pediatric assessments regarding the safety and effi-
cacy of drugs and biological products in addition to the
assessments required under that section. The authority,
if any, of the Secretary of Health and Human Services
regarding specific populations other than the pediatric
population shall be exercised in accordance with the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 301 et
seq.) as in effect on the day before the date of enactment
of this Act.
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