I 
108TH CONGRESS 
1ST SESSION H. R. 2857
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. 
IN THE HOUSE OF REPRESENTATIVES 
JULY 24, 2003
Mr. GREENWOOD (for himself, Ms. ESHOO, and Ms. PRYCE of Ohio) intro-
duced 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 
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 Representa-
tives of the United States of America in Congress assembled,
SECTION 1. SHORT TITLE. 
This Act may be cited as the ‘‘Pediatric Research Eq-
uity 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 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 new 
active ingredient, new indication, new dosage 
form, new dosing regimen, or new route of ad-
ministration; 
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 for 
each age group for which the assessment is re-
quired, that are adequate—
‘‘(i) to assess the safety and effective-
ness of the drug or the biological product 
for the claimed indications in all relevant 
pediatric subpopulations; and 
‘‘(ii) to support dosing and adminis-
tration 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 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 pediatric patients, 
such as pharmacokinetic studies.
‘‘(ii) EXTRAPOLATION BETWEEN AGE 
GROUPS.—A study may not be needed in 
each pediatric age group if data from 1 
age group can be extrapolated to another 
age group. 
‘‘(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—
‘‘(i) the drug or biological product is 
ready for approval for use in adults before 
pediatric studies are complete; 
‘‘(ii) pediatric studies should be de-
layed until additional safety or effective-
ness data have been collected; or 
‘‘(iii) there is another appropriate rea-
son for deferral; and 
‘‘(B) the applicant submits to the Sec-
retary—
‘‘(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 ap-
propriate, of the requirement to submit assess-
ments 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 exam-
ple, the number of patients is so small or 
the patients are geographically dispersed); 
‘‘(ii) there is evidence strongly sug-
gesting that the drug or biological product 
would be ineffective or unsafe in all pedi-
atric age groups; or 
‘‘(iii) the drug or biological product—
‘‘(I) does not represent a mean-
ingful therapeutic benefit over existing 
therapies for pediatric patients; and 
‘‘(II) is not likely to be used in a 
substantial number of pediatric pa-
tients.
‘‘(B) PARTIAL WAIVER.—On the initiative 
of the Secretary or at the request of an appli-
cant, the Secretary shall grant a partial waiver, 
as appropriate, of the requirement to submit as-
sessments for a drug or biological product 
under this subsection 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 exam-
ple, 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 sug-
gesting 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 mean-
ingful therapeutic 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 pa-
tients in that age group; or 
‘‘(iv) the applicant can demonstrate 
that reasonable attempts to produce a pe-
diatric formulation necessary for that age 
group have failed. 
‘‘(C) PEDIATRIC FORMULATION NOT POS-
SIBLE.—If a waiver is granted on the ground 
that it is not possible to develop a pediatric for-
mulation, the waiver shall cover only the pedi-
atric 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 prod-
uct would be ineffective or unsafe in pediatric 
populations, the information shall be included 
in the labeling for the drug or biological prod-
uct. 
‘‘(b) MARKETED DRUGS
AND BIOLOGICAL PROD-
UCTS.—
‘‘(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 advi-
sory committee meeting, the Secretary 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 prod-
uct 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 pa-
tients for the labeled indications; and 
‘‘(ii) the absence of adequate labeling could 
pose significant risks to pediatric patients; or 
‘‘(B)(i) there is reason to believe that the 
drug or biological product would represent a 
meaningful therapeutic benefit over existing 
therapies for pediatric patients for 1 or more of 
the claimed indications; and 
‘‘(ii) the absence of adequate labeling could 
pose significant risks to pediatric patients. 
‘‘(2) WAIVERS.—
‘‘(A) FULL WAIVER.—At the request of an 
applicant, the Secretary shall grant a full waiv-
er, as appropriate, of the requirement to submit 
assessments under this subsection if the appli-
cant certifies and the Secretary finds that—
‘‘(i) necessary studies are impossible 
or highly impracticable (because, for exam-
ple, the number of patients in that age 
group is so small or patients in that age 
group are geographically dispersed); or 
‘‘(ii) there is evidence strongly sug-
gesting that the drug or biological product 
would be ineffective or unsafe in all pedi-
atric 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 subsection 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 exam-
ple, 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 sug-
gesting that the drug or biological product 
would be ineffective or unsafe in that age 
group; 
‘‘(iii)(I) the drug or biological prod-
uct—
‘‘(aa) does not represent a mean-
ingful therapeutic 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 pa-
tients 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 pe-
diatric formulation necessary for that age 
group have failed. 
‘‘(C) PEDIATRIC FORMULATION NOT POS-
SIBLE.—If a waiver is granted on the ground 
that it is not possible to develop a pediatric for-
mulation, the waiver shall cover only the pedi-
atric 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 prod-
uct would be ineffective or unsafe in pediatric 
populations, the information shall be included
in the labeling for the drug or biological prod-
uct. 
‘‘(3) RELATIONSHIP TO OTHER PEDIATRIC PRO-
VISIONS.—
‘‘(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 un-
less—
‘‘(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)—