I
107TH CONGRESS
2D SESSION
H. R. 5594
To amend the Federal Food, Drug, and Cosmetic Act to require labeling
containing information applicable to pediatric patients.
IN THE HOUSE OF REPRESENTATIVES
OCTOBER 9, 2002
Mrs. MORELLA (for herself and Ms. PRYCE of Ohio) introduced the following
bill; which was referred to the Committee on the 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,
‘‘(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—
‘‘(i) the drug or biological product is
ready for approval for use in adults before
pediatric studies are complete; or
‘‘(ii) pediatric studies should be de-
layed until additional safety or effective-
ness data have been collected; and
‘‘(B) 
the 
applicant 
submits 
to 
the
Secretary—
‘‘(i) a certified description of the
planned or ongoing studies; and
‘‘(ii) evidence that the studies are
being conducted or will be conducted with
due diligence.
‘‘(4) 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—
‘‘(i) necessary studies are impossible
or highly impractical;
‘‘(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.—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—
‘‘(i) necessary studies are impossible
or highly impractical;
‘‘(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 in a
substantial number of pediatric pa-
tients in that age group; or
‘‘(iv) the applicant demonstrates that
reasonable attempts to produce a pediatric
formulation necessary for that age group
have failed.
‘‘(C) 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 and
an opportunity for written response and a meeting,
which may include an advisory committee meeting,
the Secretary may by order require the holder of an
approved application relating to a drug under sec-
tion 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) 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—
‘‘(i) necessary studies are impossible
or highly impractical; 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—
‘‘(i) necessary studies are impossible
or highly impractical;
‘‘(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 product
does not represent a meaningful thera-
peutic benefit over existing therapies for
pediatric patients in that age group;
‘‘(II) the drug or biological product is
not likely to be used in a substantial num-
ber of pediatric patients in that age group;
and
‘‘(III) the absence of adequate label-
ing could not pose significant risks to pedi-
atric patients; or
‘‘(iv) the applicant demonstrates that
reasonable attempts to produce a pediatric
formulation necessary for that age group
have failed.
‘‘(C) 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
unless—
‘‘(i) the Secretary has issued a written
request for related pediatric studies under
section 505A(d) or section 409I of the
Public Health Service Act; and
‘‘(ii)(I) if the request was made under
section 505A(d)—
‘‘(aa) the recipient of the written
request does not agree to the request;
or
‘‘(bb) the Secretary does not re-
ceive 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—
‘‘(aa) the recipient of the written
request does not agree to the request;
or
‘‘(bb) the Secretary does not re-
ceive a response as specified under
section 409I(c)(2) of that Act.
‘‘(B) NO
EFFECT
ON
OTHER
AUTHOR-
ITY.—Nothing in this subsection shall be con-
strued to alter any requirement under section
505A(d)(4) or section 409I of the Public
Health Service Act. Subject to paragraph
(2)(A), nothing in this subsection, section
505A(d)(4), or section 409I or 499 of the Pub-
lic Health Service Act shall be construed to pre-
clude the Secretary from exercising the author-
ity of the Secretary under this subsection.
‘‘(c) DELAY IN SUBMISSION OF ASSESSMENTS.—If a
person delays the submission of assessments relating to
a drug or biological product beyond a date specified in
subsection (a) or (b)—
‘‘(1) the drug or biological product—
‘‘(A) may be considered by the Secretary
to be misbranded and subject to action under
sections 302 and 304; and
‘‘(B) shall not be subject to action under
section 303; and
‘‘(2) the delay shall not be the basis for a pro-
ceeding to withdraw approval for a drug under sec-
tion 505(e) or revoke the license for a biological
product under section 351 of the Public Health
Service Act (42 U.S.C. 262).
‘‘(d) 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) 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
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.
SEC. 2. TECHNICAL CORRECTION.
Section 505A of the Federal Food, Drug, and Cos-
metic 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)’’.
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