0338 21cfr601 27 2007

§ 601.27

21 CFR Ch. I (4–1–05 Edition)

(i) Institutional review and informed
consent. Information and data submitted under this section after July 27,
1981, shall include statements regarding each clinical investigation involving human subjects, that it was conducted in compliance with the requirements for informed consent under part
50 of this chapter. Such a study is also
subject to the requirements for institutional review under part 56 of this
chapter, unless exempt under § 56.104 or
§ 56.105.

tients are complete, pediatric studies
should be delayed until additional safety or effectiveness data have been collected. If an applicant requests deferred submission, the request must
provide an adequate justification for
delaying pediatric studies, a description of the planned or ongoing studies,
and evidence that the studies are being
or will be conducted with due diligence
and at the earliest possible time.
(2) If FDA determines that there is
an adequate justification for temporarily delaying the submission of assessments of pediatric safety and effectiveness, the product may be licensed
for use in adults subject to the requirement that the applicant submit the required assessments within a specified
time.
(c) Waivers—(1) General. FDA may
grant a full or partial waiver of the requirements of paragraph (a) of this section on its own initiative or at the request of an applicant. A request for a
waiver must provide an adequate justification.
(2) Full waiver. An applicant may request a waiver of the requirements of
paragraph (a) of this section if the applicant certifies that:
(i) The product does not represent a
meaningful therapeutic benefit over
existing therapies for pediatric patients and is not likely to be used in a
substantial number of pediatric patients;
(ii) Necessary studies are impossible
or highly impractical because, e.g., the
number of such patients is so small or
geographically dispersed; or
(iii) There is evidence strongly suggesting that the product would be ineffective or unsafe in all pediatric age
groups.
(3) Partial waiver. An applicant may
request a waiver of the requirements of
paragraph (a) of this section with respect to a specified pediatric age group,
if the applicant certifies that:
(i) The product does not represent a
meaningful therapeutic benefit over
existing therapies for pediatric patients in that age group, and is not
likely to be used in a substantial number of patients in that age group;
(ii) Necessary studies are impossible
or highly impractical because, e.g., the

[47 FR 44071, Oct. 5, 1982, as amended at 64 FR
56452, Oct. 20, 1999]

§ 601.27 Pediatric studies.
(a) Required assessment. Except as provided in paragraphs (b), (c), and (d) of
this section, each application for a new
active ingredient, new indication, new
dosage form, new dosing regimen, or
new route of administration shall contain data that are adequate to assess
the safety and effectiveness of the
product for the claimed indications in
all relevant pediatric subpopulations,
and to support dosing and administration for each pediatric subpopulation
for which the product is safe and effective. Where the course of the disease
and the effects of the product are similar in adults and pediatric patients,
FDA may conclude that pediatric effectiveness can be extrapolated from adequate and well-controlled effectiveness
studies in adults, usually supplemented
with other information in pediatric patients, such as pharmacokinetic studies. In addition, studies may not be
needed in each pediatric age group, if
data from one age group can be extrapolated to another. Assessments required under this section for a product
that represents a meaningful therapeutic benefit over existing treatments
must be carried out using appropriate
formulations for the age group(s) for
which the assessment is required.
(b) Deferred submission. (1) FDA may,
on its own initiative or at the request
of an applicant, defer submission of
some or all assessments of safety and
effectiveness described in paragraph (a)
of this section until after licensing of
the product for use in adults. Deferral
may be granted if, among other reasons, the product is ready for approval
in adults before studies in pediatric pa-

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Food and Drug Administration, HHS

§ 601.29

number of patients in that age group is
so small or geographically dispersed;
(iii) There is evidence strongly suggesting that the product would be ineffective or unsafe 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.
(4) FDA action on waiver. FDA shall
grant a full or partial waiver, as appropriate, if the agency finds that there is
a reasonable basis on which to conclude that one or more of the grounds
for waiver specified in paragraphs (c)(2)
or (c)(3) of this section have been met.
If a waiver is granted on the ground
that it is not possible to develop a pediatric formulation, the waiver will
cover only those pediatric age groups
requiring that formulation. If a waiver
is granted because there is evidence
that the product would be ineffective
or unsafe in pediatric populations, this
information will be included in the
product’s labeling.
(5) Definition of ‘‘meaningful therapeutic benefit’’. For purposes of this section, a product will be considered to
offer a meaningful therapeutic benefit
over existing therapies if FDA estimates that:
(i) If approved, the product would
represent a significant improvement in
the treatment, diagnosis, or prevention
of a disease, compared to marketed
products adequately labeled for that
use in the relevant pediatric population. Examples of how improvement
might be demonstrated include, e.g.,
evidence of increased effectiveness in
treatment, prevention, or diagnosis of
disease; elimination or substantial reduction of a treatment-limiting drug
reaction; documented enhancement of
compliance; or evidence of safety and
effectiveness in a new subpopulation;
or
(ii) The product is in a class of products or for an indication for which
there is a need for additional therapeutic options.
(d) Exemption for orphan drugs. This
section does not apply to any product
for an indication or indications for
which orphan designation has been
granted under part 316, subpart C, of
this chapter.

§ 601.28 Annual
reports
of
marketing pediatric studies.

Sponsors of licensed biological products shall submit the following information each year within 60 days of the
anniversary date of approval of each
product under the license to the Director, Center for Biologics Evaluation
and Research or the Director, Center
for Drug Evaluation and Research (see
mailing addresses in § 600.2 of this chapter):
(a) Summary. A brief summary stating whether labeling supplements for
pediatric use have been submitted and
whether new studies in the pediatric
population to support appropriate labeling for the pediatric population
have been initiated. Where possible, an
estimate of patient exposure to the
drug product, with special reference to
the pediatric population (neonates, infants, children, and adolescents) shall
be provided, including dosage form.
(b) Clinical data. Analysis of available
safety and efficacy data in the pediatric population and changes proposed
in the labeling based on this information. An assessment of data needed to
ensure appropriate labeling for the pediatric population shall be included.
(c) Status reports. A statement on the
current status of any postmarketing
studies in the pediatric population performed by, or on behalf of, the applicant. The statement shall include
whether postmarketing clinical studies
in pediatric populations were required
or agreed to, and, if so, the status of
these studies shall be reported to FDA
in annual progress reports of postmarketing studies under § 601.70 rather
than under this section.
[65 FR 59718, Oct. 6, 2000, as amended at 65 FR
64618, Oct. 30, 2000; 70 FR 14984, Mar. 24, 2005]

§ 601.29

Guidance documents.

(a) FDA has made available guidance
documents under § 10.115 of this chapter
to help you comply with certain requirements of this part.
(b) The Center for Biologics Evaluation and Research (CBER) maintains a
list of guidance documents that apply
to the center’s regulations. The lists
are maintained on the Internet and are
published annually in the FEDERAL
REGISTER. You may request a copy of

[63 FR 66671, Dec. 2, 1998]

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