0338 21cfr601 28 2007

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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