[Code of Federal Regulations]
[Title 21, Volume 4]
[Revised as of April 1, 2007]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR201.23]
[Page 17-18]
TITLE 21--FOOD AND DRUGS
CHAPTER I--FOOD AND DRUG ADMINISTRATION, DEPARTMENT OF HEALTH AND HUMAN
SERVICES (CONTINUED)
PART 201 LABELING--Table of Contents
Subpart A General Labeling Provisions
Sec. 201.23 Required pediatric studies.
(a) A manufacturer of a marketed drug product, including a
biological drug product, that is used in a substantial number of
pediatric patients, or that provides a meaningful therapeutic benefit
over existing treatments for pediatric patients, as defined in
Sec. Sec. 314.55(c)(5) and 601.27(c)(5) of this chapter, but whose
label does not provide adequate information to support its safe and
effective use in pediatric populations for the approved indications may
be required to submit an application containing data adequate to assess
whether the drug product is safe and effective in pediatric populations.
The application may be required to contain adequate evidence to support
dosage and administration in some or all pediatric subpopulations,
including neonates, infants, children, and adolescents, depending upon
the known or appropriate use of the drug product in such subpopulations.
The applicant may also be required to develop a pediatric formulation
for a drug product that represents a meaningful therapeutic benefit over
existing therapies for pediatric populations for whom a pediatric
formulation is necessary, unless the manufacturer demonstrates that
reasonable attempts to produce a pediatric formulation have failed.
(b) The Food and Drug Administration (FDA) may by order, in the form
of a letter, after notifying the manufacturer of its intent to require
an assessment of pediatric safety and effectiveness of a pediatric
formulation, and after offering an opportunity for a written response
and a meeting, which may include an advisory committee meeting, require
a manufacturer to submit an application containing the information or
request for approval of a pediatric formulation described in paragraph
(a) of this section within a time specified in the order, if FDA finds
that:
(1) The drug product is used in a substantial number of pediatric
patients for the labeled indications and the absence of adequate
labeling could pose significant risks to pediatric patients; or
(2) There is reason to believe that the drug product would represent
a meaningful therapeutic benefit over existing
[[Page 18]]
treatments for pediatric patients for one or more of the claimed
indications, and the absence of adequate labeling could pose significant
risks to pediatric patients.
(c)(1) An applicant may request a full waiver of the requirements of
paragraph (a) of this section if the applicant certifies that:
(i) Necessary studies are impossible or highly impractical because,
e.g., the number of such patients is so small or geographically
dispersed, or
(ii) There is evidence strongly suggesting that the product would be
ineffective or unsafe in all pediatric age groups.
(2) An applicant may request a partial 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:
(A) Does not represent a meaningful therapeutic benefit over
existing therapies for pediatric patients in that age group, and
(B) Is not likely to be used in a substantial number of patients in
that age group, and
(C) The absence of adequate labeling could not pose significant
risks to pediatric patients; or
(ii) Necessary studies are impossible or highly impractical because,
e.g., the number of patients in that age group is so small or
geographically dispersed, or
(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.
(3) 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.
(d) If a manufacturer fails to submit a supplemental application
containing the information or request for approval of a pediatric
formulation described in paragraph (a) of this section within the time
specified by FDA, the drug product may be considered misbranded or an
unapproved new drug or unlicensed biologic.
[63 FR 66668, Dec. 2, 1998]
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