[Code of Federal Regulations]
[Title 21, Volume 4]
[Revised as of April 1, 2001]
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
 
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 
Secs. 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

[[Page 18]]

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 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]