[Code of Federal Regulations]
[Title 21, Volume 1]
[Revised as of April 1, 2003]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR99.305]

[Page 456-457]
 
                        TITLE 21--FOOD AND DRUGS
 
CHAPTER I--FOOD AND DRUG ADMINISTRATION, DEPARTMENT OF HEALTH AND HUMAN 
                                SERVICES
 
PART 99--DISSEMINATION OF INFORMATION ON UNAPPROVED/NEW USES FOR MARKETED DRUGS, BIOLOGICS, AND DEVICES--Table of Contents
 
    Subpart D--FDA Action on Submissions, Requests, and Applications
 
Sec. 99.305  Exemption from the requirement to file a supplemental application.

    (a) Within 60 days after receipt of an application for an exemption 
from the requirement of a supplemental application, FDA shall approve or 
deny the application.
    (1) If FDA does not act on the application for an exemption within 
the 60-day period, the application for an exemption shall be deemed to 
be approved.
    (2) If an application for an exemption is deemed to be approved, FDA 
may, at any time, terminate such approval if it determines that the 
requirements for granting an exemption have not been met. FDA shall 
notify the manufacturer if the approval is terminated.
    (b) In reviewing an application for an exemption, FDA shall consider 
the materials submitted by the manufacturer

[[Page 457]]

and may consider any other appropriate information, including, but not 
limited to, any pending or previously approved applications for 
exemption submitted by the manufacturer.
    (c) FDA may grant an application for an exemption if FDA determines 
that:
    (1) It would be economically prohibitive for the manufacturer to 
incur the costs necessary to submit a supplemental application for a new 
use, which at a minimum requires:
    (i) That existing data characterizing the safety and effectiveness 
of the drug or device, including data from the study described in the 
information to be disseminated are not adequate to support the 
submission of a supplemental application for the new use; and
    (ii) That the cost of the study or studies for the new use 
reasonably exceeds the expected revenue from the new use minus the cost 
of goods sold and marketing and administrative expenses attributable to 
the new use of the product, and there are not less expensive ways to 
obtain the needed information; or
    (2) It would be unethical to conduct clinical studies needed to 
support the submission of a supplemental application for the new use 
because:
    (i) Existing data characterizing the safety and effectiveness of the 
drug or device, including data from the study described in the 
information to be disseminated are not adequate to support the 
submission of a supplemental application for the new use; and
    (ii) Although available evidence would not support the submission of 
a supplemental application for the new use, the data are persuasive to 
the extent that withholding the drug or device in a controlled study 
would pose an unreasonable risk of harm to human subjects and no studies 
in different populations or of modified design can be utilized. In 
determining whether it would be unethical to conduct clinical studies, 
the agency shall consider, in addition to the persuasiveness of 
available evidence of effectiveness, whether the new use of the drug or 
device is broadly accepted as current standard medical treatment or 
therapy.