[Code of Federal Regulations]
[Title 21, Volume 1]
[Revised as of April 1, 2007]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR99.205]
[Page 451-452]
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
Subpart C Manufacturer's Submissions, Requests, and Applications
Sec. 99.205 Application for exemption from the requirement to file a
supplemental application.
(a) In certain circumstances, described in paragraph (b) of this
section, a manufacturer may submit an application for an exemption from
the requirement to submit a supplemental application for a new use for
purposes of disseminating information on that use.
(b) The manufacturer's application for an exemption shall identify
the basis for the proposed exemption and shall include materials
demonstrating that it would be economically prohibitive or that it would
be unethical to conduct the studies necessary to submit a supplemental
application for the new use.
(1) If the basis for the manufacturer's application for exemption is
that it would be economically prohibitive to incur the costs necessary
to submit a supplemental application for a new use, the manufacturer
shall, at a minimum, provide:
(i) Evidence explaining why 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.
Such evidence shall include an analysis of all data relevant to the
safety and effectiveness of the use, a summary of those data, and any
documentation resulting from prior discussions with the agency
concerning the adequacy of the existing data; and
(ii) Evidence demonstrating that the cost of the study or studies
for the new use reasonably exceeds the expected revenue from the new use
minus the costs of goods sold and marketing and administrative expenses
attributable to the new use of the product. Such evidence shall include:
(A) A description of the additional studies that the manufacturer
believes are necessary to support the submission of a supplemental
application for the new use, including documentation from prior
discussions, if any, with the agency concerning the studies that
[[Page 452]]
would be needed, and an estimate of the projected costs for such
studies;
(B) The expected patient population for the new use;
(C) The expected revenue for the new use, including an explanation
of the price at which the drug or device will be sold;
(D) Any exclusivity for the drug or device for the new use; and
(E) Any other information that the manufacturer has showing that
conducting the studies on the new use would be economically prohibitive;
and
(iii) An attestation by a responsible individual of the manufacturer
or an individual acting on the manufacturer's behalf verifying that the
estimates included with the submission are accurate and were prepared in
accordance with generally accepted accounting procedures. The data
underlying and supporting the estimates shall be made available to FDA
upon request. Alternatively, a manufacturer may submit a report of an
independent certified public accountant in accordance with the Statement
of Standards for Attestation established by the American Institute of
Certified Public Accountants and agreed upon procedures performed with
respect to the estimates submitted under this section.
(2) If the basis for the manufacturer's application for exemption is
that it would be unethical to conduct the studies necessary for the
supplemental application for a new use, the manufacturer shall provide
evidence:
(i) Explaining why 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.
Such evidence shall include an analysis of all data relevant to the
safety and effectiveness of the new use, a summary of those data, and
any documentation resulting from prior discussions with the agency
concerning the adequacy of the existing data; and
(ii) Explaining why it would be unethical to conduct the further
studies that would be necessary for the approval of the new use. Such
evidence shall establish that, notwithstanding the insufficiency of
available data to 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 (e.g., by providing no therapy,
a placebo, an alternative therapy, or an alternative dose) would pose an
unreasonable risk of harm to human subjects. In assessing the
appropriateness of conducting studies to support the new use, the
manufacturer may provide evidence showing that the new use is broadly
accepted as current standard medical treatment or therapy. The
manufacturer shall also address the possibility of conducting studies in
different populations or of modified design (e.g., adding the new
therapy to existing treatments or using an alternative dose if
monotherapy studies could not be conducted).
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