[Code of Federal Regulations]
[Title 21 Volume 5]
[Revised as of April 1, 2003]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR316.25]

[Page 175]
 
                        TITLE 21--FOOD AND DRUGS
 
CHAPTER I--FOOD AND DRUG ADMINISTRATION, DEPARTMENT OF HEALTH AND HUMAN 
                          SERVICES (CONTINUED)
 
PART 316--ORPHAN DRUGS--Table of Contents
 
                Subpart C--Designation of an Orphan Drug
 
Sec. 316.25  Refusal to grant orphan-drug designation.

    (a) FDA will refuse to grant a request for orphan-drug designation 
if any of the following reasons apply:
    (1) The drug is not intended for a rare disease or condition 
because:
    (i) There is insufficient evidence to support the estimate that the 
drug is intended for treatment of a disease or condition in fewer than 
200,000 people in the United States, or that the drug is intended for 
use in prevention or in diagnosis in fewer than 200,000 people annually 
in the United States; or
    (ii) Where the drug is intended for prevention, diagnosis, or 
treatment of a disease or condition affecting 200,000 or more people in 
the United States, the sponsor has failed to demonstrate that there is 
no reasonable expectation that development and production costs will be 
recovered from sales of the drug for the orphan indication in the United 
States. A sponsor's failure to comply with Sec. 316.21 shall constitute 
a failure to make the demonstration required in this paragraph.
    (2) There is insufficient information about the drug, or the disease 
or condition for which it is intended, to establish a medically 
plausible basis for expecting the drug to be effective in the 
prevention, diagnosis, or treatment of that disease or condition.
    (3) A drug that is otherwise the same drug as one that already has 
orphan-drug exclusive approval for the same rare disease or condition 
and the sponsor has not submitted a medically plausible hypothesis for 
the possible clinical superiority of the subsequent drug.
    (b) FDA may refuse to grant a request for orphan-drug designation if 
the request for designation contains an untrue statement of material 
fact or omits material information.