[Code of Federal Regulations]
[Title 21, Volume 5]
[Revised as of April 1, 2008]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR316.20]

[Page 178-179]
 
                        TITLE 21--FOOD AND DRUGS
 
CHAPTER I--FOOD AND DRUG ADMINISTRATION, DEPARTMENT OF 
 
PART 316_ORPHAN DRUGS--Table of Contents
 
                 Subpart C_Designation of an Orphan Drug
 
Sec. 316.20  Content and format of a request for orphan-drug designation.


    (a) A sponsor that submits a request for orphan-drug designation of 
a drug for a specified rare disease or condition shall submit each 
request in the form and containing the information required in paragraph 
(b) of this section. A sponsor may request orphan-drug designation of a 
previously unapproved drug, or of a new orphan indication for an already 
marketed drug. In addition, a sponsor of a drug that is otherwise the 
same drug as an already approved orphan drug may seek and obtain orphan-
drug designation for the subsequent drug for the same rare disease or 
condition if it can present a plausible hypothesis that its drug may be 
clinically superior to the first drug. More than one sponsor may receive 
orphan-drug designation of the same drug for the same rare disease or 
condition, but each sponsor seeking orphan-drug designation must file a 
complete request for designation as provided in paragraph (b) of this 
section.
    (b) A sponsor shall submit two copies of a completed, dated, and 
signed request for designation that contains the following:
    (1) A statement that the sponsor requests orphan-drug designation 
for a rare disease or condition, which shall be identified with 
specificity.
    (2) The name and address of the sponsor; the name of the sponsor's 
primary contact person and/or resident agent including title, address, 
and telephone number; the generic and trade name, if any, of the drug or 
drug product; and the name and address of the source of the drug if it 
is not manufactured by the sponsor.

[[Page 179]]

    (3) A description of the rare disease or condition for which the 
drug is being or will be investigated, the proposed indication or 
indications for use of the drug, and the reasons why such therapy is 
needed.
    (4) A description of the drug and a discussion of the scientific 
rationale for the use of the drug for the rare disease or condition, 
including all data from nonclinical laboratory studies, clinical 
investigations, and other relevant data that are available to the 
sponsor, whether positive, negative, or inconclusive. Copies of 
pertinent unpublished and published papers are also required.
    (5) Where the sponsor of a drug that is otherwise the same drug as 
an already-approved orphan drug seeks orphan-drug designation for the 
subsequent drug for the same rare disease or condition, an explanation 
of why the proposed variation may be clinically superior to the first 
drug.
    (6) Where a drug is under development for only a subset of persons 
with a particular disease or condition, a demonstration that the subset 
is medically plausible.
    (7) A summary of the regulatory status and marketing history of the 
drug in the United States and in foreign countries, e.g., IND and 
marketing application status and dispositions, what uses are under 
investigation and in what countries; for what indication is the drug 
approved in foreign countries; what adverse regulatory actions have been 
taken against the drug in any country.
    (8) Documentation, with appended authoritative references, to 
demonstrate that:
    (i) The disease or condition for which the drug is intended affects 
fewer than 200,000 people in the United States or, if the drug is a 
vaccine, diagnostic drug, or preventive drug, the persons to whom the 
drug will be administered in the United States are fewer than 200,000 
per year as specified in Sec. 316.21(b), or
    (ii) For a drug intended for diseases or conditions affecting 
200,000 or more people, or for a vaccine, diagnostic drug, or preventive 
drug to be administered to 200,000 or more persons per year in the 
United States, there is no reasonable expectation that costs of research 
and development of the drug for the indication can be recovered by sales 
of the drug in the United States as specified in Sec. 316.21(c).
    (9) A statement as to whether the sponsor submitting the request is 
the real party in interest of the development and the intended or actual 
production and sales of the product.
    (c) Any of the information previously provided by the sponsor to FDA 
under subpart B of this part may be referenced by specific page or 
location if it duplicates information required elsewhere in this 
section.