TITLE 21--FOOD AND DRUGS
CHAPTER I--FOOD AND DRUG ADMINISTRATION
DEPARTMENT OF HEALTH AND HUMAN SERVICES
PART 316--ORPHAN DRUGS
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.
(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pertinent unpublished and published papers are also required.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.
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