[Code of Federal Regulations]
[Title 21, Volume 5]
[Revised as of April 1, 2002]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR316.10]

[Page 176-177]
                        TITLE 21--FOOD AND DRUGS
                          SERVICES (CONTINUED)
PART 316--ORPHAN DRUGS--Table of Contents
  Subpart B--Written Recommendations for Investigations of Orphan Drugs
Sec. 316.10  Content and format of a request for written recommendations.

    (a) A sponsor's request for written recommendations from FDA 
concerning the nonclinical and clinical investigations necessary for 
approval of a marketing application shall be submitted in the form and 
contain the information required in this section. FDA may require the 
sponsor to submit information in addition to that specified in paragraph 
(b) of this section if FDA determines that the sponsor's initial request 
does not contain

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adequate information on which to base recommendations.
    (b) A sponsor shall submit two copies of a completed, dated, and 
signed request for written recommendations that contains the following:
    (1) The sponsor's name and address.
    (2) A statement that the sponsor is requesting written 
recommendations on orphan-drug development under section 525 of the act.
    (3) The name of the sponsor's primary contact person and/or resident 
agent, and the person's title, address, and telephone number.
    (4) The generic name and trade name, if any, of the drug and a list 
of the drug product's components or description of the drug product's 
formulation, and chemical and physical properties.
    (5) The proposed dosage form and route of administration.
    (6) A description of the disease or condition for which the drug is 
proposed to be investigated and the proposed indication or indications 
for use for such disease or condition.
    (7) Current regulatory and marketing status and history of the drug 
product, including:
    (i) Whether the product is the subject of an IND or a marketing 
application (if the product is the subject of an IND or a marketing 
application, the IND or marketing application numbers should be stated 
and the investigational or approved indication or indications for use 
    (ii) Known marketing experience or investigational status outside 
the United States;
    (iii) So far as is known or can be determined, all indications 
previously or currently under investigation anywhere;
    (iv) All adverse regulatory actions taken by the United States or 
foreign authorities.
    (8) The basis for concluding that the drug is for a disease or 
condition that is rare in the United States, including the following:
    (i) The size and other known demographic characteristics of the 
patient population affected and the source of this information.
    (ii) For drugs intended for diseases or conditions affecting 200,000 
or more people in the United States, or for a vaccine, diagnostic drug, 
or preventive drug that would be given to 200,000 or more persons per 
year, a summary of the sponsor's basis for believing that the disease or 
condition described in paragraph (b)(6) of this section occurs so 
infrequently that there is no reasonable expectation that the costs of 
drug development and marketing will be recovered in future sales of the 
drug in the United States. The estimated costs and sales data should be 
submitted as provided for in Sec. 316.21(c).
    (9) A summary and analysis of available data on the pharmacologic 
effects of the drug.
    (10) A summary and analysis of available nonclinical and clinical 
data pertinent to the drug and the disease to be studied including 
copies of pertinent published reports. When a drug proposed for orphan 
drug designation is intended to treat a life-threatening or severely 
debilitating illness, especially where no satisfactory alternative 
therapy exists, the sponsor may wish voluntarily to provide this 
information. A sponsor of such a drug may be entitled to expeditious 
development, evaluation, and marketing under 21 CFR part 312, subpart E.
    (11) An explanation of how the data summarized and analyzed under 
paragraphs (b)(9) and (b)(10) of this section support the rationale for 
use of the drug in the rare disease or condition.
    (12) A definition of the population from which subjects will be 
identified for clinical trials, if known.
    (13) A detailed outline of any protocols under which the drug has 
been or is being studied for the rare disease or condition and a summary 
and analysis of any available data from such studies.
    (14) The sponsor's proposal as to the scope of nonclinical and 
clinical investigations needed to establish the safety and effectiveness 
of the drug.
    (15) Detailed protocols for each proposed United States or foreign 
clinical investigation, if available.
    (16) Specific questions to be addressed by FDA in its 
recommendations for nonclinical laboratory studies and clinical 

[57 FR 62085, Dec. 29, 1992; 58 FR 6167, Jan. 26, 1993]

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