[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: 21CFR316.30]

[Page 183]
 
                        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. 316.30  Annual reports of holder of orphan-drug designation.

    Within 14 months after the date on which a drug was designated as an 
orphan drug and annually thereafter until marketing approval, the 
sponsor of a designated drug shall submit a brief progress report to the 
FDA Office of Orphan Products Development on the drug that includes:
    (a) A short account of the progress of drug development including a 
review of preclinical and clinical studies initiated, ongoing, and 
completed and a short summary of the status or results of such studies.
    (b) A description of the investigational plan for the coming year, 
as well as any anticipated difficulties in development, testing, and 
marketing; and
    (c) A brief discussion of any changes that may affect the orphan-
drug status of the product. For example, for products nearing the end of 
the approval process, sponsors should discuss any disparity between the 
probable marketing indication and the designated indication as related 
to the need for an amendment to the orphan-drug designation pursuant to 
Sec. 316.26.