[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: 21CFR316.30]

[Page 176-177]
 
                        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. 316.30  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

[[Page 177]]

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.