- Docket Number:
- Issued by:
Guidance Issuing OfficeCenter for Drug Evaluation and ResearchCenter for Biologics Evaluation and Research
The purpose of this guidance is to assist sponsors of drug and biological products for the treatment or prevention of rare diseases in conducting more efficient and successful drug development programs. Although the statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs, these issues are frequently more difficult to address in the context of a rare disease for which there is often limited medical and scientific knowledge, natural history data, and drug development experience.
You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))
If unable to submit comments online, please mail written comments to:
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852
All written comments should be identified with this document's docket number: FDA-2015-D-2818.