Rare Diseases: Natural History Studies for Drug Development Draft Guidance for Industry March 2019
Not for implementation. Contains non-binding recommendations.
- Docket Number:
- Issued by:
Guidance Issuing OfficeOffice of the Commissioner, Office of Clinical Policy and Programs, Office of Orphan Products DevelopmentCenter for Drug Evaluation and ResearchCenter for Biologics Evaluation and Research
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Rare Diseases: Natural History Studies for Drug Development.” FDA is publishing this draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases. A natural history study collects information about the natural history of a disease in the absence of an intervention, from the disease's onset until either its resolution or the individual's death. Although knowledge of a disease's natural history can benefit drug development for many disorders and conditions, natural history information is usually not available or is incomplete for most rare diseases; therefore, natural history information is particularly needed for these diseases.
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-2019-D-0481.