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Guidance Issuing OfficeCenter 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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Rare Diseases: Common Issues in Drug Development.” This draft guidance assists sponsors of drug and biological products intended to treat or prevent rare diseases in conducting more efficient and successful development programs through discussions of selected issues commonly encountered in rare disease drug development. This draft guidance addresses the following important aspects of drug development: Adequate description and understanding of the disease's natural history, adequate understanding of the pathophysiology of the disease and the drug's proposed mechanism of action, nonclinical pharmacotoxicology considerations to support the proposed clinical investigation or investigations, reliable endpoints and outcome assessment, standard of evidence to establish safety and effectiveness, drug manufacturing considerations during drug development, participation of patients, caretakers, and advocates in development programs, and interactions with the Agency. This guidance revises and replaces the draft guidance of the same name issued on August 17, 2015.
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.