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Rare Diseases: Common Issues in Drug Development Guidance for Industry Guidance for Industry February 2019


Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

Docket Number:
Issued by:
Guidance Issuing Office
Center for Drug Evaluation and Research
Center 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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Rare Diseases: Common Issues in Drug Development.” This draft guidance assists sponsors of drug and biological products intended to treat or prevent rare diseases in conducting more efficient and successful development programs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 This draft guidance addresses the following important aspects of drug development: Adequate description and understanding of the disease's natural history, adequate understanding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 This guidance revises and replaces the draft guidance of the same name issued on August 17, 2015.

Submit Comments

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:

Dockets Management
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.