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GUIDANCE DOCUMENT

Rare Diseases: Considerations for the Development of Drugs and Biological Products December 2023

Download the Final Guidance Document Read the Federal Register Notice
Final Level 1 Guidance
Docket Number:
FDA-2015-D-2818
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 final guidance for industry entitled “Rare Diseases: Considerations for the Development of Drugs and Biological Products.”  This guidance is intended to assist sponsors of drugs and biological products for treatment of rare diseases in conducting efficient and successful drug development programs through a discussion of selected issues commonly encountered in rare disease drug development.  This guidance finalizes the draft guidance entitled “Rare Diseases: Common Issues in Drug Development” issued on February 1, 2019.

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You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

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All written comments should be identified with this document's docket number: FDA-2015-D-2818.

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