Rare Diseases: Comment Period Open Soon for Revised Draft


The Food and Drug Administration (FDA) has revised the draft guidance, “Rare Diseases: Common Issues in Drug Development Guidance for Industry”. The original draft guidance was issued August 2015. The revised draft guidance is offered as an advance notice and will be published in the Federal Register at a later date.


Rare diseases are frequently serious and life-threatening. The Orphan Drug Act (ODA) allows for incentives for sponsors to develop drugs or biologics for rare diseases. The drug or biologic must meet the criteria in  21 CFR 316.20 and 316.21 to be considered for orphan status.  A new, streamlined process to increase efficiency and reduce burden with the FDA has been implemented as part of the FDA’s Orphan Drug Modernization Plan. The goal is to respond to orphan status requests within 90 days. This draft guidance will be released to aid sponsors in “conducting more efficient and successful drug development programs” for rare diseases.

The revised draft includes:

  • Updates to the “Natural History Studies” section.

  • Additional information on validation of biomarkers for surrogate endpoints.

  • Additional information about external, historical controls and early randomization.

  • Adding a safety section.

  • More information and clarification about manufacturing process and drug substance changes.

  • Adding an “Additional Considerations” section to address:

    • participation of patients, caretakers, and advocates,

    • consideration of pediatric issues,

    • and interactions with FDA.

The draft guidance is designed to address challenges to developing drugs and biologics for rare diseases where there are less data, knowledge, and experience. Comments will be available at https://www.regulations.gov/ by searching the docket number after publication in the Federal Register and need to be submitted within 60 days of publication.

- The Clinical Pathways Team 

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