Federal Register #FDA-2026-N-12214

FDA seeks input on its rare disease therapy development and review framework

The U.S. Food and Drug Administration is seeking public input to shape its approach to developing and reviewing rare disease therapies. - Government organizations: U.S. Department of Health and Human Services (HHS), Food and Drug Administration (FDA), Rare Disease Innovation Hub, Office of the Commissioner, Center for Biologics Evaluation and Research, and Center for Drug Evaluation and Research. - Named collaborating organization: The Reagan-Udall Foundation convened the public meeting in collaboration with FDA; it is not identified as a product vendor or contractor. - Purpose: Gather input for FDA’s Rare Disease Framework Initiative and a fit-for-purpose approach that considers the disease, therapy type, patient population, and evidence that can reasonably be obtained. - Topics for input: Tailored review approaches; consistent and predictable regulatory requirements; engagement with patients and external experts; and chemistry, manufacturing, and controls considerations. - Potential framework improvements: Tailored regulatory guidance or requirements, staff training, and organizational enhancements. - Procurement scope: This is a public meeting and request for information, not a solicitation for products or contracted services. No OEMs, product quantities, part numbers, or service line items are identified.

Description

The FDA announces a public meeting and requests information and comments to support its Rare Disease Framework Initiative. Convened by the Reagan-Udall Foundation in collaboration with FDA, the hybrid meeting will address developing a fit-for-purpose framework for developing and regulating rare disease therapies. FDA seeks feedback on challenges in rare disease therapy development and on tailored regulatory review, including four focus areas described in the notice. The meeting is scheduled for October 28, 2026, and comments are due by December 11, 2026.

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