FDA发布罕见病框架倡议白皮书，并定于2026年10月28日召开公开会议征求意见

FDA Rare Disease Framework Initiative Public Meeting

On October 28, 2026, the U.S. Food and Drug Administration, in partnership with the Reagan-Udall Foundation for the FDA, will convene a hybrid public meeting to seek input related to the FDA’s Rare Disease Framework Initiative to reimagine the agency’s approach to rare disease development. 

Through the Rare Disease Framework Initiative, FDA will develop a framework for rare disease drug development and review where advice and decision-making are consistently tailored to what evidence is reasonably attainable given the nature of the disease, its onset and progression, target population, patient needs, and the characterizations of the product under investigation, and clearly communicated throughout the development and review process. The FDA intends to implement and communicate these improvements as appropriate, including potentially via changes to regulations, guidance documents, and internal processes and resources, which in turn will increase efficiency and enhance consistency and predictability.

The outcome of this Framework Initiative will be a rare disease drug development
process that:

• Provides greater clarity, consistency, and predictability for drug developers, patients, disease advocates, and researchers regarding rare disease regulatory decision-making through tailored guidance and transparent communication based on the latest science.
• Embeds the patient voice both early and at clearly defined milestones in the development and review process for rare disease therapies such that both sponsors and reviewers can benefit from patient perspectives on disease burden, benefit-risk priorities, and meaningful measures of progress.
• Further leverages external expertise to support a tailored approach in regulatory review, especially for rare conditions with very small patient populations and limited understanding of natural history, as a structural feature to direct FDA reviewers in customizing their decision-making to each specific development program, product, and disease state.
• Builds shared resources, robust training, and institutional knowledge on rare disease drug development and review to ensure consistency in drug development
  and review.
• Applies risk-based approaches, particularly to chemistry, manufacturing, and controls review and pre-approval inspections, to support earlier patient access without compromising rigorous quality, safety, and effectiveness standards.
• Champions the use of cutting-edge science, novel methods and clinical trial designs, and innovative statistical approaches in the development of rare disease therapies via guidance, transparent communication, and programming that reflects FDA’s commitment to partnering with rare disease innovators to accelerate patient access to rare disease therapies.

FDA discusses its aims for the Rare Disease Framework Initiative further in a white paper, released on October 14, 2026.