FDA启动「罕见病框架倡议」 重塑罕见病药物研发规则 10月28日在白橡树园区征集意见

华盛顿——美国食品药品监督管理局（FDA）宣布启动「罕见病框架倡议」（Rare Disease Framework Initiative），计划为罕见病药物与生物制品的研发制定一套更贴合实际的新框架。美国卫生与公众服务部（HHS）发布的公告称，FDA已就此发布白皮书，新框架将逐病考量每种疾病的特点、患者需求、产品类型，以及在患者人数很少的人群中现实可得的证据。

FDA表示，该局长期以来已运用监管灵活性并设立专门项目支持罕见病药物与生物制品创新，但目前仅约5%的罕见病有经FDA批准的治疗方法，该局与患者、药物研发方及医务人员一致认为，必须加快安全有效疗法的研发。

为征集患者与疾病倡导者、业界及其他各方的意见，FDA将于10月28日在位于马里兰州银泉市的FDA白橡树园区举行会议，会议由里根-尤德尔基金会（Reagan-Udall Foundation for the FDA）协办。此后还将陆续举行分行业圆桌会、发布信息征集通知并开展FDA内部反馈环节；收集到的意见将用于制定新框架的细节，成果可能包括新订或修订的指南、法规修订、人员培训与组织调整。

FDA在公告中列出制定框架的六项承诺：通过量身定制的指南与透明沟通，使监管决策更清晰、一致且可预期；在药物研发与审评流程中，尽早并在明确的关键节点纳入患者声音；更多借助外部专业力量，使监管审评更具针对性；建设共享资源、强化培训并积累罕见病药物研发与审评的机构知识；采用基于风险的方法，特别是在化学、生产与控制审评及批准前检查方面；倡导运用前沿科学、新方法与创新统计方法。

该倡议由FDA罕见病创新中心牵头，局长办公室、生物制品审评与研究中心及药品审评与研究中心共同参与。卫生与公众服务部部长小罗伯特·F·肯尼迪在公告中表示，罕见病患者等不起一套对每种疾病一视同仁的监管体系，FDA将在坚持严格标准的同时减少不必要的障碍、运用前沿科学并加快有前景的疗法。代理FDA局长凯尔·迪亚曼塔斯表示，该局将为患者努力，构建更一致、可预期且更具创新性的罕见病药物研发路径。

（来源：美国卫生与公众服务部与FDA官方公告，宣布、HHS新闻页更新）

FDA Launches Rare Disease Framework Initiative to Reimagine Drug Development; Public Meeting Set for October 28 at White Oak Campus

WASHINGTON — The U.S. Food and Drug Administration has announced the Rare Disease Framework Initiative, an effort to reimagine the development of drugs and biologics for rare diseases. In an announcement released through the U.S. Department of Health and Human Services (HHS), the FDA said that, as outlined in a new white paper, the initiative will develop an optimized, fit-for-purpose framework that accounts for the nuances of each disease, patient needs, product type and the evidence that can realistically be generated in small patient populations.

The FDA said it has long used regulatory flexibility and targeted programs to support innovation for rare diseases, but that only about 5% of rare conditions have an FDA-approved treatment, and the agency agreed with patients, drug developers and providers that more must be done to speed the development of safe and effective treatments.

To gather feedback from patients and disease advocates, industry and others, the FDA will host a meeting on October 28 at its White Oak campus in Silver Spring, Maryland, facilitated by the Reagan-Udall Foundation for the FDA. Further opportunities to provide input will follow, including sector-specific roundtables, Requests for Information and internal FDA feedback sessions. That feedback will inform the detailed framework, which might include new or revised guidance, revisions to regulations, staff training and organizational changes.

In developing the framework, the FDA committed to six areas: providing greater clarity, consistency and predictability in regulatory decision-making through tailored guidance and transparent communication; embedding the patient's voice early and at clearly defined milestones in development and review; further leveraging external expertise to customize regulatory review; building shared resources, training and institutional knowledge; applying risk-based approaches, particularly to chemistry, manufacturing and controls review and pre-approval inspections; and championing cutting-edge science, novel methods and innovative statistical approaches.

The initiative is led by the FDA's Rare Disease Innovation Hub, partnering with the Office of the Commissioner, the Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research. HHS Secretary Robert F. Kennedy, Jr. said Americans with rare diseases cannot afford a regulatory system that treats every condition the same, and that the FDA would remove unnecessary barriers and accelerate promising treatments while upholding its rigorous standards. Acting FDA Commissioner Kyle Diamantas said the agency would build a more consistent, predictable and innovative approach while maintaining those standards.

(Source: U.S. Department of Health and Human Services and FDA announcement, announced October 14, 2026; HHS press page updated October 15, 2026)

来源：美国卫生与公众服务部、FDA

发布：GSM·美国（发布链接）

图片来源：美国食品药品监督管理局（FDA）位于马里兰州银泉市白橡树园区的1号楼主入口（局长与高级职员办公室所在地，10月28日倡议意见征集会将在该园区举行）。美国FDA官方照片，公共领域，来源：Wikimedia Commons（File:FDA Bldg 1 - Exterior With Circle (5161374950).jpg）。The main entrance of FDA Building 1 on the White Oak campus in Silver Spring, Maryland, where the October 28 feedback meeting will be held. Photo by the U.S. Food and Drug Administration, public domain, via Wikimedia Commons

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