FDA启动罕见病框架倡议，以制定一个全面、适应性强的罕见病药物开发框架
正文 · 中文译文
FDA新闻稿 FDA启动罕见病框架倡议，制定全面、适宜的罕见病药物开发框架 此项工作从征求公众意见开始 更多新闻公告 即时发布： 2026年10月14日 美国食品药品监督管理局（FDA），作为美国卫生与公众服务部（HHS）下属机构，今日宣布启动罕见病框架倡议，旨在重新构想罕见病药物及生物制品的开发方式。根据一份新白皮书所述，该倡议将制定一个优化、适宜的开发框架，通过考虑每种疾病的细微差别、患者需求、产品类型以及在小规模患者群体中现实可行的证据生成方式，来反映罕见病药物开发的实际情况。 为就罕见病框架倡议征求患者及疾病倡导者、业界及其他相关方的意见，FDA将于10月28日在FDA白橡树园区举行会议。该会议将由里根-尤德尔基金会为FDA主持。 “患有罕见病的美国人无法承受一种对所有疾病都同等对待的监管体系，”卫生与公众服务部部小罗伯特·F·肯尼迪表示。“在特朗普总统领导下，FDA正在重新思考罕见病药物开发，以患者为中心，使监管要求与小规模患者群体的实际情况相匹配。我们将消除不必要的障碍，采纳前沿科学，加速有前景的治疗方法，同时坚持FDA的严格标准。” “患有罕见病的患者及其家属几乎没有或完全没有获批的治疗选择，不确定性带来的负担可能延伸至他们生活的方方面面，”FDA代理局长Kyle Diamantas（法学博士）表示。“随着我们对罕见病了解得更多，并更深入地理解它们所造成的痛苦之广，我们必须确保美国的监管体系始终走在最前沿——准备好应对新兴科学，预见患者及其家属的需求，并帮助将安全有效的疗法带给那些需要它们的人。FDA将努力为患者争取权益，通过构建一个更加一致、可预测和创新的罕见病药物开发方法，同时维护美国民众期望于FDA的严格标准。” FDA长期以来一直运用监管灵活性，并制定了有针对性的项目，以支持罕见病药物和生物制品开发中的创新。这些努力已为患者及其家属带来了有意义的进展。尽管如此，由于仅有约5%的罕见病症拥有FDA批准的治疗方法，FDA同意患者、药物开发者和提供者的看法，即必须采取更多措施，以加快安全有效的罕见病治疗方法的开发。在制定这一框架时，FDA致力于以下方面： 通过量身定制的指导和透明的沟通，在监管决策中提供更高的清晰度、一致性和可预测性。 在药物开发和审评过程中，既在早期阶段，也在明确规定的时间节点上，融入患者的声音。 进一步利用外部专业知识，以支持监管审评的定制化。 构建共享资源、强有力的培训以及关于罕见病药物研发和审评的制度性知识。 应用基于风险的方法，特别是在化学、生产和质量控制审评以及批准前检查方面。 倡导在罕见病药物研发中采用前沿科学、新方法和创新统计学方法。 该倡议由FDA罕见病创新中心牵头，并与局长办公室、生物制品评价与研究中心以及药物评价与研究中心合作。 “罕见病患者及其家属理应获得一个能够针对其各自病情特殊性进行处理的监管流程，同时保持FDA严格的标准，”该中心主任Amy Comstock Rick（法学博士）表示。“基于关键经验和里程碑，以大胆的愿景重新构想罕见病药物研发框架，是应对当前形势所必需的。这一新框架将增强申办方和患者双方的协调性和可预测性。” 公开会议之后还将提供更多发表意见的机会，包括分领域圆桌会议、信息征询以及FDA内部反馈会议。这些反馈意见将为新框架的详细制定提供信息，该框架可能包括潜在的新指南或修订版指南、法规修订、人员培训以及组织变革。 其他资源 • FDA罕见病创新中心 • 罕见病框架 媒体： FDA 置评请求 202-690-6343 消费者： 888-INFO-FDA 美国食品药品监督管理局（FDA）是美国卫生与公众服务部下属机构，通过确保人用及兽用药品、疫苗及其他人用生物制品以及医疗器械的安全、有效和可靠来保护公众健康。该机构还负责保障本国食品供应、化妆品、膳食补充剂和辐射型电子产品的安全与可靠，并负责监管烟草制品。 ### 内容更新截至： 10/14/2026 受监管产品 生物制品 药品 医疗器械 关注 FDA 在 X 上关注 @US_FDA 在 Facebook 上关注 FDA 在 X 上关注 @FDAMedia
正文 · 英文原文
FDA News Release FDA Launches the Rare Disease Framework Initiative to Develop a Holistic, Fit-For-Purpose Framework for Rare Disease Drug Development Effort begins by Soliciting Public Feedback More Press Announcements For Immediate Release: October 14, 2026 The U.S. Food and Drug Administration, a division within the U.S. Department of Health and Human Services (HHS), today announced the Rare Disease Framework Initiative to reimagine the development of drugs and biologics for rare diseases. As outlined in a new white paper, the Initiative will develop an optimized, fit-for-purpose framework that reflects the realities of rare disease drug development by considering the nuances of each disease, patient needs, product type and the evidence that can be realistically generated in small patient populations. To solicit feedback on the Rare Disease Framework Initiative from patients and disease advocates, industry, and others, the FDA will host an October 28 meeting at the FDA’s White Oak campus. The meeting will be facilitated by the Reagan-Udall Foundation for the FDA. “Americans with rare diseases cannot afford a regulatory system that treats every condition the same,” said HHS Secretary Robert F. Kennedy, Jr. “Under President Trump, FDA is rethinking rare disease drug development by putting patients at the center and matching regulatory requirements to the realities of small patient populations. We will remove unnecessary barriers, embrace cutting-edge science, and accelerate promising treatments while upholding FDA’s rigorous standards.” “Patients and families living with rare diseases have few or no approved treatment options, and the burden of uncertainty can extend across every part of their lives,” said Acting FDA Commissioner Kyle Diamantas, J.D. “As we learn more about rare diseases and better understand the breadth of suffering they cause, we must ensure that America’s regulatory system remains at the forefront — prepared to respond to emerging science, anticipate the needs of patients and families, and help bring safe and effective therapies to those who need them. The FDA will fight hard for patients by building a more consistent, predictable, and innovative approach to rare disease drug development while maintaining the rigorous standards Americans expect from the FDA.” The FDA has long used regulatory flexibility and developed targeted programs to support innovation in the development of drugs and biologics for rare diseases. These efforts have led to meaningful advances for patients and families. Nevertheless, with only 5% or so of rare conditions having an FDA-approved treatment, the FDA agrees with patients, drug developers, and providers that more must be done to speed the development of safe and effective treatments for rare diseases. In developing the framework, the FDA is committed to the following: Providing greater clarity, consistency, and predictability in regulatory decision-making through tailored guidance and transparent communication. Embedding the patient’s voice both early, and at clearly defined milestones, in the drug development and review process. Further leveraging external expertise to support customization of regulatory review. Building shared resources, robust training, and institutional knowledge on rare disease drug development and review. Applying risk-based approaches, particularly to chemistry, manufacturing, and controls review and pre-approval inspections. Championing the use of cutting-edge science, novel methods and innovative statistical approaches in rare disease drug development. 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. “Patients with rare diseases and their families deserve a regulatory process that addresses the uniqueness of each of their conditions while maintaining the FDA’s rigorous standards,” said the Hub’s Director, Amy Comstock Rick, J.D. “A bold vision to reimagine the framework for rare disease drug development that builds on key learnings and milestones is necessary to meet this moment. This new framework will enhance consistency and predictability for both sponsors and for patients.” Further opportunities to provide input will follow the public meeting, including sector-specific roundtables, Requests for Information, and internal FDA feedback sessions. This feedback will inform the detailed development of a new framework, which might include potential new or revised guidance, revisions to regulations, staff training, and organizational changes. Additional Resources • FDA Rare Disease Innovation Hub • Rare Disease Framework Media: FDA Request for Comment 202-690-6343 Consumer: 888-INFO-FDA The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products. ### Content current as of: 10/14/2026 Regulated Product(s) Biologics Drugs Medical Devices Follow FDA Follow @US_FDA on X Follow FDA on Facebook Follow @FDAMedia on X