Agency is particularly focused on new uses to treat chronic or rare diseases
The U.S. Food and Drug Administration today announced it is soliciting input on efforts with respect to drug repurposing to help address unmet medical needs across a range of diseases and conditions.
Identifying potential new uses—such as a new indication or a new population—for FDA-approved drugs can help accelerate the availability of treatments by using existing knowledge about the drugs, including a drug's safety profile. This request for public input is part of a broader FDA initiative to update the labeling of FDA-approved drugs, when supported by sufficient evidence, to ensure that information in the labeling is clinically meaningful for health care providers and patients and scientifically up to date.
“Too many patients lack effective treatment options, even when promising science exists,” said FDA Commissioner Marty Makary, M.D., M.P.H. “Drug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need.”
The FDA is seeking information from patients, clinicians, researchers, and other stakeholders on priority disease areas and potential candidates for drug repurposing, particularly where there are scientific data that could support approval of potential new uses but there appears to be limited commercial incentives to pursue approval of those uses.
The agency is particularly interested in input on disease areas with significant unmet medical need, including metabolic diseases, neurodegenerative conditions, women’s and men’s health conditions, substance use disorders, and rare diseases, as well as other areas stakeholders believe should be prioritized.
Through this docket, the FDA is requesting information on:
- Candidates for drug repurposing where sufficient evidence may already exist to support a potential new use,
- Candidates with promising preliminary clinical data (e.g., data from case reports, case series, observational studies) that may warrant further study, and
- Candidates with promising preliminary preclinical data (e.g., findings from emerging tools such as artificial intelligence and machine learning) that may warrant further study.
The agency is also seeking feedback on innovative approaches to identifying repurposing opportunities as well as barriers that may limit the development or use of repurposed drugs, particularly in cases where there is little or no commercial incentive to pursue labeling changes supported by publicly available scientific evidence.
This effort builds on existing authorities and initiatives that support updating drug labeling when supported by scientific evidence, including the Best Pharmaceuticals for Children Act, the Making Objective Drug Evidence Revisions for New (MODERN) Labeling Act of 2020, and FDA-led initiatives such as Project Renewal, which has updated labeling for several oncology drugs to reflect current scientific understanding. Additionally, the September 2025 Make Our Children Healthy Again strategy report directed FDA to jointly investigate opportunities with the National Institutes of Health to “strengthen the use of repurposed drugs for the treatment of chronic disease, while harmonizing authorization processes through collaborative clinical trial designs to achieve FDA approval.”
Input from the public will help inform how the FDA refines its approach to evaluating and facilitating additional drug repurposing opportunities, including collaborations with federal partners such as the National Institutes of Health and the Centers for Medicare and Medicaid Services.
Interested parties may submit electronic or written comments, data, and information to the docket. Electronic submissions may be made via the Federal eRulemaking Portal at https://www.regulations.gov. Written submissions may be mailed or delivered to the Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
All submissions must include the docket number FDA-2026-N-4492 for “Drug Repurposing for Unmet Medical Needs; Request for Information.”
Additional Resources
An unofficial translation of this announcement is provided below.
翻译参考：
FDA 推动药物再利用以应对未满足的医疗需求
FDA特别关注用于治疗慢性病或罕见病的新用途
2026年5月12日，美国食品药品管理局（FDA）宣布，将就药物再利用相关工作征求意见，以帮助解决多种疾病和病症中尚未满足的医疗需求。
利用现有关于药物的信息（包括其安全性特征），识别已获FDA批准药物的潜在新用途 – 例如新的适应症或新的适用人群 —可加快治疗手段的可及性。本次征求公众意见是 FDA一项更广泛举措的一部分：即在有充分证据支持的情况下，对已获 FDA 批准药物的说明书进行更新，以确保其中的信息对医疗服务提供者和患者具有临床意义，并在科学上保持最新。
“即使相关科学研究已颇具前景，仍有太多患者缺乏有效的治疗选择，”FDA 局长 Marty Makary博士表示，“药物再利用可以更好地利用现有科学数据，为有需要的患者提供有效的治疗方案。”
FDA 正向患者、临床医生、研究人员及其他利益相关方征求信息，重点关注优先疾病领域及潜在的药物再利用候选药物，特别是那些已有科学数据支持潜在新用途的批准、但商业开发激励有限的情况。
FDA特别关注有显著未满足的医疗需求的疾病领域，包括代谢性疾病、神经退行性疾病、女性和男性健康问题、物质使用障碍以及罕见病，以及其他利益相关方认为应优先考虑的领域。
通过本次案卷征求意见，FDA 希望获得以下信息：
- 可能已有足够证据支持新用途的药物再利用候选品种；
- 具有良好的初步临床数据（例如病例报告、病例系列、观察性研究数据），值得进一步研究的候选品种；
- 具有良好的初步临床前数据（例如来自人工智能和机器学习等新兴工具的发现），值得进一步研究的候选品种。
FDA 还征求关于识别药物再利用机会的创新方法，以及限制再利用药物开发或使用的障碍方面的反馈，尤其是在缺乏商业激励、难以基于公开科学证据推动说明书变更的情形下。
这一工作建立在现有法律授权和相关举措基础之上，这些法规和举措支持在有科学证据支持时更新药物说明书，包括《儿童最佳药品法》、2020年《基于客观药品证据进行新标签修订法案》，以及 FDA 主导的“更新项目”（Project Renewal），该项目已更新多种肿瘤药物的说明书以反映最新科学认知。此外，2025年9月发布的“让我们的儿童重归健康”战略报告还指示 FDA 与美国国立卫生研究院（NIH）共同探索机会，“加强再利用药物在慢性疾病治疗中的应用，并通过协同临床试验设计协调审批流程，以推动获得FDA批准。”
公众提供的意见将有助于 FDA 完善其评估和促进药物再利用机会的方法，包括与 NIH 及医疗保险和医疗补助服务中心（CMS）等联邦合作伙伴的协作。
有意参与的各方可向该案卷提交电子或书面意见、数据和信息。电子提交可通过联邦法规制定门户网站（https://www.regulations.gov）完成。书面材料可邮寄或递交至：Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852。
所有提交材料均须注明案卷编号：FDA-2026-N-4492，主题为“Drug Repurposing for Unmet Medical Needs; Request for Information” 。
其他资源
Sincerely,
Sarah McMullen
Country Director
FDA China Office
Office of Global Operations
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