The U.S. Food and Drug Administration (FDA) announced a new initiative to gather public input on drug repurposing efforts aimed at addressing unmet medical needs across various diseases and conditions. By identifying potential new indications or patient populations for already approved drugs, the FDA hopes to leverage existing safety data and scientific knowledge to speed up the availability of effective treatments.
FDA Commissioner Marty Makary, M.D., M.P.H., emphasized the importance of drug repurposing as a way to “make better use of available scientific data” to benefit patients who currently lack effective treatment options.
Public Input Sought on Priority Diseases and Candidates
The FDA is soliciting information from patients, clinicians, researchers, and other stakeholders on disease areas that should be prioritized for drug repurposing. The agency specifically mentioned metabolic diseases, neurodegenerative disorders, women’s and men’s health conditions, substance use disorders, and rare diseases as key areas of interest. The FDA also welcomes suggestions about other conditions with significant unmet medical needs.
Submissions can include suggestions of drugs already supported by sufficient evidence for new uses, candidates with preliminary clinical data (such as case studies or observational findings), and those with promising early preclinical results, possibly identified using technologies like artificial intelligence and machine learning. The FDA is particularly interested in proposals involving potential new uses that lack commercial incentives for drug manufacturers to pursue regulatory approval.
Addressing Barriers and Innovating Approaches
Alongside identifying repurposing candidates, the FDA is requesting input on innovative methods to detect repurposing opportunities and on barriers that hinder the development or labeling updates of repurposed drugs—even when supported by public scientific evidence.
This initiative builds on existing efforts, including the Best Pharmaceuticals for Children Act, the MODERN Labeling Act of 2020, and Project Renewal, which has updated oncology drug labels to reflect current scientific data. The agency also plans to strengthen collaboration with federal partners such as the National Institutes of Health (NIH) and the Centers for Medicare and Medicaid Services (CMS).
How to Submit Input
Stakeholders may submit electronic comments through the Federal eRulemaking Portal at regulations.gov or send written submissions to the FDA Dockets Management Staff in Rockville, Maryland. All submissions must reference docket number FDA-2026-N-4492, titled “Drug Repurposing for Unmet Medical Needs; Request for Information.”
Why it matters
Drug repurposing offers a cost-effective and timely approach to expand treatment options for diseases lacking FDA-approved therapies. Using existing drugs with known safety profiles can hasten approval processes and potentially provide patients with new, effective treatments sooner. This initiative reflects the FDA’s commitment to updating drug labeling based on emerging evidence and to addressing gaps where commercial incentives have limited drug development.
Background
Drug repurposing involves finding new therapeutic uses for existing drugs. Regulatory frameworks like the Best Pharmaceuticals for Children Act and initiatives such as Project Renewal have facilitated this practice by supporting the updating of drug labeling based on new evidence. The FDA’s current request for public input also aligns with the September 2025 Make Our Children Healthy Again strategy, which called for enhanced collaboration with NIH to explore repurposing drugs for chronic diseases through harmonized clinical trial designs.
Sources
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