FDA
Got an off-label win? FDA wants to hear about it
Clinical Takeaway: Clinicians and researchers can nominate off-label uses backed by published evidence. FDA flagged metabolic disease, neurodegenerative conditions, women's and men's health, substance use disorders, and rare diseases as priorities.
Off-label prescribing fills real clinical gaps but leaves patients, payers, and prescribers without the certainty that comes from formal labeling. A new FDA request for information is aimed squarely at that mismatch. It targets cases where scientific evidence may already support a new indication or population, but where no manufacturer has sufficient financial incentive to pursue approval.
The agency is asking stakeholders to submit candidates in three categories: drugs where sufficient evidence may already support a new use, drugs with promising preliminary clinical data such as case series or observational studies, and drugs with promising preclinical data, including signals from artificial intelligence and machine-learning tools. FDA is also seeking input on barriers that limit development or adoption of repurposed drugs when commercial incentives are absent.
Priority disease areas named in the announcement include metabolic disease, neurodegenerative conditions, women's and men's health, substance use disorders, and rare diseases, with stakeholders invited to flag additional areas. The agency framed the initiative as a way to make better use of existing safety and efficacy data rather than starting from scratch with novel compounds.
The practical implication of this initiative could be twofold. Submitting strong evidence on widely used off-label therapies could accelerate formal label updates, which in turn affect insurance coverage, prescribing guidance, and legal liability. The harder question is whether the docket will translate into actual labeling changes, given that the underlying barrier, namely the lack of a sponsor with financial reason to file, is not solved by gathering evidence alone.
"Too many patients lack effective treatment options, even when promising science exists," said FDA Commissioner Marty Makary, MD, MPH. "Drug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need."
Comments may be submitted at regulations.gov under docket number FDA-2026-N-4492.
Source: U.S. Food and Drug Administration. 2026 May 11. FDA Advances Drug Repurposing to Address Unmet Medical Needs