FDA seeks public input on repurposing approved drugs for new uses

The Food and Drug Administration is asking scientists, doctors, patients and other stakeholders to help identify approved medicines that could be redeployed for new diseases, a move that could speed access to lower-cost treatments in areas where patients still have few options. The agency said it was especially focused on chronic and rare diseases, including metabolic diseases, neurodegenerative conditions, women’s and men’s health conditions, substance use disorders and rare diseases.

FDA Commissioner Marty Makary said the agency was responding to a basic problem in medicine: too many patients still lack effective treatment even when promising science already exists. The new effort would look for new indications and new patient populations, and for drugs that may deserve a second life in a different clinical setting. It would also seek examples of candidates with sufficient evidence, promising early clinical data and even promising preclinical findings, including work generated with artificial intelligence and machine learning.

Drug repurposing can shorten the path to patients because much of the groundwork is already known. Researchers do not start from zero on safety, dosing and manufacturing, which can cut years from development and lower costs. A Duke Health Policy working paper said de novo drug development can cost as much as $2.8 billion and take eight to 10 years. The same paper pointed to dexamethasone for hospitalized COVID-19 patients, thalidomide for multiple myeloma and colchicine for cardiovascular disease as examples of repurposing that found real clinical value.

The FDA’s pitch is not that any promising signal should become a new label overnight. The agency said the effort is part of a broader push to update labeling only when there is sufficient evidence, and it wants to hear about barriers that discourage companies from pursuing those changes when the medicine is off patent or there is little commercial payoff. In practice, that means the fastest wins are likely to come where there is already a strong evidence base, not just a lab hypothesis. New uses would still need enough clinical data to show the drug helps in the new condition and to define who should get it and at what dose.

The initiative builds on older FDA efforts, including the Best Pharmaceuticals for Children Act, the MODERN Labeling Act of 2020 and Project Renewal, an Oncology Center of Excellence program that has updated labeling for older cancer drugs and is now being extended to other disease areas. It also aligns with federal research efforts at the National Institutes of Health and the Advanced Research Projects Agency for Health, including NIH work on Alzheimer’s and related dementias and ARPA-H’s MATRIX project for rare diseases.

The policy stakes are especially high in rare disease care, where a May 2025 JAMA Network Open commentary said many conditions still have no FDA-approved therapy and cited nonprofit-led work on 94 potential treatments that had a reported success rate of 24.5 percent, or 23 of 94. If the FDA can turn existing evidence into new labels faster, the payoff could be lower prices, quicker access and fewer dead ends for patients who have been waiting longest.