Millions of patients with chronic and rare diseases could get faster access to treatments under a new FDA push to repurpose already-approved drugs for new uses. The agency is asking the public, researchers, and clinicians to help identify the best candidates.

The idea is straightforward: drugs already proven safe for one condition can skip much of the early testing, potentially delivering new treatment options in years instead of a decade or more. The FDA is particularly focused on diseases where commercial incentives for traditional drug development fall short.

“Too many patients lack effective treatment options, even when promising science exists,” said FDA Commissioner Marty Makary. “Drug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need.”

The agency is seeking input on three tiers of candidates: drugs where enough evidence may already exist to support a new use; drugs with promising preliminary clinical data, such as case reports or observational studies, that warrant further study; and drugs with preclinical data from emerging tools like AI and machine learning that merit investigation.

Priority areas include metabolic diseases, neurodegenerative conditions, women’s and men’s health, substance use disorders, and rare diseases. The effort builds on existing programs including the Best Pharmaceuticals for Children Act, the MODERN Labeling Act of 2020, and Project Renewal, which has already updated labeling for several oncology drugs.

The September 2025 Make Our Children Healthy Again strategy report directed the FDA to explore repurposing opportunities jointly with the National Institutes of Health.

Source: FDA Press Release