The US Food and Drug Administration has sought inputs on 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 expand the availability of treatments by using existing knowledge about the drugs, including their safety profile. This request for public input is part of a broader FDA initiative to update the labelling of FDA-approved drugs, when supported by sufficient evidence, to ensure that the information in the labelling is clinically meaningful for healthcare providers and patients, and scientifically up to date.

The regulator said that 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 is scientific data that could support approval of potential new uses but limited commercial incentive to pursue approval for those uses.

The agency is particularly interested in 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 prioritised.

The FDA is calling for information on candidates for drug repurposing where sufficient evidence may already exist to support a potential new use; candidates with promising preliminary clinical data (for instance, data from case reports, case series, and observational studies) that may warrant further study; and candidates with promising preliminary preclinical data (for example, 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 labelling changes, supported by publicly available scientific evidence.

(Source: USFDA)

More Like This

Published on May 18, 2026