The U.S. Food and Drug Administration (FDA) is seeking public input on drug repurposing initiatives aimed at addressing unmet medical needs across various diseases and conditions.
The process of identifying new uses—whether for a different indication or a new patient population—can significantly enhance treatment accessibility by leveraging existing data on FDA-approved drugs, including established safety profiles. This call for public input is part of a wider FDA initiative to revise the labeling of approved medications, ensuring that the information provided is clinically relevant and scientifically current for healthcare providers and patients alike.
According to the agency, drug repurposing can optimize the use of existing scientific data to deliver effective treatment options for patients. The FDA is particularly interested in gathering insights from patients, clinicians, researchers, and other stakeholders regarding priority disease areas and potential candidates for repurposing. This focus targets areas where substantial scientific data might support the approval of new drug uses but where there is limited financial motivation to pursue such approvals.
Areas of particular interest include metabolic diseases, neurodegenerative disorders, conditions affecting women’s and men’s health, substance use disorders, and rare diseases. The FDA encourages stakeholders to propose additional areas that merit priority attention.
The FDA is looking for information on candidates for repurposing where there is already sufficient evidence to support a potential new use, as well as candidates that show promise through preliminary clinical data—such as from case reports, case series, and observational studies—or through early preclinical data, including insights generated by emerging technologies such as artificial intelligence and machine learning.
Furthermore, the agency is soliciting feedback on innovative methods for identifying opportunities for drug repurposing, as well as barriers that may hinder the development or utilization of repurposed drugs, especially in instances where there is minimal or no commercial incentive to pursue labeling changes backed by publicly accessible scientific evidence.
(Source: U.S. FDA)
Published on May 18, 2026.
