The US Food and Drug Administration (FDA) is considering a new framework that could allow approved medicines to be repurposed for additional indications, particularly in disease areas with limited commercial incentives.
The FDA is seeking industry and public opinion on the initiative, which it hopes will accelerate availability of treatments by using pre-existing knowledge or promising upcoming data about the drug’s efficacy and safety.
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A main goal of the repurposing drive is to expand treatment options in disease areas with significant unmet need. In an initial outline, the agency highlighted metabolic diseases, neurodegenerative conditions, and health conditions that affect men and women differently as areas that could benefit most. The agency also identified substance use disorders and rare diseases as other areas it wants to prioritise.
Recent research from the Université Libre de Bruxelles highlighted the value of drug repurposing in pharmaceutical innovation. Researchers found that the average time between a drug’s first approval and its repurposed use is approximately seven years – considerably shorter than the typical development timelines for novel therapies.
A central objective of the FDA initiative is address commercial barriers that often limit investment in underserved disease categories. Rare diseases, for example, frequently affect small patient populations, making it difficult for pharma companies to recover research and development costs. According to the FDA, the availability of scientific data could support a potential new use of an approved treatment.
As such, the FDA is on the look out for candidates that fulfil this criterion, along with those that have promising preliminary clinical or preclinical data. Case reports, observational studies, and AI modelling are all potential data mediums that could hint at a new use case for a drug.
FDA commissioner Marty Makary said: “Too many patients lack effective treatment options, even when promising science exists. Drug repurposing can make better use of available scientific data to deliver effective treatment options for patients in need.”
FDA targets improved drug labelling
Makary has been a supporter of restructuring how clinical data is utilised for approved therapies. Reducing regulatory fragmentation was a key aim laid out in the agency’s fiscal year 2027 budget proposal. It was also noteworthy that the document contained a push for the rare paediatric disease priority review voucher (PRV) programme to become permanent, reflecting the agency’s priorities towards areas that have little commercial incentives.
However, drug repurposing could be one of the final initiatives overseen by Makary, after the Wall Street Journal reported that US President Donald Trump plans to dismiss the commissioner. Neither the FDA nor the White House has issued a statement.
The FDA already enacts on several initiatives that make use of drug label updating, such as Best Pharmaceuticals for Children Act and the Making Objective Drug Evidence Revisions for New (MODERN) Labeling Act of 2020. However, the current docket will help define a cohesive pathway for the agency to lean on when expanding use cases of drugs.
This request for public input on repurposing is part of a broader FDA drive on improving up-to-date labelling of approved drugs. Earlier this week, the agency debuted final industry guidance on collecting post-marketing safety data for both drugs and biologics in pregnant individuals. The framework aims to create pregnancy safety information via post-marketing data collection that can be added to labels.
