The FDA aims to save money on approved drugs. Through the newly proposed reuse initiative, the agency hopes to help address unmet medical needs by discovering new uses for previously approved medicines.
New uses for previously approved drugs, whether for new indications or new eligible patient populations, will leverage existing safety data to accelerate treatment availability, the FDA explained in a May 11 release.
“Despite promising science, too many patients lack effective treatment options,” explained outgoing FDA Commissioner Marty McCulley, M.D., in a statement. “Drug repurposing allows us to better leverage available scientific data to provide effective treatment options to patients in need.”
To advance this effort, the agency is asking patients, clinicians, and other stakeholders to provide input on priority disease areas and potential repurposing drug candidates. The disclosure document, published in the Federal Register on Tuesday, requests comments by June 11, and specifically requests proposals for FDA-approved drugs for which “there appears to be no commercial benefit in adding new uses” through a standard supplemental new drug application (sNDA).
The request is part of “a broader FDA effort to update the labels of FDA-approved drugs when supported by sufficient evidence to ensure that the information on the labels is clinically meaningful to health care providers and patients and scientifically up-to-date,” the agency said in a statement.
U.S. health officials have recently launched several moves pointing to broader labeling efforts. Related frameworks cited by the FDA in the document include Project Renewal around oncology labeling and the Make America Healthy Again (MAHA) committee’s September proposal for repurposing drugs to treat chronic conditions.
One of the most notable examples of the FDA changing drug labels outside of the standard approval pathway unfolded late last year through President Robert F. Kennedy’s efforts to address the “autism epidemic.” To offer a potential treatment for the “autistic features” of cerebral folate deficiency (CFD), the FDA has reinstated GSK’s Welcovorin (leucovorin calcium), a decades-old drug that GSK discontinued in the 1990s.
The FDA reapproved the drug in October after reviewing its potential as an autism treatment based on real-world findings. Five months later, the agency formally approved a label change that allows Welcovorin to treat CFD in patients with identified genetic mutations. Rather than basing its approval on clinical trials, the FDA bases its decisions on published literature and known data about a drug’s mechanism.
This approach is described in the document as one of the few approaches used by the FDA to support label updates for approved drugs.
The agency is currently looking for more approved drugs to make label changes, but notes that any drug that could meet the criteria must already have safety and efficacy data. However, the FDA explained that it welcomes suggestions for other drugs without such evidence, as they may lead to “promising areas for further research.”
Currently, FDA’s priority areas for this new initiative are metabolic diseases, neurodegenerative diseases, women’s and men’s health, substance use disorders, and rare diseases. Information from the request for public comment will “help inform how FDA refines its approach to evaluating and promoting additional drug repurposing opportunities, including collaboration with federal partners such as the National Institutes of Health and the Centers for Medicare and Medicaid Services,” the department said.
Monday’s announcement was co-signed by McCurry, but the former chief will no longer be in control after his resignation on Tuesday.
His resignation follows a tumultuous 14 months at the top of the FDA, during which he drew criticism for several decisions, including his positions and actions on the abortion drug mifepristone, fruit-flavored e-cigarettes, and the commissioner’s national expedited review voucher pilot program.
