Drug Repurposing for Unmet Medical Needs; Request for Information

Anna, a patient and advocate for rare ovarian and endometrial cancers, supports the FDA's drug repurposing initiative. She specifically urges the FDA to consider Anktiva as a high-priority candidate for repurposing to treat rare cancers with limited treatment options.

The Cerebral Palsy Foundation (CPF) supports the FDA's Request for Information on drug repurposing and urges the agency to include cerebral palsy as a priority disease area. They recommend that the FDA support regulatory approaches for pediatric neurodevelopmental disorders and partner with disease-specific foundations to accelerate therapeutic development.

The Lupus Foundation of America supports the FDA's initiative to identify opportunities for drug repurposing, particularly for diseases with significant unmet needs and limited commercial incentives. They advocate for streamlined regulatory pathways that incorporate real-world evidence, patient registries, and collaborative public-good models to expand treatment options for lupus patients.

Highlander Health expresses enthusiastic support for the FDA's efforts to advance drug repurposing and suggests adding liver disease as a priority area. They advocate for leveraging continuous real-world data (RWD), AI-based tools, and "clean room" analysis to identify repurposing candidates and streamline the regulatory process.

Diagnostic Green LLC, representing Renew Pharmaceuticals Ltd., supports the FDA's initiative to solicit input on drug repurposing. They specifically advocate for the repurposing of Indocyanine Green (ICG) for various surgical and diagnostic uses, citing its long safety record and the lack of commercial incentives for the drug sponsor to pursue new labeling.

The American Pharmacists Association (APhA) supports the FDA's drug repurposing efforts to accelerate patient access to therapies and suggests adding epinephrine and transplant drugs as priority areas. They urge the FDA to prioritize patient safety, maximize transparency, include pharmacists in discussions, and work with other agencies to ensure reimbursement for pharmacist-provided services.

The American Society of Transplantation supports the FDA's efforts to identify mechanisms for drug repurposing, particularly for solid organ transplant recipients. They argue that many evidence-supported therapies are currently used off-label due to small patient populations and limited commercial incentives, creating administrative burdens and potential delays in patient care.

Maxime Taquet, representing researchers from the University of Oxford and other institutions, submits evidence suggesting that the recombinant shingles vaccine is associated with a 17% relative reduction in dementia burden. They advocate for the FDA to formally evaluate this candidate for repurposing and encourage the use of natural-experiment designs on large real-world datasets to identify such candidates.

Michael B. Zemel, Chief Scientific Officer of NuSirt Sciences, Inc., submits information regarding TRIPLN(TM), a fixed-dose combination of leucine, metformin, and sildenafil, as a candidate for drug repurposing in cardiometabolic diseases. He argues that while TRIPLN(TM) is a novel combination rather than a single drug gaining a new use, it aligns with the FDA's interest in repurposing established agents and highlights specific regulatory and commercial barriers facing such programs.

The commenter supports the request for information by arguing that the current FDA regulatory framework is insufficient for drug repurposing and needs reform. They suggest specific improvements, including reducing evidentiary requirements for supplemental new drug applications (SNDAs), utilizing real-world data, and advocating for legislative changes to prioritize healthcare spending over military spending.