Drug Repurposing for Unmet Medical Needs; Request for Information

The Main Street Foundation's Center for Regulatory Analysis and Engagement (CRAE) supports the FDA's efforts to identify evidence-based drug repurposing opportunities within existing statutory frameworks. They argue for strengthening administrative processes through transparency, objective criteria, and technological modernization (like AI) while maintaining rigorous scientific standards.

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.

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 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.

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.

A parent of a child with autism expresses frustration with the current limitations of psychiatric medications and advocates for the exploration of existing drugs for repurposing. The commenter supports the action of investigating new drug repurposing options to address unmet medical needs.

Novartis Pharmaceuticals Corporation submitted comments providing a specific example of a drug candidate (clofazimine) for repurposing to address unmet medical needs. They highlight the barriers to formal approval for clofazimine despite existing scientific support and real-world use for various infections.

Dr. William E. Fitzsimmons from the University of Illinois at Chicago supports the drug repurposing initiative, specifically advocating for the inclusion of solid organ transplant immunosuppression as a priority area. He identifies several specific drugs for repurposing (such as Basiliximab and Mycophenolate mofetil) and suggests waiving user fees to overcome commercial barriers.

Reboot Rx, a nonprofit organization, supports the FDA's drug repurposing efforts and recommends prioritizing oncology as a key disease area. They provide specific drug candidates for evaluation and propose creating a "labeling-only" 505(b)(2) pathway to allow nonprofits to independently seek FDA approval for repurposed generic drugs.