Drug Repurposing for Unmet Medical Needs; Request for Information

The Children's Tumor Foundation (CTF) supports the FDA's drug repurposing initiative and recommends specific high-priority opportunities for treating neurofibromatosis. They advocate for regulatory facilitation to encourage pharmaceutical companies to pursue label expansions for drugs that show clinical benefit but lack commercial incentives for rare diseases.

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.

An individual is sharing a personal anecdote regarding the positive effects of the drug PEMGARDA on a loved one's long COVID symptoms. They are requesting that the FDA consider approving PEMGARDA for general use and as a treatment for long COVID.

A parent of a child with autism advocates for designating ASD as a priority area for drug repurposing. The commenter requests that the FDA pursue label expansion for existing drugs, create financial incentives for repurposing trials, and provide guidance on evidence for treatment-responsive subgroups.

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.

Knoa Pharma LLC supports the FDA's drug repurposing initiative, specifically advocating for the inclusion of substance use disorders (SUDs) as a priority disease area. They propose three specific candidates for repurposing: varenicline for nicotine dependence, prescription stimulants for stimulant use disorders, and nalmefene for alcohol use disorder.

AmoSey Inc., a Canadian life sciences advisory firm, supports the FDA's initiative on drug repurposing and provides specific recommendations on candidate selection and overcoming commercial incentive barriers. They advocate for prioritizing candidates with shared biological mechanisms, utilizing human genetic evidence over pure AI outputs, and creating pathways for third-party-originated proposals to find sponsors.

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.