FDA Rare Disease Innovation Hub Future Programming; Request for Comments

The Children’s Tumor Foundation (CTF) supports the FDA's Rare Disease Innovation Hub and requests that the agency include a workshop on "Unlocking Shelved Assets for Rare Disease" in its fall 2026 programming. The foundation argues for creating incentives and regulatory pathways to identify, transfer, and develop drug assets that have been de-prioritized or abandoned by pharmaceutical companies.

Dr. Richard Faris of PANTHERx Rare proposes a specific workshop topic for the RISE Fall Series focused on the role of specialty pharmacies in generating real-world evidence (RWE) for rare diseases. He argues that specialty pharmacies are uniquely positioned to provide longitudinal data on treatment effects, safety, and patient outcomes that can supplement clinical trials and support regulatory decision-making.

Emily's Entourage, an organization focused on cystic fibrosis research, supports the FDA's request for comments and proposes a specific workshop topic regarding preclinical data requirements for genetic therapies. They argue that establishing a standardized regulatory framework and master IND protocols would reduce the regulatory burden and accelerate the development of personalized treatments for patients with rare CFTR variants.

The National Ataxia Foundation (NAF), a nonprofit patient advocacy group, supports the FDA's Rare Disease Innovation Hub (RDIH) and the RISE workshop series. They propose a future workshop topic focused on enhancing and clarifying how patient input, specifically through patient-focused drug development (PFDD) and advisory committees, is integrated into the drug review process to ensure transparency and consistency.

Kyrsten Lawless, a parent and advocate for a child with alpha-mannosidosis, argues that the FDA should expand incentives and streamline pathways for AAV gene therapies and pharmacological chaperones. She highlights the clinical and economic burdens of current enzyme replacement therapies and advocates for more aggressive support for curative, one-time treatments for ultra-rare lysosomal storage disorders.

A law student interested in drug law supports the FDA Rare Disease Innovation Hub and its RISE Workshop series as a necessary initiative for modernizing rare disease regulation. The commenter argues that the Hub should focus on providing regulatory clarity, improving cross-center coordination, increasing transparency, and more directly incorporating patient input into the development process.

A law student studying food and drug regulation supports the FDA Rare Disease Innovation Hub's mission and suggests a specific focus for future workshops. They argue that the FDA should prioritize a workshop on real-world evidence (RWE) and natural history data to help navigate the challenges of conducting traditional clinical trials for small patient populations.

The commenter supports the FDA's Rare Disease Innovation Hub and suggests focusing the workshops on real-world clinical and operational challenges. They specifically advocate for addressing issues related to patient identification within EHR systems, care coordination, and expanding access to specialized care beyond major academic centers.

The commenter suggests that the FDA should use the RISE Workshop series to investigate equity and access issues in the diagnosis and treatment of rare diseases. They argue that geographical, financial, and awareness-based barriers create significant inequalities that regulatory science should help address.