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.

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.

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.

The commenter is a parent of a child with Alpha Mannosidosis, an ultra-rare lysosomal storage disorder, who argues that the FDA should loosen regulations on gene therapy for rare genetic diseases. She specifically requests a pathway for individualized gene therapy using a patient's own stem cells and advocates for broader clinical trials that cover multiple disorders rather than single specific diseases.

The commenter is a mother of a child with a rare lysosomal storage disorder who advocates for a broader regulatory approach to gene therapy. She requests that the FDA consider approving gene therapies for entire classes of diseases rather than individual disorders and asks for a pathway for individualized gene therapy using a patient's own stem cells.