FDA Rare Disease Innovation Hub Future Programming; Request for Comments

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 commenter, a caregiver for a child with an ultra-rare disease, proposes a new clinical trial framework for ultra-rare diseases that focuses on individual "N-of-1" trajectories rather than group averages. They argue that because ultra-rare diseases are highly heterogeneous, trials should measure meaningful deviations from a patient's own baseline using real-world data and personalized outcomes.