Comment from Anonymous

Equity and access in the diagnosis and treatment of rare diseases is a significant cross-cutting topic that the FDA might investigate through the RISE Workshop series. The development of treatments for uncommon diseases has advanced significantly, yet access to diagnosis and therapy is still quite unequal. Due to geographical obstacles accessing specialized care, a lack of screening technologies, or low provider awareness, many patients face diagnostic delays. Cost, insurance coverage restrictions, or availability at specialist centers may limit access to approved therapy even after diagnosis. This problem poses significant regulatory and policy issues and impacts a variety of uncommon diseases. A workshop might look at how regulatory science can better explain access discrepancies. Addressing equity and access challenges is critical to ensuring that advances in rare disease treatment benefit all patients, not just those with the resources or proximity to receive specialized care.