A Senate Aging Committee hearing and patient‑advocate protests this week put FDA rare‑disease review practices under scrutiny, as witnesses and patient groups described inconsistent regulatory decisions and long delays that threaten access. Clinicians and advocates testified that the agency’s rejections and shifting standards for some cell and gene therapies have left families and trial participants frustrated and uncertain. The sessions named specific programs and called for clearer, more predictable pathways for accelerated approvals and real‑world evidence integration.