A Senate Aging Committee hearing and related advocacy sessions put the FDA’s rare‑disease review process under scrutiny, with patient groups and clinicians pressing for consistency and clearer pathways. Witnesses cited cases where FDA decisions reversed earlier agency interactions, and rare‑disease advocates described prolonged delays and what they called a lack of engagement from agency leadership. Physicians and biotech executives testified about specific program setbacks and requested clearer guidance on real‑world evidence and external control use. The session included calls for improved communication channels between sponsors, clinicians and regulators, and prompted renewed industry debate on evidentiary standards for small‑population medicines.