A high‑profile Senate hearing and related public testimony put the FDA’s rare‑disease review record under scrutiny. Clinicians, patient advocates and company leaders testified that inconsistent communications and shifting evidentiary expectations have delayed or derailed approvals for therapies serving small, high‑need populations. Witnesses described interactions with regulators as opaque and sometimes stalled, citing cases such as Biohaven’s troriluzole and other recent rejections. At the same hearing and in media interviews, FDA Commissioner Marty Makary publicly defended the agency’s decisions, arguing the agency must hold products to evidence standards and pointing to failed trials as justification for rejections. The debate highlights tension between patient groups seeking expedited access and regulators emphasizing data robustness and safety. The dispute is likely to influence industry regulatory strategy, with sponsors weighing accelerated pathways against the risk of late‑stage refusals and the need for clearer, consistent agency guidance.