The U.S. Food and Drug Administration signaled a policy shift by endorsing approval based on a single well‑controlled trial supplemented with confirmatory evidence, authors at the agency argued in a recent New England Journal of Medicine piece. The change abandons the historical ‘two‑trial’ expectation and leans on modern analytics and trial design to establish efficacy. FDA leaders framed the move as a way to reduce development costs and accelerate patient access without lowering evidentiary standards, while industry groups urged clearer implementation guidance. Observers say the shift could shorten pivotal development timelines and alter program design, especially for indications where large, duplicate pivotal trials are impractical or costly. Regulators and sponsors will need to clarify how surrogate endpoints, advanced statistics, and external controls can complement a single pivotal study under the new stance.