FDA’s One-Pivotal-Trial Default: Scendea’s Perspective
Author:
Dr Dmitry Zamoryakhin
Principal Medical Consultant
Introduction
The US Food and Drug Administration’s (FDA’s) February 2026 announcement, made public in the New England Journal of Medicine (NEJM), outlines that one adequate and well-controlled pivotal study, together with confirmatory evidence, will now be the agency’s default basis for product approval. This differs from the FDA’s previous general expectation of two independent, adequate, and well-controlled pivotal trials to demonstrate substantial evidence of effectiveness. The FDA advises that the new requirement (of only one pivotal study) should not be interpreted as a lowering of the evidentiary bar, but instead, as a shift away from counting trials and towards judging the overall credibility of the evidence package.
Scendea considers the evidence developed by the sponsor (to demonstrate the drug’s reliability, durability, and safety) more imperative than the number of pivotal studies conducted. When a drug has a very high level of efficacy, is biologically plausible, and has a significant clinical impact, a single, well-designed study can often demonstrate all of those things sufficiently for regulatory approval. This is particularly true for rare or life-threatening diseases, where there are fewer patients to recruit and enrol, and the benefits of the drug may be much clearer…