FDA Adopts Single-Trial Default for Drug Approvals

The Food and Drug Administration announced plans to adopt a single clinical trial, supported by confirmatory evidence, as the default standard for approving new drugs and novel health products, a shift aimed at accelerating access to treatments amid advances in scientific research.

FDA Commissioner Dr. Marty Makary and his top deputy, Dr. Vinay Prasad, outlined the change in a commentary published Wednesday in the New England Journal of Medicine. The policy replaces the agency's longstanding requirement of two rigorous studies, which had been in place to verify results and ensure reproducibility. Officials stated that modern tools, including deeper biological understanding and alternative validation methods, now provide sufficient assurance of a product's effectiveness and safety without needing a second trial in most cases.

The move builds on flexibility the FDA has applied for decades, particularly for treatments of rare or life-threatening conditions like cancer, where single-trial approvals became common starting in the 1990s. Congressional laws encouraged this approach for hard-to-treat diseases, where enrolling large patient groups is challenging. In the past five years,...