FDA emphasizes study quality in shift to one-trial default for drug approval

By Amanda Conti and Ned Pagliarulo

FDA leadership has announced that one adequate and well-controlled trial, supported by confirmatory evidence, will now serve as the agency’s “default standard” for approval of novel drugs. The policy shift, articulated in a New England Journal of Medicine article, formalizes a trend the agency has increasingly adopted in practice.

The move places new emphasis on study quality, control selection, statistical methodology, and postmarket data collection — while raising questions about implementation, consistency across review divisions, and how sponsors currently in development should respond.

This AgencyIQ analysis examines the regulatory, evidentiary, and strategic implications of the new one-trial default and what it could mean for drug sponsors in the near and long term.

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