FDA offers more guidance on relying on just one trial and confirmatory evidence for a new drug approval
Although FDA isn’t changing what it requires for a new drug to win approval (that hasn’t changed since 1998), the agency on Monday released new draft guidance to better describe how one adequate and well-controlled clinical investigation and confirmatory evidence — including real-world evidence and even expanded access data — can be used to meet the agency’s substantial evidence requirement.
Building off prior guidance from 1998 and 2019, the agency says that the quantity of confirmatory evidence needed in any given development program seeking to go this route “will be impacted by the features of, and results from, the single adequate and well-controlled clinical investigation that the confirmatory evidence is intended to substantiate,” according to a Federal Register notice published Monday.
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