RE: FDA draft guidance on Substantial Evidence of Effectiveness for Human Drug and Biological Products

The Center for Science in the Public Interest respectfully submits these comments regarding the Food and Drug Administration’s draft guidance titled “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products.”

We appreciate the agency issuing a draft guidance to clarify, in part, how drug developers “can rely on one scientifically rigorous adequate and well-controlled clinical investigation with confirmatory evidence to satisfy the statutory substantial evidence of effectiveness standard.”

The new draft guidance explains that the substantial evidence standard can be met either with one adequate and well-controlled trial plus confirmatory evidence or with more than one adequate and well-controlled trial. The draft guidance then lays out two ways of meeting approval standards with only a single trial: 1) “evidence from an adequate and well-controlled trial plus a source of strong confirmatory evidence,” and 2) “evidence from a highly persuasive adequate and well-controlled trial plus early-phase confirmatory evidence.”

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