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What We Talk About When We Talk About Confirmatory Evidence

 
• By Bridget Silverman

Meaning of 'confirmatory evidence' becomes review issue in some of US FDA's most difficult choices: when a drug for a significant unmet medical need is supported by a single inadequate – but not failed – clinical trial. Neurology division director Dunn and review team leader Marler explain their interpretations in Ocrevus review memos.

Drug Review Profile regular column

FDA's decision to enshrine regulatory flexibility about the nature of "confirmatory evidence" in its landmark 1998 guidance on clinical evidence of efficacy opened paths to approval for drugs for serious unmet needs that lacked the regulatory gold standard of two adequate and well-controlled trials, but the lack of clear definitions also paved the way for multiple review disputes.

"What may constitute confirmatory evidence is left undefined in regulation (wisely so, in my estimation)," Division of Neurology Products Director Billy Dunn observed in his March 28, 2017 summary review...

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EMA Says Digital Transformation A ‘Fundamental Shift’ In Meeting Regulatory Challenges

 
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