FDA Issues Final Guidance on Clinical Trials with Multiple Endpoints

Last week, the US Food and Drug Administration (FDA) released its final guidance on multiple endpoints in clinical trials. The goal of the guidance is to provide standardized strategies for clinical trials with multiple endpoints to avoid ambiguity in study findings that might have bearing on the regulatory approval process and patient outcomes.

According to the guidance, “Making a false positive conclusion about effectiveness (i.e., falsely concluding that a drug has a positive treatment effect when it does not) is a major concern. A common approach is to control the Type I error rate at less than 5% (1 in 20 chance) for a false conclusion that there is a treatment difference or 2.5% (1 in 40 chance) for a false positive conclusion about effectiveness. As the number of endpoints or analyses increases, the Type I error rate can increase well beyond 2.5% due to multiplicity.”

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