FDA Guide on Multiple Endpoints in Clinical Trials

FDA has released a draft guidance on “Multiple Endpoints in Clinical Trials” that discusses the problems posed by multiple endpoints in the analysis and interpretation of drug/biologic study results and how these problems can be managed in clinical trials. Most drug clinical trials contain multiple endpoints to assess the effects of the drug and to document a product’s ability to favorably affect one or more disease characteristics, the document notes. “As the number of endpoints analyzed in a single trial increases, the likelihood of making false conclusions about a drug’s effects with respect to one or more of those endpoints becomes a concern if there is not appropriate adjustment for multiplicity,” it says. The guidance describes various strategies for grouping and ordering endpoints for analysis and applying statistical methods for managing multiplicity within a study in order to control the chance of making erroneous conclusions about a drug’s effects.