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New FDA Guidance on the Use of Multiple Endpoints in Clinical Trials

The U.S. Food and Drug Administration (FDA) issued a new final guidance, Multiple Endpoints in Clinical Trials Guidance for Industry. 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.” The FDA guidance describes strategies for ordering and analyzing endpoints to reduce potential erroneous or misleading conclusions about a drug’s effects. This guidance begins with background concepts like demonstrating effectiveness, type I error, and multiplicity and goes on to detail the general principles of multiple endpoints including:

  • The Hierarchy of Families of Endpoints
  • Type II Error Rate and Sample Size
  • Types of Multiple Endpoints
  • The Individual Components of Composite and MultiComponent Endpoints

View the FDA guidance snapshot for an overview and to listen to the FDA guidance recap podcast to hear highlights from FDA staff.


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Multiple Endpoints in Clinical Trials: Guidance for Industry Guidance Snapshot