Case Study: Acceptatory Phase III Trials

Design considerations are critical for confirmatory Phase III trials because they form a large part of the basis for regulatory approval and potential changes to clinical practice guidelines. For this reason, confirmatory trials should be adequate and well controlled, typically with placebo or a standard therapy as a control and a clinical benefit endpoint, or a validated surrogate as primary endpoint for full approval. For accelerated or conditional approval “likely surrogate” endpoints may be used.