A review of phase 2–3 clinical trial designs
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This article reviews phase 2–3 clinical trial designs, including their genesis and the potential role of such designs in treatment evaluation. The paper begins with a discussion of the many scientific flaws in the conventional phase 2 → phase 3 treatment evaluation process that motivate phase 2–3 designs. This is followed by descriptions of some particular phase 2–3 designs that have been proposed, including two-stage designs to evaluate one experimental treatment, a design that accommodates both frontline and salvage therapy in oncology, two-stage select-and-test designs that evaluate several experimental treatments, dose-ranging designs, and a seamless phase 2–3 design based on both early response-toxicity outcomes and later event times. A general conclusion is that, in many circumstances, a properly designed phase 2–3 trial utilizes resources much more efficiently and provides much more reliable inferences than conventional methods.
KeywordsAdaptive design Clinical trial Design Phase II clinical trial Phase III clinical trial Selection
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