Abstract:
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In the clinical phase of cancer drug development, open-label, single-arm Phase II clinical trials are conducted to evaluate safety and to assess whether a new agent or procedure shows sufficient efficacy to warrant further studies in the form of large, randomized Phase III clinical trials, i.e., to assess futility. In other clinical areas, this concept of Phase II clinical trials are still relatively novel and under-utilized. Often, they are treated as pilot studies or become under-powered Phase III clinical trials, if conducted at all. Although not all disease areas are amendable to such a design, to illustrate the value of Phase II studies in stroke, we analyzed data from a number of completed Phase III studies as if single-arm, multi-stage Phase II studies were conducted. The results show that for some of these studies, the lack of treatment effect could have been discovered prior to launching a large randomized study, and thereby preventing unnecessary use of resources as well a minimizing exposure of patients to ineffective treatments. We also show that a successful Phase II study (i.e., non-futility) does not guarantee a positive efficacy outcome in Phase III.
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