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Abstract:
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Traditionally, phase II trials are relatively small and can be expected to result in a large degree of uncertainty in the estimates based on which Phase III trials are planned. Phase II trials are also to explore dose regimens with high probability of showing effectiveness, appropriate primary efficacy endpoint(s) or patient populations. When the biology of the disease and pathophysiology of disease progression are well understood, the phase II and phase III studies may be performed in the same patient population with the same primary endpoint and the selected dose. In disease areas where the clinical outcome endpoint may not be observed in a short-term study (e.g., mortality in cancer) or the molecular pathways may not be well established, the treatment effect may be measured through an intermediate surrogate endpoint in phase II trials.
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