Abstract:
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In Phase 2b studies, it is of interest to investigate possible dose-response relationships to identify the minimum effective dose (MED) to be used in confirmatory studies. In traditional dose response studies, pairwise comparisons between placebo and each investigational dose is used to claim trial success and to make dose recommendations. Recently, a new dose ranging study design and analysis method was proposed based on a novel model-based approach--Multiple Comparison Procedures & Modeling (MCP-Mod), which detects multiple general dose response trends accounting for model uncertainty. With the same design power, MCP-Mod generally requires a smaller sample size per arm than the study design based on pairwise comparison, thus allowing for more study arms (more dose levels) given a fixed total sample size. Via simulation, we compare the performance of identifying the MED and other operating characteristics using design/analysis approaches based on pairwise comparison versus MCP-Mod under similar total study size. Furthermore, advantages and disadvantages of the different approaches will be discussed.
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