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Uniformly minimum variance conditionally unbiased estimation in multi-arm multi-stage clinical trials

机译:多臂多阶段临床试验中的一致最小方差有条件无偏估计

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摘要

Multi-arm multi-stage clinical trials compare several experimental treatments with a control treatment, with poorly performing treatments dropped at interim analyses. This leads to inferential challenges, including the construction of unbiased treatment effect estimators. A number of estimators unbiased conditional on treatment selection have been proposed, but are specific to certain selection\udrules, may ignore the comparison to the control and are not all minimum variance. We obtain estimators for treatment effects compared to the control that are uniformly minimum variance unbiased conditional on selection with any specified rule or stopping for futility.
机译:多臂多阶段临床试验将几种实验治疗方法与对照治疗方法进行了比较,在中期分析中,表现不佳的治疗方法被取消。这导致了推论性挑战,包括构建无偏治疗效果估计量。已经提出了许多不偏倚于治疗选择的估计量,但是这些估计量特定于某些选择\规则,可能会忽略与对照的比较,并且并非都是最小方差。我们获得了与对照相比的治疗效果估算值,这些估算值是一致的最小方差,无条件的条件是选择任何指定规则或因徒劳而停止。

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