ABSTRACT

In the sequential parallel comparison design (SPCD) in stage 1, placebo subjects are randomized between placebo and an experimental therapy. In stage 2, stage 1 placebo non-responders are re-randomized between placebo and an experimental therapy. We give the formula for power/sample size calculations for two test statistics for the SPCD. The first one omits the correlation between the estimated treatment effects from the two stages, and the second one does not. We discuss how to construct a confidence interval for the weighted average of the treatment effects that has proper coverage.

中文翻译：

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具有连续结果的顺序并行比较设计的功效计算

摘要

在第 1 阶段的顺序平行比较设计 (SPCD) 中，安慰剂受试者被随机分配在安慰剂和实验疗法之间。在第 2 阶段，第 1 阶段安慰剂无反应者在安慰剂和实验疗法之间重新随机分配。我们给出了 SPCD 的两个检验统计量的功效/样本量计算公式。第一个忽略了两个阶段估计的治疗效果之间的相关性，第二个没有。我们讨论如何为具有适当覆盖范围的治疗效果的加权平均值构建置信区间。