论文标题

多数结果试验,有大量有效结果

Multi-outcome trials with a generalised number of efficacious outcomes

论文作者

Law, Martin, Grayling, Michael J., Mander, Adrian P.

论文摘要

现有的多结果设计几乎完全集中于评估所有结果是否显示出疗效的证据,或者至少一个结果是否显示出疗效的证据。尽管少数作者提供了多种结果设计,以评估一般数量的结果表现出希望,但这些设计本质上仅是单阶段的。因此,我们提出了两个在多阶段设置中提供此设计特征的失败者形式的两个设计和删除失败者形式的设计。以前的这种多结果多阶段设计仅允许最多两个结果。因此,我们的设计也通过允许任何数量的结果来扩展以前的相关建议。

Existing multi-outcome designs focus almost entirely on evaluating whether all outcomes show evidence of efficacy or whether at least one outcome shows evidence of efficacy. While a small number of authors have provided multi-outcome designs that evaluate when a general number of outcomes show promise, these designs have been single-stage in nature only. We therefore propose two designs, of group-sequential and drop the loser form, that provide this design characteristic in a multi-stage setting. Previous such multi-outcome multi-stage designs have allowed only for a maximum of two outcomes; our designs thus also extend previous related proposals by permitting any number of outcomes.

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