The attributable estimand: A new approach to account for intercurrent events
The attributable estimand: A new approach to account for intercurrent events
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DOI：
10.1002/pst.2019
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发表时间：
2020-03-21
影响因子：
1.5
通讯作者：
Wright, David
中科院分区：
文献类型：
--
作者：
Darken, Patrick;Nyberg, Jack;Wright, David
“并发事件”一词最近被用来描述临床试验中可能使治疗效果估计值的定义和计算复杂化的事件。本文重点讨论使用可归因估计来解决并发事件。那些被认为与随机治疗不利相关的事件（例如，由于不良事件或缺乏疗效而停药）被认为是可归因的，并使用复合估计策略进行处理，而假设估计策略则用于不被认为与随机治疗相关的并发事件（例如，不相关的不良事件）。我们探索了如何实施这种方法的几种选择，并通过一系列模拟研究将它们与假设的“疗效”和治疗政策估计策略进行比较，这些模拟研究的设计受到最近慢性阻塞性肺疾病（COPD）试验的启发，并通过对最近完成的 COPD 试验的分析进行说明。
The term "intercurrent events" has recently been used to describe events in clinical trials that may complicate the definition and calculation of the treatment effect estimand. This paper focuses on the use of an attributable estimand to address intercurrent events. Those events that are considered to be adversely related to randomized treatment (eg, discontinuation due to adverse events or lack of efficacy) are considered attributable and handled with a composite estimand strategy, while a hypothetical estimand strategy is used for intercurrent events not considered to be related to randomized treatment (eg, unrelated adverse events). We explore several options for how to implement this approach and compare them to hypothetical "efficacy" and treatment policy estimand strategies through a series of simulation studies whose design is inspired by recent trials in chronic obstructive pulmonary disease (COPD), and we illustrate through an analysis of a recently completed COPD trial.