A Comparison of Methods for Estimating the Causal Effect of a Treatment in Randomized Clinical Trials Subject to Noncompliance
A Comparison of Methods for Estimating the Causal Effect of a Treatment in Randomized Clinical Trials Subject to Noncompliance
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DOI：
10.1111/j.1541-0420.2008.01066.x
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发表时间：
2009-06-01
期刊：
影响因子：
1.9
通讯作者：
Lin, Xihong
中科院分区：
文献类型：
--
作者：
Little, Roderick J.;Long, Qi;Lin, Xihong
我们考虑的临床试验的分析，涉及随机化到一个积极的治疗（T = 1）或对照治疗（T = 0），当积极的治疗是服从全或无依从性。在这种情况下，我们比较了三种估计治疗效果的方法：治疗分析，符合方案分析和工具变量（IV）估计，其中使用随机化指标作为IV估计治疗效果。这两个模型和基于矩的IV估计方法被认为是。这些估计的假设进行了评估，标准误差和均方误差的估计进行了比较，并检查这三种方法的设计影响。扩展的方法，包括观察到的协变量进行了讨论，强调的作用，遵守倾向的方法和对比的协变量在这些扩展的作用。方法说明的数据从妇女采取骄傲的研究，评估行为治疗妇女心脏病。
We consider the analysis of clinical trials that involve randomization to an active treatment (T = 1) or a control treatment (T = 0), when the active treatment is subject to all-or-nothing compliance. We compare three approaches to estimating treatment efficacy in this situation: as-treated analysis, per-protocol analysis, and instrumental variable (IV) estimation, where the treatment effect is estimated using the randomization indicator as an IV. Both model- and method-of-moment based IV estimators are considered. The assumptions underlying these estimators are assessed, standard errors and mean squared errors of the estimates are compared, and design implications of the three methods are examined. Extensions of the methods to include observed covariates are then discussed, emphasizing the role of compliance propensity methods and the contrasting role of covariates in these extensions. Methods are illustrated on data from the Women Take Pride study, an assessment of behavioral treatments for women with heart disease.