论文标题

基于偏好的仪器变量研究和集群随机鼓励实验:研究设计,不合格和平均聚类效应比率

Bridging preference-based instrumental variable studies and cluster-randomized encouragement experiments: study design, noncompliance, and average cluster effect ratio

论文作者

Zhang, Bo, Heng, Siyu, MacKay, Emily J., Ye, Ting

论文摘要

仪器变量方法在医学和社会科学研究中广泛使用,以得出因素和结果被未衡量的混杂变量混淆时的因果结论。此类研究的一个重要特征是,仪器变量通常在集群级别应用,例如,医院或医师偏爱每位医院或医师自然定义集群的某种治疗方法。本文建议将这种观察性仪器可变数据嵌入使用统计匹配的集群随机鼓励实验中。设计并检查了基础的潜在结果和因果假设。 Fisher的Sharp Null假设和合并效应比率的测试程序扩展到当前设置。然后,我们引入了一种新型的簇异质治疗效应模型和相关的估计:平均聚类效应比。在恒定的比例治疗效应模型中,这种新的估计比结构参数具有优势,因为它允许治疗异质性,并且在汇总效应比估计中是有利的,因为它不受辛普森的悖论的影响。我们基于解决混合整数四次约束优化问题的新估计和基于渐近有效的基于随机的测试过程。提出的设计和推论方法应用于研究在CABG手术期间使用经食管超声心动图对患者30天死亡率的影响。

Instrumental variable methods are widely used in medical and social science research to draw causal conclusions when the treatment and outcome are confounded by unmeasured confounding variables. One important feature of such studies is that the instrumental variable is often applied at the cluster level, e.g., hospitals' or physicians' preference for a certain treatment where each hospital or physician naturally defines a cluster. This paper proposes to embed such observational instrumental variable data into a cluster-randomized encouragement experiment using statistical matching. Potential outcomes and causal assumptions underpinning the design are formalized and examined. Testing procedures for two commonly-used estimands, Fisher's sharp null hypothesis and the pooled effect ratio, are extended to the current setting. We then introduce a novel cluster-heterogeneous proportional treatment effect model and the relevant estimand: the average cluster effect ratio. This new estimand is advantageous over the structural parameter in a constant proportional treatment effect model in that it allows treatment heterogeneity, and is advantageous over the pooled effect ratio estimand in that it is immune to Simpson's paradox. We develop an asymptotically valid randomization-based testing procedure for this new estimand based on solving a mixed integer quadratically-constrained optimization problem. The proposed design and inferential methods are applied to a study of the effect of using transesophageal echocardiography during CABG surgery on patients' 30-day mortality rate.

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