论文标题
合并随机试验和观察性研究的因果推理方法:综述
Causal inference methods for combining randomized trials and observational studies: a review
论文作者
论文摘要
随着数据可用性的增加,可以在不同的数据集(随机对照试验(RCT)和观察性研究中评估因果效应。 RCT将治疗的作用与不需要的(混杂性)共发生的效果分离出来,但它们可能遭受无显性性的影响,因此缺乏外部有效性。另一方面,大型观察样本通常更代表目标人群,但可能会使混杂影响与感兴趣的治疗相结合。在本文中,我们回顾了关于综合RCT和观察性研究的因果推断方法的越来越多的文献,并努力努力两全其美。我们首先讨论使用观察数据的代表性提高RCT的概括性的识别和估计方法。经典估计器包括加权,有条件结果模型之间的差异以及双重稳健的估计器。然后,我们讨论结合RCT和观察数据的方法,以确保观察性分析的毫无根据或改善(条件)平均治疗效果估计。我们还连接和对比作品在潜在结果文献和结构性因果模型文献中发展。最后,我们使用仿真研究和现实世界数据比较了主要方法,以分析曲霉素对主要创伤患者死亡率的影响。还提供了可用代码和新实施的审查。
With increasing data availability, causal effects can be evaluated across different data sets, both randomized controlled trials (RCTs) and observational studies. RCTs isolate the effect of the treatment from that of unwanted (confounding) co-occurring effects but they may suffer from unrepresentativeness, and thus lack external validity. On the other hand, large observational samples are often more representative of the target population but can conflate confounding effects with the treatment of interest. In this paper, we review the growing literature on methods for causal inference on combined RCTs and observational studies, striving for the best of both worlds. We first discuss identification and estimation methods that improve generalizability of RCTs using the representativeness of observational data. Classical estimators include weighting, difference between conditional outcome models, and doubly robust estimators. We then discuss methods that combine RCTs and observational data to either ensure uncounfoundedness of the observational analysis or to improve (conditional) average treatment effect estimation. We also connect and contrast works developed in both the potential outcomes literature and the structural causal model literature. Finally, we compare the main methods using a simulation study and real world data to analyze the effect of tranexamic acid on the mortality rate in major trauma patients. A review of available codes and new implementations is also provided.