因子实验中单一处理效应的识别

Guilherme Duarte
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引用次数: 0

摘要

尽管成本高昂,随机对照试验(RCT)仍被广泛视为从社会科学到医学等各学科的黄金标准证据。近几十年来,研究人员越来越多地寻求通过因子设计来减轻重复 RCT 的资源负担,因子设计可同时测试多个假设,例如同时评估多种药物或产品效果的实验。这是因为单一治疗效果涉及一个具有单一重点干预措施的反事实世界,允许其他变量取其自然值(这些值可能被重点干预措施混淆或改变)。在本文中,我正式提出了这些孤立量可识别性的充分条件。我表明,依赖这类设计的研究人员必须证明函数形式的线性,或者--在非参数情况下--用有向无环图(Directed Acyclic Graph)说明变量在现实世界中的关系。最后,我提出了非参数锐界--即与有限的 RCT 数据相一致的信息量最大的最佳/最差情况估计值--表明何时对效应符号的推断在经验上是合理的。这些新结果用模拟数据进行了说明。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Identification of Single-Treatment Effects in Factorial Experiments
Despite their cost, randomized controlled trials (RCTs) are widely regarded as gold-standard evidence in disciplines ranging from social science to medicine. In recent decades, researchers have increasingly sought to reduce the resource burden of repeated RCTs with factorial designs that simultaneously test multiple hypotheses, e.g. experiments that evaluate the effects of many medications or products simultaneously. Here I show that when multiple interventions are randomized in experiments, the effect any single intervention would have outside the experimental setting is not identified absent heroic assumptions, even if otherwise perfectly realistic conditions are achieved. This happens because single-treatment effects involve a counterfactual world with a single focal intervention, allowing other variables to take their natural values (which may be confounded or modified by the focal intervention). In contrast, observational studies and factorial experiments provide information about potential-outcome distributions with zero and multiple interventions, respectively. In this paper, I formalize sufficient conditions for the identifiability of those isolated quantities. I show that researchers who rely on this type of design have to justify either linearity of functional forms or -- in the nonparametric case -- specify with Directed Acyclic Graphs how variables are related in the real world. Finally, I develop nonparametric sharp bounds -- i.e., maximally informative best-/worst-case estimates consistent with limited RCT data -- that show when extrapolations about effect signs are empirically justified. These new results are illustrated with simulated data.
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