Department of Research Support for Clinical Trials, Oslo University Hospital, Oslo, Norway.
Oslo Centre for Biostatistics and Epidemiology, Institute of Basic Medical Sciences, Faculty of Medicine, University of Oslo, Oslo, Norway.
Stat Methods Med Res. 2024 Nov;33(11-12):2043-2061. doi: 10.1177/09622802241281960. Epub 2024 Oct 30.
Current instrumental variable methodology focuses mainly on estimating causal effects for a dichotomous or an ordinal treatment variable. Situations with more than two unordered treatments are less explored. The challenge is that assumptions needed to derive point-estimators become increasingly stronger with the number of relevant treatment alternatives. In this article, we aim at deriving causal point-estimators for head-to-head comparisons of the effect of multiple relevant treatments or interventions. We will achieve this with a set of plausible and well-defined rationality assumptions while only considering ordinal instruments. We demonstrate that our methodology provides asymptotically unbiased estimators in the presence of unobserved confounding effects in a simulation study. We then apply the method to compare the effectiveness of five anti-inflammatory drugs in the treatment of rheumatoid arthritis. For this, we use a clinical data set from an observational study in Norway, where price is the primary determinant of the preferred drug and can therefore be considered as an instrument. The developed methodology provides an important addition to the toolbox for causal inference when comparing more than two interventions influenced by an instrumental variable.
目前的工具变量方法主要集中于估计二分类或有序处理变量的因果效应。对于超过两种无序处理的情况,研究较少。挑战在于,随着相关处理选择的数量增加,推导出点估计量所需的假设变得越来越强。在本文中,我们旨在为多个相关处理或干预措施的效果进行头对头比较推导出因果点估计量。我们将通过一组合理且定义明确的理性假设来实现这一点,同时只考虑有序工具变量。我们通过模拟研究证明,在存在未观测到的混杂效应的情况下,我们的方法提供了渐近无偏估计量。然后,我们将该方法应用于比较五种抗炎药物在治疗类风湿关节炎方面的有效性。为此,我们使用了来自挪威一项观察性研究的临床数据集,其中价格是首选药物的主要决定因素,因此可以被视为工具变量。当比较受工具变量影响的超过两种干预措施时,所开发的方法为因果推断提供了重要的工具补充。