Leveraging Observational Data for Efficient CATE Estimation in Randomized Controlled Trials

Abstract: Randomized controlled trials (RCTs) are the gold standard for causal inference, but they are often powered only for average effects, making estimation of heterogeneous treatment effects (HTEs) challenging. Conversely, large-scale observational studies (OS) offer a wealth of data but suffer from confounding bias. Our paper presents a novel framework to leverage OS data for enhancing the efficiency in estimating conditional average treatment effects (CATEs) from RCTs while mitigating common biases. We propose an innovative approach to combine RCTs and OS data, expanding the traditionally used control arms from external sources. The framework relaxes the typical assumption of CATE invariance across populations, acknowledging the often unaccounted systematic differences between RCT and OS participants. We demonstrate this through the special case of a linear outcome model, where the CATE is sparsely different between the two populations. The core of our framework relies on learning potential outcome means from OS data and using them as a nuisance parameter in CATE estimation from RCT data. We further illustrate through experiments that using OS findings reduces the variance of the estimated CATE from RCTs and can decrease the required sample size for detecting HTEs.