Causal inference methods for combining randomized trials and observational studies: a review

Awa Dieng

Abstract: With increasing data availability, causal effects can be evaluated across different datasets, 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 struggle with inclusion biases, 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 improve (conditional) average treatment effect estimation, handling possible unmeasured confounding in the observational data. 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. Code to implement many of the methods is provided.