A causal inference framework for leveraging external controls in hybrid trials.

ABSTRACT: We consider the challenges associated with causal inference in settings where data from a randomized trial are augmented with control data from an external source to improve efficiency in estimating the average treatment effect (ATE). This question is motivated by the SUNFISH trial, which investigated the effect of risdiplam on motor function in patients with spinal muscular atrophy. While the original analysis used only data generated by the trial, we explore an alternative analysis incorporating external controls from the placebo arm of a historical trial. We cast the setting into a formal causal inference framework and show how these designs are characterized by a lack of full randomization to treatment and heightened dependency on modeling. To address this, we outline sufficient causal

SUBMITTER: Valancius M 

PROVIDER: S-EPMC11546536 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature