A two-step framework for validating causal effect estimates.
Abstract
Comparing causal effect estimates obtained using observational data to
those obtained from the gold standard (i.e., randomized controlled
trials, RCTs) helps us assess the validity of these estimates. However,
comparisons are challenging due to differences between observational
data and RCT generated data: First, the treatment assignment
mechanism is often unknown for observational data, and second, the
sampling mechanism often differs between the RCT and the
observational data. Differences in the treatment assignment mechanism
introduce potential confounding, whereas differences in the sampling
mechanism introduce sampling bias. This article proposes a two-step
framework for the validation of causal effect estimates obtained from
observational data by adjusting for both mechanisms. A simulation study
is conducted to show that our suggested two-step framework enables
observational data to produce causal effect estimates similar to those
of an RCT. An application of our approach to validate treatment effects
of adjuvant chemotherapy obtained from registry data is demonstrated.
This article establishes a novel framework for comparing causal effect
estimates between observational data and RCT data, facilitating the
assessment of the validity of causal effect estimates obtained from
observational data.