Improving trial generalizability using observational studies

Dasom Lee, Shu Yang, Lin Dong, Xiaofei Wang, Donglin Zeng and Jianwen Cai

Biometrics, 2023, vol. 79, issue 2, 1213-1225

Abstract: Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces the covariate balance between the RCT and OS, therefore improving the trial‐based estimator's generalizability. Exploiting semiparametric efficiency theory, we propose a doubly robust augmented calibration weighting estimator that achieves the efficiency bound derived under the identification assumptions. A nonparametric sieve method is provided as an alternative to the parametric approach, which enables the robust approximation of the nuisance functions and data‐adaptive selection of outcome predictors for calibration. We establish asymptotic results and confirm the finite sample performances of the proposed estimators by simulation experiments and an application on the estimation of the treatment effect of adjuvant chemotherapy for early‐stage non‐small‐cell lung patients after surgery.

Date: 2023
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