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Improving trial generalizability using observational studies.

Authors :
Lee, Dasom
Yang, Shu
Dong, Lin
Wang, Xiaofei
Zeng, Donglin
Cai, Jianwen
Source :
Biometrics. Jun2023, Vol. 79 Issue 2, p1213-1225. 13p.
Publication Year :
2023

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. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
0006341X
Volume :
79
Issue :
2
Database :
Academic Search Index
Journal :
Biometrics
Publication Type :
Academic Journal
Accession number :
164420855
Full Text :
https://doi.org/10.1111/biom.13609