Improving Trial Generalizability Using Observational Studies
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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.
Recommendations
- New methods for treatment effect calibration, with applications to non-inferiority trials
- Generalizing causal inferences from individuals in randomized trials to all trial-eligible individuals
- Globally efficient non-parametric inference of average treatment effects by empirical balancing calibration weighting
- Efficient and Robust Methods for Causally Interpretable Meta-Analysis: Transporting Inferences from Multiple Randomized Trials to a Target Population
- Sensitivity analysis for an unobserved moderator in RCT-to-target-population generalization of treatment effects
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Cited in
(28)- Sensitivity analysis for an unobserved moderator in RCT-to-target-population generalization of treatment effects
- New methods for treatment effect calibration, with applications to non-inferiority trials
- Estimating generalizability in systematic observation studies
- Efficient and Robust Methods for Causally Interpretable Meta-Analysis: Transporting Inferences from Multiple Randomized Trials to a Target Population
- Calibration Techniques Encompassing Survey Sampling, Missing Data Analysis and Causal Inference
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