Elastic integrative analysis of randomised trial and real-world data for treatment heterogeneity estimation
Shu Yang, Chenyin Gao, Donglin Zeng, Xiaofei Wang
2023Journal of the Royal Statistical Society Series B (Statistical Methodology)38 citationsDOIOpen Access PDF
Abstract
We propose a test-based elastic integrative analysis of the randomised trial and real-world data to estimate treatment effect heterogeneity with a vector of known effect modifiers. When the real-world data are not subject to bias, our approach combines the trial and real-world data for efficient estimation. Utilising the trial design, we construct a test to decide whether or not to use real-world data. We characterise the asymptotic distribution of the test-based estimator under local alternatives. We provide a data-adaptive procedure to select the test threshold that promises the smallest mean square error and an elastic confidence interval with a good finite-sample coverage property.
Topics & Concepts
EstimatorConfidence intervalReal world dataSample size determinationTest (biology)StatisticsComputer scienceMathematicsEconometricsData miningPaleontologyBiologyData scienceAdvanced Causal Inference TechniquesStatistical Methods and InferenceStatistical Methods in Clinical Trials