Elastic Integrative Analysis of Randomised Trial and Real-world Data for Treatment Heterogeneity Estimation

Overview

Journal J R Stat Soc Series B Stat Methodol

Date 2023 Jul 31

PMID 37521165

Authors

Shu Yang

Chenyin Gao

Donglin Zeng

Xiaofei Wang

Affiliations

Soon will be listed here.

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.

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