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Elastic integrative analysis of randomised trial and real-world data for treatment heterogeneity estimation.


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.

SUBMITTER: Yang S 

PROVIDER: S-EPMC10376438 | biostudies-literature | 2023 Jul

REPOSITORIES: biostudies-literature

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Elastic integrative analysis of randomised trial and real-world data for treatment heterogeneity estimation.

Yang Shu S   Gao Chenyin C   Zeng Donglin D   Wang Xiaofei X  

Journal of the Royal Statistical Society. Series B, Statistical methodology 20230406 3


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 alternativ  ...[more]

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