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

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 est...

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Bibliographic Details
Published inJournal of the Royal Statistical Society. Series B, Statistical methodology Vol. 85; no. 3; pp. 575 - 596
Main Authors Yang, Shu, Gao, Chenyin, Zeng, Donglin, Wang, Xiaofei
Format Journal Article
LanguageEnglish
Published England Oxford University Press 01.07.2023
Subjects
Elastic analysis
Heterogeneity
Tests
non-regularity
precision medicine
pre-test estimator
semiparametric efficiency
counterfactual outcome
least favourable confidence interval
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Summary: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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ISSN:1369-7412
1467-9868
1467-9868
DOI:10.1093/jrsssb/qkad017