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Elastic Integrative Analysis of Randomized Trial and Real-World Data for\n Treatment Heterogeneity Estimation

2020/05/21 by Shu Yang, Yang, Shu, Gao, Chenyin +4 · 10 citations
Mathematics · #Advanced Causal Inference Techniques #FOS: Computer and information sciences #Methodology (stat.ME) #Statistical Methods and Inference #Statistical Methods in Clinical Trials

paper · pdf · doi:10.48550/arxiv.2005.10579

openalex publication_date 2020/05/21 · openalex created_date 2022/07/26 · openalex updated_date 2026/07/28

Abstract

We propose a test-based elastic integrative analysis of the randomized trial\nand real-world data to estimate treatment effect heterogeneity with a vector of\nknown effect modifiers. When the real-world data are not subject to bias, our\napproach combines the trial and real-world data for efficient estimation.\nUtilizing the trial design, we construct a test to decide whether or not to use\nreal-world data. We characterize the asymptotic distribution of the test-based\nestimator under local alternatives. We provide a data-adaptive procedure to\nselect the test threshold that promises the smallest mean square error and an\nelastic confidence interval with a good finite-sample coverage property.\n

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