随机试验与真实世界数据的弹性整合分析用于治疗异质性估计

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

Journal of the Royal Statistical Society. Series B: Statistical Methodology · 2023
被引 38 · 同刊同年前 3%
ABS 4

中文导读

提出一种基于检验的弹性整合方法,结合随机试验和真实世界数据估计治疗异质性,通过检验决定是否使用真实世界数据,并给出自适应阈值选择和弹性置信区间。

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.

计量经济学统计学因果推断临床试验