Density and treatment effect estimation under covariate-adaptive randomization with heavy-tailed outcomes.

IF 1.6 4区 数学 Q3 BIOLOGY
Biometrics Pub Date : 2026-07-01 DOI:10.1093/biomtc/ujag159
Hongzi Li, Wei Ma, Yingying Ma, Hanzhong Liu
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引用次数: 0

Abstract

Randomized experiments are the gold standard for investigating causal relationships, with comparisons of potential outcomes under different treatment groups used to estimate treatment effects. However, outcomes with heavy-tailed distributions pose significant challenges to traditional causal inference approaches. While recent studies have explored these issues under simple randomization, their application in more complex randomization designs, such as stratified randomization or covariate-adaptive randomization, has not been adequately addressed. To fill the gap, we first investigate the performance of nonparametric kernel density estimation methods under covariate-adaptive randomization, thereby establishing theoretical guarantees for treatment effect estimators based on the estimated densities. Second, we demonstrate the application of our density estimation framework to estimate the density treatment effect and overall quantile treatment effect, deriving the consistency and asymptotic normality of the estimators. For the overall quantile treatment effect, we show that the existing variance estimator for the influence function-based M-estimator tends to overestimate the asymptotic variance, especially under more balanced designs, and lacks universal applicability across randomization methods. To remedy this, we introduce a novel stratified transformed difference-in-means estimator to enhance efficiency and propose a universally applicable variance estimator to facilitate valid inferences. Numerical results demonstrate the effectiveness of the proposed methods in finite samples.

重尾结果协变量自适应随机化下的密度和治疗效果估计。
随机实验是调查因果关系的金标准,通过比较不同治疗组的潜在结果来估计治疗效果。然而,具有重尾分布的结果对传统的因果推理方法提出了重大挑战。虽然最近的研究在简单随机化下探索了这些问题,但它们在更复杂的随机化设计中的应用,如分层随机化或协变量自适应随机化,尚未得到充分解决。为了填补这一空白,我们首先研究了非参数核密度估计方法在协变量自适应随机化下的性能,从而建立了基于估计密度的治疗效果估计的理论保证。其次,我们证明了我们的密度估计框架的应用,以估计密度处理效果和总体分位数处理效果,得到估计量的相合性和渐近正态性。对于总体分位数处理效果,我们表明现有的基于影响函数的m估计的方差估计倾向于高估渐近方差,特别是在更平衡的设计下,并且缺乏跨随机化方法的普遍适用性。为了解决这个问题,我们引入了一种新的分层变换均值差估计器来提高效率,并提出了一种普遍适用的方差估计器来促进有效的推断。数值结果证明了该方法在有限样本下的有效性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Biometrics
Biometrics 生物-生物学
CiteScore
2.70
自引率
5.30%
发文量
178
审稿时长
4-8 weeks
期刊介绍: The International Biometric Society is an international society promoting the development and application of statistical and mathematical theory and methods in the biosciences, including agriculture, biomedical science and public health, ecology, environmental sciences, forestry, and allied disciplines. The Society welcomes as members statisticians, mathematicians, biological scientists, and others devoted to interdisciplinary efforts in advancing the collection and interpretation of information in the biosciences. The Society sponsors the biennial International Biometric Conference, held in sites throughout the world; through its National Groups and Regions, it also Society sponsors regional and local meetings.
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