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In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

统计方法学 · 统计学 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

Oversubscribed treatments are often allocated using randomized waiting lists. Applicants are ranked randomly, and treatment offers are made following that ranking until all seats are filled. To estimate causal effects, researchers often…

统计方法学 · 统计学 2018-10-24 Clement de Chaisemartin , Luc Behaghel

We study the problem of estimating the average treatment effect (ATE) in adaptive experiments where treatment can only be encouraged -- rather than directly assigned -- via a binary instrumental variable. Building on semiparametric…

统计方法学 · 统计学 2025-10-30 Miruna Oprescu , Brian M Cho , Nathan Kallus

Estimation of the Average Treatment Effect (ATE) is often carried out in 2 steps, wherein the first step, the treatment and outcome are modeled, and in the second step the predictions are inserted into the ATE estimator. In the first steps,…

统计方法学 · 统计学 2023-07-21 Mehdi Rostami , Olli Saarela

Instrumental variables have been widely used to estimate the causal effect of a treatment on an outcome. Existing confidence intervals for causal effects based on instrumental variables assume that all of the putative instrumental variables…

统计方法学 · 统计学 2016-07-14 Hyunseung Kang , T. Tony Cai , Dylan S. Small

Identifying covariates that modify treatment effects is a central problem in causal inference. Yet existing data-adaptive procedures do not provide finite-sample control over the expected number of false discoveries, risking spurious…

统计方法学 · 统计学 2026-05-12 Falco J. Bargagli-Stoffi , Omar Melikechi

Effective medication management in Parkinson's Disease (PD) is challenging due to heterogeneous disease progression, variable patient response, and medication side effects. While AI models can forecast levodopa equivalent daily dose (LEDD)…

机器学习 · 计算机科学 2026-05-21 Ricardo Diaz-Rincon , Muxuan Liang , Adolfo Ramirez-Zamora , Benjamin Shickel

Instrumental variable (IV) methods allow us the opportunity to address unmeasured confounding in causal inference. However, most IV methods are only applicable to discrete or continuous outcomes with very few IV methods for censored…

统计方法学 · 统计学 2020-09-30 Youjin Lee , Edward H. Kennedy , Nandita Mitra

Investigating the causal relationship between exposure and the time-to-event outcome is an important topic in biomedical research. Previous literature has discussed the potential issues of using the hazard ratio as a marginal causal effect…

统计方法学 · 统计学 2022-05-05 Zihan Lin , Ai Ni , Bo Lu

Estimating a causal effect from observational data can be biased if we do not control for self-selection. This selection is based on confounding variables that affect the treatment assignment and the outcome. Propensity score methods aim to…

计量经济学 · 经济学 2021-09-10 Daniel Jacob

In a comprehensive cohort study of two competing treatments (say, A and B), clinically eligible individuals are first asked to enroll in a randomized trial and, if they refuse, are then asked to enroll in a parallel observational study in…

统计方法学 · 统计学 2019-10-09 Yi Lu , Daniel O. Scharfstein , Maria M. Brooks , Kevin Quach , Edward H. Kennedy

Adaptive designs dynamically update treatment probabilities using information accumulated during the experiment. Existing theory for causal inference from adaptive experiments primarily assumes the superpopulation framework with independent…

统计方法学 · 统计学 2026-02-26 Xinran Li , Anqi Zhao

Given n experiment subjects with potentially heterogeneous covariates and two possible treatments, namely active treatment and control, this paper addresses the fundamental question of determining the optimal accuracy in estimating the…

机器学习 · 统计学 2024-11-13 Jiachun Li , David Simchi-Levi , Yunxiao Zhao

To better understand effects of exposure to food allergens, food challenge studies are designed to slowly increase the dose of an allergen delivered to allergic individuals until an objective reaction occurs. These dose-to-failure studies…

应用统计 · 统计学 2019-08-30 Matthew W. Wheeler , Joost Westerhout , Joe L. Baumert , Benjamin C. Remington

In this paper, we introduce a doubly doubly robust estimator for the average and heterogeneous treatment effect for left-truncated-right-censored (LTRC) survival data. In causal inference for survival functions in LTRC survival data, two…

综合经济学 · 经济学 2024-09-04 Guanghui Pan

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

Causal inference with observational studies often relies on the assumptions of unconfoundedness and overlap of covariate distributions in different treatment groups. The overlap assumption is violated when some units have propensity scores…

统计方法学 · 统计学 2022-07-19 Shu Yang , Peng Ding

To estimate causal effects, analysts performing observational studies in health settings utilize several strategies to mitigate bias due to confounding by indication. There are two broad classes of approaches for these purposes: use of…

统计方法学 · 统计学 2023-05-01 Roy S. Zawadzki , Joshua D. Grill , Daniel L. Gillen

It is often of interest in observational studies to measure the causal effect of a treatment on time-to-event outcomes. In a medical setting, observational studies commonly involve patients who initiate medication therapy and others who do…

统计方法学 · 统计学 2019-10-07 Reagan Mozer , Mark E. Glickman

In this work, we propose an approach for assessing sensitivity to unobserved confounding in studies with multiple outcomes. We demonstrate how prior knowledge unique to the multi-outcome setting can be leveraged to strengthen causal…

统计方法学 · 统计学 2023-01-26 Jiajing Zheng , Jiaxi Wu , Alexander D'Amour , Alexander Franks
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