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How should researchers conduct causal inference when the outcome of interest is latent and measured imperfectly by multiple indicators? We develop a general nonparametric framework for identifying and estimating average treatment effects on…

统计方法学 · 统计学 2026-04-22 Jiawei Fu , Donald P. Green

In survival contexts, substantial literature exists on estimating optimal treatment regimes, where treatments are assigned based on personal characteristics to maximize the survival probability. These methods assume that a set of covariates…

统计方法学 · 统计学 2025-07-24 Junwen Xia , Zishu Zhan , Jingxiao Zhang

We argue that randomized controlled trials (RCTs) are special even among settings where average treatment effects are identified by a nonparametric unconfoundedness assumption. This claim follows from two results of Robins and Ritov (1997):…

统计方法学 · 统计学 2021-09-28 P. M. Aronow , James M. Robins , Theo Saarinen , Fredrik Sävje , Jasjeet Sekhon

Observational studies of recurrent event rates are common in biomedical statistics. Broadly, the goal is to estimate differences in event rates under two treatments within a defined target population over a specified followup window.…

统计方法学 · 统计学 2024-11-13 Arman Oganisian , Anthony Girard , Jon A. Steingrimsson , Patience Moyo

Estimating heterogeneous treatment effects with machine learning has attracted substantial attention in both academic research and industrial practice. However, the two communities often evaluate models under markedly different conditions.…

机器学习 · 计算机科学 2026-05-26 George Panagopoulos

This paper develops an empirical balancing approach for the estimation of treatment effects under two-sided noncompliance using a binary conditionally independent instrumental variable. The method weighs both treatment and outcome…

计量经济学 · 经济学 2020-07-10 Phillip Heiler

We propose a doubly robust approach to characterizing treatment effect heterogeneity in observational studies. We develop a frequentist inferential procedure that utilizes posterior distributions for both the propensity score and outcome…

统计方法学 · 统计学 2022-07-21 Heejun Shin , Joseph Antonelli

In observational studies, contingency tables provide a simple and intuitive approach to study associations between categorical variables. However, any test of association in contingency tables may be biased due to unmeasured confounders.…

统计方法学 · 统计学 2025-10-10 Elaine K. Chiu , Hyunseung Kang

A sensitivity analysis in an observational study assesses the robustness of significant findings to unmeasured confounding. While sensitivity analyses in matched observational studies have been well addressed when there is a single outcome…

统计方法学 · 统计学 2015-11-05 Colin B. Fogarty , Dylan S. Small

Externally controlled single-arm trials are critical to assess treatment efficacy across therapeutic indications for which randomized controlled trials are not feasible. A closely-related research design, the unanchored indirect treatment…

统计方法学 · 统计学 2026-02-13 Harlan Campbell , Antonio Remiro-Azócar

Scientists regularly pose questions about treatment effects on outcomes conditional on a post-treatment event. However, causal inference in such settings requires care, even in perfectly executed randomized experiments. Recently, the…

统计方法学 · 统计学 2026-02-19 Chan Park , Mats Stensrud , Eric Tchetgen Tchetgen

When drawing causal inference from observational data, there is always concern about unmeasured confounding. One way to tackle this is to conduct a sensitivity analysis. One widely-used sensitivity analysis framework hypothesizes the…

统计方法学 · 统计学 2022-06-22 Bo Zhang , Eric J. Tchetgen Tchetgen

We address causal estimation in semi-competing risks settings, where a non-terminal event may be precluded by one or more terminal events. We define a principal-stratification causal estimand for treatment effects on the non-terminal event,…

统计方法学 · 统计学 2025-06-27 Karina Gelis-Cadena , Michael Daniels , Juned Siddique

The estimand framework proposes different strategies to address intercurrent events. The treatment policy strategy seems to be the most favoured as it is closely aligned with the pre-addendum intention-to-treat principle. All data for all…

统计方法学 · 统计学 2025-07-21 Sunita Rehal , Nicky Best , Sarah Watts , Thomas Drury

Causal decomposition analysis aims to assess the effect of modifying risk factors on reducing social disparities in outcomes. Recently, this analysis has incorporated individual characteristics when modifying risk factors by utilizing…

机器学习 · 统计学 2025-09-16 Soojin Park , Suyeon Kang , Chioun Lee

With the evolution of single-cell RNA sequencing techniques into a standard approach in genomics, it has become possible to conduct cohort-level causal inferences based on single-cell-level measurements. However, the individual gene…

统计方法学 · 统计学 2025-04-23 Jin-Hong Du , Zhenghao Zeng , Edward H. Kennedy , Larry Wasserman , Kathryn Roeder

Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

统计方法学 · 统计学 2026-03-31 Zhe Chen , Xinran Li

Randomized controlled trials (RCT) are the gold standard for evaluation of the efficacy and safety of investigational interventions. If every patient in an RCT were to adhere to the randomized treatment, one could simply analyze the…

应用统计 · 统计学 2020-06-08 Yongming Qu , Linda Shurzinske , Shanthi Sethuraman

Causal inference necessarily relies upon untestable assumptions; hence, it is crucial to assess the robustness of obtained results to violations of identification assumptions. However, such sensitivity analysis is only occasionally…

统计方法学 · 统计学 2025-05-19 Tobias Freidling , Qingyuan Zhao

Detection of interactions between treatment effects and patient descriptors in clinical trials is critical for optimizing the drug development process. The increasing volume of data accumulated in clinical trials provides a unique…