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In clinical trials with recurrent events, such as repeated hospitalizations terminating with death, it is important to consider the patient events overall history for a thorough assessment of treatment effects. The occurrence of fewer…

统计方法学 · 统计学 2025-09-08 Alessandra Ragni , Torben Martinussen , Thomas Scheike

Often in follow-up studies intermediate events occur in some patients, such as reinterventions or adverse events. These intermediate events directly affect the shapes of their longitudinal profiles. Our work is motivated by two studies in…

Predictive risk scores estimating probabilities for a binary outcome on the basis of observed covariates are common across the sciences. They are frequently developed with the intent of avoiding the outcome in question by intervening in…

统计方法学 · 统计学 2022-08-26 James Liley

We develop a Bayesian nonparametric (BNP) approach to evaluate the causal effect of treatment in a randomized trial where a nonterminal event may be censored by a terminal event, but not vice versa (i.e., semi-competing risks). Based on the…

统计方法学 · 统计学 2019-07-23 Yanxun Xu , Daniel Scharfstein , Peter Müller , Michael Daniels

We consider the estimation of average treatment effects in observational studies and propose a new framework of robust causal inference with unobserved confounders. Our approach is based on distributionally robust optimization and proceeds…

统计方法学 · 统计学 2023-02-06 Dimitris Bertsimas , Kosuke Imai , Michael Lingzhi Li

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

统计方法学 · 统计学 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

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

Recurrent events are common and important clinical trial endpoints in many disease areas, e.g., cardiovascular hospitalizations in heart failure, relapses in multiple sclerosis, or exacerbations in asthma. During a trial, patients may…

统计方法学 · 统计学 2026-04-08 Jiren Sun , Tobias Mutze , Richard Cook , Tianmeng Lyu

Randomized trials are often conducted with separate randomizations across multiple sites such as schools, voting districts, or hospitals. These sites can differ in important ways, including the site's implementation, local conditions, and…

统计方法学 · 统计学 2018-03-19 Lo-Hua Yuan , Avi Feller , Luke W. Miratrix

The sufficient cause framework has been used for decades to improve our understanding of both basic and more complex causal concepts in epidemiology, such as mediation and interaction. Here, we make use of this framework to provide a…

统计方法学 · 统计学 2026-04-08 Bronner P. Gonçalves , Eiji Yamamoto , Etsuji Suzuki

This paper presents a randomization-based framework for estimating causal effects under interference between units, motivated by challenges that arise in analyzing experiments on social networks. The framework integrates three components:…

统计理论 · 数学 2018-06-21 Peter M. Aronow , Cyrus Samii

Analysis of data from randomized controlled trials in vulnerable populations requires special attention when assessing treatment effect by a score measuring, e.g., disease stage or activity together with onset of prevalent terminal events.…

Continuous outcome measurements truncated by death present a challenge for the estimation of unbiased treatment effects in randomized controlled trials (RCTs). One way to deal with such situations is to estimate the survivor average causal…

统计方法学 · 统计学 2025-12-02 Stefanie von Felten , Chiara Vanetta , Christoph M. Rüegger , Sven Wellmann , Leonhard Held

In contrast to problems of interference in (exogenous) treatments, models of interference in unit-specific (endogenous) outcomes do not usually produce a reduced-form representation where outcomes depend on other units' treatment status…

计量经济学 · 经济学 2025-06-17 Konrad Menzel

It is a common practice in randomized clinical trials with the standard survival outcome to follow patients until a prespecified number of events have been observed, a type of trial known as the event-driven trial. The event-driven design…

统计方法学 · 统计学 2026-02-10 Jingwen Zhang , Satoshi Hattori

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

统计方法学 · 统计学 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Prediction models are used amongst others to inform medical decisions on interventions. Typically, individuals with high risks of adverse outcomes are advised to undergo an intervention while those at low risk are advised to refrain from…

Many research questions involve time-to-event outcomes that can be prevented from occurring due to competing events. In these settings, we must be careful about the causal interpretation of classical statistical estimands. In particular,…

统计方法学 · 统计学 2020-09-01 Torben Martinussen , Mats Julius Stensrud

Accurate estimation of treatment effects is essential for decision-making across various scientific fields. This task, however, becomes challenging in areas like social sciences and online marketplaces, where treating one experimental unit…

机器学习 · 计算机科学 2025-02-04 Mohsen Bayati , Yuwei Luo , William Overman , Sadegh Shirani , Ruoxuan Xiong

In this paper we study approaches for dealing with treatment when developing a clinical prediction model. Analogous to the estimand framework recently proposed by the European Medicines Agency for clinical trials, we propose a…