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Network data have appeared frequently in recent research. For example, in comparing the effects of different types of treatment, network models have been proposed to improve the quality of estimation and hypothesis testing. In this paper,…

Methodology · Statistics 2020-09-04 Zhixin Zhou , Ping Li , Feifang Hu

Background: Incidence of adverse outcome events rises as patients with advanced illness approach end-of-life. Exposures that tend to occur near end-of-life, e.g., use of wheelchair, oxygen therapy and palliative care, may therefore be found…

Both cluster randomized trials and quasi-experimental designs are used to evaluate the impact of health and social policies and interventions. Stepped-wedge cluster randomized trials randomize a staggered adoption approach, while recent…

Methodology · Statistics 2026-04-15 Haidong Lu , Gregg S. Gonsalves , Fan Li , Guanyu Tong , Lee Kennedy-Shaffer

Modern longitudinal studies collect feature data at many timepoints, often of the same order of sample size. Such studies are typically affected by {dropout} and positivity violations. We tackle these problems by generalizing effects of…

Methodology · Statistics 2022-03-16 Kwangho Kim , Edward H. Kennedy , Ashley I. Naimi

The efficacy of an intervention can be assessed by randomizing patients to different diagnostic tests instead of directly to an intervention and control. This principle is applied by allocating individuals to intervention if the test result…

Applications · Statistics 2022-01-11 Huw Llewelyn

As standards of care advance, patients are living longer and once-fatal diseases are becoming manageable. Clinical trials increasingly focus on reducing disease burden, which can be quantified by the timing and occurrence of multiple…

For a trial with primary endpoint overall survival for a molecule with curative potential, statistical methods that rely on the proportional hazards assumption may underestimate the power and the time to final analysis. We show how a cure…

Applications · Statistics 2023-04-17 Kaspar Rufibach , Dominik Heinzmann , Annabelle Monnet

We address the common yet often-overlooked selection bias in interventional studies, where subjects are selectively enrolled into experiments. For instance, participants in a drug trial are usually patients of the relevant disease; A/B…

Machine Learning · Computer Science 2025-03-11 Haoyue Dai , Ignavier Ng , Jianle Sun , Zeyu Tang , Gongxu Luo , Xinshuai Dong , Peter Spirtes , Kun Zhang

Typically, a randomized experiment is designed to test a hypothesis about the average treatment effect and sometimes hypotheses about treatment effect variation. The results of such a study may then be used to inform policy and practice for…

Methodology · Statistics 2026-05-01 Elizabeth Tipton , Michalis Mamakos

Trial engagement effects are effects of trial participation on the outcome that are not mediated by treatment assignment. Most work on extending (generalizing or transporting) causal inferences from a randomized trial to a target population…

Methodology · Statistics 2024-07-23 Lawson Ung , Tyler J. VanderWeele , Issa J. Dahabreh

This paper provides a nonparametric framework for causal inference with categorical outcomes under binary treatment and binary instrument settings. I decompose the observed joint probability of outcomes and treatment into marginal…

Econometrics · Economics 2025-11-11 Onil Boussim

Recurrent events often serve as key endpoints in clinical studies but may be prematurely truncated by terminal events such as death, creating selection bias and complicating causal inference. To address this challenge, we develop a Bayesian…

Methodology · Statistics 2026-03-18 Yuki Ohnishi , Michael O. Harhay , Guangyu Tong , Fan Li

Randomized clinical trials typically aim to estimate a marginal treatment effect. While covariate adjustment can improve precision, it may change the estimand in nonlinear models due to noncollapsibility, leading to conditional rather than…

Methodology · Statistics 2026-05-25 Leticia Wuethrich , Torsten Hothorn

Path-specific effects are a broad class of mediated effects from an exposure to an outcome via one or more causal pathways with respect to some subset of intermediate variables. The majority of the literature concerning estimation of…

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…

Methodology · Statistics 2026-02-19 Chan Park , Mats Stensrud , Eric Tchetgen Tchetgen

The proximal causal inference framework enables the identification and estimation of causal effects in the presence of unmeasured confounding by leveraging two disjoint sets of observed strong proxies: negative control treatments and…

Methodology · Statistics 2025-12-16 Antonio Olivas-Martinez , Peter B. Gilbert , Andrea Rotnitzky

We consider the problem of defining the effect of an intervention on a time-varying risk factor or treatment for a disease or a physiological marker; we develop here the latter case. So, the system considered is $(Y,A,C)$, where $Y=(Y_t)$,…

Methodology · Statistics 2019-07-31 Daniel Commenges , Mélanie Prague

The traditional model specification of stepped-wedge cluster-randomized trials assumes a homogeneous treatment effect across time while adjusting for fixed-time effects. However, when treatment effects vary over time, the constant effect…

This paper studies inference on treatment effects in panel data settings with unobserved confounding. We model outcome variables through a factor model with random factors and loadings. Such factors and loadings may act as unobserved…

Econometrics · Economics 2023-12-05 Guido W. Imbens , Davide Viviano

This research was motivated by studying anti-drug antibody (ADA) formation and its potential impact on long-term benefit of a biologic treatment in a randomized controlled trial, in which ADA status was not only unobserved in the control…

Methodology · Statistics 2021-01-13 Shengchun Kong , Dominik Heinzmann , Sabine Lauer , Tian Lu
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