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Regression analyses based on transformations of cumulative incidence functions are often adopted when modeling and testing for treatment effects in clinical trial settings involving competing and semi-competing risks. Common frameworks…

Methodology · Statistics 2024-01-11 Alexandra Bühler , Richard J Cook , Jerald F Lawless

To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying…

Methodology · Statistics 2023-08-08 Liangyuan Hu , Jiayi Ji , Himanshu Joshi , Erick Scott , Fan Li

Estimating heterogeneous treatment effect (HTE) for survival outcomes has gained increasing attention, as it captures the variation in treatment efficacy across patients or subgroups in delaying disease progression. However, most existing…

Methodology · Statistics 2025-11-27 Na Bo , Ying Ding

Survival time is the primary endpoint of many randomized controlled trials, and a treatment effect is typically quantified by the hazard ratio under the assumption of proportional hazards. Awareness is increasing that in many settings this…

Methodology · Statistics 2023-10-04 Robin Ristl , Heiko Götte , Armin Schüler , Martin Posch , Franz König

Quantifying causal effects in the presence of complex and multivariate outcomes remains a key challenge in treatment evaluation. For hierarchical multivariate outcomes, the FDA recommends the Win Ratio and Generalized Pairwise Comparisons…

Methodology · Statistics 2026-03-24 Mathieu Even , Julie Josse

In many causal studies, outcomes are censored by death, in the sense that they are neither observed nor defined for units who die. In such studies, the focus is usually on the stratum of always survivors up to a single fixed time s.…

Methodology · Statistics 2024-01-02 Giulio Grossi , Marco Mariani , Alessandra Mattei , Fabrizia Mealli

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

Sensitivity analysis for unmeasured confounding under incremental propensity score interventions remains relatively underdeveloped. Incremental interventions define stochastic treatment regimes by multiplying the odds of treatment, offering…

Methodology · Statistics 2026-01-27 Shuying Shen , Valerio Bacak , Edward H. Kennedy

This work develops a flexible inferential framework for nonparametric causal inference in time-to-event settings, based on stochastic interventions defined through multiplicative scaling of the intensity governing an intermediate event…

Methodology · Statistics 2026-02-04 Helene Charlotte Wiese Rytgaard , Mark van der Laan

When the Stable Unit Treatment Value Assumption is violated and there is interference among units, there is not a uniquely defined Average Treatment Effect, and alternative estimands may be of interest. Among these are average unit-level…

Methodology · Statistics 2025-06-30 Molly Offer-Westort , Drew Dimmery

Understanding the effects of interventions is central to scientific progress, with randomized controlled trials (RCTs) regarded as the gold standard for causal inference in many applied fields. However, RCTs are costly, time-consuming, and…

Methodology · Statistics 2026-05-27 Drago Plecko , Patrik Okanovic , Torsten Hoefler , Elias Bareinboim

This Element offers a practical guide to estimating conditional marginal effects-how treatment effects vary with a moderating variable-using modern statistical methods. Commonly used approaches, such as linear interaction models, often…

Methodology · Statistics 2026-05-21 Jiehan Liu , Ziyi Liu , Yiqing Xu

Motivated by empirical studies investigating treatment effects in survival analysis, we propose a bivariate transformation model to quantify the impact of a binary treatment on a time-to-event outcome. The model equations are connected…

Methodology · Statistics 2025-02-03 Giampiero Marra , Rosalba Radice

Matching in causal inference from observational data aims to construct treatment and control groups with similar distributions of covariates, thereby reducing confounding and ensuring an unbiased estimation of treatment effects. This…

Artificial Intelligence · Computer Science 2025-04-15 Sahil Shikalgar , Md. Noor-E-Alam

Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes…

Methodology · Statistics 2021-12-21 Shuxi Zeng , Fan Li , Liangyuan Hu , Fan Li

We extend recently proposed design-based capture-recapture (CRC) methods for prevalence estimation among registry participants, in order to enhance treatment effect evaluation among a trial-eligible target population. The so-called ``anchor…

Methodology · Statistics 2025-10-28 Lin Ge , Yuzi Zhang , Lance A. Waller , Robert H. Lyles

Principal stratification is a general framework for studying causal mechanisms involving post-treatment variables. When estimating principal causal effects, the principal ignorability assumption is commonly invoked, which we study in detail…

Methodology · Statistics 2026-04-21 Minxuan Wu , Joseph Antonelli

Causal inference is the goal of randomized trials and many observational studies. The first step in a formal causal inference framework is to define the causal estimand, and in both types of study this can be intuitively defined as the…

Methodology · Statistics 2025-09-09 Margarita Moreno-Betancur , Rushani Wijesuriya , John B. Carlin

In this paper, we propose the use of causal inference techniques for survival function estimation and prediction for subgroups of the data, upto individual units. Tree ensemble methods, specifically random forests were modified for this…

Econometrics · Economics 2018-03-23 Vikas Ramachandra

Interference occurs when a unit's treatment (or exposure) affects another unit's outcome. In some settings, units may be grouped into clusters such that it is reasonable to assume that interference, if present, only occurs between…

Methodology · Statistics 2023-08-24 Chanhwa Lee , Donglin Zeng , Michael G. Hudgens