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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.…

Methodology · Statistics 2024-11-13 Arman Oganisian , Anthony Girard , Jon A. Steingrimsson , Patience Moyo

To improve precision of estimation and power of testing hypothesis for an unconditional treatment effect in randomized clinical trials with binary outcomes, researchers and regulatory agencies recommend using g-computation as a reliable…

Methodology · Statistics 2023-03-29 Ting Ye , Marlena Bannick , Yanyao Yi , Jun Shao

In survey analysis, the estimation of the cumulative distribution function (cdf) is of great interest: it allows for instance to derive quantiles estimators or other non linear parameters derived from the cdf. We consider the case where the…

Methodology · Statistics 2014-04-14 Sandrine Casanova , Eve Leconte

Two commonly used methods for improving precision and power in clinical trials are stratified randomization and covariate adjustment. However, many trials do not fully capitalize on the combined precision gains from these two methods, which…

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,…

Methodology · Statistics 2025-06-27 Karina Gelis-Cadena , Michael Daniels , Juned Siddique

The primary analysis for longitudinal randomized controlled trials (RCTs) often compares treatment groups at the last timepoint, referred to as the landmark time. Assuming data are normally distributed and missing at random, the mixed model…

Methodology · Statistics 2026-03-18 Guangyong Zou , Shi-Fang Qui , Joshua Zou , Emma Davies Smith , Yun-Hee Choi , Yuhan Bi

Marginal structural models (MSMs) are often used to estimate causal effects of treatments on survival time outcomes from observational data when time-dependent confounding may be present. They can be fitted using, e.g., inverse probability…

Methodology · Statistics 2023-12-27 Shaun R Seaman , Ruth H Keogh

Randomized experiments have been the gold standard for drawing causal inference. The conventional model-based approach has been one of the most popular ways for analyzing treatment effects from randomized experiments, which is often carried…

Methodology · Statistics 2024-11-19 Tianyi Qu , Jiangchuan Du , Xinran Li

Matching is one of the simplest approaches for estimating causal effects from observational data. Matching techniques compare the observed outcomes across pairs of individuals with similar covariate values but different treatment statuses…

Artificial Intelligence · Computer Science 2024-09-23 Abhishek Dalvi , Neil Ashtekar , Vasant Honavar

This paper presents a weighted optimization framework that unifies the binary,multi-valued, continuous, as well as mixture of discrete and continuous treatment, under the unconfounded treatment assignment. With a general loss function, the…

Econometrics · Economics 2018-08-20 Chunrong Ai , Oliver Linton , Kaiji Motegi , Zheng Zhang

The Mann-Whitney effect is an intuitive measure for discriminating two survival distributions. Here we analyze various inference techniques for this parameter in a two-sample survival setting with independent right-censoring, where the…

Statistics Theory · Mathematics 2016-09-26 Dennis Dobler , Markus Pauly

Cluster-randomized trials (CRTs) are experimental designs where groups or clusters of participants, rather than the individual participants themselves, are randomized to intervention groups. Analyzing CRT requires distinguishing between…

Methodology · Statistics 2025-10-10 Xi Fang , Bingkai Wang , Liangyuan Hu , Fan Li

Tailoring treatments to individual needs is a central goal in fields such as medicine. A key step toward this goal is estimating Heterogeneous Treatment Effects (HTE) - the way treatments impact different subgroups. While crucial, HTE…

Machine Learning · Statistics 2025-07-30 Tomer Meir , Uri Shalit , Malka Gorfine

Balanced representation learning methods have been applied successfully to counterfactual inference from observational data. However, approaches that account for survival outcomes are relatively limited. Survival data are frequently…

Machine Learning · Statistics 2021-03-04 Paidamoyo Chapfuwa , Serge Assaad , Shuxi Zeng , Michael J. Pencina , Lawrence Carin , Ricardo Henao

Real-world clinical decision making is a complex process that involves balancing the risks and benefits of treatments. Quality-adjusted lifetime is a composite outcome that combines patient quantity and quality of life, making it an…

Statistics Theory · Mathematics 2024-12-09 Hao Sun , Ashkan Ertefaie , Luke Duttweiler , Brent A. Johnson

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,…

Methodology · Statistics 2023-07-21 Mehdi Rostami , Olli Saarela

Multistate process data are common in studies of chronic diseases such as cancer. These data are ideal for precision medicine purposes as they can be leveraged to improve more refined health outcomes, compared to standard survival outcomes,…

Methodology · Statistics 2022-11-28 Giorgos Bakoyannis

In observational studies, balancing covariates in different treatment groups is essential to estimate treatment effects. One of the most commonly used methods for such purposes is weighting. The performance of this class of methods usually…

Methodology · Statistics 2021-07-07 Ruoqi Yu , Shulei Wang

Estimation of individualized treatment effects (ITE), also known as conditional average treatment effects (CATE), is an active area of methodology development. However, much less attention has been paid to the quantification of uncertainty…

Methodology · Statistics 2025-04-08 Daijiro Kabata , Nicholas C. Henderson , Ravi Varadhan

Given the unconfoundedness assumption, we propose new nonparametric estimators for the reduced dimensional conditional average treatment effect (CATE) function. In the first stage, the nuisance functions necessary for identifying CATE are…

Econometrics · Economics 2021-07-26 Qingliang Fan , Yu-Chin Hsu , Robert P. Lieli , Yichong Zhang