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Composite endpoints are widely used in cardiovascular clinical trials to improve statistical efficiency while preserving clinical relevance. The Win Ratio (WR) measure and more general frameworks of Win Statistics have emerged as…

Methodology · Statistics 2025-11-24 Yunhan Mou , Fan Li , Denise Esserman , Yuan Huang

Survival regression is widely used to model time-to-events data, to explore how covariates may influence the occurrence of events. Modern datasets often encompass a vast number of covariates across many subjects, with only a subset of the…

Methodology · Statistics 2024-09-18 Abhishek Mandal , Abhisek Chakraborty

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…

Methodology · Statistics 2025-09-08 Alessandra Ragni , Torben Martinussen , Thomas Scheike

Survival regression aims to predict the time when an event of interest will take place, typically a death or a failure. A fully parametric method [18] is proposed to estimate the survival function as a mixture of individual parametric…

Machine Learning · Computer Science 2024-04-25 Qinxin Wang , Jiayuan Huang , Junhui Li , Jiaming Liu

Conventional methods for analyzing composite endpoints in clinical trials often only focus on the time to the first occurrence of all events in the composite. Therefore, they have inherent limitations because the individual patients' first…

Methodology · Statistics 2022-11-29 Jialu Wang , Yeh-Fong Chen , Thomas Gwise

Covariate adjustment aims to improve the statistical efficiency of randomized trials by incorporating information from baseline covariates. Popular methods for covariate adjustment include analysis of covariance for continuous endpoints and…

Methodology · Statistics 2025-05-09 Zhiwei Zhang , Ya Wang , Dong Xi

Composite endpoints that combine recurrent non-fatal events with a terminal event are increasingly used in randomized clinical trials, yet conventional time-to-first event analyses may obscure clinically relevant information. We compared…

Methodology · Statistics 2025-12-16 Adrien Orué , Derek Dinart , Laurent Billot , Carine Bellera , Virginie Rondeau

The use of instrumental variables for estimating the effect of an exposure on an outcome is popular in econometrics, and increasingly so in epidemiology. This increasing popularity may be attributed to the natural occurrence of instrumental…

Methodology · Statistics 2016-08-03 T. Martinussen , S. Vansteelandt , E. J. Tchetgen Tchetgen , D. M. Zucker

When longitudinal outcomes are evaluated in mortal populations, their non-existence after death complicates the analysis and its causal interpretation. Where popular methods often merge longitudinal outcome and survival into one scale or…

Composite endpoints are widely used as primary endpoints in clinical trials. Designing trials with time-to-event endpoints can be particularly challenging because the proportional hazard assumption usually does not hold when using a…

Methodology · Statistics 2022-11-07 Jordi Cortés Martinez , Marta Bofill Roig , Guadalupe Gómez Melis

Composite endpoints are increasingly used in clinical trials to capture treatment effects across multiple or hierarchically ordered outcomes. Although inference procedures based on win statistics, such as the win ratio, win odds, and net…

Methodology · Statistics 2025-10-28 Xi Fang , Zhiqiang Cao , Fan Li

Hierarchical composite endpoints, such as those analyzed using the Finkelstein-Schoenfeld (FS) statistic, are increasingly used in clinical trials for their ability to incorporate clinically prioritized outcomes. However, adaptive design…

Methodology · Statistics 2025-04-22 Krishna Padmanabhan , Cyrus Mehta

Composite endpoints that combine multiple outcomes on different scales are common in clinical trials, particularly in chronic conditions. In many of these cases, patients will have to cross a predefined responder threshold in each of the…

Methodology · Statistics 2019-02-20 Martina McMenamin , Jessica K. Barrett , Anna Berglind , James M. S. Wason

In survival studies it is important to record the values of key longitudinal covariates until the occurrence of event of a subject. For this reason, it is essential to study the association between longitudinal and time-to-event outcomes…

Methodology · Statistics 2021-06-09 Khandoker Akib Mohammad , Yuichi Hirose , Yuan Yao , Budhi Surya

The quantile residual lifetime (QRL) regression is an attractive tool for assessing covariate effects on the distribution of residual life expectancy, which is often of interest in clinical studies. When the study subjects are exposed to…

Methodology · Statistics 2025-03-04 Tonghui Yu , Liming Xiang , Jong-Hyeon Jeong

A population-averaged additive subdistribution hazards model is proposed to assess the marginal effects of covariates on the cumulative incidence function and to analyze correlated failure time data subject to competing risks. This approach…

Methodology · Statistics 2023-08-23 Xinyuan Chen , Denise Esserman , Fan Li

We propose a generalized win fraction regression framework for prioritized composite survival outcomes. The framework models the conditional win fraction through a chosen link function (including identity, logit, or probit), thereby…

Methodology · Statistics 2026-04-08 Zhiqiang Cao , Xi Fang , Fan Li

Proportional mean residual life model is studied for analysing survival data from the case-cohort design. To simultaneously estimate the regression parameters and the baseline mean residual life function, weighted estimating equations based…

Statistics Theory · Mathematics 2019-01-18 Huijuan Ma , Jianhua Shi , Yong Zhou

There is growing interest in a hybrid control design in which a randomized controlled trial is augmented with an external control arm from a previous trial or real world data. Existing methods for analyzing hybrid control studies include…

Methodology · Statistics 2025-01-30 Zhiwei Zhang , Jialuo Liu , Wei Liu

The win ratio offers a flexible approach to incorporate the hierarchy of clinical outcomes into the analysis of a composite endpoint, enabling simultaneous consideration of multiple outcome types, unlike traditional time-to-first-event…

Methodology · Statistics 2025-07-22 David Kronthaler , Matthias Schwenkglenks , Felix Beuschlein , Ulrike Held
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