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Related papers: Random Effect Restricted Mean Survival Time Model

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Recent observations, especially in cancer immunotherapy clinical trials with time-to-event outcomes, show that the commonly used proportial hazard assumption is often not justifiable, hampering an appropriate analyse of the data by hazard…

Methodology · Statistics 2021-02-23 Marc Ditzhaus , Menggang Yu , Jin Xu

A targeted learning (TL) framework is developed to estimate the difference in the restricted mean survival time (RMST) for a clinical trial with time-to-event outcomes. The approach starts by defining the target estimand as the RMST…

Methodology · Statistics 2026-01-21 Man Jin , Yixin Fang

Several methods in survival analysis are based on the proportional hazards assumption. However, this assumption is very restrictive and often not justifiable in practice. Therefore, effect estimands that do not rely on the proportional…

Methodology · Statistics 2024-03-20 Merle Munko , Marc Ditzhaus , Dennis Dobler , Jon Genuneit

Multi-regional clinical trials (MRCTs) play an increasingly crucial role in global pharmaceutical development by expediting data gathering and regulatory approval across diverse patient populations. However, differences in recruitment…

Methodology · Statistics 2024-04-15 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

The difference in restricted mean survival times between two groups is a clinically relevant summary measure. With observational data, there may be imbalances in confounding variables between the two groups. One approach to account for such…

Applications · Statistics 2014-10-17 Andrew Wey , David Vock , John Connett , Kyle Rudser

Prediction methods for time-to-event outcomes often utilize survival models that rely on strong assumptions about noninformative censoring or on how individual-level covariates and survival functions are related. When the main interest is…

Methodology · Statistics 2024-02-29 Mahsa Ashouri , Nicholas C. Henderson

We consider a three-level meta-analysis of standardized mean differences. The standard method of estimation uses inverse-variance weights and REML/PL estimation of variance components for the random effects. We introduce new moment-based…

Methodology · Statistics 2024-11-05 Elena Kulinskaya , David C. Hoaglin

Progressive multi-state survival outcomes are common in trials with recurrent or sequential events and require treatment effect estimands that remain interpretable without proportional intensity or Markov assumptions. The restricted mean…

Methodology · Statistics 2026-01-22 Xi Fang , Bingkai Wang , Guangyu Tong , Liangyuan Hu , Shuangge Ma , Fan Li

Hazard ratios are ubiquitously used in time to event analysis to quantify treatment effects. Although hazard ratios are invaluable for hypothesis testing, other measures of association, both relative and absolute, may be used to fully…

Methodology · Statistics 2020-11-02 Federico Ambrogi , Simona Iacobelli , Per Kragh Andersen

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

Alternating recurrent events, where subjects experience two potentially correlated event types over time, are common in healthcare, social, and behavioral studies. Often there is a primary event of interest that, when triggered, initiates a…

Methodology · Statistics 2025-10-29 Abigail Loe , Susan Murray , Zhenke Wu

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

Patients with breast cancer tend to die from other diseases, so for studies that focus on breast cancer, a competing risks model is more appropriate. Considering subdistribution hazard ratio, which is used often, limited to model…

Methodology · Statistics 2023-11-21 Zhiyin Yu , Zhaojin Li , Chengfeng Zhang , Yawen Hou , Derun Zhou , Zheng Chen

Sufficient dimension reduction (SDR) is a popular class of regression methods which aim to find a small number of linear combinations of covariates that capture all the information of the responses i.e., a central subspace. The majority of…

Methodology · Statistics 2024-10-15 Linh H. Nghiem , F. K. C. Hui

What can be considered an appropriate statistical method for the primary analysis of a randomized clinical trial (RCT) with a time-to-event endpoint when we anticipate non-proportional hazards owing to a delayed effect? This question has…

Methodology · Statistics 2023-04-18 José L. Jiménez , Isobel Barrott , Francesca Gasperoni , Dominic Magirr

In recent years, cancer clinical trials have increasingly encountered non proportional hazards (NPH) scenarios, particularly with the emergence of immunotherapy. In randomized controlled trials comparing immunotherapy with conventional…

Applications · Statistics 2025-02-28 Takuto Iijima , Tomotaka Momozaki , Shuji Ando

Tests for paired censored outcomes have been extensively studied, with some justified in the context of randomization-based inference. These tests are primarily designed to detect an overall treatment effect across the entire follow-up…

Methodology · Statistics 2025-06-10 Sangjin Lee , Kwonsang Lee

Interval censoring occurs when event times are only known to fall between scheduled assessments, a common design in clinical trials, epidemiology, and reliability studies. Standard right-censoring methods, such as Kaplan-Meier and Cox…

Methodology · Statistics 2025-09-04 J. T. Korley

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

Methodology · Statistics 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

The manuscript discusses how to incorporate random effects for quantile regression models for clustered data with focus on settings with many but small clusters. The paper has three contributions: (i) documenting that existing methods may…

Methodology · Statistics 2022-02-24 Maria Laura Battagliola , Helle Sørensen , Anders Tolver , Ana-Maria Staicu