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The widely used proportional hazard assumption cannot be assessed reliably in small-scale clinical trials and might often in fact be unjustified, e.g. due to delayed treatment effects. An alternative to the hazard ratio as effect measure is…

Methodology · Statistics 2024-08-29 David Jesse , Cynthia Huber , Tim Friede

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

Comparing the survival times among two groups is a common problem in time-to-event analysis, for example if one would like to understand whether one medical treatment is superior to another. In the standard survival analysis setting, there…

Methodology · Statistics 2023-07-07 Dennis Dobler , Eni Musta

Restricted mean survival time (RMST) is gaining attention as a measure to quantify the treatment effect on survival outcomes in randomized clinical trials. Several methods to determine sample size based on the RMST-based tests have been…

Methodology · Statistics 2022-12-19 Satoshi Hattori , Hajime Uno

Interval-censored data analysis is important in biomedical statistics for any type of time-to-event response where the time of response is not known exactly, but rather only known to occur between two assessment times. Many clinical trials…

Methodology · Statistics 2019-06-12 Weichi Yao , Halina Frydman , Jeffrey S. Simonoff

In clinical studies, the illness-death model is often used to describe disease progression. A subject starts disease-free, may develop the disease and then die, or die directly. In clinical practice, disease can only be diagnosed at…

Methodology · Statistics 2026-02-02 Marta Spreafico , Anja J. Rueten-Budde , Hein Putter , Marta Fiocco

In the process of clinical diagnosis and treatment, the restricted mean survival time (RMST), which reflects the life expectancy of patients up to a specified time, can be used as an appropriate outcome measure. However, the RMST only…

Applications · Statistics 2021-06-22 Zijing Yang , Hongji Wu , Yawen Hou , Hao Yuan , Zheng Chen

Investigating the causal relationship between exposure and the time-to-event outcome is an important topic in biomedical research. Previous literature has discussed the potential issues of using the hazard ratio as a marginal causal effect…

Methodology · Statistics 2022-05-05 Zihan Lin , Ai Ni , Bo Lu

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

For right censored survival data, it is well known that the mean survival time can be consistently estimated when the support of the censoring time contains the support of the survival time. In practice, however, this condition can be…

Statistics Theory · Mathematics 2013-08-01 Ying Ding , Bin Nan

Non-parametric maximum likelihood estimation encompasses a group of classic methods to estimate distribution-associated functions from potentially censored and truncated data, with extensive applications in survival analysis. These methods,…

Methodology · Statistics 2021-08-05 Justin D. Tubbs , Lane Guolan Chen , Thuan Quoc Thach , Pak C. Sham

The distribution-free method of conformal prediction (Vovk et al, 2005) has gained considerable attention in computer science, machine learning, and statistics. Candes et al. (2023) extended this method to right-censored survival data,…

Methodology · Statistics 2025-06-04 Jing Qin , Jin Piao , Jing Ning , Yu Shen

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

Desirability Of Outcome Ranking (DOOR) methodology accounts for problems that conventional benefit:risk analyses in clinical trials ignore, such as competing risks and the trade-off relationship between efficacy and toxicity. DOOR levels…

Methodology · Statistics 2025-05-19 Shiyu Shu , Guoqing Diao , Toshimitsu Hamasaki , Scott Evans

In the context of right-censored and interval-censored data we develop asymptotic formulas to compute pseudo-observations for the survival function and the Restricted Mean Survival Time (RMST). Those formulas are based on the original…

Statistics Theory · Mathematics 2023-02-07 Olivier Bouaziz

We conducted a systematic comparison of statistical methods used for the analysis of time-to-event outcomes under various proportional and nonproportional hazard (NPH) scenarios. Our study used data from recently published oncology trials…

Applications · Statistics 2025-02-12 Xinyu Zhang , Erich J. Greene , Ondrej Blaha , Wei Wei

In the context of right-censored data, we study the problem of predicting the restricted time to event based on a set of covariates. Under a quadratic loss, this problem is equivalent to estimating the conditional Restricted Mean Survival…

Statistics Theory · Mathematics 2024-04-29 Ariane Cwiling , Vittorio Perduca , Olivier Bouaziz

Survival time prediction from medical images is important for treatment planning, where accurate estimations can improve healthcare quality. One issue affecting the training of survival models is censored data. Most of the current survival…

Computer Vision and Pattern Recognition · Computer Science 2022-05-27 Renato Hermoza , Gabriel Maicas , Jacinto C. Nascimento , Gustavo Carneiro

Restricted mean survival time (RMST) models have gained popularity when analyzing time-to-event outcomes because RMST models offer more straightforward interpretations of treatment effects with fewer assumptions than hazard ratios commonly…

Methodology · Statistics 2023-10-23 Kaiyuan Hua , Xiaofei Wang , Hwanhee Hong

Uncertainty quantification of prediction models through prediction sets is increasingly popular and successful, but most existing methods rely on directly observing the outcome and do not appropriately handle censored outcomes, such as…

Methodology · Statistics 2025-05-06 Wenwen Si , Hongxiang Qiu