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Predicting time-to-event outcomes in large databases can be a challenging but important task. One example of this is in predicting the time to a clinical outcome for patients in intensive care units (ICUs), which helps to support critical…

Computation · Statistics 2019-08-06 Yingying Xu , Joon Lee , Joel A. Dubin

In confirmatory clinical trials, survival outcomes are frequently studied and interim analyses for efficacy and/or futility are often desirable. Methods such as the log rank test and Cox regression model are commonly used to compare…

Methodology · Statistics 2024-03-27 Peter Zhang , Brent Logan , Michael Martens

Rhythm is ubiquitous in human culture and in nature, but hard to capture in all its complexity. A key dimension of rhythm, integer ratio categories occur when the relationship between temporal intervals can be expressed as small-integer…

Applications · Statistics 2025-06-23 Yannick Jadoul , Tommaso Tufarelli , Chloé Coissac , Marco Gamba , Andrea Ravignani

We consider studies where multiple measures on an outcome variable are collected over time, but some subjects drop out before the end of follow up. Analyses of such data often proceed under either a 'last observation carried forward' or…

Methodology · Statistics 2022-07-26 Oliver Dukes , David Richardson , Eric Tchetgen Tchetgen

Longitudinal modified treatment policies (LMTP) have been recently developed as a novel method to define and estimate causal parameters that depend on the natural value of treatment. LMTPs represent an important advancement in causal…

Methodology · Statistics 2025-02-06 Iván Díaz , Katherine L Hoffman , Nima S. Hejazi

Analysis of survival data with biased samples caused by left-truncation or length-biased sampling has received extensive interest. Many inference methods have been developed for various survival models. These methods, however, break down…

Statistics Theory · Mathematics 2018-12-31 Li-Pang Chen

An emerging challenge for time-to-event data is studying semi-competing risks, namely when two event times are of interest: a non-terminal event time (e.g. age at disease diagnosis), and a terminal event time (e.g. age at death). The…

Methodology · Statistics 2020-10-12 Daniel Nevo , Malka Gorfine

Many observational studies feature irregular longitudinal data, where the observation times are not common across individuals in the study. Further, the observation times may be related to the longitudinal outcome. In this setting, failing…

Methodology · Statistics 2024-05-27 Grace Tompkins , Joel A Dubin , Michael Wallace

When choosing estimands and estimators in randomized clinical trials, caution is warranted as intercurrent events, such as - due to patients who switch treatment after disease progression, are often extreme. Statistical analyses may then…

Applications · Statistics 2023-03-13 Hege Michiels , An Vandebosch , Stijn Vansteelandt

Attrition in survey and field experiments presents a challenge for social science research. Common approaches to deal with this problem -- such as complete case analysis, multiple imputation, and weighting methods -- rely on strong…

Methodology · Statistics 2026-04-13 Xiangyu Song

In clinical or epidemiological follow-up studies, methods based on time scale indicators such as the restricted mean survival time (RMST) have been developed to some extent. Compared with traditional hazard rate indicator system methods,…

Methodology · Statistics 2022-05-26 Chengfeng Zhang , Hongji Wu , Baoyi Huang , Hao Yuan , Yawen Hou , Zheng Chen

In order to estimate the proportion of `immune' or `cured' subjects who will never experience failure, a sufficiently long follow-up period is required. Several statistical tests have been proposed in the literature for assessing the…

Methodology · Statistics 2026-01-12 Tsz Pang Yuen , Eni Musta

Most statistical tests for treatment effects used in randomized clinical trials with survival outcomes are based on the proportional hazards assumption, which often fails in practice. Data from early exploratory studies may provide evidence…

Statistics Theory · Mathematics 2020-05-28 Andrea Arfé , Brian Alexander , Lorenzo Trippa

In the statistical literature, a number of methods have been proposed to ensure valid inference about marginal effects of variables on a longitudinal outcome in settings with irregular monitoring times. However, the potential biases due to…

Methodology · Statistics 2021-12-23 Janie Coulombe , Erica E M Moodie , Robert W Platt

As more Intensive Care Unit (ICU) data becomes available, the interest in developing clinical prediction models to improve healthcare protocols increases. However, the lack of data quality still hinders clinical prediction using Machine…

Intercurrent events, such as treatment switching, rescue medication, dropout, or truncation by death, frequently complicate intention-to-treat analyses in randomized clinical trials. Existing causal inference frameworks typically target…

Methodology · Statistics 2026-03-12 Georgi Baklicharov , Kelly Van Lancker , Stijn Vansteelandt

Immune checkpoint inhibitor--based therapies often produce heterogeneous survival responses, including early risk, delayed treatment benefit, and durable long-term survival in a subset of patients. In these settings, conventional summary…

Methodology · Statistics 2026-04-28 Yi-Cheng Tai , Weijing Wang , Jedd D. Wolchok , Martin T. Wells

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

Restricted mean survival time (RMST) offers a compelling nonparametric alternative to hazard ratios for right-censored time-to-event data, particularly when the proportional hazards assumption is violated. By capturing the total event-free…

Methodology · Statistics 2025-01-28 Jinghao Sun , Douglas E. Schaubel , Eric J. Tchetgen Tchetgen

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