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Estimating causal effects on time-to-event outcomes from observational data is particularly challenging due to censoring, limited sample sizes, and non-random treatment assignment. The need for answering such "when-if" questions--how the…

Machine Learning · Computer Science 2025-11-19 Jessy Xinyi Han , Devavrat Shah

The use of cumulative incidence functions for characterizing the risk of one type of event in the presence of others has become increasingly popular over the past decade. The problems of modeling, estimation and inference have been treated…

Methodology · Statistics 2021-06-25 Youngjoo Cho , Annette M. Molinaro , Chen Hu , Robert L. Strawderman

In epidemiological research, modeling the cumulative effects of time-dependent exposures on survival outcomes presents a challenge due to their intricate temporal dynamics. Conventional spline-based statistical methods, though effective,…

Machine Learning · Computer Science 2026-01-01 Kang-Chung Yang , Shinsheng Yuan

Composite binary endpoints are increasingly used as primary endpoints in clinical trials. When designing a trial, it is crucial to determine the appropriate sample size for testing the statistical differences between treatment groups for…

Applications · Statistics 2019-01-15 Marta Bofill Roig , Guadalupe Gómez Melis

Medical investigations focusing on patient survival often generate not only a failure time for each patient but also a sequence of measurements on patient health at annual or semi-annual check-ups while the patient remains alive. Such a…

Methodology · Statistics 2016-01-18 Peter McCullagh , Walter Dempsey

In a regression analysis, suppose we suspect that there are several heterogeneous groups in the population that a sample represents. Mixture regression models have been applied to address such problems. By modeling the conditional…

Methodology · Statistics 2013-07-02 Toshiya Hoshikawa

Since survival data occur over time, often important covariates that we wish to consider also change over time. Such covariates are referred as time-dependent covariates. Quantile regression offers flexible modeling of survival data by…

Methodology · Statistics 2014-05-01 Malka Gorfine , Yair Goldberg , Yaacov Ritov

Often in Phase 3 clinical trials measuring a long-term time-to-event endpoint, such as overall survival or progression-free survival, investigators also collect repeated measures on biomarkers which may be predictive of the primary…

Methodology · Statistics 2022-11-30 Abigail J. Burdon , Lisa V. Hampson , Christopher Jennison

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

Quantile regression is useful for characterizing the conditional distribution of a response variable and understanding heterogeneity in the covariate effects at different quantiles. The rise of high-dimensional physiological data in…

Methodology · Statistics 2026-03-25 Yuanzhen Yue , Stella Self , Yichao Wu , Jiajia Zhang , Rahul Ghosal

In the era of precision medicine, time-to-event outcomes such as time to death or progression are routinely collected, along with high-throughput covariates. These high-dimensional data defy classical survival regression models, which are…

Methodology · Statistics 2025-07-15 Stephen Salerno , Yi Li

In stepped wedge cluster randomized trials (SW-CRTs), observations collected under the control condition are, on average, from an earlier time than observations collected under the intervention condition. In a cohort design, participants…

Methodology · Statistics 2023-02-23 Jale Basten , Katja Ickstadt , Nina Timmesfeld

Covariate imbalance between treatment groups makes it difficult to compare cumulative incidence curves in competing risk analyses. In this paper we discuss different methods to estimate adjusted cumulative incidence curves including inverse…

Causal inference with time-to-event outcomes is fundamental in various scientific studies. In a static setup with fitted propensity scores, weighted Kaplan-Meier estimation for survival probabilities and weighted Breslow-Peto estimation for…

Methodology · Statistics 2026-05-18 Wenfu Xu , Yi Zhang , Tobias Gerhard , Zhiqiang Tan

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

Post-randomization events, also known as intercurrent events, such as treatment noncompliance and censoring due to a terminal event, are common in clinical trials. Principal stratification is a framework for causal inference in the presence…

Methodology · Statistics 2023-01-19 Bo Liu , Lisa Wruck , Fan Li

Cluster randomization trials commonly employ multiple endpoints. When a single summary of treatment effects across endpoints is of primary interest, global hypothesis testing/effect estimation methods represent a common analysis strategy.…

Methodology · Statistics 2025-05-19 E. Davies Smith , V. Jairath , G. Zou

Identifying and characterizing relationships between treatments, exposures, or other covariates and time-to-event outcomes has great significance in a wide range of biomedical settings. In research areas such as multi-center clinical…

Methodology · Statistics 2025-04-02 Hillary M. Heiling , Naim U. Rashid , Quefeng Li , Xianlu L. Peng , Jen Jen Yeh

The mean residual life function is a key functional for a survival distribution. It has a practically useful interpretation as the expected remaining lifetime given survival up to a particular time point, and it also characterizes the…

Methodology · Statistics 2018-10-11 Valerie Poynor , Athanasios Kottas

In causal inference, it is common to estimate the causal effect of a single treatment variable on an outcome. However, practitioners may also be interested in the effect of simultaneous interventions on multiple covariates of a fixed target…

Methodology · Statistics 2022-11-24 Jaime Roquero Gimenez , Dominik Rothenhäusler
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