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In oncology, conduct well-powered time-to-event randomized clinical trials may be challenging due to limited patietns number. Many designs for single-arm trials (SATs) have recently emerged as an alternative to overcome this issue. They…

应用统计 · 统计学 2026-04-10 Chloé Szurewsky , Guosheng Yin , Gwénaël Le Teuff

This work bridges the gap between staggered adoption designs and survival analysis to estimate causal effects in settings with time-varying treatments, addressing a fundamental challenge in medical research exemplified by the Stanford Heart…

统计方法学 · 统计学 2025-03-04 Xiang Meng , Iavor Bojinov

This paper introduces new effect parameters for factorial survival designs with possibly right-censored time-to-event data. In the special case of a two-sample design it coincides with the concordance or Wilcoxon parameter in survival…

统计理论 · 数学 2018-07-30 Dennis Dobler , Markus Pauly

A treatment regime is a deterministic function that dictates personalized treatment based on patients' individual prognostic information. There is a fast-growing interest in finding optimal treatment regimes to maximize expected long-term…

统计理论 · 数学 2016-11-25 Runchao Jiang , Wenbin Lu , Rui Song , Marie Davidian

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…

机器学习 · 统计学 2025-07-30 Tomer Meir , Uri Shalit , Malka Gorfine

In lifetime data, like cancer studies, theremay be long term survivors, which lead to heavy censoring at the end of the follow-up period. Since a standard survival model is not appropriate to handle these data, a cure model is needed. In…

统计方法学 · 统计学 2024-01-31 Ana López-Cheda , M. Amalia Jácome , Ingrid Van Keilegom , Ricardo Cao

Count outcomes in longitudinal studies are frequent in clinical and engineering studies. In frequentist and Bayesian statistical analysis, methods such as Mixed linear models allow the variability or correlation within individuals to be…

统计方法学 · 统计学 2024-07-15 Alejandra Estefanía Patiño Hoyos , Johnatan Cardona Jiménez

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,…

统计方法学 · 统计学 2022-05-26 Chengfeng Zhang , Hongji Wu , Baoyi Huang , Hao Yuan , Yawen Hou , Zheng Chen

Lung cancer remains one of the leading causes of cancer-related mortality, yet most survival models rely only on baseline factors and overlook posttreatment variables that reflect disease progression. To address this gap, we applied Cox…

应用统计 · 统计学 2025-10-03 Varun Vishwanathan Nair , Victor Miranda Soberanis

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…

Researchers in genetics and other life sciences commonly use permutation tests to evaluate differences between groups. Permutation tests have desirable properties, including exactness if data are exchangeable, and are applicable even when…

统计计算 · 统计学 2018-11-01 Brian Segal , Thomas Braun , Michael Elliott , Hui Jiang

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…

统计计算 · 统计学 2019-08-06 Yingying Xu , Joon Lee , Joel A. Dubin

No unmeasured confounding is often assumed in estimating treatment effects in observational data when using approaches such as propensity scores and inverse probability weighting. However, in many such studies due to the limitation of the…

应用统计 · 统计学 2019-08-06 Rong Huang , Ronghui Xu , Parambir S. Dulai

With known cause of death (CoD), competing risk survival methods are applicable in estimating disease-specific survival. Relative survival analysis may be used to estimate disease-specific survival when cause of death is either unknown or…

统计方法学 · 统计学 2021-12-10 Reuben Adatorwovor , Aurelien Latouche , Jason P. Fine

Clinical trials or studies oftentimes require long-term and/or costly follow-up of participants to evaluate a novel treatment/drug/vaccine. There has been increasing interest in the past few decades in using short-term surrogate outcomes as…

统计方法学 · 统计学 2024-12-19 Xuan Wang , Tianxi Cai , Lu Tian , Layla Parast

This article studies the estimation of the causal effect of a time-varying treatment on time-to-an-event or on some other continuously distributed outcome. The paper applies to the situation where treatment is repeatedly adapted to…

统计理论 · 数学 2008-06-19 Judith J. Lok

Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

统计方法学 · 统计学 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

Comparisons of different treatments or production processes are the goals of a significant fraction of applied research. Unsurprisingly, two-sample problems play a main role in Statistics through natural questions such as `Is the the new…

统计方法学 · 统计学 2017-09-05 P. C. Álvarez-Esteban , E. del Barrio , J. A. Cuesta-Albertos , C. Matrán

We consider after-study statistical inference for sequentially designed experiments wherein multiple units are assigned treatments for multiple time points using treatment policies that adapt over time. Our goal is to provide inference…

机器学习 · 统计学 2025-06-10 Raaz Dwivedi , Katherine Tian , Sabina Tomkins , Predrag Klasnja , Susan Murphy , Devavrat Shah

Mediation analysis for survival outcomes is challenging. Most existing methods quantify the treatment effect using the hazard ratio (HR) and attempt to decompose the HR into the direct effect of treatment plus an indirect, or mediated,…

统计方法学 · 统计学 2025-02-27 Alex Ocampo , Enrico Giudice , Dieter A. Häring , Baldur Magnusson , Theis Lange , Zachary R. McCaw