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The hazard ratio from the Cox proportional hazards model is a ubiquitous summary of treatment effect. However, when hazards are non-proportional, the hazard ratio can lose a stable causal interpretation and become study-dependent because it…

统计方法学 · 统计学 2026-02-17 Xiang Meng , Lu Tian , Kenneth Kehl , Hajime Uno

Delayed treatment effects on time-to-event outcomes have often been observed in randomized controlled studies of cancer immunotherapies. In the case of delayed onset of treatment effect, the conventional test/estimation approach using the…

统计方法学 · 统计学 2024-03-19 Miki Horiguchi , Lu Tian , Kenneth L. Kehl , Hajime Uno

The average hazard (AH), recently introduced by Uno and Horiguchi, represents a novel summary metric of event time distributions, conceptualized as the general censoring-free average person-time incidence rate on a given time window,…

应用统计 · 统计学 2024-07-02 Hong Xiong , Jean Connors , Deb Schrag , Hajime Uno

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

In a stratified clinical trial design with time to event end points, stratification factors are often accounted for the log-rank test and the Cox regression analyses. In this work, we have evaluated the impact of inclusion of stratification…

应用统计 · 统计学 2020-06-30 Madan G. Kundu , Shoubhik Mondal

Meta-analyses of survival studies aim to reveal the variation of an effect measure of interest over different studies and present a meaningful summary. They must address between study heterogeneity in several dimensions and eliminate…

统计方法学 · 统计学 2021-11-29 Joris Hautekiet , Elena-Sophie Prigge , Theresa Obermueller , Marc Arbyn , Els Goetghebeur

The Hazard Ratio (HR) is often reported as the main causal effect when studying survival data. Despite its popularity, the HR suffers from an unclear causal interpretation. As already pointed out in the literature, there is a built-in…

统计方法学 · 统计学 2022-03-08 Rachel Axelrod , Daniel Nevo

One of the most common ways researchers compare survival outcomes across treatments when confounding is present is using Cox regression. This model is limited by its underlying assumption of proportional hazards; in some cases, substantial…

应用统计 · 统计学 2021-02-02 Elizabeth A. Handorf , Marc Smaldone , Sujana Movva , Nandita Mitra

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…

应用统计 · 统计学 2025-02-12 Xinyu Zhang , Erich J. Greene , Ondrej Blaha , Wei Wei

Randomized controlled trials are the standard method for estimating causal effects, ensuring sufficient statistical power and confidence through adequate sample sizes. However, achieving such sample sizes is often challenging. This study…

统计方法学 · 统计学 2025-03-28 Keisuke Hanada , Masahiro Kojima

The treatment effects of the same therapy observed from multiple clinical trials can often be very different. Yet the patient characteristics accounting for these differences may not be identifiable in real world practice. There needs to be…

统计理论 · 数学 2015-03-20 Tinghui Yu , Yabing Mai , Sherry Liu , Xiaofei Hu

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…

统计方法学 · 统计学 2020-11-02 Federico Ambrogi , Simona Iacobelli , Per Kragh Andersen

This paper studies covariate adjusted estimation of the average treatment effect in stratified experiments. We work in a general framework that includes matched tuples designs, coarse stratification, and complete randomization as special…

计量经济学 · 经济学 2024-07-23 Max Cytrynbaum

This research was motivated by studying anti-drug antibody (ADA) formation and its potential impact on long-term benefit of a biologic treatment in a randomized controlled trial, in which ADA status was not only unobserved in the control…

统计方法学 · 统计学 2021-01-13 Shengchun Kong , Dominik Heinzmann , Sabine Lauer , Tian Lu

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…

统计方法学 · 统计学 2023-01-19 Bo Liu , Lisa Wruck , Fan Li

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…

应用统计 · 统计学 2014-10-17 Andrew Wey , David Vock , John Connett , Kyle Rudser

While well-established methods for time-to-event data are available when the proportional hazards assumption holds, there is no consensus on the best inferential approach under non-proportional hazards (NPH). However, a wide range of…

The Cox regression model and its associated hazard ratio (HR) are frequently used for summarizing the effect of treatments on time to event outcomes. However, the HR's interpretation strongly depends on the assumed underlying survival…

统计方法学 · 统计学 2021-08-10 Pablo Martinez-Camblor , Todd A. MacKenzie , A. James O'Malley

The survey experiment is widely used in economics and social sciences to evaluate the effects of treatments or programs. In a standard population-based survey experiment, the experimenter randomly draws experimental units from a target…

统计方法学 · 统计学 2026-05-11 Pengfei Tian , Jiyang Ren , Yingying Ma

Randomized experiments are the gold standard for investigating causal relationships, with comparisons of potential outcomes under different treatment groups used to estimate treatment effects. However, outcomes with heavy-tailed…

统计方法学 · 统计学 2024-07-09 Hongzi Li , Wei Ma , Yingying Ma , Hanzhong Liu
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