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Related papers: Effect measures for comparing paired event times

200 papers

To make informed health policy decisions regarding a treatment, we must consider both its cost and its clinical effectiveness. In past work, we introduced the net benefit separation (NBS) as a novel measure of cost-effectiveness. The NBS is…

Methodology · Statistics 2021-01-27 Nicholas Illenberger , Nandita Mitra , Andrew J. Spieker

Factorial analyses offer a powerful nonparametric means to detect main or interaction effects among multiple treatments. For survival outcomes, e.g. from clinical trials, such techniques can be adopted for comparing reasonable…

Methodology · Statistics 2023-02-06 Takeshi Emura , Marc Ditzhaus , Dennis Dobler , Kenta Murotani

The difference in restricted mean survival time (RMST) is a clinically meaningful measure to quantify treatment effect in randomized controlled trials, especially when the proportional hazards assumption does not hold. Several frequentist…

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…

Statistics Theory · Mathematics 2018-07-30 Dennis Dobler , Markus Pauly

This paper addresses the problem of identifying and estimating the causal effect of a treatment in the presence of unmeasured confounding and various types of right-censoring. Examples of these censoring mechanisms are administrative…

Statistics Theory · Mathematics 2025-03-19 Ilias Willems , Sara Rutten , Gilles Crommen , Ingrid Van Keilegom

In randomized trials and observational studies, it is often necessary to evaluate the extent to which an intervention affects a time-to-event outcome, which is only partially observed due to right censoring. For instance, in infectious…

Methodology · Statistics 2024-12-16 Yutong Jin , Peter B. Gilbert , Aaron Hudson

Marginal structural models are a popular tool for investigating the effects of time-varying treatments, but they require an assumption of no unobserved confounders between the treatment and outcome. With observational data, this assumption…

Methodology · Statistics 2021-06-10 Matthew Blackwell , Soichiro Yamauchi

Recent observations, especially in cancer immunotherapy clinical trials with time-to-event outcomes, show that the commonly used proportial hazard assumption is often not justifiable, hampering an appropriate analyse of the data by hazard…

Methodology · Statistics 2021-02-23 Marc Ditzhaus , Menggang Yu , Jin Xu

Objectives: Prior event rate ratio (PERR) is a promising approach to control confounding in observational and real-world evidence research. One of its assumptions is that occurrence of outcome events does not influence later event rate, or…

Methodology · Statistics 2025-07-18 Xiangmei Ma , Chetna Malhotra , Eric Andrew Finkelstein , Yin Bun Cheung

The frequency response function (FRF) is an established way to describe the outcome of experiments in posture control literature. The FRF is an empirical transfer function between an input stimulus and the induced body segment sway profile,…

Signal Processing · Electrical Eng. & Systems 2025-01-31 Vittorio Lippi

In this paper, we propose the use of causal inference techniques for survival function estimation and prediction for subgroups of the data, upto individual units. Tree ensemble methods, specifically random forests were modified for this…

Econometrics · Economics 2018-03-23 Vikas Ramachandra

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…

Methodology · Statistics 2024-12-19 Xuan Wang , Tianxi Cai , Lu Tian , Layla Parast

Background: Effective allocation of limited donor lungs in cystic fibrosis (CF) requires accurate survival predictions, so that high-risk patients may be prioritized for transplantation. In practice, decisions about allocation are made…

Applications · Statistics 2017-06-30 Aasthaa Bansal , Nicole Mayer-Hamblett , Christopher H. Goss , Patrick J. Heagerty

We propose and study a fully efficient method to estimate associations of an exposure with disease incidence when both, incident cases and prevalent cases, i.e. individuals who were diagnosed with the disease at some prior time point and…

Methodology · Statistics 2018-03-20 Marlena Maziarz , Yukun Liu , Jing Qin , Ruth Pfeiffer

Purpose: PFS is often used as a surrogate endpoint for OS in metastatic breast cancer studies. We have evaluated the association of treatment effect on PFS with significant HR$_{OS}$ (and how this association is affected by other factors)…

Applications · Statistics 2020-06-30 Madan G. Kundu , Suddhasatta Acharyya

This paper considers robust modeling of the survival time for cancer patients. Accurate prediction can be helpful for developing therapeutic and care strategies. We propose a unified Expectation-Maximization approach combined with the…

Methodology · Statistics 2019-12-23 Yi Li , Muxuan Liang , Lu Mao , Sijian Wang

Our work was motivated by a recent study on birth defects of infants born to pregnant women exposed to a certain medication for treating chronic diseases. Outcomes such as birth defects are rare events in the general population, which often…

Applications · Statistics 2017-02-24 Ronghui Xu , Jue Hou , Christina D. Chambers

Understanding how treatment effects vary on individual characteristics is critical in the contexts of personalized medicine, personalized advertising and policy design. When the characteristics are of practical interest are only a subset of…

Methodology · Statistics 2023-05-03 Peng Wu , ShaSha Han , Xingwei Tong , Runze Li

The restricted mean survival time (RMST) difference offers an interpretable causal contrast to estimate the treatment effect for time-to-event outcomes, yet a wide range of available estimators leaves limited guidance for practice. We…

Methodology · Statistics 2026-03-02 Charlotte Voinot , Clément Berenfeld , Imke Mayer , Bernard Sebastien , Julie Josse

Background: For RCTs with time-to-event endpoints, proportional hazard (PH) models are typically used to estimate treatment effects and logrank tests are commonly used for hypothesis testing. There is growing support for replacing this…

Methodology · Statistics 2024-12-10 Dominic Magirr , Craig Wang , Xinlei Deng , Tim Morris , Mark Baillie