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Survival analysis often relies on Cox models, assuming both linearity and proportional hazards (PH). This study evaluates machine and deep learning methods that relax these constraints, comparing their performance with penalized Cox models…

Machine Learning · Computer Science 2025-10-21 Ivan Rossi , Flavio Sartori , Cesare Rollo , Giovanni Birolo , Piero Fariselli , Tiziana Sanavia

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…

Applications · Statistics 2026-04-10 Chloé Szurewsky , Guosheng Yin , Gwénaël Le Teuff

A fundamental concept in two-arm non-parametric survival analysis is the comparison of observed versus expected numbers of events on one of the treatment arms (the choice of which arm is arbitrary), where the expectation is taken assuming…

Applications · Statistics 2020-07-10 Dominic Magirr

When comparing multiple groups in clinical trials, we are not only interested in whether there is a difference between any groups but rather the location. Such research questions lead to testing multiple individual hypotheses. To control…

Non-proportional hazards (NPH) have been observed in confirmatory clinical trials with time to event outcomes. Under NPH, the hazard ratio does not stay constant over time and the log-rank test is no longer the most powerful test. The…

Methodology · Statistics 2022-09-26 Bharati Kumar , Jonathan W. Bartlett

The conventional nonparametric tests in survival analysis, such as the log-rank test, assess the null hypothesis that the hazards are equal at all times. However, hazards are hard to interpret causally, and other null hypotheses are more…

Methodology · Statistics 2019-01-29 Mats Julius Stensrud , Kjetil Røysland , Pål Christie Ryalen

Proportional hazards are a common assumption when designing confirmatory clinical trials in oncology. This assumption not only affects the analysis part but also the sample size calculation. The presence of delayed effects causes a change…

Methodology · Statistics 2018-12-11 Jose L Jimenez , Viktoriya Stalbovskaya , Byron Jones

We present a new way of testing ordered hypotheses against all alternatives which overpowers the classical approach both in simplicity and statistical power. Our new method tests the constrained likelihood ratio statistic against the…

Methodology · Statistics 2018-06-26 Diaa Al Mohamad , Jelle J. Goeman , Erik W. van Zwet , Eric A. Cator

In this paper we propose a Multiple kernel testing procedure to infer survival data when several factors (e.g. different treatment groups, gender, medical history) and their interaction are of interest simultaneously. Our method is able to…

Methodology · Statistics 2022-06-16 Marc Ditzhaus , Tamara Fernández , Nicolás Rivera

We revisit the question of what randomization ratio (RR) maximizes power of the logrank test in event-driven survival trials under proportional hazards (PH). By comparing three approximations of the logrank test (Schoenfeld, Freedman,…

Methodology · Statistics 2025-05-20 Godwin Yung , Kaspar Rufibach , Marcel Wolbers , Ray Lin , Yi Liu

A formal likelihood ratio hypothesis test for the validity of a parametric regression function is proposed, using a large-dimensional, nonparametric double cone alternative. For example, the test against a constant function uses the…

Methodology · Statistics 2014-06-30 Bodhisattva Sen , Mary Meyer

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…

Methodology · Statistics 2024-03-19 Miki Horiguchi , Lu Tian , Kenneth L. Kehl , Hajime Uno

Two common concerns raised in analyses of randomized experiments are (i) appropriately handling issues of non-compliance, and (ii) appropriately adjusting for multiple tests (e.g., on multiple outcomes or subgroups). Although simple…

Methodology · Statistics 2016-05-25 Joseph J. Lee , Laura Forastiere , Luke Miratrix , Natesh S. Pillai

There is a considerable literature in case-control logistic regression on whether or not non-confounding covariates should be adjusted for. However, only limited and ad hoc theoretical results are available on this important topic. A…

Statistics Theory · Mathematics 2023-05-19 Siliang Zhang , Jinbo Chen , Zhiliang Ying , Hong Zhang

Weighted logrank tests are a popular tool for analyzing right censored survival data from two independent samples. Each of these tests is optimal against a certain hazard alternative, for example the classical logrank test for proportional…

Statistics Theory · Mathematics 2018-07-17 Marc Ditzhaus , Sarah Friedrich

In model checking for regressions, nonparametric estimation-based tests usually have tractable limiting null distributions and are sensitive to oscillating alternative models, but suffer from the curse of dimensionality. In contrast,…

Methodology · Statistics 2019-03-12 Lingzhu Li , Xuehu Zhu , Lixing Zhu

For some variants of regression models, including partial, measurement error or error-in-variables, latent effects, semi-parametric and otherwise corrupted linear models, the classical parametric tests generally do not perform well. Various…

Statistics Theory · Mathematics 2015-03-25 Pranab K. Sen , Jana Jureckova , Jan Picek

In confirmatory cancer clinical trials, overall survival (OS) is normally a primary endpoint in the intention-to-treat (ITT) analysis under regulatory standards. After the tumor progresses, it is common that patients allocated to the…

Methodology · Statistics 2022-01-19 José L. Jiménez , Julia Niewczas , Alexander Bore , Carl-Fredrik Burman

Nonparametric tests provide robust and powerful alternatives to the corresponding least squares methods. There are two approaches to nonparametric pairwise comparisons of treatment effects, the method based on pairwise rankings and the…

Methodology · Statistics 2018-02-13 Hossein Mansouri , Fangyuan Zhang

We propose a class of two-sample statistics for testing the equality of proportions and the equality of survival functions. We build our proposal on a weighted combination of a score test for the difference in proportions and a Weighted…

Methodology · Statistics 2021-12-08 Marta Bofill Roig , Guadalupe Gómez Melis