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In a randomised clinical trial, when the result of the primary endpoint shows a significant benefit, the secondary endpoints are scrutinised to identify additional effects of the treatment. However, this approach entails a risk of…

Recent years have seen the development of many novel scoring tools for disease prognosis and prediction. To become accepted for use in clinical applications, these tools have to be validated on external data. In practice, validation is…

Methodology · Statistics 2022-12-06 Matthias Schmid , Tim Friede , Nadja Klein , Leonie Weinhold

It is of special importance in the clinical trial to compare survival times between the treatment group and the control group. Propensity score methods with a logistic regression model are often used to reduce the effects of confounders.…

Statistics Theory · Mathematics 2024-12-03 Tomoya Baba , Nakahiro Yoshida

In the analysis of survival data, it is usually assumed that any unit will experience the event of interest if it is observed for a sufficient long time. However, one can explicitly assume that an unknown proportion of the population under…

Methodology · Statistics 2014-05-15 Vincent Bremhorst , Philippe Lambert

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

The hazard ratio is routinely used as a summary measure to assess the treatment effect in clinical trials with time-to-event endpoints. It is frequently assumed as constant over time although this assumption often does not hold. When the…

In many application domains, time series are monitored to detect extreme events like technical faults, natural disasters, or disease outbreaks. Unfortunately, it is often non-trivial to select both a time series that is informative about…

Methodology · Statistics 2020-05-01 Erik Scharwächter , Emmanuel Müller

In clinical studies with paired organs, binary outcomes often exhibit intra-subject correlation and may include a mixture of unilateral and bilateral observations. Under Donner's constant correlation model, we develop three likelihood-based…

Methodology · Statistics 2025-10-22 Jia Zhou , Chang-Xing Ma

In epidemiology, identifying the effect of exposure variables in relation to a time-to-event outcome is a classical research area of practical importance. Incorporating propensity score in the Cox regression model, as a measure to control…

Methodology · Statistics 2019-06-11 Yingrui Yang , Molin Wang

Frailty models are often the model of choice for heterogeneous survival data. A frailty model contains both random effects and fixed effects, with the random effects accommodating for the correlation in the data. Different estimation…

Methodology · Statistics 2019-09-17 Oodally Ajmal , Luc Duchateau , Estelle Kuhn

Hybrid clinical trials, that borrow real-world data (RWD), are gaining interest, especially for rare diseases. They assume RWD and randomized control arm be exchangeable, but violations can bias results, inflate type I error, or reduce…

Accelerated life-testing (ALT) is a very useful technique for examining the reliability of highly reliable products. It allows testing the products at higher than usual stress conditions to induce failures more quickly and economically than…

Statistics Theory · Mathematics 2020-05-15 Aida Calviño

The Cox proportional hazards model is the most widely used regression model in univariate survival analysis. Extensions of the Cox model to bivariate survival data, however, remain scarce. We propose two novel extensions based on a…

Methodology · Statistics 2025-11-12 Yael Travis-Lumer , Micha Mandel , Ido Didi Fabian , Rebecca A. Betensky , Malka Gorfine

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…

Applications · Statistics 2025-10-03 Varun Vishwanathan Nair , Victor Miranda Soberanis

A number of biomedical problems require performing many hypothesis tests, with an attendant need to apply stringent thresholds. Often the data take the form of a series of predictor vectors, each of which must be compared with a single…

Methodology · Statistics 2014-05-13 Yi-Hui Zhou , Fred Wright

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

Cardiovascular outcome trials commonly face competing risks when non-CV death prevents observation of major adverse cardiovascular events (MACE). While Cox proportional hazards models treat competing events as independent censoring,…

Methodology · Statistics 2026-02-19 Tuo Wang , Yu Du

Competing risks model time to first event and type of first event. An example from hospital epidemiology is the incidence of hospital-acquired infection, which has to account for hospital discharge of non-infected patients as a competing…

Methodology · Statistics 2013-04-09 Arthur Allignol , Jan Beyersmann , Thomas Gerds , Aurélien Latouche

In oncology the efficacy of novel therapeutics often differs across patient subgroups, and these variations are difficult to predict during the initial phases of the drug development process. The relation between the power of randomized…

Methodology · Statistics 2025-06-05 Boyu Ren , Federico Ferrari , Sandra Fortini , Steffen Ventz , Lorenzo Trippa

Effectiveness of immune-oncology chemotherapies has been presented in recent clinical trials. The Kaplan-Meier estimates of the survival functions of the immune therapy and the control often suggested the presence of the lag-time until the…

Applications · Statistics 2024-07-26 Hisato Sunami , Satoshi Hattori