English
Related papers

Related papers: Analysis and sample size calculation within the re…

200 papers

Determining the sample size of an experiment can be challenging, even more so when incorporating external information via a prior distribution. Such information is increasingly used to reduce the size of the control group in randomized…

Applications · Statistics 2019-07-10 Beat Neuenschwander , Sebastian Weber , Heinz Schmidli , Anthony O'Hagan

Adaptive subgroup enrichment design is an efficient design framework that allows accelerated development for investigational treatments while also having flexibility in population selection within the course of the trial. The adaptive…

Methodology · Statistics 2022-10-21 Liwen Wu , Qing Li , Mengya Liu , Jianchang Lin

Predictive modelling is vital to guide preventive efforts. Whilst large-scale prospective cohort studies and a diverse toolkit of available machine learning (ML) algorithms have facilitated such survival task efforts, choosing the…

Most statistical tests for treatment effects used in randomized clinical trials with survival outcomes are based on the proportional hazards assumption, which often fails in practice. Data from early exploratory studies may provide evidence…

Statistics Theory · Mathematics 2020-05-28 Andrea Arfé , Brian Alexander , Lorenzo Trippa

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

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

Although the survival rate of cancer patients has significantly increased due to advances in anti-cancer therapeutics, one of the major side effects of these therapies, particularly radiotherapy, is the potential manifestation of…

Quantitative Methods · Quantitative Biology 2014-11-07 Kamran Kaveh , Venkata S. K. Manem , Mohammad Kohandel , Siv Sivaloganathan

Composite endpoints that combine multiple outcomes on different scales are common in clinical trials, particularly in chronic conditions. In many of these cases, patients will have to cross a predefined responder threshold in each of the…

Methodology · Statistics 2019-02-20 Martina McMenamin , Jessica K. Barrett , Anna Berglind , James M. S. Wason

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…

Methodology · Statistics 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

In many phase II trials in solid tumours, patients are assessed using endpoints based on the Response Evaluation Criteria in Solid Tumours (RECIST) scale. Often, analyses are based on the response rate. This is the proportion of patients…

Methodology · Statistics 2017-03-02 Chien-Ju Lin , James Wason

The development of targeted therapies, which benefit only a subgroup of patients treated for a given type of cancer, has been extremely attractive to many investigators. Adaptive seamless phase II/III designs in oncology clinical trials…

Applications · Statistics 2016-03-08 Ryuji Uozumi , Chikuma Hamada

Advances in anticancer therapies have significantly contributed to declining death rates in certain disease and clinical settings. However, they have also made it difficult to power a clinical trial in these settings with overall survival…

Methodology · Statistics 2025-12-09 Godwin Yung , Kaspar Rufibach , Marcel Wolbers , Mark Yan , Jue Wang

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

This thesis presents Regenerative Rejection Sampling (RRS), a novel approximate sampling algorithm inspired by classical Rejection Sampling and Markov Chain Monte Carlo methods. The method constructs a continuous-time regenerative process…

Computation · Statistics 2026-04-01 Tommaso Bozzi

Massive sized survival datasets are becoming increasingly prevalent with the development of the healthcare industry. Such datasets pose computational challenges unprecedented in traditional survival analysis use-cases. A popular way for…

Methodology · Statistics 2023-05-09 Nir Keret , Malka Gorfine

Estimating individualized treatment rules is a central task for personalized medicine. [zhao2012estimating] and [zhang2012robust] proposed outcome weighted learning to estimate individualized treatment rules directly through maximizing the…

Methodology · Statistics 2017-10-02 Yifan Cui , Ruoqing Zhu , Michael Kosorok

The European Medicines Agency has in recent years allowed licensing of new pharmaceuticals at an earlier stage in the clinical trial process. When trial evidence is obtained at an early stage, the events of interest, such as disease…

Continuous outcome measurements truncated by death present a challenge for the estimation of unbiased treatment effects in randomized controlled trials (RCTs). One way to deal with such situations is to estimate the survivor average causal…

This study employs a robust analytical framework to uncover patterns in survival outcomes among breast cancer patients from diverse racial and geographical backgrounds. This research uses the SEER 2021 dataset to analyze breast cancer…

Machine Learning · Computer Science 2025-06-10 Ramisa Farha , Joshua O. Olukoya

In meta-analytic modeling, the functional relationship between a primary and surrogate endpoint is estimated using summary data from a set of completed clinical trials. Parameters in the meta-analytic model are used to assess the quality of…

Methodology · Statistics 2025-08-07 James P. Long , Abhishikta Roy , Ehsan Irajizad , Kim-Anh Do , Yu Shen