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Auxiliary information is increasingly available from administrative and other data sources, but it is often incomplete and of non-probability origin. We propose a two-step small area estimation approach in which the first step relies on…

Methodology · Statistics 2026-02-16 Donatas Šlevinskas , Ieva Burakauskaitė , Andrius Čiginas

The case-cohort design obtains complete covariate data only on cases and on a random sample (the subcohort) of the entire cohort. Subsequent publications described the use of stratification and weight calibration to increase efficiency of…

Methodology · Statistics 2023-04-10 Lola Etievant , Mitchell H. Gail

A prevalent feature of high-dimensional data is the dependence among covariates, and model selection is known to be challenging when covariates are highly correlated. To perform model selection for the high-dimensional Cox proportional…

Methodology · Statistics 2022-10-04 Pierre Bayle , Jianqing Fan

Survival analysis is a challenging variation of regression modeling because of the presence of censoring, where the outcome measurement is only partially known, due to, for example, loss to follow up. Such problems come up frequently in…

Machine Learning · Computer Science 2022-06-28 Chirag Nagpal , Steve Yadlowsky , Negar Rostamzadeh , Katherine Heller

Although there is an extensive statistical literature showing the disadvantages of discretizing continuous variables, categorization is a common practice in clinical research which results in substantial loss of information. A large…

Methodology · Statistics 2017-08-17 Márcio Augusto Diniz , Mourad Tighiouart , André Rogatko

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

Estimation of heterogeneous treatment effects is an essential component of precision medicine. Model and algorithm-based methods have been developed within the causal inference framework to achieve valid estimation and inference. Existing…

Methodology · Statistics 2021-05-10 Ruohong Li , Honglang Wang , Wanzhu Tu

Electronic Health Records (EHRs) contain extensive patient information that can inform downstream clinical decisions, such as mortality prediction, disease phenotyping, and disease onset prediction. A key challenge in EHR data analysis is…

Applications · Statistics 2026-01-01 Xin Gai , Shiyi Jiang , Anru R. Zhang

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…

Applications · Statistics 2021-02-02 Elizabeth A. Handorf , Marc Smaldone , Sujana Movva , Nandita Mitra

We develop a maximum likelihood estimating approach for time-to-event Weibull regression models with outcome-dependent sampling, where sampling of subjects is dependent on the residual fraction of the time left to developing the event of…

Applications · Statistics 2014-08-01 Brian D. M. Tom , Vernon T. Farewell , Sheila M. Bird

For biological experiments aiming at calibrating models with unknown parameters, a good experimental design is crucial, especially for those subject to various constraints, such as financial limitations, time consumption and physical…

Applications · Statistics 2014-07-22 Xiao Lin , Gabriel Terejanu

Prior to clinical applications, it is critical that risk prediction models are evaluated in independent studies that did not contribute to model development. While prospective cohort studies provide a natural setting for model validation,…

Methodology · Statistics 2017-10-13 Parichoy Pal Choudhury , Anil K. Chaturvedi , Nilanjan Chatterjee

Dynamic treatment regimes (DTRs) are critical to precision medicine, optimizing long-term outcomes through personalized, real-time decision-making in evolving clinical contexts, but require careful supervision for unsafe treatment risks.…

Machine Learning · Computer Science 2025-06-10 Yishan Shen , Yuyang Ye , Hui Xiong , Yong Chen

Adaptive designs are commonly used in clinical and drug development studies for optimum utilization of available resources. In this article, we consider the problem of estimating the effect of the selected (better) treatment using a…

Statistics Theory · Mathematics 2023-01-24 Masihuddin , Neeraj Misra

Randomized controlled trials generate experimental variation that can credibly identify causal effects, but often suffer from limited scale, while observational datasets are large, but often violate desired identification assumptions. To…

Econometrics · Economics 2023-12-27 George Z. Gui

Prognostic models in survival analysis are aimed at understanding the relationship between patients' covariates and the distribution of survival time. Traditionally, semi-parametric models, such as the Cox model, have been assumed. These…

Machine Learning · Statistics 2020-11-06 Denise Rava , Jelena Bradic

Many medical decisions involve the use of dynamic information collected on individual patients toward predicting likely transitions in their future health status. If accurate predictions are developed, then a prognostic mode can identify…

Methodology · Statistics 2018-02-22 Aasthaa Bansal , Patrick J. Heagerty

Biomedical studies that use electronic health records (EHR) data for inference are often subject to bias due to measurement error. The measurement error present in EHR data is typically complex, consisting of errors of unknown functional…

Methodology · Statistics 2020-06-16 Eric J. Oh , Bryan E. Shepherd , Thomas Lumley , Pamela A. Shaw

The extraction of relevant data from Electronic Health Records (EHRs) is crucial to identifying symptoms and automating epidemiological surveillance processes. By harnessing the vast amount of unstructured text in EHRs, we can detect…

Computation and Language · Computer Science 2025-02-10 Juliano Genari , Guilherme Tegoni Goedert

Survival analysis aims to explore the relationship between covariates and the time until the occurrence of an event. The Cox proportional hazards model is commonly used for right-censored data, but it is not strictly limited to this type of…

Methodology · Statistics 2025-07-02 Abdoulaye Dioni , Lynne Moore , Aida Eslami