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A two-stage adaptive optimal design is an attractive option for increasing the efficiency of clinical trials. In these designs, based on interim data, the locally optimal dose is chosen for further exploration, which induces dependencies…

Methodology · Statistics 2019-05-24 Zhantao Lin , Nancy Flournoy , William F. Rosenberger

Large observational datasets, including those derived from electronic health records, are a valuable resource for medical research but are often affected by missingness, measurement error, and misclassification. Two-phase sampling with…

Methodology · Statistics 2026-03-23 Jasper B. Yang , Bryan E. Shepherd , Thomas Lumley , Pamela A. Shaw

Competing risk analysis considers event times due to multiple causes, or of more than one event types. Commonly used regression models for such data include 1) cause-specific hazards model, which focuses on modeling one type of event while…

Applications · Statistics 2017-04-27 Jiayi Hou , Anthony Paravati , Ronghui Xu , James Murphy

Electronic health records (EHRs) are invaluable for clinical research, yet privacy concerns severely restrict data sharing. Synthetic data generation offers a promising solution, but EHRs present unique challenges: they contain both…

Machine Learning · Computer Science 2026-03-26 Shaonan Liu , Yuichiro Iwashita , Soichiro Nakako , Masakazu Iwamura , Koichi Kise

In many areas of systems biology, including virology, pharmacokinetics, and population biology, dynamical systems are commonly used to describe biological processes. These systems can be characterized by estimating their parameters from…

Machine Learning · Statistics 2025-11-11 Tuan Minh Ha , Binh Thanh Nguyen , Lam Si Tung Ho

In biomedical studies, it is often desirable to characterize the interactive mode of multiple disease outcomes beyond their marginal risk. Ising model is one of the most popular choices serving for this purpose. Nevertheless, learning…

Methodology · Statistics 2023-11-28 Daiqing Wu , Molei Liu

Survival analysis, or time-to-event analysis, is an important and widespread problem in healthcare research. Medical research has traditionally relied on Cox models for survival analysis, due to their simplicity and interpretability. Cox…

Machine Learning · Computer Science 2023-10-25 Mike Van Ness , Tomas Bosschieter , Natasha Din , Andrew Ambrosy , Alexander Sandhu , Madeleine Udell

A common problem in health research is that we have a large database with many variables measured on a large number of individuals. We are interested in measuring additional variables on a subsample; these measurements may be newly…

Methodology · Statistics 2022-03-22 Thomas Lumley , Tong Chen

Routinely collected data from electronic health records (EHR) provide opportunities to study effects of longitudinal treatment strategies in real-world clinical settings. A challenge presented by EHR data is that frequency of covariate…

Applications · Statistics 2026-04-14 Leah Pirondini , Karla Diaz-Ordaz , Edward Palmer , Ruth H. Keogh

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

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…

In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

Methodology · Statistics 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

The two-phase sampling design is a cost-efficient way of collecting expensive covariate information on a judiciously selected subsample. It is natural to apply such a strategy for collecting genetic data in a subsample enriched for exposure…

Applications · Statistics 2013-05-27 Jaeil Ahn , Bhramar Mukherjee , Stephen B. Gruber , Malay Ghosh

Discrete biomarkers derived as cell densities or counts from tissue microarrays and immunostaining are widely used to study immune signatures in relation to survival outcomes in cancer. Although routinely collected, these signatures are not…

Motivated by multi-center biomedical studies that cannot share individual data due to privacy and ownership concerns, we develop communication-efficient iterative distributed algorithms for estimation and inference in the high-dimensional…

Methodology · Statistics 2024-06-25 Pierre Bayle , Jianqing Fan , Zhipeng Lou

Survival time prediction from medical images is important for treatment planning, where accurate estimations can improve healthcare quality. One issue affecting the training of survival models is censored data. Most of the current survival…

Computer Vision and Pattern Recognition · Computer Science 2022-05-27 Renato Hermoza , Gabriel Maicas , Jacinto C. Nascimento , Gustavo Carneiro

With the increasing availability of electronic health records (EHR) linked with biobank data for translational research, a critical step in realizing its potential is to accurately classify phenotypes for patients. Existing approaches to…

Methodology · Statistics 2024-04-02 Molei Liu , Xinyi Wang , Chuan Hong

The health effects of environmental exposures have been studied for decades, typically using standard regression models to assess exposure-outcome associations found in observational non-experimental data. We propose and illustrate a…

Applications · Statistics 2017-09-20 Marie-Abele C. Bind , Donald B. Rubin

In clinical studies, the illness-death model is often used to describe disease progression. A subject starts disease-free, may develop the disease and then die, or die directly. In clinical practice, disease can only be diagnosed at…

Methodology · Statistics 2026-02-02 Marta Spreafico , Anja J. Rueten-Budde , Hein Putter , Marta Fiocco

Electronic medical records (EMR) contain longitudinal information about patients that can be used to analyze outcomes. Typically, studies on EMR data have worked with established variables that have already been acknowledged to be…

Machine Learning · Computer Science 2017-11-30 Prithwish Chakraborty , Vishrawas Gopalakrishnan , Sharon M. H. Alford , Faisal Farooq
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