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Suppose one wishes to estimate the effect of a binary treatment on a binary endpoint conditional on a post-randomization quantity in a counterfactual world in which all subjects received treatment. It is generally difficult to identify this…

Methodology · Statistics 2019-11-12 Alex Luedtke , Jiacheng Wu

Conventional treatment policies map patient covariates to a single recommended intervention in order to maximize expected clinical outcomes. Although a rich body of causal inference methods has been developed to estimate such policies,…

Machine Learning · Computer Science 2026-05-20 Laura Fuentes-Vicente , Mathieu Even , Gaëlle Dormion , Antoine Chambaz , Uri Shalit , Julie Josse

An individualized treatment rule (ITR) is a decision rule that aims to improve individual patients health outcomes by recommending optimal treatments according to patients specific information. In observational studies, collected data may…

Methodology · Statistics 2023-10-03 Zeyu Bian , Erica EM Moodie , Susan M Shortreed , Sylvie D Lambert , Sahir Bhatnagar

Biomarker-guided designs are increasingly used to evaluate personalized treatments based on patients' biomarker status in Phase II and III clinical trials. With adaptive enrichment, these designs can improve the efficiency of evaluating the…

Methodology · Statistics 2024-06-11 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

In biomedical Subgroup Discovery, practitioners are interested in discovering interpretable and homogeneous subgroups within a group of patients. In this paper, assuming that healthy subjects (i.e., controls) share common but irrelevant…

Machine Learning · Computer Science 2026-05-21 Robin Louiset , Edouard Duchesnay , Benoit Dufumier , Antoine Grigis , Pietro Gori

Biomarker measurements can be relatively easy and quick to obtain and they are useful to investigate whether a compound works as intended on a mechanistic, pharmacological level. In some situations, it is realistic to assume that patients,…

Methodology · Statistics 2018-06-26 Björn Bornkamp , Georgina Bermann

In the era of precision medicine, more and more clinical trials are now driven or guided by biomarkers, which are patient characteristics objectively measured and evaluated as indicators of normal biological processes, pathogenic processes,…

Methodology · Statistics 2024-01-02 Hong Zhang , Jie Pu , Shibing Deng , Satrajit Roychoudhury , Haitao Chu , Douglas Robinson

In healthcare applications, predictive uncertainty has been used to assess predictive accuracy. In this paper, we demonstrate that predictive uncertainty estimated by the current methods does not highly correlate with prediction error by…

Machine Learning · Computer Science 2021-07-08 Shi Hu , Nicola Pezzotti , Max Welling

In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

Methodology · Statistics 2018-07-11 Debashis Ghosh , Youngjoo Cho

Deep Learning (DL) can predict biomarkers from cancer histopathology. Several clinically approved applications use this technology. Most approaches, however, predict categorical labels, whereas biomarkers are often continuous measurements.…

With rapid advances in information technology, massive datasets are collected in all fields of science, such as biology, chemistry, and social science. Useful or meaningful information is extracted from these data often through statistical…

Methodology · Statistics 2021-09-22 Wenxuan Zhong , Yiwen Liu , Peng Zeng

Consider a situation where a new patient arrives in the Intensive Care Unit (ICU) and is monitored by multiple sensors. We wish to assess relevant unmeasured physiological variables (e.g., cardiac contractility and output and vascular…

Machine Learning · Statistics 2022-02-17 Ron Teichner , Ron Meir , Danny Eitan

We propose a Bayesian model selection approach that allows medical practitioners to select among predictor variables while taking their respective costs into account. Medical procedures almost always incur costs in time and/or money. These…

Methodology · Statistics 2024-05-07 Erica M. Porter , Christopher T. Franck , Stephen Adams

Discovery of novel protein biomarkers for clinical applications is an active research field across a manifold of diseases. Despite some successes and progress, the biomarker development pipeline still frequently ends in failure as biomarker…

Biomolecules · Quantitative Biology 2025-05-01 Katharina Waury

We introduce a new multiple type I error criterion for clinical trials with multiple populations. Such trials are of interest in precision medicine where the goal is to develop treatments that are targeted to specific sub-populations…

Methodology · Statistics 2021-02-05 Werner Brannath , Charlie Hillner , Kornelius Rohmeyer

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

Methodology · Statistics 2020-08-11 Yun Li , Irina Bondarenko , Michael R. Elliott , Timothy P. Hofer , Jeremy M. G. Taylor

One fundamental statistical question for research areas such as precision medicine and health disparity is about discovering effect modification of treatment or exposure by observed covariates. We propose a semiparametric framework for…

Methodology · Statistics 2020-08-04 Muxuan Liang , Menggang Yu

We propose a novel adaptive design for clinical trials with time-to-event outcomes and covariates (which may consist of or include biomarkers). Our method is based on the expected entropy of the posterior distribution of a proportional…

Applications · Statistics 2016-03-29 James E. Barrett

This study examines the application of Bayesian approach in the context of clinical trials, emphasizing their increasing importance in contemporary biomedical research. While conventional frequentist approach provides a foundational basis…

Methodology · Statistics 2026-01-16 Paramahansa Pramanik , Arnab Kumar Maity , Anjan Mandal , Haley Kate Robinson

Precision medicine leverages patient heterogeneity to estimate individualized treatment regimens, formalized, data-driven approaches designed to match patients with optimal treatments. In the presence of competing events, where multiple…

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