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Across domains such as medicine, employment, and criminal justice, predictive models often target labels that imperfectly reflect the outcomes of interest to experts and policymakers. For example, clinical risk assessments deployed to…

Machine Learning · Computer Science 2023-05-19 Luke Guerdan , Amanda Coston , Kenneth Holstein , Zhiwei Steven Wu

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

Methodology · Statistics 2026-01-22 Xinyuan Chen , Fan Li

Estimating treatment effects from observational data is of central interest across numerous application domains. Individual treatment effect offers the most granular measure of treatment effect on an individual level, and is the most useful…

Machine Learning · Statistics 2024-08-06 Hengrui Cai , Huaqing Jin , Lexin Li

In this study, we present a novel clinical decision support system and discuss its interpretability-related properties. It combines a decision set of rules with a machine learning scheme to offer global and local interpretability. More…

Methodology · Statistics 2021-07-16 Francisco Valente , Simão Paredes , Jorge Henriques

Propensity scores are commonly used to reduce the confounding bias in non-randomized observational studies for estimating the average treatment effect. An important assumption underlying this approach is that all confounders that are…

Methodology · Statistics 2022-08-02 Youfei Yu , Jiacong Du , Min Zhang , Zhenke Wu , Andrew M. Ryan , Bhramar Mukherjee

Predicting risks of chronic diseases has become increasingly important in clinical practice. When a prediction model is developed in a given source cohort, there is often a great interest to apply the model to other cohorts. However, due to…

Methodology · Statistics 2020-03-05 Zheng Jiayin , Zheng Yingye , Hsu Li

Individualizing treatment assignment can improve outcomes for diseases with patient-to-patient variability in comparative treatment effects. When a clinical trial demonstrates that some patients improve on treatment while others do not, it…

Methodology · Statistics 2022-11-02 Nina Galanter , Marco Carone , Ronald C. Kessler , Alex Luedtke

The amount of data collected from patients involved in clinical trials is continuously growing. All patient characteristics are potential covariates that could be used to improve clinical trial analysis and power. However, the restricted…

Methodology · Statistics 2021-03-29 Samuel Branders , Alvaro Pereira , Guillaume Bernard , Marie Ernst , Adelin Albert

In several medical decision-making problems, such as antibiotic prescription, laboratory testing can provide precise indications for how a patient will respond to different treatment options. This enables us to "fully observe" all potential…

Machine Learning · Computer Science 2020-08-14 Soorajnath Boominathan , Michael Oberst , Helen Zhou , Sanjat Kanjilal , David Sontag

Doctors use statistics to advance medical knowledge; we use a medical analogy to introduce statistical inference "from scratch" and to highlight an improvement. Your doctor, perhaps implicitly, predicts the effectiveness of a treatment for…

Methodology · Statistics 2015-11-29 Keli Liu , Xiao-Li Meng

Disease prediction holds considerable significance in modern healthcare, because of its crucial role in facilitating early intervention and implementing effective prevention measures. However, most recent disease prediction approaches…

Computation and Language · Computer Science 2024-05-07 Zhixiang Su , Yinan Zhang , Jiazheng Jing , Jie Xiao , Zhiqi Shen

Problem definition: Personalized medicine (PM) seeks the best treatment for each patient among a set of available treatment methods. Since a specific treatment does not work well on all patients, traditionally, the best treatment was…

Optimization and Control · Mathematics 2023-08-03 Jianzhong Du , Siyang Gao , Chun-Hung Chen

Assessing the quality of cancer care administered by US health providers poses numerous challenges due to meaningful heterogeneity in patient populations. Patients undergoing oncology treatment exhibit substantial variation in disease…

Applications · Statistics 2025-02-17 Yige Li , Nancy L. Keating , Mary Beth Landrum , Jose R. Zubizarreta

Composite endpoints are commonly used with an anticipation that clinically relevant endpoints as a whole would yield meaningful treatment benefits. The win ratio is a rank-based statistic to summarize composite endpoints, allowing…

Methodology · Statistics 2022-12-14 Di Zhang , Stephen R. Wisniewski , Jong-Hyeon Jeong

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent guidelines and initiatives of regulatory agencies. Among the factors contributing to modernized drug development and…

A central question in many fields of scientific research is to determine how an outcome would be affected by an action, or to measure the effect of an action (a.k.a treatment effect). In recent years, a need for estimating the heterogeneous…

Methodology · Statistics 2021-08-24 Weijia Zhang , Jiuyong Li , Lin Liu

Automatically reasoning with conflicting generic clinical guidelines is a burning issue in patient-centric medical reasoning where patient-specific conditions and goals need to be taken into account. It is even more challenging in the…

Artificial Intelligence · Computer Science 2019-02-21 Kristijonas Čyras , Tiago Oliveira

Estimating individualized treatment rules (ITRs) is crucial for tailoring interventions in precision medicine. Typical ITR estimation methods rely on conditional average treatment effects (CATEs) to guide treatment assignments. However,…

Methodology · Statistics 2025-10-20 Peng Wu , Qing Jiang , Shanshan Luo , Zhi Geng

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

Methodology · Statistics 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song