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In causal inference, measuring treatment heterogeneity is crucial as it provides scientific insights into how treatments influence outcomes and guides personalized decision-making. In this work, we study semi-supervised settings where a…

Methodology · Statistics 2025-09-08 Yilizhati Anniwaer , Yuqian Zhang

Individualized treatment rules (ITRs) are considered a promising recipe to deliver better policy interventions. One key ingredient in optimal ITR estimation problems is to estimate the average treatment effect conditional on a subject's…

Methodology · Statistics 2021-03-16 Hongming Pu , Bo Zhang

Prediction algorithms that quantify the expected benefit of a given treatment conditional on patient characteristics can critically inform medical decisions. Quantifying the performance of treatment benefit prediction algorithms is an…

Methodology · Statistics 2023-05-17 Yuan Xia , Paul Gustafson , Mohsen Sadatsafavi

Treatment effect estimation is essential for informed decision-making in many fields such as healthcare, economics, and public policy. While flexible machine learning models have been widely applied for estimating heterogeneous treatment…

Machine Learning · Computer Science 2025-09-29 Pascal Memmesheimer , Vincent Heuveline , Jürgen Hesser

Robust validation of Machine Learning (ML) models is essential, but traditional data partitioning approaches often ignore the intrinsic quality of each instance. This study proposes the use of Item Response Theory (IRT) parameters to…

Machine Learning · Computer Science 2025-08-15 Lucas Cardoso , Vitor Santos , José Ribeiro Filho , Ricardo Prudêncio , Regiane Kawasaki , Ronnie Alves

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

We consider the problem of estimating the average treatment effect (ATE) when both randomized control trial (RCT) data and external real-world data (RWD) are available. We decompose the ATE estimand as the difference between a pooled-ATE…

Methodology · Statistics 2025-01-22 Mark van der Laan , Sky Qiu , Jens Magelund Tarp , Lars van der Laan

Observational health data can be leveraged to measure the real-world use and potential benefits or risks of existing medical interventions. However, lack of programming proficiency and advanced knowledge of causal inference methods excludes…

Methodology · Statistics 2022-03-22 Collin Sakal , Hon Hwang , Juan C Quiroz , Blanca Gallego

Estimating causal quantities from observational data is crucial for understanding the safety and effectiveness of medical treatments. However, to make reliable inferences, medical practitioners require not only estimating averaged causal…

Machine Learning · Computer Science 2026-03-13 Valentyn Melnychuk , Stefan Feuerriegel , Mihaela van der Schaar

Subgroup analyses of randomized controlled trials (RCTs) constitute an important component of the drug development process in precision medicine. In particular, subgroup analyses of early-stage trials often influence the design and…

Methodology · Statistics 2025-02-12 Daniel Schwartz , Riddhiman Saha , Steffen Ventz , Lorenzo Trippa

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

Identifying heterogeneous treatment effects (HTEs) in randomized controlled trials is an important step toward understanding and acting on trial results. However, HTEs are often small and difficult to identify, and HTE modeling methods…

Methodology · Statistics 2020-11-24 Erin Craig , Donald A Redelmeier , Robert J Tibshirani

Developing new drugs for target diseases is a time-consuming and expensive task, drug repurposing has become a popular topic in the drug development field. As much health claim data become available, many studies have been conducted on the…

Machine Learning · Computer Science 2023-02-22 Yaobin Ling , Pulakesh Upadhyaya , Luyao Chen , Xiaoqian Jiang , Yejin Kim

We study the statistical complexity of estimating partition functions given sample access to a proposal distribution and an unnormalized density ratio for a target distribution. While partition function estimation is a classical problem,…

Machine Learning · Statistics 2026-03-02 Adam Block , Abhishek Shetty

Cardiothoratic ratio (CTR) estimated from chest radiographs is a marker indicative of cardiomegaly, the presence of which is in the criteria for heart failure diagnosis. Existing methods for automatic assessment of CTR are driven by Deep…

Image and Video Processing · Electrical Eng. & Systems 2019-08-09 Roman Solovyev , Iaroslav Melekhov , Timo Lesonen , Elias Vaattovaara , Osmo Tervonen , Aleksei Tiulpin

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

Ratio-based biomarkers (RBBs), such as the proportion of necrotic tissue within a tumor, are widely used in clinical practice to support diagnosis, prognosis, and treatment planning. These biomarkers are typically estimated from…

Computer Vision and Pattern Recognition · Computer Science 2026-04-03 Jiameng Li , Teodora Popordanoska , Aleksei Tiulpin , Sebastian G. Gruber , Frederik Maes , Matthew B. Blaschko

Individualized treatment rules (ITR) can improve health outcomes by recognizing that patients may respond differently to treatment and assigning therapy with the most desirable predicted outcome for each individual. Flexible and efficient…

Methodology · Statistics 2017-09-25 Brent R. Logan , Rodney Sparapani , Robert E. McCulloch , Purushottam W. Laud

In most medical research, the average treatment effect is used to evaluate a treatment's performance. However, precision medicine requires knowledge of individual treatment effects: What is the difference between a unit's measurement under…

Computation · Statistics 2022-08-30 Mingyang Cai , Stef van Buuren , Gerko Vink

The aim of clinical effectiveness research using repositories of electronic health records is to identify what health interventions 'work best' in real-world settings. Since there are several reasons why the net benefit of intervention may…

Methodology · Statistics 2020-06-19 Jie Zhu , Blanca Gallego
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