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Clinical data informs the personalization of health care with a potential for more effective disease management. In practice, this is achieved by subgrouping, whereby clusters with similar patient characteristics are identified and then…

Applications · Statistics 2024-04-17 Christof Naumzik , Alice Kongsted , Werner Vach , Stefan Feuerriegel

Inferring individualised treatment effects from observational data can unlock the potential for targeted interventions. It is, however, hard to infer these effects from observational data. One major problem that can arise is covariate shift…

Machine Learning · Computer Science 2024-04-25 Damian Machlanski , Spyros Samothrakis , Paul Clarke

An important goal of precision medicine is to personalize medical treatment by identifying individuals who are most likely to benefit from a specific treatment. The Likely Responder (LR) framework, which identifies a subpopulation where…

Methodology · Statistics 2026-03-13 Annan Deng , Carole Siegel , Hyung G. Park

This paper considers the identification of dynamic treatment effects with panel data, in complex designs where the treatment may not be binary and may not be absorbing. We first show that under no-anticipation and parallel-trends…

Econometrics · Economics 2025-12-23 Clément de Chaisemartin , Xavier D'Haultfœuille

Plotting the residuals is a recommended procedure to diagnose deviations from linear model assumptions, such as non-linearity, heteroscedasticity, and non-normality. The presence of structure in residual plots can be tested using the lineup…

Machine Learning · Statistics 2024-11-05 Weihao Li , Dianne Cook , Emi Tanaka , Susan VanderPlas , Klaus Ackermann

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

Multiple sclerosis (MS) is a chronic autoimmune disease that affects the central nervous system. The progression and severity of MS varies by individual, but it is generally a disabling disease. Although medications have been developed to…

Applications · Statistics 2013-03-06 Joyce C. Ho , Joydeep Ghosh , KP Unnikrishnan

The progression of chronic diseases often follows highly variable trajectories, and the underlying factors remain poorly understood. Standard mixed-effects models typically represent inter-patient differences as random deviations around a…

Real-world observational datasets and machine learning have revolutionized data-driven decision-making, yet many models rely on empirical associations that may be misleading due to confounding and subgroup heterogeneity. Simpson's paradox…

Machine Learning · Computer Science 2026-03-03 Xian Teng , Yu-Ru Lin

Decision tree learning is increasingly being used for pointwise inference. Important applications include causal heterogenous treatment effects and dynamic policy decisions, as well as conditional quantile regression and design of…

Machine Learning · Statistics 2024-02-08 Matias D. Cattaneo , Jason M. Klusowski , Peter M. Tian

One of the key challenges when developing a predictive model is the capability to describe the domain knowledge and the cause-effect relationships in a simple way. Decision rules are a useful and important methodology in this context,…

Machine Learning · Computer Science 2021-10-19 Francisco Valente , Jorge Henriques , Simão Paredes , Teresa Rocha , Paulo de Carvalho , João Morais

The paper concerns the problem of predicting the effect of actions or interventions on a system from a combination of (i) statistical data on a set of observed variables, and (ii) qualitative causal knowledge encoded in the form of a…

Artificial Intelligence · Computer Science 2012-07-02 Carlos Brito , Judea Pearl

Subgroup analysis is a frequently used tool for evaluating heterogeneity of treatment effect and heterogeneity in treatment harm across observed baseline patient characteristics. While treatment efficacy and adverse event measures are often…

Applications · Statistics 2018-08-14 Nicholas C. Henderson , Ravi Varadhan

Model-based clustering is a powerful tool that is often used to discover hidden structure in data by grouping observational units that exhibit similar response values. Recently, clustering methods have been developed that permit…

Methodology · Statistics 2025-06-24 Sally Paganin , Garritt L. Page , Fernando Andrés Quintana

Precision oncology aims to prescribe the optimal cancer treatment to the right patients, maximizing therapeutic benefits. However, identifying patient subgroups that may benefit more from experimental cancer treatments based on randomized…

Methodology · Statistics 2026-01-06 Xingyu Li , Qing Liu , Tony Jiang , Hong Amy Xia , Peng Wei , Brian P. Hobbs

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

Methodology · Statistics 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

The identification of most relevant clinical criteria related to low back pain disorders may aid the evaluation of the nature of pain suffered in a way that usefully informs patient assessment and treatment. Data concerning low back pain…

Methodology · Statistics 2018-02-06 Michael Fop , Keith Smart , Thomas Brendan Murphy

Complex diseases are caused by a multitude of factors that may differ between patients. As a result, hypothesis tests comparing all patients to all healthy controls can detect many significant variables with inconsequential effect sizes. A…

Machine Learning · Statistics 2022-05-25 Eric V. Strobl , Thomas A. Lasko

We propose a causal hidden Markov model to achieve robust prediction of irreversible disease at an early stage, which is safety-critical and vital for medical treatment in early stages. Specifically, we introduce the hidden variables which…

Computer Vision and Pattern Recognition · Computer Science 2021-03-31 Jing Li , Botong Wu , Xinwei Sun , Yizhou Wang

The statistical analysis of group studies in neuroscience is particularly challenging due to the complex spatio-temporal nature of the data, its multiple levels and the inter-individual variability in brain responses. In this respect,…

Methodology · Statistics 2025-05-15 Nicolò Margaritella , Vanda Inácio , Ruth King
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