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Adaptive enrichment trials aim to identify and recruit participants most likely to benefit from treatment based on evolving biomarker evidence, with the goal of informing individualized treatment recommendations. Bayesian methods are well…

Methodology · Statistics 2026-03-11 Lara Maleyeff , Shirin Golchi , Erica E. M. Moodie

Hierarchical model fitting has become commonplace for case-control studies of cognition and behaviour in mental health. However, these techniques require us to formalise assumptions about the data-generating process at the group level,…

Computers and Society · Computer Science 2020-11-04 Vincent Valton , Toby Wise , Oliver J. Robinson

Motivated by the study of state opioid policies, we propose a novel approach that uses autoregressive models for causal effect estimation in settings with panel data and staggered treatment adoption. Specifically, we seek to estimate the…

Major depressive disorder (MDD) is a heterogeneous condition; multiple underlying neurobiological substrates could be associated with treatment response variability. Understanding the sources of this variability and predicting outcomes has…

Commonly, clinical trials report effects not only for the full study population but also for patient subgroups. Meta-analyses of subgroup-specific effects and treatment-by-subgroup interactions may be inconsistent, especially when trials…

Methodology · Statistics 2025-12-23 Renato Panaro , Christian Röver , Tim Friede

We consider the problem of identifying sub-groups of participants in a clinical trial that have enhanced treatment effect. Recursive partitioning methods that recursively partition the covariate space based on some measure of between groups…

Methodology · Statistics 2018-06-22 Jon Arni Steingrimsson , Jiabei Yang

The identification of patient subgroups with differential treatment effects is the first step towards individualised treatments. A current draft guideline by the EMA discusses potentials and problems in subgroup analyses and formulated…

Methodology · Statistics 2020-01-22 Heidi Seibold , Achim Zeileis , Torsten Hothorn

When treating depression, clinicians are interested in determining the optimal treatment for a given patient, which is challenging given the amount of treatments available. To advance individualized treatment allocation, integrating data…

Motivation: Opioid use disorder (OUD) often arises after prescription opioid exposure and follows transitions among onset, remission, and relapse. Linked EHR-survey resources such as the All of Us Research Program enable stage-specific risk…

Quantitative Methods · Quantitative Biology 2026-01-23 Mengman Wei , Stanislav Listopad , Qian Peng

INTRODUCTION: The pharmacological treatment of Major Depressive Disorder (MDD) relies on a trial-and-error approach. We introduce an artificial intelligence (AI) model aiming to personalize treatment and improve outcomes, which was deployed…

The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the…

Methodology · Statistics 2022-02-22 Janie Coulombe , Erica E. M. Moodie , Susan M. Shortreed , Christel Renoux

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

Methodology · Statistics 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

Incarceration-diversion treatment programs aim to improve societal reintegration and reduce recidivism, but limited capacity forces policymakers to make prioritization decisions that often rely on risk assessment tools. While predictive,…

Computers and Society · Computer Science 2026-01-21 Chuwen Zhang , Pengyi Shi , Amy Ward

An active challenge in developing multimodal machine learning (ML) models for healthcare is handling missing modalities during training and deployment. As clinical datasets are inherently temporal and sparse in terms of modality presence,…

Machine Learning · Computer Science 2026-05-08 Andrew Wang , Ellie Pavlick , Ritambhara Singh

Model-based recursive partitioning (MOB) can be used to identify subgroups with differing treatment effects. The detection rate of treatment-by-covariate interactions and the accuracy of identified subgroups using MOB depend strongly on the…

Applications · Statistics 2022-09-07 Cynthia Huber , Norbert Benda , Tim Friede

Adverse childhood experiences (ACEs) are categories of childhood abuse, neglect, and household dysfunction. Screening by a single additive ACE score (e.g., a $\ge 4$ cutoff) has poor individual-level discrimination. We instead identify…

Applications · Statistics 2025-11-26 Ruizhe Zhang , Jooyoung Kong , Dylan S. Small , William Bekerman

Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

Computation · Statistics 2016-06-28 Marius Thomas , Björn Bornkamp

In the fight against hard-to-treat diseases such as cancer, it is often difficult to discover new treatments that benefit all subjects. For regulatory agency approval, it is more practical to identify subgroups of subjects for whom the…

Methodology · Statistics 2014-10-09 Wei-Yin Loh , Xu He , Michael Man

The Early diagnosis and treatment of depression is essential for effective treatment. Depression, while being one of the most common mental illnesses, is still poorly understood in both research and clinical practice. Among different…

Signal Processing · Electrical Eng. & Systems 2023-03-13 Abdolkarim Saeedi , Arash Maghsoudi , Fereidoun Nowshiravan Rahatabad

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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