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Related papers: Measuring Effects of Medication Adherence on Time-…

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Many rare diseases offer limited established treatment options, leading patients to switch therapies when new medications emerge. To analyze the impact of such treatment switches within the low sample size limitations of rare disease…

In cohort studies, non-random medication use can pose barriers to estimation of the natural history trend in a mean biomarker value (namely, the association between a predictor of interest and a biomarker outcome that would be observed in…

Methodology · Statistics 2021-01-25 Andrew J. Spieker , Joseph A. C. Delaney , Robyn L. McClelland

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying…

Methodology · Statistics 2023-08-08 Liangyuan Hu , Jiayi Ji , Himanshu Joshi , Erick Scott , Fan Li

Consider a subject or unit in a longitudinal biomedical, public health, engineering, economic, or social science study which is being monitored over a possibly random duration. Over time this unit experiences competing recurrent events and…

Methodology · Statistics 2024-12-30 Lili Tong , Piaomu Liu , Edsel Pena

Identifying prognostic factors for disease progression is a cornerstone of medical research. Repeated assessments of a marker outcome are often used to evaluate disease progression, and the primary research question is to identify factors…

Computation · Statistics 2022-10-24 Brian Kwan , Lin Liu , David Strong , H. Irene Su , Loki Natarajan

Chronic diseases, including diabetes, hypertension, asthma, HIV-AIDS, epilepsy, and tuberculosis, necessitate rigorous adherence to medication to avert disease progression, manage symptoms, and decrease mortality rates. Adherence is…

Computer Vision and Pattern Recognition · Computer Science 2025-11-20 Md Asaduzzaman Jabin , Hanqi Jiang , Yiwei Li , Patrick Kaggwa , Eugene Douglass , Juliet N. Sekandi , Tianming Liu

A dynamic treatment regime is a sequence of medical decisions that adapts to the evolving clinical status of a patient over time. To facilitate personalized care, it is crucial to assess the probability of each available treatment option…

Methodology · Statistics 2024-11-05 Jiefeng Bi , Matteo Borrotti , Bernardo Nipoti

Researchers are increasingly turning to machine learning (ML) algorithms to investigate causal heterogeneity in randomized experiments. Despite their promise, ML algorithms may fail to accurately ascertain heterogeneous treatment effects…

Methodology · Statistics 2024-04-23 Kosuke Imai , Michael Lingzhi Li

This paper considers how to classify the effects of interventions in causal models for outcomes and exposures observed over time. First, we demonstrate the limitations of the most common uses of potential outcomes and causal directed…

Methodology · Statistics 2026-05-29 Russell Steele , Naftali Weinberger , Tess Baker , Ian Shrier

Understanding the effect of a particular treatment or a policy pertains to many areas of interest, ranging from political economics, marketing to healthcare. In this paper, we develop a non-parametric algorithm for detecting the effects of…

Methodology · Statistics 2022-08-24 Davide Viviano , Jelena Bradic

Large-scale longitudinal molecular profiling is now firmly established in biomedical research, prompted by the need to uncover coordinated biomarker trajectories reflecting the dynamics of underlying biological mechanisms and characterise…

Methodology · Statistics 2026-03-24 Salima Jaoua , Daniel Temko , Hélène Ruffieux

Deciding on an appropriate intervention requires a causal model of a treatment, the outcome, and potential mediators. Causal mediation analysis lets us distinguish between direct and indirect effects of the intervention, but has mostly been…

Machine Learning · Computer Science 2023-06-19 Çağlar Hızlı , ST John , Anne Juuti , Tuure Saarinen , Kirsi Pietiläinen , Pekka Marttinen

Electronic health records contain valuable information for monitoring patients' health trajectories over time. Disease progression models have been developed to understand the underlying patterns and dynamics of diseases using these data as…

Estimating the impact of continuous treatment variables (e.g., dosage amount) on binary outcomes presents significant challenges in modeling and estimation because many existing approaches make strong assumptions that do not hold for…

Methodology · Statistics 2025-05-08 Muyan Jiang , Yunkai Zhang , Anil Aswani

Hypertension is a leading cause of cardiovascular diseases and morbidity, with antihypertensive drugs and blood pressure management strategies having heterogeneous effects on patients. Previous authors exploited this heterogeneity to…

Methodology · Statistics 2025-01-15 Janie Coulombe , Dany El-Riachi , Fanxing Du , Tianze Jiao

The systematic collection of longitudinal data is very common in practice, making mixed models widely used. Most developments around these models focus on modeling the mean trajectory of repeated measurements, typically under the assumption…

In Oncology, trials evaluating drug combinations are becoming more common. While combination therapies bring the potential for greater efficacy, they also create unique challenges for ensuring drug safety. In Phase-I dose escalation trials…

Applications · Statistics 2023-02-23 Lukas A. Widmer , Andrew Bean , David Ohlssen , Sebastian Weber

Mobile health is a rapidly developing field in which behavioral treatments are delivered to individuals via wearables or smartphones to facilitate health-related behavior change. Micro-randomized trials (MRT) are an experimental design for…

Methodology · Statistics 2019-05-13 Tianchen Qian , Predrag Klasnja , Susan A. Murphy

Background and Objectives: Multidrug Resistance (MDR) is a critical global health issue, causing increased hospital stays, healthcare costs, and mortality. This study proposes an interpretable Machine Learning (ML) framework for MDR…