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Medication adherence is a well-known problem for pharmaceutical treatment of chronic diseases. Understanding how nonadherence affects treatment efficacy is made difficult by the ethics of clinical trials that force patients to skip doses of…

Quantitative Methods · Quantitative Biology 2021-12-30 Elijah D Counterman , Sean D Lawley

Estimating the joint effect of a multivariate, continuous exposure is crucial, particularly in environmental health where interest lies in simultaneously evaluating the impact of multiple environmental pollutants on health. We develop novel…

The International Council for Harmonization (ICH) E9 (R1) addendum provides the estimand framework to formulate treatment effects in a clinical trial. One of the attributes of an estimand the framework describes is intercurrent events.…

Many problems within personalized medicine and digital health rely on the analysis of continuous-time functional biomarkers and other complex data structures emerging from high-resolution patient monitoring. In this context, this work…

Machine Learning · Statistics 2025-01-14 Marcos Matabuena

Uncertainty quantification of causal effects is crucial for safety-critical applications such as personalized medicine. A powerful approach for this is conformal prediction, which has several practical benefits due to model-agnostic…

Machine Learning · Computer Science 2026-02-04 Maresa Schröder , Dennis Frauen , Jonas Schweisthal , Konstantin Heß , Valentyn Melnychuk , Stefan Feuerriegel

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment…

Methodology · Statistics 2022-10-05 Dasom Lee , Shu Yang , Xiaofei Wang

Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

Methodology · Statistics 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

With the availability of massive amounts of data from electronic health records and registry databases, incorporating time-varying patient information to improve risk prediction has attracted great attention. To exploit the growing amount…

Applications · Statistics 2022-08-29 Yifei Sun , Sy Han Chiou , Colin O. Wu , Meghan McGarry , Chiung-Yu Huang

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

Because different patients may response quite differently to the same drug or treatment, there is increasing interest in discovering individualized treatment rule. In particular, people are eager to find the optimal individualized treatment…

Methodology · Statistics 2016-04-14 Wei Xiao , Hao Helen Zhang , Wenbin Lu

Not only does mobile health technology enable researchers to track changes in multiple longitudinal outcomes of interest and to record the occurrence of health-related events over time, but it also allows for the delivery of repeated…

Multi-regional clinical trials (MRCTs) play an increasingly crucial role in global pharmaceutical development by expediting data gathering and regulatory approval across diverse patient populations. However, differences in recruitment…

Methodology · Statistics 2024-04-15 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

With a large number of baseline covariates, we propose a new semi-parametric modeling strategy for heterogeneous treatment effect estimation and individualized treatment selection, which are two major goals in personalized medicine. We…

Methodology · Statistics 2021-08-12 Wenchuan Guo , Xiao-hua Zhou , Shujie Ma

Estimating the effects of interventions on patient outcome is one of the key aspects of personalized medicine. Their inference is often challenged by the fact that the training data comprises only the outcome for the administered treatment,…

Treatment specific survival curves are an important tool to illustrate the treatment effect in studies with time-to-event outcomes. In non-randomized studies, unadjusted estimates can lead to biased depictions due to confounding. Multiple…

Methodology · Statistics 2023-04-25 Robin Denz , Renate Klaaßen-Mielke , Nina Timmesfeld

When modelling competing risks survival data, several techniques have been proposed in both the statistical and machine learning literature. State-of-the-art methods have extended classical approaches with more flexible assumptions that can…

In recent years, the growing availability of biomedical datasets featuring numerous longitudinal covariates has motivated the development of several multi-step methods for the dynamic prediction of survival outcomes. These methods employ…

Methodology · Statistics 2026-01-14 Mirko Signorelli , Sophie Retif

Beyond conditional average treatment effects, treatments may impact the entire outcome distribution in covariate-dependent ways, for example, by altering the variance or tail risks for specific subpopulations. We propose a novel estimand to…

Machine Learning · Statistics 2026-03-18 Saksham Jain , Alex Luedtke

Objective: In randomized clinical trials, prediction models can be used to explore the relationships between patients' variables (e.g., clinical, pathological, or lifestyle variables, and also biomarker or genomic data) and treatment effect…

Quantitative Methods · Quantitative Biology 2026-02-03 Elvire Roblin , Paul-Henry Cournède , Stefan Michiels

Precision medicine has the potential to tailor treatment decisions to individual patients using machine learning (ML) and artificial intelligence (AI), but it faces significant challenges due to complex biases in clinical observational data…

Machine Learning · Computer Science 2024-11-26 Michael Vollenweider , Manuel Schürch , Chiara Rohrer , Gabriele Gut , Michael Krauthammer , Andreas Wicki