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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

Routinely collected clinical blood tests are an emerging molecular data source for large-scale biomedical research but inherently feature irregular sampling and informative observation. Traditional approaches rely on imputation, which can…

Quantitative Methods · Quantitative Biology 2025-03-20 Andrea Zerio , Maya Bechler-Speicher , Tine Jess , Aleksejs Sazonovs

In many applications, it is of interest to identify a parsimonious set of features, or panel, from multiple candidates that achieves a desired level of performance in predicting a response. This task is often complicated in practice by…

Methodology · Statistics 2025-10-23 B. D. Williamson , Y. Huang

Optimal treatment rules are mappings from individual patient characteristics to tailored treatment assignments that maximize mean outcomes. In this work, we introduce a conditional potential benefit (CPB) metric that measures the expected…

Methodology · Statistics 2024-08-29 Alexander W. Levis , Eli Ben-Michael , Edward H. Kennedy

In recent years we have been able to gather large amounts of genomic data at a fast rate, creating situations where the number of variables greatly exceeds the number of observations. In these situations, most models that can handle a…

Methodology · Statistics 2025-02-07 Andrea Bratsberg , Abhik Ghosh , Magne Thoresen

Free energy calculations are rapidly becoming indispensable in structure-enabled drug discovery programs. As new methods, force fields, and implementations are developed, assessing their expected accuracy on real-world systems…

Estimation of individual treatment effects is commonly used as the basis for contextual decision making in fields such as healthcare, education, and economics. However, it is often sufficient for the decision maker to have estimates of…

Machine Learning · Computer Science 2020-08-13 Maggie Makar , Fredrik D. Johansson , John Guttag , David Sontag

In precision medicine, one of the most important problems is estimating the optimal individualized treatment rules (ITR), which typically involves recommending treatment decisions based on fully observed individual characteristics of…

Methodology · Statistics 2025-10-15 Yue Zhang , Shanshan Luo , Zhi Geng , Yangbo He

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

For more than two decades, advances in personalised medicine and precision healthcare have largely been based on genomics and other omics data. These strategies aim to tailor interventions to individual patient profiles, promising greater…

The problem of dynamic prediction with time-dependent covariates, given by biomarkers, repeatedly measured over time, has received much attention over the last decades. Two contrasting approaches have become in widespread use. The first is…

Methodology · Statistics 2021-03-31 Hein Putter , Hans C. van Houwelingen

Randomized clinical trials are the gold standard for analyzing treatment effects, but high costs and ethical concerns can limit recruitment, potentially leading to invalid inferences. Incorporating external trial data with similar…

Methodology · Statistics 2024-09-09 Yujia Gu , Hanzhong Liu , Wei Ma

To address an important risk classification issue that arises in clinical practice, we propose a new mixture model via latent cure rate markers for survival data with a cure fraction. In the proposed model, the latent cure rate markers are…

Applications · Statistics 2009-10-12 Sungduk Kim , Yingmei Xi , Ming-Hui Chen

An individualized dose rule recommends a dose level within a continuous safe dose range based on patient level information such as physical conditions, genetic factors and medication histories. Traditionally, personalized dose finding…

Methodology · Statistics 2020-07-21 Liangyu Zhu , Wenbin Lu , Michael R. Kosorok , Rui Song

We propose a new procedure for inference on optimal treatment regimes in the model-free setting, which does not require to specify an outcome regression model. Existing model-free estimators for optimal treatment regimes are usually not…

Methodology · Statistics 2020-07-06 Yunan Wu , Lan Wang

Personalizing drug prescriptions in cancer care based on genomic information requires associating genomic markers with treatment effects. This is an unsolved challenge requiring genomic patient data in yet unavailable volumes as well as…

Predictive models are often introduced to decision-making tasks under the rationale that they improve performance over an existing decision-making policy. However, it is challenging to compare predictive performance against an existing…

Machine Learning · Computer Science 2024-06-13 Luke Guerdan , Amanda Coston , Kenneth Holstein , Zhiwei Steven Wu

Predictive biomarkers are playing an essential role in precision medicine. Identifying an optimal cutoff to select patient subsets with greater benefit from treatment is critical and more challenging for predictive biomarkers measured with…

Applications · Statistics 2025-04-29 Chi Zhang , Wei Shi , Spencer Woody , Qing Liu

Machine learning models are often trained to predict the outcome resulting from a human decision. For example, if a doctor decides to test a patient for disease, will the patient test positive? A challenge is that historical decision-making…

Machine Learning · Computer Science 2024-04-23 Sidhika Balachandar , Nikhil Garg , Emma Pierson

In order for clinicians to manage disease progression and make effective decisions about drug dosage, treatment regimens or scheduling follow up appointments, it is necessary to be able to identify both short and long-term trends in…

Quantitative Methods · Quantitative Biology 2016-12-06 Norman Poh , Simon Bull , Santosh Tirunagari , Nicholas Cole , Simon de Lusignan