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Survival time is the primary endpoint of many randomized controlled trials, and a treatment effect is typically quantified by the hazard ratio under the assumption of proportional hazards. Awareness is increasing that in many settings this…

Methodology · Statistics 2023-10-04 Robin Ristl , Heiko Götte , Armin Schüler , Martin Posch , Franz König

Background: Comparative effectiveness research using network meta-analysis can present a hierarchy of competing treatments, from the least to most preferable option. However, the research question associated with the hierarchy of multiple…

Individualized treatment rules (ITRs) are deterministic decision rules that recommend treatments to individuals based on their characteristics. Though ubiquitous in medicine, ITRs are hardly ever evaluated in randomized controlled trials.…

Methodology · Statistics 2023-08-22 François Grolleau , Francois Petit , Raphaël Porcher

When confronted with an undesired cell population, such as bacterial infections or tumors, we seek the most effective treatment, designed to eliminate the population as rapidly as possible. A common practice is to monitor the cells…

Biological Physics · Physics 2025-01-23 Uzi Harush , Ravid Straussman , Baruch Barzel

In this article, we develop nonparametric inference methods for comparing survival data across two samples, which are beneficial for clinical trials of novel cancer therapies where long-term survival is a critical outcome. These therapies,…

Methodology · Statistics 2024-09-05 Yi-Cheng Tai , Weijing Wang , Martin T. Wells

We present nonparametric algorithms for estimating optimal individualized treatment rules. The proposed algorithms are based on the XGBoost algorithm, which is known as one of the most powerful algorithms in the machine learning literature.…

Machine Learning · Statistics 2020-02-04 Duzhe Wang , Haoda Fu , Po-Ling Loh

Dynamic treatment regimes or policies are a sequence of decision functions over multiple stages that are tailored to individual features. One important class of treatment policies in practice, namely multi-stage stationary treatment…

Machine Learning · Statistics 2025-01-09 Daiqi Gao , Yufeng Liu , Donglin Zeng

In personalized medicine, the ability to predict and optimize treatment outcomes across various time frames is essential. Additionally, the ability to select cost-effective treatments within specific budget constraints is critical. Despite…

Machine Learning · Computer Science 2024-10-14 Thomas Schwarz , Cecilia Casolo , Niki Kilbertus

Parkinson's disease (PD) is a chronic, degenerative neurological disorder. PD cannot be prevented, slowed or cured as of today but highly effective symptomatic treatments are available. We consider relevant estimands and treatment effect…

In many applied optimization settings, parameters that define the constraints may not guarantee the best possible solution, and superior solutions might exist that are infeasible for the given parameter values. Removing such constraints,…

Optimization and Control · Mathematics 2024-07-22 Farzin Ahmadi , Todd R. McNutt , Kimia Ghobadi

The problem of chemotherapy treatment optimization can be defined in order to minimize the size of the tumor without endangering the patient's health; therefore, chemotherapy requires to achieve a number of objectives, simultaneously. For…

Neural and Evolutionary Computing · Computer Science 2023-03-21 Mina Jafari , Behnam Ghavami , Vahid Sattari Naeini

Biomarker-guided designs are increasingly used to evaluate personalized treatments based on patients' biomarker status in Phase II and III clinical trials. With adaptive enrichment, these designs can improve the efficiency of evaluating the…

Methodology · Statistics 2024-06-11 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

While many novel therapies have been approved in recent years for treating patients with multiple myeloma, there is still no established curative regimen, especially for patients with high risk disease. In this work, we use a mathematical…

Optimization and Control · Mathematics 2022-11-18 Mahya Aghaee , Urszula Ledzewicz , Michael Robbins , Natalie Bezman , Hearn Jay Cho , Helen Moore

Estimating individualized treatment rules (ITRs) is crucial for tailoring interventions in precision medicine. Typical ITR estimation methods rely on conditional average treatment effects (CATEs) to guide treatment assignments. However,…

Methodology · Statistics 2025-10-20 Peng Wu , Qing Jiang , Shanshan Luo , Zhi Geng

Precision medicine seeks to discover an optimal personalized treatment plan and thereby provide informed and principled decision support, based on the characteristics of individual patients. With recent advancements in medical imaging, it…

Methodology · Statistics 2023-04-26 Xinyi Li , Michael R. Kosorok

Prediction of survival for cancer patients is an open area of research. However, many of these studies focus on datasets with a large number of patients. We present a novel method that is specifically designed to address the challenge of…

Machine Learning · Computer Science 2015-09-30 Hamid Reza Hassanzadeh , John H. Phan , May D. Wang

With the advancement in drug development, multiple treatments are available for a single disease. Patients can often benefit from taking multiple treatments simultaneously. For example, patients in Clinical Practice Research Datalink (CPRD)…

Applications · Statistics 2018-04-17 Muxuan Liang , Ye Ting , Haoda Fu

Overall survival (OS) is the gold standard for assessing patient benefit and cost-effectiveness of new cancer drugs. However, it is often difficult to use OS as the primary endpoint in randomized clinical trials (RCTs) for patients with…

Methodology · Statistics 2026-04-23 Sidi Wang , Kelley Kidwell , Bo Huang , Satrajit Roychoudhury

One of the most common ways researchers compare survival outcomes across treatments when confounding is present is using Cox regression. This model is limited by its underlying assumption of proportional hazards; in some cases, substantial…

Applications · Statistics 2021-02-02 Elizabeth A. Handorf , Marc Smaldone , Sujana Movva , Nandita Mitra

The accurate prediction of patient prognosis is a critical challenge in clinical practice. With the availability of various patient information, physicians can optimize medical care by closely monitoring disease progression and therapy…

Applications · Statistics 2023-11-28 He Weiyi
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