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Detection of interactions between treatment effects and patient descriptors in clinical trials is critical for optimizing the drug development process. The increasing volume of data accumulated in clinical trials provides a unique…

Applications · Statistics 2017-12-25 Baptiste Goujaud , Eric W. Tramel , Pierre Courtiol , Mikhail Zaslavskiy , Gilles Wainrib

Survival is poor for patients with metastatic cancer, and it is vital to examine new biomarkers that can improve patient prognostication and identify those who would benefit from more aggressive therapy. In metastatic prostate cancer, two…

Methodology · Statistics 2024-08-07 Glenn Heller , Sean M. Devlin

Renal cell carcinoma represents a significant global health challenge with a low survival rate. This research aimed to devise a comprehensive deep-learning model capable of predicting survival probabilities in patients with renal cell…

Computer Vision and Pattern Recognition · Computer Science 2023-07-10 Maryamalsadat Mahootiha , Hemin Ali Qadir , Jacob Bergsland , Ilangko Balasingham

In cancer biomarker development, a key objective is to evaluate whether a new biomarker, when combined with an established one, improves early cancer detection compared to using the established biomarker alone. Incremental value is often…

Methodology · Statistics 2025-11-21 Indrila Ganguly , Ying Huang

In pharmaceutical R&D, predicting the efficacy of a pharmaceutical in treating a particular disease prior to clinical testing or any real-world use has been challenging. In this paper, we propose a flexible and modular machine…

Machine Learning · Computer Science 2025-09-10 Adrien Couetoux , Thomas Devenyns , Lise Diagne , David Champagne , Pierre-Yves Mousset , Chris Anagnostopoulos

Insufficiently precise diagnosis of clinical disease is likely responsible for many treatment failures, even for common conditions and treatments. With a large enough dataset, it may be possible to use unsupervised machine learning to…

Precision medicine is currently a topic of great interest in clinical and intervention science. One way to formalize precision medicine is through a treatment regime, which is a sequence of decision rules, one per stage of clinical…

Methodology · Statistics 2016-06-07 Yichi Zhang , Eric B. Laber , Anastasios Tsiatis , Marie Davidian

Balancing influential covariates is crucial for valid treatment comparisons in clinical studies. While covariate-adaptive randomization is commonly used to achieve balance, its performance can be inadequate when the number of baseline…

Methodology · Statistics 2024-12-30 Ziqing Guo , Yang Liu , Lucy Xia

Appropriate dosing of radiation is crucial to patient safety in radiotherapy. Current quality assurance depends heavily on a peer-review process, where the physicians' peer review on each patient's treatment plan, including dose and…

Machine Learning · Computer Science 2021-12-01 Qiongge Li , Jean Wright , Russell Hales , Ranh Voong , Todd McNutt

Variable selection is a procedure to attain the truly important predictors from inputs. Complex nonlinear dependencies and strong coupling pose great challenges for variable selection in high-dimensional data. In addition, real-world…

Methodology · Statistics 2023-07-04 Keyao Wang , Huiwen Wang , Jichang Zhao , Lihong Wang

Clinical biosensors with low detection limit hold significant promise in the early diagnosis of debilitating diseases. Recent progress in sensor development has led to the demonstration of detection capable of detecting target molecules…

Medical Physics · Physics 2019-12-05 Tuhin Chakrabortty , Chandra R. Murthy , Manoj Varma

Patient-reported outcome (PRO) measures are increasingly collected as a means of measuring healthcare quality and value. The capability to predict such measures enables patient-provider shared decision making and the delivery of…

Methodology · Statistics 2023-03-01 Jaeyoung Park , Muxuan Liang , Ying-Qi Zhao , Xiang Zhong

In 2023, the U.S. Food and Drug Administration issued guidance for adjustment of covariates in randomized clinical trials, emphasizing its role in enhancing precision and power through prognostic baseline variables. Despite its potential,…

Methodology · Statistics 2026-05-28 Kelly Van Lancker , Iván Díaz , Stijn Vansteelandt

In early-phase clinical trials, a predictive biomarker may identify subgroups that benefit from an experimental therapy, even when the overall average treatment effect is negligible. Recently proposed nonparametric interaction tests such as…

Methodology · Statistics 2025-08-15 Nan Miles Xi , Lin Wang

The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent guidelines and initiatives of regulatory agencies. Among the factors contributing to modernized drug development and…

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

Biomarker discovery is vital in advancing personalized medicine, offering insights into disease diagnosis, prognosis, and therapeutic efficacy. Traditionally, the identification and validation of biomarkers heavily depend on extensive…

Machine Learning · Computer Science 2024-09-25 Wangyang Ying , Dongjie Wang , Xuanming Hu , Ji Qiu , Jin Park , Yanjie Fu

A treatment benefit predictor (TBP) is a function that maps patient characteristics to an estimate of the treatment benefit for that patient. Such predictors support optimizing individualized treatment decisions, which are central to…

Methodology · Statistics 2025-09-30 Yuan Xia , Mohsen Sadatsafavi , Paul Gustafson

In routine care, individuals identified a priori as high-risk are usually tested for conditions more frequently. Protected attributes, such as sex or ethnicity may also determine testing frequency. Such heterogeneous detection rates across…

Applications · Statistics 2026-05-08 Jose Benitez-Aurioles , Ricardo Silva , Brian McMillan , Matthew Sperrin

Recently, interest has grown in the use of proxy variables of unobserved confounding for inferring the causal effect in the presence of unmeasured confounders from observational data. One difficulty inhibiting the practical use is finding…

Machine Learning · Computer Science 2024-05-28 Feng Xie , Zhengming Chen , Shanshan Luo , Wang Miao , Ruichu Cai , Zhi Geng