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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

Predicting drug response in patients from preclinical data remains a major challenge in precision oncology due to the substantial biological gap between in vitro cell lines and patient tumors. Rather than aiming to improve absolute in vitro…

Machine Learning · Computer Science 2026-03-18 Camille Jimenez Cortes , Philippe Lalanda , German Vega

Cancer pathology is unique to a given individual, and developing personalized diagnostic and treatment protocols are a primary concern. Mathematical modeling and simulation is a promising approach to personalized cancer medicine. Yet, the…

Tissues and Organs · Quantitative Biology 2020-08-03 Alvaro Köhn-Luque , Xiaoran Lai , Arnoldo Frigessi

Biomarker measurements can be relatively easy and quick to obtain and they are useful to investigate whether a compound works as intended on a mechanistic, pharmacological level. In some situations, it is realistic to assume that patients,…

Methodology · Statistics 2018-06-26 Björn Bornkamp , Georgina Bermann

Adaptive designs are commonly used in clinical and drug development studies for optimum utilization of available resources. In this article, we consider the problem of estimating the effect of the selected (better) treatment using a…

Statistics Theory · Mathematics 2023-01-24 Masihuddin , Neeraj Misra

Unsupervised patient stratification is essential for disease subtype discovery, yet, despite growing evidence of molecular heterogeneity of non-oncological diseases, popular methods are benchmarked primarily using cancers with mutually…

Cancer remains a global challenge due to its growing clinical and economic burden. Its uniquely personal manifestation, which makes treatment difficult, has fuelled the quest for personalized treatment strategies. Thus, genomic profiling is…

Domain shift is a common problem in clinical applications, where the training images (source domain) and the test images (target domain) are under different distributions. Unsupervised Domain Adaptation (UDA) techniques have been proposed…

Image and Video Processing · Electrical Eng. & Systems 2023-09-06 Jiajin Zhang , Hanqing Chao , Amit Dhurandhar , Pin-Yu Chen , Ali Tajer , Yangyang Xu , Pingkun Yan

Multi-arm trials are gaining interest in practice given the statistical and logistical advantages they can offer. The standard approach uses a fixed allocation ratio, but there is a call for making it adaptive and skewing the allocation of…

Methodology · Statistics 2026-01-16 Gianmarco Caruso , Pavel Mozgunov

We propose a frequentist adaptive phase 2 trial design to evaluate the safety and efficacy of three treatment regimens (doses) compared to placebo for four types of helminth (worm) infections. This trial will be carried out in four…

Vision transformer has demonstrated great potential in abundant vision tasks. However, it also inevitably suffers from poor generalization capability when the distribution shift occurs in testing (i.e., out-of-distribution data). To…

Computer Vision and Pattern Recognition · Computer Science 2022-12-07 Xin Li , Cuiling Lan , Guoqiang Wei , Zhibo Chen

Bayesian response adaptive clinical trials are currently evaluating experimental therapies for several diseases. Adaptive decisions, such as pre-planned variations of the randomization probabilities, attempt to accelerate the development of…

Methodology · Statistics 2021-05-25 Marta Bonsaglio , Sandra Fortini , Steffen Ventz , Lorenzo Trippa

Cancer is responsible for millions of deaths worldwide every year. Although significant progress has been achieved in cancer medicine, many issues remain to be addressed for improving cancer therapy. Appropriate cancer patient…

Machine Learning · Computer Science 2021-03-31 David Oniani , Chen Wang , Yiqing Zhao , Andrew Wen , Hongfang Liu , Feichen Shen

Adaptive enrichment allows for pre-defined patient subgroups of interest to be investigated throughout the course of a clinical trial. Many trials which measure a long-term time-to-event endpoint often also routinely collect repeated…

Methodology · Statistics 2024-02-26 Abigail J. Burdon , Richard D. Baird , Thomas Jaki

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

Melanoma is a type of skin cancer developed from melanocytes. It is one of the most lethal types of cancer, accounting for approximately 75% of skin cancer deaths. Late stage melanoma is very difficult to treat, since the cancer cells are…

Quantitative Methods · Quantitative Biology 2018-11-28 Xue Teng , Fuad Gwadry , Haley McConkey , Scott Ernst , Femida Gwadry-Sridhar

Radiation response in cancer is shaped by complex, patient specific biology, yet current treatment strategies often rely on uniform dose prescriptions without accounting for tumor heterogeneity. In this study, we introduce a meta learning…

Medical Physics · Physics 2025-08-12 Hao Peng , Yuanyuan Zhang , Steve Jiang , Robert Timmerman , John Minna

In this paper, we develop new methods for estimating average treatment effects in observational studies, focusing on settings with more than two treatment levels under unconfoundedness given pre-treatment variables. We emphasize…

Methodology · Statistics 2017-10-11 Shu Yang , Guido W. Imbens , Zhanglin Cui , Douglas Faries , Zbigniew Kadziola

Due to the high heterogeneity and clinical characteristics of cancer, there are significant differences in multi-omic data and clinical characteristics among different cancer subtypes. Therefore, accurate classification of cancer subtypes…

Quantitative Methods · Quantitative Biology 2023-08-23 Liangrui Pan , Dazheng Liu , Zhichao Feng , Wenjuan Liu , Shaoliang Peng

Broadening eligibility criteria in cancer trials has been advocated to represent the true patient population more accurately. While the advantages are clear in terms of generalizability and recruitment, novel dose-finding designs are needed…

Applications · Statistics 2023-01-12 Rebecca B. Silva , Bin Cheng , Richard D. Carvajal , Shing M. Lee