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Targeted therapies on the basis of genomic aberrations analysis of the tumor have shown promising results in cancer prognosis and treatment. Regardless of tumor type, trials that match patients to targeted therapies for their particular…

Applications · Statistics 2018-04-18 Yanxun Xu , Peter Mueller , Apostolia M Tsimberidou , Donald Berry

Bayesian sample size calculations in clinical trials usually rely on complex Monte Carlo simulations in practice. Obtaining bounds on Bayesian notions of the false-positive rate and power often lack closed-form or approximate numerical…

Methodology · Statistics 2026-03-03 Riko Kelter

The win ratio (WR) statistic is increasingly used to evaluate treatment effects based on prioritized composite endpoints, yet existing Bayesian adaptive designs are not directly applicable because the WR is a summary statistic derived from…

Methodology · Statistics 2026-02-20 Di Zhu , Yong Zang

Phase I-II cancer clinical trial designs are intended to accelerate drug development. In cases where efficacy cannot be ascertained in a short period of time, it is common to divide the study in two stages: i) a first stage in which dose is…

Methodology · Statistics 2022-12-13 José L. Jiménez , Mourad Tighiouart

Unblinded sample size re-estimation (SSR) is often planned in a clinical trial when there is large uncertainty about the true treatment effect. For Proof-of Concept (PoC) in a Phase II dose finding study, contrast test can be adopted to…

Methodology · Statistics 2022-11-11 Qingyang Liu , Guanyu Hu , Binqi Ye , Susan Wang , Yaoshi Wu

In this article a generalized mathematical model describing the interactions between malignant tumour and immune system with discrete time delay incorporated into the system is considered. Time delay represents the time required to generate…

Tissues and Organs · Quantitative Biology 2015-11-05 Monika Joanna Piotrowska

Laser-directed-energy deposition (DED) offers advantages in additive manufacturing (AM) for creating intricate geometries and material grading. Yet, challenges like material inconsistency and part variability remain, mainly due to its…

Machine Learning · Computer Science 2024-02-28 Vispi Karkaria , Anthony Goeckner , Rujing Zha , Jie Chen , Jianjing Zhang , Qi Zhu , Jian Cao , Robert X. Gao , Wei Chen

Phase I dose-escalation trials constitute the first step in investigating the safety of potentially promising drugs in humans. Conventional methods for phase I dose-escalation trials are based on a single treatment schedule only. More…

Applications · Statistics 2021-03-04 Burak Kürsad Günhan , Sebastian Weber , Abdelkader Seroutou , Tim Friede

Treatment effect heterogeneity refers to the systematic variation in treatment effects across subgroups. There is an increasing need for clinical trials that aim to investigate treatment effect heterogeneity and estimate subgroup-specific…

Methodology · Statistics 2026-03-06 Xianglin Zhao , Shirin Golchi , Jean-Philippe Gouin , Kaberi Dasgupta

Randomized clinical trials are considered the gold standard for estimating causal effects. Nevertheless, in studies that are aimed at examining adverse effects of interventions, such trials are often impractical because of ethical and…

Methodology · Statistics 2020-01-20 Anthony D. Scotina , Andrew R. Zullo , Robert J. Smith , Roee Gutman

Most Bayesian response-adaptive designs unbalance randomization rates towards the most promising arms with the goal of increasing the number of positive treatment outcomes during the study, even though the primary aim of the trial is…

Applications · Statistics 2018-07-02 Steffen Ventz , Matteo Cellamare , Sergio Bacallado , Lorenzo Trippa

In response to the U.S.\ Food and Drug Administration's (FDA) Project Optimus, a paradigm shift is underway in the design of early-phase oncology trials. To accelerate drug development, seamless Phase I/II designs have gained increasing…

Methodology · Statistics 2025-11-04 Kana Makino , Natsumi Makigusa , Masahiro Kojima

Radiotherapy treatment planning often relies on time-consuming, trial-and-error adjustments that heavily depend on the expertise of specialists, while existing deep learning methods face limitations in generalization, prediction accuracy,…

Computer Vision and Pattern Recognition · Computer Science 2026-01-06 Hui Xie , Haiqin Hu , Lijuan Ding , Qing Li , Yue Sun , Tao Tan

Bayesian optimization (BO) is a widely-used sequential method for zeroth-order optimization of complex and expensive-to-compute black-box functions. The existing BO methods assume that the function evaluation (feedback) is available to the…

Machine Learning · Computer Science 2022-06-22 Arun Verma , Zhongxiang Dai , Bryan Kian Hsiang Low

We investigate delay effects on dominant transition pathways (DTP) between metastable states of stochastic systems. A modified version of the Maier-Stein model with linear delayed feedback is considered as an example. By a stability…

Applications · Statistics 2012-09-19 Huijun Jiang , Zhonghuai Hou

There is a growing interest in the implementation of platform trials, which provide the flexibility to incorporate new treatment arms during the trial and the ability to halt treatments early based on lack of benefit or observed…

Methodology · Statistics 2023-08-25 Peter Greenstreet , Thomas Jaki , Alun Bedding , Pavel Mozgunov

Immune checkpoint inhibitors (ICIs) have transformed cancer treatment, yet patient responses remain highly variable, and the biological mechanisms underlying resistance are poorly understood. While machine learning models hold promise for…

Machine Learning · Computer Science 2025-08-27 Ifrah Tariq , Ernest Fraenkel

In oncology, phase II studies are crucial for clinical development plans as such studies identify potent agents with sufficient activity to continue development in the subsequent phase III trials. Traditionally, phase II studies are…

Methodology · Statistics 2023-08-08 Takuya Yoshimoto , Satoru Shinoda , Kouji Yamamoto , Kouji Tahata

Determining the extent to which a patient is benefiting from cancer therapy is challenging. Criteria for quantifying the extent of "tumor response" observed within a few cycles of treatment have been established for various types of solid…

Methodology · Statistics 2020-09-18 Jie Zhou , Xun Jiang , H. Amy Xia , Peng Wei , Brian P. Hobbs

In the first stage of a two-stage study, the researcher uses a statistical model to impute the unobserved exposures. In the second stage, imputed exposures serve as covariates in epidemiological models. Imputation error in the first stage…

Applications · Statistics 2021-07-19 Ron Sarafian , Itai Kloog , Jonathan D. Rosenblatt
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