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While cancer has traditionally been considered a genetic disease, mounting evidence indicates an important role for non-genetic (epigenetic) mechanisms. Common anti-cancer drugs have recently been observed to induce the adoption of…

Populations and Evolution · Quantitative Biology 2025-07-04 Einar Bjarki Gunnarsson , Benedikt Vilji Magnússon , Jasmine Foo

Traditional phase I dose finding cancer clinical trial designs aim to determine the maximum tolerated dose (MTD) of the investigational cytotoxic agent based on a single toxicity outcome, assuming a monotone dose-response relationship.…

Methodology · Statistics 2024-11-14 Hao Sun , Hsin-Yu Lin , Jieqi Tu , Revathi Ananthakrishnan , Eunhee Kim

In recent years new cancer treatments improved survival in multiple histologies. Some of these therapeutics, and in particular treatment combinations, are often associated with severe treatment-related adverse events (AEs). It is therefore…

Applications · Statistics 2022-03-10 Steffen Ventz , Lorenzo Trippa

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

Bayesian Optimal Experimental Design (BOED) is a powerful tool to reduce the cost of running a sequence of experiments. When based on the Expected Information Gain (EIG), design optimization corresponds to the maximization of some…

Machine Learning · Statistics 2025-03-14 Jacopo Iollo , Christophe Heinkelé , Pierre Alliez , Florence Forbes

Adaptive radar waveform design grounded in information-theoretic principles is critical for advancing cognitive radar performance in complex environments. This paper investigates the optimization of phase-coded waveforms under constant…

Signal Processing · Electrical Eng. & Systems 2025-05-01 Ruofeng Yu , Caiguang Zhang , Chenyang Luo , Mengdi Bai , Shangqu Yan , Wei Yang , Yaowen Fu

In Oncology, trials evaluating drug combinations are becoming more common. While combination therapies bring the potential for greater efficacy, they also create unique challenges for ensuring drug safety. In Phase-I dose escalation trials…

Applications · Statistics 2023-02-23 Lukas A. Widmer , Andrew Bean , David Ohlssen , Sebastian Weber

Effective therapy of complex diseases requires control of highly non-linear complex networks that remain incompletely characterized. In particular, drug intervention can be seen as control of signaling in cellular networks. Identification…

Quantitative Methods · Quantitative Biology 2009-09-03 Jacob D. Feala , Jorge Cortes , Phillip M. Duxbury , Carlo Piermarocchi , Andrew D. McCulloch , Giovanni Paternostro

Dose-response models express the effect of different dose or exposure levels on a specific outcome. In meta-analysis, where aggregated-level data is available, dose-response evidence is synthesized using either one-stage or two-stage models…

Accurate models of clinical actions and their impacts on disease progression are critical for estimating personalized optimal dynamic treatment regimes (DTRs) in medical/health research, especially in managing chronic conditions.…

Methodology · Statistics 2021-02-19 William Hua , Hongyuan Mei , Sarah Zohar , Magali Giral , Yanxun Xu

Antimicrobial resistance is a threat to public health with millions of deaths linked to drug resistant infections every year. To mitigate resistance, common strategies that are used are combination therapies and therapy switching. However,…

Populations and Evolution · Quantitative Biology 2025-06-02 Juan Magalang , Javier Aguilar , Jose Perico Esguerra , Édgar Roldán , Daniel Sanchez-Taltavull

One of the challenges in the design of confirmatory trials is to deal with uncertainties regarding the optimal target population for a novel drug. Adaptive enrichment designs (AED) which allow for a data-driven selection of one or more…

Applications · Statistics 2020-09-15 Anh Nguyen Duc , Dominik Heinzmann , Claude Berge , Marcel Wolbers

Pharmacodynamic (PD) models are mathematical models of cellular reaction networks that include drug mechanisms of action. These models are useful for studying predictive therapeutic outcomes of novel drug therapies in silico. However, PD…

Molecular Networks · Quantitative Biology 2023-09-27 Natalie M. Isenberg , Susan D. Mertins , Byung-Jun Yoon , Kristofer Reyes , Nathan M. Urban

Opportunistic pharmacokinetic (PK) studies have sparse and imbalanced clinical measurement data, and the impact of sample time errors is an important concern when seeking accurate estimates of treatment response. We evaluated an approximate…

Applications · Statistics 2023-10-25 Hannah L Weeks , Matthew S Shotwell

Novel dose-finding designs, using estimation to assign the best estimated maximum- tolerated-dose (MTD) at each point in the experiment, most commonly via Bayesian techniques, have recently entered large-scale implementation in Phase I…

Methodology · Statistics 2017-01-24 Assaf P. Oron , Peter D. Hoff

This paper introduces a new Phase I design aimed at enhancing the performance of existing methods, including algorithm-based, model-based, and model-assisted designs. The design, developed by integrating the concept of Fisher information,…

Applications · Statistics 2024-12-11 Xiaojun Zhu

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

Despite considerable progress in genome- and proteome-based high-throughput screening methods and rational drug design, the number of successful single target drugs did not increase appreciably during the past decade. Network models suggest…

Molecular Networks · Quantitative Biology 2007-05-23 Peter Csermely , Vilmos Agoston , Sandor Pongor

Objective: We propose a semiautomatic pipeline for radiation therapy treatment planning, combining ideas from machine learning-automated planning and multicriteria optimization (MCO). Approach: Using knowledge extracted from historically…

Medical Physics · Physics 2022-02-16 Tianfang Zhang , Rasmus Bokrantz , Jimmy Olsson

An unprecedented number of new cancer targets are in development, and most are being developed in combination therapies. Early oncology development is strategically challenged in choosing the best combinations to move forward to late stage…

Applications · Statistics 2021-01-08 Linda Z. Sun , Cai , Wu , Xiaoyun , Li , Cong Chen , Emmett V. Schmidt