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We study the problem of finding the optimal dosage in early stage clinical trials through the multi-armed bandit lens. We advocate the use of the Thompson Sampling principle, a flexible algorithm that can accommodate different types of…

Machine Learning · Statistics 2020-04-09 Maryam Aziz , Emilie Kaufmann , Marie-Karelle Riviere

Adaptive experimental designs have gained popularity in clinical trials and online experiments. Unlike traditional, fixed experimental designs, adaptive designs can dynamically adjust treatment randomization probabilities and other design…

Methodology · Statistics 2025-08-19 Wenxin Zhang , Mark van der Laan

In this paper, the issue of designing an iterative-detection-and-decoding (IDD)-aided receiver, relying on the low-complexity probabilistic data association (PDA) method, is addressed for turbo-coded multiple-input-multiple-output (MIMO)…

Information Theory · Computer Science 2013-11-08 Shaoshi Yang , Li Wang , Tiejun Lv , Lajos Hanzo

We propose the BayesDose-Framework, a Bayesian approach for fast and accurate dose prediction in proton therapy. Our framework is based on a previously published deterministic LSTM model and is trained and evaluated on simulated beamlet…

Medical Physics · Physics 2023-07-04 Luke Voss , Ahmad Neishabouri , Tim Ortkamp , Andrea Mairani , Niklas Wahl

Model-informed precision dosing (MIPD) is a quantitative dosing framework that combines prior knowledge on the drug-disease-patient system with patient data from therapeutic drug/ biomarker monitoring (TDM) to support individualized dosing…

Optimization and Control · Mathematics 2021-06-08 Corinna Maier , Jana de Wiljes , Niklas Hartung , Charlotte Kloft , Wilhelm Huisinga

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

Benchmark dose analysis aims to estimate the level of exposure to a toxin that results in a clinically-significant adverse outcome and quantifies uncertainty using the lower limit of a confidence interval for this level. We develop a novel…

The main objective of dose finding trials is to find an optimal dose amongst a candidate set for further research. The trial design in oncology proceeds in stages with a decision as to how to treat the next group of patients made at every…

Methodology · Statistics 2025-10-21 Andrew Hall , Duncan Wilson , Stuart Barber , Sarah R Brown

Nonlinear regression models addressing both efficacy and toxicity outcomes are increasingly used in dose-finding trials, such as in pharmaceutical drug development. However, research on related experimental design problems for corresponding…

Methodology · Statistics 2016-01-06 Holger Dette , Katrin Kettelhake , Kirsten Schorning , Weng Kee Wong , Frank Bretz

Dose-finding clinical trials in oncology aim to estimate the maximum tolerated dose (MTD), based on safety traditionally obtained from the clinician's perspective. While the collection of patient-reported outcomes (PROs) has been advocated…

Applications · Statistics 2023-04-03 Anaïs Andrillon , Lucie Biard , Shing M. Lee

In this paper we consider two-stage adaptive dose-response study designs, where the study design is changed at an interim analysis based on the information collected so far. In a simulation study, two approaches will be compared for these…

Methodology · Statistics 2016-02-08 Emma McCallum , Björn Bornkamp

Cancer drug response prediction (DRP) models present a promising approach towards precision oncology, tailoring treatments to individual patient profiles. While deep learning (DL) methods have shown great potential in this area, models that…

An important objective in environmental risk assessment is estimation of minimum exposure levels, called Benchmark Doses (BMDs) that induce a pre-specified Benchmark Response (BMR) in a target population. Established inferential approaches…

Methodology · Statistics 2014-06-12 Qijun Fang , Walter W. Piegorsch , Katherine Y. Barnes

We propose a rule-based statistical design for combination dose-finding trials with two agents. The Ci3+3 design is an extension of the i3+3 design with simple decision rules comparing the observed toxicity rates and equivalence intervals…

Applications · Statistics 2023-03-29 Shijie Yuan , Tianjian Zhou , Yawen Lin , Yuan Ji

We consider design issues for toxicology studies when we have a continuous response and the true mean response is only known to be a member of a class of nested models. This class of non-linear models was proposed by toxicologists who were…

Statistics Theory · Mathematics 2010-11-29 Holger Dette , Andrey Pepelyshev , Piter Shpilev , Weng Kee Wong

Purpose: We propose a general framework for quantifying predictive uncertainties of dose-related quantities and leveraging this information in a dose mimicking problem in the context of automated radiation therapy treatment planning.…

Medical Physics · Physics 2021-09-08 Tianfang Zhang , Rasmus Bokrantz , Jimmy Olsson

In the era of targeted therapy, there has been increasing concern about the development of oncology drugs based on the "more is better" paradigm, developed decades ago for chemotherapy. Recently, the US Food and Drug Administration (FDA)…

Methodology · Statistics 2022-09-07 Beibei Guo , Ying Yuan

We propose BaySize, a sample size calculator for phase I clinical trials using Bayesian models. BaySize applies the concept of effect size in dose finding, assuming the MTD is defined based on an equivalence interval. Leveraging a decision…

Methodology · Statistics 2023-03-29 Xiaolei Lin , Jiaying Lyu , Shijie Yuan , Sue-Jane Wang , Yuan Ji

Optimal design of a Phase I cancer trial can be formulated as a stochastic optimization problem. By making use of recent advances in approximate dynamic programming to tackle the problem, we develop an approximation of the Bayesian optimal…

Methodology · Statistics 2010-12-01 Jay Bartroff , Tze Leung Lai

Rapid and accurate identification of Venous thromboembolism (VTE), a severe cardiovascular condition including deep vein thrombosis (DVT) and pulmonary embolism (PE), is important for effective treatment. Leveraging Natural Language…

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