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The goal of uplift modeling is to recommend actions that optimize specific outcomes by determining which entities should receive treatment. One common approach involves two steps: first, an inference step that estimates conditional average…

Machine Learning · Computer Science 2025-05-21 Simon De Vos , Christopher Bockel-Rickermann , Stefan Lessmann , Wouter Verbeke

A master protocol trial uses a single overarching protocol to test multiple therapies, often across several diseases or subtypes. Although such trials offer considerable flexibility and efficiency, their constrained and non-uniform…

Methodology · Statistics 2026-04-29 Shiyu Wan , Yuhan Qian , Yanyao Yi , Nicole Mayer-Hamblett , Patrick J. Heagerty , Ting Ye

Consider an experiment, where a new drug is tested for the first time on human subjects - healthy volunteers. Such experiments are often performed as dose-escalation studies: a set of increasing doses is pre-selected, individuals are…

Statistics Theory · Mathematics 2016-08-17 Samuel Rosa , Radoslav Harman

An accurately identified maximum tolerated dose (MTD) serves as the cornerstone of successful subsequent phases in oncology drug development. Bayesian logistic regression model (BLRM) is a popular and versatile model-based dose-finding…

Methodology · Statistics 2021-05-17 Hongtao Zhang , Alan Y Chiang , Jixian Wang

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

This work introduces the Burdened Bayesian Logistic Regression Model (BBLRM), an enhancement of the Bayesian Logistic Regression Model (BLRM) for dose-finding in phase I oncology trials. The BLRM determines the maximum tolerated dose (MTD)…

Methodology · Statistics 2025-08-18 Andrea Nizzardo , Luca Genetti , Marco Pergher

We consider the optimal design problem for identifying effective dose combinations within drug combination studies where the effect of the combination of two drugs is investigated. Drug combination studies are becoming increasingly…

Exploratory cancer drug studies test multiple tumor cell lines against multiple candidate drugs. The goal in each paired (cell line, drug) experiment is to map out the dose-response curve of the cell line as the dose level of the drug…

Machine Learning · Statistics 2021-03-23 Wesley Tansey , Christopher Tosh , David M. Blei

A major practical impediment when implementing adaptive dose-finding designs is that the toxicity outcome used by the decision rules may not be observed shortly after the initiation of the treatment. To address this issue, we propose the…

Applications · Statistics 2014-01-09 Suyu Liu , Guosheng Yin , Ying Yuan

In parametric Bayesian designs of early phase cancer clinical trials with drug combinations exploring a discrete set of partially ordered doses, several authors claimed that there is no added value in including an interaction term to model…

Methodology · Statistics 2022-08-12 Mourad Tighiouart , José L. Jiménez , Marcio A. Diniz , André Rogatko

Two useful strategies to speed up drug development are to increase the patient accrual rate and use novel adaptive designs. Unfortunately, these two strategies often conflict when the evaluation of the outcome cannot keep pace with the…

Methodology · Statistics 2018-07-24 Ruitao Lin , Ying Yuan

An individualized dose rule recommends a dose level within a continuous safe dose range based on patient level information such as physical conditions, genetic factors and medication histories. Traditionally, personalized dose finding…

Methodology · Statistics 2020-07-21 Liangyu Zhu , Wenbin Lu , Michael R. Kosorok , Rui Song

The Project Optimus initiative by the FDA's Oncology Center of Excellence is widely viewed as a groundbreaking effort to change the $\textit{status quo}$ of conventional dose-finding strategies in oncology. Unlike in other therapeutic areas…

Applications · Statistics 2023-04-14 Zhenghao Jiang , Gu Mi , Ji Lin , Christelle Lorenzato , Yuan Ji

Adaptive therapy is a dynamic cancer treatment protocol that updates (or "adapts") treatment decisions in anticipation of evolving tumor dynamics. This broad term encompasses many possible dynamic treatment protocols of patient-specific…

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

The effects of molecularly targeted drug perturbations on cellular activities and fates are difficult to predict using intuition alone because of the complex behaviors of cellular regulatory networks. An approach to overcoming this problem…

Systems and Control · Computer Science 2019-01-15 Afroza Shirin , Isaac Klickstein , Song Feng , Yen Ting Lin , William S. Hlavacek , Francesco Sorrentino

We investigate adaptive protocols for the elimination or reduction of the use of medications or addictive substances. We formalize this problem as online optimization, minimizing the cumulative dose subject to constraints on well-being. We…

Optimization and Control · Mathematics 2023-09-22 Paula Gradu , Benjamin Recht

Phase I clinical trials are essential to bringing novel therapies from chemical development to widespread use. Traditional approaches to dose-finding in Phase I trials, such as the '3+3' method and the Continual Reassessment Method (CRM),…

Methodology · Statistics 2024-03-04 Luka Kovacevic , Thomas Jaki , Helen Barnett , Pavel Mozgunov

Many drugs used therapeutically or recreationally induce tolerance: the effect of the substance decreases with repeated use. This phenomenon may reduce the efficacy of the substance unless dosage is increased beyond what is healthy for the…

Dynamical Systems · Mathematics 2024-03-19 Golrokh Nouri , Paul Tupper

Purpose: Radiation therapy treatment planning is a trial-and-error, often time-consuming process. An optimal dose distribution based on a specific anatomy can be predicted by pre-trained deep learning (DL) models. However, dose…

Medical Physics · Physics 2021-09-15 Jianhui Ma , Dan Nguyen , Ti Bai , Michael Folkerts , Xun Jia , Weiguo Lu , Linghong Zhou , Steve Jiang
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