English
Related papers

Related papers: A Bayesian Optimal Phase II Design for Randomized …

200 papers

In this paper, we study the design and analysis of experiments conducted on a set of units over multiple time periods where the starting time of the treatment may vary by unit. The design problem involves selecting an initial treatment time…

Econometrics · Economics 2023-09-27 Ruoxuan Xiong , Susan Athey , Mohsen Bayati , Guido Imbens

We propose an adaptive design for early phase drug combination cancer trials with the goal of estimating the maximum tolerated dose (MTD). A nonparametric Bayesian model, using beta priors truncated to the set of partially ordered dose…

Applications · Statistics 2019-10-22 Zahra S. Razaee , Galen Wien-Cook , Mourad Tighiouart

It is increasingly common for therapies in oncology to be given in combination. In some cases, patients can benefit from the interaction between two drugs, although often at the risk of higher toxicity. A large number of designs to conduct…

This article develops new closed-form variance expressions for power analyses for commonly used difference-in-differences (DID) and comparative interrupted time series (CITS) panel data estimators. The main contribution is to incorporate…

Methodology · Statistics 2021-10-18 Peter Z. Schochet

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

The conventional more-is-better dose selection paradigm, which targets the maximum tolerated dose (MTD), is not suitable for the development of targeted therapies and immunotherapies as the efficacy of these novel therapies may not increase…

Methodology · Statistics 2023-08-31 Peng Yang , Daniel Li , Ruitao Lin , Bo Huang , Ying Yuan

The primary goal of dose allocation in phase I trials is to minimize patient exposure to subtherapeutic or excessively toxic doses, while accurately recommending a phase II dose that is as close as possible to the maximum tolerated dose…

Methodology · Statistics 2025-03-21 Jiapeng Xu , Dehua Bi , Shenghua Kelly Fan , Bee Leng Lee , Ying Lu

In most clinical trials, patients are randomized with equal probability among treatments to obtain an unbiased estimate of the treatment effect. Response-adaptive randomization (RAR) has been proposed for ethical reasons, where the…

Applications · Statistics 2019-09-16 Thevaa Chandereng , Rick Chappell

Experimental design is crucial for inference where limitations in the data collection procedure are present due to cost or other restrictions. Optimal experimental designs determine parameters that in some appropriate sense make the data…

Machine Learning · Statistics 2016-03-11 Panagiotis Tsilifis , Roger G. Ghanem , Paris Hajali

Datasets used in immunotherapy response prediction are typically small in size, as well as diverse in cancer type, drug administered, and sequencer used. Models often drop in performance when tested on patient cohorts that are not included…

Machine Learning · Computer Science 2026-04-02 Sayed Hashim , Frank Soboczenski , Paul Cairns

Impulse-to-peak response (I2P) analysis for state-space ordinary differential equation (ODE) systems is a well-studied classical problem. However, the techniques employed for I2P optimal control of ODEs have not been extended to partial…

Optimization and Control · Mathematics 2026-04-07 Tristan Thomas , Sachin Shivakumar , Javad Mohammadpour Velni

Phase-3 clinical trials provide the highest level of evidence on drug safety and effectiveness needed for market approval by implementing large randomized controlled trials (RCTs). However, 30-40% of these trials fail mainly because such…

Applications · Statistics 2024-01-09 Sayeri Lala , Niraj K. Jha

In early-phase cancer clinical trials, the limited availability of data presents significant challenges in developing a framework to efficiently quantify treatment effectiveness. To address this, we propose a novel utility-based Bayesian…

Methodology · Statistics 2025-06-10 Saurabh Bhandari , Michael J. Daniels , Chenguang Wang

Phase I clinical trials are designed to test the safety (non-toxicity) of drugs and find the maximum tolerated dose (MTD). This task becomes significantly more challenging when multiple-drug dose-combinations (DC) are involved, due to the…

Machine Learning · Computer Science 2021-01-27 Hyun-Suk Lee , Cong Shen , William Zame , Jang-Won Lee , Mihaela van der Schaar

Traditionally, optimization of radiation therapy (RT) treatment plans has been done before the initiation of RT course, using population-wide estimates for patients' response to therapy. However, recent technological advancements have…

Medical Physics · Physics 2021-02-15 Stefan C. M. ten Eikelder , Ali Ajdari , Thomas Bortfeld , Dick den Hertog

Randomized discontinuation design (RDD) is an enrichment strategy commonly used to address limitations of traditional placebo-controlled trials, particularly the ethical concern of prolonged placebo exposure. RDD consists of two phases: an…

Methodology · Statistics 2025-06-03 Ayon Mukherjee , Oleksandr Sverdlov , Ngoc-Thuy Ha , Yu Deng

One of the main goals of sequential, multiple assignment, randomized trials (SMART) is to find the most efficacious design embedded dynamic treatment regimes. The analysis method known as multiple comparisons with the best (MCB) allows…

Methodology · Statistics 2020-08-07 William J. Artman , Ashkan Ertefaie , Kevin G. Lynch , James R. McKay

Background: We aimed to design a Bayesian adaption trial through extensive simulations to determine values for key design parameters, demonstrate error rates, and establish the expected sample size. The complexity of the proposed outcome…

Optimizing doses for multiple indications is challenging. The pooled approach of finding a single optimal biological dose (OBD) for all indications ignores that dose-response or dose-toxicity curves may differ between indications, resulting…

Methodology · Statistics 2024-10-07 Shuqi Wang , Peter F. Thall , Kentaro Takeda , Ying Yuan

With the development of novel therapies such as molecularly targeted agents and immunotherapy, the maximum tolerated dose paradigm that "more is better" does not necessarily hold anymore. In this context, doses and schedules of novel…