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A sequential multiple assignment randomized trial (SMART) facilitates comparison of multiple adaptive treatment strategies (ATSs) simultaneously. Previous studies have established a framework to test the homogeneity of multiple ATSs by a…

统计方法学 · 统计学 2022-11-04 Liwen Wu , Junyao Wang , Abdus S. Wahed

Multi-arm bandits are gaining popularity as they enable real-world sequential decision-making across application areas, including clinical trials, recommender systems, and online decision-making. Consequently, there is an increased desire…

统计方法学 · 统计学 2023-03-01 Dae Woong Ham , Iavor Bojinov , Michael Lindon , Martin Tingley

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…

应用统计 · 统计学 2023-04-14 Zhenghao Jiang , Gu Mi , Ji Lin , Christelle Lorenzato , Yuan Ji

Clinical trials are an integral component of medical research. Trials require careful design to, for example, maintain the safety of participants, use resources efficiently and allow clinically meaningful conclusions to be drawn. Adaptive…

统计方法学 · 统计学 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

Factorial designs are frequently used in different fields of science, e.g. psychological, medical or biometric studies. Standard approaches, as the ANOVA $F$-test, make different assumptions on the distribution of the error terms, the…

统计方法学 · 统计学 2018-02-21 Maria Umlauft

A multi-arm multi-stage trial is a multi-arm trial which includes interim analyses - analysing the data at certain specified points, generally discontinuing treatments which are concluded to not work and proceeding with the remainder. It is…

统计方法学 · 统计学 2022-11-14 Martin Law

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…

统计方法学 · 统计学 2023-08-25 Peter Greenstreet , Thomas Jaki , Alun Bedding , Pavel Mozgunov

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…

统计方法学 · 统计学 2010-12-01 Jay Bartroff , Tze Leung Lai

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

统计方法学 · 统计学 2010-12-01 Peter F. Thall

Background: Advanced adaptive randomised clinical trials are increasingly used. Compared to their conventional counterparts, their flexibility may make them more efficient, increase the probability of obtaining conclusive results without…

Ever since the seminal work of R. A. Fisher and F. Yates, factorial designs have been an important experimental tool to simultaneously estimate the effects of multiple treatment factors. In factorial designs, the number of treatment…

统计方法学 · 统计学 2024-03-21 Lei Shi , Jingshen Wang , Peng Ding

Online healthcare communities provide users with various healthcare interventions to promote healthy behavior and improve adherence. When faced with too many intervention choices, however, individuals may find it difficult to decide which…

机器学习 · 计算机科学 2020-09-15 Tongxin Zhou , Yingfei Wang , Lu , Yan , Yong Tan

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

统计方法学 · 统计学 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

Adaptive seamless designs combine confirmatory testing, a domain of phase III trials, with features such as treatment or subgroup selection, typically associated with phase II trials. They promise to increase the efficiency of development…

统计方法学 · 统计学 2019-01-25 Tim Friede , Nigel Stallard , Nicholas Parsons

In a sequential multiple-assignment randomized trial (SMART), a sequence of treatments is given to a patient over multiple stages. In each stage, randomization may be done to allocate patients to different treatment groups. Even though…

统计方法学 · 统计学 2024-01-09 Rik Ghosh , Bibhas Chakraborty , Inbal Nahum-Shani , Megan E. Patrick , Palash Ghosh

We propose a multiple cohort expansion (MUCE) approach as a design or analysis method for phase 1b multiple expansion cohort trials, which are novel first-in-human studies conducted following phase 1a dose escalation. The MUCE design is…

统计方法学 · 统计学 2025-05-21 Jiaying Lyu , Tianjian Zhou , Shijie Yuan , Wentian Guo , Yuan Ji

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,…

应用统计 · 统计学 2024-12-11 Xiaojun Zhu

Multi-arm trials are gaining interest in practice given the statistical and logistical advantages they can offer. The standard approach uses a fixed allocation ratio, but there is a call for making it adaptive and skewing the allocation of…

统计方法学 · 统计学 2026-01-16 Gianmarco Caruso , Pavel Mozgunov

Experimentation is crucial for managers to rigorously quantify the value of a change and determine if it leads to a statistically significant improvement over the status quo. As companies increasingly mandate that all changes undergo…

统计方法学 · 统计学 2024-10-16 Biyonka Liang , Iavor Bojinov

Nowadays, more and more clinical trials choose combinational agents as the intervention to achieve better therapeutic responses. However, dose-finding for combinational agents is much more complicated than single agent as the full order of…

应用统计 · 统计学 2022-08-05 Shu Wang , Ji-Hyun Lee