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Crossover designs are an extremely useful tool to investigators, whilst group sequential methods have proven highly proficient at improving the efficiency of parallel group trials. Yet, group sequential methods and crossover designs have…

Methodology · Statistics 2017-10-11 Michael Grayling , James Wason , Adrian Mander

Background. Designing trials to reduce treatment duration is important in several therapeutic areas, including TB and antibiotics. We recently proposed a new randomised trial design to overcome some of the limitations of standard two-arm…

Clinical trials usually involve sequential patient entry. When designing a clinical trial, it is often desirable to include a provision for interim analyses of accumulating data with the potential for stopping the trial early. We review…

Methodology · Statistics 2023-03-13 Tianjian Zhou , Yuan Ji

Platform trials evaluate multiple experimental treatments against a common control group (and/or against each other), which often reduces the trial duration and sample size. Bayesian platform designs offer several practical advantages,…

Methodology · Statistics 2025-07-18 Luke Hagar , Lara Maleyeff , Shirin Golchi , Dick Menzies

The two-stage preference design (TSPD) enables the inference for treatment efficacy while allowing for incorporation of patient preference to treatment. It can provide unbiased estimates for selection and preference effects, where a…

Methodology · Statistics 2023-11-28 Ruyi Liu , Fan Li , Denise Esserman , Mary M. Ryan

For indications where only unstable reference treatments are available and use of placebo is ethically justified, three-arm `gold standard' designs with an experimental, reference and placebo arm are recommended for non-inferiority trials.…

Methodology · Statistics 2022-10-18 Werner Brannath , Martin Scharpenberg , Sylvia Schmidt

The Ramsey sequence is a canonical example of a quantum phase measurement for a spin qubit. In Ramsey measurements, the measurement efficiency can be optimized through careful selection of settings for the phase accumulation time setting,…

Quantum Physics · Physics 2021-10-27 Robert D. McMichael , Sergey Dushenko , Sean M. Blakley

Background: Well-designed phase II trials must have acceptable error rates relative to a pre-specified success criterion, usually a statistically significant p-value. Such standard designs may not always suffice from a clinical perspective…

Applications · Statistics 2020-02-10 Satrajit Roychoudhury , Nicolas Scheuer , Beat Neuenschwander

We use the lens of weak signal asymptotics to study a class of sequentially randomized experiments, including those that arise in solving multi-armed bandit problems. In an experiment with $n$ time steps, we let the mean reward gaps between…

Statistics Theory · Mathematics 2023-06-26 Xu Kuang , Stefan Wager

Group sequential design (GSD) is widely used in clinical trials in which correlated tests of multiple hypotheses are used. Multiple primary objectives resulting in tests with known correlations include evaluating 1) multiple experimental…

Methodology · Statistics 2021-03-22 Keaven M. Anderson , Zifang Guo , Jing Zhao , Linda Z. Sun

The Bayes factor, the data-based updating factor from prior to posterior odds, is a principled measure of relative evidence for two competing hypotheses. It is naturally suited to sequential data analysis in settings such as clinical trials…

Methodology · Statistics 2026-01-07 Samuel Pawel , Leonhard Held

An important tool to evaluate the performance of any design is an optimal benchmark proposed by O'Quigley and others (2002, Biostatistics 3(1), 51-56) that provides an upper bound on the performance of a design under a given scenario. The…

Statistics Theory · Mathematics 2018-03-06 Pavel Mozgunov , Thomas Jaki , Xavier Paoletti

This paper addresses the following general scenario: A scientist wishes to perform a battery of experiments, each generating a sequential stream of data, to investigate some phenomenon. The scientist would like to control the overall error…

Methodology · Statistics 2014-05-12 Jay Bartroff , Jinlin Song

This article deals with the sequential design of experiments for (deterministic or stochastic) multi-fidelity numerical simulators, that is, simulators that offer control over the accuracy of simulation of the physical phenomenon or system…

Delayed outcomes are ubiquitous in online experimentation. When such a temporal dimension is present, treatment influences not only the outcome value but also the outcome timing, which can move in opposite directions. Motivated by the…

Methodology · Statistics 2026-03-30 Michael Lindon , Nathan Kallus

In this paper, we propose a general method for testing composite hypotheses. Our idea is to use confidence limits to define stopping and decision rules. The requirements of operating characteristic function can be satisfied by adjusting the…

Statistics Theory · Mathematics 2012-02-10 Xinjia Chen

In sequential experiments, subjects become available for the study over a period of time, and covariates are often measured at the time of arrival. We consider the setting where the sample size is fixed but covariate values are unknown…

Methodology · Statistics 2020-05-28 Mia S. Tackney , David C. Woods , Ilya Shpitser

Clinicians and researchers alike are increasingly interested in how best to personalize interventions. A dynamic treatment regimen (DTR) is a sequence of pre-specified decision rules which can be used to guide the delivery of a sequence of…

Adaptive designs(AD) are a broad class of trial designs that allow preplanned modifications based on patient data providing improved efficiency and flexibility. However, a delay in observing the primary outcome variable can harm this added…

Methodology · Statistics 2025-09-26 Aritra Mukherjee , Michael J. Grayling , James M. S. Wason

For a trial with primary endpoint overall survival for a molecule with curative potential, statistical methods that rely on the proportional hazards assumption may underestimate the power and the time to final analysis. We show how a cure…

Applications · Statistics 2023-04-17 Kaspar Rufibach , Dominik Heinzmann , Annabelle Monnet
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