scispace - formally typeset
Open AccessJournal ArticleDOI

A simulation study for comparing testing statistics in response-adaptive randomization

Reads0
Chats0
TLDR
The Cook's correction to chi-square test and Williams' correction to log-likelihood-ratio test are generally recommended for hypothesis test in response-adaptive randomization, especially when sample sizes are small.
Abstract
Background Response-adaptive randomizations are able to assign more patients in a comparative clinical trial to the tentatively better treatment. However, due to the adaptation in patient allocation, the samples to be compared are no longer independent. At large sample sizes, many asymptotic properties of test statistics derived for independent sample comparison are still applicable in adaptive randomization provided that the patient allocation ratio converges to an appropriate target asymptotically. However, the small sample properties of commonly used test statistics in response-adaptive randomization are not fully studied.

read more

Content maybe subject to copyright    Report

Citations
More filters
Posted Content

Response adaptive designs for binary responses: how to offer patient benefit while being robust to time trends?

TL;DR: In this paper, the authors discuss and address a major criticism of RAR: the undesirable type I error inflation due to unknown time trends in the trial, and make recommendations of which correction methods are most suitable in the rare disease context for several RAR rules, differentiating between two-armed and the multi-armed case.
Posted Content

Response-adaptive randomization in clinical trials: from myths to practical considerations

TL;DR: This work aims to address a persistent gap in understanding of response-adaptive randomization by providing a critical, balanced and updated review of methodological and practical issues to consider when debating the use of RAR in clinical trials.
Journal ArticleDOI

Looking ahead: clinical trial design in adult congenital heart disease.

TL;DR: In looking ahead, examples of creative methodological solutions to maximizing the efficiency of clinical trials for rare diseases are discussed, including Bayesian analyses, outcome-adaptive randomization and internal pilot studies.
Journal ArticleDOI

Frequentist operating characteristics of Bayesian optimal designs via simulation

TL;DR: This paper proposes a general simulation‐based approach to compare frequentist designs with Bayesian adaptive designs based on frequentist criteria such as power and to compute valid frequentist p‐values and results quantify the trade‐off between power and the optimal assignment of patients to treatments within the trial.
Journal ArticleDOI

Adaptive Clinical Trial Design: An Overview and Potential Applications in Dermatology.

TL;DR: This article defines adaptive trials; gives examples of the most common types; highlight the pros, cons, and ethical considerations of these designs; and illustrate how these tools can be applied to drug development in dermatology.
References
More filters
Book

Group Sequential Methods with Applications to Clinical Trials

TL;DR: A short history of sequential and group sequential methods can be found in this paper, where the authors present a road map for the application of two-sided tests for comparing two treatments with normal response of known variance.
Journal ArticleDOI

The Randomized Play-the-Winner Rule in Medical Trials

TL;DR: In this article, a simple randomized treatment assignment rule is proposed and analyzed in a sequential medical trial, and on the average this rule assigns more patients to the better treatment, and it is applicable to the case where patients have delayed responses to treatments.
Journal ArticleDOI

Play the Winner Rule and the Controlled Clinical Trial

TL;DR: Investigation of the conduct of a clinical trial where the “Play the Winner Rule” (PWR) is used to assign patients to the different therapies shows that over a wide range of situations this rule leads to near optimum results when used in a two-stage manner.

Teacher's Corner Simple and Effective Confidence Intervals for Proportions and Differences of Proportions Result from Adding Two Successes and Two Failures

Alan Agresti, +1 more
TL;DR: In this article, simple adjustments of these confidence intervals based on adding four pseudo observations, half of each type, perform surprisingly well even for small samples, and one can bypass awkward sample size guidelines and use the same formulas with small and large samples.
Related Papers (5)