UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs

Burnett, T; Mozgunov, P; Pallmann, P; Villar, SS; Wheeler, GM; Jaki, T; (2020) Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs. BMC Medicine , 18 , Article 352. 10.1186/s12916-020-01808-2. Green open access

[thumbnail of Adding flexibility to clinical trial designs - an example-based guide - Burnett et al 2020.pdf]
Preview
Text
Adding flexibility to clinical trial designs - an example-based guide - Burnett et al 2020.pdf - Published Version

Download (1MB) | Preview

Abstract

Adaptive designs for clinical trials permit alterations to a study in response to accumulating data in order to make trials more flexible, ethical, and efficient. These benefits are achieved while preserving the integrity and validity of the trial, through the pre-specification and proper adjustment for the possible alterations during the course of the trial. Despite much research in the statistical literature highlighting the potential advantages of adaptive designs over traditional fixed designs, the uptake of such methods in clinical research has been slow. One major reason for this is that different adaptations to trial designs, as well as their advantages and limitations, remain unfamiliar to large parts of the clinical community. The aim of this paper is to clarify where adaptive designs can be used to address specific questions of scientific interest; we introduce the main features of adaptive designs and commonly used terminology, highlighting their utility and pitfalls, and illustrate their use through case studies of adaptive trials ranging from early-phase dose escalation to confirmatory phase III studies.

Type: Article
Title: Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs
Open access status: An open access version is available from UCL Discovery
DOI: 10.1186/s12916-020-01808-2
Publisher version: https://doi.org/10.1186/s12916-020-01808-2
Language: English
Additional information: This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
Keywords: Novel designs, Innovative trials, Efficient methods, Enrichment designs, Multi-arm multi-stage platform trials
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > CRUK Cancer Trials Centre
URI: https://discovery.ucl.ac.uk/id/eprint/10115464
Downloads since deposit
23Downloads
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item