The University of Southampton
University of Southampton Institutional Repository

The development of a set of key points to aid clinicians and researchers in designing and conducting n-of-1 trials

The development of a set of key points to aid clinicians and researchers in designing and conducting n-of-1 trials
The development of a set of key points to aid clinicians and researchers in designing and conducting n-of-1 trials

Introduction: n-of-1 trials are undertaken to optimise the evaluation of health technologies in individual patients. They involve a single patient receiving treatments, both interventional and control, consecutively over set periods of time, the order of which is decided at random. Although n-of-1 trials are undertaken in medical research it could be argued they have the utility to be undertaken more frequently. We undertook the National Institute for Health Research (NIHR) commissioned DIAMOND (Development of generalisable methodology for n-of-1 trials delivery for very low volume treatments) project to develop key points to assist clinicians and researchers in designing and conducting n-of-1 trials.

Methods: the key points were developed by undertaking a stakeholder workshop, followed by a discussion within the study team and then a stakeholder dissemination and feedback event. The stakeholder workshop sought to gain the perspectives of a variety of stakeholders (including clinicians, researchers and patient representatives) on the design and use of n-of-1 trials. A discussion between the study team was held to reflect on the workshop and draft the key points. Lastly, the stakeholders from the workshop were invited to a dissemination and feedback session where the proposed key points were presented and their feedback gained.

Results: a set of 22 key points were developed based on the insights from the workshop and subsequent discussions. They provide guidance on when an n-of-1 trial might be a viable or appropriate study design and discuss key decisions involved in the design of n-of-1 trials, including determining an appropriate number of treatment periods and cycles, the choice of comparator, recommended approaches to randomisation and blinding, the use of washout periods and approaches to analysis.

Conclusions: the key points developed in the project will support clinical researchers to understand key considerations when designing n-of-1 trials. It is hoped they will support the wider implementation of the study design.

Clinical Trials as Topic/methods, Consensus, Humans, Research Design, Research Personnel, Stakeholder Participation, Technology Assessment, Biomedical, Treatment Outcome, Key points, Cross-over trials, Rare diseases, n-of-1, Personalised medicine
1745-6215
Chatters, Robin
751cd9f6-f06f-4f96-b3e9-ece8aed53468
Hawksworth, Olivia
62a8ba73-7a27-41c7-a8a8-087f69b9e936
Julious, Steven
f43c38eb-8aaa-4f4c-a770-a68bc2321360
Cook, Andrew
ab9c7bb3-974a-4db9-b3c2-9942988005d5
Chatters, Robin
751cd9f6-f06f-4f96-b3e9-ece8aed53468
Hawksworth, Olivia
62a8ba73-7a27-41c7-a8a8-087f69b9e936
Julious, Steven
f43c38eb-8aaa-4f4c-a770-a68bc2321360
Cook, Andrew
ab9c7bb3-974a-4db9-b3c2-9942988005d5

Chatters, Robin, Hawksworth, Olivia, Julious, Steven and Cook, Andrew (2024) The development of a set of key points to aid clinicians and researchers in designing and conducting n-of-1 trials. Trials, 25 (1), [473]. (doi:10.1186/s13063-024-08261-z).

Record type: Article

Abstract

Introduction: n-of-1 trials are undertaken to optimise the evaluation of health technologies in individual patients. They involve a single patient receiving treatments, both interventional and control, consecutively over set periods of time, the order of which is decided at random. Although n-of-1 trials are undertaken in medical research it could be argued they have the utility to be undertaken more frequently. We undertook the National Institute for Health Research (NIHR) commissioned DIAMOND (Development of generalisable methodology for n-of-1 trials delivery for very low volume treatments) project to develop key points to assist clinicians and researchers in designing and conducting n-of-1 trials.

Methods: the key points were developed by undertaking a stakeholder workshop, followed by a discussion within the study team and then a stakeholder dissemination and feedback event. The stakeholder workshop sought to gain the perspectives of a variety of stakeholders (including clinicians, researchers and patient representatives) on the design and use of n-of-1 trials. A discussion between the study team was held to reflect on the workshop and draft the key points. Lastly, the stakeholders from the workshop were invited to a dissemination and feedback session where the proposed key points were presented and their feedback gained.

Results: a set of 22 key points were developed based on the insights from the workshop and subsequent discussions. They provide guidance on when an n-of-1 trial might be a viable or appropriate study design and discuss key decisions involved in the design of n-of-1 trials, including determining an appropriate number of treatment periods and cycles, the choice of comparator, recommended approaches to randomisation and blinding, the use of washout periods and approaches to analysis.

Conclusions: the key points developed in the project will support clinical researchers to understand key considerations when designing n-of-1 trials. It is hoped they will support the wider implementation of the study design.

Text
s13063-024-08261-z - Version of Record
Available under License Creative Commons Attribution.
Download (1MB)

More information

Accepted/In Press date: 18 June 2024
Published date: 11 July 2024
Additional Information: Publisher Copyright: © The Author(s) 2024.
Keywords: Clinical Trials as Topic/methods, Consensus, Humans, Research Design, Research Personnel, Stakeholder Participation, Technology Assessment, Biomedical, Treatment Outcome, Key points, Cross-over trials, Rare diseases, n-of-1, Personalised medicine

Identifiers

Local EPrints ID: 492778
URI: http://eprints.soton.ac.uk/id/eprint/492778
ISSN: 1745-6215
PURE UUID: 6ec2115c-7a5f-4fa3-ad84-1ba8408a0846
ORCID for Andrew Cook: ORCID iD orcid.org/0000-0002-6680-439X

Catalogue record

Date deposited: 13 Aug 2024 17:13
Last modified: 14 Aug 2024 01:40

Export record

Altmetrics

Contributors

Author: Robin Chatters
Author: Olivia Hawksworth
Author: Steven Julious
Author: Andrew Cook ORCID iD

Download statistics

Downloads from ePrints over the past year. Other digital versions may also be available to download e.g. from the publisher's website.

View more statistics

Atom RSS 1.0 RSS 2.0

Contact ePrints Soton: eprints@soton.ac.uk

ePrints Soton supports OAI 2.0 with a base URL of http://eprints.soton.ac.uk/cgi/oai2

This repository has been built using EPrints software, developed at the University of Southampton, but available to everyone to use.

We use cookies to ensure that we give you the best experience on our website. If you continue without changing your settings, we will assume that you are happy to receive cookies on the University of Southampton website.

×