Stakeholders’ views on the routine use of n-of-1 trials to improve clinical care and to make resource allocation decisions for drug use
Jane Nikles A D , Geoffrey K. Mitchell A , Alexandra Clavarino A , Michael J. Yelland B and Christopher B. Del Mar CA School of Medicine, The University of Queensland, Brisbane, QLD 4006, Australia.
B School of Medicine, Griffith University, Brisbane, QLD 4131, Australia.
C Faculty of Health Sciences and Medicine, Bond University, Gold Coast, QLD 4229, Australia.
D Corresponding author. Email: uqjnikle@uq.edu.au
Australian Health Review 34(1) 131-136 https://doi.org/10.1071/AH09654
Submitted: 27 May 2008 Accepted: 1 April 2009 Published: 25 March 2010
Abstract
N-of-1 trials are empirical formal tests using a within-patient randomised, double-blind, cross-over comparison of drug and placebo (or another drug), which we adapted to study individual patients’ responses as a clinical tool to guide clinical management. We administered semi-structured interviews to gauge stakeholder perspectives on the possibility of using routine n-of-1 trials for this purpose. Stakeholders included government and non-government health care sector, and patient, clinician and consumer, organisations. Stakeholders supported more widespread implementation of n-of-1 trials, in a targeted fashion, with some caveats. Barriers to their widespread implementation included constraints on doctors’ time, doctors’ acceptance, drug company acceptance, patient willingness, and cost. Strategies for overcoming barriers included conditional Pharmaceutical Benefits Scheme listing if cost-effective. There was little consensus on which model of n-of-1 trial implementation would be most effective. We discuss different approaches to addressing the several concerns raised to enable widespread introduction of n-of-1 trials into routine clinical practice as a decision tool.
What is known about the topic? No previous publications have explored methods of implementation and gathered stakeholder’s views on widespread use of n-of-1 trials.
What does this paper add? N-of-1 trials could have a role in making resource allocation decisions for medications, and in guiding treatment choices for patients.
What are the implications for practitioners? There is potential for n-of-1 trials to be used more widely, including as part of normal medical practice for targeted illnesses and groups of patients. This could deliver clinical and economic benefits throughout the health care system.
Acknowledgement
We thank the Australian Health Ministers’ Advisory Council for funding; Richard Clark and Brita Pekarsky for their contributions in the early stages of the project; Richard Clark for conducting some interviews; and Brita Pekarsky for contributing the model shown in the Box.
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