Using N-of-1 Trials to improve patient management and save costs

Scuffham, Paul A., Nikles, Jane, Mitchell, Geoffrey K., Yelland, Michael J., Vine, Norma, Poulos, Christopher J., Pillans, Peter I., Bashford, Guy, del Mar, Chris, Schluter, Philip J. and Glasziou, Paul (2010) Using N-of-1 Trials to improve patient management and save costs. Journal of General Internal Medicine, 25 9: 906-913. doi:10.1007/s11606-010-1352-7

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Author Scuffham, Paul A.
Nikles, Jane
Mitchell, Geoffrey K.
Yelland, Michael J.
Vine, Norma
Poulos, Christopher J.
Pillans, Peter I.
Bashford, Guy
del Mar, Chris
Schluter, Philip J.
Glasziou, Paul
Title Using N-of-1 Trials to improve patient management and save costs
Journal name Journal of General Internal Medicine   Check publisher's open access policy
ISSN 0884-8734
Publication date 2010-09
Sub-type Article (original research)
DOI 10.1007/s11606-010-1352-7
Volume 25
Issue 9
Start page 906
End page 913
Total pages 8
Editor Mitchell D. Feldman
Richard L. Kravitz
Place of publication New York, U.S.A.
Publisher Springer
Collection year 2011
Language eng
Formatted abstract
BACKGROUND: N-of-1 trials test treatment effectiveness within an individual patient. OBJECTIVE: To assess (i) the impact of three different N-of-1 trials on both clinical and economic outcomes over 12 months and (ii) whether the use of N-of-1 trials to target patients' access to high-cost drugs might be cost-effective in Australia. DESIGN: Descriptive study of management change, persistence, and costs summarizing three N-of-1 trials.
PARTICIPANTS: Volunteer patients with osteoarthritis, chronic neuropathic pain or ADHD whose optimal choice of treatment was uncertain.
INTERVENTIONS: Double-blind cyclical alternative medications for the three conditions.
MEASURES: Detailed resource use, treatment and health outcomes (response) data collected by postal and telephone surveys immediately before and after the trial and at 3, 6 and 12 months. Estimated costs to the Australian healthcare system for the pre-trial vs. 12 months post-trial.
RESULTS: Participants persisting with the joint patient-doctor decision 12 months after trial completion were 32% for osteoarthritis, 45% for chronic neuropathic pain and 70% for the ADHD trials. Cost-offsets were obtained from reduced usage of non-optimal drugs, and reduced medical consultations. Drug costs increased for the chronic neuropathic pain and ADHD trials due to many patients being on either low-cost or no pharmaceuticals before the trial.
CONCLUSIONS: N-of-1 trials are an effective method to identify optimal treatment in patients in whom disease management is uncertain. Using this evidence-based approach, patients and doctors tend to persist with optimal treatment resulting in cost-savings. N-of-1 trials are clinically acceptable and may be an effective way of rationally prescribing some expensive long-term medicines.
© 2010 Society of General Internal Medicine.
Keyword N-of-1 trials
Follow-up study
Rational prescribing
Attention-deficit /hyperactivity disorder
Clinical practice
Randomized trials
Adherence to diabetes guidelines
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

Document type: Journal Article
Sub-type: Article (original research)
Collections: Official 2011 Collection
School of Medicine Publications
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Citation counts: TR Web of Science Citation Count  Cited 18 times in Thomson Reuters Web of Science Article | Citations
Scopus Citation Count Cited 22 times in Scopus Article | Citations
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Created: Sun, 29 Aug 2010, 00:03:19 EST