Date of this Version

9-1-2010

Document Type

Journal Article

Publication Details

Accepted Version.

Scuffham, P.A., Nikles, J., Mitchell, G.K., Yelland, M.J., Vine, N., Poulos, C.J., Pillans, P.I., Bashford, G., Del Mar, C., Schluter, P.J. & Glasziou, P. (2010). Using N-of-1 trials to improve patient management and save costs. Journal of General Internal Medicine, 25(9), 906-913.

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2010 HERDC submission. FoR Code: 111717, 110399

© Copyright Society of General Internal Medicine, 2010.

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.

This document has been peer reviewed.