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Randomized Controlled Trial
. 2010 Sep;25(9):906-13.
doi: 10.1007/s11606-010-1352-7. Epub 2010 Apr 13.

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

Paul A Scuffham  1 , Jane Nikles, Geoffrey K Mitchell, Michael J Yelland, Norma Vine, Christopher J Poulos, Peter I Pillans, Guy Bashford, Chris del Mar, Philip J Schluter, Paul Glasziou
Affiliations
Randomized Controlled Trial

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

Paul A Scuffham et al. J Gen Intern Med. 2010 Sep.

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.

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Figures

Figure 1
Figure 1
Typical N-of-1 trial. The order of treatment and placebo are randomly assigned for each cycle.
Figure 2
Figure 2
Scenarios evaluated for use of higher cost pharmaceuticals.
See this image and copyright information in PMC

Comment in

  • N-of-1 trials: a new future?
    Larson EB. Larson EB. J Gen Intern Med. 2010 Sep;25(9):891-2. doi: 10.1007/s11606-010-1440-8. J Gen Intern Med. 2010. PMID: 20632123 Free PMC article. No abstract available.
  • N-of-1 trials: not just for academics.
    Federman DG, Shelling ML, Kirsner RS. Federman DG, et al. J Gen Intern Med. 2011 Feb;26(2):115. doi: 10.1007/s11606-010-1554-z. J Gen Intern Med. 2011. PMID: 21042877 Free PMC article. No abstract available.

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