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

J Gen Intern Med. 2010 Sep;25(9):906-13. doi: 10.1007/s11606-010-1352-7. Epub 2010 Apr 13.


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.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Attention Deficit Disorder with Hyperactivity / drug therapy
  • Australia
  • Child
  • Child, Preschool
  • Chronic Disease / drug therapy
  • Chronic Disease / economics
  • Cost-Benefit Analysis
  • Cross-Over Studies
  • Double-Blind Method
  • Evidence-Based Medicine / economics*
  • Female
  • Humans
  • Male
  • Medication Adherence*
  • Middle Aged
  • Neuralgia / drug therapy
  • Osteoarthritis / drug therapy
  • Precision Medicine / economics*
  • Research Design*
  • Treatment Outcome
  • Young Adult