Journal of General Internal Medicine

, Volume 25, Issue 9, pp 906–913 | Cite as

Using N-of-1 Trials to Improve Patient Management and Save Costs

  • Paul A. Scuffham
  • 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
Original Research

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.

KEY WORDS

N-of-1 trials cost-effectiveness follow-up study rational prescribing 

Abbreviations

ADHD

attention-deficit hyperactivity disorder

BCa

bias-corrected and accelerated

GP

general practitioner i.e. family physician

IQR

inter-quartile range

OA

osteoarthritis

PBS

pharmaceutical benefits scheme

RCT

randomized controlled trial

SD

standard deviation

SR

slow-release

95% CI

95% confidence interval

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Copyright information

© Society of General Internal Medicine 2010

Authors and Affiliations

  • Paul A. Scuffham
    • 1
    • 11
  • Jane Nikles
    • 2
  • Geoffrey K. Mitchell
    • 3
  • Michael J. Yelland
    • 1
  • Norma Vine
    • 4
  • Christopher J. Poulos
    • 5
  • Peter I. Pillans
    • 6
  • Guy Bashford
    • 5
  • Chris del Mar
    • 7
  • Philip J. Schluter
    • 8
    • 9
  • Paul Glasziou
    • 10
  1. 1.School of MedicineGriffith UniversityBrisbaneAustralia
  2. 2.School of Medicinethe University of QueenslandBrisbaneAustralia
  3. 3.Discipline of General Practicethe University of QueenslandBrisbaneAustralia
  4. 4.Australian Red Cross Blood ServiceBrisbaneAustralia
  5. 5.Port Kembla HospitalPort KemblaAustralia
  6. 6.Princess Alexandra HospitalBrisbaneAustralia
  7. 7.Faculty of Health Science and MedicineBond UniversityGold CoastAustralia
  8. 8.School of Public Health and Psychosocial StudiesAuckland University of TechnologyAucklandNew Zealand
  9. 9.School of Nursing and MidwiferyThe University of QueenslandBrisbaneAustralia
  10. 10.Department of Primary Health Care and Director, Centre for Evidence-Based PracticeOxford UniversityOxfordUK
  11. 11.Health Economics, School of MedicineGriffith UniversityMeadowbrookAustralia

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