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

Research output: Contribution to journalArticleResearchpeer-review

47 Citations (Scopus)
26 Downloads (Pure)

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.

Original languageEnglish
Pages (from-to)906-913
Number of pages8
JournalJournal of General Internal Medicine
Volume25
Issue number9
DOIs
Publication statusPublished - Sep 2010

Fingerprint

Costs and Cost Analysis
Neuralgia
Chronic Pain
Drug Costs
Osteoarthritis
Cost Savings
Disease Management
Telephone
Pharmaceutical Preparations
Volunteers
Therapeutics
Referral and Consultation
Joints
Economics
Delivery of Health Care
Health

Cite this

Scuffham, P. A., Nikles, J., Mitchell, G. K., Yelland, M. J., Vine, N., Poulos, C. 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. https://doi.org/10.1007/s11606-010-1352-7
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. / Using N-of-1 trials to improve patient management and save costs. In: Journal of General Internal Medicine. 2010 ; Vol. 25, No. 9. pp. 906-913.
@article{e404a1c6064f4ac396a49052ad405486,
title = "Using N-of-1 trials to improve patient management and save costs",
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.",
author = "Scuffham, {Paul A.} and Jane Nikles and Mitchell, {Geoffrey K.} and Yelland, {Michael J.} and Norma Vine and Poulos, {Christopher J.} and Pillans, {Peter I.} and Guy Bashford and {Del Mar}, Chris and Schluter, {Philip J.} and Paul Glasziou",
year = "2010",
month = "9",
doi = "10.1007/s11606-010-1352-7",
language = "English",
volume = "25",
pages = "906--913",
journal = "Journal of General Internal Medicine",
issn = "0884-8734",
publisher = "Springer",
number = "9",

}

Scuffham, PA, Nikles, J, Mitchell, GK, Yelland, MJ, Vine, N, Poulos, CJ, Pillans, PI, Bashford, G, Del Mar, C, Schluter, PJ & Glasziou, P 2010, 'Using N-of-1 trials to improve patient management and save costs' Journal of General Internal Medicine, vol. 25, no. 9, pp. 906-913. https://doi.org/10.1007/s11606-010-1352-7

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.; Glasziou, Paul.

In: Journal of General Internal Medicine, Vol. 25, No. 9, 09.2010, p. 906-913.

Research output: Contribution to journalArticleResearchpeer-review

TY - JOUR

T1 - Using N-of-1 trials to improve patient management and save costs

AU - Scuffham, Paul A.

AU - Nikles, Jane

AU - Mitchell, Geoffrey K.

AU - Yelland, Michael J.

AU - Vine, Norma

AU - Poulos, Christopher J.

AU - Pillans, Peter I.

AU - Bashford, Guy

AU - Del Mar, Chris

AU - Schluter, Philip J.

AU - Glasziou, Paul

PY - 2010/9

Y1 - 2010/9

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=77956418835&partnerID=8YFLogxK

U2 - 10.1007/s11606-010-1352-7

DO - 10.1007/s11606-010-1352-7

M3 - Article

VL - 25

SP - 906

EP - 913

JO - Journal of General Internal Medicine

JF - Journal of General Internal Medicine

SN - 0884-8734

IS - 9

ER -

Scuffham PA, Nikles J, Mitchell GK, Yelland MJ, Vine N, Poulos CJ et al. Using N-of-1 trials to improve patient management and save costs. Journal of General Internal Medicine. 2010 Sep;25(9):906-913. https://doi.org/10.1007/s11606-010-1352-7