Skip to main content
Erschienen in: Journal of General Internal Medicine 9/2010

01.09.2010 | Original Research

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

verfasst von: Paul A. Scuffham, PhD, Jane Nikles, PhD, Geoffrey K. Mitchell, PhD, Michael J. Yelland, PhD, Norma Vine, BA, Hons, Christopher J. Poulos, FAFRM, Peter I. Pillans, MD, Guy Bashford, FAFRM, Chris del Mar, MD, Philip J. Schluter, PhD, Paul Glasziou, PhD

Erschienen in: Journal of General Internal Medicine | Ausgabe 9/2010

Einloggen, um Zugang zu erhalten

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.
Literatur
2.
Zurück zum Zitat Chodick G, Heymann A, Wood F, Kokia E. The direct medical costs of diabetes in Isreal. Eur J Health Econ. 2005;6(2):166–171.CrossRefPubMed Chodick G, Heymann A, Wood F, Kokia E. The direct medical costs of diabetes in Isreal. Eur J Health Econ. 2005;6(2):166–171.CrossRefPubMed
3.
Zurück zum Zitat Guyatt G, Keller L, Jaeschke R, Rosenbloom D, Adachi J, Newhouse M. The N-of-1 randomized controlled trial: clinical usefulness. Our three-year experience. Ann Intern Med. 1990;112(4):293–299.PubMed Guyatt G, Keller L, Jaeschke R, Rosenbloom D, Adachi J, Newhouse M. The N-of-1 randomized controlled trial: clinical usefulness. Our three-year experience. Ann Intern Med. 1990;112(4):293–299.PubMed
4.
Zurück zum Zitat Guyatt G, Sackett D, Taylor D. Determining optimal therapy. Randomized trials in individual patients. N Engl J Med. 1986;314(14):889–892.CrossRefPubMed Guyatt G, Sackett D, Taylor D. Determining optimal therapy. Randomized trials in individual patients. N Engl J Med. 1986;314(14):889–892.CrossRefPubMed
5.
Zurück zum Zitat Kravitz R, Duan N, Niedzinski E, Hay M, Subramanian S, Weisner T. What ever happened to N-of-1 trials? Insiders’ perspectives and a look to the future. Milbank Q. 2008;86(4):533–555.CrossRefPubMed Kravitz R, Duan N, Niedzinski E, Hay M, Subramanian S, Weisner T. What ever happened to N-of-1 trials? Insiders’ perspectives and a look to the future. Milbank Q. 2008;86(4):533–555.CrossRefPubMed
6.
Zurück zum Zitat Zucker D, Schmid C, McIntosh M, D’Agostino R, Selker H, Lau J. Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment. J Clin Epidemiol. 1997;50(4):401–410.CrossRefPubMed Zucker D, Schmid C, McIntosh M, D’Agostino R, Selker H, Lau J. Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment. J Clin Epidemiol. 1997;50(4):401–410.CrossRefPubMed
7.
Zurück zum Zitat Scuffham P, Yelland M, Nikles C, Pietrzak E, Wilkinson D. Are N-of-1 trials an economically viable option to improve access to selected high cost medications? The Australian experience. Value Health. 2008;11(1):97–109.PubMed Scuffham P, Yelland M, Nikles C, Pietrzak E, Wilkinson D. Are N-of-1 trials an economically viable option to improve access to selected high cost medications? The Australian experience. Value Health. 2008;11(1):97–109.PubMed
8.
Zurück zum Zitat Nikles C, Mitchell G, del Mar C, McNairn N, Clavarino Z. Long-term changes in management following n-of-1 trials of stimulants in attention-deficit/hyperactivity disorder. Eur J Clin Pharmacol. 2007;63(11):985–989.CrossRefPubMed Nikles C, Mitchell G, del Mar C, McNairn N, Clavarino Z. Long-term changes in management following n-of-1 trials of stimulants in attention-deficit/hyperactivity disorder. Eur J Clin Pharmacol. 2007;63(11):985–989.CrossRefPubMed
9.
Zurück zum Zitat Yelland M, Poulos C, Pillans P, et al. N-of-1 randomized trials to assess the efficacy of gabapentin for chronic neuropathic pain. Pain Med. 2009;10(4):754–761.CrossRefPubMed Yelland M, Poulos C, Pillans P, et al. N-of-1 randomized trials to assess the efficacy of gabapentin for chronic neuropathic pain. Pain Med. 2009;10(4):754–761.CrossRefPubMed
10.
Zurück zum Zitat Yelland M, Nikles C, McNairn N, del Mar C, Schluter P, Brown R. Celecoxib compared with sustained-release paracetamol for osteoarthritis: a series of n-of-1 trials. Rheumatology. 2007;46:135–140.CrossRefPubMed Yelland M, Nikles C, McNairn N, del Mar C, Schluter P, Brown R. Celecoxib compared with sustained-release paracetamol for osteoarthritis: a series of n-of-1 trials. Rheumatology. 2007;46:135–140.CrossRefPubMed
11.
Zurück zum Zitat Cramer J, Rosenheck R, Kirk G, Krol W, Krystal J, VA Naltrexone Study Group 425. Medication compliance feedback and monitoring in a clinical trial: predictors and outcomes. Value Health. 2003;6:566–573.CrossRefPubMed Cramer J, Rosenheck R, Kirk G, Krol W, Krystal J, VA Naltrexone Study Group 425. Medication compliance feedback and monitoring in a clinical trial: predictors and outcomes. Value Health. 2003;6:566–573.CrossRefPubMed
13.
Zurück zum Zitat Department of Health and Ageing. Schedule of Pharmaceutical Benefits for Approved Paharmacists and Medical Practitioners. Canberra: Commonwealth of Australia; 2006. Department of Health and Ageing. Schedule of Pharmaceutical Benefits for Approved Paharmacists and Medical Practitioners. Canberra: Commonwealth of Australia; 2006.
14.
Zurück zum Zitat Efron B, Tibshirani R. The Bootstrap Method for standard errors, confidence intervals, and other measures of statistical accuracy. Stat Sci. 1986;1(1):1–35. Efron B, Tibshirani R. The Bootstrap Method for standard errors, confidence intervals, and other measures of statistical accuracy. Stat Sci. 1986;1(1):1–35.
15.
Zurück zum Zitat Efron B, Tibshirani R. An Introduction to the Bootstrap. New York: Chapman Hall; 1993. Efron B, Tibshirani R. An Introduction to the Bootstrap. New York: Chapman Hall; 1993.
16.
Zurück zum Zitat Nikles C, Yelland M, Glasziou P, del Mar C. Do individualized medication effectiveness tests (N-of-1 trials) change joint patient-doctor decisions about which drug to use for osteoarthritis and chronic pain? Am J Ther. 2005;12:92–97.CrossRefPubMed Nikles C, Yelland M, Glasziou P, del Mar C. Do individualized medication effectiveness tests (N-of-1 trials) change joint patient-doctor decisions about which drug to use for osteoarthritis and chronic pain? Am J Ther. 2005;12:92–97.CrossRefPubMed
17.
Zurück zum Zitat Nikles C, Clavarino Z, del Mar C. Using N-of-1 trials as a clinical tool to improve prescribing. Br J Gen Pract. 2005;55(512):175–180.PubMed Nikles C, Clavarino Z, del Mar C. Using N-of-1 trials as a clinical tool to improve prescribing. Br J Gen Pract. 2005;55(512):175–180.PubMed
18.
Zurück zum Zitat Huser M, Evans T, Berger V. Medication adherence trends with statins. Adv Ther. 2005;22:163–171.CrossRefPubMed Huser M, Evans T, Berger V. Medication adherence trends with statins. Adv Ther. 2005;22:163–171.CrossRefPubMed
19.
20.
Zurück zum Zitat Tsapas A, Matthews D. Using N-of-1 trials in evidence-based clinical practice. JAMA. 2009;301(10):1022–1023.CrossRefPubMed Tsapas A, Matthews D. Using N-of-1 trials in evidence-based clinical practice. JAMA. 2009;301(10):1022–1023.CrossRefPubMed
21.
Zurück zum Zitat Suri R. The use of human deoxyribonuclease (rhDNase) in the management of cystic fibrosis. BioDrugs. 2005;19(3):135–144.CrossRefPubMed Suri R. The use of human deoxyribonuclease (rhDNase) in the management of cystic fibrosis. BioDrugs. 2005;19(3):135–144.CrossRefPubMed
22.
Zurück zum Zitat Pocock S. Current issues in the design and interpretation of clinical trials. Br Med J. 1985;290:39–42.CrossRef Pocock S. Current issues in the design and interpretation of clinical trials. Br Med J. 1985;290:39–42.CrossRef
23.
Zurück zum Zitat Sheather-Reid R, Cohen M. Efficacy of analgesics in chronic pain: A series of N-of-one studies. J Pain Symptom Manage. 1998;15:244–252.CrossRefPubMed Sheather-Reid R, Cohen M. Efficacy of analgesics in chronic pain: A series of N-of-one studies. J Pain Symptom Manage. 1998;15:244–252.CrossRefPubMed
24.
Zurück zum Zitat Guyatt G. Clinical care and clinical research: A false dichotomy. In: Daly J, ed. Ethical Intersections: Health Research, Methods and Research Responsibility. Sydney: Allen & Unwin; 1996:66–73. Guyatt G. Clinical care and clinical research: A false dichotomy. In: Daly J, ed. Ethical Intersections: Health Research, Methods and Research Responsibility. Sydney: Allen & Unwin; 1996:66–73.
25.
Zurück zum Zitat Montori V, Guyatt G. Using N-of-1 trials in evidence-based clinical practice-Reply. JAMA. 2009;301(10):1023.CrossRef Montori V, Guyatt G. Using N-of-1 trials in evidence-based clinical practice-Reply. JAMA. 2009;301(10):1023.CrossRef
26.
Zurück zum Zitat Tsapas A, Matthews D. N of 1 trials in diabetes: Making individual therapeutic decisions. Diabetologia. 2008;51(6):921–923.CrossRefPubMed Tsapas A, Matthews D. N of 1 trials in diabetes: Making individual therapeutic decisions. Diabetologia. 2008;51(6):921–923.CrossRefPubMed
27.
Zurück zum Zitat Nikles C, Mitchell G, del Mar C, Clavarino Z, McNairn N. An N-of-1 trial service in clinical practice: Testing the effectiveness of stimulants for attention deficit hyperactivity disorder. Pediatrics. 2006;117(6):2040–2046.CrossRefPubMed Nikles C, Mitchell G, del Mar C, Clavarino Z, McNairn N. An N-of-1 trial service in clinical practice: Testing the effectiveness of stimulants for attention deficit hyperactivity disorder. Pediatrics. 2006;117(6):2040–2046.CrossRefPubMed
Metadaten
Titel
Using N-of-1 Trials to Improve Patient Management and Save Costs
verfasst von
Paul A. Scuffham, PhD
Jane Nikles, PhD
Geoffrey K. Mitchell, PhD
Michael J. Yelland, PhD
Norma Vine, BA, Hons
Christopher J. Poulos, FAFRM
Peter I. Pillans, MD
Guy Bashford, FAFRM
Chris del Mar, MD
Philip J. Schluter, PhD
Paul Glasziou, PhD
Publikationsdatum
01.09.2010
Verlag
Springer-Verlag
Erschienen in
Journal of General Internal Medicine / Ausgabe 9/2010
Print ISSN: 0884-8734
Elektronische ISSN: 1525-1497
DOI
https://doi.org/10.1007/s11606-010-1352-7

Weitere Artikel der Ausgabe 9/2010

Journal of General Internal Medicine 9/2010 Zur Ausgabe

Leitlinien kompakt für die Innere Medizin

Mit medbee Pocketcards sicher entscheiden.

Seit 2022 gehört die medbee GmbH zum Springer Medizin Verlag

Update Innere Medizin

Bestellen Sie unseren Fach-Newsletter und bleiben Sie gut informiert.