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Relative effectiveness assessment of listed drugs (REAL): A new method for an early comparison of the effectiveness of approved health technologies

Published online by Cambridge University Press:  08 January 2010


Bruno Falissard
Affiliation:
South Paris University
Valérie Izard
Affiliation:
Haute autorité de santé
Bertrand Xerri
Affiliation:
Haute autorité de santé
Gilles Bouvenot
Affiliation:
Hôpital Sainte-Marguerite and Haute autorité de santé
François Meyer
Affiliation:
Haute autorité de santé
Laurent Degos
Affiliation:
Hôpital Saint Louis and Haute autorité de santé

Abstract

Objectives: Post-listing assessment of pharmaceuticals depends on national habits. In England, the assessment is based on estimates of cost per quality-adjusted life-year. These are made some considerable time after listing (negative list). In France, effectiveness, and then efficiency, is assessed immediately after listing (positive list). We propose a new formal method—the REAL method—that can help make early comparisons of the effectiveness of medical treatments.

Methods: Relative efficacies are first obtained from randomized controlled trials (RCTs). Members of the Transparency Committee (French National Authority for Health) are then consulted by questionnaire on the transposability of these results to real life. The RCT results and experts’ ratings are entered into an effect model to obtain estimates of relative effectiveness, using unidimensional scaling, and bootstrap procedures.

Results: Application of the REAL method to the example of a new drug to treat Parkinson's disease and three comparators used in the same indication provided graphs of the distributions of their relative efficacy and relative effectiveness. The new drug was found to provide no added value.

Conclusions: The REAL method is a rational, transparent, and practical procedure for comparing the effectiveness of pharmaceuticals in an immediate post-listing setting.


Type
METHODS
Copyright
Copyright © Cambridge University Press 2010

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References

1. Agresti, A. An introduction to categorical data analysis. New York: John Wiley & Sons; 1996.Google Scholar
2. Boissel, JP, Collet, JP, Lievre, M, Girard, P. An effect model for the assessment of drug benefit: Example of antiarrhythmic drugs in postmyocardial infarction patients. J Cardiovasc Pharmacol. 1993;22:356363.CrossRefGoogle ScholarPubMed
3. Bombardier, C, Maetzel, A. Pharmacoeconomic evaluation of new treatments: Efficacy versus effectiveness studies? Ann Rheum Dis. 1999;58 (Suppl 1):I82I85.CrossRefGoogle ScholarPubMed
4. Brannigan, M. Oregon's experiment. Health Care Anal. 1993;1:1532.CrossRefGoogle ScholarPubMed
5. Danis, M, Patrick, DL, Southerland, LI, Green, ML. Patients’ and families’ preferences for medical intensive care. JAMA. 1988;260:797802.CrossRefGoogle ScholarPubMed
6. Davis, CE. Generalizing from clinical trials. Control Clin Trials. 1994;15:1114.CrossRefGoogle ScholarPubMed
7. De Leeuw, J. Unidimensional scaling. In: Everitt, BS, Howell, D, eds. Encyclopedia of statistics in behavioral science. New York: John Wiley & Sons; 2005.Google Scholar
8. Gelman, A. Prior distributions for variance parameters in hierarchical models (Comment on an Article by Browne and Draper). Bayesian Anal. 2006;1:515534.CrossRefGoogle Scholar
9. Glenny, AM, Altman, DG, Song, F, et al. International Stroke Trial Collaborative Group: Indirect comparisons of competing interventions. Health Technol Assess. 2005;9:1134.CrossRefGoogle Scholar
10. Harris, J. It's not NICE to discriminate. J Med Ethics. 2005;31:373375.CrossRefGoogle ScholarPubMed
11. Hill, S, Garattini, S, van Loenhout, J, O'Brien, BJ, de Joncheere, K. Technology appraisal programme of the National Institute for Clinical Excellence: A review by WHO. World Health Organization; 2003.Google Scholar
12. La Puma, J. Quality-adjusted life years: Ethical implications and the Oregon plan. Issues Law Med. 1992;7:429441.Google ScholarPubMed
13. LeWitt, PA, Lyons, KE, Pahwa, R. Advanced Parkinson disease treated with rotigotine transdermal system: PREFER Study. Neurology. 2007;68:12621267.CrossRefGoogle ScholarPubMed
14. Lu, G, Ades, AE. Combination of direct and indirect evidence in mixed treatment comparisons. Stat Med. 2004;23:31053124.CrossRefGoogle ScholarPubMed
15. Lumley, T. Network meta-analysis for indirect treatment comparisons. Stat Med. 2002;21:23132324.CrossRefGoogle ScholarPubMed
16. National Institute for Clinical Excellence (NICE). Measuring effectiveness and cost effectiveness: The QALY. http://www.nice.org.uk/newsevents/infocus/measuringeffectivenessandcosteffectivenesstheQALY.jsp. Accessed October 17, 2008.Google Scholar
17. Poewe, WH, Rascol, O, Quinn, N, et al. Efficacy of pramipexole and transdermal rotigotine in advanced Parkinson's disease: A double-blind, double-dummy, randomised controlled trial. Lancet Neurol. 2007;6:513520.CrossRefGoogle ScholarPubMed
18. PricewaterhouseCoopers. Pharma 2020: The vision — Which path will you take? 2007; http://www.pwc.com/gx/eng/about/ind/pharma. Accessed October 17, 2008.Google Scholar
19. Rascol, O, Brooks, DJ, Melamed, E, et al. Rasagiline as an adjunct to levodopa in patients with Parkinson's disease and motor fluctuations (LARGO, Lasting effect in Adjunct therapy with Rasagiline Given Once daily, study): A randomised, double-blind, parallel-group trial. Lancet. 2005;365:947954.CrossRefGoogle ScholarPubMed
20. Schlander, M. The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): No(t yet an) exemplar of a deliberative process. J Med Ethics. 2008;34:534539.CrossRefGoogle Scholar
21. Schmid, CH, Lau, J, McIntosh, MW, Cappelleri, JC. An empirical study of the effect of the control rate as a predictor of treatment efficacy in meta-analysis of clinical trials. Stat Med. 1998;17:19231942.3.0.CO;2-6>CrossRefGoogle ScholarPubMed
22. Song, F, Altman, DG, Glenny, AM, Deeks, JJ. Validity of indirect comparison for estimating efficacy of competing interventions: Empirical evidence from published meta-analyses. BMJ. 2003;326:472.CrossRefGoogle ScholarPubMed
23. Thompson, SG, Higgins, JP. Treating individuals 4: Can meta-analysis help target interventions at individuals most likely to benefit? Lancet. 2005;365:341346.CrossRefGoogle Scholar
24. World Bank. Gross domestic product 2004. 2005. http://www.worldbank.org/.Google Scholar

Falissard et al. supplementary material

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