Hostname: page-component-78c5997874-mlc7c Total loading time: 0 Render date: 2024-11-05T15:41:44.967Z Has data issue: false hasContentIssue false
  • English
  • Français

A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study

Published online by Cambridge University Press:  21 December 2010

Emmanouil Mentzakis ,
Patricia Stefanowska  and
Jeremiah Hurley
Emmanouil Mentzakis*
Affiliation:
Department of Economics, McMaster University, Hamilton, Ontario, Canada
Patricia Stefanowska
Affiliation:
Centre for Health Economics and Policy Analysis, McMaster University, Hamilton, Ontario, Canada
Jeremiah Hurley
Affiliation:
Department of Economics, McMaster University, Hamilton, Ontario, Canada and Centre for Health Economics and Policy Analysis, McMaster University, Hamilton, Ontario, Canada
*
Correspondence to: Emmanouil Mentzakis, Department of Economics, McMaster University, 1280 Main Street West, Hamilton, Ontario, L8S 4M4, Canada. Email: [email protected]
Get access Rights & Permissions [Opens in a new window]

Abstract

Policy debate about funding criteria for drugs used to treat rare, orphan diseases is gaining prominence. This study presents evidence from a discrete choice experiment using a convenience sample of university students to investigate individual preferences regarding public funding for drugs used to treat rare diseases and common diseases. This pilot study finds that: other things equal, the respondents do not prefer to have the government spend more for drugs used to treat rare diseases; that respondents are not willing to pay more per life year gained for a rare disease than a common disease; and that respondents weigh relevant attributes of the coverage decisions (e.g. costs, disease severity and treatment effectiveness) similarly for both rare and common diseases. The results confirm the importance of severity and treatment effectiveness in preferences for public funding. Although this is the first study of its kind, the results send a cautionary message regarding the special treatment of orphan drugs in coverage decision-making.

Type
Articles
Information
Health Economics, Policy and Law , Volume 6 , Issue 3 , 16 June 2011 , pp. 405 - 433
Copyright
Copyright © Cambridge University Press 2010

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

Bateman, I., Carson, R. T., Day, B., Hanemann, W. M., Hanley, N., Hett, T., Jones, A., Loomes, G., Mourato, S., Ozdemiroglu, E., Pearce, D. W., Sugden, R., Swanson, J. (2002), Economic Valuation with Stated Preferences Techniques: A Manual, Cheltenham, UK: Edward Elgar.Google Scholar
Bennett, J.Adamowicz, W. (2001), ‘Some Fundamentals of Environmental Choice Modelling’, in J. Bennett and R. Blamey (eds), The Choice Modelling Approach to Environmental Valuation, Northampton: Edward Elgar, 37–69.Google Scholar
Canadian Organization for Rare Disorders (2008), http://www.cord.ca/ [5 September 2008].Google Scholar
Cheung, R. Y., Cohen, J. C.Illingworth, P. (2004), ‘Orphan drug policies: implications for the United States, Canada, and developing countries’, Health Law Journal, 12: 183–200.Google ScholarPubMed
Cookson, R.Dolan, P. (1999), ‘Public views on health care rationing: a group discussion study’, Health Policy, 49(1–2): 63–74.Google Scholar
Danielis, R.Marcucci, E. (2007), ‘Attribute cut-offs in freight service selection’, Transportation Research Part E: Logistics and Transportation Review, 43(5): 506–515.Google Scholar
Dawes, R. M. (1964), ‘Social selection based on multidimensional criteria’, Journal of Abnormal Psychology, 68: 104–109.Google Scholar
Dear, J. W., Lilitkarntakul, P.Webb, D. J. (2006), ‘Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products’, British Journal of Clinical Pharmacology, 62(3): 264–271.CrossRefGoogle ScholarPubMed
Denis, A., Simoens, S., Fostier, C., Mergaert, L., Cleemput, I. (2009), ‘Policies for Orphan Diseases and Orphan Drugs’, KCE Reports 112C, Belgian Health Care Knowledge Centre.Google Scholar
Depositario, D. P. T., Jr.Nayga, R. M., Wu, X.Laude, T. P. (2009), ‘Should students be used as subjects in experimental auctions?’, Economics Letters, 102(2): 122–124.Google Scholar
DiMasi, J. A., Hansen, R. W., Grabowski, H. G.Lasagna, L. (1991), ‘Cost of innovation in the pharmaceutical industry’, Journal of Health Economics, 10(2): 107–142.Google Scholar
Dolan, P. (1997), ‘Modeling valuations for EuroQol health states’, Medical Care, 35(11): 1095–1108.CrossRefGoogle ScholarPubMed
Dolan, P., Edlin, R., Tsuchiya, A. (2008), ‘The Relative Societal Value of Health Gains to Different Beneficiaries’, Final Report, RM03/JH11, University of Birmingham.Google Scholar
Dolan, P., Gudex, C., Kind, P., Williams, A. (1995), ‘A social tariff for EuroQoL: Results from a UK general population survey’, Discussion paper No. 138, Centre for Health Economics, University of York.Google Scholar
Dolan, P.Olsen, J. A. (2002), Distributing Health Care: Economic and Ethical Issues, New York: Oxford University Press Inc.CrossRefGoogle Scholar
Dolan, P., Shaw, R., Tsuchiya, A.Williams, A. (2005), ‘QALY maximisation and people's preferences: a methodological review of the literature’, Health Economics, 14(2): 197–208.Google Scholar
Donaldson, C., Baker, R., Bateman, I., Jones-Lee, M., Lancsar, E., Loomes, G., Mason, H., Odejar, M., Prades, J. L. P., Robinson, A., Ryan, M., Shackley, P., Smith, R., Sugden, R., Wildman, J. (2008), ‘Weighting and valuing quality adjusted life years: preliminary results from the social value of a QALY project’, Working Paper JH12, University of Birmingham.Google Scholar
Drummond, M. F., Wilson, D. A., Kanavos, P., Ubel, P.Rovira, J. (2007a), ‘Assessing the economic challenges posed by orphan drugs’, International Journal of Technology Assessment in Health Care, 23(01): 36–42.CrossRefGoogle ScholarPubMed
Drummond, M. F., Wilson, D. A., Kanavos, P., Ubel, P.Rovira, J. (2007b), ‘Assessing the economic challenges posed by orphan drugs: a response to McCabe et al.’, International Journal of Technology Assessment in Health Care, 23(03): 401–404.CrossRefGoogle Scholar
Gerard, K., Shanahan, M.Louviere, J. (2003), ‘Using stated preference discrete choice modelling to inform health care decision-making: a pilot study of breast screening participation’, Applied Economics, 35(9): 1073–1085.CrossRefGoogle Scholar
Greene, W. H.Hensher, D. A. (2003), ‘A latent class model for discrete choice analysis: contrasts with mixed logit’, Transportation Research Part B: Methodological, 37(8): 681–698.Google Scholar
Hannan, E. J.Quinn, B. G. (1979), ‘The determination of the order of an autoregression’, Journal of the Royal Statistical Society. Series B (Methodological), 41(2): 190–195.Google Scholar
Hensher, D. A., Rose, J. M.Greene, W. H. (2005), Applied Choice Analysis: A Primer, Cambridge, UK: Cambridge University Press.CrossRefGoogle Scholar
Hole, A. R. (2008), ‘Modelling heterogeneity in patients’ preferences for the attributes of a general practitioner appointment’, Journal of Health Economics, 27(4): 1078–1094.Google Scholar
Huber, J.Klein, N. M. (1991), ‘Adapting cutoffs to the choice environment: the effects of attribute correlation and reliability’, The Journal of Consumer Research, 18(3): 346–357.Google Scholar
Hughes, D. A., Tunnage, B.Yeo, S. T. (2005), ‘Drugs for exceptionally rare diseases: do they deserve special status for funding? [see comment]’, The Quarterly Journal of Medicine, 98(11): 829–836.Google Scholar
King, M. T., Hall, J., Lancsar, E., Fiebig, D., Hossain, I., Louviere, J., Reddel, H. K.Jenkins, C. R. (2007), ‘Patient preferences for managing asthma: results from a discrete choice experiment’, Health Economics, 16(7): 703–717.Google Scholar
Kuhfeld, W. F. (2005), ‘Marketing Research Methods in SAS’, TS-722, Cary, NC, USA: SAS Institute Inc.Google Scholar
Lancaster, K. (1966), ‘A new approach to consumer theory’, The Journal of Political Economy, 74(2): 132–157.Google Scholar
Lancsar, E.Louviere, J. (2008), ‘Conducting discrete choice experiments to inform healthcare decision making: a user's guide’, Pharmaco Economics, 26(8): 661–677.Google Scholar
Louviere, J. J., Hensher, D. A.Swait, J. D. (2000), Stated Choice Methods: Analysis and Applications, Cambridge, UK: Cambridge University Press.CrossRefGoogle Scholar
Maguire, K. B., Taylor, L. O.Gurmu, S. (2003), ‘Do students behave like adults? Evidence from valuation experiments’, Applied Economics Letters, 10: 753–756.Google Scholar
McCabe, C., Tsuchiya, A., Claxton, K.Raftery, J. (2006), ‘Orphan drugs revisited’, The Quarterly Journal of Medicine, 99(5): 341–345.Google Scholar
McFadden, D. (1974), ‘Conditional Logit Analysis of Qualitative Choice Behavior’, in P. Zarembka (ed.), Frontiers in Econometrics, New York: Academic Press, 105–142.Google Scholar
Medecins Sans Frontieres (2001), ‘Fatal imbalance. the crisis in research and development for drugs for neglected diseases’, Medecins Sans Frontieres Access to Essential Medicines Campaign and the Drugs for Neglected Diseases Working Group, Geneva.Google Scholar
Menon, D.Stafinski, T. (2008), ‘Engaging the public in priority-setting for health technology assessment: findings from a citizens’ jury’, Health Expectations, 11(3): 282–293.Google Scholar
NICE Citizens Council (2004), NICE Citizens Council Report: Ultra Orphan Drugs, London: National Institute for Health and Clinical Excellence.Google Scholar
Nord, E. (1993), ‘The trade-off between severity of illness and treatment effect in cost-value analysis of health care’, Health Policy, 24(3): 227–238.CrossRefGoogle ScholarPubMed
Ryan, M., Gerard, K.Amaya-Amaya, M. (2008), Using Discrete Choice Experiments to Value Health and Health Care, The Netherlands: Springer.CrossRefGoogle Scholar
Schlander, M. (2008), ‘The use of cost-effectiveness by the national institute for health and clinical excellence (NICE): no(t yet an) exemplar of a deliberative process’, Journal of Medical Ethics, 34(7): 534–539.CrossRefGoogle Scholar
Swait, J. (2001), ‘A non-compensatory choice model incorporating attribute cutoffs’, Transportation Research Part B: Methodological, 35(10): 903–928.Google Scholar
Swait, J.Adamowicz, W. (2001), ‘The influence of task complexity on consumer choice: a latent class model of decision strategy switching’, Journal of Consumer Research, 28(1): 135–148.CrossRefGoogle Scholar
Tversky, A. (1972), ‘Elimination-by-aspects: a theory of choice’, Psychology Review, 79: 281–299.Google Scholar
Ubel, P. A., Spranca, M. D., Dekay, M. L., Hershey, J. C.Asch, D. A. (1998), ‘Public preferences for prevention versus cure: what if an ounce of prevention is worth only an ounce of cure?’, Medical Decision Making, 18(2): 141–148.CrossRefGoogle ScholarPubMed
Wastfelt, M., Fadeel, B.Henter, J. I. (2006), ‘A journey of hope: lessons learned from studies on rare diseases and orphan drugs’, Journal of Internal Medicine, 260(1): 1–10.CrossRefGoogle ScholarPubMed
Zwerina, K., Huber, J., Kuhfeld, W. F. (1996), ‘A general method for constructing efficient choice designs’, SAS Technical Papers: TS-722E.Google Scholar