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

  • Emmanouil Mentzakis (a1), Patricia Stefanowska (a2) and Jeremiah Hurley (a3)
  • DOI: http://dx.doi.org/10.1017/S1744133110000344
  • Published online: 21 December 2010
Abstract
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.

Copyright
Corresponding author
Correspondence to: Emmanouil Mentzakis, Department of Economics, McMaster University, 1280 Main Street West, Hamilton, Ontario, L8S 4M4, Canada. Email: mentzak@mcmaster.ca
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This list contains references from the content that can be linked to their source. For a full set of references and notes please see the PDF or HTML where available.

I. Bateman , R. T. Carson , B. Day , W. M. Hanemann , N. Hanley , T. Hett , A. Jones , G. Loomes , S. Mourato , E. Ozdemiroglu , D. W. Pearce , R. Sugden , J. Swanson (2002), Economic Valuation with Stated Preferences Techniques: A Manual, Cheltenham, UK: Edward Elgar.

R. Cookson P. Dolan (1999), ‘Public views on health care rationing: a group discussion study’, Health Policy, 49(1–2): 6374.

R. Danielis E. Marcucci (2007), ‘Attribute cut-offs in freight service selection’, Transportation Research Part E: Logistics and Transportation Review, 43(5): 506515.

R. M. Dawes (1964), ‘Social selection based on multidimensional criteria’, Journal of Abnormal Psychology, 68: 104109.

J. W. Dear , P. Lilitkarntakul D. J. Webb (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): 264271.

D. P. T. Depositario , R. M. Nayga Jr., X. Wu T. P. Laude (2009), ‘Should students be used as subjects in experimental auctions?’, Economics Letters, 102(2): 122124.

J. A. DiMasi , R. W. Hansen , H. G. Grabowski L. Lasagna (1991), ‘Cost of innovation in the pharmaceutical industry’, Journal of Health Economics, 10(2): 107142.

P. Dolan (1997), ‘Modeling valuations for EuroQol health states’, Medical Care, 35(11): 10951108.

P. Dolan , R. Shaw , A. Tsuchiya A. Williams (2005), ‘QALY maximisation and people's preferences: a methodological review of the literature’, Health Economics, 14(2): 197208.

K. Gerard , M. Shanahan J. Louviere (2003), ‘Using stated preference discrete choice modelling to inform health care decision-making: a pilot study of breast screening participation’, Applied Economics, 35(9): 10731085.

W. H. Greene D. A. Hensher (2003), ‘A latent class model for discrete choice analysis: contrasts with mixed logit’, Transportation Research Part B: Methodological, 37(8): 681698.

A. R. Hole (2008), ‘Modelling heterogeneity in patients’ preferences for the attributes of a general practitioner appointment’, Journal of Health Economics, 27(4): 10781094.

J. Huber N. M. Klein (1991), ‘Adapting cutoffs to the choice environment: the effects of attribute correlation and reliability’, The Journal of Consumer Research, 18(3): 346357.

D. A. Hughes , B. Tunnage S. T. Yeo (2005), ‘Drugs for exceptionally rare diseases: do they deserve special status for funding? [see comment]’, The Quarterly Journal of Medicine, 98(11): 829836.

M. T. King , J. Hall , E. Lancsar , D. Fiebig , I. Hossain , J. Louviere , H. K. Reddel C. R. Jenkins (2007), ‘Patient preferences for managing asthma: results from a discrete choice experiment’, Health Economics, 16(7): 703717.

K. Lancaster (1966), ‘A new approach to consumer theory’, The Journal of Political Economy, 74(2): 132157.

E. Lancsar J. Louviere (2008), ‘Conducting discrete choice experiments to inform healthcare decision making: a user's guide’, Pharmaco Economics, 26(8): 661677.

K. B. Maguire , L. O. Taylor S. Gurmu (2003), ‘Do students behave like adults? Evidence from valuation experiments’, Applied Economics Letters, 10: 753756.

C. McCabe , A. Tsuchiya , K. Claxton J. Raftery (2006), ‘Orphan drugs revisited’, The Quarterly Journal of Medicine, 99(5): 341345.

D. Menon T. Stafinski (2008), ‘Engaging the public in priority-setting for health technology assessment: findings from a citizens’ jury’, Health Expectations, 11(3): 282293.

E. Nord (1993), ‘The trade-off between severity of illness and treatment effect in cost-value analysis of health care’, Health Policy, 24(3): 227238.

M. Ryan , K. Gerard M. Amaya-Amaya (2008), Using Discrete Choice Experiments to Value Health and Health Care, The Netherlands: Springer.

M. Schlander (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): 534539.

J. Swait (2001), ‘A non-compensatory choice model incorporating attribute cutoffs’, Transportation Research Part B: Methodological, 35(10): 903928.

J. Swait W. Adamowicz (2001), ‘The influence of task complexity on consumer choice: a latent class model of decision strategy switching’, Journal of Consumer Research, 28(1): 135148.

A. Tversky (1972), ‘Elimination-by-aspects: a theory of choice’, Psychology Review, 79: 281299.

P. A. Ubel , M. D. Spranca , M. L. Dekay , J. C. Hershey D. A. Asch (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): 141148.

M. Wastfelt , B. Fadeel J. I. Henter (2006), ‘A journey of hope: lessons learned from studies on rare diseases and orphan drugs’, Journal of Internal Medicine, 260(1): 110.

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Health Economics, Policy and Law
  • ISSN: 1744-1331
  • EISSN: 1744-134X
  • URL: /core/journals/health-economics-policy-and-law
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