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


  • Mentzakis, Emmanouil
  • Stefanowska, Patricia
  • Hurley, Jeremiah


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.

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  • Mentzakis, Emmanouil & Stefanowska, Patricia & Hurley, Jeremiah, 2011. "A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study," Health Economics, Policy and Law, Cambridge University Press, vol. 6(03), pages 405-433, June.
  • Handle: RePEc:cup:hecopl:v:6:y:2011:i:03:p:405-433_00

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    Cited by:

    1. Christopher McCabe & Mike Paulden & James O'Mahony & Richard Edlin & Anthony Culyer, 2014. "Life at a premium: considering an end-of-life premium in Value Based Reimbursement," Working Papers 1407, Academic Unit of Health Economics, Leeds Institute of Health Sciences, University of Leeds.
    2. Jennifer Whitty & Emily Lancsar & Kylie Rixon & Xanthe Golenko & Julie Ratcliffe, 2014. "A Systematic Review of Stated Preference Studies Reporting Public Preferences for Healthcare Priority Setting," The Patient: Patient-Centered Outcomes Research, Springer;Johns Hopkins Bloomberg School of Public Health, vol. 7(4), pages 365-386, December.
    3. Desser, Arna S., 2013. "Prioritizing treatment of rare diseases: A survey of preferences of Norwegian doctors," Social Science & Medicine, Elsevier, vol. 94(C), pages 56-62.
    4. Moser, Riccarda & Raffaelli, Roberta, 2014. "Does attribute cut-off elicitation affect choice consistency? Contrasting hypothetical and real-money choice experiments," Journal of choice modelling, Elsevier, vol. 11(C), pages 16-29.
    5. Monika Wagner & Hanane Khoury & Jacob Willet & Donna Rindress & Mireille Goetghebeur, 2016. "Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation," PharmacoEconomics, Springer, vol. 34(3), pages 285-301, March.
    6. LUYTEN, Jeroen & KESSELS, Roselinde & GOOS, Peter & BEUTELS, Philippe, 2013. "Public preferences for prioritizing preventive and curative health care interventions: A discrete choice experiment," Working Papers 2013032, University of Antwerp, Faculty of Applied Economics.
    7. repec:spr:sochwe:v:49:y:2017:i:1:d:10.1007_s00355-017-1053-9 is not listed on IDEAS
    8. repec:spr:pharmo:v:1:y:2017:i:1:d:10.1007_s41669-016-0002-3 is not listed on IDEAS
    9. Mortimer, Duncan & Peacock, Stuart, 2012. "Social welfare and the Affordable Care Act: Is it ever optimal to set aside comparative cost?," Social Science & Medicine, Elsevier, vol. 75(7), pages 1156-1162.
    10. Mike Paulden & Tania Stafinski & Devidas Menon & Christopher McCabe, 2015. "Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework," PharmacoEconomics, Springer, vol. 33(3), pages 255-269, March.
    11. Michael Clark & Domino Determann & Stavros Petrou & Domenico Moro & Esther Bekker-Grob, 2014. "Discrete Choice Experiments in Health Economics: A Review of the Literature," PharmacoEconomics, Springer, vol. 32(9), pages 883-902, September.
    12. repec:eee:hepoli:v:121:y:2017:i:7:p:731-744 is not listed on IDEAS
    13. repec:spr:pharme:v:36:y:2018:i:2:d:10.1007_s40273-017-0575-4 is not listed on IDEAS

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