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Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

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  • Mike Paulden
  • Tania Stafinski
  • Devidas Menon
  • Christopher McCabe

Abstract

Our framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision. Copyright The Author(s) 2015

Suggested Citation

  • 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.
    • Handle: RePEc:spr:pharme:v:33:y:2015:i:3:p:255-269
    DOI: 10.1007/s40273-014-0235-x
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    References listed on IDEAS

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    1. 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(3), pages 405-433, July.
    2. McCabe, C & Claxton, K & Culyer, AJ, 2008. "The NICE Cost-Effectiveness Threshold: What it is and What that Means," MPRA Paper 26466, University Library of Munich, Germany.
    3. Karl Claxton & Steve Martin & Marta Soares & Nigel Rice & Eldon Spackman & Sebastian Hinde & Nancy Devlin & Peter C Smith & Mark Sculpher, 2013. "Methods for the estimation of the NICE cost effectiveness threshold," Working Papers 081cherp, Centre for Health Economics, University of York.
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    Citations

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    Cited by:

    1. Degtiar, Irina, 2017. "A review of international coverage and pricing strategies for personalized medicine and orphan drugs," Health Policy, Elsevier, vol. 121(12), pages 1240-1248.
    2. Jeff Round & Mike Paulden, 2018. "Incorporating equity in economic evaluations: a multi-attribute equity state approach," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 19(4), pages 489-498, May.
    3. Marialuisa Saviano & Sergio Barile & Francesco Caputo & Mattia Lettieri & Stefania Zanda, 2019. "From Rare to Neglected Diseases: A Sustainable and Inclusive Healthcare Perspective for Reframing the Orphan Drugs Issue," Sustainability, MDPI, vol. 11(5), pages 1-21, March.
    4. Jason C Hsu & Huai-Chueh Wu & Wen-Chia Feng & Chih-Ho Chou & Edward Chia-Cheng Lai & Christine Y Lu, 2018. "Disease and economic burden for rare diseases in Taiwan: A longitudinal study using Taiwan’s National Health Insurance Research Database," PLOS ONE, Public Library of Science, vol. 13(9), pages 1-14, September.
    5. López-Bastida, J. & Ramos-Goñi, J.M. & Aranda-Reneo, I. & Trapero-Bertran, M. & Kanavos, P. & Rodriguez Martin, B., 2019. "Using a stated preference discrete choice experiment to assess societal value from the perspective of decision-makers in Europe. Does it work for rare diseases?," Health Policy, Elsevier, vol. 123(2), pages 152-158.
    6. Kleinhout-Vliek, Tineke & de Bont, Antoinette & Boer, Bert, 2017. "The bare necessities? A realist review of necessity argumentations used in health care coverage decisions," Health Policy, Elsevier, vol. 121(7), pages 731-744.
    7. Elena Nicod, 2017. "Why do health technology assessment coverage recommendations for the same drugs differ across settings? Applying a mixed methods framework to systematically compare orphan drug decisions in four Europ," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 18(6), pages 715-730, July.
    8. Nicod, Elena & Annemans, Lieven & Bucsics, Anna & Lee, Anne & Upadhyaya, Sheela & Facey, Karen, 2019. "HTA programme response to the challenges of dealing with orphan medicinal products: Process evaluation in selected European countries," Health Policy, Elsevier, vol. 123(2), pages 140-151.

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