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Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation

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  • Monika Wagner
  • Hanane Khoury
  • Jacob Willet
  • Donna Rindress
  • Mireille Goetghebeur

Abstract

The adapted framework measures value in its widest sense, while being responsive to rare disease issues and policies. It provides an operationalizable platform to integrate values, competing ethical dilemmas, and uncertainty in appraising healthcare interventions. Copyright The Author(s) 2016

Suggested Citation

  • 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.
    • Handle: RePEc:spr:pharme:v:34:y:2016:i:3:p:285-301
    DOI: 10.1007/s40273-015-0340-5
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    References listed on IDEAS

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    1. Javier Orofino & Javier Soto & Miguel Casado & Itziar Oyagüez, 2010. "Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007," Applied Health Economics and Health Policy, Springer, vol. 8(5), pages 301-315, September.
    2. 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.
    3. Warren G. Linley & Dyfrig A. Hughes, 2013. "Societal Views On Nice, Cancer Drugs Fund And Value‐Based Pricing Criteria For Prioritising Medicines: A Cross‐Sectional Survey Of 4118 Adults In Great Britain," Health Economics, John Wiley & Sons, Ltd., vol. 22(8), pages 948-964, August.
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    Cited by:

    1. Mónica D. Oliveira & Inês Mataloto & Panos Kanavos, 2019. "Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 20(6), pages 891-918, August.
    2. Belousova, Olga A. & Groen, Aard J. & Ouendag, Aniek M., 2020. "Opportunities and barriers for innovation and entrepreneurship in orphan drug development," Technological Forecasting and Social Change, Elsevier, vol. 161(C).
    3. 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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