IDEAS home Printed from https://ideas.repec.org/a/spr/pharme/v34y2016i3d10.1007_s40273-015-0340-5.html
   My bibliography  Save this article

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

Author

Listed:
  • Monika Wagner

    (LASER Analytica)

  • Hanane Khoury

    (LASER Analytica)

  • Jacob Willet

    (LASER Analytica)

  • Donna Rindress

    (LASER Analytica)

  • Mireille Goetghebeur

    (LASER Analytica
    University of Montreal, School of Public Health)

Abstract

Background The multiplicity of issues, including uncertainty and ethical dilemmas, and policies involved in appraising interventions for rare diseases suggests that multicriteria decision analysis (MCDA) based on a holistic definition of value is uniquely suited for this purpose. The objective of this study was to analyze and further develop a comprehensive MCDA framework (EVIDEM) to address rare disease issues and policies, while maintaining its applicability across disease areas. Methods Specific issues and policies for rare diseases were identified through literature review. Ethical and methodological foundations of the EVIDEM framework v3.0 were systematically analyzed from the perspective of these issues, and policies and modifications of the framework were performed accordingly to ensure their integration. Results Analysis showed that the framework integrates ethical dilemmas and issues inherent to appraising interventions for rare diseases but required further integration of specific aspects. Modification thus included the addition of subcriteria to further differentiate disease severity, disease-specific treatment outcomes, and economic consequences of interventions for rare diseases. Scoring scales were further developed to include negative scales for all comparative criteria. A methodology was established to incorporate context-specific population priorities and policies, such as those for rare diseases, into the quantitative part of the framework. This design allows making more explicit trade-offs between competing ethical positions of fairness (prioritization of those who are worst off), the goal of benefiting as many people as possible, the imperative to help, and wise use of knowledge and resources. It also allows addressing variability in institutional policies regarding prioritization of specific disease areas, in addition to existing uncertainty analysis available from EVIDEM. Conclusion 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.

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:d:10.1007_s40273-015-0340-5
    DOI: 10.1007/s40273-015-0340-5
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s40273-015-0340-5
    File Function: Abstract
    Download Restriction: Access to the full text of the articles in this series is restricted.
    File URL: https://libkey.io/10.1007/s40273-015-0340-5?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    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.
    Full references (including those not matched with items on IDEAS)

    Citations

    Citations are extracted by the CitEc Project, subscribe to its RSS feed for this item.
    as


    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.

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. 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.
    2. 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.
    3. Eline Picavet & Marc Dooms & David Cassiman & Steven Simoens, 2011. "Drugs for rare diseases: Influence of orphan designation status on price," Applied Health Economics and Health Policy, Springer, vol. 9(4), pages 275-279, July.
    4. 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.
    5. Marta Trapero-Bertran & Beatriz Rodríguez-Martín & Julio López-Bastida, 2019. "What attributes should be included in a discrete choice experiment related to health technologies? A systematic literature review," PLOS ONE, Public Library of Science, vol. 14(7), pages 1-15, July.
    6. 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.
    7. Jeremiah Hurley & Emmanouil Mentzakis & Mita Giacomini & Deirdre DeJean & Michel Grignon, 2017. "Non-market resource allocation and the public’s interpretation of need: an empirical investigation in the context of health care," Social Choice and Welfare, Springer;The Society for Social Choice and Welfare, vol. 49(1), pages 117-143, June.
    8. Li, Zili & Washington, Simon P. & Zheng, Zuduo & Prato, Carlo G., 2023. "A Bayesian hierarchical approach to the joint modelling of Revealed and stated choices," Journal of choice modelling, Elsevier, vol. 47(C).
    9. 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.
    10. Oehlmann, Malte & Glenk, Klaus & Lloyd-Smith, Patrick & Meyerhoff, Jürgen, 2021. "Quantifying landscape externalities of renewable energy development: Implications of attribute cut-offs in choice experiments," Resource and Energy Economics, Elsevier, vol. 65(C).
    11. 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.
    12. 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.
    13. Veronika Kalouguina & Joël Wagner, 2020. "Challenges and Solutions for Integrating and Financing Personalized Medicine in Healthcare Systems: A Systematic Literature Review," JRFM, MDPI, vol. 13(11), pages 1-22, November.
    14. 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;International Academy of Health Preference Research, vol. 7(4), pages 365-386, December.
    15. 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.
    16. Tania Stafinski & Jacqueline Street & Andrea Young & Devidas Menon, 2022. "Moving beyond the Court of Public Opinion: A Citizens’ Jury Exploring the Public’s Values around Funding Decisions for Ultra-Orphan Drugs," IJERPH, MDPI, vol. 20(1), pages 1-13, December.
    17. Johanna Wiss & Lars-Ake Levin & David Andersson & Gustav Tinghög, 2017. "Prioritizing Rare Diseases: Psychological Effects Influencing Medical Decision Making," Medical Decision Making, , vol. 37(5), pages 567-576, July.
    18. Mo Zhou & Winter Maxwell Thayer & John F. P. Bridges, 2018. "Using Latent Class Analysis to Model Preference Heterogeneity in Health: A Systematic Review," PharmacoEconomics, Springer, vol. 36(2), pages 175-187, February.
    19. 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 Business and Economics.
    20. Alain Denis & Lut Mergaert & Christel Fostier & Irina Cleemput & Steven Simoens, 2010. "Issues surrounding orphan disease and orphan drug policies in Europe," Applied Health Economics and Health Policy, Springer, vol. 8(5), pages 343-350, September.

    More about this item

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:spr:pharme:v:34:y:2016:i:3:d:10.1007_s40273-015-0340-5. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.springer.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.