IDEAS home Printed from https://ideas.repec.org/a/eee/hepoli/v100y2011i1p25-34.html
   My bibliography  Save this article

Priority setting for orphan drugs: An international comparison

Author

Listed:
  • Rosenberg-Yunger, Zahava R.S.
  • Daar, Abdallah S.
  • Thorsteinsdóttir, Halla
  • Martin, Douglas K.

Abstract

Objectives To describe the process of priority setting for two orphan drugs - Cerezyme and Fabrazyme - in Canada, Australia and Israel, in order to understand and improve the process based on stakeholder perspectives.Methods We conducted qualitative case studies of how three independent drug advisory committees made decisions relating to the funding of Cerezyme and Fabrazyme. Interviews were conducted with 22 informants, including committee members, patient groups and industry representatives.Results (1) Description: Orphan drugs reimbursement recommendations by expert panels were based on clinical evidence, cost and cost-effectiveness analysis. (2) Evaluation: Committee members expressed an overall preference for the current drug review process used by their own committee, but were concerned with the fairness of the process particularly for orphan drugs. Other informants suggested the inclusion of other relevant values (e.g. lack of alternative treatments) in order to improve the priority setting process. Some patient groups suggested the use of an alternative funding mechanism for orphan drugs.Conclusions Priority setting for drugs is not solely a technical process (involving cost-effective analysis, evidence-based medicine, etc.). Understanding the process by which reimbursement decisions are made for orphan drugs may help improve the system for future orphan drugs.

Suggested Citation

  • Rosenberg-Yunger, Zahava R.S. & Daar, Abdallah S. & Thorsteinsdóttir, Halla & Martin, Douglas K., 2011. "Priority setting for orphan drugs: An international comparison," Health Policy, Elsevier, vol. 100(1), pages 25-34, April.
    • Handle: RePEc:eee:hepoli:v:100:y:2011:i:1:p:25-34
    as

    Download full text from publisher

    File URL: http://www.sciencedirect.com/science/article/pii/S0168-8510(10)00269-1
    Download Restriction: Full text for ScienceDirect subscribers only
    ---><---

    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. Shani, Segev & Siebzehner, Miriam Ines & Luxenburg, Osnat & Shemer, Joshua, 2000. "Setting priorities for the adoption of health technologies on a national level -- the Israeli experience," Health Policy, Elsevier, vol. 54(3), pages 169-185, December.
    2. Gallego, Gisselle & Taylor, Susan Joyce & Brien, Jo-anne Elizabeth, 2007. "Priority setting for high cost medications (HCMs) in public hospitals in Australia: A case study," Health Policy, Elsevier, vol. 84(1), pages 58-66, November.
    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. 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. Iskrov, Georgi & Miteva-Katrandzhieva, Tsonka & Stefanov, Rumen, 2012. "Challenges to orphan drugs access in Eastern Europe: The case of Bulgaria," Health Policy, Elsevier, vol. 108(1), pages 10-18.
    3. Todd Gammie & Christine Y Lu & Zaheer Ud-Din Babar, 2015. "Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries," PLOS ONE, Public Library of Science, vol. 10(10), pages 1-24, October.
    4. Pace, Jessica & Ghinea, Narcyz & Kerridge, Ian & Lipworth, Wendy, 2018. "An ethical framework for the creation, governance and evaluation of accelerated access programs," Health Policy, Elsevier, vol. 122(9), pages 984-990.
    5. 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.

    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. Rosenberg-Yunger, Zahava R.S. & Thorsteinsdóttir, Halla & Daar, Abdallah S. & Martin, Douglas K., 2012. "Stakeholder involvement in expensive drug recommendation decisions: An international perspective," Health Policy, Elsevier, vol. 105(2), pages 226-235.
    2. Jana Rogge & Bernhard Kittel, 2016. "Who Shall Not Be Treated: Public Attitudes on Setting Health Care Priorities by Person-Based Criteria in 28 Nations," PLOS ONE, Public Library of Science, vol. 11(6), pages 1-15, June.
    3. Gallagher, Siun & Little, Miles, 2019. "Procedural justice and the individual participant in priority setting: Doctors' experiences," Social Science & Medicine, Elsevier, vol. 228(C), pages 75-84.
    4. Chinitz, David & Meislin, Rachel & Alster-Grau, Ilana, 2009. "Values, institutions and shifting policy paradigms: Expansion of the Israeli National Health Insurance Basket of Services," Health Policy, Elsevier, vol. 90(1), pages 37-44, April.
    5. Brayan V. Seixas & François Dionne & Craig Mitton, 2021. "Practices of decision making in priority setting and resource allocation: a scoping review and narrative synthesis of existing frameworks," Health Economics Review, Springer, vol. 11(1), pages 1-11, December.
    6. Shavit, Oren, 2009. "Utilization of health technologies--Do not look where there is a light; shine your light where there is a need to look!: Relating national health goals with resource allocation decision-making; illust," Health Policy, Elsevier, vol. 92(2-3), pages 268-275, October.
    7. Adele Diederich & Jeannette Winkelhage & Norman Wirsik, 2011. "Age as a Criterion for Setting Priorities in Health Care? A Survey of the German Public View," PLOS ONE, Public Library of Science, vol. 6(8), pages 1-10, August.
    8. Jeannette Winkelhage & Adele Diederich, 2012. "The Relevance of Personal Characteristics in Allocating Health Care Resources—Controversial Preferences of Laypersons with Different Educational Backgrounds," IJERPH, MDPI, vol. 9(1), pages 1-21, January.
    9. Jennifer Whitty & Sharyn Rundle-Thiele & Paul Scuffham, 2012. "Insights from triangulation of two purchase choice elicitation methods to predict social decision making in healthcare," Applied Health Economics and Health Policy, Springer, vol. 10(2), pages 113-126, March.
    10. Douglas, Conor M.W. & Wilcox, Elizabeth & Burgess, Michael & Lynd, Larry D., 2015. "Why orphan drug coverage reimbursement decision-making needs patient and public involvement," Health Policy, Elsevier, vol. 119(5), pages 588-596.
    11. Hammerman, Ariel & Greenberg, Dan, 2009. "Estimating the budget impact of new technologies added to the National List of Health Services in Israel: Stakeholders' incentives for adopting a financial risk-sharing mechanism," Health Policy, Elsevier, vol. 89(1), pages 78-83, January.
    12. Arieh Gavious & Dan Greenberg & Ariel Hammerman & Ella Segev, 2014. "Impact of a financial risk-sharing scheme on budget-impact estimations: a game-theoretic approach," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 15(5), pages 553-561, June.
    13. Dale, Elina & Peacocke, Elizabeth F. & Movik, Espen & Voorhoeve, Alex & Ottersen, Trygve & Kurowski, Christoph & Evans, David B. & Norheim, Ole Frithjof & Gopinathan, Unni, 2023. "Criteria for the procedural fairness of health financing decisions: a scoping review," LSE Research Online Documents on Economics 119799, London School of Economics and Political Science, LSE Library.
    14. Lesley Chim & Glenn Salkeld & Patrick Kelly & Wendy Lipworth & Dyfrig A Hughes & Martin R Stockler, 2017. "Societal perspective on access to publicly subsidised medicines: A cross sectional survey of 3080 adults in Australia," PLOS ONE, Public Library of Science, vol. 12(3), pages 1-24, March.
    15. Lianne Barnieh & Fiona Clement & Anthony Harris & Marja Blom & Cam Donaldson & Scott Klarenbach & Don Husereau & Diane Lorenzetti & Braden Manns, 2014. "A Systematic Review of Cost-Sharing Strategies Used within Publicly-Funded Drug Plans in Member Countries of the Organisation for Economic Co-Operation and Development," PLOS ONE, Public Library of Science, vol. 9(3), pages 1-10, March.
    16. Kapiriri, Lydia & Razavi, Donya, 2017. "How have systematic priority setting approaches influenced policy making? A synthesis of the current literature," Health Policy, Elsevier, vol. 121(9), pages 937-946.
    17. Golan, Ofra & Hansen, Paul & Kaplan, Giora & Tal, Orna, 2011. "Health technology prioritization: Which criteria for prioritizing new technologies and what are their relative weights?," Health Policy, Elsevier, vol. 102(2), pages 126-135.
    18. Factor, Roni & Kang, Minah, 2014. "Priority setting in health care as portrayed in South Korean and Israeli newspapers," Health Policy, Elsevier, vol. 114(2), pages 226-235.

    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:eee:hepoli:v:100:y:2011:i:1:p:25-34. 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: Catherine Liu or the person in charge (email available below). General contact details of provider: http://www.elsevier.com/locate/healthpol .

    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.