IDEAS home Printed from https://ideas.repec.org/a/spr/eujhec/v18y2017i6d10.1007_s10198-016-0823-0.html
   My bibliography  Save this article

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 European countries

Author

Listed:
  • Elena Nicod

    (London School of Economics and Political Science)

Abstract

Purpose Health technology assessment (HTA) coverage recommendations differ across countries for the same drugs. Unlike previous studies, this study adopts a mixed methods research design to investigate, in a systematic manner, these differences. Methods HTA recommendations for ten orphan drugs appraised in England (NICE), Scotland (SMC), Sweden (TLV) and France (HAS) (N = 35) were compared using a validated methodological framework that breaks down these complex decision processes into stages facilitating their understanding, analysis and comparison, namely: (1) the clinical/cost-effectiveness evidence, (2) its interpretation (e.g. part of the deliberative process) and (3) influence on the final decision. This allowed qualitative and quantitative identification of the criteria driving recommendations and highlighted cross-country differences. Results Six out of ten drugs received diverging HTA recommendations. Reasons for cross-country differences included heterogeneity in the evidence appraised, in the interpretation of the same evidence, and in the different ways of dealing with the same uncertainty. These may have been influenced by agency-specific evidentiary, risk and value preferences, or stakeholder input. “Other considerations” (e.g. severity, orphan status) and other decision modulators (e.g. patient access schemes, lower discount rates, restrictions, re-assessments) also rendered uncertainty and cost-effectiveness estimates more acceptable. The different HTA approaches (clinical versus cost-effectiveness) and ways identified of dealing with orphan drug particularities also had implications on the final decisions. Conclusions This research contributes to better understanding the drivers of these complex decisions and why countries make different decisions. It also contributed to identifying those factors beyond the standard clinical and cost-effectiveness tools used in HTA, and their role in shaping these decisions.

Suggested Citation

  • 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.
    • Handle: RePEc:spr:eujhec:v:18:y:2017:i:6:d:10.1007_s10198-016-0823-0
    DOI: 10.1007/s10198-016-0823-0
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s10198-016-0823-0
    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/s10198-016-0823-0?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. Nicod, Elena & Kanavos, Panos, 2016. "Developing an evidence-based methodological framework to systematically compare HTA coverage decisions: A mixed methods study," Health Policy, Elsevier, vol. 120(1), pages 35-45.
    2. Brousselle, Astrid & Lessard, Chantale, 2011. "Economic evaluation to inform health care decision-making: Promise, pitfalls and a proposal for an alternative path," Social Science & Medicine, Elsevier, vol. 72(6), pages 832-839, March.
    3. Paul Dolan & Rebecca Shaw & Aki Tsuchiya & Alan Williams, 2005. "QALY maximisation and people's preferences: a methodological review of the literature," Health Economics, John Wiley & Sons, Ltd., vol. 14(2), pages 197-208, February.
    4. Mikael Svensson & Fredrik Nilsson & Karl Arnberg, 2015. "Reimbursement Decisions for Pharmaceuticals in Sweden: The Impact of Disease Severity and Cost Effectiveness," PharmacoEconomics, Springer, vol. 33(11), pages 1229-1236, November.
    5. 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.
    6. Banta, David, 2003. "The development of health technology assessment," Health Policy, Elsevier, vol. 63(2), pages 121-132, February.
    7. Nicod, Elena & Kanavos, Panos, 2012. "Commonalities and differences in HTA outcomes: A comparative analysis of five countries and implications for coverage decisions," Health Policy, Elsevier, vol. 108(2), pages 167-177.
    8. Gauvin, Francois-Pierre & Abelson, Julia & Giacomini, Mita & Eyles, John & Lavis, John N., 2010. ""It all depends": Conceptualizing public involvement in the context of health technology assessment agencies," Social Science & Medicine, Elsevier, vol. 70(10), pages 1518-1526, May.
    9. Michael Drummond & Gerard Pouvourville & Elizabeth Jones & Jennifer Haig & Grece Saba & Hélène Cawston, 2014. "A Comparative Analysis of Two Contrasting European Approaches for Rewarding the Value Added by Drugs for Cancer: England Versus France," PharmacoEconomics, Springer, vol. 32(5), pages 509-520, May.
    10. Mitton, Craig R. & McMahon, Meghan & Morgan, Steve & Gibson, Jennifer, 2006. "Centralized drug review processes: Are they fair?," Social Science & Medicine, Elsevier, vol. 63(1), pages 200-211, 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. Anderson, Michael & Drummond, Michael & Taylor, David & McGuire, Alistair & Carter, Paul & Mossialos, Elias, 2022. "Promoting innovation while controlling cost: The UK's approach to health technology assessment," Health Policy, Elsevier, vol. 126(3), pages 224-233.
    2. Visintin, Erica & Tinelli, Michela & Kanavos, Panos, 2019. "Value assessment of disease-modifying therapies for Relapsing-Remitting Multiple Sclerosis: HTA evidence from seven OECD countries," Health Policy, Elsevier, vol. 123(2), pages 118-129.
    3. Maynou, Laia & Cairns, John, 2018. "What is driving HTA decision-making? Evidence from cancer drug reimbursement decisions from 6 European countries," LSE Research Online Documents on Economics 90877, London School of Economics and Political Science, LSE Library.
    4. 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.
    5. Kanavos, Panos & Visintin, Erica & Gentilini, Arianna, 2023. "Algorithms and heuristics of health technology assessments: A retrospective analysis of factors associated with HTA outcomes for new drugs across seven OECD countries," Social Science & Medicine, Elsevier, vol. 331(C).
    6. Victoria Serra-Sastre & Simona Bianchi & Jorge Mestre-Ferrandiz & Phill O’Neill, 2021. "Does NICE influence the adoption and uptake of generics in the UK?," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 22(2), pages 229-242, March.
    7. Maynou, Laia & Cairns, John, 2019. "What is driving HTA decision-making? Evidence from cancer drug reimbursement decisions from 6 European countries," Health Policy, Elsevier, vol. 123(2), pages 130-139.
    8. 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.
    9. Kyle, Margaret K., 2022. "Incentives for pharmaceutical innovation: What’s working, what’s lacking," International Journal of Industrial Organization, Elsevier, vol. 84(C).
    10. Serra-Sastre, Victoria & Bianchi, Simona & Mestre-Ferrandiz, Jorge & O’Neill, Phill, 2021. "Does NICE influence the adoption and uptake of generics in the UK?," LSE Research Online Documents on Economics 113639, London School of Economics and Political Science, LSE Library.

    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. Aris Angelis & Ansgar Lange & Panos Kanavos, 2018. "Using health technology assessment to assess the value of new medicines: results of a systematic review and expert consultation across eight European countries," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 19(1), pages 123-152, January.
    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. 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.
    4. 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.
    5. Erik Nord & Jose Luis Pinto & Jeff Richardson & Paul Menzel & Peter Ubel, 1999. "Incorporating societal concerns for fairness in numerical valuations of health programmes," Health Economics, John Wiley & Sons, Ltd., vol. 8(1), pages 25-39, February.
    6. Bombard, Yvonne & Abelson, Julia & Simeonov, Dorina & Gauvin, Francois-Pierre, 2011. "Eliciting ethical and social values in health technology assessment: A participatory approach," Social Science & Medicine, Elsevier, vol. 73(1), pages 135-144, July.
    7. Francesca Iandolo & Pietro Vito & Irene Fulco & Francesca Loia, 2018. "From Health Technology Assessment to Health Technology Sustainability," Sustainability, MDPI, vol. 10(12), pages 1-22, December.
    8. 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.
    9. Whitty, Jennifer A. & Littlejohns, Peter, 2015. "Social values and health priority setting in Australia: An analysis applied to the context of health technology assessment," Health Policy, Elsevier, vol. 119(2), pages 127-136.
    10. Maynou, Laia & Cairns, John, 2019. "What is driving HTA decision-making? Evidence from cancer drug reimbursement decisions from 6 European countries," Health Policy, Elsevier, vol. 123(2), pages 130-139.
    11. 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.
    12. Fischer, Katharina E. & Rogowski, Wolf H. & Leidl, Reiner & Stollenwerk, Björn, 2013. "Transparency vs. closed-door policy: Do process characteristics have an impact on the outcomes of coverage decisions? A statistical analysis," Health Policy, Elsevier, vol. 112(3), pages 187-196.
    13. Mills, Mackenzie & Kanavos, Panos, 2022. "How do HTA agencies perceive conditional approval of medicines? Evidence from England, Scotland, France and Canada," Health Policy, Elsevier, vol. 126(11), pages 1130-1143.
    14. Livio Garattini & Anna Padula, 2020. "HTA for pharmaceuticals in Europe: will the mountain deliver a mouse?," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(1), pages 1-5, February.
    15. Maynou, Laia & Cairns, John, 2018. "What is driving HTA decision-making? Evidence from cancer drug reimbursement decisions from 6 European countries," LSE Research Online Documents on Economics 90877, London School of Economics and Political Science, LSE Library.
    16. Anderson, Michael & Drummond, Michael & Taylor, David & McGuire, Alistair & Carter, Paul & Mossialos, Elias, 2022. "Promoting innovation while controlling cost: The UK's approach to health technology assessment," Health Policy, Elsevier, vol. 126(3), pages 224-233.
    17. Jennifer A. Whitty & Julie Ratcliffe & Gang Chen & Paul A. Scuffham, 2014. "Australian Public Preferences for the Funding of New Health Technologies," Medical Decision Making, , vol. 34(5), pages 638-654, July.
    18. Nicod, Elena & Kanavos, Panos, 2016. "Developing an evidence-based methodological framework to systematically compare HTA coverage decisions: A mixed methods study," Health Policy, Elsevier, vol. 120(1), pages 35-45.
    19. Fischer, Katharina Elisabeth & Heisser, Thomas & Stargardt, Tom, 2016. "Health benefit assessment of pharmaceuticals: An international comparison of decisions from Germany, England, Scotland and Australia," Health Policy, Elsevier, vol. 120(10), pages 1115-1122.
    20. Fontrier, Anna-Maria & Visintin, Erica & Kanavos, Panos, 2022. "Similarities and differences in Health Technology Assessment systems and implications for coverage decisions: evidence from 32 countries," LSE Research Online Documents on Economics 112969, London School of Economics and Political Science, LSE Library.

    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:eujhec:v:18:y:2017:i:6:d:10.1007_s10198-016-0823-0. 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.