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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

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  • 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.

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  • 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
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    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.
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    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.

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