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Surrogate Endpoints in Health Technology Assessment: An International Review of Methodological Guidelines

Author

Listed:
  • Bogdan Grigore

    (Institute of Health Research, University of Exeter)

  • Oriana Ciani

    (Institute of Health Research, University of Exeter
    SDA Bocconi)

  • Florian Dams

    (University of Bern)

  • Carlo Federici

    (SDA Bocconi)

  • Saskia Groot

    (Erasmus University Rotterdam)

  • Meilin Möllenkamp

    (Universität Hamburg)

  • Stefan Rabbe

    (Universität Hamburg)

  • Kosta Shatrov

    (University of Bern)

  • Antal Zemplenyi

    (Syreon Research Institute
    University of Pécs)

  • Rod S. Taylor

    (Institute of Health Research, University of Exeter
    University of Glasgow)

Abstract

In the drive towards faster patient access to treatments, health technology assessment (HTA) agencies are increasingly faced with reliance on evidence from surrogate endpoints, leading to increased decision uncertainty. This study undertook an updated survey of methodological guidance for using surrogate endpoints across international HTA agencies. We reviewed HTA and economic evaluation methods guidance from European, Australian and Canadian HTA agencies. We considered how guidelines addressed the methods for handling surrogate endpoints, including (1) level of evidence, (2) methods of validation, and (3) thresholds of acceptability. Across the 73 HTA agencies surveyed, 29 (40%) had methodological guidelines that made specific reference to consideration of surrogate outcomes. Of the 45 methods documents analysed, the majority [27 (60%)] were non-technology specific, 15 (33%) focused on pharmaceuticals and three (7%) on medical devices. The principles of the European network for Health Technology Assessment (EUnetHTA) guidelines published in 2015 on the handling of surrogate endpoints appear to have been adopted by many European HTA agencies, i.e. preference for final patient-relevant outcomes and reliance on surrogate endpoints with biological plausibility and epidemiological evidence of the association between the surrogate and final endpoint. Only a small number of HTA agencies (UK National Institute for Care and Excellence; the German Institute for Medical Documentation and Information and Institute for Quality and Efficiency in Health Care; the Australian Pharmaceutical Benefits Advisory Committee; and the Canadian Agency for Drugs and Technologies in Health) have developed more detailed prescriptive criteria for the acceptance of surrogate endpoints, e.g. meta-analyses of randomised controlled trials showing strong association between the treatment effect on the surrogate and final outcomes. As the decision uncertainty associated with reliance on surrogate endpoints carries a risk to patients and society, there is a need for HTA agencies to develop more detailed methodological guidance for consistent selection and evaluation of health technologies that lack definitive final patient-relevant outcome evidence at the time of the assessment.

Suggested Citation

  • Bogdan Grigore & Oriana Ciani & Florian Dams & Carlo Federici & Saskia Groot & Meilin Möllenkamp & Stefan Rabbe & Kosta Shatrov & Antal Zemplenyi & Rod S. Taylor, 2020. "Surrogate Endpoints in Health Technology Assessment: An International Review of Methodological Guidelines," PharmacoEconomics, Springer, vol. 38(10), pages 1055-1070, October.
  • Handle: RePEc:spr:pharme:v:38:y:2020:i:10:d:10.1007_s40273-020-00935-1
    DOI: 10.1007/s40273-020-00935-1
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    References listed on IDEAS

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    1. Ruof, Jörg & Knoerzer, Dietrich & Dünne, Anja-Alexandra & Dintsios, Charalabos-Markos & Staab, Thomas & Schwartz, Friedrich Wilhelm, 2014. "Analysis of endpoints used in marketing authorisations versus value assessments of oncology medicines in Germany," Health Policy, Elsevier, vol. 118(2), pages 242-254.
    2. Karl Claxton & Mark Sculpher & Chris McCabe & Andrew Briggs & Ron Akehurst & Martin Buxton & John Brazier & Tony O'Hagan, 2005. "Probabilistic sensitivity analysis for NICE technology assessment: not an optional extra," Health Economics, John Wiley & Sons, Ltd., vol. 14(4), pages 339-347, April.
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    As found by EconAcademics.org, the blog aggregator for Economics research:
    1. Chris Sampson’s journal round-up for 12th October 2020
      by Chris Sampson in The Academic Health Economists' Blog on 2020-10-12 11:00:03

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