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Use of surrogate endpoints in health technology assessment: a review of selected NICE technology appraisals in oncology

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  • Lorna Wheaton
  • Sylwia Bujkiewicz

Abstract

Objectives: Surrogate endpoints, used to substitute for and predict final clinical outcomes, are increasingly being used to support submissions to health technology assessment agencies. The increase in use of surrogate endpoints has been accompanied by literature describing frameworks and statistical methods to ensure their robust validation. The aim of this review was to assess how surrogate endpoints have recently been used in oncology technology appraisals by the National Institute for Health and Care Excellence (NICE) in England and Wales. Methods: This paper identified technology appraisals in oncology published by NICE between February 2022 and May 2023. Data are extracted on methods for the use and validation of surrogate endpoints. Results: Of the 47 technology appraisals in oncology available for review, 18 (38 percent) utilised surrogate endpoints, with 37 separate surrogate endpoints being discussed. However, the evidence supporting the validity of the surrogate relationship varied significantly across putative surrogate relationships with 11 providing RCT evidence, 7 providing evidence from observational studies, 12 based on clinical opinion and 7 providing no evidence for the use of surrogate endpoints. Conclusions: This review supports the assertion that surrogate endpoints are frequently used in oncology technology appraisals in England and Wales. Despite increasing availability of statistical methods and guidance on appropriate validation of surrogate endpoints, this review highlights that use and validation of surrogate endpoints can vary between technology appraisals which can lead to uncertainty in decision-making.

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  • Lorna Wheaton & Sylwia Bujkiewicz, 2024. "Use of surrogate endpoints in health technology assessment: a review of selected NICE technology appraisals in oncology," Papers 2412.02380, arXiv.org, revised Dec 2024.
    • Handle: RePEc:arx:papers:2412.02380
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