Lorna Wheaton, Sylwia Bujkiewicz
arXiv 3 Dec 2024 · Statistics — Applications · publishedInternational Journal of Technology Assessment in Health Care (2025) · 6 citations (OpenAlex)
arXiv:2412.02380 · PDF · DOI · OpenAlex · Extracted main text
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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The works this paper leans on most, across its whole bibliography — not restricted to papers in our corpus. Ranked by composite intensity, which combines how often a work is mentioned, how many sections mention it, and how much of that falls in the main text rather than the appendix.
| Reference | Intensity | Mentions | Sections | Main text | |
|---|---|---|---|---|---|
| 1 | Ciani O, Buyse M, Drummond M, Rasi G, Saad ED, Taylor RS (2017) Time to review the role of surrogate end points in health policy: state of the art and the way forward | 1.000 | 6 | 4 | 100% |
| 2 | Pembrolizumab for adjuvant treatment of completely resected stage 3… (2022) https://www.nice.org.uk/guidance/ta766 | 0.874 | 6 | 2 | 100% |
| 3 | Niraparib for maintenance treatment of relapsed, platinum-sensitive… (2022) https://www.nice.org.uk/guidance/ta784 | 0.693 | 6 | 1 | 100% |
| 4 | Asciminib for treating chronic myeloid leukaemia after 2 or more tyr… (2022) https://www.nice.org.uk/guidance/ta813 | 0.693 | 6 | 1 | 100% |
| 5 | Daratumumab in combination for untreated multiple myeloma when a ste… (2022) https://www.nice.org.uk/guidance/ta763 | 0.693 | 5 | 1 | 100% |
| 6 | Ibrutinib for treating Waldenstrom’s macroglobulinaemia , Technology… (2022) https://www.nice.org.uk/guidance/ta795 | 0.693 | 5 | 1 | 100% |
| 7 | Venetoclax for treating chronic lymphocytic leukaemia , Technology A… (2022) https://www.nice.org.uk/guidance/ta796 | 0.693 | 5 | 1 | 100% |
| 8 | Nivolumab with chemotherapy for neoadjuvant treatment of resectable… (2023) https://www.nice.org.uk/guidance/ta876 | 0.693 | 5 | 1 | 100% |
| 9 | Nivolumab for adjuvant treatment of invasive urothelial cancer at hi… (2022) https://www.nice.org.uk/guidance/ta817 | 0.644 | 4 | 1 | 100% |
| 10 | Atezolizumab for adjuvant treatment of resected non-small-cell lung… (2022) https://www.nice.org.uk/guidance/ta823 | 0.644 | 4 | 1 | 100% |
Showing the top 10 of 75 scored citations.