{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T09:23:32Z","timestamp":1793179412904,"version":"4.5.0"},"reference-count":74,"publisher":"Cambridge University Press (CUP)","issue":"1","license":[{"start":{"date-parts":[[2025,2,19]],"date-time":"2025-02-19T00:00:00Z","timestamp":1739923200000},"content-version":"unspecified","delay-in-days":49,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100000265","name":"Medical Research Council","doi-asserted-by":"publisher","award":["MR\/T025166\/1"],"award-info":[{"award-number":["MR\/T025166\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Int J Technol Assess Health Care"],"published-print":{"date-parts":[[2025]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec id=\"S0266462325000017_sec1\">\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>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 the use of surrogate endpoints has been accompanied by literature describing the 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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"S0266462325000017_sec2\" sec-type=\"methods\">\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This article identifies technology appraisals in oncology published by NICE between February 2022 and May 2023. Data are extracted on the use and validation of surrogate endpoints including purpose, evidence base, and methods used.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"S0266462325000017_sec3\" sec-type=\"results\">\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Of the 47 technology appraisals in oncology available for review, 18 (38 percent) utilized 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 randomized controlled trial evidence, 7 providing evidence from observational studies, 12 based on the clinical opinion, and 7 providing no evidence for the use of surrogate endpoints.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"S0266462325000017_sec4\" sec-type=\"conclusions\">\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>This review supports the assertion that surrogate endpoints are frequently used in oncology technology appraisals in England and Wales and despite the 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.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1017\/s0266462325000017","type":"journal-article","created":{"date-parts":[[2025,2,19]],"date-time":"2025-02-19T00:59:51Z","timestamp":1739926791000},"source":"Crossref","is-referenced-by-count":9,"title":["Use of surrogate endpoints in health technology assessment: a review of selected NICE technology appraisals in oncology"],"prefix":"10.1017","volume":"41","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2318-7109","authenticated-orcid":false,"given":"Lorna","family":"Wheaton","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylwia","family":"Bujkiewicz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"56","published-online":{"date-parts":[[2025,2,19]]},"reference":[{"key":"S0266462325000017_r72","unstructured":"72. Pembrolizumab with carboplatin and paclitaxel for untreated metastatic squamous non-small-cell lung cancer, Technology Appraisal Guidance [TA770], Published: 9th February 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta770."},{"key":"S0266462325000017_r41","doi-asserted-by":"publisher","DOI":"10.1177\/0272989X231162852"},{"key":"S0266462325000017_r39","doi-asserted-by":"publisher","DOI":"10.1002\/sim.8465"},{"key":"S0266462325000017_r58","unstructured":"58. Olaparib for previously treated BRCA mutation-positive hormone-relapsed metastatic prostate cancer, Technology Appraisal Guidance [TA831], Published: 5th October 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta831."},{"key":"S0266462325000017_r28","unstructured":"28. Asciminib for treating chronic myeloid leukaemia after 2 or more tyrosine kinase inhibitors, Technology Appraisal Guidance [TA813], Published: 3rd August 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta813."},{"key":"S0266462325000017_r53","unstructured":"53. Mobocertinib for treating EGFR exon 20 insertion mutation-positive advanced non-small-cell lung cancer after platinum-based chemotherapy, Technology Appraisal Guidance [TA855], Published: 4th January 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta855."},{"key":"S0266462325000017_r51","unstructured":"51. Nivolumab with platinum- and fluoropyrimidine-based chemotherapy for untreated HER2-negative advanced gastric, gastro-oesophageal junction or oesophageal adenocarcinoma, Technology Appraisal Guidance [TA857], Published: 11th January 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta857."},{"key":"S0266462325000017_r54","unstructured":"54. Cabozantinib for previously treated advanced hepatocellular carcinoma, Technology Appraisal Guidance [TA849], Published: 14th December 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta849."},{"key":"S0266462325000017_r24","unstructured":"24. Pembrolizumab for adjuvant treatment of renal cell carcinoma, Technology Appraisal Guidance [TA830], Published: 19th October 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta830."},{"key":"S0266462325000017_r6","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2016.2240"},{"key":"S0266462325000017_r42","doi-asserted-by":"publisher","DOI":"10.1016\/j.jclinepi.2023.06.013"},{"key":"S0266462325000017_r30","unstructured":"30. Venetoclax for treating chronic lymphocytic leukaemia, Technology Appraisal Guidance [TA796], Published: 15th June 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta796."},{"key":"S0266462325000017_r11","doi-asserted-by":"publisher","DOI":"10.1080\/10543406.2015.1094811"},{"key":"S0266462325000017_r25","unstructured":"25. Zanubrutinib for treating Waldenstrom\u2019s macroglobulinaemia, Technology Appraisal Guidance [TA833], Published: 19th October 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta833."},{"key":"S0266462325000017_r38","volume-title":"Institute for Quality and Efficiency in Health Care (IQWiG): Executive Summaries [Internet].","year":"2011"},{"key":"S0266462325000017_r49","unstructured":"49. Nivolumab with fluoropyrimidine- and platinum-based chemotherapy for untreated unresectable advanced, recurrent, or metastatic oesophageal squamous cell carcinoma, Technology Appraisal Guidance [TA865], Published: 8th February 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta865."},{"key":"S0266462325000017_r56","unstructured":"56. Palbociclib with fulvestrant for treating hormone receptor-positive, HER2-negative advanced breast cancer after endocrine therapy, Technology Appraisal Guidance [TA836], Published: 26th October 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta836."},{"key":"S0266462325000017_r57","unstructured":"57. Oral azacitidine for maintenance treatment of acute myeloid leukaemia after induction therapy, Technology Appraisal Guidance [TA827], Published: 5th October 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta827."},{"key":"S0266462325000017_r40","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2023.09.2168"},{"key":"S0266462325000017_r65","unstructured":"65. Avelumab for maintenance treatment of locally advanced or metastatic urothelial cancer after platinum-based chemotherapy, Technology Appraisal Guidance [TA788], Published: 11th May 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta788."},{"key":"S0266462325000017_r10","doi-asserted-by":"publisher","DOI":"10.1007\/s40273-020-00935-1"},{"key":"S0266462325000017_r66","unstructured":"66. Tucatinib with trastuzumab and capecitabine for treating HER2-positive advanced breast cancer after 2 or more anti-HER2 therapies, Technology Appraisal Guidance [TA786], Published: 27th April 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta786."},{"key":"S0266462325000017_r73","unstructured":"73. Venetoclax with Azacitidine for untreated acute myeloid leukaemia when intensive chemotherapy is unsuitable, Technology Appraisal Guidance [TA765], Published: 2nd February 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta765."},{"key":"S0266462325000017_r9","doi-asserted-by":"publisher","DOI":"10.1177\/0272989X21994553"},{"key":"S0266462325000017_r1","doi-asserted-by":"publisher","DOI":"10.1038\/nrd.2016.81"},{"key":"S0266462325000017_r60","unstructured":"60. Pralsetinib for treating RET fusion-positive advanced non-small-cell lung cancer, Technology Appraisal Guidance [TA812], Published: 3rd August 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta812."},{"key":"S0266462325000017_r36","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(13)70158-X"},{"key":"S0266462325000017_r5","doi-asserted-by":"publisher","DOI":"10.1001\/jamainternmed.2019.0462"},{"key":"S0266462325000017_r3","doi-asserted-by":"publisher","DOI":"10.1002\/hec.4524"},{"key":"S0266462325000017_r15","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2021-052186"},{"key":"S0266462325000017_r21","unstructured":"21. Trastuzumab deruxtecan for treating HER2-positive unresectable or metastatic breast cancer after 1 or more anti-HER2 treatments, Technology Appraisal Guidance [TA862], Published: 1st February 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta862."},{"key":"S0266462325000017_r34","unstructured":"34. Daratumumab in combination for untreated multiple myeloma when a stem cell transplant is suitable, Technology Appraisal Guidance [TA763], Published: 2nd February 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta763."},{"key":"S0266462325000017_r48","unstructured":"48. Ixazomib with lenalidomide and dexamethasone for treating relapsed or refractory multiple myeloma, Technology Appraisal Guidance [TA870], Published: 22nd February 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta870."},{"key":"S0266462325000017_r32","unstructured":"32. Niraparib for maintenance treatment of relapsed, platinum-sensitive ovarian, fallopian tube and peritoneal cancer, Technology Appraisal Guidance [TA784], Published: 20th April 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta784."},{"key":"S0266462325000017_r20","unstructured":"20. Polatuzumab vedotin in combination for untreated diffuse large B-cell lymphoma, Technology Appraisal Guidance [TA874], Published: 1st March 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta874."},{"key":"S0266462325000017_r46","unstructured":"46. Bouvy, J . How \u201csurrogate outcomes\u201d influence long-term health outcomes. 2023. Available from: https:\/\/www.nice.org.uk\/news\/blog\/how-surrogate-outcomes-influence-long-term-health-outcomes."},{"key":"S0266462325000017_r16","doi-asserted-by":"publisher","DOI":"10.1002\/sim.5403"},{"key":"S0266462325000017_r29","unstructured":"29. Abemaciclib with endocrine therapy for adjuvant treatment of hormone receptor-positive, HER2-negative, node-positive early breast cancer at high risk of recurrence, Technology Appraisal Guidance [TA810], Published: 20th July 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta810."},{"key":"S0266462325000017_r67","unstructured":"67. Venetoclax with low dose cytarabine for untreated acute myeloid leukaemia when intensive chemotherapy is unsuitable, Technology Appraisal Guidance [TA787], Published: 27th April 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta787."},{"key":"S0266462325000017_r7","doi-asserted-by":"publisher","DOI":"10.1186\/s12916-017-0902-9"},{"key":"S0266462325000017_r55","unstructured":"55. Amivantamab for treating EGFR exon 20 insertion mutation-positive advanced non-small-cell lung cancer after platinum-based chemotherapy, Technology Appraisal Guidance [TA850], Published: 14th December 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta850."},{"key":"S0266462325000017_r18","unstructured":"18. Pembrolizumab plus chemotherapy with or without bevacizumab for persistent, recurrent or metastatic cervical cancer, Technology Appraisal Guidance [TA885], Published: 3rd May 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta885."},{"key":"S0266462325000017_r71","unstructured":"71. Dostarlimab for previously treated advanced or recurrent endometrial cancer with high microsatellite instability or mismatch repair deficiency, Technology Appraisal Guidance [TA779], Published: 16th March 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta779."},{"key":"S0266462325000017_r74","unstructured":"74. Tafasitamab with lenalidomide for treating relapsed or refractory diffuse large B-cell lymphoma, Technology Appraisal Guidance [TA883], Published: 3rd May 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta883."},{"key":"S0266462325000017_r12","unstructured":"12. Bujkiewicz, S , Achana, F , Papanikos, T , Riley, R , Abrams, K . NICE DSU Technical Support Document 20: Multivariate meta-analysis of summary data for combining treatment effects on correlated outcomes and evaluating surrogate endpoints. 2019. Available from: https:\/\/www.sheffield.ac.uk\/sites\/default\/files\/2022-02\/TSD20-mvmeta-final.pdf."},{"key":"S0266462325000017_r2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2016.10.011"},{"key":"S0266462325000017_r4","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2020.1176"},{"key":"S0266462325000017_r70","unstructured":"70. Nivolumab with ipilimumab for untreated advanced renal cell carcinoma, Technology Appraisal Guidance [TA780], Published: 24th March 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta780."},{"key":"S0266462325000017_r31","unstructured":"31. Ibrutinib for treating Waldenstrom\u2019s macroglobulinaemia, Technology Appraisal Guidance [TA795], Published: 8th June 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta795."},{"key":"S0266462325000017_r69","unstructured":"69. Sotorasib for previously treated KRAS G12C mutation-positive advanced non-small-cell lung cancer, Technology Appraisal Guidance [TA781], Published: 30th March 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta781."},{"key":"S0266462325000017_r47","unstructured":"47. Ripretinib for treating advanced gastrointestinal stromal tumour after 3 or more treatments, Technology Appraisal Guidance [TA881], Published: 3rd May 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta881."},{"key":"S0266462325000017_r8","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.n2191"},{"key":"S0266462325000017_r33","unstructured":"33. Pembrolizumab for treating relapsed or refractory classical Hodgkin lymphoma after stem cell transplant or at least 2 previous therapies, Technology Appraisal Guidance [TA772], Published: 23rd February 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta772."},{"key":"S0266462325000017_r13","unstructured":"13. Welton, NJ , Phillippo, D , Owen, R , Jones, H , Dias, S , Bujkiewicz, S , Ades, A , Abrams, K. CHTE2020 sources and synthesis of evidence: update to evidence synthesis methods. 2020."},{"key":"S0266462325000017_r14","unstructured":"14. NICE publishes new combined methods and processes manual and topic selection manual. published: 31st January 2022. Available from: https:\/\/www.nice.org.uk\/news\/articles\/nice-publishes-new-combined-methods-and-processes-manual-and-topic-selection-manual-for-its-health-technology-evaluation-programmes."},{"key":"S0266462325000017_r52","unstructured":"52. Lenvatinib with pembrolizumab for untreated advanced renal cell carcinoma, Technology Appraisal Guidance [TA858], Published: 11th January 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta858."},{"key":"S0266462325000017_r64","unstructured":"64. Tepotinib for treating advanced non-small-cell lung cancer with MET gene alterations, Technology Appraisal Guidance [TA789], Published: 18th May 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta789."},{"key":"S0266462325000017_r61","unstructured":"61. Pembrolizumab plus chemotherapy for untreated, triple-negative, locally recurrent unresectable or metastatic breast cancer, Technology Appraisal Guidance [TA801], Published: 29th June 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta801."},{"key":"S0266462325000017_r50","unstructured":"50. Regorafenib for previously treated metastatic colorectal cancer, Technology Appraisal Guidance [TA866], Published: 8th February 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta866."},{"key":"S0266462325000017_r68","unstructured":"68. Daratumumab monotherapy for treating relapsed and refractory multiple myeloma, Technology Appraisal Guidance [TA783], Published: 13th April 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta783."},{"key":"S0266462325000017_r45","unstructured":"45. Surrogate Endpoint Statistical Evaluation for HTA Decision Making. 2024. Available from: https:\/\/www.ispor.org\/member-groups\/task-forces\/surrogate-endpoint-statistical-assessment-for-hta-decision-making."},{"key":"S0266462325000017_r44","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2022-064304"},{"key":"S0266462325000017_r23","unstructured":"23. Pembrolizumab for adjuvant treatment of resected stage 2B or 2C melanoma, Technology Appraisal Guidance [TA837], Published: 26th October 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta837."},{"key":"S0266462325000017_r26","unstructured":"26. Atezolizumab for adjuvant treatment of resected non-small-cell lung cancer, Technology Appraisal Guidance [TA823], Published: 28th September 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta823."},{"key":"S0266462325000017_r62","unstructured":"62. Cemiplimab for treating advanced cutaneous squamous cell carcinoma, Technology Appraisal Guidance [TA802], Published: 29th June 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta802."},{"key":"S0266462325000017_r43","doi-asserted-by":"publisher","DOI":"10.1016\/j.eclinm.2023.102283"},{"key":"S0266462325000017_r63","unstructured":"63. Durvalumab for maintenance treatment of unresectable non-small-cell lung cancer after platinum-based chemoradiation, Technology Appraisal Guidance [TA798], Published: 22nd June 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta798."},{"key":"S0266462325000017_r19","unstructured":"19. Nivolumab with chemotherapy for neoadjuvant treatment of resectable non-small-cell lung cancer, Technology Appraisal Guidance [TA876], Published: 22nd March 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta876."},{"key":"S0266462325000017_r59","unstructured":"59. Alpelisib with fulvestrant for treating hormone receptor-positive, HER2-negative, PIK3CA-mutated advanced breast cancer, Technology Appraisal Guidance [TA816], Published: 10th August 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta816."},{"key":"S0266462325000017_r37","unstructured":"37. Tafasitamab with lenalidomide for treating relapsed or refractory diffuse large B-cell lymphoma, Technology Appraisal Guidance [TA883], Published: 3rd May 2023. Available from: https:\/\/www.nice.org.uk\/guidance\/ta883."},{"key":"S0266462325000017_r35","unstructured":"35. Pembrolizumab for adjuvant treatment of completely resected stage 3 melanoma, Technology Appraisal Guidance [TA766], Published: 2nd February 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta766."},{"key":"S0266462325000017_r27","unstructured":"27. Nivolumab for adjuvant treatment of invasive urothelial cancer at high risk of recurrence, Technology Appraisal Guidance [TA817], Published: 10th August 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta817."},{"key":"S0266462325000017_r17","unstructured":"17. Sacituzumab govitecan for treating unresectable triple-negative advanced breast cancer after 2 or more therapies, Technology Appraisal Guidance [TA819], Published: 17th August 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta819."},{"key":"S0266462325000017_r22","unstructured":"22. Pembrolizumab for neoadjuvant and adjuvant treatment of triple-negative early or locally advanced breast cancer, Technology Appraisal Guidance [TA851], Published: 14th December 2022. Available from: https:\/\/www.nice.org.uk\/guidance\/ta851."}],"container-title":["International Journal of Technology Assessment in Health Care"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.cambridge.org\/core\/services\/aop-cambridge-core\/content\/view\/S0266462325000017","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,2,19]],"date-time":"2025-02-19T01:00:14Z","timestamp":1739926814000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.cambridge.org\/core\/product\/identifier\/S0266462325000017\/type\/journal_article"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025]]},"references-count":74,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2025]]}},"alternative-id":["S0266462325000017"],"URL":"https:\/\/doi.org\/10.1017\/s0266462325000017","relation":{},"ISSN":["0266-4623","1471-6348"],"issn-type":[{"value":"0266-4623","type":"print"},{"value":"1471-6348","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025]]},"article-number":"e11"}}