ArticleMedical decision making : an international journal of the Society for Medical Decision Making2021
Validity of Surrogate Endpoints and Their Impact on Coverage Recommendations: A Retrospective Analysis across International Health Technology Assessment Agencies.
Article in Medical decision making : an international journal of the Society for Medical Decision Making, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 48 citations in OpenAlex.
- Predicting overall survival benefit in previously untreated, unresectable or metastatic melanoma from improvement in progression-free survival: a correlation meta-analysis.Frontiers in oncology · 2025Pooled it
- UK National Screening Committee position statement on surrogate outcomes in cancer screening trials.BMJ (Clinical research ed.) · 2026Article
- Is Innovation Worth the Price? Cost-Effectiveness Analysis in Interventional Oncology.Cardiovascular and interventional radiology · 2026Review
- A multi-stakeholder survey on communicating cost-effectiveness uncertainties to stakeholders: a case study of ICER.Health affairs scholar · 2026Article
- Radiographic progression-free survival as a surrogate endpoint for overall survival in first-line ARPi naïve metastatic castration-resistant prostate cancer.The oncologist · 2026Article
- Addressing clinical uncertainties in ATMP reimbursement: a review of methodological guidelines and European practice.Frontiers in pharmacology · 2026Review
- Use of surrogate endpoints in health technology assessment and reimbursement of treatments for the management of chronic kidney disease.EClinicalMedicine · 2025Review
- Review
- Use of surrogate endpoints in health technology assessment: a review of selected NICE technology appraisals in oncology.International journal of technology assessment in health care · 2025Review
- A Causal Inference Approach to Mediation Analysis in Vitreomacular Traction: How Much Does Traction Resolution Mediate Functional Outcomes?Journal of market access & health policy · 2024Article
- Surrogacy and the Valuation of ATMPs: Taking Our Place in the Evidence Generation/Assessment Continuum.PharmacoEconomics · 2024Article
- UK Stakeholder Perspectives on Surrogate Endpoints in Cancer, and the Potential for UK Real-World Datasets to Validate Their Use in Decision-Making.Cancer management and research · 2024Article
- Article
- Development of a framework and decision tool for the evaluation of health technologies based on surrogate endpoint evidence.Health economics · 2022Article
- Coverage with evidence development for medical devices in Europe: Can practice meet theory?Health economics · 2022Article
- Outcome-based reimbursement in Central-Eastern Europe and Middle-East.Frontiers in medicine · 2022Article
- Value Insider Season 1 Episode 1: The Importance of Payers and HTA: How Did We End Up Here? (Introduction to Value) [Podcast].International journal of general medicine · 2022Article
- The Impasse on Overall Survival in Oncology Reimbursement Decision-Making: How Can We Resolve This?Cancer management and research · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 4 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundSurrogate endpoints (i.e., intermediate endpoints intended to predict for patient-centered outcomes) are increasingly common. However, little is known about how surrogate evidence is handled in the context of health technology assessment (HTA).
objectives1) To map methodologies for the validation of surrogate endpoints and 2) to determine their impact on acceptability of surrogates and coverage decisions made by HTA agencies.
methodsWe sought HTA reports where evaluation relied on a surrogate from 8 HTA agencies. We extracted data on the methods applied for surrogate validation. We assessed the level of agreement between agencies and fitted mixed-effects logistic regression models to test the impact of validation approaches on the agency's acceptability of the surrogate endpoint and their coverage recommendation.
resultsOf the 124 included reports, 61 (49%) discussed the level of evidence to support the relationship between the surrogate and the patient-centered endpoint, 27 (22%) reported a correlation coefficient/association measure, and 40 (32%) quantified the expected effect on the patient-centered outcome. Overall, the surrogate endpoint was deemed acceptable in 49 (40%) reports (
conclusionsHandling of surrogate endpoint evidence in reports varied greatly across HTA agencies, with inconsistent consideration of the level of evidence and statistical validation. Our findings call for careful reconsideration of the issue of surrogacy and the need for harmonization of practices across international HTA agencies.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.