Cancer Management and Research (Jul 2024)
UK Stakeholder Perspectives on Surrogate Endpoints in Cancer, and the Potential for UK Real-World Datasets to Validate Their Use in Decision-Making
Abstract
David Baldwin,1 Jonathan Carmichael,2 Gordon Cook,3 Neal Navani,4,5 James Peach,6 Ruth Slater,7 Pete Wheatstone,8 Julia Wilkins,7 Nicola Allen-Delingpole,9 Cicely EP Kerr,10 Khalid Siddiqui11 1Department of Respiratory Medicine, Nottingham University Hospitals NHS Trust and the University of Nottingham, Nottingham, UK; 2Department of Oncology, The National Institute for Health Research Leeds In Vitro Diagnostics Co-Operative (NIHR Leeds MIC), Leeds, UK; 3Cancer Research UK Trials Unit, LICTR, University of Leeds & NIHR (Leeds) IVD MIC, Leeds, UK; 4Lungs for Living Research Centre, UCL Respiratory, University College London, London, UK; 5Department of Thoracic Medicine, University College London Hospital, London, UK; 6Human Centric Drug Discovery, Wood Centre for Innovation, Oxford, UK; 7Health Economics Unit, London, UK; 8Patient and Public Involvement and Engagement Group, DATA-CAN, London, UK; 9The Association of the British Pharmaceutical Industry, London, UK; 10Research Data Scotland, Bayes Centre, Edinburgh, UK; 11Janssen-Cilag Ltd, High Wycombe, UKCorrespondence: James Peach, Human Centric Drug Discovery, Wood Centre for Innovation, Quarry Road, Oxford, OX3 8SB, UK, Email [email protected]: Duration of overall survival in patients with cancer has lengthened due to earlier detection and improved treatments. However, these improvements have created challenges in assessing the impact of newer treatments, particularly those used early in the treatment pathway. As overall survival remains most decision-makers’ preferred primary endpoint, therapeutic innovations may take a long time to be introduced into clinical practice. Moreover, it is difficult to extrapolate findings to heterogeneous populations and address the concerns of patients wishing to evaluate everyday quality and extension of life. There is growing interest in the use of surrogate or interim endpoints to demonstrate robust treatment effects sooner than is possible with measurement of overall survival. It is hoped that they could speed up patients’ access to new drugs, combinations, and sequences, and inform treatment decision-making. However, while surrogate endpoints have been used by regulators for drug approvals, this has occurred on a case-by-case basis. Evidence standards are yet to be clearly defined for acceptability in health technology appraisals or to shape clinical practice. This article considers the relevance of the use of surrogate endpoints in cancer in the UK context, and explores whether collection and analysis of real-world UK data and evidence might contribute to validation.Keywords: cancer, surrogate endpoints, real-world data, multiple myeloma, lung cancer, quality of life
