UK Stakeholder Perspectives on Surrogate Endpoints in Cancer, and the Potential for UK Real-World Datasets to Validate Their Use in Decision-Making.

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.

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© 2024 Baldwin et al.

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Dr Khalid Siddiqui is a current employee of Johnson and Johnson Innovative Medicine, UK. Dr David Baldwin reports personal fees, non-financial support from Janssen during the conduct of the study; personal fees from AstraZeneca, personal fees from MSD, and personal fees from Roche; and honoraria from Bristol Myers Squibb, Roche, Astra Zeneca, and MSD outside the submitted work. Dr Jonathan Carmichael reports personal fees from Janssen, during the conduct of the study. Dr Gordon Cook reports grants, personal fees from Janssen, grants, personal fees from Takeda, grants, personal fees from BMS, personal fees from Pfizer, and personal fees from Amgen, during the conduct of the study. Dr Neal Navani reports grants from Medical Research Council, personal fees, non-financial support from Amgen, grants, personal fees, non-financial support from Astra Zeneca, personal fees, non-financial support from Boehringer Ingelheim, personal fees from Bristol Meyers Squibb, personal fees from EQRx, personal fees, non-financial support from Fujifilm, personal fees from Guardant Health, personal fees from Intuitive, personal fees from Janssen, grants, personal fees from Lilly, personal fees from Merck Sharp Dohme, personal fees, non-financial support from Olympus, personal fees from Roche, during the conduct of the study. Mr James Peach reports grants from Jansen & Jansen, grants from ABPI, during the conduct of the study; Shareholding from Univ8 Genomics, personal fees from IQVIA, personal fees from Roche, personal fees from ABPI, personal fees from Health Data Research UK, personal fees from Sensyne Health, personal fees from Novartis, personal fees from Medicines Discovery Catapult, outside the submitted work; and Voluntary (unpaid) advisor to Cancer Research UK and sit on funding committee. Dr Nicola Allen-Delingpole reports that ABPI is a trade association representing the pharmaceutical industry in the UK funded by annual subscriptions from member companies from ABPI, outside the submitted work; Dr Cicely Kerr was an employee of Janssen-Cilag at the time the work was commissioned by this company and currently holds shares in the company. The authors report no other conflicts of interest in this work.