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Real-world outcomes associated with new cancer medicines approved by the Food and Drug Administration and European Medicines Agency: A retrospective cohort study

Research output: Contribution to journalArticlepeer-review

Jemma M. Boyle, Gemma Hegarty, Christopher Frampton, Elizabeth Harvey-Jones, Joanna Dodkins, Katharina Beyer, Gincy George, Richard Sullivan, Christopher Booth, Ajay Aggarwal

Original languageEnglish
Pages (from-to)136-144
Number of pages9
JournalEuropean Journal of Cancer
PublishedSep 2021

Bibliographical note

Funding Information: This work was supported by a National Institute for Health Research (NIHR) Advanced Fellowship [ NIHR300599 ] to [AA] and by the UK Research and Innovation Economic and Social Research Council [ES/P010962/1] to [RS]. These grant sources had no involvement in the study design, collection, analysis and interpretation of data, in the writing of the report, or the decision to submit the article for publication. Publisher Copyright: © 2021 The Authors Copyright: Copyright 2021 Elsevier B.V., All rights reserved.

King's Authors


Purpose: Real-World Data (RWD) studies are increasingly used to support regulatory approvals, reimbursement decisions, and changes in clinical practice for novel cancer drugs. However, few studies have systematically appraised their quality or compared outcomes to pivotal trials. Methods: All RWD studies (2010–2019) for drugs approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) from 2010 to 2015 for solid organ tumours in the non-curative setting were identified. Quality assessment was undertaken using the Newcastle Ottawa Scale. Survival differences between each RWD study and the pivotal trial were determined using a related sample Wilcoxon signed-rank test. Results: 293 RWD studies for 45 of the 57 drug indications approved by the FDA/EMA were identified. The most common tumour types were prostate cancer (29%, n = 86) and melanoma (15%, n = 43). A quarter of the studies had industry funding. No high-quality studies were identified, and 78% were low quality. Comparative survival analysis between RWD and pivotal trials was possible for 224 studies (37 drug indications). Differences in median survival between the RWD studies and their corresponding trial ranged from −32 months to 21 months (IQR –4·2 months to 1·6 months). Low-quality studies were more likely to report superior survival outcomes (23%) compared to higher quality studies (8%) (p = 0.02). Conclusion: RWD study quality for novel cancer drugs is low and of insufficient rigour to inform reimbursement decisions and clinical practice. RWD studies seeking publication should provide a completed quality assessment tool on submission. Greater investment in properly designed RWD studies is required.

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