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. 2023 Oct;28(10):103719.
doi: 10.1016/j.drudis.2023.103719. Epub 2023 Jul 17.

Regulatory benefit-risk assessment of oncology drugs: A systematic review of FDA and EMA approvals

Cathy Anne Pinto  1 , Zaneta Balantac  2 , Shahrul Mt-Isa  3 , Xinyue Liu  4 , Oswaldo L Bracco  5 , Harrison Clarke  2 , Tommi Tervonen  2
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Free article

Regulatory benefit-risk assessment of oncology drugs: A systematic review of FDA and EMA approvals

Cathy Anne Pinto et al. Drug Discov Today. 2023 Oct.
Free article

Abstract

The European Medicines Agency (EMA) and FDA have policy goals of strengthening benefit-risk (B-R) capabilities; but how this has been translating into regulatory practice is unclear. A systematic review of oncology drug approvals between 2015 and 2020 was conducted with approvals identified through review of FDA and EMA annual reports, with extraction of information on submission, clinical program and B-R assessment from publicly available review documents. Data were extracted from 236 reviews (EMA: 66 new submissions, 100 label extensions; FDA: 70 new submissions). The standard of evidence for B-R assessments seems to have diversified over time; yet, despite policy targets to extend their use, these assessments rarely include patient experience or real-world data.

Keywords: benefit–risk assessment; oncology drugs; regulatory.

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Conflict of interest statement

Declaration of Competing Interest CAP, XL, SMI and OB are employees of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Rahway, NJ, USA (MSD), which funded this study and develops and manufactures oncologic therapies. ZB and HC are salaried employees of Evidera, which received funds from MSD to conduct this study. TT was a salaried employee of Evidera at the time of the study.

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