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

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.

Automated summary

The policies of EMA and FDA aim to strengthen benefit-risk capabilities, but how they are implemented in regulatory practice is unclear. A systematic review of oncology drug approvals between 2015 and 2020 revealed that the standard of evidence for benefit-risk assessments has diversified over time, but patient experience and real-world data are rarely included in these assessments despite policy goals to increase their use.