OBJECTIVES: The rising costs, expedited approvals, and negative added benefit ratings of oncology drugs have raised concerns about the alignment of regulatory and reimbursement incentives. This study aimed to evaluate the association between added benefit and revenues of oncology drugs, and to examine potential disparities in added benefit and revenues among different approval pathways of the European Medicines Agency (EMA) for these drugs.

METHODS: We identified oncology drugs and their indications approved by the EMA (1995-2020) and retrieved corresponding added benefit ratings published by seven organizations, including health technology assessment agencies from the United States, France, Germany, and Italy, along with two medical oncology societies and a medical journal. Revenue data were extracted from publicly available financial reports. We standardized the obtained added benefit ratings, compared cumulative revenues to estimated R&D costs, and explored the association between added benefit and revenues. In subgroup analyses, we distinguished between standard marketing authorization (SMA), conditional marketing authorization (CMA), and authorization under exceptional circumstances (AEC).

RESULTS: For 166 indications, 458 added benefit ratings were collected; 189 (41%) were negative/non-quantifiable. The median time to offset median R&D costs was three years; 90% of drugs recovered these costs within eight years. Drugs with higher added benefit ratings generally had greater revenues. Negative/non-quantifiable added benefit ratings were more frequent for CMAs and AECs than for SMAs (RR 1.53, 95%-CI 1.23-1.89). CMAs generated lower revenues and took longer to offset R&D costs than SMAs (four versus three years).

CONCLUSIONS: While revenues seemingly align with added benefit, most oncology drugs recover R&D costs within a few years despite providing little added benefit. This is particularly true for drugs approved through CMA, which inherently lack comprehensive evidence. Policymakers should evaluate whether current regulatory and reimbursement incentives effectively promote the best drugs for high-need patients.