OBJECTIVES: To examine the relationship between the use of surrogate measures in pivotal trials underpinning cancer drug approvals by the European Medicines Agency (EMA) between 2017 and 2022 and health technology assessment (HTA) recommendations made the National Centre for Pharmacoeconomics in Ireland (NCPE).

METHODS: A previously published methodology was used to identify cancer drug indications that received (conditional) marketing authorization between 2017 and 2022, inclusive. EMA-approved cancer drugs were categorized using the following benefit categories, based on pivotal trial endpoints: overall survival (OS); progression-free survival (PFS); disease response (DR) and single-arm trials (SATs). The NCPE website was searched to identify indications that had undergone, at least, a Rapid Review (RR) assessment. Additional data were extracted for indications that had undergone a full HTA.

RESULTS: 108 cancer drugs indications were identified, comprising of 68 unique cancer drugs. There were 14 cancer drug approvals identified in 2017, rising to 28 in 2022. In 2017, OS, PFS and SAT benefit underpinned equal proportions of approvals (28.6% each). In 2022, SAT underpinned the largest proportion of EMA cancer-drug approvals (53.6%). As of June 2023, 77 indications (71.3%) had undergone at least a RR assessment; 31 indications had completed a full HTA appraisal. All of the indications underpinned by SAT evidence (n=7) received a conditional negative recommendation; 75% of indications with PFS benefit (n=12) received a conditional negative recommendation and 66.7% of indications with an OS benefit (n=12) received a conditional negative recommendation. Indications with SAT evidence had a mean incremental QALY gain of 1.88 (SD 1.20), whereas indications with an OS benefit had a mean incremental QALY gain of 0.81 (SD 0.36).

CONCLUSIONS: The use of SATs to underpin regulatory approval of cancer drugs in the EU is increasing. This results in additional uncertainty in the comparative benefit of cancer-drugs supported by SAT evidence.