Presentation (CTI)

OBJECTIVES: To review and compare the clinical evidence, differentiation versus comparators, and supporting data submitted across therapies that have received PRIME designation from the EMA, to identify transferable learnings for future products.
METHODS: A list of all EMA approvals designated as PRIME priority medicines (n=46) was extracted from the EMA website. For these drugs, the corresponding HTA assessments were downloaded from the G‑BA and NICE websites. These assessments were reviewed to evaluate the clinical evidence, PROs, ITCs, and other relevant data included in the evidence package. The evidence packages were then compared across drugs to identify key themes, differences, and resulting HTA outcomes. For the 46 drugs identified, no HTA assessments were identified in Germany and the UK for 12. For the remaining drugs, a total of 146 assessments were downloaded.
RESULTS: After removing duplicates and retaining only those aligned with the indication for which PRIME designation was awarded, 118 assessments were included in the analysis. Our findings show that the majority of drugs relied on single-arm trials and often lacked endpoints such as overall survival. Similarly, many also lacked the inclusion of PROs, and while ITCs were conducted in several cases, their acceptability was limited due to methodological limitations. Our analysis suggests that while all PRIME designated drugs in Germany were reimbursed, only 75% of were reimbursed in the UK
CONCLUSIONS: Our analysis highlights that while the EMA awards PRIME designation to drugs based on high unmet need, HTA bodies rely on the submitted evidence, which in many cases does not meet their expectations, leading to sub-optimal HTA outcomes. There is a need for regulators and HTA bodies to establish a balanced and pragmatic framework for therapies targeting high unmet need indications, in order to better guide manufacturers and ensure adequate patient access.