Presentation (CTI)

OBJECTIVES: This study aimed to identify determinants of patient access for oncology medicines that were evaluated through the most extensive health technology process (HTA) in the Netherlands, the so-called ‘’lock procedure’’.
METHODS: This retrospective observational study included all oncology medicine-indications authorized by the European Medicines Agency (EMA) throughout 2020-2025 that entered the lock procedure. Data were extracted from EMA reports, HTA reports, and official reimbursement decisions. Variables across the reimbursement process were extracted and analyzed descriptively to identify drivers of reimbursement outcomes. A separate analysis examined the impact of revised clinical benefit criteria introduced in 2023.
RESULTS: Among 270 EMA-authorized oncology medicine-indications, 67 underwent the lock procedure, including 6 reassessments and 8 sub-indications. Forty-nine percent (33/67) achieved patient access, 37% (25/67) did not (5 failed negotiations; 20/21 negative recommendations [1 obtained conditional reimbursement]), 13% (9/67) are still under negotiation. Before revised clinical benefit criteria in 2023, negative pharmacotherapeutic conclusions represented 6% (n=1/18), compared to 35% (n=9/26) afterwards, particularly among dossiers using surrogate endpoints. Uncertain pharmacotherapeutic evidence 67%, (n=14/21), or pharmacoeconomic model uncertainty 33% (n=7/21) drove negative reimbursement recommendations. Among non-cost-effective medicine-indications, 70% (n=32/46) achieved patient access through managed entry agreements (MEAs). Negotiation timelines showed greater variability (standard deviation [SD]: 135 days) than HTA timelines (SD: 61 days). Among medicine-indications with positive pharmacotherapeutic conclusions, 45% (n=24/53) had budget impact estimates below the eligibility threshold for the lock procedure, suggesting these combinations could have undergone a less extensive HTA process.
CONCLUSIONS: Clinical benefit assessment stringency emerged as a key access barrier, with revised clinical benefit criteria increasing the proportion of medicine-indication combinations that did not meet the efficacy threshold for reimbursement. MEAs frequently mitigated unfavorable cost-effectiveness outcomes, highlighting the importance of price negotiations. Discrepancies between early horizon-scanning estimates and final budget impact assessments suggest opportunities to improve proportionality and efficiency of the reimbursement process.