Presentation (CTI)

OBJECTIVES: Despite numerous European Medicines Agency (EMA) approvals, patient access to cell and gene therapies across Europe remains delayed - averaging 578 days per the EFPIA W.A.I.T. indicator. In the Netherlands, “lock procedure” treatments take around 600 days to become available. This study compared the number of reimbursed cell and gene indications in the Netherlands versus select European countries, analyzed reimbursement timelines, and identified key Health Technology Assessment (HTA) challenges.
METHODS: Cell and gene therapies entering the lock between January 1, 2015, and December 31, 2024, were identified. Reimbursement timelines were sourced from EMA and Ministry of Health, Welfare, and Sport (VWS) websites; HTA challenges from publicly available assessment reports.
RESULTS: 13 therapies covering 21 indications entered the lock. At data cut-off (1-5-2025), 48% of these indications were reimbursed in the Netherlands with an average time between European Commission (EC) decision and reimbursement of 759 days. Timelines differed between added (517) and equal value (1022) claims and were similar for cell (751 days) and gene (765 days) therapies. Full HTA assessments were completed in 67% of the cases. Pharmacotherapeutic challenges (i.e., single-arm trials, immature data) were present in all cases (100%). Pharmacoeconomic challenges appeared in 70%, predominantly linked to therapeutic uncertainty. Budget impact concerns were noted in 46%, often tied to uncertain patient numbers. Availability of cell and gene therapies varies significantly across the EU with Ireland (29%), the Netherlands (48%), and Denmark (48%) trailing Germany and Austria (both 95%).
CONCLUSIONS: Cell and gene therapy availability varies widely across the EU, with the Netherlands among the lowest. The requirement for long-term, published data delays submissions and patient access. While price reductions are often seen as a solution to accelerate access, this study shows that in the Netherlands, therapeutic uncertainties are central to HTA evaluations, driving downstream economic concerns, ultimately resulting in conservative price recommendations.