Presentation (CTI)

OBJECTIVES: Gene therapies are expanding rapidly in number and clinical scope, but the different HTA and reimbursement processes, and subsequent high price expectations create challenges for patient access and healthcare affordability (Harrison, 2023). This study compares HTA outcomes and reimbursement/funding pathways for launched gene and cell therapies across the EU4 and UK markets, and identifies key factors influencing payer decisions and patient access.
METHODS: This study identified 22 gene therapies and CAR-T cell therapies approved by the EMA and MHRA since 2017. Information was compiled on products’ indication, treatment type, orphan drug status, approval date, and key clinical trial design - collected from official HTA agencies. Outcomes were coded by category and compared descriptively across markets.
RESULTS: Overall, 86% (19/22) received a positive or conditional HTA recommendation in at least one EU4+UK market, primarily reflecting orphan-disease status and high unmet need for severely disabling conditions. At the country level, Germany approved 61% of evaluated indications (n=23), but 8.7% achieved a ‘considerable’ or ‘major’ added-benefit rating; 52.2% received a non-quantifiable hint of benefit and 39.1% were rated ‘not proven’. France showed a similar pattern (n=27), with only 3.7% achieving a top-tier ASMR/CAV II rating, while most received lower ASMR/CAV ratings or were not reimbursed. Italy was more favourable, reimbursing 86% of evaluations (n=14). Spain reimbursed 67% (n=21), with 9.5% pending confirmed reimbursement and 24% negative. The UK recommended 40% (n=25) for routine NHS use, with 28% receiving conditional access through the CDF or Managed Access Agreement.
CONCLUSIONS: The analysis shows variation in ATMP HTA outcomes across the EU4+UK markets. Italy and Spain showed stronger access, the UK was more selective, and Germany and France had low clear-positive rates but frequent conditional recognition. These findings highlight the importance of evidence maturity, comparator selection, pricing arrangements and managed access mechanisms in optimising reimbursement and accelerating patient access.