REVIEW OF HEALTH TECHNOLOGY ASSESSMENTS FOR CELL AND GENE THERAPIES IN EUROPE
Author(s)
Mark Chalmers, PhD1, Deepti Rai, BDS2, PRATEEK KANADE, M. Pharm3, Darata Zemaitaityte, Masters in Health Economics, Policy & Law4.
1Partner, EY, Dublin, Ireland, 2EY, Hyderabad, India, 3EY, BENGALURU, India, 4EY, Dublin, Ireland.
1Partner, EY, Dublin, Ireland, 2EY, Hyderabad, India, 3EY, BENGALURU, India, 4EY, Dublin, Ireland.
OBJECTIVES: To examine how major European HTA bodies (NICE, HAS, and G‑BA) appraise cell and gene therapies (CGTs), with a focus on the key challenges associated with demonstrating their clinical value
METHODS: All CGTs granted marketing authorization by the European Medicines Agency (EMA) up to May 2026 were identified and reviewed. HTA decisions across the UK, France, and Germany were subsequently analyzed. Clinical and economic evidence submissions, appraisal outcomes, and underlying decision drivers were collated and examined to identify consistent and divergent patterns across countries
RESULTS: Approximately 27 CGTs have been authorized by EMA, with around 20 achieving reimbursement in at least one European market. Despite increasing regulatory approvals, HTA outcomes vary widely across countries. Common HTA concerns include a lack of long-term evidence, uncertainty regarding durability of effect, reliance on single-arm trials, and insufficient quality-of-life data. In France, most therapies achieve moderate clinical benefit ratings; however, uncertainties often result in restricted use. In Germany, added benefit assessments are frequently limited or conditional, with full evaluations in some cases deferred. In England, access is enabled through specialized pathways, but only a subset of therapies receives routine commissioning
CONCLUSIONS: A persistent gap remains between regulatory approval and HTA acceptance for CGTs. High upfront costs, combined with unresolved long-term uncertainty, continue to challenge value demonstration and consistent patient access. Addressing these challenges will require strengthened evidence generation, greater use of post-launch data collection, and continued evolution of HTA frameworks to better accommodate one-time, potentially curative treatments. From a manufacturer perspective, early integration of payer-relevant evidence, including long-term outcomes and comparative benchmarks, will be critical to strengthening value demonstration in HTA evaluations
METHODS: All CGTs granted marketing authorization by the European Medicines Agency (EMA) up to May 2026 were identified and reviewed. HTA decisions across the UK, France, and Germany were subsequently analyzed. Clinical and economic evidence submissions, appraisal outcomes, and underlying decision drivers were collated and examined to identify consistent and divergent patterns across countries
RESULTS: Approximately 27 CGTs have been authorized by EMA, with around 20 achieving reimbursement in at least one European market. Despite increasing regulatory approvals, HTA outcomes vary widely across countries. Common HTA concerns include a lack of long-term evidence, uncertainty regarding durability of effect, reliance on single-arm trials, and insufficient quality-of-life data. In France, most therapies achieve moderate clinical benefit ratings; however, uncertainties often result in restricted use. In Germany, added benefit assessments are frequently limited or conditional, with full evaluations in some cases deferred. In England, access is enabled through specialized pathways, but only a subset of therapies receives routine commissioning
CONCLUSIONS: A persistent gap remains between regulatory approval and HTA acceptance for CGTs. High upfront costs, combined with unresolved long-term uncertainty, continue to challenge value demonstration and consistent patient access. Addressing these challenges will require strengthened evidence generation, greater use of post-launch data collection, and continued evolution of HTA frameworks to better accommodate one-time, potentially curative treatments. From a manufacturer perspective, early integration of payer-relevant evidence, including long-term outcomes and comparative benchmarks, will be critical to strengthening value demonstration in HTA evaluations
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA356
Topic
Clinical Outcomes, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Genetic, Regenerative & Curative Therapies, Personalized & Precision Medicine