Access and Evolving Evidence: A European HTA Analysis of Innovative Medicines Under Evidence Generation Agreements
Author(s)
Marine Mathias, PharmD1, Katarzyna Lasota, MSc2, Sanja Stanisic, MSc3, Jelena Sostar, MSc4.
1Certara, Paris, France, 2Certara, Kraków, Poland, 3Certara, Inc, Milan, Italy, 4Certara Evidence & Access, Correggio, Italy.
1Certara, Paris, France, 2Certara, Kraków, Poland, 3Certara, Inc, Milan, Italy, 4Certara Evidence & Access, Correggio, Italy.
OBJECTIVES: The EMA’s PRIME (PRIority MEdicines) designation accelerates the development of innovative therapies addressing unmet needs. However, health technology assessment (HTA) bodies often require further evidence post-approval. The aim of this analysis was to describe the HTA outcomes and evidence requirements in the 3 largest European markets: Germany, France, and the UK.
METHODS: This analysis focused on EMA PRIME-designated medicines authorized between January 2023 and April 2025, examining HTA reports from HAS (France), G-BA (Germany) and NICE (UK) to evaluate submitted evidence, key uncertainties, access conditions and post-launch data requirements.
RESULTS: A total of 9 medicines with PRIME designation were approved between January 1, 2023, and April 9, 2025, of which 7 held orphan drug designation and 6 received conditional marketing authorisation, requiring post-approval evidence generation. Completed HTA assessments were available from NICE for 6/9, from HAS for 7/9, and from G-BA for 6/9 of these medicines. In France and in the UK, 4 of 6 and 5 of 7 assessed therapies were respectively granted positive recommendation conditional to further evidence generation. In Germany, the G-BA required application-related data collection for 6 of 9 assessed medicines, though implementation has been confirmed in only one case. Across the countries, key uncertainties prompting post-launch evidence generation requirements included uncertainties around long-term effects, in particular effect durability and comparative effectiveness. HTA bodies mandated evidence with long-term follow-up, and real-world data collection over 3-5 years, focusing on clinical durability (7 of 10 evidence generation requests), safety (7/10), survival (4/10), and comparative data (6/10).
CONCLUSIONS: Manufacturers launching innovative therapies with conditional regulatory approval in Europe, should expect conditional access with reimbursement tied to post-marketing data collection. Proactive post-approval evidence generation planning and infrastructure for local patient registries is essential to support positive HTA recommendations, and patient access.
METHODS: This analysis focused on EMA PRIME-designated medicines authorized between January 2023 and April 2025, examining HTA reports from HAS (France), G-BA (Germany) and NICE (UK) to evaluate submitted evidence, key uncertainties, access conditions and post-launch data requirements.
RESULTS: A total of 9 medicines with PRIME designation were approved between January 1, 2023, and April 9, 2025, of which 7 held orphan drug designation and 6 received conditional marketing authorisation, requiring post-approval evidence generation. Completed HTA assessments were available from NICE for 6/9, from HAS for 7/9, and from G-BA for 6/9 of these medicines. In France and in the UK, 4 of 6 and 5 of 7 assessed therapies were respectively granted positive recommendation conditional to further evidence generation. In Germany, the G-BA required application-related data collection for 6 of 9 assessed medicines, though implementation has been confirmed in only one case. Across the countries, key uncertainties prompting post-launch evidence generation requirements included uncertainties around long-term effects, in particular effect durability and comparative effectiveness. HTA bodies mandated evidence with long-term follow-up, and real-world data collection over 3-5 years, focusing on clinical durability (7 of 10 evidence generation requests), safety (7/10), survival (4/10), and comparative data (6/10).
CONCLUSIONS: Manufacturers launching innovative therapies with conditional regulatory approval in Europe, should expect conditional access with reimbursement tied to post-marketing data collection. Proactive post-approval evidence generation planning and infrastructure for local patient registries is essential to support positive HTA recommendations, and patient access.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR14
Topic
Health Policy & Regulatory, Health Technology Assessment, Medical Technologies
Topic Subcategory
Approval & Labeling, Coverage with Evidence Development & Adaptive Pathways, Reimbursement & Access Policy
Disease
Genetic, Regenerative & Curative Therapies, Rare & Orphan Diseases