Authors: Marlies Boeren, Maud Brijs, Kristof Theys, Ben Barron-Millar, Malcolm Boyd, Malte Glüsen, Ingo Hantke, Priscila Mazzola, Melanie van Riemsdijk
Tuesday 11th Nov 2025
Poster Presentation: 10:30 - 13:30
Discussion Period: 11:30 - 12:15
OBJECTIVES
ATMPs have the potential to provide significant clinical benefits, and potentially cure, for patients with debilitating and life-threatening diseases. However, access to ATMPs across Western Europe remains unequal. With the introduction of Joint Clinical Assessments (JCA), this research aims to explore whether this initiative can help reduce access disparities by identifying both country-specific and cross-national factors that influence successful reimbursement and those contributing to access disparities across different countries.
METHODS
For EU-authorized ATMPs, national HTA assessments and outcomes were analyzed by screening HTA databases from Belgium, the Netherlands, France, Germany, England, Ireland, and Spain. The analysis involves qualitative in-depth review of the submitted evidence and HTA outcomes both within and across the listed countries.
RESULTS
A total of 28 ATMPs have received EU marketing authorization from 2009 until May 2025, of which 8 have been withdrawn or not renewed. Among the countries investigated, availability is highest in Germany (n=17) and lowest in Ireland (n=5). Despite the similarity of the submitted clinical evidence, reimbursement outcomes varied significantly across countries. Disparities in access to ATMPs were driven by factors such as the availability of standard of care, the appraisal of clinical evidence, and flexibility in financial arrangements. Cross-country access was particularly successful for indications with a high unmet need, where no targeted treatment alternatives were available, regardless of price.
CONCLUSIONS
Reimbursement outcomes are determined by the local availability of standard of care, the appraisal of clinical evidence, and the flexibility of financial arrangements. Consequently, a streamlined relative effectiveness assessment through JCA alone is unlikely to fully address access disparities, as appraisal occurs at the national level. Therefore, additional measures at the country level are necessary to ensure faster and equitable access to ATMPs, in alignment with the objectives of the HTA regulation.
Who will be attending?
Marlies Boeren
Senior Medical and Market Access Manager
Maud Brijs
Medical & Market Access Manager
Kristof Theys
Health Economics & Data Science Manager
Ben Barron-Millar
Senior Consultant, Value Communication
Malcolm Boyd
Consultant, Policy and Public Affairs
Malte Glüsen
Senior Research Analyst
Ingo Hantke
Director Market Access
Priscila Mazzola
Director, Head of Value Communication
