This paper is only available as a PDF. To read, Please Download here.
Highlights:
- What is already known about the topic?
- •Advanced therapy medicinal products (ATMPs) pose a major challenge for reimbursement decisions.
- What does the paper add to existing knowledge?
- •An updated review on the financing considerations for the approved ATMPs in eight EU countries.
- •Most ATMPs were reimbursed with differences in how the magnitude of added therapeutic benefit is considered among countries.
- •Most ATMPs were associated with high costs, requiring managed entry agreements and taking 9-17 months to HTA recommendation.
- What insights does the paper provide for informing healthcare-related decision making?
- •Harmonisation of cost-effectiveness analysis and added therapeutic value criteria could lead to an aligned access and EU market attractiveness.
Abstract
Objectives
The uncertainty on cost-benefit of advanced therapy medicinal products (ATMPs) is being a current challenge for their reimbursement in health systems. The aim of this study was to provide a comparative analysis of the national health authorities (NHAs) reimbursement recommendations issued in different European countries. Methods: The NHA reimbursement recommendations for the approved ATMPs were compared among 8 European Countries (EU8: Ireland, England/Wales, Scotland, Netherlands, France, Germany, Spain and Italy). The search was carried out until December 31st, 2021. Results: Nineteen approved ATMPs and 76 appraisal reports were analysed. The majority of the ATMPs were reimbursed, although with uncertainty on added therapeutic value. No relationship between the type of the European Medicines Agency approval and reimbursement was found. Managed entry agreements such as payment by results were necessary to ensure market access. The main issue during the evaluation was to base the cost effectiveness analyses on assumptions due to the limited long-term data. The estimated incremental cost-effectiveness ratio among countries reveals high variability. Overall, the median time to NHA recommendation for the EU8 is in the range of 9-17 months.
Conclusions
Transparent, harmonised and systematic assessments across the EU NHAs in terms of cost-effectiveness, added therapeutic value, and grade of innovativeness are needed. This could lead to a more aligned access, increasing the EU market attractiveness, and raising public fairness in terms of patient access and pricing.
Keywords
Article info
Publication history
Accepted:
December 27,
2022
Received in revised form:
December 1,
2022
Received:
May 28,
2022
Publication stage
In Press Accepted ManuscriptFootnotes
Author Contributions Statement
Concept and design: Carolina Iglesias-López (C.I-L.), Antònia Agustí (A.A.), Antonio Vallano (A.V.), Mercè Obach (M.O.)
Acquisition of data: C.I-L.
Analysis and interpretation of data, C.I-L., A.A., A.V., M.O.
Drafting and revising the manuscript, C.I-L.
Critical revision of the paper for important intellectual content: C.I-L., A.A., A.V., M.O.
All authors have approved the final article.
Conflict of Interest
The authors have nothing to disclose.
Funding/Support
This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.
ATMPs are reimbursed, in spite of some uncertainty on their efficacy, with high costs requiring managed entry agreements frequently
Acknowledgments
None
Identification
Copyright
© 2023 International Society for Pharmacoeconomics and Outcomes Research, Inc. Published by Elsevier Inc.
User license
Creative Commons Attribution – NonCommercial – NoDerivs (CC BY-NC-ND 4.0) | How you can reuse 
Elsevier's open access license policy
Creative Commons Attribution – NonCommercial – NoDerivs (CC BY-NC-ND 4.0)
Permitted
For non-commercial purposes:
- Read, print & download
- Redistribute or republish the final article
- Text & data mine
- Translate the article (private use only, not for distribution)
- Reuse portions or extracts from the article in other works
Not Permitted
- Sell or re-use for commercial purposes
- Distribute translations or adaptations of the article
Elsevier's open access license policy
