Reimbursement Challenges for ATMPs in EU5: Learnings From Product Withdrawals and Implications for Future Development of Innovative Therapies
Author(s)
Viktor Danev, BSc MSc1, Gerdi Strydom, MBA, PharmD1, Anna L. McCormick, DPhil1, Stephen Hall, DPhil1, Ines Oliveira, BSc MSc2.
1Valid Insight (part of the Bioscript group), Macclesfield, United Kingdom, 2Valid Insight (Bioscript Group), Macclesfield, United Kingdom.
1Valid Insight (part of the Bioscript group), Macclesfield, United Kingdom, 2Valid Insight (Bioscript Group), Macclesfield, United Kingdom.
OBJECTIVES: The objective of this study was to analyse the pricing and reimbursement (P&R) status of all advanced therapy medicinal products (ATMP) approved by the European Medicines Agency (EMA) to date, identify the main reasons for ATMP withdrawal from the European market, and determine the key implications for manufacturers in the future.
METHODS: A targeted search was conducted to capture all EMA-approved ATMPs from October 2009 to June 2025. EMA and health technology assessment (HTA) reports were retrieved to analyse ATMP marketing authorisation and reimbursement status, HTA outcomes, pricing, and contractual agreements with public health insurers in EU5. Press releases and company position statements were reviewed to identify reasons for ATMP discontinuations in Europe.
RESULTS: The EMA approved 29 ATMPs from 2009 to 2025. Nine (31.0%) have withdrawn their marketing authorisation for different reasons: Of them, five (55.6%) were discontinued due to a combination of commercial and reimbursement challenges, two (22.2%) due to commercial reasons alone, and the remaining two (22.2%) because of clinical concerns. The majority of the ATMPs are reimbursed across the EU5 markets; however, there are restrictions and managed entry agreements (MEAs) in place for many. Manufacturers often need to give substantial confidential discounts on their ATMPs to secure public reimbursement.
CONCLUSIONS: Manufacturers of ATMPs need to overcome regulatory and reimbursement challenges to launch their products on the European market. ATMPs are usually associated with significant price tags compared to chronic therapies and high uncertainty, making payer decision-making difficult. Hence, these products are often reimbursed conditionally pending additional data collection or with financial- or outcome-based MEAs in place. P&R outcomes are not always commercially viable for manufacturers, and as a result, some withdraw their products from the European market. It is yet to be seen if the joint clinical assessment (JCA) will improve patient access to ATMPs in Europe.
METHODS: A targeted search was conducted to capture all EMA-approved ATMPs from October 2009 to June 2025. EMA and health technology assessment (HTA) reports were retrieved to analyse ATMP marketing authorisation and reimbursement status, HTA outcomes, pricing, and contractual agreements with public health insurers in EU5. Press releases and company position statements were reviewed to identify reasons for ATMP discontinuations in Europe.
RESULTS: The EMA approved 29 ATMPs from 2009 to 2025. Nine (31.0%) have withdrawn their marketing authorisation for different reasons: Of them, five (55.6%) were discontinued due to a combination of commercial and reimbursement challenges, two (22.2%) due to commercial reasons alone, and the remaining two (22.2%) because of clinical concerns. The majority of the ATMPs are reimbursed across the EU5 markets; however, there are restrictions and managed entry agreements (MEAs) in place for many. Manufacturers often need to give substantial confidential discounts on their ATMPs to secure public reimbursement.
CONCLUSIONS: Manufacturers of ATMPs need to overcome regulatory and reimbursement challenges to launch their products on the European market. ATMPs are usually associated with significant price tags compared to chronic therapies and high uncertainty, making payer decision-making difficult. Hence, these products are often reimbursed conditionally pending additional data collection or with financial- or outcome-based MEAs in place. P&R outcomes are not always commercially viable for manufacturers, and as a result, some withdraw their products from the European market. It is yet to be seen if the joint clinical assessment (JCA) will improve patient access to ATMPs in Europe.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA284
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Genetic, Regenerative & Curative Therapies