Disparities in Market Access of ATMPs: A Comparative Analysis of HTA Outcomes Across Europe
Author(s)
Steve Horsburgh, PhD1, Audrey Elaine Fulthorp, PhD1, Georgia Hollier-Hann, PhD1, Stephen Ralston, MSc2.
1Coronado Research, Newcastle upon Tyne, United Kingdom, 2Coronado Research, Guildford, United Kingdom.
1Coronado Research, Newcastle upon Tyne, United Kingdom, 2Coronado Research, Guildford, United Kingdom.
OBJECTIVES: Advanced Therapy Medicinal Products (ATMPs - gene therapies and cell/tissue therapies) offer transformative potential for treating rare and life-threatening diseases. The availability of these therapies differs between US and Europe, however, patient access to these therapies may also vary within Europe due to differences in national HTA body mechanisms and manufacturer strategies. The objective of this study was to explore disparities in approval outcomes in Europe.
METHODS: ATMPs with EMA marketing authorisation were identified via the EMA website (no time limit; all currently approved ATMPs included). Searches were conducted for HTA bodies NICE (England), G-BA (Germany), HAS (France), AIFA (Italy) and AEMPS (Spain). Key outcomes included whether an assessment was conducted, frequency of positive outcomes, time from EMA approval to HTA, and the use of conditional access mechanisms.
RESULTS: Of the 18 ATMPs currently with EMA marketing authorisation, eight are gene therapies, six are CAR-T therapies, and four are other cell therapies. AIFA assessed all 18 (100%) ATMPS, AEMPS and NICE assessed 14 (78%), G-BA assessed 12 (67%), and HAS assessed nine (50%). Nineteen HTAs have been conducted by AIFA across multiple indications, 14 (74%) resulting in reimbursement. Of the 14 ATMPs assessed by NICE, there are currently 18 HTAs; all but one resulted in a positive recommendation (94%). AEMPS have conducted 19 HTAs, with 12 (63%) resulting in funding (with restrictions). G-BA have conducted 18 HTAs, with nine (50%) resulting in ‘hint of non-quantifiable benefit’ and six (33%) resulting in ‘additional benefit not proven’. Of the 15 HTAs conducted by HAS, one resulted in an unfavourable decision.
CONCLUSIONS: These data show that reimbursement of ATMPs vastly differs amongst key EU markets, both in terms of which ATMPs have been assessed and the frequency of positive outcomes. These findings demonstrate the importance of tailored market access strategies to optimise patient access across Europe.
METHODS: ATMPs with EMA marketing authorisation were identified via the EMA website (no time limit; all currently approved ATMPs included). Searches were conducted for HTA bodies NICE (England), G-BA (Germany), HAS (France), AIFA (Italy) and AEMPS (Spain). Key outcomes included whether an assessment was conducted, frequency of positive outcomes, time from EMA approval to HTA, and the use of conditional access mechanisms.
RESULTS: Of the 18 ATMPs currently with EMA marketing authorisation, eight are gene therapies, six are CAR-T therapies, and four are other cell therapies. AIFA assessed all 18 (100%) ATMPS, AEMPS and NICE assessed 14 (78%), G-BA assessed 12 (67%), and HAS assessed nine (50%). Nineteen HTAs have been conducted by AIFA across multiple indications, 14 (74%) resulting in reimbursement. Of the 14 ATMPs assessed by NICE, there are currently 18 HTAs; all but one resulted in a positive recommendation (94%). AEMPS have conducted 19 HTAs, with 12 (63%) resulting in funding (with restrictions). G-BA have conducted 18 HTAs, with nine (50%) resulting in ‘hint of non-quantifiable benefit’ and six (33%) resulting in ‘additional benefit not proven’. Of the 15 HTAs conducted by HAS, one resulted in an unfavourable decision.
CONCLUSIONS: These data show that reimbursement of ATMPs vastly differs amongst key EU markets, both in terms of which ATMPs have been assessed and the frequency of positive outcomes. These findings demonstrate the importance of tailored market access strategies to optimise patient access across Europe.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA113
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Genetic, Regenerative & Curative Therapies, Rare & Orphan Diseases