Viability of European cross-border access opportunities to streamline access to ATMPs

Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries

“…The new European regulation on HTA will help shape the a landscape for ATMPs in the EU, since it stipulates that from 2025 onwards, ATMPs will be required to undergo joint clinical assessments, with the potential of significantly mitigating current differences between national comparative effectiveness assessments ( Julian et al, 2022 ; Angelillo et al, 2023 ). However, launching and filing for reimbursement and funding decisions will remain at a national level so the overall impact is difficult to assess at this stage.…”

“…The results of our study highlight considerable variation in the approaches used by individual countries to provide access to ATMPs and the scope for voluntary collaborations to overcome some of the existing barriers, particularly for smaller countries. For example, some of the options available to them include joint P&R negotiations for new medicines for demand pooling (to increase the volume), collaboration on the administration of ATMPs (through joint treatment centers), or cross-country collaboration on real-world-evidence generation ( Angelillo et al, 2023 ). There are a number of good examples of collaboration in the European region: FINOSE (Finland, Norway, Sweden), BENELUXA or the Valletta Declaration, or bilateral arrangements such as those between Malta and the United Kingdom (i.e., Malta has an agreement for hematology patients in need of an ATMP to be treated in the United Kingdom, as presented at the beginning of the results section).…”

“…The development of detailed treatment protocols (including all associated costs), and clear communication of it to stakeholders, would facilitate cross-border collaboration enabling international multidisciplinary care teams to build on existing infrastructures such as the European Reference Networks (ERNs) to deliver care and to collect evidence, which would provide a European instrument to collaborate towards mitigating uncertainties ( Angelillo et al, 2023 ). The view of patient representatives is that, although pooled procurement of ATMPs has not yet been extensively explored, it should be considered more widely ( Benvenuti et al, 2021 ).…”

“…The view of patient representatives is that, although pooled procurement of ATMPs has not yet been extensively explored, it should be considered more widely ( Benvenuti et al, 2021 ). Options suggested to boost cross-border collaboration in Europe to enhance access to ATMPs include innovative solutions that are yet to be tried, such as providing care through regional expert treatment centers ( Angelillo et al, 2023 ).…”

“…Demand pooling and pooled procurement of ATMPs has not yet been frequently used, should be considered more widely ( Benvenuti et al, 2021 ) and could facilitate evaluation, evidence generation, pricing and ultimately access in all countries due to the stronger negotiating position they would acquire, but particularly in small countries ( Angelillo et al, 2023 ).…”