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

Objectives: To investigate the viability of several cross-border access opportunities to streamline patient access to advanced therapy medicinal products (ATMPs) in the context of the new Regulation on Health Technology assessment (HTA). The regulation, proposed by the European Commission and adopted in December 2021strongly advocates joint European collaboration to make innovative, life-changing therapies, such as ATMPs, more widely available to patients in Europe. ATMPsoffer ground-breaking new opportunities for the treatment of various diseases. However, healthcare systems are often required to undergo significant adaptation in terms of infrastructure development, adoption of new legislation or implementation of alternative reimbursement solutions before access to patients can be granted. This calls for new cross-border access pathways to streamline access, especially in non-EU4+UK countries. Methods: Desk research and 60-minute interviews with regulatory and payer experts were conducted to evaluate new access pathways for ATMPs. Results: Some pathways, such as cross country collaborations (e.g. joint treatment centers) have the potential to provide long-term access to ATMPs. However, several important challenges were identified that currently prevent cross-border access from being viable options for routine access to ATMPs, especially for SMEs. Conclusions: Through open communication and collaboration between manufacturers, payers and policy makers to overcome challenges associated with currently available access pathways for ATMPs, cross-border solutions could represent real opportunities for manufacturers, including SMEs, to expedite reimbursed patient access to ATMPs. Public Interest Summary: In December 2021, a new regulation was adopted on a European level to promote crossEuropean collaboration to make innovative treatments more widely available to patients. Making sure patients have access to innovative, potentially life-changing, treatments is associated with many challenges. As a result, patients, especially those who live outside of the four biggest countries in the European Union (EU), also known as the EU4: (France, Germany, Italy and Spain) and the UK where the necessary infrastructure is often lacking, can experience difficulty accessing the innovative treatments they need. We conducted interviews with experts (payers and regulators) to evaluate new pathways to make sure (non-EU4 + UK) patients get better access to innovative therapies. Collaboration and open communication between countries, manufacturers, payers and regulators presents a viable way of achieving this.