Conditional reimbursement schemes have the potential to mitigate uncertainty on duration of effect based on data available at time of regulatory approval. This approach is in use in several countries and a wider application in Europe for ATMPs is recommended.

Develop pan-European initiatives to create:

1. Real-World-Evidence infrastructure: Real-World Evidence (RWE) development is instrumental in addressing uncertainties on long-term effect, safety, health-related quality of life, and use of healthcare resources. There is a need to develop RWE infrastructure and a common framework at the European level to support long-term evidence generation and procedures to enhance the quality of evidence collected specifically for ATMPs.
2. New early-dialogue opportunities: There is a need for more opportunities for early-dialogue activities through additional EU and National funding. This would offer developers (and in particular SMEs) early insight on ways to address product specific uncertainties and how to mitigate them.
3. Timely and effective access to cross-border healthcare for all EU patients: Despite existing legislation to facilitate cross-border healthcare in Europe, there are still barriers limiting access to ATMPs as they are most often delivered through centers of excellence which are not always present at the country or regional level. In particular, there is urgent need for measures to coordinate and fund access to cross-border ATMP treatment at European level.

New payment models are needed to ensure timely patient access to innovation while preserving sustainability of healthcare systems. Without the adoption of these new models, some transformative therapies may not reach patients in some or all European countries and some may be at risk of withdrawal from the market.

Innovative access and funding arrangements include:

• Pay-for-performance
• Annuity payments
• Special funds for transformative treatments