The Challenge

The recent years have seen a number of high-end innovative therapies becoming accessible to patients in Europe. These products offer ground-breaking new opportunities to patients living with rare diseases or cancer, with the capacity of often one-time disease-modifying and potentially curative treatments.

These Advanced Therapies Medicinal Products (ATMPs) bring new hope for the treatment of disease, pushing the boundaries of innovation, and transforming the lives of patients. Some of these therapies are already authorised in the EU, and the next years will see numerous new treatments becoming available for patients. However, there are questions around the pricing of these therapies and whether the national healthcare systems are ready to accommodate these transformative therapies.

The FIPRA Approach

Only an open and transparent debate can lay the foundation for a better understanding of ATMPs. In an effort to raise awareness of these therapies and set the foundations for an educated discussion with MEPs and stakeholders, FIPRA provides the Secretariat to an informal MEP Interest Group that consults and engages with a multi-stakeholder expert Alliance composed of patient groups, medical experts, researchers, academics, industry and other relevant decision-makers. TRANSFORM aims to foster effective dialogue and provide evidence-based policy recommendations to enable safe and timely patient access to cell and gene therapies, whilst ensuring sustainability of healthcare systems.

The Outcome

FIPRA developed a series of Policy Asks, which are the result of consensus building amongst the different stakeholder members of the Alliance:
• Policy Asks on Access to Authorised ATMPs (March 2022)
• Policy Asks on the Regulatory Framework for ATMPs (June 2022)
• Policy Asks to Optimize Cross-border Patient Access to Transformative Therapies (Nov 2021)
• TRANSFORM Recommendations for Action in the EU Pharmaceutical Strategy:
The Policy Asks were launched at public events (online or in the European Parliament), with MEPs or national policy-makers. They were summarized in the TRANSFORM MEP Charter under 7 Recommendations, which were endorsed by the 20+ MEPs members of the TRANSFORM MEP Interest Group, and will feed into the legislative review of the general pharmaceutical legislation and other dossiers (SoHO, EHDS, HTA).