From Breakthrough to Access: Delivering ATMP Value through EU Cooperation  

Hosted by MEP Carlo Cicciolo (ECR, Italy)

The EU’s Pharmaceutical Package, currently in Trilogue negotiations, presented the opportunity for a significant shift in the EU’s positioning as a global pharmaceutical leader. While the legislative package will have a substantial impact on the competitiveness of the EU biotech sector and the future of patient access to medical innovation, multiple bottlenecks along the market access pathway for medical biotechnologies – including cell and gene therapies and other Advanced Therapy Medicinal Products (ATMPs) – remain beyond the scope of the proposal.

The European Commission’s agenda for the Life Sciences, including the Life Sciences Strategy, EU Biotech Act, and revision of the Clinical Trials Regulation, recognises the potential of life sciences innovation in driving EU competitiveness, well-being, and prosperity. As noted in the Draghi Report, post-authorisation bottlenecks limit the uptake of medical biotechnologies and, in turn, the prospect of returns on investment. The complexity of health technology assessment for ATMPs, coupled with the high degree of fragmentation among the 27 pricing and reimbursement (P&R) regimes developers must anticipate in their market entry strategies, creates significant uncertainty, places substantial administrative and financial burden on ATMP startups, and ultimately discourages innovation and European launches – with dire consequences to patient access to life-saving treatments.

Achieving the shared of objective of establishing the EU as the world’s most attractive place for life sciences by 2030 will require ambitious and coordinated actions at EU and national levels. To ensure the EU becomes a global ATMP hub, from lab to patients,

The forthcoming Biotech Acts offers EU policymakers the chance to revisit the key hurdles in market access and develop fit-for-purpose solutions to streamlined evaluation, adaptive P&R processes, and sustainable uptake across the EU. Member States have the responsibility to exchange learnings from best practices in innovative payment models and early access frameworks, in order to progress towards equitable access to health innovation for all patients.

In light of the Commission’s forthcoming proposal for the Biotech Acts, as well as Italy’s pioneering role in ATMP research and development, the TRANSFORM MEP Interest Group and Alliance convened a closed-door roundtable with policymakers from Italy.

Why a multi-stakeholder dialogue now?

2025 offers a prime opportunity for policymakers to gather input across stakeholders on the state of play of biotech innovation in the EU, measures to track progress and success in ATMP research, development and access, and the solutions that can drive forward the sector’s competitiveness while fostering a conducive environment for medical innovation.

TRANSFORM seeks to facilitate this stock-taking exercise and convey the perspective of patients, clinicians, researchers and industry. Our Alliance has recently issued a paper with our multistakeholder perspective on what tools European policymakers should leverage in upcoming proposals to meet the EU’s biotech competitiveness targets by fostering a viable market for these innovative technologies.

During this meeting, each of our members will present the barriers to the uptake of ATMPs from their respective sectoral perspectives. TRANSFORM is also keen to understand the Italian Government’s aspirations for implementing the Competitiveness Compass and how TRANSFORM can help facilitate the identification and exchange of national best practices in support for the ATMP ecosystem, such as Italy’s particular approach to a highly-integrated public-private research, development and clinical delivery ecosysem. Following the event, TRANSFORM hopes to set the groundwork for continued engagement with the Permanent Representation of Italy to the EU on shared policy priorities.

Read our event report to find out more: