Presidency event - Lithuania. Strengthening the European clinical research ecosystem for advanced therapy medicinal products and substances of human origin

This action will cover the organisation of a high-level conference by the Lithuanian presidency, focusing on strengthening the European clinical trials ecosystem with a particular emphasis on Advanced Therapy Medicinal Products (ATMPs) and Substances of Human Origin (SoHO). These innovative therapies which offer the potential for regeneration or repair, involve complex trials but are central to Europe’s ambition to become a global leader in cutting-edge medical research and patient-centred care. The decline of clinical research activity in Europe not only impacts economic competitiveness but also limits patient access to transformative therapies, especially in areas such as rare diseases, oncology, and regenerative medicine.

ATMPs and SoHO-based interventions -including cell and gene therapies, tissue-engineered products, and treatments derived from blood, plasma, or other human sources- face unique scientific, regulatory and operational challenges. These include heightened ethical considerations, complex trial logistics, and fragmented regulatory interpretations. As the EU continues to lag significantly in ATMP clinical trial activity, this event aims to build momentum for targeted action, drawing on recent EU initiatives such as the “Biotechnology and Biomanufacturing Communication”[[Commission Communication on Building the future with nature: Boosting Biotechnology and Biomanufacturing in the EU; COM(2024) 137 final: https://research-and-innovation.ec.europa.eu/document/download/47554adc-dffc-411b-8cd6-b52417514cb3_en]], the “Strategy for European Life Sciences”[[https://research-and-innovation.ec.europa.eu/strategy/strategy-research-and-innovation/jobs-and-economy/towards-strategy-european-life-sciences_en; https://ec.europa.eu/commission/presscorner/detail/en/ip_25_1686]] and the upcoming Biotech Act[[https://ec.europa.eu/info/law/better-regulation/have-your-say/initiatives/14627-Biotech-Act_en]].

This conference will bring together policymakers, regulators, academic and clinical researchers, patient representatives, and industry experts in the field of regenerative medicine to align efforts on advancing innovation, ensuring robust regulation, accelerating patient access, and fostering collaboration across the European health ecosystem.

The conference should aim to address the following goals:

• Identify key obstacles to effective implementation of ATMP and SoHO clinical research in the EU and Associated Countries, including regulatory fragmentation, variability in ethics review, manufacturing, site activation delays, funding and lack of trial-readiness.
• Highlight best practices from countries supporting innovation-friendly environments, including national strategies and reimbursement policies.
• Explore enabling infrastructures for ATMP and SoHO product development, such as academic clinical trial networks and decentralised, hospital-based manufacturing of ATMPs.
• Advance policy recommendations aimed at supporting pan-European ATMP/SoHO networks, fast-track regulatory pathways, and strategic investment in trial and manufacturing capacity and capabilities.

The outcomes of the conference will directly inform EU-level policy discussions on the future of ATMP and SoHO clinical research ecosystems, the next Research and Innovation (R&I) framework programme, the European Health Union[[https://commission.europa.eu/document/download/98c6e4dc-0fc3-4ec6-8ec2-bfcdcb2f018a_en?filename=policy_com-2024-206_en.pdf]], and the European Medicines Regulatory Network[[https://www.ema.europa.eu/en/about-us/how-we-work/european-medicines-regulatory-network]], contributing to a resilient and innovation-driven health system across the EU.

By focusing on ATMPs and SoHO-based interventions, this initiative underscores the EU’s commitment to the EU’s competitiveness as well as scientific excellence, health sovereignty, and equitable patient access to frontier therapies.

This grant will be awarded without a call for proposals according to Article 198(e) of the Financial Regulation and Article 24(3)(b) of the Horizon Europe Regulation to the legal entity identified below.

Award criteria:

The criteria are described in General Annex D. The following exceptions apply: The thresholds for each criterion will be 4 (Excellence), 4 (Impact) and 4 (Implementation). The cumulative threshold will be 12.

Procedure:

The evaluation committee will be composed fully by representatives of EU institutions.

Legal and financial set-up of the Grant Agreements:

Eligible costs will take the form of a lump sum as defined in the Decision of 7 July 2021 authorising the use of lump sum contributions under the Horizon Europe Programme – the Framework Programme for Research and Innovation (2021-2027) – and in actions under the Research and Training Programme of the European Atomic Energy Community (2021-2025)[[This decision is available on the Funding and Tenders Portal, in the reference documents section for Horizon Europe, under ‘Simplified costs decisions’ or through this link: https://ec.europa.eu/info/funding-tenders/opportunities/docs/2021-2027/horizon/guidance/ls-decision_he_en.pdf]].

The funding rate will be 100%.

Subcontracting is not restricted to a limited part of the action.