Press release
NEW PROJECT “JOIN4ATMP”
The JOIN4ATMP project aims to accelerate and de-risk European Advanced Therapy Medicinal Product (ATMP) development and ensure wide-spread ATMP access, through the mapping of obstacles to such development, the audit of real-world-based solutions and the definition of new paths forward.
Berlin, 25 of January 2024 – Advanced Therapy Medicinal Products (ATMPs) are medicines based on genes, cells and tissues for human use that can sustainably improve or even cure diseases that currently have no or inadequate standard-of-care options. However, several hurdles remain unsolved and prevent the implementation of these innovative therapy options into effective clinical application.
JOIN4ATMP aims to accelerate European ATMP development and ensure wide-spread ATMP access while fostering the expansion of strong Good Manufacturing Practices (GMP) on a European level. To achieve this ambitious goal, the project will focus on three processes: categorizing obstacles to ATMP progress, mapping potential solutions based on real-world use cases and designing actionable recommendations. Bringing together specialists, physicians and researchers from fourteen partners based in Europe, JOIN4ATMP is coordinated by Charité - Universitätsmedizin Berlin supported by Berlin Institute of Health (BIH), and will be funded with EUR 3 million from the European Union’s Horizon Europe Framework Programme - Coordination and support action (CSA) over the next three years. JOIN4ATMP joins all members of the European University Hospital Alliance (EUHA), the existing EU-funded T2EVOLVE and RESTORE networks, Fraunhofer Gesellschaft, the European Organisation for Rare Disease Association (EURORDIS) and EURICE.
Obstacles delaying effective clinical breakthrough
Advanced Therapy Medicinal Products (ATMPs) are effective medicine alternatives presenting impactful solutions for rare genetic diseases, common immune diseases, cancer and tissue injury. Patients in the EU and associated countries should be ensured safe, efficient and equitable ATMP access. However, significant challenges remain for the scientific, clinical and patient communities as well as commercial entities and regulatory, policy-making and technology-assessing bodies.
Various initiatives in the EU and from the European Medicines Agency (EMA) are aimed to improve ATMP growth and use. The RESTORE initiative focused on the development of a sustainable pan-European ecosystem, while the T2EVOLVE consortium works towards a facilitated access to safe and effective (curative) T cell therapies and products. Finally, the EMA just launched a pilot program to support academic and not-for-profit ATMP developers during attainment of marketing authorization for their medicinal products. Yet, several regulatory, clinical, economic and ethical issues remain unsolved.
JOIN4ATMP as initiative for ATMP access facilitator
The JOIN4ATMP initiative sets out to expand ATMP access in Europe by focusing on three axes:
- a novel information package on currently existing and potential future hurdles preventing rapid and streamlined ATMP development and clinical translation
- a defined set of recommendations, including proposals for changes to laws and regulations, with concrete guidance on how to overcome these hurdles and encourage equitable ATMP access for patients throughout Europe
- a sustainable model for long-term consortium operation (GMP coordination platform and EU ATMP expert platform) beyond CSA funding to coordinate activities in Europe for this important field
Mapping obstacles and operable solutions will offer a comprehensive yet dynamic framework describing specific characteristics of ATMPs and empowering developers to energize and broaden ATMP growth and application. Compiling (pre)clinical datasets using standardised parameters and machine-readable format will increase its ease of use while improving data comparability for standard evaluation of new methods and models. JOIN4ATMP will publish its findings in journals and disseminate them in meetings and conferences.
The project aims to foster ATMP access and implementation in clinical practices, significantly reducing predictable (long-term treatment) costs and overall lighting up the burden put on healthcare systems. In 2022, only 16 of the globally initiated 144 clinical ATMP trials in 2022 were conducted in Europe. ATMPs are inherently more expensive to produce, putting pressure on healthcare budgets and making sustainable ATMP reimbursement a major challenge. Thanks to models and tools established within the project, JOIN4ATMP will allow scientists to estimate ATMP value and cost of development, increasing the likelihood of successful market entry. The facilitated ATMP clinical transfer will eventually participate in the alleviation of socio-economic costs for society thanks to an earlier treatment of ill patients inducing an accelerated return to work.
Through early stakeholder interaction, well-informed ATMP developers and harmonized approval procedures, the time to market for new ATMP products will shorten without sacrificing safety and efficacy of the therapies. Standardization in ATMP production and application will ensure patient safety and ATMP quality, while harmonised and regulatory frameworks will strengthen EC policy implementation and health equity around Europe.
“Advancements in European ATMPs represent a progressively significant domain that will shape the landscape of medical and research sectors in Europe. Through the JOIN4ATMP project, we want to give to the European society an easier access to efficient and safe treatment options to specific diseases.”, says Prof. Dr. med. Annette Künkele-Langer, Pediatric Oncologist and ATMP developer at Charité - Universitätsmedizin Berlin. “Working collaboratively with 14 partners around nine European countries, we aim to provide structured and standardized data while offering actionable solutions to overcome existing barriers in the efficient development of ATMPs in Europe.”
The JOIN4ATMP consortium holds its Kick-off meeting in Berlin on January 25-26, 2024.
Key Facts
- Title
Map, join and drive European activities for advanced therapy medicinal product development and implementation for the benefit of patients and society (JOIN4ATMP)
- Start
1 January 2024
- Duration
36 months
- Budget
2,999,958.75 €
- Coordinator
Charité – University Hospital Berlin
Website: www.JOIN4ATMP.eu
Social Media: LinkedIn, X (formerly Twitter)
Project Partners
- Austria
- - University Hospital AKH Wien & MedUni Wien (MUV)
- Belgium
- - Katholieke Universiteit Leuven (UZL)
- Denmark
- - Aarhus University Hospital (AUH)
- France
- - Assistance Publique – Hôpitaux de Paris (AHP)
- European Organisation for Rare Diseases Association (EURORDIS) - Germany
- - Charité – University Hospital Berlin (CHARITE)
- European Research and Project Office GmbH (EURICE)
- Fraunhofer Gesellschaft zur Förderung der Angewandten Forschung e.V (FHOFER)
- University Hospital Würzburg (UKW) - Italy
- - Fondazione Telethon ETS (FTELE)
- Università Vita Salute San Raffaele (UNISR) - Netherlands
- - Erasmus Universitair Medisch Centrum Rotterdam (ErasmusMC)
- Spain
- - Vall d’Hebron Institute of Oncology (VHIO)
- Sweden
- -Region Stockholm - Karolinska University Hospital (KUH)
Contact
Project Coordination
Charité – University Hospital Berlin
Annette Künkele
Email
Project Management
Eurice GmbH
Lorena Consolino
Email