Periodic Reporting for period 1 - JOIN4ATMP (MAP, JOIN AND DRIVE EUROPEAN ACTIVITIES FOR ADVANCED THERAPY MEDICINAL PRODUCT DEVELOPMENT AND IMPLEMENTATION FOR PATIENT AND SOCIETY BENEFIT)
Periodo di rendicontazione: 2024-01-01 al 2025-06-30
Advanced Therapy Medicinal Products (ATMPs) are innovative medicines made from genes, cells, or tissues. They offer new possibilities to treat – and in some cases cure – serious diseases such as rare genetic disorders, certain cancers, and immune system diseases, especially where no effective treatments exist today. However, many obstacles still limit their wider use in Europe. These include complex and differing regulations between countries, high costs, limited manufacturing capacity, and unequal access for patients.
The JOIN4ATMP project aims to speed up the safe and fair delivery of ATMPs to patients. It brings together leading experts from research, hospitals, industry, and patient organisations to identify the main barriers, gather proven solutions from real-world cases, and develop practical recommendations, guidelines, and ready-to-use procedures. By sharing this knowledge openly, JOIN4ATMP will help improve European regulations, strengthen manufacturing capabilities, and build a sustainable system so that advanced therapies can reach all patients who need them, when they need them.
The JOIN4ATMP project aims to speed up the safe and fair delivery of ATMPs to patients. It brings together leading experts from research, hospitals, industry, and patient organisations to identify the main barriers, gather proven solutions from real-world cases, and develop practical recommendations, guidelines, and ready-to-use procedures. By sharing this knowledge openly, JOIN4ATMP will help improve European regulations, strengthen manufacturing capabilities, and build a sustainable system so that advanced therapies can reach all patients who need them, when they need them.
Over the first 18 months, JOIN4ATMP has made major progress in identifying the barriers that limit the development and fair access to Advanced Therapy Medicinal Products (ATMPs) in Europe, and in creating practical solutions to overcome them.
WP1 built a robust, searchable database of ATMP preclinical and clinical trials in Europe, identifying nearly 1,000 ATMP-related studies from the EU Clinical Trials Register. This mapping highlights early-phase trials where translation to patient use is often delayed. Surveys and expert interviews revealed how preclinical testing is performed, the models used, and where improvements are possible - such as better use of alternative methods and earlier regulatory engagement. A user-friendly web application was developed to share and update this data dynamically.
WP2 studied ATMP manufacturing under Good Manufacturing Practice (GMP) across the EU, revealing challenges such as limited facility access, inconsistent regulations, and shortages of GMP-grade materials. Recommendations include harmonising GMP rules, creating an EU-wide raw material database, supporting developers, and introducing a “parent–child” regulatory approach to speed approvals. A concept for an EU-wide GMP coordination hub was also developed to improve efficiency, reduce costs, and enhance patient access.
WP3 analysed real-world regulatory and clinical hurdles through case studies and a cross-country SWOT analysis. These showed barriers such as fragmented regulations, difficulties in trial design for small patient groups, and limited comparability of products. WP3 also explored alternative financing models to help non-commercial developers bring ATMPs for rare and ultra-rare diseases to patients.
WP4 focused on the economic and evidence aspects of ATMP access. It studied how real-world data is used in decision-making, tested a financial model (risk-adjusted Net Present Value model) to help early developers assess viability, and advanced proposals for sustainable reimbursement models that link payment to patient outcomes.
WP5 mapped regulatory barriers for academic and SME developers, compared support systems in the EU and other regions, and began designing a pan-European expert platform to connect regulators, developers, clinicians, and patients. It also examined public awareness and trust in ATMPs, finding strong variation between countries, and launched high-profile webinars to start a structured dialogue with patient communities.
Together, these achievements provide the scientific evidence, regulatory insight, and policy tools needed to accelerate ATMP development and ensure that innovative therapies can reach all European patients in a safe, timely, and equitable way.
WP1 built a robust, searchable database of ATMP preclinical and clinical trials in Europe, identifying nearly 1,000 ATMP-related studies from the EU Clinical Trials Register. This mapping highlights early-phase trials where translation to patient use is often delayed. Surveys and expert interviews revealed how preclinical testing is performed, the models used, and where improvements are possible - such as better use of alternative methods and earlier regulatory engagement. A user-friendly web application was developed to share and update this data dynamically.
WP2 studied ATMP manufacturing under Good Manufacturing Practice (GMP) across the EU, revealing challenges such as limited facility access, inconsistent regulations, and shortages of GMP-grade materials. Recommendations include harmonising GMP rules, creating an EU-wide raw material database, supporting developers, and introducing a “parent–child” regulatory approach to speed approvals. A concept for an EU-wide GMP coordination hub was also developed to improve efficiency, reduce costs, and enhance patient access.
WP3 analysed real-world regulatory and clinical hurdles through case studies and a cross-country SWOT analysis. These showed barriers such as fragmented regulations, difficulties in trial design for small patient groups, and limited comparability of products. WP3 also explored alternative financing models to help non-commercial developers bring ATMPs for rare and ultra-rare diseases to patients.
WP4 focused on the economic and evidence aspects of ATMP access. It studied how real-world data is used in decision-making, tested a financial model (risk-adjusted Net Present Value model) to help early developers assess viability, and advanced proposals for sustainable reimbursement models that link payment to patient outcomes.
WP5 mapped regulatory barriers for academic and SME developers, compared support systems in the EU and other regions, and began designing a pan-European expert platform to connect regulators, developers, clinicians, and patients. It also examined public awareness and trust in ATMPs, finding strong variation between countries, and launched high-profile webinars to start a structured dialogue with patient communities.
Together, these achievements provide the scientific evidence, regulatory insight, and policy tools needed to accelerate ATMP development and ensure that innovative therapies can reach all European patients in a safe, timely, and equitable way.
JOIN4ATMP has produced important results that can directly influence how Europe develops, regulates, and delivers ATMPs. The project has created a comprehensive database of preclinical and clinical ATMP trials, mapped manufacturing and regulatory hurdles across Member States, and gathered real-world evidence from developers, regulators, and patients. These findings highlight clear needs for action to ensure wider uptake and long-term success.
Key potential impacts include:
· Faster and fairer access through harmonised Good Manufacturing Practice (GMP) rules, early regulatory dialogue, and a proposed EU-wide GMP coordination hub to improve efficiency and avoid duplication.
· Stronger innovation support for academic and SME developers via targeted advice systems, better access to GMP facilities, and flexible approval models (e.g. “parent–child” approach) that speed up similar product authorisations.
· Improved decision-making by using real-world data and performance-based payment models, helping health systems manage high-cost treatments while ensuring patient benefit.
· Enhanced public trust by promoting transparent communication, patient engagement, and inclusive policy tools.
To achieve further uptake, the project identifies key needs: sustained EU-level investment in GMP infrastructure, regulatory harmonisation across Member States, integration of regulatory and HTA evidence requirements, dedicated funding and Intellectual Property Rights (IPR) support for non-commercial developers, and policies that actively involve patients in therapy development.
With these measures, JOIN4ATMP’s outcomes can help shape an agile, innovation-friendly EU policy framework that delivers cutting-edge therapies to all patients who need them.
Key potential impacts include:
· Faster and fairer access through harmonised Good Manufacturing Practice (GMP) rules, early regulatory dialogue, and a proposed EU-wide GMP coordination hub to improve efficiency and avoid duplication.
· Stronger innovation support for academic and SME developers via targeted advice systems, better access to GMP facilities, and flexible approval models (e.g. “parent–child” approach) that speed up similar product authorisations.
· Improved decision-making by using real-world data and performance-based payment models, helping health systems manage high-cost treatments while ensuring patient benefit.
· Enhanced public trust by promoting transparent communication, patient engagement, and inclusive policy tools.
To achieve further uptake, the project identifies key needs: sustained EU-level investment in GMP infrastructure, regulatory harmonisation across Member States, integration of regulatory and HTA evidence requirements, dedicated funding and Intellectual Property Rights (IPR) support for non-commercial developers, and policies that actively involve patients in therapy development.
With these measures, JOIN4ATMP’s outcomes can help shape an agile, innovation-friendly EU policy framework that delivers cutting-edge therapies to all patients who need them.