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The JOIN4ATMP project aims to accelerate and de-risk European ATMP development and ensure wide-spread access of ATMPs, through the mapping of obstacles to such development, the audit of real-world-based solutions and the definition of new paths forward.


EURORDIS - Eurordis

EURORDIS – Rare Diseases Europe, is a unique, non-profit alliance of over 1000 rare disease patient organisations from 74 countries that work together to improve the lives of over 300 million people living with a rare disease globally. By connecting patients, families, and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies, and patient services. Our mission is to work across borders and diseases to improve the lives of all persons living with a rare disease. This is done through the three-pronged strategy of: advocate, empower and partner. The contribution of EURORDIS has been key to the adoption of important rare disease and orphan medicine legislations at the European level, including the EU Regulation on Orphan Medicinal Products, the EU Regulation on Paediatric Drugs, the EU Regulation on Advanced Therapies, the Council Recommendation on a European action in the field of rare diseases, the EU Directive on Patients’ Rights in Cross-border Healthcare, and others. By partnering with rare disease national alliances, EURORDIS also empowers national processes, and facilitates the adoption and implementation of national plans and strategies for rare diseases in European countries.

Role within JOIN4ATMP

EURORDIS is a Core Partner and is a member of the Steering Committee and active in all 7 Work Packages and supporting tasks, bring the patient voice into the research activities and shaping the emerging findings and recommendations. EURORDIS will support mapping the regulatory challenges experienced in the application of clinical trials through to market authorisation and price reimbursement for innovative therapies for rare diseases and rare cancers. We will draw on the experience gained from the rare disease community and patient involvement in clinical trials and regulatory affairs, ensuring the hurdles in academic and SME development address the 95% of rare conditions without a treatment. Within WP1, 2 & 3, we will be involved with European regulatory frameworks are adapted for novel scientific progress and decentralized ATMP manufacture is made consistent across healthcare centres. In WP4 support JOIN4ATMP to benefit from the experience gains to assessment and pricing, to inform alternative proposal for adapted pricing and reimbursement schemes that support equitable access to novel ATMPs. In WP5 we will be involved engaging with the academic and SME developers and manufacturers, clinicians, patients & the public have an increased knowledge of the regulatory aspects and increased acceptance of ATMPs. Lastly, we will support the engagement and communication of JOIN4ATMP activities across the rare disease community.

Main contact(s)

Photo of Matt Bolz-Johnson

Matt Bolz-Johnson

Mental Health & Wellbeing Lead and Healthcare Advisor at EURORDIS, Partner