Patients at the Heart of Innovation: EURORDIS-Led Webinar Sheds Light on ATMP Development Challenges and Priorities
Within the framework of the JOIN4ATMP project, EURORDIS – Rare Diseases Europe – chaired a highly engaging and insightful webinar last week titled “Streamlining the ATMP Development Pathway in the EU”. This session placed a strong focus on the patient perspective in the evolving landscape of Advanced Therapeutic Medicinal Products (ATMPs), exploring both recent advancements and enduring obstacles in the field.
The webinar featured a panel of distinguished speakers who brought a wide array of expertise to the discussion:
- Maria Pia Cicalese – San Raffaele Telethon Institute for Gene Therapy
- Lerie Sollis – International Patient Organisation for Primary Immunodeficiencies (IPOPI)
- Silvia Martin Lluesma – Vall d’Hebron Institute of Oncology & CEU San Pablo University
Together, they explored the intersections between clinical research, ATMP development, and patient advocacy, leading to a dynamic exchange that revealed several shared challenges and priorities:
- The critical role of patient involvement in all stages of development—from study design to communication strategies and access frameworks.
- Cross-sector collaboration and innovative solutions, such as adaptive trials, centralised manufacturing models, and sustainable funding mechanisms.
- Regulatory and clinical trial complexities, including protracted approval processes, multicentre trial coordination and difficult patient recruitment.
- Improving patient access to ATMPs, particularly in cross-border settings where logistical and financial barriers remain a significant obstacle.
- The urgent call for robust EU-level funding schemes to support the development and accessibility of these high-impact, but often costly, therapies.
The session marked a significant step in promoting patient-centred approaches within the ATMP field and highlighted the essential need for policy innovation and inclusive dialogue.
Looking ahead, EURORDIS will host a follow-up webinar on 12th of May 2025 titled “Streamlining the CAR-T Development Pathway in the EU”. This session is open to all stakeholders and interested participants can register here.
Additionally, a full interview with Matt Bolz-Johnson, Team Leader at EURORDIS, is now available on the JOIN4ATMP website and LinkedIn page, offering further insight into the patient-led vision for transforming the ATMP development landscape.