AUTHOR=Berishvili Ekaterine , Piemonti Lorenzo , de Koning Eelco J. P. , Lindstedt Sandra , Scholz Hanne , Scott William E. , Auxenfans Celine , Johnson Paul , Martin Dominique E. , Gunther Penilla , Mey Devi , Potena Luciano , Thaunat Olivier TITLE=ESOT Roadmap for Advanced Therapy Medicinal Products in Transplantation: Navigating Regulatory Challenges to Enhance Access and Care JOURNAL=Transplant International VOLUME=37 YEAR=2024 URL=https://www.frontierspartnerships.org/journals/transplant-international/articles/10.3389/ti.2024.13485 DOI=10.3389/ti.2024.13485 ISSN=1432-2277 ABSTRACT=

The field of organ transplantation is experiencing a transformative shift with the rise of Advanced Therapy Medicinal Products (ATMPs), which include gene therapies, somatic cell therapies, and tissue-engineered products. These therapies offer new, potentially curative treatments for longstanding medical challenges, impacting numerous patients. However, their adoption is hindered by complex regulatory frameworks, high production costs, and inconsistent access across Europe. The ESOT ATMP Task Force’s position paper analyzes these challenges from research to clinical application, advocating for a coordinated strategy to position Europe as a leader in ATMP development. It proposes specific actions such as streamlining regulatory pathways to accelerate approvals, boosting funding for ATMP research, and creating specialized facilities for development and implementation. The paper also highlights the critical roles of patient engagement and real-world evidence in optimizing clinical and regulatory practices.