Fermín Sánchez-Guijo, MD, PhD.
Head, Hematology Department & Professor of Medicine, University of Salamanca, University Hospital of Salamanca, Salamanca, Spain
Vice-President, ISCT Europe and Middle East Region
Anna Sureda, MD, PhD.
Head, Hematology Department, Institut Català d’Oncologia – L’Hospitalet, IDIBELL, Universitat de Barcelona, Barcelona, Spain
President, European Group for Blood and Marrow Transplantation (EBMT)
The ever-changing field of cell and gene therapy sometimes requires a joint reflection on present and future challenges in order to better address them. This is what the executive committee of ISCT Europe and the EBMT have been doing in collaboration over the last year and a half, and the fruit of these discussions and consensus has recently been published in Cytotherapy. The manuscript addresses critical aspects such as quantification and registration of real-world cell and gene therapy activity, center accreditation, patient accessibility, regulatory challenges, and workforce development, providing some insights into the complexities of this rapidly advancing field.
We have underscored the importance of comprehensive data collection through registries, particularly the established EBMT registry, which has been instrumental in evaluating hematopoietic transplant activities. The integration of a "Cell Therapy Form" into this registry, along with plans to include other cellular and gene therapies, highlights the need for a broader understanding of real-world outcomes. Collaborative efforts with ISCT, TERMIS-EU, and others to gather data beyond hematopoietic transplantation demonstrate a commitment to comprehensive assessment.
The emphasis on accreditation, exemplified by the success of JACIE (another collaborative effort between EBMT and ISCT), illustrates the critical role of standardized procedures in ensuring patient safety. The correlation between accreditation and improved patient outcomes in hematopoietic transplant centers highlights the necessity for quality management standards across all cellular therapy processes.
The manuscript sheds light on the considerable variation in access to approved ATMPs among European countries, emphasizing the need for equitable reimbursement approaches. The concept of "outcome-based reimbursement" adopted in some European countries presents an innovative solution to ensure patient access while aligning payments with treatment outcomes. Harmonizing reimbursement policies across Europe, as advocated for by EUnetHTA, could significantly enhance access to these transformative therapies.
Challenges in commercial product accessibility, particularly highlighted in the context of CAR-T therapy, call attention to the role of academic centers in filling gaps. The discussion on Hospital Exemption as a mechanism to increase patient access to ATMPs, exemplified by experiences in Spain, points to a potential avenue for personalized therapies. The manuscript also touches upon the evolving landscape of academic product development and the need for collaboration among stakeholders.
As the European ATMPs legislation undergoes review, the manuscript calls for standardized criteria for Hospital Exemption and mandatory reporting within registries. The focus on ensuring patient safety and streamlining access within regulatory frameworks reflects a commitment to balanced oversight.
The manuscript addresses the crucial need to combat unproven therapies, emphasizing collaboration among scientific societies, regulatory bodies, and patient groups. Additionally, the discussion on workforce development highlights the anticipated surge in demand for specialized professionals within the ATMP field. Initiatives by ISCT, EBMT, and other entities to augment training programs underscore the proactive approach to meet these growing demands.
In conclusion, this manuscript serves as a roadmap for addressing the multifaceted challenges within the European cell and gene therapy landscape. The collaborative efforts of scientific societies, academic institutions, industry stakeholders, regulatory bodies, and patient groups are paramount in advancing these transformative therapies. By navigating the complexities of real-world assessment, accreditation, access, regulatory frameworks, and workforce development, the field is poised for continued growth and innovation.
As cell and gene therapy professionals, it is essential to stay informed about these challenges, as they directly impact patient care, treatment accessibility, and the future of the field. Collaboration and knowledge-sharing across scientific societies and stakeholders will be instrumental in overcoming challenges and advancing the promise of cell and gene therapies for patients across Europe and beyond.
References
1. Sanchez-Guijo F, Vives J, Ruggeri A, et al. Current challenges in cell and gene therapy: a joint view from the European Committee of the International Society for Cell & Gene Therapy (ISCT) and the European Society for Blood and Marrow Transplantation (EBMT). Cytotherapy. 2024 Feb 17:S1465-3249(24)00054-9. doi: 10.1016/j.jcyt.2024.02.007. Epub ahead of print. PMID: 38416085.