Perspectives on the use and availability of chimeric antigen receptor T cells (CAR-T) and cell therapies: A worldwide cross-sectional survey by the worldwide network for blood and marrow transplantation (WBMT)
Year: 2025
Type: article
Study design: Observational Study
Abstract: Chimeric antigen receptor T cell therapy (CAR-T) cells represent a new generation of autologous, allogeneic and personalised cell-based therapies that have revolutionised the treatment of B cell haematological malignancies. Despite their significant effectiveness in treating challenging relapsed and refractory diseases, access to this cutting-edge treatment remains a critical issue globally, even in high income countries. To gain insights into these challenges, the Worldwide Network for Blood & Marrow Transplantation (WBMT) initiated a survey focused on the state of CAR-T and cellular therapy availability worldwide. The survey aimed to identify the accessibility, manufacturing capabilities, apheresis, accreditation, reimbursement, presence of regulatory frameworks and legal oversight of these cell-based therapies. The survey included questions on demographics, the respondent's centre, CAR-T availability, details about haematopoietic stem cell transplant programs, supply and indications for CAR-T, quality assurance, and information about other cell and gene therapy products beside CAR-T. Conducted online over three months in 2023, the survey garnered 181 complete responses from various geographical regions, from North America, Asia, Europe, South and Central America, Australia and New Zealand, and Africa. Our findings suggested a promising level of awareness and interest in CAR-T therapy globally, even in lower-income regions. However, survey respondents cited cost as the primary barrier to access, alongside infrastructure and governmental support issues. The survey also highlighted the varying reimbursement strategies across regions, with costs in Europe and North America being relatively similar while Asia showed more variability. There was also variability in the regulatory and accreditation frameworks associated with delivery of these novel therapies As CAR-T therapy continues to grow, innovative solutions such as global partnerships, in-house production, and the establishment of cellular therapy centres in developing countries are essential. Addressing the challenges of access requires a comprehensive approach that combines efforts to lower costs, enhance healthcare infrastructure, and foster international collaborations, ensuring that CAR-T therapy becomes available to all who need it.
Authors: Eddie HP Tan, Mahmoud Aljurf, Fazal Hussain, Christian Chabannon, Nina Worel, Daniel Weisdorf, Ibrahim Yakoub-Agha, Sebastian Galeano, Fermin Sanchez-Guijo, Laurent Garderet, Yoshiko Atsuta, Annalisa Ruggeri, Nada Hamad, Sharukh Hashmi, Cristobal Frutos, Yoshihisa Kodera, Adriana Seber, Carmem Bonfim, Dietger Niederwieser, Damiano Rondelli, Hildegard Greinix, Mickey BC Koh
Institutions: Health Sciences Authority, King Faisal Specialist Hospital & Research Centre, The University of Texas at San Antonio Health Science Center, Inserm, Aix-Marseille Université, Institut Paoli-Calmettes, Medical University of Vienna, University of Minnesota, Université de Lille, Centre Hospitalier Universitaire de Lille, Institute for Translational Research in Inflammation, Hospital Británico, Universidad de Salamanca, Instituto de Investigación Biomédica de Salamanca, Sorbonne Université, Pitié-Salpêtrière Hospital, Aichi Medical University Hospital, IRCCS Ospedale San Raffaele, St Vincent's Hospital Sydney, Khalifa University of Science and Technology, Sheikh Shakhbout Medical City, IPS Central, Aichi Medical University, Universidade Federal de São Paulo, Lithuanian University of Health Sciences, Leipzig University, University of Illinois Chicago, Medical University of Graz, City St George's, University of London
Language: English
Topic: CAR-T cell therapy research
Subfield: Oncology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $3,700
OpenAlex ID: W4409464248