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) - 15/04/25

Doi : 10.1016/j.retram.2025.103515

Eddie HP Tan ¹, Mahmoud Aljurf ², Fazal Hussain ³, Christian Chabannon ⁴, Nina Worel ⁵, Daniel Weisdorf ⁶, Ibrahim Yakoub-Agha ⁷, Sebastian Galeano ⁸, Fermin Sanchez-Guijo ⁹, Laurent Garderet ¹⁰, Yoshiko Atsuta ¹¹, Annalisa Ruggeri ¹², Nada Hamad ¹³, Sharukh Hashmi ¹⁴, Cristobal Frutos ¹⁵, Yoshihisa Kodera ¹⁶, Adriana Seber ¹⁷, Carmem Bonfim ¹⁸, Dietger Niederwieser ¹⁹, Damiano Rondelli ²⁰, Hildegard Greinix ²¹, Mickey BC Koh ^22,^⁎
¹ Cell and Gene Therapy Facility, Health Sciences Authority Singapore HSA, Singapore
² King Faisal Specialist Hospital and Research Centre, Riyadh, KSA
³ Department of Medicine, University of Texas Health Science Center San Antonio, Texas USA
⁴ Institut Paoli-Calmettes, Inserm CBT-1409 & Aix-Marseille Université, Marseille, France
⁵ Department of Transfusion Medicine and Cell Therapy, Medical University of Vienna, Vienna, Austria
⁶ Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis, MN, USA
⁷ Centre Hospitalier Universitaire de Lille, Université de Lille, INSERM U1286, Lille, France
⁸ Hospital Britanico, Montevideo, Uruguay
⁹ Hematology Department, IBSAL-University Hospital of Salamanca and University of Salamanca, Salamanca, Spain
¹⁰ Sorbonne Université, Hôpital Pitié Salpêtrière, Service d'hématologie, APHP, Paris, France
¹¹ Center for Hematopoietic Stem Cell Transplantation, Aichi Medical University Hospital, Nagakute, Japan
¹² San Raffaele Scientific Institute, Milano, Italy
¹³ Australasian Bone Marrow Transplant Recipient Registry (ABMTRR), St. Vincent´s Hospital Sydney, Australia
¹⁴ Khalifa University, Department of Medicine, SSMC, Abu Dhabi, and College of Medical and Health Sciences, Abu Dhabi, United Arab Emirates
¹⁵ Instituto de Prevision Social, Asuncion, Paraguay
¹⁶ Department of Promotion for Blood and Marrow Transplantation, Aichi Medical University School of Medicine, Nagakute, Japan
¹⁷ Department of Medicine, Universidad de Federal de Sao Paulo Escola Paulista de Medicina, Sao Paulo, Brazil
¹⁸ Pele Pequeno Principe, Research Institute/Pediatric Blood and Marrow Transplantation Program Hospital Pequeno Principe, Curitiba, Brazil
¹⁹ University of Leipzig, Leipzig, Germany; Aichi Medical University School of Medicine, Nagakute, Japan; Lithuanian University of Health Sciences, Kaunas, Lithuania
²⁰ University of Illinois, Chicago, Illinois, USA
²¹ Division of Hematology, Medical University of Graz, Austria
²² Dept of Haematology, St George's University Hospital and City St George's, University of London, United Kingdom; Cell Therapy Facility, Blood Services Group, Health Sciences Authority, Singapore

^⁎Corresponding Author.

Sous presse. Manuscrit accepté. Disponible en ligne depuis le Tuesday 15 April 2025

Highlights

•	Survey revealed global CAR-T therapy awareness, but potential bias exists.
•	Cost, need for infrastructure and lack of government support limit CAR-T access.
•	Improvements needed on global reimbursement strategies and regulatory frameworks.
•	Global partnerships and local capability development key to universal CAR-T access.

Le texte complet de cet article est disponible en PDF.

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.

Le texte complet de cet article est disponible en PDF.