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)

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...

ver descrição completa

Detalhes bibliográficos
Autores: Tan, Eddie, Aljurf, Mahmoud, Hussain, Fazal, Chabannon, Christian, Worel, Nina, Weisdorf, Daniel J., Yakoub-Agha, Ibrahim, Galeano, Sebastian, Sánchez-Guijo, Fermín M., Garderet, Laurent, Atsuta, Yoshiko, Ruggeri, Annalisa, Hamad, Nada, Hashmi, Shahruk, Frutos, Cristobal, Kodera, Yoshihisa, Seber, Adriana, Bonfim, Carmem, Niederwieser, Dietger, Rondelli, Damiano, Koh, Mickey
Formato: artículo
Estado:Versión publicada
Fecha de publicación:2025
País:España
Recursos:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:dnet:digitalcsic_::e97db8c96c5a2ec661f29d556fedc5b2
Acesso em linha:http://hdl.handle.net/10261/426305
Access Level:acceso abierto
Palavra-chave:CAR-T
Chimeric antigen T cells
Cellular therapy
Haematopoietic stem cell transplant
HSCT
id ES_8b02439cc5b15ff2984d869d799cfaaa
oai_identifier_str oai:dnet:digitalcsic_::e97db8c96c5a2ec661f29d556fedc5b2
network_acronym_str ES
network_name_str España
repository_id_str
spelling 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)Tan, EddieAljurf, MahmoudHussain, FazalChabannon, ChristianWorel, NinaWeisdorf, Daniel J.Yakoub-Agha, IbrahimGaleano, SebastianSánchez-Guijo, Fermín M.Garderet, LaurentAtsuta, YoshikoRuggeri, AnnalisaHamad, NadaHashmi, ShahrukFrutos, CristobalKodera, YoshihisaSeber, AdrianaBonfim, CarmemNiederwieser, DietgerRondelli, DamianoKoh, MickeyCAR-TChimeric antigen T cellsCellular therapyHaematopoietic stem cell transplantHSCTChimeric 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.Peer reviewedElsevierConsejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]202620262025info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Publisher's versioninfo:eu-repo/semantics/publishedVersionapplication/pdfhttp://hdl.handle.net/10261/426305reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Ingléshttps://doi.org/10.1016/j.retram.2025.103515Síinfo:eu-repo/semantics/openAccessoai:dnet:digitalcsic_::e97db8c96c5a2ec661f29d556fedc5b22026-05-22T06:33:51Z
dc.title.none.fl_str_mv 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)
title 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)
spellingShingle 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)
Tan, Eddie
CAR-T
Chimeric antigen T cells
Cellular therapy
Haematopoietic stem cell transplant
HSCT
title_short 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)
title_full 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)
title_fullStr 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)
title_full_unstemmed 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)
title_sort 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)
dc.creator.none.fl_str_mv Tan, Eddie
Aljurf, Mahmoud
Hussain, Fazal
Chabannon, Christian
Worel, Nina
Weisdorf, Daniel J.
Yakoub-Agha, Ibrahim
Galeano, Sebastian
Sánchez-Guijo, Fermín M.
Garderet, Laurent
Atsuta, Yoshiko
Ruggeri, Annalisa
Hamad, Nada
Hashmi, Shahruk
Frutos, Cristobal
Kodera, Yoshihisa
Seber, Adriana
Bonfim, Carmem
Niederwieser, Dietger
Rondelli, Damiano
Koh, Mickey
author Tan, Eddie
author_facet Tan, Eddie
Aljurf, Mahmoud
Hussain, Fazal
Chabannon, Christian
Worel, Nina
Weisdorf, Daniel J.
Yakoub-Agha, Ibrahim
Galeano, Sebastian
Sánchez-Guijo, Fermín M.
Garderet, Laurent
Atsuta, Yoshiko
Ruggeri, Annalisa
Hamad, Nada
Hashmi, Shahruk
Frutos, Cristobal
Kodera, Yoshihisa
Seber, Adriana
Bonfim, Carmem
Niederwieser, Dietger
Rondelli, Damiano
Koh, Mickey
author_role author
author2 Aljurf, Mahmoud
Hussain, Fazal
Chabannon, Christian
Worel, Nina
Weisdorf, Daniel J.
Yakoub-Agha, Ibrahim
Galeano, Sebastian
Sánchez-Guijo, Fermín M.
Garderet, Laurent
Atsuta, Yoshiko
Ruggeri, Annalisa
Hamad, Nada
Hashmi, Shahruk
Frutos, Cristobal
Kodera, Yoshihisa
Seber, Adriana
Bonfim, Carmem
Niederwieser, Dietger
Rondelli, Damiano
Koh, Mickey
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]
dc.subject.none.fl_str_mv CAR-T
Chimeric antigen T cells
Cellular therapy
Haematopoietic stem cell transplant
HSCT
topic CAR-T
Chimeric antigen T cells
Cellular therapy
Haematopoietic stem cell transplant
HSCT
description 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.
publishDate 2025
dc.date.none.fl_str_mv 2025
2026
2026
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_6501
Publisher's version
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/426305
url http://hdl.handle.net/10261/426305
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv https://doi.org/10.1016/j.retram.2025.103515

dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Elsevier
publisher.none.fl_str_mv Elsevier
dc.source.none.fl_str_mv reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC
instname:Consejo Superior de Investigaciones Científicas (CSIC)
instname_str Consejo Superior de Investigaciones Científicas (CSIC)
reponame_str DIGITAL.CSIC. Repositorio Institucional del CSIC
collection DIGITAL.CSIC. Repositorio Institucional del CSIC
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869412775656161281
score 15,81155