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...
| Autores: | , , , , , , , , , , , , , , , , , , , , |
|---|---|
| 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 |
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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 |
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article |
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publishedVersion |
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http://hdl.handle.net/10261/426305 |
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http://hdl.handle.net/10261/426305 |
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Inglés |
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Inglés |
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https://doi.org/10.1016/j.retram.2025.103515 Sí |
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info:eu-repo/semantics/openAccess |
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openAccess |
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application/pdf |
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Elsevier |
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Elsevier |
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reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC instname:Consejo Superior de Investigaciones Científicas (CSIC) |
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