文献检索文档翻译深度研究
Suppr Zotero 插件Zotero 插件
邀请有礼套餐&价格历史记录

新学期,新优惠

限时优惠:9月1日-9月22日

30天高级会员仅需29元

1天体验卡首发特惠仅需5.99元

了解详情
不再提醒
插件&应用
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
高级版
套餐订阅购买积分包
AI 工具
文献检索文档翻译深度研究
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2025

为嵌合抗原受体T细胞铺平道路:美国移植与细胞治疗学会80/20特别工作组关于提高商业批准的免疫效应细胞疗法临床中心认证效率及维持运营的挑战与解决方案的共识。

Paving the Road for Chimeric Antigen Receptor T Cells: American Society for Transplantation and Cellular Therapy 80/20 Task Force Consensus on Challenges and Solutions to Improving Efficiency of Clinical Center Certification and Maintenance of Operations for Commercially Approved Immune Effector Cell Therapies.

作者信息

Nikiforow Sarah, Frigault Matthew J, Frey Noelle V, Gardner Rebecca A, Komanduri Krishna V, Perales Miguel-Angel, Kebriaei Partow, Warkentin Phyllis Irene, Pasquini Marcelo, Aho Joy Lynn, Levine Bruce L, Heslop Helen E, Hlucky Tracey L, Habucky Karen, Gharibo Mecide, Jagasia Madan, Locke Frederick L

机构信息

Hematologic Malignancies, Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.

Hematopoietic Cell Transplant and Cell Therapy Program, Massachusetts General Hospital, Boston, Massachusetts.

出版信息

Transplant Cell Ther. 2023 Apr;29(4):228-239. doi: 10.1016/j.jtct.2023.01.021. Epub 2023 Jan 26.


DOI:10.1016/j.jtct.2023.01.021
PMID:36709800
Abstract

As the number and type of regulatory authority-approved cellular therapies grow, clinical treatment centers face a heavy burden of duplicative documentation around initial qualification, ongoing auditing, and reporting, with overlapping requirements from each manufacturer to ensure safe use of their specific product, which in the United States are stipulated under individual Food and Drug Administration (FDA) Biologic License Applications. The American Society for Transplantation and Cellular Therapy (ASTCT) convened the 80/20 Task Force to consider challenges and potential solutions to these issues. The Task Force proposed that 80% of manufacturers' requirements for onboarding and ongoing operations of commercially available products could be standardized and streamlined. Task Force members interviewed dozens of stakeholders, including clinicians at large academic medical centers already using commercial and investigational immune effector cell (IEC) products, regulators, members of accrediting bodies and professional cellular therapy societies, and manufacturers of IEC therapies for oncologic indications. In November 2021, the Task Force organized and led virtual discussions in a public forum and at a private ASTCT 80/20 Workshop at the online AcCELLerate Forum, a cellular-therapy stakeholders' meeting organized by the ASTCT, National Marrow Donor Program (NMDP), and Center for International Blood and Marrow Transplant Research (CIBMTR). At the workshop, approximately 60 stakeholders worked to identify and prioritize common challenges in onboarding and maintenance of operations at clinical sites for commercial FDA-approved and future IEC therapies and ways to streamline the process. It was agreed that standardization would improve efficiency of onboarding, allowing more cost-effective, sustainable growth of approved IEC therapies at treatment centers, and facilitate wider access while maintaining safety and clinical success. This early but extensive survey of stakeholders resulted in 5 overarching suggestions for both established and emerging treatment centers: (1) eliminate duplication in accreditation and auditing of clinical sites; (2) define expectations for the education about and management of CAR-T therapy toxicities to potentially replace product-specific REMS programs; (3) streamline current REMS education, testing, and data reporting; (4) standardize information technology (IT) platforms supporting enrollment, clinical site-manufacturer communication, and logistics of maintaining chain of identity/chain of custody across multiple transportation steps; and (5) encourage the use of universal nomenclature by cell therapy manufacturers. Future discussions need to engage a broader range of stakeholders, including administrators, pharmacists, nurses, data coordinators, surgeons, pathologists, and those developing promising cellular therapies for solid tumors, as well as teams from smaller academic or community cancer center settings. Continued collaboration with stakeholders outside of clinical sites will include accrediting bodies/auditors, established and emerging cell therapy companies, software developers, professional societies, and the patients who receive these therapies. Active dialog with government regulators remains essential. Such joint efforts are critical as the number of IEC therapies for myriad oncologic and nononcologic indications grows.

摘要

随着监管机构批准的细胞疗法数量和种类不断增加,临床治疗中心在初始资格认定、持续审核和报告方面面临着大量重复文档的沉重负担,每个制造商都有重叠的要求,以确保其特定产品的安全使用,在美国,这些要求在各个食品药品监督管理局(FDA)生物制品许可申请中都有规定。美国移植与细胞治疗学会(ASTCT)召集了80/20特别工作组来考虑这些问题的挑战和潜在解决方案。特别工作组提议,80%的制造商对市售产品入职和持续运营的要求可以标准化和简化。特别工作组成员采访了数十名利益相关者,包括已经在使用商业和研究性免疫效应细胞(IEC)产品的大型学术医疗中心的临床医生、监管机构、认证机构和专业细胞治疗协会的成员,以及用于肿瘤适应症的IEC疗法的制造商。2021年11月,特别工作组在一个公共论坛以及由ASTCT、国家骨髓捐赠计划(NMDP)和国际血液与骨髓移植研究中心(CIBMTR)组织的细胞治疗利益相关者会议——在线AcCELLerate论坛的ASTCT 80/20研讨会上组织并主持了虚拟讨论。在研讨会上,大约60名利益相关者努力确定并优先考虑临床场所为FDA批准的商业和未来IEC疗法进行入职和运营维护方面的常见挑战,以及简化流程的方法。大家一致认为,标准化将提高入职效率,使治疗中心已批准的IEC疗法能够实现更具成本效益、可持续的增长,并在保持安全性和临床成功率的同时促进更广泛的可及性。这项对利益相关者进行的早期但广泛的调查为既有治疗中心和新兴治疗中心提出了5条总体建议:(1)消除临床场所认证和审核中的重复;(2)明确对CAR-T疗法毒性教育和管理的期望,以潜在取代特定产品的风险评估和缓解策略(REMS)计划;(3)简化当前的REMS教育、测试和数据报告;(4)标准化支持入组、临床场所与制造商沟通以及在多个运输步骤中维护身份链/监管链物流的信息技术(IT)平台;(5)鼓励细胞治疗制造商使用通用命名法。未来的讨论需要让更广泛的利益相关者参与进来,包括管理人员、药剂师、护士、数据协调员、外科医生、病理学家,以及那些为实体瘤开发有前景的细胞疗法的人员,还有来自较小学术或社区癌症中心的团队。与临床场所之外的利益相关者持续合作将包括认证机构/审核人员、既有和新兴的细胞治疗公司、软件开发人员、专业协会,以及接受这些疗法的患者。与政府监管机构进行积极对话仍然至关重要。随着用于众多肿瘤和非肿瘤适应症的IEC疗法数量不断增加,这种联合努力至关重要。

相似文献

[1]
Paving the Road for Chimeric Antigen Receptor T Cells: American Society for Transplantation and Cellular Therapy 80/20 Task Force Consensus on Challenges and Solutions to Improving Efficiency of Clinical Center Certification and Maintenance of Operations for Commercially Approved Immune Effector Cell Therapies.

Transplant Cell Ther. 2023-4

[2]
The future of Cochrane Neonatal.

Early Hum Dev. 2020-11

[3]
ACT To Sustain: Adoptive Cell Therapy To Sustain Access to Non-Commercialized Genetically Modified Cell Therapies.

Transplant Cell Ther. 2024-8

[4]
Paediatric Strategy Forum for medicinal product development of chimeric antigen receptor T-cells in children and adolescents with cancer: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration.

Eur J Cancer. 2022-1

[5]
American Society for Blood and Marrow Transplantation Pharmacy Special Interest Group Survey on Chimeric Antigen Receptor T Cell Therapy Administrative, Logistic, and Toxicity Management Practices in the United States.

Biol Blood Marrow Transplant. 2018-9-26

[6]
Considerations for immune effector cell therapy collections: a white paper from the American Society for Apheresis.

Cytotherapy. 2022-9

[7]
Developing and Monitoring a Standard-of-Care Chimeric Antigen Receptor (CAR) T Cell Clinical Quality and Regulatory Program.

Biol Blood Marrow Transplant. 2020-8

[8]
The Why, what, and How of the New FACT standards for immune effector cells.

J Immunother Cancer. 2017-4-18

[9]
APhA 2011 REMS white paper: Summary of the REMS stakeholder meeting on improving program design and implementation.

J Am Pharm Assoc (2003). 2011

[10]
Accommodating clinical trials and other externally manufactured cellular therapy products: challenges, lessons learned and creative solutions.

Cytotherapy. 2022-1

引用本文的文献

[1]
Harnessing the promises of cell therapy, gene therapy, and regenerative medicine in Québec, Canada.

Front Med (Lausanne). 2025-6-3

[2]
High-Risk Neuroblastoma Challenges and Opportunities for Antibody-Based Cellular Immunotherapy.

J Clin Med. 2024-8-13

[3]
Development of a Safety Surveillance Plan for the Academic Medicine Sponsor Performing First-in-Human Cellular Therapy Clinical Trials: A Report from the Consortium for Pediatric Cellular Immunotherapy.

Transplant Cell Ther. 2024-5

[4]
Updates and Challenges in ENS Cell Therapy for the Treatment of Neurointestinal Diseases.

Biomolecules. 2024-2-16

[5]
Bispecific antibodies and CAR-T cells: dueling immunotherapies for large B-cell lymphomas.

Blood Cancer J. 2024-2-8

[6]
Healthcare center-based cell therapy laboratories supporting off-site manufactured cell therapies: The experiences of a single academic cell therapy laboratory.

Transfusion. 2024-2

[7]
A robust quality infrastructure is key to safe and effective delivery of immune effector cells: how FACT-finding can help.

Blood Adv. 2024-2-27

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

推荐工具

医学文档翻译智能文献检索