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Chimeric Antigen Receptor T-Cell Therapies: Barriers and Solutions to Access. ​

文献信息 ​

DOI10.1200/OP.22.00315
PMID36130152
期刊JCO oncology practice
影响因子4.6
JCR 分区Q1
发表年份2022
被引次数45
关键词嵌合抗原受体T细胞疗法, 患者获取障碍, 治疗可及性
文献类型Review, Journal Article
ISSN2688-1527
页码800-807
期号18(12)
作者Joseph Mikhael, Jessica Fowler, Nina Shah

一句话小结 ​

CAR-T疗法为重度血液肿瘤患者提供了新的治疗选择，但患者在获取治疗时面临诸多障碍，如临床试验的严格标准、高昂费用和复杂的治疗过程。文章呼吁关键利益相关者合作，采取创新措施改善患者的治疗获取机会，以应对疫情期间加剧的挑战。

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嵌合抗原受体T细胞疗法 · 患者获取障碍 · 治疗可及性

摘要 ​

嵌合抗原受体T细胞（CAR-T）疗法是针对重度预处理的血液肿瘤患者的相对新型治疗方法。虽然这些创新疗法能够为治疗选择有限的患者提供显著益处，但患者在获取治疗时仍面临障碍。传统临床试验耗时较长，并可能受到严格的患者资格标准、资源和入组名额可用性等限制。由于CAR-T治疗过程的复杂性，治疗交付可能给患者带来额外负担，包括要求患者居住在治疗中心附近，以及在输注后需与照护者共同生活。CAR-T细胞的制造在专业设施中完成，取决于试剂的可用性、制造劳动力和及时运输。CAR-T治疗费用高昂，许多美国健康计划限制对细胞和基因疗法的覆盖。由于这些障碍，现有挑战在COVID-19疫情期间更加严重。本文讨论了这些障碍，并提出了一些改善患者获取治疗的潜在解决方案，包括临床试验设计和制造的创新、治疗交付地点的选择，以及关键利益相关者对治疗和报销的看法。我们呼吁关键利益相关者群体采取行动，解决CAR-T治疗的障碍，以扩大患者的治疗获取机会。未来，关键利益相关者之间的合作，包括支付方、监管机构以及产业/学术界，将对持续解决这些障碍并增强患者对这些疗法的获取至关重要。

英文摘要 ​

Chimeric antigen receptor T-cell (CAR-T) therapies are relatively new treatments for patients with heavily pretreated hematologic malignancies. Although these innovative therapies can offer substantial benefit to patients with limited alternative treatment options, patient-access barriers exist. Conventional clinical trials are time-consuming and may be limited by strict patient eligibility criteria, resources, and availability of enrollment slots. Because of the complexity of the CAR-T administration process, treatment delivery can be associated with additional burden for the patient, including requiring patients to reside close to treatment centers and remain with a caregiver after infusion. Manufacturing of CAR-T cells is completed in specialized facilities and depends on the availability of reagents, manufacturing workforce, and timely transportation. CAR-T therapy is costly, and many US health plans restrict coverage of cell and gene therapies. Several of the existing challenges because of these barriers have been exacerbated during the COVID-19 pandemic. This review discusses these barriers and proposes some potential solutions to improving patient access, including innovation in clinical trial design and manufacturing, location of treatment delivery, and key stakeholder opinions regarding treatment and reimbursement. We propose a call to action for key stakeholder groups to address these barriers to CAR-T therapy to expand treatment access for patients. Future collaboration between key stakeholders, including payers, regulatory agencies, and industry/academia, will be critical to continue to address these barriers and enhance patient access to these therapies.

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主要研究问题 ​

  1. 在CAR-T疗法的实施过程中，如何优化患者的治疗体验以减少其负担？
  2. 针对CAR-T疗法的高成本，是否有可能通过新的支付模式或保险政策来改善患者的经济负担？
  3. 如何评估不同地区对CAR-T疗法的可及性差异，以及这些差异对患者选择治疗的影响？
  4. 在CAR-T疗法的临床试验设计中，是否有可能采用更灵活的标准以提高患者的参与率？
  5. 针对COVID-19疫情对CAR-T疗法获取的影响，未来的应对策略应包括哪些关键要素？

核心洞察 ​

  1. 研究背景和目的
    近年来，嵌合抗原受体T细胞（CAR-T）疗法作为针对重度预处理血液恶性肿瘤的创新治疗方法，受到广泛关注。尽管CAR-T疗法为患者提供了新的治疗希望，但患者获取这些疗法的障碍依然存在。本研究旨在探讨影响患者获取CAR-T疗法的主要障碍，并提出解决方案，以改善患者的治疗可及性。

  2. 主要方法和发现
    本研究通过文献回顾，分析了CAR-T疗法的临床试验设计、治疗交付过程及制造环节中的障碍。发现传统临床试验往往耗时较长，且受限于严格的患者筛选标准、资源短缺及名额限制。CAR-T细胞的生产需要高专业化的设施，依赖于原料、制造人力及及时运输。此外，治疗费用高昂以及美国许多健康保险计划对细胞和基因疗法的覆盖限制，进一步加剧了患者的获取难度。COVID-19疫情期间，相关挑战愈加明显，影响了患者的治疗机会。

  3. 核心结论
    研究认为，改善CAR-T疗法的患者获取需要创新的临床试验设计和制造流程，以及合理的治疗交付地点。同时，呼吁关键利益相关者（包括支付方、监管机构、产业界和学术界）共同努力，主动解决现存的障碍，以扩大患者的治疗选择。

  4. 研究意义和影响
    本研究强调了CAR-T疗法在治疗重度血液恶性肿瘤中的潜在价值，同时指出了影响患者获取治疗的多重因素。通过提出具体的解决方案，研究为未来CAR-T疗法的推广和患者的可及性提供了重要参考。这不仅有助于提高患者的生活质量，也为相关政策制定者和医疗服务提供者在优化治疗流程、降低治疗壁垒方面提供了切实的指导，具有重要的社会和经济意义。

引用本文的文献 ​

  1. Innovation in BCMA CAR-T therapy: Building beyond the Model T. - Rahul Banerjee;Sarah S Lee;Andrew J Cowan - Frontiers in oncology (2022)
  2. Patient Perceptions of CAR-T Therapy in the USA: Findings from In-Depth Interviews. - Todd J Bixby;Christine J Brittle;Patricia A Mangan;Edward A Stadtmauer;Lisa R Kallenbach - Oncology and therapy (2023)
  3. Real-time semantic segmentation and anomaly detection of functional images for cell therapy manufacturing. - Rui Qi Chen;Benjamin Joffe;Paloma Casteleiro Costa;Caroline Filan;Bryan Wang;Stephen Balakirsky;Francisco Robles;Krishnendu Roy;Jing Li - Cytotherapy (2023)
  4. Recent advances in cellular immunotherapy for lymphoid malignancies. - Haerim Chung;Hyunsoo Cho - Blood research (2023)
  5. An updated cost-effectiveness analysis of axicabtagene ciloleucel in second-line large B-cell lymphoma patients in the United States. - Olalekan O Oluwole;Anik R Patel;Sachin Vadgama;Nathaniel J Smith;Rob Blissett;Chaoling Feng;Michael Dickinson;Patrick B Johnston;Miguel-Angel Perales - Journal of medical economics (2024)
  6. Mosunetuzumab with polatuzumab vedotin in relapsed or refractory aggressive large B cell lymphoma: a phase 1b/2 trial. - Lihua E Budde;Adam J Olszewski;Sarit Assouline;Izidore S Lossos;Catherine Diefenbach;Manali Kamdar;Nilanjan Ghosh;Dipenkumar Modi;Waleed Sabry;Seema Naik;Amitkumar Mehta;Shazia K Nakhoda;Stephen D Smith;Kathleen Dorritie;Ting Jia;Song Pham;Ling-Yuh Huw;Jing Jing;Hao Wu;Wahib S Ead;Iris To;Connie Lee Batlevi;Michael C Wei;Julio C Chavez - Nature medicine (2024)
  7. Circulating-tumor DNA Assessment in Diffuse Large B-cell Lymphoma to Determine Up-front Stem Cell Transplantation: A Pilot Study. - Juhyung Kim;Tan Minh LE;Donghyeon Lee;Hong Duc Thi Nguyen;Hee Jeong Cho;Sang Kyun Sohn;Jong Gwang Kim;Shin-Young Jeong;Ji Yeon Ham;Ji Yun Jeong;Hyung Soo Han;Joon Ho Moon;Dong Won Baek - In vivo (Athens, Greece) (2024)
  8. Cost-effectiveness of second-line lisocabtagene maraleucel in relapsed or refractory diffuse large B-cell lymphoma. - Jee H Choe;Tianzhou Yu;Jeremy S Abramson;Mohamed Abou-El-Enein - Blood advances (2024)
  9. Engineered Adoptive T-Cell Therapies for Breast Cancer: Current Progress, Challenges, and Potential. - Diego F Chamorro;Lauren K Somes;Valentina Hoyos - Cancers (2023)
  10. Autologous stem cell transplant in fit patients with refractory or early relapsed diffuse large B-cell lymphoma that responded to salvage chemotherapy. - Aung M Tun;Yucai Wang;Seth Maliske;Ivana Micallef;David J Inwards;Thomas M Habermann;Luis Porrata;Jonas Paludo;Jose Villasboas Bisneto;Allison Rosenthal;Mohamed A Kharfan-Dabaja;Stephen M Ansell;Grzegorz S Nowakowski;Umar Farooq;Patrick B Johnston - Haematologica (2024)

... (35 更多 篇文献)

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