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从加拿大角度理解实施嵌合抗原受体 T 细胞疗法的可行性。

Understanding the Feasibility of Implementing CAR T-Cell Therapies from a Canadian Perspective.

机构信息

Graduate Student, School of Pharmacy, University of Waterloo, Waterloo, ON.

Associate Professor, School of Pharmacy, University of Waterloo, Waterloo, ON.

出版信息

Healthc Policy. 2021 Feb;16(3):89-105. doi: 10.12927/hcpol.2021.26430.

DOI:10.12927/hcpol.2021.26430
PMID:33720827
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7957352/
Abstract

In Canada, chimeric antigen receptor (CAR) T-cell therapy was recommended for funding for the treatment of select hematological cancers. Canadian hospitals have limited experience and capacity in administrating this therapy. We conducted a qualitative interview-based study with stakeholders in Canada. Questions were asked related to the development, administration, implementation and logistical planning of CAR T-cell therapy. Results were summarized into four main themes: (i) novel; (ii) patient characteristics and the delivery of care; (iii) processes from "bench-to-bedside"; and (iv) the future state, including both challenges and recommendations to ensure sustainability. Valuable perspectives from stakeholders highlight some of the unique challenges to implementing a highly personalized and expensive-to-deliver therapy.

摘要

在加拿大,嵌合抗原受体 (CAR) T 细胞疗法被推荐用于资助某些血液系统癌症的治疗。加拿大医院在管理这种疗法方面经验有限,能力有限。我们对加拿大的利益相关者进行了一项基于定性访谈的研究。问题涉及 CAR T 细胞疗法的开发、管理、实施和后勤规划。结果总结为四个主要主题:(i) 新颖性;(ii) 患者特征和护理提供;(iii) “从实验室到病床”的过程;以及 (iv) 未来状态,包括确保可持续性的挑战和建议。利益相关者的宝贵观点强调了实施这种高度个性化和昂贵的治疗方法所面临的一些独特挑战。

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本文引用的文献

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Cost and Access Implications of Defining CAR-T Therapy as a Drug.将嵌合抗原受体T细胞(CAR-T)疗法定义为药物对成本及可及性的影响
JAMA Health Forum. 2020 Jul 1;1(7):e200868. doi: 10.1001/jamahealthforum.2020.0868.
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Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries.基因疗法基于结果的实际报销情况:欧洲主要国家近期推出的嵌合抗原受体T细胞(CAR-T)疗法的经验
J Mark Access Health Policy. 2020 Jan 15;8(1):1715536. doi: 10.1080/20016689.2020.1715536. eCollection 2020.
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Systematic review protocol: an assessment of the post-approval challenges of autologous CAR-T therapy delivery.系统评价方案:自体嵌合抗原受体T细胞(CAR-T)疗法批准后实施挑战的评估
BMJ Open. 2019 Jul 4;9(7):e026172. doi: 10.1136/bmjopen-2018-026172.
4
Chimeric antigen receptor-T cell therapy manufacturing: modelling the effect of offshore production on aggregate cost of goods.嵌合抗原受体-T 细胞疗法的生产:模拟离岸生产对药品总成本的影响。
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Tisagenlecleucel in Adult Relapsed or Refractory Diffuse Large B-Cell Lymphoma.Tisagenlecleucel 治疗成人复发或难治性弥漫性大 B 细胞淋巴瘤。
N Engl J Med. 2019 Jan 3;380(1):45-56. doi: 10.1056/NEJMoa1804980. Epub 2018 Dec 1.
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Characterising and justifying sample size sufficiency in interview-based studies: systematic analysis of qualitative health research over a 15-year period.基于访谈的研究中样本量充足性的特征描述和论证:对 15 年来定性健康研究的系统分析。
BMC Med Res Methodol. 2018 Nov 21;18(1):148. doi: 10.1186/s12874-018-0594-7.
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Saturation in qualitative research: exploring its conceptualization and operationalization.定性研究中的饱和度:探索其概念化与操作化
Qual Quant. 2018;52(4):1893-1907. doi: 10.1007/s11135-017-0574-8. Epub 2017 Sep 14.
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Ther Clin Risk Manag. 2018 May 31;14:1007-1017. doi: 10.2147/TCRM.S145039. eCollection 2018.
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N Engl J Med. 2018 Feb 1;378(5):439-448. doi: 10.1056/NEJMoa1709866.