Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada
BMJ Open. 2021 Aug 12;11(8):e046707. doi: 10.1136/bmjopen-2020-046707.
Chimeric antigen receptor T-cell (CAR-T) therapy is a class of immunotherapy. An economic evaluation conducted at an early stage of development of CAR-T therapy for treatment of adult relapsed or refractory acute lymphoblastic leukaemia could provide insight into factors contributing to the cost of treatment, the potential clinical benefits, and what the health system can afford. Traditionally, stakeholders are engaged in certain parts of health technology assessment processes, such as in the identification and selection of technologies, formulation of recommendations, and implementation of recommendations; however, little is known about processes for stakeholder engagement during the conduct of the assessment. This is especially the case for economic evaluations. Stakeholders, such as clinicians, policy-makers, patients, and their support networks, have insight into factors that can enhance the validity of an economic evaluation model. This research outlines a specific methodology for stakeholder engagement and represents an avenue to enhance health economic evaluations and support the use of these models to inform decision making for resource allocation. This protocol may inform a tailored framework for stakeholder engagement processes in future economic evaluation model development.
We will involve clinicians, healthcare researchers, payers, and policy-makers, as well as patients and their support networks in the conduct and verification of an early economic evaluation of a novel health technology to incorporate stakeholder-generated knowledge. Three stakeholder-specific focus groups will be conducted using an online adaptation of the nominal group technique to elicit considerations from each. This study will use CAR-T therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia as a basis for investigating broader stakeholder engagement processes.
This study received ethics approval from the Ottawa Hospital Research Institute Research Ethics Board (REB 20200320-01HT) and the results will be shared via conference presentations, peer-reviewed publications, and ongoing stakeholder engagement.
嵌合抗原受体 T 细胞（CAR-T）疗法是一种免疫疗法。在 CAR-T 疗法治疗成人复发或难治性急性淋巴细胞白血病的早期开发阶段进行经济评估，可以深入了解治疗成本、潜在临床获益以及医疗体系承受能力的因素。传统上，利益相关者参与卫生技术评估过程的某些部分，例如识别和选择技术、制定建议和实施建议；然而，对于评估过程中的利益相关者参与过程知之甚少。对于经济评估尤其如此。临床医生、政策制定者、患者及其支持网络等利益相关者深入了解可以提高经济评估模型有效性的因素。这项研究概述了一种具体的利益相关者参与方法，代表了一种增强健康经济评估和支持使用这些模型为资源分配决策提供信息的途径。本方案可能为未来经济评估模型开发中的利益相关者参与过程制定一个定制框架。
我们将邀请临床医生、医疗保健研究人员、支付者和政策制定者以及患者及其支持网络参与一项新的卫生技术的早期经济评估的进行和验证，以纳入利益相关者产生的知识。将使用在线名义小组技术的变体对三个特定于利益相关者的焦点小组进行，以从每个小组中引出考虑因素。本研究将以 CAR-T 疗法治疗成人复发或难治性 B 细胞急性淋巴细胞白血病为基础，调查更广泛的利益相关者参与过程。
这项研究获得了渥太华医院研究所研究伦理委员会（REB 20200320-01HT）的伦理批准，研究结果将通过会议演讲、同行评议出版物和持续的利益相关者参与来分享。
