Canadian Centre for Applied Research in Cancer Control (CC-ARCC), BC Cancer, Vancouver, British Columbia, Canada; Faculty of Health Sciences, Simon Fraser University, Burnaby, British Columbia, Canada.
Canadian Centre for Applied Research in Cancer Control (CC-ARCC), BC Cancer, Vancouver, British Columbia, Canada; School of Population and Public Health, University of British Columba, Vancouver, British Columbia, Canada.
J Cancer Policy. 2021 Dec;30:100304. doi: 10.1016/j.jcpo.2021.100304. Epub 2021 Sep 2.
Chimeric antigen receptor T-cell (CAR-T) therapy represents a novel approach to cancer treatment, particularly advanced cancer. Much of the current evidence for the effectiveness of these therapies is associated with considerable uncertainty. This uncertainty poses a challenge for decision-makers and health systems responsible for granting patients access to these therapies. While the stage of development of the technology is a component of this uncertainty, it can be reduced with relevant data collection alongside clinical trials that is meaningful to patients and decision-makers. The objective of this research was to investigate the frequency with which HRQoL data is collected in currently registered clinical trials investigating CAR-T cancer treatment.
We searched for current CAR-T clinical trials at a registry compiled at United States National Institutes of Health National Library of Medicine (NLM) database. Trials were required to be active, recruiting, or completed. Trials were required to be phase I-IV, listed as 'interventional', and specific to cancer treatment.
There were 424 clinical trials that were included in our analysis. The majority of these trials (76 %) were investigating CAR-T therapy in haematological malignancies. Of the included studies, only 29 (6.8 %) included HRQoL as a primary or secondary outcome measure. Only 25 (5.9 %) trials reported collecting data on overall survival.
This investigation into clinical trials for CAR-T therapies has shown a failure to collect valuable HRQoL data. Sponsors of clinical trials need to appreciate that clinical trials for novel therapies need to collect relevant data that is paramount to informing decision-making and providing access to patients.
The effectiveness of innovative cancer therapies, such as CAR-T, remains associated with considerable uncertainty. This uncertainty can be reduced for decision-makers via the collection of critical HRQoL data. Sponsors of clinical trials should be incentivized to collect these data, particularly where the intention is to use that trial for a reimbursement submission and decision..
嵌合抗原受体 T 细胞（CAR-T）疗法代表了一种治疗癌症的新方法，尤其是治疗晚期癌症。这些疗法的有效性的大部分现有证据都存在很大的不确定性。这种不确定性给负责批准患者使用这些疗法的决策者和医疗系统带来了挑战。虽然该技术的开发阶段是这种不确定性的一个组成部分，但通过在临床试验中收集与患者和决策者都相关的有意义的数据，可以降低这种不确定性。本研究的目的是调查目前正在注册的调查 CAR-T 癌症治疗的临床试验中收集 HRQoL 数据的频率。
我们在美国国立卫生研究院国家医学图书馆（NLM）数据库中搜索了当前的 CAR-T 临床试验。试验必须是活跃的、正在招募或已完成。试验必须是 I-IV 期，列为“干预性”，并专门用于癌症治疗。
有 424 项临床试验被纳入我们的分析。这些试验中大多数（76%）是在血液恶性肿瘤中研究 CAR-T 疗法。在所纳入的研究中，只有 29 项（6.8%）将 HRQoL 作为主要或次要结局指标。只有 25 项（5.9%）试验报告收集了总生存数据。
对 CAR-T 疗法临床试验的调查表明，未能收集有价值的 HRQoL 数据。临床试验的赞助商需要认识到，新型疗法的临床试验需要收集相关数据，这对于告知决策和为患者提供治疗至关重要。
CAR-T 等创新癌症疗法的有效性仍然存在很大的不确定性。决策者可以通过收集关键的 HRQoL 数据来降低这种不确定性。临床试验的赞助商应受到激励，收集这些数据，特别是在打算将该试验用于报销申请和决策的情况下。
