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SCI

17 September 2024

Externally Controlled Studies Using Real-World Data in Patients With Hematological Cancers: A Systematic Review

(JAMA Oncology, IF: 22.5)

  • Sjoerd J. F. Hermans, MD; Niek G. van der Maas, MD; Yvette van Norden, PhD; Avinash G. Dinmohamed, BASc, MSc, PhD; Elizabeth Berkx, PhD; Peter C. Huijgens, MD, PhD; Donna R. Rivera, PharmD; R. Angelo de Claro, MD; Francesco Pignatti, MD, MSc; Jurjen Versluis, MD, PhD; Jan J. Cornelissen, MD, PhD

  • CORRESPONDENCE TO: j.cornelissen@erasmusmc.nl

IMPORTANCE 重要性

The use of real-world data (RWD) external control arms in prospective studies is increasing. The advantages, including the immediate availability of a control population, must be balanced with the requirements of meeting evidentiary standards.

在前瞻性研究中使用真实世界数据（RWD）外部对照臂的情况正在增加。这一设计的优势包括控制组的即时可用性，必须与满足证据标准的要求相平衡。

 

OBJECTIVE 目的

To address the question of whether and to what extent the methods of RWD studies compare to standard methods used in randomized clinical trials.

解决RWD研究是否以及在多大程度上与随机临床试验中使用的标准方法进行比较的问题。

 

EVIDENCE REVIEW 证据审查

A systematic search across 4 electronic databases and Google Scholar was conducted from January 1, 2000, to October 23, 2023. Studies were included in the systematic review if they compared an intervention arm in a clinical trial to an RWD control arm in patients with hematological cancers and if they were published between 2000 and 2023.

从2000年1月1日到2023年10月23日，对4个电子数据库和谷歌学术进行了系统搜索。如果研究将临床试验中的干预组与血液系统癌症患者的RWD对照组进行比较，并且这些研究是在2000年至2023年间发表的，则这些研究被纳入系统评价。

 

FINDINGS 调查结果

Thirty-two prospective intervention studies incorporating external control data from RWD sources of patients with hematological cancers were identified. A total of 4306 patients from intervention arms and 10 594 from RWD control arms were included across all studies. Only 2 studies (6%) included prospectively collected RWD. The complete trial inclusion criteria were applied to the RWD cohort in 7 studies (22%). Four studies (13%) published the statistical analysis plan and prespecified use of RWD. A total of 23 studies (72%) applied matching algorithms for trial and RWD cohorts, including matching for demographic, disease, and/or therapy-related characteristics. The end point criteria were the same as the trial in 8 studies (25%). In contrast, 12 studies (38%) used different end points, and 12 (38%) did not provide an end point definition for the RWD. Twelve studies (38%) had a median follow-up difference of less than a year between arms. Eight studies (25%) reported toxic effect data for the trial arm, of which 5 studies reported toxic effect data for the RWD arm.

确定了32项前瞻性干预研究，这些研究纳入了来自血液系统癌症患者RWD来源的外部对照数据。所有研究共纳入了干预组的4306名患者和RWD对照组的10594名患者。只有2项研究（6%）囊括了前瞻性收集的RWD。七项研究（22%）中将完整的试验纳入标准应用于RWD队列。四项研究（13%）公布了统计分析计划和预先指定使用的RWD。共有23项研究（72%）和RWD队列应用了匹配算法，包括人口统计学、疾病和/或治疗相关特征的匹配。八项研究（25%）的终点标准与试验相同。相比之下，12项研究（38%）使用了不同的终点，12项（38%）没有为RWD提供终点定义。十二项研究（38%）的中位随访差异不到一年。八项研究（25%）报告了试验组的毒性作用数据，其中5项研究报告了RWD组的毒性反应数据。

 

CONCLUSIONS AND RELEVANCE 结论和相关性

In this systematic review, limitations were observed in the application of clinical trial eligibility criteria to RWD, statistical rigor and application of matching methods, the definition of end points, follow-up, and reporting of adverse events, which may challenge the conclusions reported in studies using RWD.

在这项系统综述中，观察到在将临床试验资格标准应用于RWD、统计严谨性和匹配方法的应用、终点的定义、随访和不良事件的报告方面存在局限性，这可能会对使用RWD的研究报告结论提出质疑。