Supplementary materials: Single-arm oncology trials and the nature of external controls arms

This is a peer-reviewed supplementary table for the article 'Single-arm oncology trials and the nature of external controls arms' published in the Journal of Comparative Effectiveness Research.Supplementary Table 1: Comparing cancer types of focus among the 15 example studies.Summary: Aim: Single-arm trials with external control arms (ECAs) have gained popularity in oncology. ECAs may consist of primary data from previous trials, electronic health records (EHRs) or aggregate data from the literature. We sought to provide a description of how such studies achieve similarity of patients, comparability of data quality and outcome assessment. Materials & methods: In a stratified convenience sample of 15 studies, five used primary data from trials as ECAs, five used secondary data from EHRs and five used aggregate data from the literature. Data were collected from the published literature and public web resources, blinded to the eventual approval decision. Results: Studies using ECAs from primary data and EHR data displayed methods to achieve comparability of information, including matched baseline characteristics. Aggregate data from published studies did not attempt to match covariates. The EHR controls often showed calendar time overlap for collecting information while trial data were mostly historic. Outcome data were not consistently reported across studies. US FDA approval was only seen when primary data from trials or EHR data were used as the ECA, however no ECA in this sample directly contributed to approval. Discussion: In this nonsystematic review of ECAs for single-arm trials, the ECAs derived from primary data collected by other trials or EHRs show patterns of patient comparability, time overlap, and realistic methodological approaches to achieving balance between treatment arms. They are often submitted to regulators while literature-derived aggregate findings as ECA may serve as benchmarks for pipeline decisions.

此为发表于《比较有效性研究杂志》之文章《单臂临床试验与外部对照臂的本质》的同行评审补充表格。补充表格1：比较15项示例研究中关注的癌症类型。摘要：目的：单臂临床试验配以外部对照臂（ECAs）在肿瘤学领域日益受到青睐。外部对照臂可能包括先前试验的原始数据、电子健康记录（EHRs）或文献中的汇总数据。本研究旨在描述此类研究如何实现患者相似性、数据质量可比性和结果评估。材料与方法：在15项分层便利样本研究中，其中五项采用试验的原始数据作为外部对照臂，五项采用电子健康记录的次级数据，五项采用文献中的汇总数据。数据收集自已发表的文献和公共网络资源，对最终审批决定保持盲审。结果：使用来自原始数据和EHR数据的ECAs的研究显示了实现信息可比性的方法，包括匹配基线特征。已发表研究的汇总数据并未尝试匹配协变量。电子健康记录的对照通常在收集信息时存在日历时间重叠，而试验数据则多为历史数据。结果数据在各研究中报告并不一致。美国食品药品监督管理局（US FDA）的审批仅在试验的原始数据或EHR数据用作外部对照臂时出现，然而在本样本中，没有任何ECAs直接促成审批。讨论：在本对单臂临床试验中ECAs的非系统综述中，来自其他试验或EHR收集的原始数据所导出的ECAs显示出患者可比性、时间重叠以及实现治疗臂之间平衡的现实方法学途径。它们通常提交给监管机构，而文献导出的汇总发现作为ECAs可能作为管线决策的基准。