Harvey Catherine, Woodcock Ashley, Vestbo Jørgen, Crim Courtney, Frith Lucy, Bakerly Nawar Diar, New John P, Williams Claire, Elkhenini Hanaa, Majeed Nasir, Cardwell Glenn, Collier Susan, Jacques Loretta, Fletcher Joanne
GlaxoSmithKline plc, Uxbridge, Middlesex, UK.
Manchester Academic Health Sciences Centre, The University of Manchester, Manchester, UK.
ERJ Open Res. 2021 May 31;7(2). doi: 10.1183/23120541.00966-2020. eCollection 2021 Apr.
Evidence to support clinical decision making must be based on safety data that have been captured, analysed and interpreted in a robust and reliable way. Randomised real-world evidence (RRWE) studies provide the opportunity to evaluate the use of medicines in patients and settings representative of routine clinical practice. However, elements that underpin the design of RRWE studies can have a significant impact upon the analysis, interpretation and implications of safety data. In this narrative review, we use data from the Salford Lung Study; two prospective, 12-month, open-label, parallel-group, phase III randomised controlled trials conducted in primary care in the UK; to highlight the importance of capturing treatment modifications when attempting to evaluate safety events according to actual treatment exposure. We demonstrate that analysing safety data by actual treatment received ( accounting for the treatment modifications that occur routinely in the primary care setting) provides additional insight beyond analysing according to randomised treatment strategy only. It is therefore proposed that understanding of safety data from RRWE trials can be optimised by analysing both by randomised group and by actual treatment received.
支持临床决策的证据必须基于以稳健可靠的方式收集、分析和解读的安全数据。随机真实世界证据（RRWE）研究提供了在代表常规临床实践的患者和环境中评估药物使用情况的机会。然而，RRWE研究设计的基础要素可能会对安全数据的分析、解读及意义产生重大影响。在这篇叙述性综述中，我们使用了索尔福德肺部研究的数据；这是在英国初级医疗中进行的两项为期12个月的前瞻性、开放标签、平行组III期随机对照试验；以突出在根据实际治疗暴露评估安全事件时记录治疗调整的重要性。我们证明，按实际接受的治疗分析安全数据（考虑到初级医疗环境中常规发生的治疗调整）比仅按随机治疗策略分析能提供更多见解。因此，建议通过按随机分组和按实际接受的治疗进行分析来优化对RRWE试验安全数据的理解。
