US Food and Drug Administration, Bethesda, Maryland, USA.
OptumLabs, Cambridge, Massachusetts, USA.
Clin Pharmacol Ther. 2022 Jan;111(1):283-292. doi: 10.1002/cpt.2453. Epub 2021 Nov 11.
The purpose of this study was to evaluate the potential collective opportunities and challenges of transforming real-world data (RWD) to real-world evidence for clinical effectiveness by focusing on aligning analytic definitions of oncology end points. Patients treated with a qualifying therapy for advanced non-small cell lung cancer in the frontline setting meeting broad eligibility criteria were included to reflect the real-world population. Although a trend toward improved outcomes in patients receiving PD-(L)1 therapy over standard chemotherapy was observed in RWD analyses, the magnitude and consistency of treatment effect was more heterogeneous than previously observed in controlled clinical trials. The study design and analysis process highlighted the identification of pertinent methodological issues and potential innovative approaches that could inform the development of high-quality RWD studies.
本研究旨在通过关注肿瘤终点分析定义的一致性，评估将真实世界数据（RWD）转化为临床疗效真实世界证据的潜在机遇和挑战。在广泛的入组标准下，符合条件的接受一线治疗的晚期非小细胞肺癌患者被纳入研究以反映真实世界人群。尽管在 RWD 分析中观察到 PD-（L）1 治疗组患者的结局改善呈趋势，但治疗效果的幅度和一致性比之前在对照临床试验中观察到的更为混杂。该研究设计和分析过程突出了确定相关方法学问题和潜在创新方法的重要性，这些方法可能为高质量 RWD 研究的开展提供信息。
