真实世界数据：在癌症临床试验中的应用和相关性。
Real-World Data: Applications and Relevance to Cancer Clinical Trials.
发表日期：2023 Oct
作者：
Andrew J Gross, Courtney E Pisano, Chachrit Khunsriraksakul, Daniel E Spratt, Henry S Park, Yilun Sun, Ming Wang, Nicholas G Zaorsky
来源：
SEMINARS IN RADIATION ONCOLOGY
摘要：
随机对照试验（RCTs）是比较效果研究（CER）的黄金标准。自20世纪80年代以来，大型国家肿瘤数据库的建立和利用不断增加，以提供可随时访问的“真实世界数据”（RWD）。本综述文章讨论了RCT在肿瘤学中的作用以及国家肿瘤数据库中RWD在CER中的作用。RCTs仍然是CER的首选研究类型，因为它们能够最小化混杂因素和偏倚。RCTs存在一些挑战，包括耗时和资源，但这些因素不会影响结果的内部效度。RCTs的普遍性和外部效度可能存在限制。RWD非常适合研究肿瘤流行病学、治疗模式、护理差异、护理质量评估以及RCT数据在被排除在RCT之外的特定人群中的适用性。然而，具有RWD的回顾性数据库在CER中存在一些限制，因为未测量的混杂因素，并且通常无法准确确定因果治疗效应。版权所有 © 2023 Elsevier Inc. All rights reserved.
Randomized controlled trials (RCTs) are the gold standard for comparative-effectiveness research (CER). Since the 1980s, there has been a rise in the creation and utilization of large national cancer databases to provide readily accessible "real-world data" (RWD). This review article discusses the role of RCTs in oncology, and the role of RWD from the national cancer database in CER. RCTs remain the preferred study type for CER because they minimize confounding and bias. RCTs have challenges to conduct, including extensive time and resources, but these factors do not impact the internal validity of the result. Generalizability and external validity are potential limitations of RCTs. RWD is ideal for studying cancer epidemiology, patterns of care, disparities in care delivery, quality-of-care evaluation, and applicability of RCT data in specific populations excluded from RCTs. However, retrospective databases with RWD have limitations in CER due to unmeasured confounders and are often suboptimal in identifying causal treatment effects.Copyright © 2023 Elsevier Inc. All rights reserved.