Real-World Data: Applications and Relevance to Cancer Clinical Trials

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-ofcare 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.(c) 2023 Elsevier Inc. All rights reserved.

Automated summary

This article discusses the role of randomized controlled trials (RCTs) in oncology and the use of real-world data (RWD) from national cancer databases in comparative-effectiveness research (CER). RCTs remain the preferred study type for CER due to their ability to minimize confounding and bias. However, RCTs also have limitations in terms of generalizability and external validity. RWD is valuable for studying cancer epidemiology, care patterns, disparities, and applicability of RCT data in specific populations. Retrospective databases with RWD, though, may not accurately identify causal treatment effects in CER.