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.

随机对照试验(RCT) 是比较有效性研究 (CER) 的黄金标准。自 20 世纪 80 年代以来，大型国家癌症数据库的创建和利用不断增加，以提供易于访问的“真实世界数据”(RWD)。这篇综述文章讨论了 RCT 在肿瘤学中的作用，以及国家癌症数据库中 RWD 在 CER 中的作用。RCT 仍然是 CER 的首选研究类型，因为它们可以最大限度地减少混杂因素和偏倚。随机对照试验的实施面临挑战，包括大量的时间和资源，但这些因素不会影响结果的内部有效性。普遍性和外部有效性是随机对照试验的潜在局限性。RWD 非常适合研究癌症流行病学、护理模式、护理提供的差异、护理质量评估以及 RCT 数据在 RCT 之外的特定人群中的适用性。然而，由于未测量的混杂因素，RWD 回顾性数据库在 CER 方面存在局限性，并且在识别因果治疗效果方面往往不是最佳的。