Conventional cancer clinical trials can be time-consuming and expensive, often yielding results with limited applicability to real-world scenarios and presenting challenges for patient participation. Real-world data (RWD) studies offer a promising solution to address evidence gaps and provide essential information about the effects of cancer treatments in real-world settings. The distinction between RWD and data derived from randomized clinical trials lies in the method of data collection, as RWD by definition are obtained at the point of care. Experimental designs resembling those used in traditional clinical trials can be utilized to generate RWD, thus offering multiple benefits including increased efficiency and a more equitable balance between internal and external validity. Real-world data can be utilized in the field of pharmacovigilance to facilitate the understanding of disease progression and to formulate external control groups. By utilizing prospectively collected RWD, it is feasible to conduct pragmatic clinical trials (PCTs) that can provide evidence to support randomized study designs and extend clinical research to the patient’s point of care. To ensure the quality of real-world studies, it is crucial to implement auditable data abstraction methods and develop new incentives to capture clinically relevant data electronically at the point of care. The treatment landscape is constantly evolving, with the integration of front-line immune checkpoint inhibitors (ICIs), either alone or in combination with chemotherapy, affecting subsequent treatment lines. Real-world effectiveness and safety in underrepresented populations, such as the elderly and patients with poor performance status (PS), hepatitis, or human immunodeficiency virus, are still largely unexplored. Similarly, the cost-effectiveness and sustainability of these innovative agents are important considerations in the real world.
传统的癌症临床试验往往耗时且成本高昂，其研究结果在真实世界场景中的适用性有限，同时也给患者参与带来挑战。真实世界数据研究为填补证据缺口提供了可行方案，能够提供关于癌症治疗在真实世界环境中效果的关键信息。真实世界数据与随机临床试验数据的本质区别在于数据收集方式——真实世界数据本质上是在诊疗点采集获得的。借鉴传统临床试验的实验设计方法可用于生成真实世界数据，从而带来多重优势，包括提升研究效率，并在内部效度与外部效度之间实现更合理的平衡。在药物警戒领域，真实世界数据可用于促进对疾病进展的理解，并构建外部对照组。通过利用前瞻性收集的真实世界数据，开展实用性临床试验具有可行性，这类试验既能为随机研究设计提供证据支持，又能将临床研究延伸至患者诊疗现场。为确保真实世界研究的质量，实施可审计的数据提取方法、建立新型激励机制以电子化方式在诊疗点采集临床相关数据至关重要。当前治疗格局持续演进，一线免疫检查点抑制剂（无论是单药还是联合化疗）的应用正在影响后续治疗路径的选择。在代表性不足的人群中（如老年患者、体能状态较差者、肝炎或人类免疫缺陷病毒感染者），真实世界疗效与安全性仍有待深入探索。同样，这些创新药物的成本效益与可持续性也是真实世界中需要重点考量的因素。
Real-World Data and Evidence in Lung Cancer: A Review of Recent Developments
肿瘤(癌症)患者之家