Nagasaka Misako, Roy Upal Basu, Berk Alexandra, Liu Geoffrey, Nadler Eric, Abrahami Devin
Division of Hematology and Oncology, Department of Medicine, Chao Family Comprehensive Cancer Center, University of California Irvine School of Medicine, Orange, CA, USA.
Division of Neurology, Department of Medicine, St. Marianna University School of Medicine, Kawasaki, Japan.
Future Oncol. 2025 Apr;21(9):1005-1011. doi: 10.1080/14796694.2025.2475728. Epub 2025 Mar 14.
With the ongoing discovery of various oncogenic driver mutations in metastatic non-small cell lung cancer (mNSCLC), a precision medicine approach has emerged, characterized by targeted therapies for select patient populations. Randomized controlled trials (RCT) remain the gold standard for evaluating efficacy and safety of such therapies; however, RCTs evaluating treatments for rare oncogenic drivers still face limitations, given small populations, potentially long-time horizon for outcome events to occur, and underrepresentation of certain subgroups. For these targeted therapies, the complementary nature between real-world evidence (RWE) and RCT may expand the totality of evidence available, to better inform treatment decision-making. In particular, treatments for rare oncogenic drivers can benefit from RWE that provides additional, generalizable clinical insights for subgroups underrepresented or ineligible for RCT, or confirms outcomes observed in RCT. As a discipline, RWE has seen significant advances in methodology and healthcare stakeholder acceptability, with potential for even greater innovation, and presents a valuable opportunity to support decision-making around access and use of targeted therapies for rare oncogenic drivers in mNSCLC.
随着转移性非小细胞肺癌（mNSCLC）中各种致癌驱动基因突变的不断发现，一种精准医学方法应运而生，其特点是针对特定患者群体进行靶向治疗。随机对照试验（RCT）仍然是评估此类疗法疗效和安全性的金标准；然而，鉴于患者群体规模小、结局事件可能需要较长时间才能发生以及某些亚组代表性不足，评估针对罕见致癌驱动因素治疗的RCT仍面临局限性。对于这些靶向疗法，真实世界证据（RWE）与RCT之间的互补性可能会扩大现有证据的总量，从而更好地为治疗决策提供信息。特别是，针对罕见致癌驱动因素的治疗可以从RWE中受益，RWE为在RCT中代表性不足或不符合条件的亚组提供了额外的、可推广的临床见解，或证实了在RCT中观察到的结局。作为一门学科，RWE在方法学和医疗保健利益相关者的可接受性方面取得了重大进展，具有更大创新的潜力，并为支持围绕mNSCLC中罕见致癌驱动因素的靶向疗法的获取和使用的决策提供了宝贵机会。
