The value of real-world evidence in supporting targeted therapies for patients with rare oncogenic drivers in mNSCLC.

Abstract

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.