The Role of Real-World Evidence in Oncology Drug Development: Current Capabilities and Future Potential

Real-world evidence (RWE) has been a keenly desired complement to randomized controlled trials (RCTs) in oncology drug development, offering essential information on the treatment's efficacy in real-world clinical practice environments. The review systematically reviewed the peer-reviewed literature published 2019 through 2024 in order to characterize the role of RWE in drug development, regulatory clearance, and methodological refinement. The evidence demonstrates that RWE is increasingly being applied along the continuum of drug development, and regulatory authorities are increasingly placing value on it - 13 of 706 oncology drug approvals in 2015 through 2020 were approved on the basis of RWE. Its most important applications are external control arms in single-arm trials, post-market surveillance, and evidence in support of clearance in the rare tumors in which RCTs are typically not practical. Even though RWE potentially facilitates expedited clearance of a drug, cost reduction, and evidence in support of underrepresented populations, in order to be fully utilized, challenges with data standardization, quality control, and methodological consistency are needed to be overcome.