Presentation (CTI)

OBJECTIVES: Real-world evidence (RWE), derived from real-world data (RWD), complements randomized clinical trial findings by capturing outcomes in broader, more diverse patient populations. In oncology, treatments are often tailored to small, heterogeneous patient populations, and the therapeutic landscape is constantly evolving. RWE in oncology can provide valuable insights into real-world effectiveness and safety, economic and humanistic implications, and regulatory and reimbursement decisions. This study aims to characterize RWE generation in oncology and its growing role in healthcare decision-making over the last decade.
METHODS: A targeted literature review was performed to identify articles published between 2015 and 2024 using a PubMed search with keywords and MeSH terms related to “real-world evidence,” “real-world data,” “cancer”, “regulatory”, and “reimbursement”. Articles were characterized to assess their role in healthcare decision-making.
RESULTS: Between 2015 and 2024, the number of articles addressing RWE and RWD in cancer increased by 1.4-fold, rising from 6,197 to 8,629. Approximately one-third of these articles focused on the application of RWE/RWD to healthcare decision-making, while another third were observational studies. Articles discussing the application of RWE in regulatory and health technology assessment (HTA)/reimbursement decision-making increased by 2-fold and 1.9-fold, respectively. Observational studies examining treatment patterns saw a nearly 4-fold increase, while those reporting economic outcomes grew by 1.2-fold. About one in ten observational studies assessed real-world effectiveness and safety.
CONCLUSIONS: The increasing volume of RWE articles underscores its growing importance in oncology decision-making. RWE informs regulatory approvals, HTA/reimbursement processes, treatment pattern analyses, and economic evaluations. Despite its expanding role, further standardization of methodologies and improved data quality are needed to ensure robust and actionable insights. As oncology continues to evolve with personalized medicine and innovative therapies, RWE will remain pivotal in bridging evidence gaps and guiding policy and clinical practice.