Dr Benjamin on Using Real-World Data to Bridge Knowledge Gaps in Urothelial Cancer Care

"We have data from clinical trials to look at, but there are issues with some of these trials. With real-world data, we can supplement and complement the clinical trial data where there are gaps."

David J. Benjamin, MD, a medical oncologist at Hoag Family Cancer Institute, discussed how real-world evidence can be leveraged to bridge the gaps left by clinical trial data in urothelial cancer.

Although substantial real-world data have replicated findings from the phase 3 JAVELIN Bladder 100 trial (NCT02603432), data from the phase 3 EV-302 trial (NCT04223856) combination of enfortumab vedotin-ejfv (Padcev) plus pembrolizumab (Keytruda) are relatively limited, Benjamin asserted. The retrospective UNITE study, which included data from 171 patients outside of the clinical trial setting who received this combination, is the most notable contribution to date, Benjamin explained. In this study, the overall response rate (ORR) among patients with advanced urothelial carcinoma treated with enfortumab vedotin plus pembrolizumab in the real-world setting (n = 171) was 51% (95% CI, 42%-60%). Conversely, the ORR with the combination (n = 437) in EV-302 was higher, at 67.7% (95% CI, 63.1%-72.1%).

Despite the important contributions of UNITE, more robust real-world data are needed to understand not only efficacy but also the adverse effects associated with this new regimen, Benjamin noted.

Although data from clinical trials are valuable, they have inherent limitations, Benjamin stated. For example, in the platinum-based chemotherapy arm of EV-302, over 60% of patients who were eligible for maintenance avelumab (Bavencio) did not receive it, he reported. This introduces a gap in the knowledge base when discussing survival benefits with patients, as the trial data may not fully reflect the effect of standard-of-care maintenance therapy, he added.

Benjamin went on to underscore the role of real-world evidence in addressing these gaps. He explained that real-world evidence can help evaluate the survival benefit of platinum-based chemotherapy followed by maintenance avelumab by capturing real-world treatment patterns and outcomes. Additionally, in EV-302, patients with an ECOG performance status of 2 represented only approximately 3% of the study population. In contrast, patients with an ECOG performance status of 2 or higher are seen much more frequently in real-world practice, Benjamin detailed. Integrating real-world data with clinical trial results can provide a more comprehensive understanding of treatment effectiveness and safety, ultimately enhancing clinical decision-making for patients with urothelial cancer, he concluded.