How patient-reported outcomes can transform bladder cancer clinical trials

In a recent video interview with Urology Times discussing the BCG vs gemcitabine/docetaxel (gem/doce) bridge trial, Angela B. Smith, MD, MS, vice chair of Academic Affairs and a professor of urology at the University of North Carolina School of Medicine, Chapel Hill, emphasizes that integrating patient-reported outcomes (PROs) into bladder cancer research is not just beneficial but essential.

She notes that the trial, led by Max Kates, MD, and supported by a dedicated PRO committee, illustrates how embedding patient perspectives enriches clinical evidence. According to Smith, when studies focus solely on traditional end points such as recurrence or survival, they often miss critical insights into patient experience—insights that may explain real-world uptake or rejection of an intervention. Incorporating PROs, she argues, completes the “full story” by capturing how patients perceive treatment burdens, trade-offs, and quality-of-life impacts that ultimately shape adherence and implementation.

Smith stresses that balancing PROs with traditional clinical end points begins with engaging patients early in the trial design process. Patient advocates can help researchers determine which outcomes matter most and ensure that PRO measures are designated appropriately as primary or secondary endpoints. Too often, she notes, PROs are added late and retroactively, weakening their value. Early involvement—simply asking patients what matters—provides clarity, requires minimal extra effort, and significantly strengthens both scientific rigor and patient relevance.

Regarding funding, Smith explains that the incremental cost of adding PROs to an already-planned clinical trial is relatively small. For studies centered primarily on PROs, she highlights the Patient-Centered Outcomes Research Institute (PCORI) as a particularly robust funding source, noting its support for engagement awards, comparative effectiveness studies, and dissemination and implementation research. She adds that industry partners increasingly recognize the value of patient engagement and are more willing than in years past to support PRO integration, including funding patient advocate involvement in study design. Collectively, these strategies make PRO-driven research more feasible and help ensure that bladder cancer trials capture outcomes that truly matter to patients.