Implementation of patient reported outcomes as nonmuscle-invasive bladder cancer clinical trial endpoints.

722 Background: Nonmuscle-invasive bladder cancer (NMIBC) represents a highly recurrent malignancy requiring repeated transurethral surgical intervention and intravesical therapies which impact health-related quality of life. Thus, traditional clinical trial endpoints, including recurrence- and progression-free survival, may not fully capture the patient experience. Patient-reported outcomes (PROs) provide a validated framework to measure symptoms, treatment burden, and overall well-being directly from the patient’s perspective. In 2021 the Food & Drug Administration (FDA) issued guidance on the use of PROs in cancer clinical trials. This study evaluates the frequency at which PROs are incorporated as NMIBC clinical trial endpoints. Methods: Interventional trials limited to patients with NMIBC were queried from clinicaltrials.gov from 1994-2026 (start of trial). Observational studies or those without a discrete intervention were excluded. The incorporation of PROs as primary and secondary endpoints was recorded. PROs were defined as any information about a patient’s health/experience coming directly from the patient without interpretation by a provider/investigator. Categorical variables were analyzed with Chi-square and Fisher’s exact tests, when applicable, and continuous variables were analyzed with the Mann-Whitney U test. Results: Ninety-four clinical trials were included, of which 21 (22%) incorporated PROs. PROs were included in 3% and 20% of trials as primary and secondary endpoints, respectively. The most commonly used PROs include the EORTC QLQ-C30 and EORTC QLQ-NMIBC24 (incorporated in 48% and 43% of NMIBC trials using PROs, respectively). Trials that incorporated PROs as endpoints included higher median anticipated patient enrollments (166 patients (IQR 461) vs. 46 patients (IQR 101)). PROs were more likely included in randomized controlled trials (62% vs. 41% in non-randomized trials, P = 0.057). There was no difference in funding mechanism (industry-sponsored vs. cooperative group/federally funded) between trials that did and did not incorporate PROs. There was little change in PRO incorporation as trial endpoints over the study period. Conclusions: Despite recent emphasis from the FDA, patient advocacy groups, and the scientific community on the use of PRO measurements to capture the holistic, lived experience of patients on investigational cancer therapeutics, there is limited incorporation of PROs in NMIBC interventional trials. Moving forward, a standardized framework of validated PRO measurements should be incorporated into early clinical trial design.