(UroToday.com) The 2026 GU ASCO annual meeting featured a urothelial carcinoma session and a presentation by Dr. Spencer Bell discussing the implementation of patient-reported outcomes as non-muscle invasive bladder cancer clinical trial endpoints. Non-muscle invasive bladder cancer 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 provide a validated framework to measure symptoms, treatment burden, and overall well-being directly from the patient’s perspective. In 2021, the FDA issued guidance on the use of patient-reported outcomes in cancer clinical trials. This study evaluates the frequency at which patient-reported outcomes are incorporated as non-muscle invasive bladder cancer clinical trial endpoints.
Interventional trials limited to patients with non-muscle invasive bladder cancer were queried from clinicaltrials.gov from 1994-2026 (start of trial). Observational studies or those without a discrete intervention were excluded. The incorporation of patient-reported outcomes as primary and secondary endpoints was recorded. Patient-reported outcomes were defined as any information about a patient’s health/experience coming directly from the patient without interpretation by a provider/investigator.
Ninety-four clinical trials were included, of which 21 (22%) incorporated patient-reported outcomes. Patient-reported outcomes were included in 3% and 20% of trials as primary and secondary endpoints, respectively. The most commonly used patient-reported outcomes include the EORTC QLQ-C30 and EORTC QLQ-NMIBC24, which were incorporated in 48% and 43% of non-muscle invasive bladder cancer trials using patient-reported outcomes, respectively.
Trials that incorporated patient-reported outcomes as endpoints included higher median anticipated patient enrollments: 166 patients (IQR 461) versus 46 patients (IQR 101). Additionally, patient-reported outcomes were more likely to be included in randomized controlled trials (62% versus 41% in non-randomized trials, p = 0.057). There was no difference in funding mechanism (industry-sponsored versus cooperative group/federally funded) between trials that did and did not incorporate patient-reported outcomes. Finally, there was little change in patient-reported outcome incorporation as trial endpoints over the study period.
Dr. Bell concluded his presentation discussing the implementation of patient-reported outcomes as non-muscle invasive bladder cancer clinical trial endpoints with the following take-home points:
- Despite recent emphasis from the FDA, patient advocacy groups, and the scientific community on the use of patient-reported outcome measurements to capture the holistic, lived experience of patients on investigational cancer therapeutics, there is limited incorporation of patient-reported outcomes in non-muscle invasive bladder cancer interventional trials
- Moving forward, a standardized framework of validated patient-reported outcome measurements should be incorporated into early clinical trial design
Presented by: Spencer Bell, MD, University of Kentucky, Lexington, KY
Written by: Zachary Klaassen, MD, MSc – Urologic Oncologist, Associate Professor of Urology, Georgia Cancer Center, Wellstar MCG Health, @zklaassen_md on Twitter during the 2026 Genitourinary (GU) American Society of Clinical Oncology (ASCO) Annual Meeting, San Francisco, CA, Thurs, Feb 26 – Sat, Feb 28, 2026.
Related content: Patient-Reported Outcomes in NMIBC Trials: A 30-Year Analysis of Incorporation Trends - Patrick Hensley