Patient-reported outcomes as non-muscle-invasive bladder cancer clinical trial endpoints
In brief
Only 22% of non-muscle-invasive bladder cancer trials track patient-reported outcomes
Among 94 interventional trials, just 21 included patient-reported outcomes; only 3% used them as a primary endpoint, and uptake changed little over time. These measures capture patients' experiences directly, but their limited and inconsistent use makes it harder to compare how treatments affect quality of life - a gap the authors say calls for shared tools and broader adoption.
- Journal
- BJU international (Q1)
- Published
- 28 September 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Kimberly Toumazos, Spencer Bell, Katelyn Spencer, Darrell Nakagawa, Kelly K Bree, Ashish M Kamat, et al.
- PMID
- 42806610
- DOI
- 10.1111/bju.70461
Why clinicians should know about it
- Picked for Urology (top studies of the week, 4 October 2026): PROs as endpoints in NMIBC trials
Abstract
OBJECTIVE: To evaluate the frequency at which patient-reported outcomes (PROs) are incorporated as nonmuscle-invasive bladder cancer (NMIBC) clinical trial endpoints. PATIENTS AND METHODS: Interventional trials limited to patients with NMIBC were queried from clinicaltrials.gov from 1994 to 2025 (start of trial). Observational studies or those without a discrete intervention were excluded. The incorporation of all PROs as primary and secondary endpoints was recorded. PROs were not limited to validated tools. They were defined according to United States Food and Drug Administration (FDA) guidance as any information about a patient's health/experience coming directly from the patient without interpretation by a provider/investigator. Categorical variables were analysed with chi-square and Fisher's exact tests, when applicable, and continuous variables were analysed with the Mann-Whitney U test. RESULTS: A total of 94 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 European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) and EORTC QLQ-Nonmuscle-Invasive Bladder Cancer 24 module (incorporated in 48% and 43% of NMIBC trials using PROs, respectively). Trials that incorporated PROs as endpoints included higher median anticipated patient enrolments (median [interquartile range] number of patients 166 [461] vs 46 [101], P = 0.002) and were more likely to be randomised controlled trials (62% vs 41%, 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 time. CONCLUSION: 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. These data serve as a call to action for PRO tool harmonisation and universal incorporation in NMIBC clinical trial design.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.