Patient outcomes

Are NMIBC Trials Capturing What Matters to Patients?

For patients with non-muscle-invasive bladder cancer (NMIBC), the burden of disease extends beyond traditional measures of recurrence and progression. Repeated cystoscopies, intravesical treatments, urinary symptoms, sexual dysfunction, and concern about recurrence can affect patients throughout years of surveillance. Yet a systematic review suggests that commonly used patient-reported outcome (PRO) instruments do not consistently capture the full NMIBC experience, raising important questions about how quality of life and treatment burden are assessed in clinical trials.

Researchers reviewed 39 studies evaluating eight PRO instruments used in adults with NMIBC. The instruments were assessed for factors including reliability, validity, responsiveness, interpretability, and their coverage of NMIBC-relevant concerns. The review also examined how well the tools may support contemporary clinical trials and regulatory requirements. Among the instruments evaluated, the EORTC QLQ-NMIBC24 and Bladder Cancer Index had the strongest overall evidence base for use in NMIBC.

The EORTC QLQ-NMIBC24 was developed specifically for NMIBC and captures urinary symptoms, intravesical treatment burden, sexual function, and concerns about future health. The Bladder Cancer Index also demonstrated strong measurement properties and assesses urinary, bowel, and sexual function. Other commonly used cancer questionnaires can provide valuable information about fatigue, physical functioning, emotional well-being, and general quality of life, but their ability to capture issues specific to NMIBC is more limited.

One of the review's most notable findings was what existing questionnaires may be missing. Recurrence anxiety and the cumulative burden of repeated treatment and surveillance remain incompletely measured across available instruments. This may be particularly important in NMIBC, where recurrence rates are high and patients can undergo repeated cystoscopy, intravesical therapy, and long-term follow-up. The authors also noted that patient-reported outcome data during maintenance therapy and long-term survivorship remain underrepresented, leaving uncertainty about how well current instruments reflect the experience of patients living with NMIBC over time.

For urologists and investigators, the findings reinforce the importance of looking beyond traditional clinical endpoints when evaluating NMIBC care. No single questionnaire appears to capture every aspect of the patient experience, and the authors suggest that combining validated NMIBC-specific instruments with broader cancer measures and targeted questions may provide a more complete assessment. As new treatments and surveillance strategies are evaluated, incorporating measures of recurrence anxiety and cumulative treatment burden could help ensure that clinical benefit is considered alongside the experience of living with and being treated for NMIBC.