Objectives: In June 2021, the FDA released updated guidelines on core patient-reported outcomes (PROs) in cancer clinical trials (1). The guidelines emphasized the need to focus on five concepts: 1) ‘disease related symptoms’, 2) ‘symptomatic adverse events (AEs)’, 3) ‘overall side effect impact’, 4) ‘physical function’ and 5) ‘role function’ to facilitate a better understanding of treatment benefits by including patient reports. Non-muscle invasive bladder cancer (NMIBC) and associated treatments have been found to impair quality of life (QoL) including physical and role functioning (QoL) (2). This research aims to explore the extent to which clinical trials in NMIBC that included QoL assessments, naturally covered the concepts outlined in the guidance before its publication.

Methods: The following terms were used in PubMed: 'non-muscle invasive’ AND ‘cancer’ AND ‘bladder’ AND (‘quality of life’ or 'PRO' or 'questionnaire') AND ‘clinical' and 'trial’. Studies published in English describing NMIBC trials and including QoL assessments were identified and examined.

Results: Eleven trials from PubMed met the inclusion criteria. The five concepts recommended by the FDA were covered to a varying degree and by a plethora of PROs (e.g. quality of life of cancer patients QLQ-C30, bladder cancer index – BCI). Patient-reported assessment of symptoms and role function were reported in 9 of 11 studies, physical function was reported in all studies, yet patient-reported symptomatic AEs and overall side effect impact were not included in any of the trials.

Conclusions: The development of a standardized approach to assess the concepts recommended by the FDA in NMIBC, may improve the comparability of the QoL findings across trials. The adoption of the FDA guidelines across all NMIBC clinical trials could facilitate a better understanding of the impact of therapies on patients and allow for a more comprehensive analysis of risk/benefits of cancer treatments.