OBJECTIVES : Non-randomized trials are prevalent in cancer, and patient-reported symptoms and function are of interest to patients, regulators and other stakeholders. We describe the use and reporting of patient-reported outcomes (PROs) in non-randomized trials submitted to the US Food and Drug Administration to support product approval.

METHODS : We examined application documents including protocols and clinical study reports submitted to FDA to support new applications or efficacy supplements for anticancer products from 2007 – 2017. We identified the PRO measures collected, and assessed whether completion information (% of patients on-study completing the PROs) was provided.

RESULTS : We identified 169 trials for anti-cancer products, of which 64 (38%) were non-randomized. Of these 64 trials, 19 (30%) included PROs. Six of the 19 (32%) trials that included PROs reported PRO completion rates. For the 6 trials with available PRO completion information, the median pre-treatment completion rates were 85% at screening (range 80 – 90%) and 92% at baseline (range 91% - 99%). At 6 months, median completion was 87% (range 70% - 96%).

CONCLUSIONS : Although many cancer trials are non-randomized, PROs have been infrequently used in these designs. When PROs are used, completion information is rarely provided by the applicants. However, when reported, completion rates were reasonably high. It is unclear whether trials with unreported PRO completion would follow this trend or not. Data from PRO measures of symptoms and function can be informative, even in non-randomized trials, and FDA will review this data as part of the totality of evidence. When PROs are assessed in trials, the expectation should be that study reports include all PRO results, including completion rate, to inform the quality of the data collected.