OBJECTIVES: Patient Reported Outcomes (PROs) are particularly relevant in therapeutic areas where clinical diagnosis and management is based primarily on how the patient responds to healthcare provider questions. In those therapeutic areas, PROs are generally included in randomized clinical trials as the primary endpoint; however, for almost all other therapeutic areas, PROs are included only as secondary or exploratory objectives. Current PRO guidance does not address scientific limitations resulting from the lack of a comparator in single arm (SA) registrational clinical trials. This review focuses on the utilization of PROs in oncology SA registrational trials incorporating an ECA to better understand the scope of the problem.

METHODS: Cortellis for Regulatory Intelligence by Clarivate was used to identify and review FDA and EMA submissions from 2013-2023. SA oncology submissions were searched for the keyword “oncology” in title, abstract, and further limited to those containing “single arm” within the text. Two reviewers independently screened regulatory submissions for mentions of PROs, type of endpoint, trial phase, ECA type, PRO inclusion in ECA, and mentions of PROs in the label.

RESULTS: 66 oncology product approvals were identified and reviewed (EMA, n=4; FDA, n=62). 18 were excluded as they represented only minor updates to original submissions; 48 were included in the final review. 21 (44%) included PROs as secondary or exploratory endpoints; only two (10%) of which included an ECA. However, neither ECA incorporated PROs. PROs most frequently cited were: EORTC QLQ-C30 (n=10, 48%) and EQ-5D (n=6, 29%). PROMIS Cancer Fatigue and PRO-CTCAE, both FDA recommended scales, were each used only once.

CONCLUSIONS: This review demonstrated that PROs are included in oncology SA trials, but not in the corresponding ECAs or product labels. Identification of data sources and methodology to incorporate PROs in ECAs for contextualization are needed.