Presentation (CTI)

OBJECTIVES: The US Food and Drug Administration (FDA) encourages the use of patient-reported outcome (PRO) measures, including PRO-CTCAE items and global tolerability items such as FACT-GP5 or EORTC Q168, to capture patients’ perceptions of treatment tolerability and support benefit-risk assessment in oncology. This study assessed the extent to which tolerability PROs have been incorporated into recent FDA oncology drug approvals and reflected in regulatory review documents and product labeling.
METHODS: FDA oncology drug approvals issued between 2021 and 2025 were systematically identified. Public FDA multidisciplinary review documents and approved product labels were reviewed to determine inclusion of tolerability PROs. Data extracted included indication, trial phase and design, PRO instruments used, number of PRO-CTCAE items included in confirmatory trials, and the presence and location of PRO-related statements in product labeling. Results were summarized descriptively.
RESULTS: Of 258 oncology approvals identified, 19 (7.4%) included tolerability-related PROs, the majority of which were for solid tumors (n=16). PRO-CTCAE items alone were used in six approvals, FACT-GP5 alone in nine, and both in four approvals. No approvals included EORTC Q168. The number of PRO-CTCAE items included in confirmatory trials ranged from one to 19. Tolerability PROs appeared in product labeling for three approvals, located in the safety section for two products and in the efficacy section for one.
CONCLUSIONS: Despite FDA emphasis on importance of tolerability assessment in oncology drug development, their incorporation into regulatory review documents and product labeling remains limited. Greater and more consistent integration of tolerability PROs into oncology clinical trials and regulatory submissions could strengthen and support more comprehensive benefit-risk evaluations, as tolerability becomes increasingly central to oncology drug approval.