Presentation (CTI)

OBJECTIVES: Patient-reported outcomes (PROs) are critical for capturing patient experiences in cancer clinical trials and informing regulatory decisions. FDA provided guidance in 2021 on PRO use in Cancer Clinical Trials. Adherence of Cancer trials with this guidance remains unclear and is the main objective of this study.
METHODS: Trials Cancer Clinical Trials conducted over 2019 to 2024 were reviewed for all US FDA approved Oncology Therapies. Data was extracted into a proprietary database from CT.gov, pivotal publications and prescriber information (PI). A checklist was created using the FDA guidance and adherence assessed.
RESULTS: Since 2019, US FDA approved 68 oncology therapies which were studied in 206 clinical trials. Of these 76 (36.9%) did not report any PRO measures used. The remaining 130 studies were further analyzed. Among these, 57 trials (43.8%) provided a PRO plan without reporting results, while 73 trials (56.2%) included detailed PRO outcomes. Baseline PRO assessments were conducted in 115 trials (88.5%), however only one trial provided a missing data plan. PRO assessment frequency aligned with treatment cycles in 91 trials (70%). The evaluation of adherence to FDA guidelines was conducted at the Cancer therapy Level. Only 46% of the therapies met General Trial Design requirements, hampered by low Missing data scores. Core PROs collected achieved 62% adherence (lack of standardized AE measures) and Instrument Selection and Validation fared the best with 92% adherence. Data Collection Documentation compliance was 38% and Labeling Considerations adherence was largely absent across therapies.
CONCLUSIONS: While some oncology trials demonstrate strong adherence to FDA recommendations, significant gaps persist, particularly in minimizing missing data, documenting adverse events, and incorporating PRO findings into labeling. Enhanced adoption of FDA-recommended practices is needed to ensure standardized and meaningful PRO integration in oncology trials.