PCN299 A REVIEW OF PATIENT-REPORTED OUTCOMES LABELING FOR ONCOLOGY PRODUCTS APPROVED BY FDA AND EMA FROM 2017 TO 2018

To compare the patient-reported outcomes (PRO) labeling for oncology products approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) between 2017 and 2018. FDA Drug Approval Packages (DAP) and EMA European Public Assessment Reports (EPAR) were reviewed for drugs with oncology indications approved by both FDA and EMA between 2017 and 2018. PRO labeling was defined as any description of treatment-related benefit or risk in the FDA package insert (PI) or EMA summary of product characteristics (SmPC) based on PRO data. A total of 121 oncology indications were identified and reviewed, among which, 21 indications (for 21 products) received PRO labeling, including 15 (71.4%) for solid tumors and 6 (28.6%) for hematological malignancies. Among these 21 drugs, 2 (9.5%) had PRO language in the PI and 20 (95.2%) had PRO language in the SmPC. As for study design, 17 (90.0%) indications were evaluated using randomized controlled trials (RCTs), and 13 (61.9%) employed open-label designs. Frequently used PRO measures (PROMs) in EMA labeling included the EQ-5D (65%), the European Organization for Research and Treatment of Cancer Core 30 Items (EORTC QLQ-C30, 55%), and the Functional Assessment of Cancer Therapy (FACT, 35%). However, only disease-specific PROMs were included in FDA labeling. As for PRO concepts in EMA labeling, the majority (90%) mentioned HRQoL/global health status, more than half (55%) mentioned symptoms, and 30% had functioning concepts. Whereas in FDA labeling, only disease-specific symptoms were used. Key comments from FDA reviewers regarding PRO data submissions failing to receive PRO labeling include inappropriate PROMs, excessive missing data, uncontrolled or open-label study design, and lack of clinical/statistical significance. FDA and EMA applied different standards assessing PRO data for drug labeling. Broad concepts such as HRQoL and global health status were not accepted by the FDA.