Patient-reported outcomes in advanced breast cancer: inside the label and approval documents

ABSTRACT Patient-reported outcome (PRO) measures are used in clinical research and practice for the assessment of disease-related symptoms and impacts as well as treatment-related side effects, from the patient perspective. However, a systematic examination of the role of PROs in metastatic breast cancer treatment approvals is lacking. A review of FDA labels and historical drug approval documents for metastatic breast cancer treatments was conducted to determine how PROs had been used or pursued to support labeling claims. In the historical drug approval documents, PROs were often being implemented by sponsors, and regulatory reviewers noted several issues limiting their suitability to support label claims. The findings suggest there is much room for improvement in how sponsors develop, implement, and report PRO measurement strategies as part of drug approval.