A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved by the FDA and the EMA (2012-2016).

•In recent years, the Food and Drug Administration (FDA) has granted patient-reported outcome (PRO) labeling to very few oncology drugs.•The FDA and the European Medicines Agency (EMA) use different evidentiary standards to assess PRO data from oncology studies. PRO labeling by the EMA was frequently based on open-label studies, on broad concepts such as health-related quality of life, and on PRO measures that may be outdated and unsuitable for contemporary oncology clinical trials.•There are key differences in evidentiary standards between the FDA and the EMA. An understanding of these differences may be useful to guide PRO measurement strategies as study sponsors pursue PRO labeling. Sponsors pursuing labeling from both agencies should include in clinical trials PRO measures that assess patient-focused proximal concepts of core disease symptoms, treatment-related symptoms, and impacts on physical functioning. The addition of health-related quality-of-life assessments may also be useful for EMA reviews.