Objectives: In 2009, the United States (US) Food and Drug Administration (FDA) issued a formal guidance for the use of patient-reported outcomes (PRO) in support of labeling claims, whereas the European Medicines Agency (EMA) offers insight in a 2007 reflection paper in lieu of formal guidance. To evaluate and describe decision making by the FDA and EMA, a review of PRO label claims granted for new molecular entities and biologic license applications from 2006 through 2010 was conducted. The purpose of this research was to evaluate consistencies and discrepancies and to highlight trends in the acceptance of PRO claims across agencies.
Methods: A listing was created of drug approvals granted by both the FDA and the EMA. PRO claims were compared using US Drug Approval Packages and European Public Assessment Reports packages to determine any instances where claims made for the same product by the same company were similar or different.
Results: A total of 75 products were identified as having been approved by both agencies. Of these, a total of 35 (40%) were granted at least one PRO claim by the EMA, as compared with 14 (19%) by the FDA. Most claims in the US focused on signs and symptoms; however, claims in the European Union were more likely to include higher-order concepts such as health-related quality of life (HRQL) and functioning (29% EMA, 9% FDA). Only a small number of products (∼10%) had the same claims granted by both agencies.
Conclusions: The EMA is more likely than the FDA to grant PRO claims and to grant claims for higher-order constructs such as HRQL and functioning. Additionally, there appears to be poor concordance between claims granted by both agencies, which may demonstrate a need for sponsors to develop agency-specific PRO strategies.
