Assessment of PRO label claims granted by the FDA as compared to the EMA (2006-2010)

Value Health. 2013 Dec;16(8):1150-5. doi: 10.1016/j.jval.2013.08.2293. Epub 2013 Oct 17.


Background: The US Food and Drug Administration (FDA) provides formal guidance for the use of patient-reported outcomes (PROs) in support of labeling claims, whereas the European Medicines Agency (EMA) offers insight in a reflection paper relating to health-related quality of life in lieu of formal guidance.

Objectives: PRO label claims granted for new molecular entities and biologic license applications from 2006 through 2010 were reviewed to evaluate consistencies and discrepancies in PRO label claims granted by the FDA and the EMA and to highlight trends in the acceptance of PRO claims across agencies.

Methods: Products approved by both the FDA and the EMA were identified. By using US Drug Approval Packages and European Public Assessment Reports packages, any PRO label claims made for the same product by the same company were compared.

Results: Both agencies approved a total of 75 products. Of these, 35 (47%) had at least one EMA-granted PRO label claim compared with 14 (19%) by the FDA. Most FDA-grated claims focused on symptoms; however, EMA-granted claims were more likely to include higher order concepts. Few (~12%) were granted the same label claims. Despite this discordance between the two agencies, where PRO label claims were granted by both the FDA and the EMA, there was similarity in the type of label claim.

Conclusions: The EMA is more likely than the FDA to grant PRO claims and for higher order constructs. On a macro level, there appears to be poor concordance between claims granted by both agencies. On close examination, however, there appears to be greater concordance than previously recognized, which may be instructive in formulating future PRO strategies. Further research to create strategic alignment across agencies may be beneficial.

Keywords: EMA; FDA; labeling; patient-reported outcome.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Drug Approval / statistics & numerical data*
  • Drug Labeling / statistics & numerical data*
  • Europe
  • Humans
  • Pharmaceutical Preparations*
  • Quality of Life
  • Treatment Outcome*
  • United States
  • United States Food and Drug Administration / statistics & numerical data*


  • Pharmaceutical Preparations