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

Value Health. 2019 Feb;22(2):203-209. doi: 10.1016/j.jval.2018.09.2842.


Objectives: To compare US Food and Drug Administration (FDA) and European Medicines Agency (EMA) labeling for evidence based on patient-reported outcomes (PROs) of new oncology treatments approved by both agencies.

Methods: Oncology drugs and indications approved between 2012 and 2016 by both the FDA and the EMA were identified. PRO-related language and analysis reported in US product labels and drug approval packages and EMA summaries of product characteristics were compared for each indication.

Results: In total, 49 oncology drugs were approved for a total of 64 indications. Of the 64 indications, 45 (70.3%) included PRO data in either regulatory submission. No FDA PRO labeling was identified. PRO language was included in the summary of product characteristics for 21 (46.7%) of 45 indications. European Organisation for Research and Treatment of Cancer and Functional Assessment of Cancer Therapy measures were used frequently in submissions. FDA's comments suggest that aspects of study design (eg, open labels) or the validity of PRO measures was the primary reason for the lack of labeling based on PRO endpoints. Both agencies identified missing PRO data as problematic for interpretation.

Conclusions: During this time period, the FDA and the EMA used different evidentiary standards to assess PRO data from oncology studies, with the EMA more likely to accept data from open-label studies and broad concepts such as health-related quality of life. An understanding of the key differences between the agencies may guide sponsor PRO strategy when pursuing labeling. Patient-focused proximal concepts are more likely than distal concepts to receive positive reviews.

Keywords: European Medicines Agency (EMA); Food and Drug Administration (FDA); labeling; oncology; patient-reported outcome (PRO).

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Antineoplastic Agents / standards*
  • Drug Approval*
  • Drug Labeling / legislation & jurisprudence
  • Drug Labeling / standards*
  • Europe / epidemiology
  • Humans
  • Neoplasms / drug therapy
  • Neoplasms / epidemiology
  • Patient Reported Outcome Measures*
  • Randomized Controlled Trials as Topic / standards
  • United States / epidemiology
  • United States Food and Drug Administration / legislation & jurisprudence
  • United States Food and Drug Administration / standards*


  • Antineoplastic Agents