Quantifying Diversity and Representation in Pivotal Trials Leading to Marketing Authorization in Europe

Ther Innov Regul Sci. 2022 Sep;56(5):795-804. doi: 10.1007/s43441-022-00421-0. Epub 2022 Jun 9.


Background: Following up on a study from 2019, Tufts CSDD collected and analyzed data on demographic disparities and representation in pivotal trials supporting the marketing authorization of novel drugs and biologics approved in Europe between 2007 and 2019.

Methods: Data were collected from products' EPAR, the EUDRACT database, and other publicly available sources, and compared to indication-specific demographic data or a census estimate. In total, data were collected on 446 drugs and 943 pivotal trials.

Results: Results indicated that gender demographic data were only reported for 80.7% of pivotal trials, and that racial and ethnicity demographic data were reported less often (64.1% and 29.9% of pivotal trials, respectively). Results also indicated that non-white racial identities were under-represented by more than 20% in nearly half or more of pivotal trials.

Conclusions: Guidelines encouraging the reporting of patient demographic data are insufficient and availability of the data is problematic. The available data suggest that under-representation in pivotal trials is widespread.

Keywords: Demographics; Diversity; Ethnicity; Pivotal trials; Race; Representation.

MeSH terms

  • Drug Approval*
  • Ethnicity
  • Europe
  • Humans
  • Marketing
  • Racial Groups*