New Benchmarks on Demographic Disparities in Pivotal Trials Supporting FDA-Approved Drugs and Biologics

Ther Innov Regul Sci. 2023 Sep 29. doi: 10.1007/s43441-023-00579-1. Online ahead of print.


Background: A lack of diversity and representation in clinical trials is an established issue in drug development, and the COVID-19 pandemic increased awareness of the problem among the general public. This awareness has led to increased pressure on drug development sponsors, as well as additional attention and regulation from federal bodies, to improve the diversity of clinical trials. This study updates existing baselines regarding demographic disparities, as well as detecting early signs that the situation may be starting to improve.

Methods: Building on an existing dataset, this study collected and analyzed pivotal trial demographic data for drugs and biologics approved by the FDA between 2007 and 2021. Demographic data were collected from applications on the FDA website and, and compared to indication-specific demographic data when available, or US census estimates when they were not. Regression analyses were used to test for significant trends in reporting of demographic data and representation in pivotal trials, as well as the effect of representation on clinical trial duration and FDA review.

Results: Reporting of demographic data has improved significantly for all three demographic categories (sex, racial identity, and ethnic identity) over the observed time period (p < 0.0001). During this time period, overrepresentation of white participants has decreased significantly (p < 0.0001), and representation of Black participants has increased (p = 0.0003). Other racial and ethnic identities did not show significant trends. Representation of demographic subgroups was not significant predictors of trial duration except for the representation of Black participants, which was a negative correlation, indicating that as representation of Black participants increases, trial duration decreases (p = 0.0350).

Keywords: Clinical trials; DEI; Diversity; Representation.