DIVERSITY IN CLINICAL TRIALS: A REVIEW OF DRUGS APPROVED BY THE FDA IN 2024
Author(s)
Sophia Cassim, BA, Abigail Wright, PhD, Foluso O Agboola, MPH, MD.
Institute for Clinical and Economic Review, Boston, MA, USA.
Institute for Clinical and Economic Review, Boston, MA, USA.
OBJECTIVES: To report and discuss the clinical trial diversity in pivotal trials of drugs approved by the US Food and Drug Administration (FDA) in 2024 using the Clinical trial Diversity Rating (CDR) tool developed by the Institute for Clinical and Economic Review (ICER).
METHODS: This project examined the pivotal trials included in the FDA approval packages for novel drugs approved by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research in 2024. Clinical trial demographic data were abstracted from the FDA Drug Trial Snapshots, which provides aggregate data by drug. Clinical trial diversity was assessed using the CDR tool, which quantitatively compares trial participant characteristics to disease-specific prevalence estimates. Using pre-defined thresholds, each drug was assigned a rating of good, fair, or poor based on the diversity of its trial participants across demographic characteristics: race/ethnicity, sex, and age (older adults).
RESULTS: We evaluated the pivotal trials of 54 novel drugs. The FDA Drug Trial Snapshots reported data on trial participants aged 65 and older (data not typically available in clinical trial manuscripts), enabling assessment of this demographic characteristic. The representation of sex and age was predominantly rated as good. However, representation of race/ethnicity was predominantly rated as fair, with only a few achieving a good rating. There was significant underrepresentation of Black/African American and Hispanic participants. This was indicated by a “Participant to Disease-prevalence Representation Ratio” (PDRR) of less than 0.8 in most trials, only 10% (6/54) had a PDRR greater than 0.8 for Black/African American participants, and 19% (10/54) for Hispanic participants.
CONCLUSIONS: The CDR tool can objectively shed light on the diversity of participants across race, age, and sex within pivotal trials using disease-specific prevalence estimates. The underrepresentation of particular demographic groups underscores the importance of continued discussion on clinical trial diversity.
METHODS: This project examined the pivotal trials included in the FDA approval packages for novel drugs approved by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research in 2024. Clinical trial demographic data were abstracted from the FDA Drug Trial Snapshots, which provides aggregate data by drug. Clinical trial diversity was assessed using the CDR tool, which quantitatively compares trial participant characteristics to disease-specific prevalence estimates. Using pre-defined thresholds, each drug was assigned a rating of good, fair, or poor based on the diversity of its trial participants across demographic characteristics: race/ethnicity, sex, and age (older adults).
RESULTS: We evaluated the pivotal trials of 54 novel drugs. The FDA Drug Trial Snapshots reported data on trial participants aged 65 and older (data not typically available in clinical trial manuscripts), enabling assessment of this demographic characteristic. The representation of sex and age was predominantly rated as good. However, representation of race/ethnicity was predominantly rated as fair, with only a few achieving a good rating. There was significant underrepresentation of Black/African American and Hispanic participants. This was indicated by a “Participant to Disease-prevalence Representation Ratio” (PDRR) of less than 0.8 in most trials, only 10% (6/54) had a PDRR greater than 0.8 for Black/African American participants, and 19% (10/54) for Hispanic participants.
CONCLUSIONS: The CDR tool can objectively shed light on the diversity of participants across race, age, and sex within pivotal trials using disease-specific prevalence estimates. The underrepresentation of particular demographic groups underscores the importance of continued discussion on clinical trial diversity.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
SA23
Topic
Study Approaches
Disease
No Additional Disease & Conditions/Specialized Treatment Areas