Evaluation of worldwide clinical trials by gender: An FDA perspective

Introduction: The US Food and Drug Administration (FDA) has undertaken efforts to promote representation of women in clinical trials. The objectives of this research are to assess women's participation in clinical trials from a global perspective and to analyze the demographic characteristics of clinical trial participants. Methods: FDA's Center for Drug Evaluation and Research-Professional Affairs and Stakeholder Engagement (CDER/PASE) and Office of Women's Health (OWH) collaborated to evaluate demographic data (race, ethnicity, gender, and age) of pivotal trials of New Molecular Entities (NMEs) approved in 2015-2016 by geographic location. One hundred fifty-four pivotal clinical trials supporting 66 NMEs were identified, and the research team analyzed demographic characteristics of 131,749 participants from 70 countries. Results: U.S. sites contributed 31% of the 131,749 study participants. On the country level, the United States contributed the largest number of participants and other individual countries contributed 5% or less of the total trial population. Overall, 43% (n = 56,272) of the 131,747 clinical trial participants were women. Of the 40,833 U.S. participants, 49% were women as compared to 40% of the 90,914 non-U.S. participants. Similar levels of participation were seen after the exclusion of sex-specific drug indications, and by therapeutic area for U.S. and non-U.S. sites. Conclusions: Clinical trials are becoming increasingly multi-national, and the increasing representation of women across countries is promising. FDA approval processes ensure that global data used in the drug approval process meets regulatory standards and that data can be generalized to the U.S. population.