Author + information
- Received November 15, 2017
- Revision received January 26, 2018
- Accepted February 11, 2018
- Published online April 30, 2018.
- Pamela E. Scott, PhD, MAa,∗ (, )
- Ellis F. Unger, MDb,
- Marjorie R. Jenkins, MD, MEdHPa,
- Mary Ross Southworth, PharmDb,
- Tzu-Yun McDowell, PhDb,
- Ruth J. Geller, MHSa,
- Merina Elahi, BSa,
- Robert J. Temple, MDb and
- Janet Woodcock, MDb
- aU.S. Food and Drug Administration Office of Women’s Health, Silver Spring, Maryland
- bU.S. Food and Drug Administration Center for Drug Evaluation and Research, Silver Spring, Maryland
- ↵∗Address for correspondence:
Dr. Pamela Scott, U.S. Food and Drug Administration Office of Women’s Health, 10903 New Hampshire Avenue, Building 32, Room 2326, Silver Spring, Maryland 20993.
Background Concerns exist that women are underrepresented in trials of cardiovascular medications.
Objectives The authors sought to examine women’s participation and the reported safety and efficacy by gender for pivotal cardiovascular disease (CVD) trials submitted to the U.S. Food and Drug Administration (FDA) supporting marketing applications.
Methods On the basis of publicly available FDA reviews, the authors assessed enrollment of women in trials supporting 36 drug approvals from 2005 to 2015. Prevalence-corrected estimates for the participation of women were calculated as the percentage of women among trial participants divided by the percentage of women in the disease population (participation to prevalence ratio [PPR]), with a range between 0.8 and 1.2 reflecting similar representation of women in the trial and disease population. Sex differences in efficacy and safety were assessed.
Results The proportion of women enrolled ranged from 22% to 81% (mean 46%). The calculated PPR by disease area was within or above the desirable range for atrial fibrillation (0.8 to 1.1), hypertension (0.9), and pulmonary arterial hypertension (1.4); PPR was <0.8 for heart failure (0.5 to 0.6), coronary artery disease (0.6), and acute coronary syndrome/myocardial infarction (0.6). The authors found little indication of clinically meaningful gender differences in efficacy or safety. Gender differences in efficacy or safety were described in labeling for 4 drugs.
Conclusions Women were well represented in trials of drugs for hypertension and atrial fibrillation, and overrepresented for pulmonary arterial hypertension. Representation of women fell below a PPR of 0.8 for trials in heart failure, coronary artery disease, and acute coronary syndrome. Minimal gender differences in drug efficacy and safety profiles were observed.
- cardiovascular diseases/therapy
- clinical trials
- drug efficacy
- drug safety
- Food and Drug Administration
- women’s health
This project was supported, in part, by appointments of Merina Elahi, Ruth Geller, and Dania Shafei to the Research Participation Program at the Office of Women’s Health, U.S. Food and Drug Administration, administered by the Oak Ridge Institute for Science and Education through an interagency agreement between the U.S. Department of Energy and FDA. The views expressed in this paper are those of the authors and do not necessarily reflect the views of the FDA. The authors have reported that they have no relationships relevant to the contents of this paper to disclose.
- Received November 15, 2017.
- Revision received January 26, 2018.
- Accepted February 11, 2018.