TY - JOUR
T1 - Ten-year trends in enrollment of women and minorities in pivotal trials supporting recent us food and drug administration approval of novel cardiometabolic drugs
AU - Khan, Muhammad Shahzeb
AU - Shahid, Izza
AU - Siddiqi, Tariq Jamal
AU - Khan, Safi U.
AU - Warraich, Haider J.
AU - Greene, Stephen J.
AU - Butler, Javed
AU - Michos, Erin D.
N1 - Funding Information:
S.J.G. has received a Young Investigator Award from the Heart Failure Society of America/Emergency Medicine Foundation Acute Heart Failure, funded by Novartis; has received research support from Amgen, AstraZeneca Bristol-Myers Squibb, and Novartis; serves on an advisory board for Amgen Cytokinetics; and serves as a consultant for Amgen and Merck. J.B. is a consultant for Abbott, Amgen, Applied Therapeutics, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CVRx, Janssen, LivaNova, Luitpold, Medtronic, Merck, Novartis, Relypsa, and Vifor. The remaining authors have no disclosures to report.
Funding Information:
Dr Michos is supported by the Amato Fund for Women's Cardiovascular Health Research at Johns Hopkins University.
Publisher Copyright:
© 2020 The Authors.
PY - 2020/6/2
Y1 - 2020/6/2
N2 - BACKGROUND: In 1993, the US Food and Drug Administration established guidelines to increase diversity by sex and race/ ethnicity of participants in clinical trials supporting novel drug approvals. In this study we investigated the 10-year trends of participation of women and minorities in pivotal trials supporting approval of new molecular entities in cardiometabolic drugs from January 2008 to December 2017. METHODS AND RESULTS: A list of new molecular entities was abstracted from publicly available data at Drugs@Fda. Sex and race/ethnicity data were collected from trial publications. Linear regression analysis was performed to assess the relation between drug approval year and proportion of women and minorities enrolled. Thirty-five novel cardiovascular (n=24) and diabetes mellitus (n=11) drugs were approved by the US Food and Drug Administration during the study period. The median number of participants supporting each drug was 5930 (interquartile range, 3175–10 942). Women represented 36% (n=108 052) of trial participants (n=296 163). Women were underrepresented compared with their proportion of the disease population in trials of coronary heart disease (participation-to-prevalence ratio, 0.52), heart failure (participation-to-prevalence ratio, 0.58), and acute coronary syndrome (participation-to-prevalence ratio, 0.68). Among trial participants, 81% were white, 4% black, 12% Asian, and 11% Hispanic/Latino. There was no significant association between enrollment of women (P=0.29) or under-represented minorities (P=0.45) with the drug approval year. CONCLUSIONS: Over the past decade (2008–2017), women and minorities, particularly blacks, have continued to be inad-equately represented in pivotal cardiometabolic clinical trials that support US Food and Drug Administration approval of new molecular entities. This may have major implications in determining efficacy of such therapies in these groups, and may impair generalizability of trial results to routine clinical practice.
AB - BACKGROUND: In 1993, the US Food and Drug Administration established guidelines to increase diversity by sex and race/ ethnicity of participants in clinical trials supporting novel drug approvals. In this study we investigated the 10-year trends of participation of women and minorities in pivotal trials supporting approval of new molecular entities in cardiometabolic drugs from January 2008 to December 2017. METHODS AND RESULTS: A list of new molecular entities was abstracted from publicly available data at Drugs@Fda. Sex and race/ethnicity data were collected from trial publications. Linear regression analysis was performed to assess the relation between drug approval year and proportion of women and minorities enrolled. Thirty-five novel cardiovascular (n=24) and diabetes mellitus (n=11) drugs were approved by the US Food and Drug Administration during the study period. The median number of participants supporting each drug was 5930 (interquartile range, 3175–10 942). Women represented 36% (n=108 052) of trial participants (n=296 163). Women were underrepresented compared with their proportion of the disease population in trials of coronary heart disease (participation-to-prevalence ratio, 0.52), heart failure (participation-to-prevalence ratio, 0.58), and acute coronary syndrome (participation-to-prevalence ratio, 0.68). Among trial participants, 81% were white, 4% black, 12% Asian, and 11% Hispanic/Latino. There was no significant association between enrollment of women (P=0.29) or under-represented minorities (P=0.45) with the drug approval year. CONCLUSIONS: Over the past decade (2008–2017), women and minorities, particularly blacks, have continued to be inad-equately represented in pivotal cardiometabolic clinical trials that support US Food and Drug Administration approval of new molecular entities. This may have major implications in determining efficacy of such therapies in these groups, and may impair generalizability of trial results to routine clinical practice.
KW - Cardiometabolic drugs
KW - Clinical trials
KW - Minorities
KW - Women
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U2 - 10.1161/JAHA.119.015594
DO - 10.1161/JAHA.119.015594
M3 - Article
C2 - 32427023
AN - SCOPUS:85085904669
SN - 2047-9980
VL - 9
JO - Journal of the American Heart Association
JF - Journal of the American Heart Association
IS - 11
M1 - e015594
ER -