Participation of women in clinical trials for new drugs approved by the food and drug administration in 2000-2002.
OBJECTIVE
This study aimed to track the inclusion of women in clinical trials for new drugs approved by the Food and Drug Administration (FDA) between 2000 and 2002 and to evaluate the extent of analyses by sex.


METHODS
Data were extracted from FDA reviewers' reports, summaries of clinical trials in New Drug Applications (NDAs), and product labeling and organized into a Microsoft Access database. The information collected includes subject enrollment by sex per clinical phase and sex differences in pharmacokinetics, safety, and efficacy as determined by either sponsors or reviewers.


RESULTS
There were 67 New Molecular Entities (NMEs) approved by the FDA between 2000 and 2002. A total of 397,825 subjects were enrolled in 2,323 clinical trials. If 9 sex-specific NMEs are excluded, 297,697 subjects were enrolled in 1,974 clinical trials. Forty-seven percent of participants were male, 49% were female, and 4% of subjects were not specified. Of the 58 sex-nonspecific products in the study, 71% (41 of 58) of sex analyses were performed either by the sponsor or FDA reviewers. Twenty-five NMEs were found to have sex differences in pharmacokinetics, efficacy or adverse events. However, no recommendation was made to adjust dosage based on sex differences.


CONCLUSIONS
The percentages of women and men participating in clinical trials varied by year, phase, and product type. However, the overall participation by women and men was comparable, suggesting an improvement in including more women in clinical trials when compared with the previous FDA study evaluating women's participation from 1995 through 1999. As with the previous study, however, a significant underrepresentation of women in early phase trials and in certain areas, such as cardiovascular products, was observed and continues to be an issue of concern. Lack of appropriate analyses by sex should also be noted as an issue of concern.
Yang
Yongsheng
Y
Food and Drug Administration, Center for Drug Evaluation and Research, Silver Spring, Maryland 20993, USA. yongsheng.yang@fda.hhs.gov
Carlin
Alan S
AS
Faustino
Patrick J
PJ
Motta
Mónica I Pagán
MI
Hamad
Mazen L
ML
He
Ruyi
R
Watanuki
Y
Y
Pinnow
E E
EE
Khan
Mansoor A
MA
D000328:Adult; D017677:Age Distribution; D000368:Aged; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D017277:Drug Approval / Q000706:statistics & numerical data*; D005260:Female; D007722:Health Knowledge, Attitudes, Practice; D006297:Health Services Accessibility / Q000706:statistics & numerical data*; D006805:Human Experimentation / Q000706:statistics & numerical data*; D006801:Humans; D008297:Male; D008875:Middle Aged; D018579:Patient Selection; D017678:Sex Distribution; D014481:United States / Q000453:epidemiology; D014486:United States Food and Drug Administration; D016387:Women's Health; D055815:Young Adult