Women Still Underrepresented in Drug Trials, Report Says

Although the FDA has made efforts to increase the number of female participants in drug trials, women "continue to be underrepresented" and differences in how the sexes respond to new treatments continue to be "unexamined during drug development," a new General Accounting Office report released yesterday states. In 1992, the GAO found that the FDA did not ensure representation of women in drug studies or that drug studies accounted for sex differences. To that end, a 1993 federal guidance recommended that clinical studies include enough men and women to detect sex differences. In 1998, the Clinton administration issued a federal regulation calling for already collected safety and efficacy data to be presented separately for men and women. In addition, the regulation required investigational new drug annual reports to include the number of men and women who participated in the trial. Further, a 2000 federal regulation permitted the FDA to halt drug studies for life-threatening conditions if "otherwise eligible men or women are excluded from participation in studies based solely on their reproductive potential." As a follow up to its 1992 report, the GAO reviewed new drug applications submitted to the FDA between Aug. 10, 1998, and Dec. 31, 2000, and summary documents submitted by the drug's sponsor. GAO researchers also randomly sampled 100 annual reports for new drugs and interviewed FDA officials, pharmacology and drug safety experts and pharmaceutical industry representatives. The GAO report found that overall, women represented 52% of study participants, but the proportion of female participants "varied greatly" at different stages of drug development. For instance, women accounted for 22% of participants in initial, small-scale safety trials and for more than 50% of participants in larger trials.

The GAO report found that nearly one-third of summary documents failed to meet the requirement that safety and efficacy information be presented by sex, and 39% of annual reports did not include required demographic information. The FDA "has not effectively overseen the presentation and analysis of data related to sex differences in drug development," the report states, noting that there exists no system to record and track women's participation in studies or to monitor companies' compliance with federal regulations. Further, the report says that the FDA fails to "routinely review" required counts of demographic data by sex and lacks procedures that would determine whether new drug applications "adequately discuss sex differences." The report recommends that the FDA "implement management tools" to ensure effective enforcement of current regulations and that reviewers "consistently and systematically document and discuss sex differences in their written reviews" (GAO, "Women's Health: Women Sufficiently Represented in New Drug Testing, But FDA Oversight Needs Improvement," 7/16). In repsonse to the report, the FDA said yesterday that it is "taking steps to further improve oversight of both gender and racial differences in medication" (AP/Las Vegas Sun, 8/6). The GAO report, GAO-01-754, can be found on the GAO Web site under the new reports section.