Early testing of new drugs remains mostly a guy's game

Female participation in phase III studies has improved, but required information about gender differences frequently is missing from new drug applications.

By Victoria Stagg Elliott, AMNews staff. Sept. 3, 2001.

More than half of all subjects in large-scale drug studies are now female.

This accomplishment is primarily the result of guidelines and regulations issued by the Food and Drug Administration and National Institutes of Health requiring sex-specific information on safety and efficacy and encouraging increased participation by women.

Gender parity has not, however, been achieved in the smaller phase I trials, where only 22% of subjects are female. Many new drug applications to the FDA also lack the gender-specific information required by the agency, according to a report issued by the General Accounting Office last month. And this continuing disconnect is a deficiency that still must be overcome.

"If you don't analyze the sex differences, it doesn't make any difference to enroll women," said Denise Faustman, MD, PhD, associate professor of medicine at Harvard Medical School, Boston. "And phase III, from a scientific viewpoint, is way too late."

The GAO report is part of an increasing body of work critical of how clinical trials are designed and how the information that results from them is presented in respect to gender differences. A previous GAO report concluded that in many cases in which drugs were withdrawn from the market because of adverse events, these events were more dangerous for women than for men. A report released by the Institute of Medicine in April found that sex differences should be considered at all levels of biomedical research.

"This is a global problem at all levels of the investigative community from the basic science field all the way to the clinical trial field," said Dr. Faustman. "It spans all disciplines of investigation, and the FDA needs to enforce its rules." [...]