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Highlights

This report reviews the Food and Drug Administration's (FDA) inclusion of women in clinical drug trials. GAO found that women were a majority of the clinical trial participants in the new drug applications (NDA) it examined and that every NDA included enough women in the pivotal studies to be able to statistically demonstrate that the drug is effective in women. Although these findings are welcome, GAO also found three areas of concern. The first is the relatively small proportion of women in early small-scale safety studies. These early studies provide important information on drugs' toxicity and safe dosing levels for later stages of clinical development, and many of the NDAs GAO examined found significant sex differences in a drug's pharmacokinetics, or how it is absorbed, distributed, metabolized, excreted, and concentrated in the bloodstream. Second, GAO is not confident that either NDA sponsors or FDA's reviewers took full advantage of the available data to learn more about the effects of the drug in women and to explore potential sex differences in dosing. This is because NDA summary documents are not required to include analyses of sex differences, and many of them do not. Third, FDA lacks appropriate management systems to monitor how many women are in clinical trials, to be certain that NDAs and investigational new drug applications (IND) annual reports comply with regulations for presenting outcome data by sex and tabulating the number of women included in ongoing trials, and to confirm that its medical officers have adequately addressed sex-related issues in their reviews. Although FDA has taken some promising initial steps to address these deficiencies, it is important that the agency finalize the pilot programs it has underway and give sustained attention to these management issues.

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Recommendations

Recommendations for Executive Action

Agency Affected Recommendation Status
Food and Drug Administration 1. The FDA should adopt management tools that will ensure drug sponsors' compliance with current regulations regarding the presentation of data by sex and that its reviewers' consistently and systematically discuss sex differences in their written reviews of NDAs. Specifically, the Acting Principal Deputy Commissioner, FDA, should promptly implement management tools , such as the proposed demographic worksheet and the standardized template for Medical Officer Reviews, that will allow the agency to determine if NDAs and IND annual reports are in compliance with regulations that mandate the presentation of available safety and efficacy outcome data for women in NDAs and the tabulation of study participants by sex in the annual reports.
Closed - Implemented
In August 2011, FDA reported that it had established a standardized demographic template for the required use of its medical reviewers. FDA reported that it is now routine for analyses to be conducted for subpopulations, including by sex.
Food and Drug Administration 2. The FDA should adopt management tools that will ensure drug sponsors' compliance with current regulations regarding the presentation of data by sex and that its reviewers' consistently and systematically discuss sex differences in their written reviews of NDAs. Specifically, the Acting Principal Deputy Commissioner, FDA, should fully implement the proposed template for Medical Officer Reviews or take other actions to ensure that FDA's medical officers consistently and systematically consider and discuss sex differences in their written reviews of NDAs.
Closed - Implemented
The Food and Drug Administration mandated the use of the clinical review template for collection of demographic data. In addition, training courses have been implemented to ensure that all Medical Review Officers are aware of the regulatory requirements for drug sponsors to report data separately for men and women.

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