Women's Health:

Women Sufficiently Represented in New Drug Testing, but FDA Oversight Needs Improvement

GAO-01-754: Published: Jul 6, 2001. Publicly Released: Aug 6, 2001.

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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.

Recommendations for Executive Action

  1. Status: Open

    Comments: On September 21, 2005, FDA noted that a demographic worksheet was piloted, but it was found not to be useful because the demographic data were not being submitted in a format that readily allows an accurate, standardized, and timely assessment of the number of participants by gender, age, and race. The Center for Drug Evaluation and Research plans to issue guidance to industry within the next year that will describe the preferred submission format of demographic data to facilitate the documentation and evaluation of this information. In September 2007, FDA reported that they are still working on guidance that will describe the preferred submission format of demographic data to facilitate documentation and evaluation of study participants by sex in IND annual reports. In July 2009, no follow up information was provided by the FDA in regard to this recommendation.

    Recommendation: 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.

    Agency Affected: Department of Health and Human Services: Public Health Service: Food and Drug Administration

  2. Status: Closed - Implemented

    Comments: 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.

    Recommendation: 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.

    Agency Affected: Department of Health and Human Services: Public Health Service: Food and Drug Administration

 

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