FDA today proposed a rule that would ensure that women are not excluded from participating in early studies of drugs and biologics to treat life-threatening diseases just because of their reproductive potential. The following may be used to respond to inquiries.
In a notice to be published in tomorrow's Federal Register, the agency is requesting public comment on a proposal that would prevent the routine exclusion of either men or women from clinical studies because of potential risks of toxicity to offspring or reproductive organs.
The proposed rule would permit FDA to place a trial on clinical hold (not allow the study to be conducted or to continue) if a sponsor proposes to exclude or is excluding women from a trial for inappropriate reasons.
FDA's proposal reflects a significant evolution of thought about participation of women of reproductive potential in clinical studies during the past two decades, and is in keeping with the agency's l993 guideline encouraging women's participation in all phases of clinical trials.
FDA prepared this proposal after the National Task Force on AIDS Drug Development and the Presidential Advisory Council on HIV/AIDS recommended that women with HIV or AIDS, particularly those of reproductive potential, not be excluded from investigational trials intended to study a therapy for a life-threatening disease. Many of the women who are affected by HIV and AIDS are young women with reproductive potential.
The council's recommendation, along with limited surveys of some applications showing the exclusion of women of reproductive age in some clinical trials, including some trials of drugs for life-threatening illness, led to today's proposal.
In developing this proposal FDA focused on several important factors: First, FDA is committed to expanding access to and accelerating approval of new therapies for life-threatening diseases. FDA recognizes that physicians and patients are generally willing to accept greater potential risks or side effects from medical products to treat life-threatening diseases.
Second, FDA believes that patients, including women of reproductive potential, in concert with their partners and doctors, can make their own risk/benefit decision when provided with thorough information about risks and potential benefits by the clinical investigator. The informed consent process, required by law for participation in all studies, provides information intended to describe known and potential risks, including potential risks to the embryo or fetus, should a woman become pregnant.
By encouraging diversity at all stages of a drug's development, FDA hopes that drug sponsors will generate better data about how the drug will affect the populations that will receive it once it is marketed. The agency believes it is important to identify important information about a drug during the investigational phase, such as dosages for different age groups, genders, and racial subgroups, and to use that information to refine labeling information, patient selection,and dose selection.
It is important to note that the proposed rule would not require specific recruitment goals for women, nor would it apply to all clinical trials. For example, it would not apply to:
-- Clinical trials designed to look at healthy volunteers exclusively;
--trials designed to test drugs for special circumstances, such as in gender specific populations such as drugs for prostate cancer; or
-- trials designed exclusively for men so long as a companion study in women of reproductive potential has been planned or is being conducted.
Food and Drug Administration U.S. Department of Health and Human Services Public Health Service 5600 Fishers Lane Rockville, MD 20857
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