FDA is receiving inquiries about a planned new policy on the inclusion of women in clinical trials that manufacturers must conduct before new drugs or biological products can be approved.
A new FDA guideline will encourage companies both to include women in reasonable numbers in studies and provide the agency information about any significant differences found between men and women in their responses to drugs. It will also change a policy in effect since l977 that has resulted in the exclusion of most women capable of becoming pregnant from participating in the earliest phases of clinical trials.
The agency believes that the new policy will permit earlier detection of gender differences that could lead to better selection of doses and monitoring procedures in later studies. The gender-specific data will also result in better labeling information for prescribing physicians.
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Based on several surveys of new drug applications, FDA found that women have generally participated in substantial numbers in clinical trials, usually reflecting the gender prevalence of the disease for which the drugs were being studied. However, women capable of becoming pregnant have been excluded in many cases from phase I trials, the earliest studies in humans intended to give a preliminary assessment of how well the drug is tolerated, and from early phase II trials, the first controlled studies of drug efficacy.
In addition, results of animal reproduction studies have been required before women could be included in trials. This policy was intended to protect a fetus from any possibility of unnecessary exposure to potentially toxic agents. However, the policy has not excluded women capable of becoming pregnant from trials of drugs for life-threatening diseases such as AIDS and cancer.
In recent years, the policy has been re-evaluated and there has been a growing belief that it should be changed. First, the policy has been viewed by some as being paternalistic because it denies women the right to make their own decisions on risks they wish to take. Second, while FDA still believes that protecting fetuses from potentially toxic agents is an important principle, the agency has concluded that fetal protection can be achieved by measures short of excluding women from early trials. The new guidelines call for precautions such as pre-enrollment pregnancy testing; use of contraception and behavioral measures that minimize the possibility of pregnancy; and providing women with appropriate information about potential risks.
FDA is also planning to address the issue of the participation of pregnant women in clinical trials.