New FDA guidance on clinical trial inclusion

Key takeaways:

• Patients who are pregnant or breastfeeding are often excluded, but that creates risk for these patients.
• An expert said it is important to include them when it is safe to do so for better data generalization.

Including pregnant and breastfeeding women in clinical trials is a step in the right direction to gathering better, more generalizable data, according to an expert.

The FDA recently released a draft guidance document on the appropriate inclusion of women who are pregnant and breastfeeding in clinical trials.

“Pregnant and breastfeeding women are often excluded from clinical trials and those who become pregnant while participating in a clinical trial are frequently discontinued from the clinical trial,” according to the guidance. “As a result, pregnancy- as well as breastfeeding-specific information in the product labeling on benefits and risks of medicinal product use is, at best, sparse and treatment decisions need to be made in the absence of this information.”

That has consequences for pregnant and breastfeeding women, like health care providers inadvertently choosing treatments harmful to a patient or pregnancy, according to the guidance.

The draft guidance is open for comment through Sept. 19.

Healio spoke with Eve Espey, MD, MPH, Distinguished Professor and Chair of the University of New Mexico’s Department of Obstetrics and Gynecology, to learn more about the draft guidance and how it will benefit clinical trials.

Healio: Many treatments are understudied in pregnant and lactating patients. What are some specific examples that stand out to you? Why is it particularly important to have more data in these areas?

Espey: For an important recent example, in July, an FDA advisory panel raised concerns about the impact of antidepressants in pregnancy, citing harms to both the pregnant person and the baby. Strong reactions from the medical community, including the American College of Obstetricians and Gynecologists, followed quickly because there are also major concerns about harms related to discontinuing antidepressants or nontreatment of debilitating depression. The reality is that we need more data on the balance of risks and benefits to adequately inform clinician counseling and patient decision-making.

Healio: How do you feel about the FDA releasing this draft guidance?

Espey: I’m glad that the FDA is addressing the inclusion of pregnant and breastfeeding women in clinical trials. Just as a sea change was required to include women in clinical trials and again to include women with other typically excluded characteristics (smoking, high BMI) from contraceptive trials, it is important to replicate real-life circumstances to the extent that it is safe to do so, in order to generate the data that will help with counseling and decision-making.

Healio: What is particularly beneficial about the draft guidance? Will it make an impact?

Espey: If pregnant and breastfeeding women are included in clinical trials, it will allow better generalization of results — ie, the results will apply more broadly than current studies that exclude pregnant and breastfeeding women.

Healio: Is there anything you would change? If so, what?

Espey: The language in the guidance appears appropriately cautious while encouraging inclusion — for example, it covers a situation where pregnancy occurs in a clinical trial participant who is required to use contraception because of a specific concern about the medication, suggesting a review of risks and benefits to continuing the participant in the trial and continuing the medication. This is a big change from prior guidance that would have required immediate removal of the participant simply on account of having become pregnant.

Healio: What other efforts are needed to strengthen research in pregnant and lactating patients?

Espey: More research must be specifically designed to identify the impacts of medications in pregnancy and lactation. Additional research would require more investment in women’s health research in general, which is currently substantially underfunded.

For more information:

Eve Espey, MD, MPH, can be reached at [email protected].

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