FDA Releases Guidance on Pregnancy Safety Studies for Drug Development

The FDA released final guidance aimed at bridging the data gap regarding medication safety during pregnancy. The document titled, "Postapproval Pregnancy Safety Studies," provides a comprehensive framework for industry sponsors to design investigations that generate much-needed human safety data in the post-market setting.^1,2

For pharmacists, this shift directly impacts the reliability and depth of information available in drug labeling for patient counseling. The FDA emphasized that clinicians need clear, reliable information to help patients make informed treatment decisions during pregnancy.^1,2

“Hopefully this guidance will result in better data to inform the relevant sections of the label, including the Pregnancy section,” Natalie DiPietro Mager, PharmD, PhD, professor of pharmacy practice at Ohio Northern University, said in an interview. “Pharmacists can use this information to provide more robust counseling to patients about the potential risks and benefits of medication use during pregnancy.”

History of Pregnant Women in Clinical Trials

Historically, pregnant women were excluded from clinical trials to protect the fetus from potentially harmful exposures, which inadvertently shifted the risk of unknown adverse effects (AEs) to the postmarketing phase, where real-world use occurs. With approximately 3.6 million births in the United States annually and nearly 42% of pregnancies being unintended, the potential for inadvertent drug exposure before a woman realizes she is pregnant is a pressing public health concern, according to the authors.^1,2

Former Center for Drug Evaluation Research Director Tracy Beth Hoeg noted that many medical products are currently recommended by providers despite a lack of sufficient clinical trial data to assess pregnancy-related risks. The new guidance finalizes a 2019 draft and provides specific recommendations on how postmarketing data can be leveraged to promptly identify these risks.^1-3

“Given that most safety data about medication use in pregnancy is currently obtained through post-marketing surveillance, it is important for pharmacists to submit high-quality adverse event reports if their patient experiences an adverse event and encourage pregnant patients to enroll in pregnancy exposure registries,” DiPietro Mager said.

About the New Guidance

The guidance outlines 3 primary methodologies for data collection: case reports and case series, prospective pregnancy registries, and complementary studies utilizing electronic health care data. Spontaneous AE reports remain a vital source of initial safety signals, though they often suffer from limitations such as underreporting and incomplete information. Pharmacists and other health care providers play a crucial role in identifying unique patterns of congenital malformations through high-quality case reporting, which often serves as the start of an iterative process for deeper safety investigations.¹

Pregnancy registries offer a more structured approach by actively enrolling patients exposed to a drug before birth outcomes are known, which helps reduce recall bias and exposure misclassification. Although these registries are efficient for collecting data on rare exposures, they frequently face significant challenges related to participant recruitment and retention.

To address these limitations, the FDA encourages the use of complementary studies that leverage real-world data from electronic health records and medical claims. These large-scale electronic health care databases allow for mother-infant linkage and can help evaluate rare exposures or outcomes that smaller registries might miss.

The Pharmacist’s Role

“Women of childbearing age should ask their pharmacist about the safety profile of the medication during pregnancy. If it is a medication that may cause fetal harm or increase the risk of adverse pregnancy outcomes, she should talk to her pharmacist about using an effective form of contraception to prevent pregnancy while taking that medication,” DiPietro Mager said.

She added that women who are using contraceptive methods should be using them correctly and consistently, emphasizing the role of the pharmacist in education and counseling.

The goal of these studies is to generate human safety data that can be included in the pregnancy subsection of drug labels. This updated labeling provides pharmacists with standardized information on potential risks, such as major congenital malformations, preterm delivery, or measures of fetal growth deficiency.

The guidance also suggests that sponsors should include registry contact information, such as toll-free numbers or websites, in product labeling to facilitate patient-initiated recruitment and provider awareness. By refining how post-approval data is collected and analyzed, the FDA aims to ensure that the medication counseling provided at the pharmacy counter is grounded in robust, clinically relevant human evidence.^1,2

“Women experiencing [adverse] effects should talk to their pharmacist, and there should be a plan in place when wanting to switch methods to minimize the chance of an unintended pregnancy,” DiPietro Mager concluded. “Women should be proactive in planning when they want to conceive and work with their pharmacist and other health care providers in advance to see if there are modifications that can be made to their medication regimen to minimize the risk.”

READ MORE: Women’s Health Resource Center

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REFERENCES

1. US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research, Center for Biologics Evaluation. Postapproval pregnancy safety studies. May 2026. Accessed May 18, 2026. https://www.fda.gov/media/124746/download

2. FDA issues guidance to improve collection of pregnancy safety data for drugs and biologics. News release. May 8, 2026. Accessed May 18, 2026. https://www.fda.gov/news-events/press-announcements/fda-issues-guidance-improve-collection-pregnancy-safety-data-drugs-and-biologics

3. Federal Registry. Postapproval pregnancy safety studies; guidance for industry; available. May 11, 2026. Accessed May 18, 2026. https://www.federalregister.gov/documents/2026/05/11/2026-09268/postapproval-pregnancy-safety-studies-guidance-for-industry-availability