FDA finalizes guidance for postapproval pregnancy safety studies

The FDA has issued final guidance, Postapproval Pregnancy Safety Studies, detailing how sponsors and investigators should design investigations to assess the safety of drug and biological products during pregnancy. The May 2026 document aims to standardize the collection of clinically relevant human safety data for inclusion in the "Pregnancy" subsection of product labeling, according to a news release from the federal agency.^1,2

With approximately 3.6 million births annually in the United States and 41.6% of pregnancies being unintended in 2019, many patients are inadvertently or necessarily exposed to medical products, stated the FDA in the guidance. Because pregnant women are historically excluded from pre-approval clinical trials, the FDA often requires postmarketing studies to identify potential risks to the developing fetus or the mother.

Diverse methodologies for data collection

The guidance highlights 3 general approaches for post-approval safety assessment: case reports, pregnancy registries, and complementary data sources. Each method offers unique clinical insights:

• Case reports and series: Used for identifying signals of rare outcomes. While spontaneous reporting has limitations like underreporting, well-documented cases are critical for detecting distinctive patterns of congenital malformations.
• Pregnancy registries: These prospective observational studies enroll women before the pregnancy outcome is known. This reduces recall bias and allows for detailed patient-level data collection, including physical examinations of the newborn.
• Complementary studies: These utilize electronic health data, such as insurance claims and electronic health records, to evaluate outcomes in larger populations. They are particularly useful for validating safety signals and assessing outcomes across multiple healthcare settings.

New technical recommendations

The guidance introduces specific recommendations for study design, emphasizing the need for concurrent internal comparator groups—pregnant women with the same disease who are unexposed to the drug. It also outlines 3 critical windows for data collection: drug exposure prior to pregnancy (to assess early loss or ectopic pregnancy), exposure during pregnancy (to monitor maternal complications like preeclampsia), and postpartum monitoring (to track recovery and lactation safety).

FDA Commissioner Marty Makary, MD, MPH, stated that the guidance provides clinicians with more useful information for counseling patients.

“Pregnant women and their healthcare providers need clear, reliable information to make informed treatment decisions,” said Makary in a statement. “This guidance supports better ways to collect safety data, so clinicians have more useful information when counseling patients during pregnancy.”

Tracy Beth Hoeg, MD, PhD, Acting Director of the Center for Drug Evaluation and Research, added that the framework allows for prompt identification of pregnancy-related risks.

“Currently many medical products may be recommended to pregnant women by healthcare providers in spite of the fact that data from the clinical trials used for FDA approval were insufficient to assess safety during pregnancy,” said Hoeg.

“This guidance provides specific recommendations about how post-marketing data can be leveraged and studies can be designed so clinicians and the public can be better informed about product safety and pregnancy-related risks can be more promptly identified.”

To view the full guidance, as published by the FDA on May 8, 2026, click here.

References:

1. FDA Issues Guidance to Improve Collection of Pregnancy Safety Data for Drugs and Biologics. FDA. Published May 8, 2026. Accessed May 8, 2026. https://content.govdelivery.com/accounts/USFDA/bulletins/4166d77
2. Postapproval Pregnancy Safety Studies. US Department of Health and Services. FDA. Published May 8, 2026. Accessed May 8, 2026. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/postapproval-pregnancy-safety-studies