The US Food and Drug Administration (FDA) has debuted its final industry guidance on collecting post-marketing safety data for both drugs and biologics in pregnant individuals.
The framework, which aims to create pregnancy safety information that drugmakers can include on medicine labels, touches upon how sponsors can best design a pregnancy registry. The agency also details how to approach case-control studies, electronic health record (EHR)-guided complementary studies and population-level surveillance protocols in the post-marketing context.
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When running these pregnancy studies, the FDA advises sponsors to develop flexible, multi-pronged recruitment and retention strategies that focus on early enrolment. To achieve this, the agency recommends engaging with patient advocacy groups and medical societies to boost awareness, while communicating directly with patients to encourage continued participation.
Many medications that could benefit pregnant women are not offered to them due to a lack of safety data. The FDA’s recommendations outline better ways to collect safety information after approval and understand potential risks to the mother or developing foetus.
The guidance also discusses how sponsors should incorporate statistics into their safety studies, with the FDA advising operators to focus on prespecified, descriptive statistics that effectively summarise patient characteristics. When performing statistical analyses, the FDA suggests that sponsors perform separate analyses for each pregnancy outcome – including events like miscarriage, stillbirth, termination and live birth.
Alongside these suggestions, the FDA recommends that operators seek input from paediatric, statistics, genetics and obstetrics experts to guide operations, and that companies consider best practices for observational research and real-world data collection.
According to the FDA’s commissioner, Marty Makary, the ‘Postapproval Pregnancy Safety Studies’ framework will allow companies to give healthcare providers more clear and reliable information – helping them to better counsel pregnant patients and make informed decisions on treatment.
The FDA debuts this guidance amid swirling rumours around Makary’s departure from the agency, as US President Donald Trump has reportedly signed off on the commissioner’s ousting after months of turmoil within the agency, as per a report from the Wall Street Journal. The FDA or the White House are yet to confirm this decision.
Closing the pregnancy knowledge gap
The FDA has prioritised the Postapproval Pregnancy Safety Studies framework because pregnant women still remain largely excluded from clinical trials, with the European Medicines Agency (EMA) estimating that less than 0.4% of all clinical trials currently submitted within the EU region include pregnant people, and only 0.1% are lactating, as per data from its Clinical Trials Information System (CTIS).
This disparity has resulted in notable knowledge gaps around treatment efficacy and safety within this patient population, which has led several global regulators, including the FDA, the EMA and the World Health Organization (WHO) to call for better inclusion of pregnant individuals in clinical trials and research.
In previous conversation with Clinical Trials Arena, sister publication to Pharmaceutical Technology Sheila Diamond, director of scientific engagement at Medidata, noted that closing the pregnancy gap in this context will require “multi-stakeholder collaboration” and adaptive trial designs incorporating real-time adjustments.
