PDUFA VII Pregnancy Demonstration Projects
Funder: United States Food and Drug Administration (FDA)
Leadership: Judy Maro
Year Funded: 2024
Project Summary
To advance optimal approaches to efficient generation of high-quality human safety data for drug products used during pregnancy, the U.S. Food and Drug Administration (FDA) made a commitment, under the latest reauthorization of the Prescription Drug User Fee Act (PDUFA VII), to develop a framework describing how to optimally use data from different types of postapproval pregnancy safety studies.
The FDA will conduct six demonstration projects to address gaps in knowledge about performance characteristics of different study designs. The projects’ findings will contribute to the ongoing development of the draft framework.
Project Details
Projects 1-3 will assess the performance of pregnancy registries and electronic healthcare database studies:
- Project One will focus on a signal detection approach in a relatively common exposure.
- Project Two will focus on a signal evaluation approach in a relatively common exposure.
- Project Three will focus on a single arm safety studies in a relatively rare exposure.
Project 4 will assess the performance and usefulness of major congenital malformations (MCM) as a composite outcome in signal detection and signal evaluation when there is true risk for some but not all specific malformations.
Project 5 will develop a prediction model to estimate the magnitude of exposure.
Project 6 will conduct a chart review for cases of major congenital malformations identified in Projects 1-3.
Optimizing the Use of Postapproval Pregnancy Safety Studies
The Duke-Margolis Center for Health Policy, under a cooperative agreement with the FDA, convened a two-day Public Workshop that gathered stakeholder input on this topic.