For many conditions affecting newborn babies, or neonates, there is an unmet need for safe and effective therapeutics. This is due in part to the unique challenges of conducting clinical trials in neonates. Researchers often struggle to define study outcomes that are clinically meaningful and relevant to providers, patients, and families. This meeting aims to promote discussion and collaboration between researchers, clinicians, industry, and regulators on efficacy endpoint considerations for neonatal randomized clinical trials, and to provide a forum for patients and families to share what clinical benefits they find important.

Real-world evidence (RWE) generated from real-world data (RWD) is increasingly being utilized to address scientific and regulatory questions at the Food and Drug Administration (FDA), including both product safety and effectiveness. Evaluating RWE for regulatory use, however, depends on a robust causal inference framework, and there is growing stakeholder interest to understand how methodological advances with negative controls can improve causal inference.

On March 8, 2023, the Duke-Margolis Center for Health Policy, under cooperative agreement with the FDA, will convene a workshop on how negative controls could support new methodological approaches for causal inference in the Sentinel System. Discussion will inform the methods development projects aimed at: 1) establishing empirical methods to automate the negative controls identification in Sentinel and integrate it into the Sentinel Initiative tools, and 2) developing approaches to use a double-negative control adjustment to reduce unmeasured confounding in studying effectiveness of vaccines. This workshop will fulfill a Prescription Drug User Fee Act (PDUFA) VII Commitment.

This public workshop is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award [U19FD006602] totaling $4,241,714 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.