Measuring Clinical Benefit in Neonatal Randomized Clinical Trials: Challenges and Opportunities

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.