Journal Article
Measuring Clinical Benefit in Neonatal Randomized Clinical Trials: Challenges and Opportunities
Many of the therapeutics used in neonatal intensive care units (NICUs) are not specifically labeled or approved for use in neonates and there is a lack of research and development targeting neonatal conditions. There are numerous inherent challenges in conducting trials that have impeded the development of safe and effective therapies for neonates. Measuring clinical benefit in response to treatment is one particular and crucial challenge that researchers and clinicians face in clinical trials for neonatal therapies.
The Duke–Margolis Center for Health Policy, under a cooperative agreement with the FDA, held a public workshop on March 23, 2023, to better understand the challenges in measuring clinical benefit in neonatal clinical trials and to discuss ways to optimize methods for establishing efficacy of medical products intended for use in neonates. This paper summarizes the key takeaways from the discussions among researchers, clinicians, industry, regulators, and patient-family advocates on the challenges, current approaches, and promising new approaches to measuring clinical benefit in neonatal randomized controlled clinical trials, including next steps for capturing meaningful clinical outcomes to support regulatory decisions for neonatal therapeutics.
The Duke–Margolis Center for Health Policy, under a cooperative agreement with the FDA, held a public workshop on March 23, 2023, to better understand the challenges in measuring clinical benefit in neonatal clinical trials and to discuss ways to optimize methods for establishing efficacy of medical products intended for use in neonates. This paper summarizes the key takeaways from the discussions among researchers, clinicians, industry, regulators, and patient-family advocates on the challenges, current approaches, and promising new approaches to measuring clinical benefit in neonatal randomized controlled clinical trials, including next steps for capturing meaningful clinical outcomes to support regulatory decisions for neonatal therapeutics.
