Program seeks Council approval for an initiative entitled “Neonatal Research Network”.
In the United States, of the approximately 4 million babies born every year, 1 in 10 infants are born preterm, with more than 25,000 of them born before 28 weeks of gestation. Babies born this early have higher rates of death and disability. Data from the Neonatal Research Network’s (NRN) Generic Database shows that 64% of these infants die; those that survive up to 12 hours experience serious, life-threatening conditions, including breathing problems (72% have bronchopulmonary dysplasia), severe brain hemorrhages (29%), vision problems from retinopathy of prematurity (23%), gastrointestinal problems (13% with necrotizing enterocolitis), cerebral palsy, and developmental delays. Only 20% survive without neurodevelopmental impairments. Most of these infants also end up spending months in neonatal intensive care units (NICUs).
In the NICUs, treatments and management strategies vary greatly with limited evidence of efficacy between different practices. For instance, treatment of bronchopulmonary dysplasia involved many combinations of drugs and ventilation techniques. Furthermore, up to 96% of newborns are given off-label drugs – without clinical trials to test their safety and efficacy – particularly in this patient population.
To help address these problems, we propose to continue support for the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)’s Neonatal Research Network (NRN) and update it to adopt the four ‘Guiding Principles” for clinical trial infrastructure as outlined by NICHD leadership (NOT-HD-19-034). The objective of this program is to facilitate the advancement of neonatal care by establishing a network of academic centers and a data coordinating center that, by rigorous patient evaluation using common protocols, can study the required numbers of patients and provide answers more rapidly than individual centers acting alone.
The NRN infrastructure has been and continues to be set up for carefully designed randomized double-blinded placebo controlled as well as management trials with the ability to follow both short-term (clinical effect) and long-term (neurodevelopmental outcome) measures. The infrastructure is also set up for observational, longitudinal studies in the neonatal intensive care unit setting. Many randomized clinical trials involve the need for baseline information regarding disease incidence and outcome, which is available from the generic database of neonates <29 weeks gestation in the current NRN. These data also provide the backbone of results for updating the NICHD Extremely Preterm Birth Outcomes estimator tool.
Importantly, the NRN will also adopt the four ‘Guiding Principles” for clinical trial infrastructure as outlined by NICHD leadership (NOT-HD-19-034):
- Enhancing rigor and reproducibility;
- Promoting greater availability of infrastructure;
- Facilitating data sharing and access to biospecimens; and
- Facilitating greater involvement of diverse populations in clinical trials.
This initiative also aligns with the current NICHD Strategic Plan and its aspiration goal of enhancing the survival and healthy development of preterm infants.
Pregnancy & Perinatology Branch (PPB)
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