Friday, May 13, 2016
Billy Long: Promoting new therapies for newborn care
With approximately 200,000 newborns in the United States requiring neonatal intensive care each year, it’s safe to say that mostly everybody is connected to or directly knows someone – a family member, neighbor, co-worker, or other acquaintance – who has had to experience their child being born premature or ill.
Prematurity can be devastating to parents and their newborns alike. It is the leading cause of newborn mortality and the second leading cause of infant mortality. Furthermore, those children who survive their premature births have a higher susceptibility to lifelong health challenges like cerebral palsy, chronic lung disease, deafness, and myriad intellectual disabilities.
Scientifically, providing care for premature and newborn infants is vastly different than treating any other age group due to their early developmental stage. A neonate – an infant less than four weeks old – is undergoing the rapid generation of new organs and tissue which can cause their reaction to any test treatment to return drastically different results than if it were administered to a more developed person.
This is a problem for current US Food and Drug Administration (FDA) regulatory models, which tie treatment incentives and obligations more tightly to adult medical care than neonatal treatment innovation. This has carved a widening gap between less desirable patient outcomes among neonates and older populations.
The good news is that past legislation to advance care has spurred increased submissions of scientific pediatric research to the FDA. This research has given us an idea of where we’re starting. The bad news, however, is that it’s shown us that most medications being given to preterm infants lack the data to certify their safety and quality – with more than 90 percent not approved by the FDA. Also, few of these drugs were actually used for their intended patient populations and the FDA has been remarkably bogged down when it comes to approving neonatal treatments. In fact, the last time the agency approved a drug for these newborns was 1999.
We need to rethink our total approach in meeting the needs of these children. That’s why I’ve introduced the “Promoting Life-Saving New Therapies for Neonates Act of 2016,” which will stimulate the development of safe and effective drugs for the neonatal field. First, it induces collaboration among medical experts to identify a list of specific priority neonatal caregiver needs, and – based on those priorities – grants authority for the FDA to offer better incentives to neonate therapy developers to stimulate development.
This crucial bill would undo federal barriers to treatment innovation so that researchers and medical experts across the country can find new ways to stay ahead of threats to newborns and reduce preventable infant mortality. Under this new incentive model, medical researchers can begin closing the treatment gap for neonatal patients and their families.
Many of our nation’s infant deaths are preventable and, together, we can make strides toward saving these young lives. The well-being of these children should be our top priority and I will continue fighting in Congress to ensure that they have resources and safe therapy options to overcome their ailments and go on to live healthy lives.