It was the year of the original Blackberry, Napster music downloads and AOL Instant Messaging — all ancient technology by today’s standards. Yet 1998 was the most recent year that the FDA approved more than one drug therapy specifically for newborns. Since that time, there has only been one approved, and it is no longer in production. Almost two decades represents an eternity in the fields of science, medicine and technology. So why don’t we have more and newer drugs to improve survival for newborns?
Obstacles to care
One obstacle is the lack of clinical trials for premature newborns. The solution is not as easy as it may seem. Adequate funding, appropriate facilities and knowledgeable clinicians are necessary to begin studies. Parents must also be willing to let their babies participate in research to test innovative therapies designed expressly for neonates.
In the United States, approximately 200,000 newborns annually are treated for prematurity, the leading cause of newborn mortality. According to the Newborn Health Initiative, “Among those who survive, one in five faces health problems that persist for life, such as cerebral palsy, intellectual disabilities, chronic lung disease and deafness.”
The hospital-based health care these newborns need is expensive. Treating newborns in neonatal intensive care units costs more than $26 billion per year, nationally. New clinical trials are necessary to address the urgent need for infant-specific treatments that can help decrease hospital stays and improve long-term health.
Attempts at progress
Meanwhile, clinicians are stuck trying to scale techniques, devices and drugs designed for adults to address the needs of premature newborns. The approach does not work. Although many adults may be big babies, babies are not little adults. Precision decreases and risk increases.
For example, dosing of many medications is based on a baby’s weight. The common practice of “dosing down” a medicine for a lower weight can be fraught with miscalculation and lead to administration of an inaccurate amount of medication. Given the tiny size of some newborns, exact, individualized dosing can mean the difference between proper treatment and an overdose.
Congress has introduced legislation to stimulate the development of safe and effective drugs for neonates. The Promoting Life-Saving New Therapies for Neonates Act of 2017 will reward drug sponsors who successfully develop new products for neonates with a one-year extension of their exclusivity period on another drug. But, researchers will need to trial these drugs on infants for any of these prospective therapies to come to fruition.
Increased awareness can help new parents understand that participating in a clinical trial is not just for the very desperate or the terminally ill. Clinical trials allow people of all ages and stages of life to invest in the health of future generations.
Parents often describe their newborn as a gift. We must see clinical trial participation as a gift that keeps giving — protecting these precious newborns, providing appropriate therapies and allowing them to thrive.