Background
Annually, approximately 200,000 newborns in the United States require admission to a neonatal intensive care unit for treatment of prematurity, costing more than 26 billion dollars per year. Prematurity is the leading cause of newborn mortality and the second leading cause of infant mortality. 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.
Federal legislation implementing a combination of pediatric incentives and obligations has led to an increase in pediatric studies submitted to the FDA, resulting in over 500 product labeling changes since the enactment of the Food and Drug Administration Modernization Act (FDAMA) in 1997. Despite these policy efforts, few drug labeling changes have included neonates (premature and full term infants up to 28 days of age); and the last new drug for neonates was approved in 1999.
Current incentives have not been sufficient to stimulate innovation for the neonatal population due to numerous challenges. For example, very few patients are available for study at any given time so it is difficult and costly to get sufficient numbers. The patients are also very sick so it is difficult to get informed consent from caregivers. Furthermore, many conditions for which drug therapy is needed in the neonatal population do not occur in older children and adults.
Federal legislation implementing a combination of pediatric incentives and obligations has led to an increase in pediatric studies submitted to the FDA, resulting in over 500 product labeling changes since the enactment of the Food and Drug Administration Modernization Act (FDAMA) in 1997. Despite these policy efforts, few drug labeling changes have included neonates (premature and full term infants up to 28 days of age); and the last new drug for neonates was approved in 1999.
Current incentives have not been sufficient to stimulate innovation for the neonatal population due to numerous challenges. For example, very few patients are available for study at any given time so it is difficult and costly to get sufficient numbers. The patients are also very sick so it is difficult to get informed consent from caregivers. Furthermore, many conditions for which drug therapy is needed in the neonatal population do not occur in older children and adults.
The Solution
Fortunately, legislation has been recently introduced to stimulate drug development specifically for the neonatal population. Specifically, the legislation will:
- Close the treatment gap by stimulating the development of safe and effective drugs for a challenging and neglected pediatric population.
- Ensure that new neonatal drugs address the most critical needs in the neonatal population by collaborating with multiple stakeholders, such as the NIH, the Critical Path Institute, and patient advocacy groups, to identify priority conditions.
- Create a new incentive model by providing a transferrable "exclusivity voucher" to drug sponsors who successfully develop products for neonates. This voucher would enable the product sponsor to extend the exclusivity period on another drug by one year.
