The Newborn Health Initiative October 2017

Prematurity Often Leads to Lifelong Health Problems 200,000 newborns in the United States require admission to a NICU every year. Prematurity is the leading cause of newborn 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.

Current Incentives Have Done Little to Benefit Preemies Federal legislation implementing a combination of pediatric incentives has led to an increase in pediatric studies submitted to the FDA Over 500 product labeling changes since the enactment of the FDA Modernization Act 1997. Despite these policy efforts, few drug labeling changes have included neonates. The last new drug for neonates was approved in 1999.

Neonates Present Unique Challenges for Innovators Getting informed consent while families are under immense emotional pressure. Very few newborn patients are available for study at any given time. Numerous research sites are often required to participate. Many conditions for which drug therapies are needed in neonates do not occur in older children and adults.

The Promoting Life-Saving New Therapies Act for Neonates Act H.R. 2641 would stimulate drug development for neonates. Stakeholder Engagement: Important provisions would ensure that new drugs address the most critical needs in the neonatal population by collaborating with stakeholders to identify priority conditions. National Institutes of Health Critical Path Institute Patient Advocacy Groups New Incentive Model: The legislation creates a transferrable "exclusivity voucher" to drug sponsors who successfully develop products specifically for neonates.

Director, Newborn Health Initiative Join Us! www.NewbornHealth.org Andrew Rosenberg Director, Newborn Health Initiative arosenberg@thornrun.com