May 11, 2016

Lujan and Long Introduce Bipartisan Legislation to Encourage Development of Life-Saving Treatments for Newborns

Washington, D.C. – Congressman Ben Ray Luján of New Mexico’s Third District has partnered with Congressman Billy Long of Missouri to introduce bipartisan legislation this week to encourage the development of life-saving treatments for newborns.  The bill addresses the fact that no new drugs developed specifically to treat newborns have been approved since 1999.  The Promoting Live-Saving New Therapies for Neonates Act aims to spur development of drugs that can improve outcomes for devastating neonatal conditions.

Approximately 200,000 newborns in the United States annually require admission to a neonatal intensive care unit for treatment when born premature.  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.  Current incentives, however, have not been sufficient to stimulate innovation for the neonatal population.

“When it comes to the development of new treatments that have the potential and promise to save lives, our most vulnerable children are too often left behind.  The fact is a variety of factors and challenges have hindered the development of much-needed therapies and stood in the way of progress treating newborns,” Congressman Luján said.  “This legislation will provide incentives to stimulate innovation and encourage research and development in this area of great need.  In recent years, we have seen tremendous medical advancements, and with this bill, we can see that our youngest children can benefit from the next breakthrough.”

“Families with children who are born prematurely or sick simply do not have enough options to save their newborns,” said Congressman Long. “Only one drug has been approved to help treat these infant patients in the past 16 years, while about 200,000 children are still being born each year that need intensive neonatal care in the US.  The well-being of these children should be our top priority. 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 this threat and reduce preventable infant mortality.”

“I see first-hand how innovation in neonatal health is often thwarted due to the difficulty of clinical trials and the economic challenges of developing treatments for this patient population,” said Dr. Kristi Watterberg, Professor of Pediatrics/Neonatology at the University of New Mexico School of Medicine.  “The lack of adequate treatment options for vulnerable premature babies leaves doctors without the right tools to confront the serious problems they face.  This bill provides incentives to industry to take on the vital research and clinical trials that would otherwise be too complex and costly to perform.  As a doctor who has dedicated my life to helping these tiny and fragile patients, I believe strongly that we must do more to encourage innovation in neonatal medicine, and this legislation takes an important step forward.”

Federal legislation implementing a combination of pediatric incentives and obligations led to an increase in pediatric studies, but did not properly addressed the challenges that have limited treatments for newborns.  For example, very few patients are available for study at any given time so it is difficult and costly to get sufficient numbers.  In addition, many conditions for which drug therapy is needed in the neonatal population do not occur in older children and adults, and even if a drug has been shown to be safe and effective in older populations, the data cannot be extrapolated to newborns because of their distinctive developmental status and immature physiology.

The Promoting Life-Saving New Therapies for Neonates Act aims to close the treatment gap by stimulating the development of safe and effective drugs for a challenging and neglected pediatric population.  The bill will ensure that new neonatal drugs address the most critical needs by collaborating with multiple stakeholders, such as the National Institutes of Health, the Critical Path Institute, and patient advocacy groups to identify priority condition.  In addition, it creates a new incentive model by providing a transferrable “exclusivity voucher” to drug sponsors who successfully develop products for newborns.  This voucher would enable the product sponsor to extend the exclusivity period on another drug by one year.

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