According to a panel of experts who presented at a briefing this week in the Senate Dirksen Office Building on Capitol Hill, new legislation is needed to improve the quality of treatment options available to premature newborns. While scores of new treatment options have become available for the pediatric patient population as a whole—thanks in large part to incentives implemented over the past two decades – panelists noted that only one drug has been approved specifically for use in neonates – infants in the first 28 days of life, who often face rare conditions that don’t exist in the adult population or even older infants.
Dr. Jonathan Davis The vulnerability of this population and the significant barriers to performing clinical trials on newborns has led to an unprecedented “innovation desert” in this space. “We can do better,” said Dr. Jonathan Davis, Chief of Newborn Medicine at Tufts University School of Medicine. “We have the worst rates of prematurity of any developed country… but people don’t want to do (clinical) studies on babies.” Further, with a dedicated effort to improving treatments for preemies, Dr. Davis estimated that a significant dent could be made in the $29 billion spent annually on treating prematurity and related conditions.
The deficit in treatment options hits particularly close to home for those who have first-hand experience with preemies. Panelist Jennifer Degl spoke eloquently about her daughter Joy, who was born 17-weeks early and developed a rare lung condition known as bronchopulmonary dysplasia. Ms. Degl, whose book From Hope to Joy tells the story of her daughter’s remarkable journey, provided an emotional account of her experience in a neonatal intensive care unit (NICU). She recalled the breathing tubes, the incubator, even her daughters translucent skin and fused eyelids, as she explained how she couldn’t even touch her baby for the first month of her life.
The deficit in treatment options hits particularly close to home for those who have first-hand experience with preemies. Panelist Jennifer Degl spoke eloquently about her daughter Joy, who was born 17-weeks early and developed a rare lung condition known as bronchopulmonary dysplasia. Ms. Degl, whose book From Hope to Joy tells the story of her daughter’s remarkable journey, provided an emotional account of her experience in a neonatal intensive care unit (NICU). She recalled the breathing tubes, the incubator, even her daughters translucent skin and fused eyelids, as she explained how she couldn’t even touch her baby for the first month of her life.
Ms. Jennifer Degl While many babies, including Joy, are fortunate enough to survive their premature births, they are also likely to face ongoing medical challenges related to their treatment as an infant. Joy “has been on more medications than I can recall,” Ms. Degl noted as she described the challenges her now-three-year-old daughter continues to face due to her premature birth. Chronic conditions, including lung disease and extreme asthma, are frequently associated with children who were born prematurely, but few medical therapies currently exist to curb their prevalence. “I was surprised when I heard that a new drug hasn’t been developed for neonates in almost 20 years,” Ms. Degl said. “As a science teacher, it’s remarkable that Apple can release a new iPhone every year, yet every medication being used to treat my daughter was developed when I was still in high school.”
Ms. Allyson Kayton But it’s not just parents of preemies who are hoping for new methods to improve care in NICUs – healthcare providers in these settings are often just as invested in the survival of these infants as the parents themselves. Recalling her first day working in a NICU, nurse practitioner Allyson Kayton, who was representing the National Association of Neonatal Nurses and Nurse Practitioners, said that after hearing a newborn baby cry and fight for his life, “I became a patient advocate every day.” Yet despite wanting to provide the best care possible for her patient, Ms. Kayton said that “never in [her] career” has there been a new drug developed to improve the care provided for her patients. And she noted that focusing on the most prevalent conditions in preemies—such as lung conditions associated with breathing on ventilators for extended periods of time—will allow innovators to “be proactive in recognizing what’s needed for these patients.”
Dr. Christina Bucci-Rechtweg While the differences between treating adults and infants are apparent on the surface, they may be even more stark than many policymakers realize at first glance. Dr. Christina Bucci-Rechtweg, Head of Pediatric & Maternal Health Policy at Novartis, explained that many treatments developed for adults are designed with the recognition that organs develop and then fail over time. For infants, however, most organs haven’t even had time to mature. And while treatments utilized in adults are often formulated to help the patients “stay on the shelf” and manage their chronic illness, those same therapies “can be toxic in the neonatal population.”
Senator Bill Cassidy (R-LA) Despite these challenges, though, the panel sounded a hopeful note, pointing to federal legislation that could introduce new incentives to spur the development of groundbreaking drugs for this population. Indeed, Sen. Bill Cassidy (R-LA), an original cosponsor of the Promoting Life Saving New Therapies for Neonates Act (S. 2041) and a doctor himself, outlined his legislative proposal which would do just that. “It’s difficult to do research in NICUs,” the senator said. “That’s why we need to change the paradigm to give pharmaceutical companies the incentive to invest in a drug that will only treat a small number of patients.” Sen. Cassidy introduced the bipartisan bill late in 2015 with Sen. Bob Casey (D-PA), who is among Congress’ staunchest champions for pediatric health.
As Sen. Cassidy explained at the briefing, his legislation would take a series of steps to identify priority conditions for researchers to target, while providing a unique incentive to allow drug developers to achieve profitability if they manage to bring a new product to market. Among the legislation’s provisions, Sen. Cassidy noted that the bill would:
Here’s how you can help promote new cures for our newborns. To sign a letter to your senators on cosponsoring the vital legislation, S.2041 – The Promoting Life Saving New Therapies for Neonates Act, click here. For more information on the gap in innovation for neonates and the Newborn Health Initiative, please visit www.newbornhealth.org.
As Sen. Cassidy explained at the briefing, his legislation would take a series of steps to identify priority conditions for researchers to target, while providing a unique incentive to allow drug developers to achieve profitability if they manage to bring a new product to market. Among the legislation’s provisions, Sen. Cassidy noted that the bill would:
- 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.
Here’s how you can help promote new cures for our newborns. To sign a letter to your senators on cosponsoring the vital legislation, S.2041 – The Promoting Life Saving New Therapies for Neonates Act, click here. For more information on the gap in innovation for neonates and the Newborn Health Initiative, please visit www.newbornhealth.org.
