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For every new parent, the ritual of preparing for the arrival of a baby presents an opening to an entirely new world of special, baby-approved products and supplies. There are countless toys, clothes, training aids, bathing supplies and furniture designed exclusively for babies and infants based on their size and specific needs.
But what about the medicines that are used on babies in the hospital? Shouldn’t they be designed specifically for the unique needs of newborns too?
Too often, the answer to that question is no. Over the past two decades, drug development for neonates – a scientific term for premature or full-term infants up to 28 days old – has lagged seriously behind the significant advances medical innovators have made in other fields. Since 1999, there has not been a single new therapy approved by the Food and Drug Administration (FDA) for the newborn population.
In fact, over 90% of medicines currently given to newborns are “off-label,” meaning that the medication is being used in a manner that has not been explicitly tested or endorsed by the FDA. Given that newborns can often be exposed to over 60 drugs in a single Neonatal Intensive Care Unit (NICU) stay, this glaring treatment gap presents serious concerns of unanticipated adverse effects from drug interaction and improper dosing.
The problem has been magnified by the prevalence of pre-term births, with hundreds of thousands of infants being admitted to NICUs ever year due to the challenges of prematurity. Nearly one in ten infants in the United States are born before reaching full-term, while prematurity remains the leading cause of newborn mortality.
For those who survive, one in five premature infants will face health challenges that can persist for a lifetime – including debilitating conditions such as cerebral palsy, lung disease, and deafness. And unfortunately, the dedicated health professionals in NICUs across the country lack treatments that are both effective in treating these problems and carefully tailored for newborns’ fragile physiology.
There are compelling explanations for why researchers have been unable to produce any revolutionary therapies for newborns, despite the clear need for improvement. Testing is incredibly difficult and parents are understandably wary of providing consent for experimental treatments. The market for neonatal drugs – particularly those that treat rare diseases – is small and unprofitable, leaving little incentive for the type of private investment necessary to produce a breakthrough. And simply administering medications and studying outcomes for newborn patients is exceedingly challenging; pharmacological combinations can be difficult to evaluate and the exact efficacy of each treatment will change as an infant grows.
Statistics bear out the impact this severe shortage in treatment options has on the vulnerable newborn population, particularly in this country. With the United States’ infant mortality rate among the worst in the industrialized world, new treatments for newborns has the potential to save young lives across the country.
This evidence all points to an urgently needed restructuring of the system we use to promote the development of novel therapies for our newborns. That’s why we are proud to be the House sponsors of the Promoting Life-Saving New Therapies for Neonates Act. In partnership with our Senate colleagues, Sens. Bill Cassidy (R-LA) and Bob Casey (D-PA), we aim to create a new approach in encouraging our medical community to discover breakthrough treatments for healthcare’s most precious patients.
Our bill, H.R. 5182, would first engage stakeholders at the National Institutes of Health and elsewhere to identify the most pressing needs in newborn health. Working with the FDA, these identified problem areas would be outlined in a priority list, helping manufacturers target specific challenges and develop tools that will help neonatal physicians. Finally, the legislation provides for a transferrable “exclusivity voucher” as a fresh incentive for manufacturers who successfully address an FDA-identified priority in neonatal health, helping spur the significant private investment required to revolutionize care for newborns.
It is only through this creative, bipartisan approach that we can unlock the successes of modern medicine for the newborn population. Previous legislative efforts, such as the Food and Drug Administration Modernization Act (FDAMA) in 1997, have led to additional pediatric studies, but none have produced a coinciding growth in FDA-approved medicines for newborns. Our legislation differs by capitalizing on the expertise of both advocacy groups and industry, while also activating additional resources through the provision of an exclusivity voucher.
From baby food to baby shoes, we’ve made sure our infants have products that meet their special needs. Isn’t it time we did the same for their medicines?
Reps. Billy Long (R-MO) and Ben Ray Luján (D-NM) represent Missouri’s 7th congressional district and New Mexico’s 3rd congressional district, respectively. Both serve on the House Energy and Commerce Committee’s Health Subcommittee, where their bill, the Promoting Life-Saving New Therapies for Neonates Act of 2016 (H.R. 5182), is currently being considered. You can follow both on Twitter, @USRepLong and @repbenraylujan.