Amy Judge DeLong is manager of Federal Government Relations at Boston Children’s Hospital.
In the midst of a seismic shift in Presidential administrations and anticipation of the incoming Congress, a landmark medical research bill with several provisions important to children cleared the lame duck session of Congress. The 21st Century Cures Act (Cures) is the end result of nearly three years of bipartisan Congressional activity. Last week, it was signed into law.
Cures includes scores of provisions aimed at strengthening National Institutes of Health funding for medical research and accelerating review efforts at the Food and Drug Administration. The law cleared Congress with overwhelming majorities, an example of bipartisanship that may be challenged in the months ahead. …
On the minds of everyone involved in the care of sick children is the pressing need for more pediatric research funding. Last November, Congress finally passed the National Pediatric Research Act. It authorizes the National Institutes of Health to support a nationwide network of up to 20 pediatric research consortia, but it falls short of actually increasing NIH spending. Indeed, the next step in implementing the Act is to secure a specific funding commitment from the NIH or Congress.
The prospects for such alliances are good. Patient advocacy foundations are increasingly active in research, and academic-industry partnerships are on the rise. At Boston Children’s Hospital alone, sponsored research and collaborations with foundations and industry have tripled from nine in 2009 to 29 in 2013. Alan Crane, MBA, a partner at Polaris Partners and an advisor to Boston Children’s Technology and Innovation Development Office, points out that drug discovery is much harder and more complex today than it used to be—just as many products are coming off patent.
Fifty years ago, a baby born to President John F. Kennedy and his wife, Jacqueline, died after just 39 hours of life. Born just slightly premature at 37 weeks and weighing only 4lbs, 10½oz, Patrick Bouvier Kennedy developed respiratory distress syndrome, then a frequent cause of death and long-term lung disability in infants born prematurely. At the time, this condition killed about 25,000 children each year.
Today, thanks to National Institutes of Health (NIH)-funded research, we know that babies born prematurely or at very low weights may lack an important protein called surfactant in their lungs. This discovery led to the development here at Boston Children’s Hospital by Mary Ellen Avery, MD, of surfactant replacement therapy, which has revolutionized the care of premature infants. As a direct result of research into this pediatric disease, premature infants born as early as 23 or 24 weeks are viable. The incidence and severity of respiratory distress is extremely reduced: Fewer than 1,000 babies will die from it in the United States this year.
WE WANT YOU! Tell your senators to sponsor the National Pediatric Research Network Act. Here’s how.