How Congress could help fulfill the promise of pediatric research

By David A. Williams, MD, and Amy DeLong

United_States_Capitol_-_west_frontFifty 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.

Despite such evidence that addressing chronic conditions and diseases of childhood can positively impact both health outcomes and future medical spending, pediatric research has been underfunded at the NIH for years. Among the many reasons for this is the reality that most pediatric diseases are rare, which limits available resources. In recent years, the disproportionate spending between pediatric and adult research has become more pronounced, with only about 5 percent of a $30 billion budget going to pediatrics.

Boston Children’s and other pediatric research institutions are working together in support of the National Pediatric Research Network Act to address this problem. This legislation, introduced by U.S. Representatives Lois Capps (Democrat – CA) and Cathy McMorris Rogers (Republican – WA) and U.S. Senators Sherrod Brown (Democrat – OH) and Roger Wicker (Republican – MS), would address the shortfall in pediatrics by using a proven model to foster greater collaboration, coordination and sharing of resources.

In recent years, the disproportionate spending between pediatric and adult research has become more pronounced, with only about 5 percent of a $30 billion budget going to pediatrics.

The bill would have the NIH establish “pediatric research consortia” throughout the nation. Each consortium would focus on a particular pediatric disease and involve researchers from multiple institutions working together to combat that disease.

One important way these researchers would work together across institutions is sharing of core research infrastructure—the lifeblood of basic clinical investigations. For example, Boston Children’s is part of a small group of institutions developing an experimental approach to cell engineering for the cure of rare genetic diseases, called gene therapy. Boston Children’s researchers developed and provided to other participating institutions the complex methods required for manipulation and testing of engineered cells in a specialized cell manufacturing laboratory, while another institution in the group developed, safety tested and distributed the “transfer vehicle” or “vector” used to insert therapeutic genes into patients’ stem cells. By pooling our resources and expertise, we have been able to advance gene therapy at a much faster pace than if we all worked independently.

Historical graph of NIH budget and NIH pediatric spending
Source: Gitterman DP, Hay WW Jr., Pediatric Research, 2006 (

Through the National Pediatric Research Network legislation, the NIH would be able to both increase and maximize the value of funds committed to pediatric research. Indeed, this kind of collaboration works. For more than 40 years, the National Cancer Institute has made it possible for pediatric research institutions to work together in a similar consortium model to develop the most effective treatments for childhood leukemia. Prior to this collaboration, childhood leukemia was uniformly fatal. Today, the survival rate for the most common form of childhood leukemia is 85 percent.

A robust pediatric research infrastructure is necessary to improve the health and well-being of our nation’s children. The National Pediatric Research Network Act has already passed the House of Representatives with overwhelming support. The next step is to move the bill through the United States Senate, and for that, we need your support.

The Children’s Advocacy Network, an arm of the Office of Government Relations at Boston Children’s, is actively working with your senators and representatives to turn this bill into law. Please help us gain the legislative support we need by clicking here to email your senators and ask that they add their names to the National Pediatric Research Network Act (S. 424) as a cosponsor.

David A. Williams, MD, is Division Chair of Hematology/Oncology and Associate Chair of Pediatric Oncology at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and the Leland Fikes Professor of Pediatrics at Harvard Medical School. Amy DeLong is the Manager of Federal Government Relations in Boston Children’s Office of Government Relations