FightSMA (http://www.fightsma.org) is tremendously proud to announce that after years of passionate advocacy by families across the country the United States Senate passed the National Pediatric Research Network Act (NPRNA) Nov. 14 by unanimous consent. The legislation, which the U.S. House approved on Tuesday, Nov.12, now heads to the President’s desk for his signature. The NPRNA was approved as part of a health package known as the PREEMIE reauthorization act (S.252).
The legislation authorizes the National Institutes of Health (NIH) to establish a network of research consortia that will conduct basic through clinical research on a variety of pediatric disorders. The NPRNA requires that an appropriate number of consortia focus on research conducted on pediatric rare diseases such as spinal muscular atrophy (SMA), including clinical trials evaluating potential therapies and treatments. It will help create the collaborative infrastructure necessary to conduct leading-edge pediatric research today and well into the future, including support for promising young investigators beginning their careers in pediatrics.
“We are currently in an exciting period of SMA therapeutics with the advent of a number of promising interventions,” said Dr. Alex MacKenzie, Co-Chair of FightSMA’s Scientific Advisory Committee. “But from a clinical trial point of view it really is just the beginning. The next decade will witness the introduction and trialing of these as well as a number of additional SMA therapeutic modalities; needed for all of these, in addition to a comparison of relative effectiveness, is a sense of which types of SMA will benefit the most from which intervention as well as a clear picture of when, for the optimal outcome, treatment needs to start and whether therapy needs to be continued for the life of the individual affected by SMA. The NPRNA will be critical to achieving these goals. It is important to underline the fact that the interventions as well as the trial methodologies configured for SMA will serve to inform approaches for many of the hundreds of rare and ultra rare disorders still awaiting effective treatment.”
FightSMA is extraordinarily grateful to our lead Senate sponsors, Senators Sherrod Brown (D-OH) and Roger Wicker (R-MS), for introducing and championing the bill through the U.S. Senate. Without their steadfast dedication to our cause we would not be celebrating this great victory. We extend our gratitude to Senate Health, Education, Labor and Pensions Committee chairman Tom Harkin (D-IA) and ranking member Lamar Alexander (R-TN) for shepherding the bill to final passage.
“This is excellent news for the health and well-being of Ohio’s—and America’s—children,” said Sen. Brown. “Pediatric research institutions and pediatric researchers face real financial challenges that threaten their important work, especially in their efforts to combat and cure rare pediatric diseases. But this new law will leverage investments into pediatric research through enhanced partnerships and data-sharing among research institutions, to the benefit of our children and families.”
“Passage of the National Pediatric Research Network Act is a monumental step towards fighting childhood diseases, such as spinal muscular atrophy,” said Sen. Wicker. “NPRNA will provide researchers greater opportunity to share and coordinate pediatric research in ways that could help save children’s lives and more efficiently use federal dollars. This legislation will fundamentally change how findings on pediatric diseases are shared and will equip researchers with the resources to develop better treatment and possible cures for thousands of American children.”
FightSMA would also like to again express our profound thanks to our House sponsors and champions who pushed the NPRNA bill through that chamber four times in various forms over the past year as part of their fierce determination to enact this bill: Lead sponsors Lois Capps (D-CA) and Cathy McMorris Rodgers (R-WA); House Energy and Commerce chairman Fred Upton (R-MI) and ranking member Henry Waxman (D-CA); and House Majority Leader Eric Cantor (R-VA).
“This bill will increase coordination and improve pediatric research – especially research of rare and complex diseases – while developing new treatments to fight them,” said Congresswoman Capps. “Children have unique health care experiences, treatment needs, and research challenges; and while public and private research has come a long way on pediatric diseases over the years, we know that we are still far behind on important diagnostics, cures, and treatments for far too many ailing children. We see the importance in legislation like this right here at home, where our neighbors Bill and Victoria Strong work every day to make their daughter Gwendolyn’s life a little better as she grows up with SMA. We owe it to the Strongs and to every parent to do what we can to find treatments and cures for illnesses in children.”
Rep. McMorris Rodgers, who co-sponsored the NPRNA with Rep. Capps, said, “The National Pediatric Research Network Act will authorize the establishment of a well proven, and evidence-based approach for addressing pediatric research. It will enable the National Institutes of Health (NIH) to support multi-institute research to coordinate and streamline this important research. Most importantly, it will help to speed cures to the youngest patients.”
We look forward to enactment of the NPRNA in the coming days, and to the National Institutes of Health’s implementation of a national pediatric research network that will bring hope to children living with devastating disorders by expanding clinical trials to help find treatments for SMA and other pediatric diseases.