A bill that could impact eventual treatments for rare diseases and conditions found in children, including spinal muscular atrophy, has passed in the U.S. House of Representatives.
H.R. 6163 calls for amending title IV of the Public Health Service Act to provide for a National Pediatric Research Network focusing on pediatric rare diseases and conditions.
The Network would be empowered to award grants and other forms of funding to public or private non-profit organizations that conduct “basic, clinical, behavioral or translational research to meet unmet needs for pediatric research and training researchers in pediatric research techniques,” the bill says.
Entities receiving such funding would “focus primarily on pediatric rare diseases or conditions, including any such diseases or conditions that are genetic disorders, such as spinal muscular atrophy and Duchenne muscular dystrophy, or are related to birth defects, such as Down syndrome and Fragile X,” the bill adds. Researchers would be required to conduct or coordinate clinical trials regarding disease prevention, diagnosis or treatment and “rapidly and efficiently disseminate scientific findings resulting from such trials.”
FightSMA, an organization devoted to finding a treatment and cure for the illness, called the bill’s Sept. 19 passage “an unprecedented step forward for the advancement of clinical research on rare pediatric disorders.”
In a statement about the bill’s passage, the organization added, “Legislation directly impacting the fight against spinal muscular atrophy has never before experienced this level of success in Congress, and many thanks must be given to the bill’s lead sponsors, Reps. Cathy McMorris Rodgers (R-Wash.), Lois Capps (D-Calif.) and Diana DeGette (D-Colo.). Their leadership, along with the strong support of Majority Leader Eric Cantor (R-Va.), led to the bill passing through the Energy and Commerce Committee and the full House with overwhelming bipartisan support.”
The focus now turns to a companion bill in the Senate, S. 3461, introduced in July by Sen. Sherrod Brown (D-Ohio). The bill, which had seven co-sponsors at press time, is currently in committee. The latest senators to sign on — Roy Blunt (R-Mo.) and Chuck Grassley (R-Iowa) -— added their support the day H.R. 6163 passed in the House.
