United States Senate Unanimously Passes the National Pediatric Research Network Act (NPRNA) Supporting SMA Research

United States Senate Unanimously Passes the National Pediatric Research Network Act (NPRNA) Supporting SMA Research

Headed to the President’s desk today for signature, the legislation establishes a network of research consortia to focus on pediatric rare diseases, specifically including SMA.

Friday, November 15, 2013

Unanimously passed by the U.S. Senate on Thursday night, the National Pediatric Research Network Act (NPRNA) was approved within the PREEMIE Reauthorization Act (S.252).  The NPRNA authorizes the establishment of an infrastructure of research consortia focused on pediatric rare diseases. The act mandates that funding be allotted to public or private nonprofit entities to address unmet needs through basic, clinical, behavioral, and translational research and training of investigators in pediatric research techniques.  Awarded consortia will be required to conduct multi-site clinical trials of therapies for the prevention, diagnosis, and/or treatment of pediatric rare conditions. A data coordinating center will be created to assist in collaborative research projects, management of data, and distribution of findings.

The NPRNA additionally emphasizes collaboration with the Centers for Disease Control (CDC) for establishment of appropriate patient registries, as well as communication with the Commissioner of Food and Drugs (FDA) on the state of the consortia research and status of clinical trials.

This act represents an exciting and pivotal step in the fight against a number of devastating pediatric diseases. The legislation, which specifically includes mention of SMA, can be read in full here: https://www.govtrack.us/congress/bills/113/hr225/text