The National Pediatric Research Network Act (NPRNA) of 2013 is expected to be introduced in the U.S. Senate in the coming weeks, but many still don’t know why it’s such a big deal for the Spinal Muscular Atrophy Community.

Simply put, the NPRNA promises to directly benefit the fight against SMA by supporting collaborative pediatric research including multi-site clinical trials for rare pediatric disorders like SMA.  Here’s more from the Congressional Budget Office, writing about the house version (H.R. 225) that passed on February 4, 2013:

H.R. 225 would authorize the Director of the National Institutes of Health (NIH) to establish a National Pediatric Research Network that could provide support for research and training at up to 20 pediatric research consortia for up to five years. The bill would require the Director of NIH to establish a data coordinating center for the consortia. Upon request by the Centers for Disease Control and Prevention (CDC), consortia participating in the program would be required to provide assistance to the CDC to establish or expand surveillance systems, such as patient registries.

NIH currently supports many research networks that support research and training focused on pediatric health care needs and operates data coordinating centers for those networks. Those networks perform essentially the same activities as the consortia described in the bill. Existing networks do not routinely provide assistance to the CDC to establish surveillance systems. Based on information provided by NIH, CBO estimates that implementing H.R. 225 would have no effect on the number of research consortia or data coordinating centers that NIH would support. CBO expects that CDC would request assistance from a few networks to establish surveillance systems. Based on past coordination involving patient registries, CBO expects that the cost of providing such support would total about $1 million over five years. Thus, CBO estimates that implementing H.R. 225 would cost $1 million over the 2014-2018 period, assuming the availability of appropriated funds.

Enacting the bill would not affect direct spending or revenues; therefore, pay-as-you-go procedures do not apply.

H.R. 225 contains no intergovernmental or private-sector mandates as defined in the Unfunded Mandates Reform Act and would not affect the budgets of state, local, or tribal governments.