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Advocating for Policy

FightSMA engages government leaders and policy makers globally to build support for pediatric research. For ten years, FightSMA has worked closely with members of Congress to develop legislation supporting pediatric SMA research. As a direct result of FightSMA’s consistent effort, the National Pediatric Research Network Act (H.R. 225) was signed into law on November 27, 2013.

• DOWNLOAD THE BILL (H.R. 225, FEB 2013 VERSION)

What is the National Pediatric Research Network Act (NPRNA)?

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.

Why is the NPRNA Important?

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.

For ten years, FightSMA has worked closely with members of Congress to develop legislation supporting pediatric SMA research.

Why is this such an Achievement?

Only 4% of bills sent to the Senate from the House actually pass—and ours was one of them! That’s a result of FightSMA’s persistent efforts to raise awareness of SMA on the hill and our long-term, bipartisan relationships with policymakers and government leaders.

Who is Championing NPRNA on the Hill?

Congressman Fred Upton (R-MI) and Senator Sherrod Brown (DOH) are widely regarded as the bill’s bipartisan champions (honored by NORD for their tireless efforts). FightSMA is extremely grateful for NORD’s active endorsement of the NPRNA, which gave the legislation a critical boost. Enacted in November 2013 after years of consideration on Capitol Hill, the NPRNA is a tremendous opportunity for the advancement of SMA and rare disease research. The bill simply wouldn’t have become law without the commitment by Upton and Brown to get the legislation to the President’s desk. FightSMA is so thankful for the opportunity to work with all of the NPRNA’s champions, including Sen. Roger Wicker (R-MS) and Reps. Lois Capps (D-CA) and Cathy McMorris Rodgers (R-WA).

H.R. 225, FEB 2013 VERSION

  • Winter-Spring 2007:  FightSMA recruits original House and Senate sponsors for first-ever federal legislation supporting SMA clinical trials
  • August 2, 2007: SMA Treatment Acceleration Act introduced in the House by Reps. Patrick Kennedy (D-RI) and Eric Cantor (R-VA)
  • September 12, 2007: SMA Treatment Acceleration Act introduced in the Senate by Sens. Debbie Stabenow (D-MI) and Johnny Isakson (R-GA)

  • April 28, 2009: SMA Treatment Acceleration Act of 2009 introduced in the House by Reps. Patrick Kennedy (D-RI) and Eric Cantor (R-VA)
  • May 21, 2009: SMA Treatment Acceleration Act of 2009 introduced in the Senate by Sen. Debbie Stabenow (D-MI) and Johnny Isakson (R-GA)

  • 2011-2012:  FightSMA works with Reps. Cathy McMorris Rodgers (R-WA), Lois Capps (D-CA), and Eric Cantor (R-VA) to develop the National Pediatric Research Network Act
  • July 19, 2012: National Pediatric Research Network Act of 2012 introduced in the House by Reps. Cathy McMorris Rodgers (R-WA) and Lois Capps (D-CA)
  • July 31, 2012: National Pediatric Research Network Act of 2012 introduced in the Senate by Sens. Sherrod Brown (D-OH) and Roger Wicker (R-MS)
  • September 11, 2012: National Pediatric Research Network Act of 2012 marked up by House Energy and Commerce committee
  • September 19, 2012: National Pediatric Research Network Act of 2012 passes House by voice vote
  • December 19, 2012: House passes PREEMIE Reauthorization Act by voice vote; bill contains National Pediatric Research Network Act language

  • January 14, 2013: National Pediatric Research Network Act of 2013 introduced in the House by Reps. Lois Capps (D-CA) and Cathy McMorris Rodgers (R-WA)
  • January 22, 2013: National Pediatric Research Network Act of 2013 marked up by the House Energy and Commerce committee
  • February 4, 2013: National Pediatric Research Network Act of 2013 passes House by vote of 275-27
  • February 28, 2013: National Pediatric Research Network Act of 2013 introduced in the Senate by Sens. Sherrod Brown (D-OH) and Roger Wicker (R-MS)
  • September 25, 2013: House passes PREEMIE Reauthorization Act, which includes National Pediatric Research Network Act
  • November 14, 2013: Senate passes PREEMIE Reauthorization Act, which includes National Pediatric Research Network Act
  • November 27, 2013: PREEMIE Reauthorization Act signed by President Obama; National Pediatric Research Network Act becomes law
  • February 27, 2014, Sen. Sherrod Brown (D-OH) introduces S.Res.368, a resolution designating February 28, 2014, as "Rare Disease Day".  Bill mentions SMA among a select group of diseases, along with a reference to the NPRNA.  The bill passed the Senate by unanimous consent the same day.