A rare childhood disease non-profit needed federal support in its search for a cure.
An international non-profit organization dedicated to accelerating a treatment for spinal muscular atrophy (SMA) engaged PSW to boost federal funding for research on this devastating disease, the leading inherited killer of children under two. PSW attacked this challenge both through original authorizing legislation and in the appropriations process.
First, leading a coalition of patient organizations and families across the country, PSW crafted legislation authorizing the National Institutes of Health to expand support for SMA clinical trials, and secured introduction of bipartisan bills in the House and Senate with dozens of cosponsors. PSW then partnered with a coalition of children’s hospitals and several of our House and Senate allies to draft the National Pediatric Research Network Act (NPRNA), a broad bill to establish collaborative research networks including clinical trials for rare pediatric diseases. PSW quarterbacked a grassroots/grasstops campaign with families and hospitals to build support for the bill, and after multiple fly-ins and extensive lobbying both in D.C. and in Congressional districts, the NPRNA was cleared by the House and Senate and enacted into law in November 2013.
On the appropriations front, PSW targeted the Defense Health accounts in the Senate Defense Appropriations bill. We identified key members of the Defense Appropriations subcommittee with whom we enjoy strong relationships and arranged constituent meetings with these offices. We also worked with a Senate office to arrange an informational call between a small group of SMA researchers and Department of Defense (DOD) medical research personnel. PSW ultimately secured the inclusion of spinal muscular atrophy in the list of eligible diseases for the Peer-Reviewed Medical Research Program (PRMRP) in the Senate’s Defense Appropriations bill. We have continued to work to ensure that SMA remains on the list for the past three fiscal years. As a result, SMA researchers in multiple states have competed for and won millions of dollars in DOD support for SMA research.
An international non-profit sought to increase their federal funding for research on a devastating disease.
Targeting Defense Health accounts, PSW maneuvered between congressional offices and boosted research dollars.