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Reinvigorating the Fight Against Rare Diseases: Op-Ed by CLSA & EveryLife Foundation in The Hill
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March 8, 2016

On Feb. 29, the California Life Sciences Association (CLSA) and the EveryLife Foundation for Rare Diseases authored an op-ed in The Hill. “Reinvigorating the Fight Against Rare Diseases” was published on World Rare Disease Day, and highlights the importance of continued investment into research and development of new therapies and cures for rare diseases, which affect approximately 1 in 10 Americans and their families.

Since the Orphan Drug Act was passed in 1983, innovators in California and across the nation have developed therapies for cystic fibrosis, Pompe disease and other debilitating diseases – but the work is not yet done.

During the 114th Congress, CLSA supported advancement of several pieces of legislation that would have a demonstrable impact on treating rare diseases and disorders, including the 21st Century Cures Act, the Advancing Targeted Therapies for Rare Diseases Act, the Creating Hope Act, and the Orphan Product Extensions Now (OPEN) Act. CLSA continues to work with our membership, the California delegation and other  stakeholders to advocate in support of these pieces of legislation and continue to inform policy makers of the need for new incentives for research and investment into therapies and cures for rare diseases.

CLSA was also pleased to support a congressional briefing on March 3, sponsored by the bipartisan, bicameral Congressional Rare Disease Caucus, “The Rare Disease Ecosystem: Fostering Patient Engagement & Driving Biomedical Innovation.” Several CLSA members participated in the panel discussion, including expert panelists from Raptor Pharmaceuticals, Stanford University, and Ultragenyx, Inc. All four of the Rare Disease Congressional Caucus co-chairs attended to offer remarks:  Representatives Leonard Lance (R-N.J.), and Joe Crowley (D-N.Y.), and Senators Orrin Hatch (R-Utah) and Amy Klobuchar (D-Minn.).

Questions? Please contact Jenny Carey, CLSA’s Vice President, Federal Government Relations & Alliance Development (jcarey@califesciences.org or 202-743-7559) or Meghan Drenan, CLSA’s Associate Director of Federal Government Relations (mdrenan@califesciences.org or 202-743-7560).