U.S. Representative Leonard Lance (R-NJ) applauded advocates of rare disease research during a Rare Disease Caucus briefing on Capitol Hill on Wednesday, February 28. Rep. Lance thanked the National Institutes of Health (NIH) “for all that it does in rare diseases” and noted that “in the bipartisan budget agreement, the NIH saw a funding boost of $2 billion.”
The Everylife Foundation for Rare Diseases sponsored the caucus, marking the occasion of Rare Disease Day. Speakers included Gina Szanuk, cofounder and director, Rare and Undiagnosed Network, and Robert Selliah, founder and CEO, American Med Chem, among others.
Selliah called for more funding for the NIH to support continued collaboration with industry partners. Szanuk agreed, noting there is much work to be done to address rare diseases, which currently affects 30 million Americans. The 21st Century Cures Act and Orphan Drug Act, she said, have accelerated the development of new therapies. She urged policymakers to approve other measures including the OPEN Act, which would provide incentives to increase market potential for companies that repurpose previously approved drugs for the treatment of rare diseases, and the Advancing Access to Precision Medicine Act to allow states to apply for exceptions to Medicaid payment rules to cover genome sequencing clinical services for children.
The EveryLife Foundation will be honored at Research!America’s Advocacy Awards Dinner on March 14 in Washington, D.C.