We are excited to announce that\u00a0U.S. lawmakers have finally reached an agreement on the 21st Century Cures Act, a game-changer for patients with rare diseases. This agreement comes more than one year after the House passed its original version of the bill. The first vote is expected to take place tomorrow.<\/p>\n
Late last week, Chairmen Fred Upton (R-MI) and Lamar Alexander (R-TN) of the House Energy and Commerce Committee and the Senate Health, Education, Labor and Pensions Committee (HELP) Committee, respectively, announced a bicameral agreement<\/a>\u00a0on an updated 21st Century Cures Act. The House is scheduled to vote on the package tomorrow, and the Senate is expected to vote soon after.<\/p>\n Overall we are quite pleased with the package, and fully support its passage. There are many provisions that will improve the discovery, development, and delivery of orphan therapies for rare disease patients, including:<\/p>\n While not everything we supported has been included (such as the OPEN Act\u00a0<\/strong>additional resources for developing natural history registries<\/strong> for rare diseases, and more robust funding for the NIH and FDA<\/strong>), the package still represents a substantial step forward for the development of therapies for rare diseases, the majority of which still have no treatment.<\/p>\n NORD looks forward to continuing to advocate for a strong Rare Pediatric Disease PRV program, appropriate FDA funding to ensure a strong FDA orphan therapy review process, and the replenishment of funds aimed at prevention to address important unmet needs.<\/p>\n Most of all we want to thank our fellow rare disease patient advocates for joining us in this fight. Thank you to the 185 organizations<\/a>\u00a0that supported a long-term reauthorization of the Rare Pediatric Disease PRV program, and the more than 1,000 advocates who called their Congressmen to support the 21st Century Cures Act on the unified rare disease day of action earlier this month.<\/p>\n\n