Good News For Cystic Fibrosis As US Senate Passes Bill

The Cystic Fibrosis Foundation applauds passage of a bill in the United States Senate to expand cooperation between the Food and Drug Administration (FDA) and outside rare disease experts and patient advocates during the FDA drug approval process.   The bill, passed yesterday, is known as the Expanding and Promoting Expertise in Review of Rare Treatments Act (EXPERRT). Its passage in the Senate is the last hurdle before the bill goes to President Obama for his signature. He is expected to sign it into law. Introduced by Sen. Sheldon Whitehouse (D-RI),  EXPERRT is part of the FDA Safety and Innovation Act, which reauthorizes the FDA's user-fee program that funds its drug and device evaluation.  "The Cystic Fibrosis Foundation commends the Senate for its action on this issue, which is critical to the 30,000 Americans with cystic fibrosis and millions of others who struggle with complex conditions for which there are little or no available treatments," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation.  "Senator Whitehouse has been an invaluable champion for the CF community, and we are grateful to him for his work on this critical legislation."  "I've heard from so many people with rare diseases who are frustrated with waiting for promising new treatments to gain approval, and who want to help speed up the process,"  said Whitehouse. "This bill accomplishes that goal by encouraging the FDA to take advantage of the wisdom and insights of rare disease experts and patient advocates in approving new treatments.  It's an important step toward providing faster access to promising new therapies, and I thank everyone who supported this bill for helping me to push it across the finish line." Passage of this legislation follows the recent approval of Kalydeco™, a cystic fibrosis drug developed by Vertex Pharmaceuticals with financial, scientific and clinical support from the Cystic Fibrosis Foundation.  Kalydeco's swift approval, announced by the FDA just three months after it was submitted for review, demonstrates how a well-informed, collaborative FDA review process involving reviewers, industry partners, patients and cystic fibrosis experts can efficiently move lifesaving therapies to market.  Kalydeco is the first CF drug to address the underlying cause of the disease in select CF patient groups. The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more cystic fibrosis research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The Cystic Fibrosis Foundation is a donor-supported nonprofit organization.