U.S. Senate Passes EXPERRT Act — Bill Increases Patient and Expert Participation in FDA Review of CF and Other Rare Disease Drugs
June 27, 2012
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.
Passage of this legislation follows the recent approval of Kalydeco™, a CF drug developed by Vertex Pharmaceuticals with financial, scientific and clinical support from the CF 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 collaborators, patients and cystic fibrosis experts can efficiently move lifesaving therapies to market.