Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments
BETHESDA, Md., May 10, 2012 /PRNewswire-USNewswire/ -- The Cystic Fibrosis Foundation issued the following statement regarding the House Energy and Commerce Committee's Health Subcommittee approval of legislation to include consultation with rare disease experts and patient advocates as part of the U.S. Food and Drug Administration's (FDA) systematic drug review process. [Details provided below.]
Statement from Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation:
"On behalf of the millions of Americans with cystic fibrosis and other rare diseases, we are heartened that the voice of patients and experts will be heard as more sophisticated therapies for complex rare diseases are advanced. The Cystic Fibrosis Foundation applauds members of the House Energy and Commerce Committee's Health Subcommittee and bill sponsors Representatives Edward Markey, Tom Marino and Cliff Stearns for their support of the EXPERRT Act. This legislation will ensure that FDA reviewers have the most complete information when evaluating new treatments and help bring these therapies more quickly to the patients who need them."
Comments from Bill Sponsors
- According to the lead sponsor of the EXPERRT Act, Congressman Edward J. Markey (D-MA): "The inclusion of the EXPERRT Act in the FDA Reform Act ensures that sharing knowledge amongst the FDA and individuals with expertise in rare diseases will become common practice.