At the hearing, Beall discussed the CF Foundation's recent successes in drug development and made recommendations for ways Congress can help advance treatments for rare diseases. In particular, his remarks emphasized the importance of patient involvement throughout the development of new treatments.

“Congress should make sure patients have a seat at the table, because no one understands a disease better than the people who live and fight it every day,” said Beall.

Co-sponsored by House Energy and Commerce Committee Chairman Fred Upton (R-Mich.) and Representative Diana DeGette (D-Colo.), 21st Century Cures is aimed at developing policies that will accelerate the discovery, development and delivery of treatments and speed biomedical innovation. Through this initiative, the Energy and Commerce Committee is soliciting input from industry, patients, researchers, government agencies and other stakeholders about ways the government can incentivize and accelerate the development of cures.

Other panelists at the hearing included Janet Woodcock, M.D., Director of the U.S. Food and Drug Administration's Center for Drug Evaluation and Research and representatives from the American Cancer Society, Parent Project Muscular Dystrophy, the American Cancer Society and the National Organization for Rare Disorders.