The roundtable discussion, hosted by Representative Diana DeGette (D-CO), focused on personalized medicine and its implications for research, drug development and patients. Dr. Accurso advocated for people with cystic fibrosis and suggested ways that Congress can support biomedical innovation and drug development. He emphasized the need for greater collaboration in drug discovery and development, the development of patient registries and clinical trial networks like those employed in the CF community and increased patient involvement in the process. He also urged policymakers to sustain increased funding for the NIH and the Food and Drug Administration (FDA).

Dr. Accurso and others pointed to Kalydeco, the first drug to treat the underlying cause of CF, as a great success story in personalized medicine. Kalydeco was approved in one of the fastest and most efficient FDA approval processes to date.

This event was held as part of the House of Representatives' “21st Century Cures” initiative. Co-sponsored by Rep. DeGette and House Energy and Commerce Committee Chair Fred Upton (R-MI), the initiative is focused on developing policies to accelerate the discovery, development and delivery of treatments and promote medical innovation. Robert J. Beall, Ph.D., president and CEO of the CF Foundation, also testified at a “21st Century Cures” hearing on patient participation in drug discovery and development in Washington, D.C., in July.

To learn more about the roundtable or the 21st Century Cures Initiative, visit http://energycommerce.house.gov/cures.