LANDMARK RARE DISEASE LEGISLATION SIGNED INTO LAW BY THE PRESIDENT

President Obama signed into law yesterday the U.S. Food and Drug Administration (FDA) Safety and Innovation Act of 2012 which includes the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983. The Act will provide more than $6 billion in industry user fees to the FDA over the next five years to fund a share of the agency's review of drugs and medical devices. For rare disease patients, including pachyonychia congenita (PC) patients,  it also includes provisions of critical importance. These come as a result of years of advocacy, education and relationship-building by a coalition of rare disease patient organizations, including PC Project. These provisions will accelerate rare disease patient access to new medical treatments, speed-up development of "breakthrough therapies", and improve consultation between the government and rare disease experts.

This legislation passed with bipartisan support in both the House and Senate. The FDA Commissioner, Margaret Hamburg, MD, as well told Congress that the FDA was committed to greater support of the rare disease program and closer collaboration with all those affected by rare disease research. 

People with pachyonychia congenita, a rare disease affecting roughy several thousand world-wide, will benefit from this increased rare disease focus by the U.S. Government.  PC Project's ongoing efforts to get cllinical trials underway for  PC patients will be greatly assisted by this Act's provisions.   We are thrilled to know we will be working in a governmental environment that will help us overcome regulatory and financial hurdles that are present in the drug development and approval process. We are grateful to everyone involved who worked on behalf of making these new rules a reality.