LANDMARK RARE DISEASE LEGISLATION APPROVED BY CONGRESS!

Landmark legislation passed the U.S. Senate today aimed at helping to promote the development of rare disease therapies. Senate Bill 3187, the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) will improve the FDA regulatory process, provide incentives to invest in rare disease therapies, and expand scientific capability to develop cutting-edge therapies. The House of Representatives approved the bill last week and it will now go to President Obama for his signature. 

The bill's features important to rare disease patients will (1) enhance accelerated patient access to new medical treatments, (2) encourage the development of Humanitarian Use Devices, or medical devices for small patient populations, (3) provide accelerated development of "breakthrough therapies" - or ones that show early promise, (4) enhance consultation with rare disease medical experts, and (5) establish a rare pediatric disease priority review voucher incentive program. 

PC Project has been working with the rare disease coalitions that have been providing input on, and supporting, this legislation.