The Burrill Report


The Burrill Report (July 9, 2012): Rare Disease Community Scores a Big Victory (.MP3,11.8 Mb)

Congress has passed the Food and Drug Administration Safety and Innovation Act, which renews the Prescription Drug User Fee Act for another five years. That allows the FDA to continue to collect user fees from industry to fund the review of new drugs. But perhaps the biggest winner in this new incarnation of PDUFA is the rare disease community, which won significant concessions in the legislation. We spoke to Emil Kakkis, president of the Everylife Foundation for Rare Diseases about the legislation, why rare disease advocates were so successful in shaping the law, and how it will change the landscape for rare disease drug development.

July 06, 2012
http://www.burrillreport.com/article-rare_disease_community_scores_a_big_victory.html