About FDA

Description: The purpose of the workshops is to directly facilitate the development of Orphan Drug designation applications. Two workshops will be held: one in California in February 2010 and the other in Minnesota in August 2010. Both workshops are co-sponsored with academia and patient organizations. FDA staff will be on hand at these workshops to answer questions as applicants construct their applications.

Accomplishment: In co-sponsoring the workshops, OOPD was successful in meeting its goal to increase the number of promising applications for orphan drug status by offering detailed explanations and hands-on guidance to academic researchers, small biotechnology companies, pharmaceutical firms and other potential drug sponsors. 65 sponsor groups and an estimated 101 participants joined the workshops which were specifically designed to help simplify and clarify the orphan drug designation process for those actually working on a designation application in accordance with applicable regulatory criteria. The workshops also increased the positive interactions of FDA with each of the co-sponsorship organizations that focus on rare diseases and orphan drugs as well as educating more potential drug sponsors about the designation process. As a result of this workshop, attendees submitted 44 orphan drug designation applications for review.