Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including 7 years’ marketing exclusivity and tax breaks. Last year, just 250 requests for orphan-drug designation were filed, and 160 were successful. There are roughly 350 orphan drugs approved, covering about 150 rare diseases. Timothy Coté, director of the FDA’s Office of Orphan Products Development, said that his office is offering 2 workshops with on-the-spot regulatory advice to try to elicit more orphan drug applications. Over the course of the 2-day workshop, each company met 4 times with FDA staffers who offered advice on 9 critical issues in filling out an application. Orphan drug applications are 6-7 pages. At the conclusion of the first workshop, only 14 of the 29 companies submitted applications, although they can still submit at any time. It usually takes the FDA 60 days to determine whether the designation will be given. Up to 50 more organizations can attend a second workshop to be held at the University of Minnesota in August 2010. Dr. Coté said he is considering a workshop in Europe. Next time, he wants only applicants who can file, without the delay of company approval, at the end of the 2 days to attend.
Source: Wall Street Journal (digital)