FDA Sponsoring Workshops To Increase Rare Disease Treatment Options

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The Wall Street Journal: FDA is putting on workshops for pharmaceutical manufacturers to increase the pool of applicants for approved "orphan drugs," that treat rare diseases. Currently, there are about 7,000 so-called orphan diseases in the U.S. that have few or no FDA-approved treatments.

"Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including seven years' marketing exclusivity and tax breaks. Last year, just 250 requests for orphan-drug designation were filed, and 160 received it." The workshops help companies through the application process by offering regulatory advice. The first, held last month in Claremont, Calif., attracted "29 potential sponsors, from major drug companies to academic centers, small biotechs and even some patient advocates. In a follow-up survey, 74% said they had never before filed an application for orphan drug designation" (Marcus, 3/10).

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