As highlighted in a recent News Feature, Rare incentives (Nature Rev. Drug Discov. 7, 190–191; 2008), 2008 saw the 25th anniversary of the US Orphan Drug Act (ODA), which was promulgated in recognition of the economic barriers to developing drugs for rare diseases (defined as those that affect fewer than 200,000 persons in the United States). The ODA encourages the development of promising new compounds by providing significant fiscal benefits: FDA-enforced marketing exclusivity, tax credits and fee exemptions. These products are designated ‘orphan products’ by the FDA’s Office of Orphan Products Development, and if shown to be safe and effective can be approved for marketing. As of 20 July 2009, 343 orphan products have been marketed to treat rare diseases. By contrast, only 10 such products were approved in the 10 years preceding the ODA4.
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