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Incentives For Orphan Drug Research And Development In The United States, Rosa Rodriguez-Monguio, Enrique Seoane-Vazquez, Sheryl Szeinbach, Jay Visaria

Rosa Rodriguez-Monguio

Background The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions. This study analyzed the characteristics of OD designations, approvals, sponsors, and evaluated the effective patent and market exclusivity life of orphan new molecular entities (NMEs) approved in the US between 1983 and 2007. Methods Primary data sources were the FDA Orange Book, the FDA Office of Orphan Drugs Development, and the US Patent and Trademark Office. Data included all orphan designations and approvals listed by the FDA and all NMEs approved by the FDA during the study …