Orphan Drug Designation

Orphan Drug Designation (ODD) in the U.S. means that a drug has been designated by the U.S. FDA as an “orphan drug” pursuant to the Orphan Drug Act of 1983. This law was passed to facilitate the development of drugs for rare diseases that affect small numbers of people living in the U.S. (which is defined as affecting less than 200,000 people in the U.S.). Orphan drug designation does not mean that the drug is safe or effective, and does not mean that it is legal to market or sell the drug in the U.S. Rather, the designation means only that the drug’s sponsor qualifies for certain benefits from the U.S. government, including tax incentives and market exclusivity for 7 years. Orphan drugs normally follow the same regulatory approval process as other drugs (including dosing, safety and efficacy). However, some statistical requirements are lessened (such as not requiring the same number of patients in a Phase III clinical trial as would be required for a non-orphan drug), in order to try to provide a more streamlined process for gaining ultimate approval. Japan adopted a similar law in 1993; the European Union adopted a similar law in 2000.

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