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KMID : 1142220220170020137
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Regulatory Research on Food, Drug & Cosmetic
2022 Volume.17 No. 2 p.137 ~ p.149
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Orphan Drug Designation: A Comparison of Regulatory Systems among United States, European Union, and Republic of Korea
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Kim Chan-Ho
Lee Jang-Ik
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Abstract
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This article reports a comparison of regulatory rules and procedures for acquiring an orphan drug designation (ODD) from the Food and Drug Administration (FDA) in the United States, the European Medicines Agency (EMA) in the European Union, and the Ministry of Food and Drug Safety (MFDS) in Korea for the pharmaceutical companies that develop orphan drug products. The authors investigated, analyzed and compared the regulations and guidelines related to ODD. The regulations on an ODD by FDA, EMA and MFDS are very similar. In receiving an ODD, required are general information, descriptions of the target disease, sufficient scientific evidence showing the possibility that the drug can be applied to the target disease, and the estimation for the number of patients of the target disease. Public or scientific data on the number of patients and the pathophysiological characteristics of the target disease, and the mechanism of action and the clinical trial data to support the effectiveness of the drug need to be submitted to regulators for an ODD.
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KEYWORD
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rare disease, orphan drug, orphan medicine, orphan drug under development, orphan drug designation, orphan designation
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