Define Orphan Drug Act. Orphan Drug Act synonyms, Orphan Drug Act pronunciation, Orphan Drug Act translation, English dictionary definition of Orphan Drug Act. n. a drug that remains undeveloped or is neglected because of limited potential for commercial
4The wide range of conditions that fall within the definition of ‘orphan diseases’ has led to the emergence of an informal subcategory – called ultra-orphan diseases – to describe extremely rare conditions. The term has no formal legal definition but treatments for these very rare – ultra...
1. Introduction Orphan drugs, by definition, are intended for the treatment, prevention, or diagnosis of a rare disease (RD) or condition [1]. For the Food and Drug Administration (FDA), "supporting the development and evaluation of new treatments for rare diseases is a key priority" [2]...
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