Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies.

Background: The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding of rare disease drug development, as well as the repositioning business models of developers.

Research Design And Methods: We used a dataset containing all orphan drug designations between 1983 and 2019.