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By Sarah Rickwood
The 2020s are the age of orphan medicines.
Orphan medicines are available for a larger number of diseases and patients than ever before, a testament to the success of legislation established decades ago encouraging the development of these medicines in both the U.S. and European Union.
Orphan medicines were the majority of European Medicines Agency (EMA) approvals for the first time ever in 2016 (59%) and the majority of U.S. Food and Drug Administration (FDA) approvals for the first time ever in 2018 (58%).