2021 in Review: Orphan drug development

By Dr Nicola Davies

In March 2021, the US Food and Drug Administration (FDA) announced its continued support for rare disease programs.1 So far, giant strides have been made in the search to find cures for diseases that affect a small percentage of the population, but are devastating in effect. The number of rare diseases is estimated to be between 5,000 and 8,000, and approximately 263-446 million people around the world are living with one or more of the diseases.2,3 Up to 71.9% of the diseases are genetic, often requiring personalized treatment, and 69.9% are first diagnosed in childhood.3 Quite often, these rare diseases are chronic and life-threatening.

Drug approvals