Rare Disease Drug Development

It is increasingly recognized that while each rare disease may affect a small number of individuals the collective burden of rare or orphan diseases is a major global healthcare challenge. The World Economic Forum estimates that 475 million people worldwide are affected by a rare disease. To mark World Rare Disease Day (28 February 2021), in this article Stephanie Finnegan, Cromos Pharma’s COO, explores the opportunities and challenges for rare disease drug development.