Rare Disease Day: What is pharma doing for rare diseases?

In 2023, more than 50% (28 of 55) of the novel drugs reviewed by the US Food and Drug Administration (FDA) received orphan drug designation for the prevention, diagnosis, or treatment of a rare disease.1

However, there are a reported 6,000 to 10,000 identified rare diseases, affecting over 400 million people worldwide2,3 – so, while rare diseases are a growing field of interest to pharma, they remain an unmet clinical need.

When compared to more common illnesses, rare diseases have historically received less attention, funding, and treatment development.4 Yet, if the pharmaceutical industry's increasing focus on these lesser-known conditions continues, this narrative is going to change. For Rare Disease Day, February 29, 2024, this article examines the different layers of pharma’s commitment to rare diseases.