The Value Of Orphan Drug Designation

By Veeranna Lolla, Head of Regulatory Affairs, Pharmaceutics International

This year marked the 14th annual Rare Disease Day, which highlighted the reality that more than 90% of rare diseases still have no FDA-approved treatment.

The orphan drug market is dedicated to treating diseases that are considered rare and life-threatening to a small patient population. In the U.S., a rare disease is one that affects fewer than 200,000 people; approximately 7,000 rare diseases have been identified. While a rare disease affects a small number of people, in totality, rare diseases affect more than 25 million Americans, according to the National Organization for Rare Disorders.¹