Rare diseases impact millions of people worldwide

Rare diseases, while individually uncommon, collectively affect a large portion of the population, leading to a substantial public health impact. More than 10,000 rare diseases affect nearly 30 million people in the United States, and about 400 million worldwide. When you add up all the related direct and indirect costs, rare diseases carry a U.S. economic burden of nearly $1 trillion every year. They often present complex challenges in diagnosis, treatment, and management.  Many of the diseases have a chronic clinical course and with impact on affected patients and their families, including physical and intellectual disabilities and premature death.