Rare diseases affect more than 30 million Americans. According to the FDA, a rare disease is defined as a disease or condition that has a prevalence of fewer than 200,000 affected individuals in the United States. An orphan product is a drug, biologic, device or medical food used for the prevention, diagnosis, or treatment of a rare disease.
We are on the verge of a medical renaissance, a period of accelerated progress in the treatment and management of rare diseases. This reawakening also brings with it challenges that we must grapple with.
Concerted effort by governments, regulators, and industry is rapidly expanding development of new therapies for the more than 7,000 known rare diseases.