Orphan Drug Development: David and Goliath

Orphan products, which include drugs and biologics, are an often-neglected area of clinical research. Orphan products are used to treat rare diseases or conditions that by definition, affect fewer than 200,000 people (or up to 1 in 1300) in the United States. Approximately 5000 orphan diseases have been identified, most of which lack effective drug treatment (1).