Rare And Orphan Diseases: Choosing A Path To Regulatory Approval And Market Success

By Sharon Ayd, Vice President, Principal Regulatory Center of Excellence, Advarra

Medical science has identified about 7,000 rare or “orphan” diseases. These conditions only affect a small percentage of the population: in the United States, we consider a rare disease as one affecting fewer than 200,000 people.

Historically, the small number of affected persons has meant a lack of market demand to encourage industry investment toward treatment. As a result, drug makers have typically paid less attention to rare diseases. Despite increased support from governments, regulators, and public opinion, significant challenges still continue for sponsors willing to bring these drugs to market. This white paper examines those challenges within the U.S. system and provides some guidance to sponsors on meeting them.