The Orphan Drug Act of 1983 provides critical incentives and resources for those developing medicines for rare diseases. Under this legislation, a rare or “orphan” disease became defined as a disease or disorder affecting fewer than 200,000 people in the U.S. Since the passage of the Orphan Drug Act, the pace of development of new medicines for rare diseases has increased significantly. Since 1983, more than 350 new medicines to treat rare diseases have been approved FDA. Even with this progress, we still face a huge challenge. Almost 95% of all patients afflicted with a rare disease still have no treatment options. Despite nearly thirty years of effort by academia, government agencies, rare disease patients groups, the medical community and the private sector, we still have only about 420 approved therapies.