Trends in Development of Orphan Drugs - A Review

Orphan drugs are pharmaceutical agents that treat rare medical condition known as orphan or rare disease. According to U.S Rare Disease Act of 2002, any disease that affects less than 200,000 patients or 1 in 1500 is termed as a rare disease [1-3]. European Union defines rare disease as a life threatening or chronically debilitating disease which is of such low prevalence that special efforts are needed to address it.” It should affect 1 in 2000 persons [2,3]. European Union definition for rare disease also includes some tropical diseases found in developing countries [3]. There are around 5000-8000 rare diseases reported, out of which only 10% have cures [4]. According to the European Organisation for Rare Disease (EURORDIS) 80% of rare diseases are genetic in nature [5].