Rare diseases overview for a clinical trial perspective

Rare diseases are those diseases which affect a very few number of people. They are also termed as orphan diseases and are associated with improper management, long term deterioration, adverse effects and fatalities as well. As per the Committee for Orphan Medicinal Products (COMP), European Medicines Agency (EMA), a disease can be categorized as a ‘rare disease’(RD) when the number of affected individuals is less than 5 members across 10,000 individuals in the European Union EU (1). The United States Food and Drug Administration (USFDA) designates a disease as ‘rare’ if the number of people in the USA suffering from it are less than 2,00,000 i.e. less than 6.4 persons in 10,000 (2).