Education Forum - Orphan diseases and drugs

The World Health Organization defines orphan diseases, as all pathological conditions, affecting 0.65-1 out of every 1000 inhabitants. They are usually not studied for their pathophysiology or for newer therapeutic options, as these are not economically viable. The Orphan Drug Act was passed on January 28, 1983 by USA to stimulate the research, development and approval of those products that treat orphan diseases. Till date, 11 drugs (4.87%) for tropical infectious diseases have been designated with orphan drug status and as many drugs for other infectious diseases. Several drugs with orphan status are used in the treatment of diseases that no longer meet orphan status criteria, such as AIDS and end-stage renal disease. Understanding of the human genome, nuclear cloning, rational drug designing and application of high throughput screening in drug discovery programs, might lead to new drug discoveries for orphan diseases. Hence, there is hope in future for patients neglected by for-profit drug discovery efforts.

Keywords
Drugs for neglected diseases initiative, neglected diseases orphan drugs.