A review of orphan drugs and rare diseases
Journal Title: International Journal of Research in Pharmacology & Pharmacotherapeutics (IJRPP) - Year 2016, Vol 5, Issue 2
Abstract
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 path physiology 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 has 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 the future for patients neglected by for-profit drug discovery efforts.
Authors and Affiliations
Venkat Paluri
Keywords
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