Rare diseases are defined as diseases or disorders that cause genetic origin, low incidence (life-threatening or chronic depressive, resulting in premature deaths and visible decline in the quality of life or socio-economic potential of the individual. Orphan drug; Is the term used for the treatment of rare disorders; Because the number of patients affected is so low that it is not profitable to invest or market research and development and marketing. Orphan medicines are considered the most expensive medicines in the world. Rare diseases entered the health agenda when the Orphan Drug Regulation was issued in the US in 1983. As a result of the investigations, it has been found that there are no regulations on orphan medicines in Turkey and that it is necessary to develop orphan medicines are brought from abroad by Turkish Pharmacists Association (TPA) and it is not taken into the scope of Social Security.
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