RECENT DEVELOPMENTS ON ORPHAN DRUG AND LEGAL ARRANGEMENTS CURRENT SITUATION
Project no. 21148 · Status: Kapanmış · 2019
Summary
Rare diseases are those in which a very low number of people are affected within the total population. Rarely, 6-8 thousand rare diseases are determined today and they are added to the literature regularly as new diseases are discovered. Rare diseases are very chronic and progressive diseases with very serious consequences. There is very little scientific and medical knowledge about rare diseases. Most of the rare diseases do not have a definitive treatment, but it is aimed to increase the quality of life of the patient by reducing the symptoms as much as possible with appropriate drug treatment and care. Orphan drugs are medicinal products developed for use in the diagnosis, treatment and prevention of rare diseases. Pharmaceutical manufacturers are often reluctant to produce and develop orphan drugs. The reason for this is that the production processes of orphan drugs are quite long and expensive.