Drugs required for the treatment of rare diseases are often referred to as “orphan drugs” since they specifically target a limited group of patients. Diseases affecting very few people in a country or region may not be financially attractive for pharmaceutical companies. Consequently, government-funded research, private sector incentives, or other financial encouragements may stimulate the development of drugs for these Rare Diseases. It is widely recognized that some pharmaceutical companies are turning towards orphan drug production with the aim of enhancing patients’ quality of life and prolong their life spans, driven by a sense of social responsibility. However, unfortunately, this effort is not yet at a sufficient level.
In our country, there is no designated orphan drug status in the laws. Orphan drugs are subject to the same licensing and reimbursement processes as other drugs. The majority of orphan drugs in Turkey are produced abroad. Orphan drugs that are not licensed in Turkey can be delivered to patients through the Turkish Pharmacists’ Association.