Orphan drugs are medicinal products intended for the diagnosis, prevention, or treatment of life-threatening or very serious diseases or disorders that are rare. A disease or disorder is defined as rare when it affects less than 1 in 2,000 citizens, some rare diseases include Moebius syndrome, Stoneman syndrome, alkaptonuria.
These drugs are called “orphans” because, under normal market conditions, the pharmaceutical industry has little interest in developing and marketing products intended for only a small number of patients. For drug companies, the extremely high cost of bringing a medicinal product to market would not be recovered by the expected sales of the product.
As a result, the potential market for new drug treatment is also small and the drug companies’ industry would incur a financial loss.
Therefore, governments and rare disease patient organizations advocate for economic incentives to encourage drug companies to develop and market medicines for rare disease treatment.
