Every patient has the right to the best possible treatment, and in particular patients suffering from a rare disease. Rare diseases are often genetic, metabolic diseases but a rare disease can exist in any medical field and concern patients of any age, sex or ethnicity. Due to the broad range of rare diseases doctors in any specialty, as well as general practitioners, may one day meet a patient with a rare disease. Orphan Europe, which is dedicated to the development, registration, marketing and distribution of unique treatments for rare diseases, currently provides treatment for a number of these in Europe and in other countries. Recordati Rare Diseases provides treatments for rare diseases in the U.S.A.
An orphan drug is a medicinal product developed for the treatment of a rare disease. A rare disease is a disease affecting less than 5 per 10, 000 inhabitants, is fatal, or severely debilitating. Currently over 6000 rare diseases are known, which means that several million people are affected all over the world –over 25 million in Europe alone. Today, treatment exists for only 200-300 of these 6000 diseases. Rare diseases are often genetic, meaning that newborns, children, and young adults often are affected. Physicians may never see a patient with a rare disease. For that reason there’s always a risk that when a baby is born with a rare disease, a correct diagnosis may not be made and appropriate treatment may not be given.