An orphan drug is a medicinal product developed for the treatment of a rare disease. In Europe, the official definition of 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.

Additionally because the number of patients is so small a rare disease is often not “adopted” by the pharmaceutical industry (hence the expression orphan drug). It normally takes about 10 years to develop a new drug and for a traditional pharmaceutical company the diseases affecting much greater patient populations often take priority.

To provide care for these “forgotten patients” and to encourage pharmaceutical and biotechnology companies to invest in treatment for rare diseases, governments have created various legal and financial incentives. In 1983 the Orphan Drug Act was introduced in the US and in 1999 the “Regulation on orphan medicinal products” was approved by the European Parliament. This regulation grants, among other things, a ten-year market exclusivity for any new orphan drug.

Orphan Europe has been developing and providing orphan drugs for the treatment of rare diseases since 1990, well before the orphan drug legislation was introduced in Europe. We have the conviction that every patient has the right to the best possible treatment, especially patients suffering from a rare disease.