Rare Diseases & Orphan Drugs

An orphan drug is a medicinal product developed for the treatment of a rare disease. A rare disease is defined as a condition that affects a very small percentage of the population, and is fatal or severely debilitating. For instance, in Europe, it is defined for less than 1 patient per 2,000 inhabitants; in the USA, it is no more than 200,000. There are over 7,000 known rare diseases, which means that several million people are affected all over the world – over 25 million in Europe alone. Today, treatment exists for only 200 to 300 diseases. Rare diseases are often genetic, meaning that newborn babies, children, and young adults are often 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 and timely diagnosis may not be made and appropriate treatment may not be given.

Also, patient numbers are so small that a rare disease is often not 'adopted' by the pharmaceutical industry (hence the expression orphan drug).

To provide care for people with a rare disease 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 USA and in 1999 legislation was approved by the European Parliament. This legislation grants, among other things, market exclusivity for any new orphan drug.

Recordati Rare Diseases 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 believe that every patient has the right to the best possible treatment, especially patients with rare diseases.