Rare Diseases & Orphan Drugs

An orphan drug is a medicinal product developed for the treatment of a rare disease. The MHRA defines a condition or disease as rare, if it affects fewer than 5 in 10,000 people in Great Britain.

There are over 6,000 known rare diseases, with at least 80% of these having an identified genetic origin. But the total number of identified rare diseases is increasing as genetic research is beginning to explain disease patterns that we didn’t understand before.

This means that 1 in 17 people will suffer from a rare disease at some point – which equates to nearly 4 million people in the UK and 30 million people across Europe. Because they are predominately genetic, most rare diseases are present at birth with estimates citing that 50% of new cases of rare disease are in children.

Rare diseases are therefore not actually that rare at all. They represent a significant cause of illness, making considerable demands on the resources and capacity of health care services .

• On average it takes over 4 years to receive an accurate diagnosis
• The majority of rare diseases currently have no effective treatment

Recordati Rare Diseases has been developing and providing orphan drugs for the treatment of rare diseases since 1990, We believe that every patient has the right to the best possible treatment, especially patients with rare diseases.