Specialty pharmaceuticals refer to pharmaceuticals used to treat diseases diagnosed by a specialist, often connected to a hospital, and where the patient’s treatment is controlled by the specialists. Patients can be treated either in open care or in hospitals.
Orphan medicinal products
Orphan medicinal products or orphan drugs are part of the market for specialty pharmaceuticals and refer to drugs used for diagnosis, prevention or treatment of rare diseases that are chronic, debilitating and often life-threatening.
An increasing number of treatments
In 2015, the European Commission approved more medicines for rare diseases than ever before – of 93 newly approved medicines, 18 had an orphan designation. Similarly, the US Food and Drug Administration (FDA) approved 45 new therapies in 2015, of which 21 were orphan drugs. However, the key aspect of securing timely and sustainable patient access to these newly approved therapies remains.
The main driver of this growth is successful legislation combined with the availability of infrastructure and technological advancements that have helped to spur momentum worldwide.