Article: Balancing Expectations: An Unmet Need In The Orphan Disease Arena

There are between 6000 and 8000 rare diseases in the world, and one in ten people in Europe and the US have a rare disease; 75% of rare diseases affect children, and 30% of rare disease patients die before the age of 51. Over 95% of these rare diseases have no treatment options available—instilling a devastating burden on patients and their families2. While the pharmaceutical industry is focusing on innovation to address this significant unmet need among patients, such dedication is not entirely altruistic. Commercial opportunity in orphan medicinal products (OMPs) is estimated to reach $209bn by 2022 and constitute one of the largest growing parts within the prescription drug segment3, continuously drawing investment from small, medium and large companies alike.