Rare Disease Day

At APC, we’re very proud of the fact that we help companies accelerate the development of therapeutics for rare diseases.  The impact that this work can have on the lives of real people desperately in need of a therapeutic is what inspires our  “Process Advancing Medicine” philosophy.

Here are some facts and figures which you may not know about rare diseases:

• A disease or disorder is defined as “rare” in Europe when it affects less than 1 in 2,000 people.  In the US, the Orphan Drug Act defines a “rare disease” as a disease or condition that affects less than 200,000 people in the United States.

• The total number of Americans living with a rare disease is estimated at between 25-30 million, whilst in the EU about there are about 30 million people suffering from a Rare Disease.

• It has been estimated that approximately  1 in 20 people will live with a Rare Disease at some point in their life.

• The exact cause for many rare diseases remains unknown.  However 80% of Rare Diseases are thought to be genetic in origin but environmental factors, such as diet, smoking, or exposure to chemicals, also can play a role in rare diseases. Such factors may directly cause disease, or interact with genetic factors to cause or increase the severity of disease. 

• Approximately 75% of rare diseases affect children.

Currently 7,000 Rare Diseases are known but only 5% have treatments.