EVERY YEAR, ON THE LAST DAY OF FEBRUARY, OVER 85 COUNTRIES AND REGIONS FROM AROUND THE WORLD TAKE PART IN RARE DISEASE DAY
A disease or disorder is defined as rare in Europe when it affects fewer than 1 in 2000.
A disease or disorder is defined as rare in the USA when it affects fewer than 200,000 Americans at any given time.
One rare disease may affect only a handful of patients in the EU (European Union), and another touch as many as 245,000. In the EU, as many as 30 million people alone may be affected by one of over 6000 rare diseases existing.
80% of rare diseases have identified genetic origins while others are the result of infections (bacterial or viral), allergies and environmental causes, or are degenerative and proliferative.
50% of rare diseases affect children.
(Source for all of the above: http://www.rarediseaseday.org
It takes, on average, 7.6 years in the US and 5.6 years in the UK for a patient with a rare disease to receive a proper diagnosis. http://www.geneticalliance.org.uk/d…report.pdf
There are approximately 25 to 30 million individuals in the United States living with a rare disease. This amounts to almost 8% to 10% of the population, or 1 of every 10 to 12 persons.http://www.raredr.com/resource-guid…2bvKC.dpuf
In a survey of US patients and caregivers, 37% said they had to borrow money from family/friends to pay for expenses. http://www.geneticalliance.org.uk/d…report.pdf
Since the Orphan Drug Act became law in 1983, about 500 orphan drugs have been approved for a rare condition by the FDA, and 3500 drugs have been given an orphan designation (not approved, but being investigated). In 2014, the FDA approved a record 46 orphan drugs. http://www.raredr.com/resource-guid…2bvKC.dpuf
Worldwide Clinical Trials has participated in the design, execution, and/or analysis of more than 50 studies in orphan diseases. Therapies have included small-molecule enzyme inhibitors, immunotherapy (monoclonal antibodies), hormone therapy, and enzyme replacement therapy http://www.worldwide.com