World "Rare Disease Day"

The last day of February has been designated as World “Rare Disease Day” to call attention to the public health issues associated with rare diseases. The Project Charity — The Children’s Rare Disease Network has compiled some facts and figures about rare disease that we thought would be of interest. If you have other facts and figures not on our list, please send them to us. We are particularly interested in international facts on rare disease that do not seem to be available.

DID YOU KNOW… Approximately 7,000 rare disorders are known to exist and new ones are discovered each year

Rare disease affects between 25-30 million people in the United States and approximately 30 million people in the European Union

One in 10 Americans is living with a rare disease

Children represent the vast majority of those afflicted with rare disease

Approximately 80 percent of rare diseases are not acquired; they are inherited. They are caused by mutations or defects in genes

In the United States, rare diseases are defined as those affecting 200,000 or fewer people or about 1 per 1,000

Rare disease is often referred to as an “orphan” disease

Orphan or rare diseases are often not pursued by the pharmaceutical industry because they provide little financial incentive for the private sector to make and market new medications to treat or prevent them and because there are not enough patients to make research cost-effective

Research on rare diseases can often lead to advances in our understanding of common diseases such as cancer, heart disease, diabetes, stroke and other major health problems
As a whole, rare diseases represent a large medical challenge. Combine this with the lack of financial incentives to treat or cure rare diseases, and a serious public health issue is created

The US Orphan Drug Act (ODA) of 1983 has been one of the most successful pieces of health related legislation ever enacted in the United States. Through a system of tax credits, government grants, assistance for clinical research, as well as seven years marketing exclusivity, the Orphan Drug Act has resulted in hundreds of approved orphan medicines, treating over millions of patients worldwide. Similar legislation has been adopted in Japan, Australia and the UK