Rare Diseases

A look at some key statistics on rare diseases in the US

The global pharmaceutical industry is make huge investments in treatments for rare diseases, which once drew little interest from major drugmakers. 

A look at some key statistics on rare diseases:

- Patients with a rare disease in the U.S.: 30 million, nearly 1 in 10 people.

- Rare disease patients worldwide: 350 million, more than cancer and AIDS patients combined.

- Percentage of rare disease patients who are children: 50 percent.

- Threshold to be classified as a rare disease: fewer than 200,000 Americans affected.

- Number of known rare diseases: 7,000, with more discovered every year.

- Percentage of rare diseases caused by abnormal genes: 80 percent.

- Percentage of rare diseases with an FDA-approved treatment: 5 percent.

- Rare-disease drugs approved in the 1970s: fewer than 10.

- Rare-disease drugs approved since the 1983 Orphan Drug Act: 220.

- Rare-disease drugs approved in 2014: 17.

- New medicines in development for rare diseases: more than 450.

- U.S. venture capital funding of companies developing rare-disease drugs in 2003: $268 million. In 2013, $557 million.