Fact Sheet

Great Strides Against Rare Diseases

Great Strides Against Rare Diseases

PhRMA February 25, 2016

Although rare diseases each may individually only impact a relatively small number of patients, their impact on public health is far reaching. Rare diseases affect 30 million Americans, but many may go undiagnosed or misdiagnosed. Approximately 7,000 different rare diseases are known today, with likely many more still to be identified.

Developing medicines to treat rare diseases is particularly challenging, but America’s biopharmaceutical researchers have leveraged new technologies and the growing scientific understanding of many rare diseases to develop groundbreaking therapies over the past 10 years.