The numbers may surprise you. It is known that there are about 7000 rare diseases, and approximately 30 new diseases are identified each year. Also known as orphan diseases, they are known as diseases that affect less than 200,000 patients in America. Although it may be true that every rare disease affects only a few patients, millions of people depend on the development of innovative treatments and medicines.
In a series of new charts, BIO demonstrates how far we have come – and how far we have to go – in fighting rare diseases. You can see them here and here.
The orphan drug law gave families and individuals suffering from rare diseases such as Hodgkin's lymphoma and cystic fibrosis hope and optimism. Since the passage of the law in 1983, hundreds of new treatments have been approved by the US Food and Drug Administration for the treatment of these rare diseases – compared to only 34 testimonials before their age.
Prominent scientists, experienced businessmen, business leaders, committed investment communities, universities and world-class research institutions are essential for the development of great medicines and treatments. But as the success of the orphan drug law demonstrates, the environment and public policy incentives are also crucial factors in stimulating innovation in rare diseases. In order to build on the historical successes of the orphan drug law, it is important that the incentives provided by the law to encourage the development of treatments for rare diseases be maintained.
To learn more, visit www.bio.org/toolkit/disease-area.