On Rare Disease Day, celebrating 30 years of orphan drugs


Tony Jewell

By Rachelle Benson

Rare Disease Day is an international advocacy day to raise awareness of rare diseases as a global health challenge.

Thirty years ago, President Ronald Reagan signed into law the Orphan Drug Act (ODA), named for its focus on orphan—or rare—diseases.

Individuals with rare diseases—more than 25 million in the US alone—may have struggled to obtain a proper diagnosis, find information, and get treatment due to the rarity of their conditions and lack of relevant medical research. With its approval in 1983, however, the ODA encouraged companies to develop medicines and diagnostic tools for rare diseases by providing financial incentives.

Between 1973 and 1983, less than 10 products for rare diseases were developed without government assistance. Since the Orphan Drug Act was enacted, more than 400 orphan products have been approved by the Food and Drug Administration (FDA). With the help of patient advocacy organizations, especially the National Organization for Rare Disorders, advances have been made in areas such as research, legislation, and patient support.

Even with recent developments, however, there are approximately 6800 rare diseases that do not have an FDA-approved therapy. AstraZeneca is committed to fighting rare diseases through ongoing research, and in fact, AstraZeneca achieved FDA approval on its first orphan drug in 2011. We encourage you to join us by participating in Rare Disease Day on February 28. For more information, please visit http://rarediseaseday.us