FierceBiotech Special Report: Rare and Orphan Disease Drug Development


There’s a reason rare diseases are called orphans. Small disease populations represent small customer bases. Given that it can cost billions of dollars to bring a new drug to market, pharmaceutical companies have traditionally tended to shy away from such conditions in favor of more common indications.

Buoyed by financial incentives, dedicated regulatory paths, and highly engaged patient advocates, drug developers are devoting considerable resources to rare indications, and adopting some clever science to bring those orphan diseases home.

Companies like Pfizer, GlaxoSmithKline, and Shire Pharmaceuticals have established units dedicated to rare disease drug development. Last year Sanofi scored one of the largest rare diseases pipelines and businesses in the world via its acquisition of Genzyme for $20.1 billion. 

To read more about the challenges and opportunities associated with bringing drugs for rare diseases to market, download the complete report today.