Since the Orphan Drug Act of 1983, the profile of rare diseases and the search for orphan drugs has increased significantly. In recent years this has been reflected by big pharma companies, such as Pfizer, GlaxoSmithKline and Novartis, establishing their own rare disease units and renewing research efforts into rare diseases.
In an article by the American Council on Science and Health, Ed Mascioli, Head of Pfizer’s new rare disease R&D unit, indicates that “researching drugs for rare diseases have a single, known cause, which can be directly targeted. Thus the drugs treating them tend to be more effective and less risky”.
Big pharma have now joined the numerous smaller companies and patient organisations that have long existed in the orphan drug space and endeavoured to raise the profile of rare diseases for the patients in need.
Interestingly, Ed Mascioli along with leading figures from the rare disease and patient community will ascend on the World Orphan Drug Congress 2011 in Geneva on the 29th November – 1st December.