Of the more than 6000 diseases classified as orphan diseases, fewer than ten per cent have therapies that directly address the underlying disease. Additionally, the level of investment in rare disease research has traditionally been low, resulting in limited advances.
Pfizer said its Rare Disease Research Unit will work across all disease areas, discovering small molecule and biopharmaceutical drugs. All existing research relating to rare disease will be folded into the unit, to be based in Cambridge, in the US.
The company said it also intends to work closely with patient advocacy groups as it develops and advances the unit’s research strategy.
Peter Saltonstall, President and CEO of US group, the National Organization for Rare Disorders (NORD), welcomed the move. “Approximately 30 million Americans, 30 million Europeans and millions more around the world have rare diseases and, for most of those people, there is no specific treatment. As the voice of the U.S. rare-disease patient community, NORD applauds Pfizer’s commitment to expanding its research with the goal of development new treatments for this medically underserved population.”
The Rare Diseases Research Unit will be lead by Edward Mascioli, the founder and CEO of Dapis Capital, a private equity firm specialising in healthcare and the life sciences. He is also a former Vice President of Clinical Affairs at the biotech Peptimmune and Senior Medical Director at the contract research company Paraxel.