Pfizer Inc. announced the creation of a new research unit focused on rare diseases. This new unit will significantly expand Pfizer's presence in rare disease research with the goal of discovering novel, life-saving medicines for diseases affecting less than 200,000 patients. Its creation within Worldwide Research and Development represents a further step in Pfizer's strategy to apply our strong science and technology base to find innovative new treatments for patients with large unmet medical need.
"We are very excited about our new Rare Diseases Research Unit," said Jose Carlos Gutierrez-Ramos, PhD, senior vice president, BioTherapeutics Research and Development. "We are coupling Pfizer's existing experience in rare diseases, such as haemophilia, with our advanced protein technologies, resources and world-class scientific team to focus on becoming a driving force in rare disease research. Pfizer has a long history in discovering, developing and commercializing medicines that treat rare diseases and we are hopeful that this research unit will lead to additional new medicines for patients suffering from devastating illnesses for which there is no cure."
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 needed scientific advancements in this field. Pfizer's Rare Disease Research Unit will pursue treatments across all therapeutic areas and modalities and will serve as the focal point for the company's existing research on rare diseases. The company intends to work closely with patient advocacy groups as it develops and advances the unit's research strategy.
"NORD is very happy that Pfizer is establishing a Rare Diseases Research Unit," said Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD). "Approximately 30 million Americans, 30 million Europeans and millions more around the world have rare diseases and, for mores of those people, there is no specific treatment. As the voice of the US 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, M.D. Dr. Mascioli was most recently the founder and CEO of Dapis Capital, a private equity firm focused on healthcare and the life sciences. He has extensive experience in clinical development from his time as vice president of clinical affairs at Peptimmune Inc and senior medical director at Paraxel. Dr Mascioli also has a strong background in both business development and academia. He will be based in Cambridge, Mass and will report into Dr Gutierrez-Ramos.