Pfizer has formed a new rare disease unit to “significantly expand” its roster of medicines for uncommon but lucrative to treat diseases. The division will serve as an umbrella for rare disease programs the company is already pursuing, such as treatments for the rare genetic disorder Gaucher disease, hemophilia and Ewing's sarcoma, an unusual bone and tissue cancer.

 

“We are coupling Pfizer's existing experience in rare diseases, such as hemophilia, with our advanced protein technologies, resources, and world-class scientific team to focus on becoming a driving force in rare disease research,” says Jose Carlos Gutierrez-Ramos, senior vice president of Pfizer’s biotherapeutics research and development group.

 

Pfizer did not disclose how much of its nearly $8 billion annual research budget it intends to devote to the new unit, but Anne Wilson, a company spokesperson, says it would be “proportional to the medical need” in each rare indication addressed, with a focus on what Pfizer does best, protein design and manufacture and clinical development.

 

GSK, Novartis, and a growing number of venture firms, such as Third Rock Ventures, are beginning to see the potential for profit in niche diseases, a segment in which Genzyme and Shire have found proven riches. The high price tag generally attached to specialty treatments, made in small batches for patient populations of fewer than 200,000, is also drawing Big Pharma interest as it becomes clear that fewer blockbusters are going to be padding their bottom lines.

 

Of course, that very same attribute is beginning to generate “significant payer interest in developing cost containment strategies targeted to this sector” too, notes Pfizer in its most recent annual report.

 

“While the impact on Pfizer of payers’ efforts to control access and pricing of specialty pharmaceuticals has been limited to date, our growing portfolio of specialty products, combined with the increasing use of health technology assessment in markets around the world and the deteriorating finances of governments, may lead to a more significant adverse business impact in the future," it says.

 

The company's new rare diseases research unit will be lead by Edward Mascioli. Mascioli was most recently the founder and CEO of Dapis Capital, a private equity firm focused on healthcare and the life sciences.