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12:00 AM
 | 
Apr 16, 2007
 |  BioCentury  |  Strategy

Alexion's pricing rationale

At $389,000 per year, the cost of Alexion Pharmaceuticals Inc.'s Soliris eculizumab, newly approved for paroxysmal nocturnal hemoglobinuria, comes in at the high end of drugs for ultra-orphan diseases. Still, the company does not expect payer coverage to be a problem, and Kaiser Permanente, for one, says it is less concerned about the prices of these kinds of drugs than the high prices of more widely used drugs with only marginal efficacy.

David Hallal, VP of U.S. commercial operations at ALXN (Cheshire, Conn.), told BioCentury the company took four main factors into consideration before establishing the price. These included the rarity and life-threatening nature of the disease; the "compelling clinical benefits" afforded by the drug; the costs of discovery, development, production and ongoing research; and "an ongoing commitment to the PNH community," including programs to help both uninsured and underinsured patients pay for treatment.

ALXN estimates there are 8,000-10,000 patients in Europe and North America with PNH, a debilitating genetic disorder characterized by hemolysis, or the chronic destruction of red blood cells. Although there is no formal definition of "ultra-orphan," the U.K.'s National Institute for Health and Clinical Excellence (NICE) uses the term for conditions occurring in less than 1,000 people in the U.K.

The estimated median survival for PNH...

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