These days, if pharma executives aren't talking about targeted drugs and their companion diagnostics, they're touting the promise of rare-disease treatments. In what might be Exhibit A for that argument, Forbes profiles Alexion Pharmaceuticals, which made its name--and built its income statement--on Soliris, a drug now approved for a variety of rare-and-frightening disorders.
Alexion launched Soliris in 2007 for one indication--to treat patients with a rare and terminal form of anemia. Since then, it has broadened the drug's market and pumped up its sales by 600%, Forbes points out. Expected 2012 sales? $1.1 billion.
So, Soliris is one of Big Pharma's next new breed: The very expensive treatment for very few patients. At $440,000 per year, Alexion only has to reach a few thousand patients to hit blockbuster territory. And payers can hardly balk at covering a treatment that can relieve intense suffering and prolong patients' lives. Even penny-pinching governments fork over the money, Alexion CEO Leonard Bell told Forbes.
That's not to say that super-expensive products that target super-small niches offer infinite promise. Insurers and government payers can't foot the bill for an unlimited number of $200,000-and-up drugs, no matter how effective.
- read the Forbes story