NCPA - National Center for Policy Analysis


March 19, 2008

When a drug can cost more than $300,000 a year, the right dose becomes a matter of public debate, say observers.  Cerezyme, the drug in question, is used to treat a rare inherited enzyme deficiency called Gaucher disease.  Some experts say that for most patients, as little as one-fourth the standard top dose would work, which could save the health care system more than $200,000 a year per Gaucher patient.

The dispute over Cerezyme could be a sign of the increased scrutiny that dosing will receive as drugs become more expensive.  Pharmaceutical companies have faced complaints for years over prices, but now they might have to defuse efforts to use less of their drugs to cut costs, and to rebut accusations that doses are inflated to bolster profits.

There are also economic and social questions about how much burden taxpayers and co-workers should be expected to bear; ultra-expensive drugs, especially in full doses, can raise the cost of everyone's insurance.  With Cerezyme, which is made by Genzyme, the profits are sizable:

  • Gaucher disease, which can have complications like ruined joints, is rare; only about 1,500 people in the United States are on the drug and about 5,000 worldwide.
  • Sales of Cerezyme totaled $1.1 billion last year, making it a blockbuster by industry standards.

Genzyme is not the only company feeling some pressure:

  • Some lung cancer doctors are using only half the approved dose of Genentech's drug Avastin, after a recent study suggested the half-dose might be as effective as the full one, which can cost $100,000 a year.
  • Scientists reported last year that a two-hour infusion of Schering-Plough's anticlotting drug Integrilin worked just as well for some patients as the standard 18-hour infusion, saving hundreds of dollars per treatment.
  • And doctors are sharply reducing their use of anemia drugs sold by Amgen and Johnson & Johnson.

Source: Andrew Pollack, "Cutting Dosage of Costly Drug Spurs a Debate," New York Times, March 16, 2008.

For text: 


Browse more articles on Health Issues