Environmental Valuation & Cost-Benefit News

When a drug can cost more than $300,000 a year, the right dose becomes a matter of public debate.

The drug in question, Cerezyme, 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, saving the health care system more than $200,000 a year per Gaucher patient.
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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.

In a letter to The Journal of Clinical Oncology last September, Dr. Ian E. Haines of Monash University in Australia argued that the doses of all three Genentech cancer drugs — Herceptin, Rituxan and Avastin — appeared to be higher than necessary, perhaps to bolster sales.

Dr. David P. Schenkein, a Genentech executive, said the doses were chosen solely for maximum effectiveness in fighting cancer.

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.
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According to Genzyme, the average dose in the United States is 51 units per kilogram every two weeks. At that dose, it costs about $350,000 a year to treat an adult weighing 70 kilograms, or 154 pounds.

But the worldwide average, even factoring in United States patients, is 34 units. And in Israel, the standard dose is 15 units per kilogram every two weeks.

“Patients are doing just as well on a treatment that is 75 percent less expensive,” said Dr. Ari Zimran, an Israeli Gaucher expert.

A study in the journal Blood in 2006 compared patients in the Netherlands, where a low dose is used, to those in Germany, where a high dose is typical. In the usual measures of disease severity — liver and spleen size and blood counts — there was no difference between the groups. But the German patients did better in two indirect indices of bone health.

Dr. Pramod Mistry, a Gaucher expert at Yale who receives some research funding from Genzyme, said he was caring for some of Dr. Beutler’s former patients who had become “very, very sick” because of under-treatment.
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By Andrew Pollack
The New York Times www.NYTimes.com
FOR FULL STORY GO TO:
http://www.nytimes.com/2008/03/16/business/16gaucher.html?th&emc=th
March 16, 2008