The marketing of Cerezyme, a drug used to treat the rare disorder Gaucher disease, provides an interesting case study of the methods used by drug makers to influence the treatment decisions made by patients and doctors.
Gaucher disease is a rare genetic disorder in which a person lacks an enzyme called glucocerebrosidase. Lack of the glucocerebrosidase enzyme causes harmful substances to build up in the liver, spleen, bones, and bone marrow and prevents cells and organs from working properly. The only drug available in the US to treat the disorder—Cerezyme— is made by one company called Genzyme. The only other drug approved in the US and overseas for Gaucher’s is Zavesca, sold by Switzerland’s Actelion. It is only approved for those patients for whom Cerezyme is unsuitable. Britain’s Shire and Israel’s Protalix Bio Therapeutics are in final clinical trials of enzymes similar to Cerezyme but made in ways that should avoid Genzyme’s patents. New Jersey’s Amicus Therapeutics is in mid-stage testing of another pill that works by a different means.
Because of the disease’s rarity and the drug’s treatment monopoly, many patients contact Genzyme directly for help. And sometimes, Genzyme tries to help Gaucher disease patients – whether they want it or not. Dr. Peter A. Kouides, a hematologist at Rochester General Hospital, said one of his Gaucher patients declined Cerezyme treatment for years. Genzyme’s representatives “were always bugging us about why we weren’t treating this guy.” Although most drug makers do not know who takes their medications, Genzyme knows just about everyone with Gaucher’s. Critics feel Genzyme disproportionately influences treatment and encourages high drug doses.
Many Gaucher disease patients have to come to Genzyme at some point for help in negotiating insurance coverage or arranging other financial assistance and Genzyme has a staff of about 50 on hand to help with these matters. Genzyme has also helped set up Gaucher treatment centers nationwide, many at teaching hospitals with specialists with far more experience than nonspecialists, who might see only one case of Gaucher’s in a lifetime.
Critics are concerned about the doctors who run these centers and argue that they have become indebted to Genzyme. “Genzyme wisely created their own opinion leaders,” said Dr. Ari Zimran, an Israeli expert on Gaucher disease who has been a consultant to some emerging competitors of Genzyme. The company has “monopolized the disease itself, not just the medicine,” Dr. Zimran said.
Three doctors who run treatment centers claim Genzyme’s financial support did not influence their medical decisions. “If I decide not to treat someone, that’s too bad for Genzyme,” said Dr. Barry E. Rosenbloom, who runs the Gaucher center in Los Angeles. Dr. Rosenbloom said his center receives $150,000 from Genzyme annually to pay for one full- and one part-time person, in part to contribute data to a patient registry.
Dr. David P. Meeker, president of the Genzyme division that sells Cerezyme, said it was simply looking to support patients and physicians. “We are not here to play doctors,” he said. “We’re not out there to advocate that patients do anything other than what is in their best medical interest.” Meanwhile, Genzyme’s seven years of monopoly under the Orphan Drug Act—a federal law meant to encourage companies to develop medicines for rare diseases—expired in 2001, but Genzyme has patents on Cerezyme’s manufacturing method until 2019.