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Executive Summary

Genzyme's Ceredase may be administered initially at lower doses without compromising efficacy, leading to a 75% savings over the current annual cost of treating Gaucher's disease patients, a study published in the Dec. 3 issue of the New England Journal of Medicine concludes. Conducted by Ernest Beutler, MD, et al., Scripps Research Institute, the study found that a Ceredase (alglucerase) dose of 30 mg/kg per month given in two-hour infusions of "2.3 U per kilogram three times weekly [may be] a reasonable compromise between high costs and the convenience of the patient." The estimated annual cost of treating a 70 kg person at the lower dose of 30 U/kg per month would be about $88,000 compared to the $382,000 price-tag of the current recommended dose of 60 U/kg every two weeks. The study compared improvements in 14 moderately severe to severe Gaucher's disease patients given fractionated doses of Ceredase with those described in the literature for 18 patients receiving a Ceredase dose of 50-60 U/kg every two weeks. Nine patients in the study received 30 U/kg a month given as two-hour infusions of 2.3 U/kg three times weekly. Two patients, who were physicians, treated themselves with daily two-hour infusions totaling 30 U/kg per month. The remaining three patients received 120 U/kg of the drug per month given as two-hour infusions of 9.2 U/kg three times weekly. All patients in the study had decreases in the size of their livers and spleens (if they had one) comparable to those seen in the patients treated with a 50-60 U/kg twice monthly Ceredase regimen. The article points out that even though the more frequent administration of Ceredase will lead to more ancillary fees, the total cost of treatment will still be less than that of high-dose intermittent administration. The authors estimated that an additional 130 infusions per year at $150 per infusion would add $20,000 to the cost of low- dose treatment. "Even this large expense is dwarfed, however, by the savings in the amount of the enzyme given to all but the smallest children," the authors maintained. If the dose of Ceredase can be reduced to a maintenance dose of 7.5 U/kg every two weeks after a patient has been stabilized for the first six months as suggested in a study by Barton et al. published in NEJM last December ("The Pink Sheet" Dec. 23, 1991, p. 16) the cost of the first year of drug therapy could be reduced to about $68,000. Genzyme maintains that reductions in dose after a certain initiation period could result in annual drug costs of between $20,000-$60,000. The Scripps dosing regimen was cited in a recent Office of Technology Assessment report on federal and private roles in drug development as a way of lowering the cost of the expensive orphan drug therapy. OTA estimates annual costs for the drug may range from $57,980 (based on the 30 U/kg dose three times weekly for a 50 kg person) to $546,000. OTA also projects that between one- third and one-half of Ceredase recipients risk exhausting or critically reducing their insurance benefits over time ("The Pink Sheet" Oct. 12, T&G-12). OTA continues to maintain that the true R&D costs for Ceredase are lower than Genzyme's estimates, even though the company's accounting practices are legitimate. At an NIH Director's advisory committee meeting on drug pricing, OTA Senior Associate Judith Wagner used Ceredase as an example of how information can be viewed differently by companies and government agencies. "When you come to sit down with [a] company and companies present information, it is so easy to be misled even with data that are true," she said. Wagner reported that when a group from OTA, accompanied by a Harvard University professor of accounting and operations, visited Genzyme, the company "treated us wonderfully and [shared] a great deal of proprietary data." However, while the company's R&D estimate included a $20 mil. payback to its R&D limited partnership, OTA considered that "double counting of R&D costs," Wagner said. "We are right [in our estimates] in a true resource [sense about] what it actually cost that company in terms of paying scientists, [etc.]," she asserted. OTA estimates of the cost of Ceredase development are around $29.4 mil., while Genzyme's figures total $48.6 mil. The company will be able to supply much larger amounts of Ceredase once its recombinant alglucerase product is approved. However, Genzyme predicts that approval for recombinant product will not occur until mid-1994, as the company plans at the earliest to file a PLA for the recombinant version sometime in the first half of 1993. OTA's Wagner suggested that the company's orphan exclusivity on the purified form is delaying development of the recombinant form of the drug.

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