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Executive Summary

Changes in the Orphan Drug Act to allow approval of Fisons' aerosol pentamidine, Pneumopent, is supported by five senators on the Appropriations/Labor and HHS Subcommittee in a May 3 letter to HHS Secretary Sullivan. The five senators -- Bumpers (D-Ark.), Inouye (D-Hawaii), Burdick (D-N.D.), Adams (D-Wash.), and Hatfield (R-Ore.) -- asked Sullivan "whether the Administration will support an amendment to the Orphan Drug Act to permit the marketing of the Sloan-Kettering/Fisons regimen of pentamidine." If the White House does not plan to endorse a change in the Orphan Drug Act, the senators requested that Sullivan explain the Administration's "rationale for declining to extend support" to their proposal "as soon as possible." The letter was sparked in part by the development of Rep. Waxman's (D-Calif.) bill to amend the Orphan Drug Act. Waxman's bill would effectively allow FDA to approve Fisons' product. The Administration has been opposed to changing the marketing exclusivity of the Orphan Drug Act. As members of the HHS appropriations subcommittee, the senators have been involved in finding supplemental funding to help AIDS patients defray the cost of treatments. Fisons' product is potentially a more cost-effective treatment for Pneumocystis carinii pneumonia. An identical Senate counterpart to the Waxman bill has been introduced by Sen. Metzenbaum (D-Ohio). The senators explained that their interest in Pneumopent was due to the lower cost of the regimen vis-a-vis Lyphomed's product, Nebupent. An NDA for Pneumopent has been blocked since last spring by FDA's approval of Lyphomed's product as an orphan drug, which bestowed seven years of marketing exclusivity to Nebupent under the marketing incentives of the Orphan Drug Act. "In the case of aerosol pentamidine," the senators wrote, "there is evidence that a nebulizing regimen developed at Memorial Sloan-Kettering Cancer Center and tested by the Fisons Corp. offers tangible advantages over the product currently on the market in terms of enhanced patient convenience and cost-effectiveness." The senators said it had been reported to them that the Fisons Pneumopent regimen, "because of the efficiency of the device it employs, can result in savings of 50% or more on drug costs alone." The senators also suggested that the nebulizer used in the Fisons' regimen, which is portable, "could achieve additional savings for the Medicare and Medicaid programs by avoiding the charges for administration in hospitals, clinics, or physicians offices or through home health agencies, which are associated with the more cumbersome existing regimen." While declaring their support for the Orphan Drug Act, the five senators noted that the act "does not seem to address circumstances in which different devices may be used in combination with the same drug...[or] deal with situations in which more than one company devotes substantial resources to development of an orphan drug product only to find the investment wasted because of the vagaries of the approval process." In addition, the letter suggests that Memorial Sloan-Kettering began development of the Fisons product before Lyphomed. Sen. Pryor (D-Ark.) floated a proposal to change the Orphan Drug Act late last year that was specifically directed at eliminating the obstacles to Fisons' marketing of its pentamidine product. That suggestion never materialized in the form of legislation, possibly due to the opposition expressed by the National Organization for Rare Disorders to the Pryor proposal.

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