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Executive Summary

INVESTIGATIONAL ORPHAN DRUGS WITH MULTIPLE SPONSORS "racing to the finish line" of approval and marketing demonstrate "that more blockbuster drugs are in the pipeline," the National Organization for Rare Disorders maintained in a May 30 letter to Rep. Waxman (D-Calif.). "The few abuses that have occurred to date," NORD asserted, "foreshadow a long line of more severe abuses that will ...inevitably occur during the next two or three years." Acknowledging that the association in the past has argued to "forestall any substantive changes" to the Orphan Drug Act, NORD explained its change of heart. This year "we have come to realize that the act must be changed" to prevent further abuse in the future. "We genuinely fear that these abuses may become so extreme as to force Congress in the future to put so many strictures in the act that it would, in effect, become inoperative." Waxman has introduced legislation (HR 4638) to amend the act by providing simultaneous developers of an orphan product to share market rights granted to the manufacturer of the product approved first ("The Pink Sheet" April 30, p. 10). He is expected to schedule a markup of the bill before his Health Subcommittee during June. NORD and Waxman are apparently concerned that a budget-conscious Congress will object to Medicare's spending more than$200 mil. in 1990 on Amgen's Epogen (erythropoietin). The association's Executive Director Abbey Meyers, who co-signed the letter with NORD President Jess Thoene, MD, was on Capitol Hill to lobby for the Waxman amendments. The association distinguishes HR 4638 from orphan law amendments previously proposed by Waxman and opposed by NORD. The 1987 proposal broadly provided shared exclusivity for subsequent developers of orphan products on the grounds that marketing interest by multiple companies constituted a per se indication that the product was not a true orphan. However, the current bill restricts products that may compete against an original orphan to those shown to have been developed simultaneously throughout the R&D process. Contrary to arguments that the risk of lost exclusivity will discourage R&D into orphan products, NORD says it believes Waxman's legislation will enhance current incentives. Under the proposed amendments, the association argues, small biotechnology companies need not fear being locked out of a market and thereby losing its R&D investment because a large pharmaceutical company with far greater resources managed to win the race to approval by a day.

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