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Executive Summary

SEN. HATCH WILL ENCOURAGE ADOPTION OF $ 8 MIL. INCREASE FOR ORPHAN grant funding program of FDA's Office of Orphan Products Development. Hatch (R-Utah) told an April 12 press conference held by the National Commission on Orphan Diseases that "one specific recommendation, which I will ask the Appropriations Committee to adopt immediately, is to fully fund the grants program of [the] Office of Orphan Products Development. This would mean an increase from $ 6 mil. to $ 14 mil." The press conference was held to announce the availability of the commission's report to Congress. The report includes recommendations for facilitating research and development of orphan drugs for rare diseases ("The Pink Sheet" April 3, T&G-5). Draft copies of the report had been submitted to Congress and HHS. Asked whether he and Sen. Kennedy (D-Mass.) still plan to endorse legislation that would limit the profits companies can earn under the exclusivity provisions of the Orphan Drug Act, Hatch said: "We have to address this problem . . . We're basically today discussing how to derive a sensible and equitable solution." Rep. Waxman (D-Calif.), who also appeared at the briefing, added: "We tried to deal with that in a proposal last year, the year before. We haven't developed a consensus on it." Capitol Hill concern has centered on the possibility of companies using the act to obtain monopolies for products with significantly larger market potential than that intended by the Orphan Drug Act. Attention has been focused on human growth hormone, Lilly's Humatrope and Genentech's Protropin, Burroughs Wellcome's Retrovir (AZT) and Amgen's as-yet-unapproved erythropoietin (Epogen). Hatch and Kennedy sent a joint letter to FDA at the end of last year asking the agency to delay awarding exclusivity for any orphan products until the middle of 1989, in order to permit the senators time to consider amendments to the act. Congressional efforts to curb exclusivity runs counter to a recommendation in the commission report that suggests increasing the period of exclusive marketing from the current seven years ("The Pink Sheet" Jan. 30, p. 12). With the recent arrival of new biotech products, Hatch noted, "we have seen some amazing uses of the Orphan Drug Act and I don't blame them for using it this way." The senator added that "some of these companies are not protected by a current patent . . . and I think we've got to provide those protections and once the protections are provided, they won't be seeking to use the Orphan Drug Act to solve their problems." Hatch is working on legislation to provide up to 10 years of exclusivity for newly approved biotech drugs that would be unpatentable under current law because they are based on substances produced in the human body ("The Pink Sheet" Feb. 20, p. 3). Commission Chairman Jess Thoene, MD, discussed the report's recommendation for granting patent extension to a company's already marketed non-orphan product if that company receives approval for an orphan drug. "In essence we're saying, 'if you're going to devote substantial corporate resources to coming up with a new orphan drug . . . we're [going to give] you a movable wild card on a patented product,'" Thoene said. He emphasized, however, that patent extension would only be given to companies that have a systematic drug discovery and development program for non-AIDS and noncancer rare diseases.

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