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Executive Summary

PMA COMMISSION LOOKING AT WAYS TO SUPPORT ORPHAN PRODUCT RESEARCH EARLIER in the development process, the assn.'s Commission on Rare Diseases Chairman Theodore Cooper told Rep. Waxman's (D-Calif.) House Health Subcmte. at a March 23 hearing. "There is an apparent need for greater support of basic and preclinical research in the field of rare diseases," Cooper said. He added that the PMA group "has undertaken a study of its scope and procedures" which could lead to an expansion of its charter. Cooper said that the commission "has received a number of inquiries and proposals for such support, but most are not within the scope of the commission's charter." He added that "the current provisions and incentives of the Orphan Drug Act do not provide support for this type of research. The subcmte. may wish to consider addressing this need." The Generic Pharmaceutical Industry Assn.'s Orphan Drugs Institute President William Haddad also suggested increased Congressional involvement in establishing procedures for supporting early research. Haddad said that "our greatest long-range challenge is to reach the academic researcher and interest him in the development of new drugs for rare diseases. That will happen only when there is a continuity of funding." To fill the need, Haddad suggested, a natl. institute for testing orphan drugs should be established. Such an organization "will encourage academic research because most of the extraordinary expenses come from testing." Establishment of an institute, Haddad observed, "is something Congress can do."

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