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Executive Summary

Rep. Waxman's bill to change the Orphan Drug Act is scheduled for markup by the California Democrat's House Energy and Commerce/Health Subcommittee on June 19 or 20. Subcommittee counsel William Schultz told a June 14 Food & Drug Law Institute meeting in Washington that the subcommittee has planned a markup for June 19. However, six bills are on the agenda for that day, and discussion of the orphan drug bill (HR 4638) may be delayed until June 20. Noting that the FDA grant program and the R&D tax incentives under the Orphan Drug Act must be reauthorized this year, Schultz predicted that both the House and Senate will pass legislation affecting the law. A companion bill to Waxman's legislation, introduced by Sen. Metzenbaum (D-Ohio), is pending in the Senate. metzenbaum's subcommittee staff director is Waxman's former chief aide for drug issues, William Corr. Schultz acknowledged that Waxman's proposal has a difficult row to hoe through the competing special interests involved in the orphan drug area. Observing that twelve former congressional staffers were counted at the subcommittee's hearing in February to present views of the legislation, Schultz speculated that the hearing might have set a Capitol Hill record. The White House Council on Competitiveness, through a working group, is recommending opposition to any changes in the law, according to a June 1 letter from Lawrence Lindsey, special assistant to the President for policy development, to the Pharmaceutical Manufacturers Association. PMA strongly opposes changes to the law, as does the Industrial Biotechnology Association. Individual companies including Upjohn and Enzon, as well as the Association of Biotechnology Companies and the National Organization for Rare Disorders, are among the proponents for change ("The Pink Sheet" May 14, p. 6). The competitiveness working group has agreed to four "principles": (1) "The Orphan Drug Act has been an effective mechanism in furthering research and development on drugs for rare diseases and conditions and should not be altered in any fundamental manner;" (2) "The Council on Competitiveness does not believe that current legislative proposals will prove successful at improving the act's administration or effectiveness;" (3) "The Council on Competitiveness is strongly opposed to any measure that would retroactively remove rights assigned under the act;" (4) "The Council on Competitiveness urges the [FDA], as additional information becomes available, to continue to refine its efforts with respect to future implementation of the act in a manner that limits its special provisions to the development of treatments for patient populations of fewer than 200,000 people." The group added: "The incentives created under the program are critically important to the development of drugs that in many cases would be unavailable. Any effort to make changes in the program that are retroactive both would be counter to fundamental notions of fairness and would undermine future efforts to implement the program. We basically believe the Orphan Drug Act is working well, and we oppose any legislative changes to the program at this time." Earlier speeches by Bush Administration officials have indicated that the Administration would be opposed to Orphan Drug Act amendments although no definitive statement has been made. For example, HHS Assistant Secretary for Health James Mason told a May 3 symposium in Washington, D.C. he did not believe the law needs to be amended. However, he added that if problems are brought to light during the debate on the law, "by all means let's fix what's wrong."

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