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ORPHAN DISEASE COMMISSION REPORT: IMPLEMENTATION COSTS OF $ 45.6 MIL

Executive Summary

ORPHAN DISEASE COMMISSION REPORT: IMPLEMENTATION COSTS OF $ 45.6 MIL. are estimated for the first year alone. The report, which offers a total of 54 recommendations, states: "The cost of implementing all recommendations is estimated at $ 45.6 million for the first year. This amount does not include recommendations is estimated at $ 45.6 million for the first year. This amount does not include recommended increases for funding for research on rare diseases and biomedical research in general." The report is expected to be submitted to Congress during the week ending April 8. After two years of preparation, the congressionally-appointed National Commission on Orphan Diseases is expected to announce its recommendations at an April 11 press conference. Reportedly, Sens. Kennedy (D-Mass.), Hatch (R-Utah), Kassebaum (R-Kans.) and Rep. Waxman (D-Calif.) will be present at the briefing. The reports's final version contains no substantive changes from a draft approved by the Commission in January ("The Pink Sheet" Jan. 30, p. 12). Recommendations include: across-the-board funding increases by Congress and the private sector for basic and clinical research of rare diseases (including $ 40 mil. over four years for Clinical Research Centers); additional resources for FDA review and extramural activities; and increased coverage for rare diseases by insurers. Some of the more substantive changes recommended are in the area of orphan product approvals. For example, the commission advises increasing the period of orphan exclusivity from the current seven years, classifying all orphan products for 1A "fast-track" approval and extending the patent on a company's non-orphan product if they receive approval for an orphan drug. The report further suggests that Congress provide "by statute and appropriate funds for a Central Office of Orphan and Rare Diseases in the Office of the Assistant Secretary for Health." Among other things, the office would foster the implementation of the commission's recommendations, respond to proposals for legislation and regulations affecting rare disease patients, replace the current Orphan Products Board and report to Congress on federal activities related to rare diseases.
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