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Executive Summary

ORPHAN DRUG ACT AT 10 YEARS: REP. WAXMAN PREPARED TO MOVE ON AMENDMENTS to the act during the 103rd Congress, the California Democrat has indicated. Speaking as a featured guest at a Feb. 23 banquet sponsored by the National Organization for Rare Disorders to celebrate the 10th anniversary of the enactment of the legislation, Waxman said "there are some kinks" in the act, and "we're going to work on it." Waxman authored the legislation. The NORD celebration, held in Washington, D.C., also honored Sen. Kassebaum (R-Kan.), the Senate sponsor. She has and is expected again to cosponsor with Sen. Metzenbaum (D-Ohio) legislation to place a $200 mil. sales threshold on the act's market exclusivity provision. Other honorees were actor Jack Klugman, of the television series "Quincy, M.E.," which in the early 1980s featured two episodes on orphan drugs and generated much public support for the legislation; Melvin Van Woert, MD, a researcher at New York's Mt. Sinai School of Medicine who serves on NORD's Medical Advisory Board; Bruce Fried, who is a senior VP and deputy general counsel of the Washington lobbying firm The Wexler Group and served as a health policy advisor to President Clinton's campaign and transition team; and NORD Executive Director Abbey Meyers, who successfully lobbied for passage of the Orphan Drug Act as the mother of a Tourette's Syndrome patient. NORD also honored Bristol-Myers Squibb "for its commitment to upholding the spirit and intent of the Orphan Drug Act by voluntarily relinquishing its orphan drug designations for" the AIDS drug Videx and the cancer agent Taxol. NORD noted that when the company dropped the designations, it had "learned more about the potential uses for the two drugs," and felt that retaining orphan status for them "was not in keeping with the purpose of the act." Speaking at a Feb. 22 meeting of the Orphan Products Board, FDA Office of Orphan Products Development Director Marlene Haffner reported that since passage of the Orphan Drug Act in 1983, 87 orphan drugs and biologics have been approved and used to treat over 2 mil. patients. Haffner said that 20% of the 87 approved orphan products are for pediatric indications, while 50% of the 508 orphan drugs that are still in research or under review by FDA are intended for pediatric indications. Other patient populations highlighted by Haffner were the elderly and those suffering from rare cancers. Since 1983, "there have been 58 designations [of orphan status] and eight approvals for rare cancers," Haffner said, and "there have been 71 designations for the treatment of the geriatric population." She added that "24 out of the 28 treatment INDs are for orphan indications." Fourteen orphan products were approved in calendar year 1992, the same number as in 1991. The average review time for the 14 orphan products approved last year was 17.6 months, versus 30.2 months for new molecular entities. "I'm not sure why there is that difference," she said, adding that "it could be that the number of patients in trials [of orphan products] are so much smaller that there are fewer data points to be collected and reviewed." Haffner said the agency feels "that the act has been very successful in carrying out its mission of making available to patients with rare diseases drugs to treat those diseases." Mission Pharmacal President Neill Walsdorf gave FDA much of the credit for the success of the Orphan Drug Act. Speaking at a Feb. 23 Food & Drug Law Institute seminar on the 10-year history of the act, Walsdorf said the "most important" aspect of the orphan drug regulations is "the appropriate attitude of the FDA personnel to help and assist these drugs through the approval process to get the products to the patients who need them." Four other firms who presented their experience with moving orphan drugs through the FDA approval process -- Burroughs Wellcome, Marion Merrell Dow, Enzon and Gynex -- also emphasized the importance of FDA's receptive attitude toward their products. The National Institutes of Health has estimated that Mission Pharmacal's "small group of drugs and diagnostics" to treat kidney stones has generated savings in hospital and surgery costs of more than $400 mil. annually, Walsdorf told the FDLI audience. The average hospital cost ranges from $3,000 to $15,000 for an occurrence of kidney stones, Walsdorf said, noting that the San Antonio, Tex.-based company's products are capable of "70% to 80% stone remission regardless of stone recurring stone formers."

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