WASHINGTON _ The Orphan Drug Act Amendments of 1994(ODAA) _ compromise legislation hammered out in six years ofnegotiations between politicians, the biotechnology industry andpatient advocacy groups _ was voted out of the Senate Labor andHuman Resources committee on Wednesday without amendments. Thebill (S 1981) now proceeds to the full Senate.A companion bill in the House (HR 4160) has been referred to theEnergy and Commerce Committee's subcommittee on Health and theEnvironment and has yet to be voted on.Biotechnology Industry Organization (BIO) President Carl Feldbaumhas predicted that both bills will move quickly through the legislativeprocess.Sen. Judd Gregg (R-N.H.) offered several amendments to the ODAA,but his proposals were defeated. Gregg said he thought the bill wenttoo far in scaling back incentives for companies to develop orphandrugs. He charged that the bill won the support of BIO only becausebiotechnology companies were "scared to death" of health care reformand wanted to save all their resources to fight that battle.But his colleagues argued that the bill should remain unchanged sinceit is the culmination of years of delicate negotiations and Sen. EdwardKennedy (D-Mass.) repeatedly praised BIO's cooperative attitude incrafting a compromise.The ODAA legislation contains several significant changes to theoriginal Orphan Drug Act enacted in 1983:

A guarantee of four years of exclusive marketing rights for an orphandrug, down from seven;

A three-year extension of exclusive marketing rights if the FDAdetermines that an orphan drug has "limited commercial potential" (thecommercial potential of a drug would be determined by the Secretaryof Health and Human Services using criteria such as sales informationand other 'relevant' data);

A loss of exclusivity if the orphan drug treats a patient populationwhich exceeds 200,000 people;

A grant of exclusivity to two or more companies if they develop adrug simultaneously.The simultaneous development provision stipulates that once the firstcompany has been granted orphan drug status by the FDA, othercompanies developing a similar drug may qualify for shared marketexclusivity if the following conditions are met: 1) an application fororphan drug status is received by the Department of Health and HumanServices (DHHS) within six months after publication of the firstcompany's designation; 2) human clinical trials are initiated within 12months of the first company's initiation of human clinical trials; and 3)a product license application is submitted less than one year after thefirst company's PLA.Other features of the new legislation include a plan to replace theexisting DHHS's Orphan Products Board with an Office for OrphanDiseases and Conditions. The new office would be advised by an 11-member advisory committee which would include five representativesfrom organizations of persons with rare diseases, three researchscientists and three representatives of health-related companies.

-- Lisa Piercey Washington Editor

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