Page:United States Statutes at Large Volume 116 Part 3.djvu/400

 116 STAT. 1992 PUBLIC LAW 107-281—NOV. 6, 2002 Public Law 107-281 107th Congress An Act Nov. 6, 2002 To amend the Federal Food, Drug, and Cosmetic Act with respect to the development [H R 4014] ^^ products for rare diseases. Be it enacted by the Senate and House of Representatives of Rare Diseases the United States of America in Congress assembled, Orphan Product Development Act SECTION 1. SHORT TITLE. 2i^USC 301 note '^^^^ ^^^ "^^^ ^^ ^^^^^ ^^ ^^^ "Rare Diseases Orphan Product • Development Act of 2002". 21 USC 360ee SEC. 2. FINDINGS AND PURPOSES. (a) FINDINGS. — Congress makes the following findings: (1) Rare diseases and disorders are those which affect small patient populations, typically populations smaller than 200,000 individuals in the United States. Such diseases and conditions include Huntington's disease, amyotrophic lateral sclerosis (Lou Gehrig's disease), Tourette syndrome, Crohn's disease, cystic fibrosis, cystinosis, and Duchenne muscular dystrophy. (2) For many years, the 25,000,000 Americans suffering from the over 6,000 rare diseases and disorders were denied access to effective medicines because prescription drug manufacturers could rarely make a profit from marketing drugs for such small groups of patients. The prescription drug industry did not adequately fund research into such treatments. Despite the urgent health need for these medicines, they came to be known as "orphan drugs" because no companies would commercialize them. (3) During the 1970s, an organization called the National Organization for Rare Disorders (NORD) was founded to provide services and to lobby on behalf of patients with rare diseases and disorders. NORD was instrumental in pressing Congress for legislation to encourage the development of orphan drugs. (4) The Orphan Drug Act created financial incentives for the research and production of such orphan drugs. New Federal programs at the National Institutes of Health and the Food and Drug Administration encouraged clinical research and commercial product development for products that target rare diseases. An Orphan Products Board was established to promote the development of drugs and devices for rare diseases or disorders. (5) Before 1983, some 38 orphan drugs had been developed. Since the enactment of the Orphan Drug Act, more than 220 new orphan drugs have been approved and marketed in the

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