Orphan Drugs: The United States Experience
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0093--8615/99 Copyright 0 1999 Dnig Information Association Inc.
ORPHAN DRUGS: THE UNITED STATES EXPERIENCE MARLENEE.
HAFFNER,
MD, MPH
Director. Office of Orphan Products Development, U.S. Food and Drug Administration, Rockville, Maryland
This paper provides an overview of o r p h m drug legislation in the United States. Since passage ofthe Orphan Drug Act in 1983, 182 designated orphan products have received Food and Drug Administration approval for rare disease treatment or diagnosis. Incentives provided in the act-seven years of marketing exclusivi@ protocol assistance, tax credits, the orphan products grant program which authorizes grant moneys to support pivotal clinical trials, and user fee waiver-are discussed. Issues, challenges, and successes are outlined. Important international initiatives in orphan drug legislation triggered bv the United States Orphan Drug Act are highlighted. Key Words: Orphan drugs; Rare diseases; Orphan Drug Act; Food and Drug Administra-
tion
THE STORY OF ORPHAN drug legislation in the United States serves as dramatic testimony to the power of patients with rare diseases, who-although individually few in number-were able to use their combined strength and ability to influence the law. The time had come, in America, to respond to their need. Voluntary organizations worked aggressively to promote legislation to encourage the development of products necessary and often life-saving for patients with rare diseases. Despite their potential usefulness, these drugs and biologics, known as orphan products, often remained inadequately tested andor unavailable to patients, due to the small number of patients with a particular disease.
Presented at the DIA 34th Annual Meeting ‘Thinking Globally: Product Development, Registration, and Marketing in the New Millennium” June 7-1 l , 1998, Boston, Massachusetts. Reprint address: Marlene E. Haffner, MD, MPH, Director. Office of Orphan Products Development, Food and Drug Aministration, 5600 Fishers Lane. Room 8-73, Rockville, MD 20857.
The Orphan Drug Act (ODA) was passed by the United States Congress with rare unanimity, and was signed into law by President Ronald Reagan on January 4, 1983. This legislation amended the United States Federal Food, Drug, and Cosmetic Act, and provided incentives for the pharmaceutical industry to develop drugs that otherwise have limited commercial value and consequently generate little industry support. Amendments to the ODA in 1984 attributed a population definition to the qualification for orphan designation. The now 15-year-old legislation provides official orphan designation from the Office of Orphan Products Development (0PD)the Food and Drug Administration (FDA) office which administers the act-for qualified products to treat or diagnose very rare diseases.
MARKETING EXCLUSIVITY Once a sponsor’s designated orphan product has received market approval from the FDA, the manufacturer is entitled to a period of marketing exclusivity that continues for
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