The Orphan Drug Act of 1983

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As a pharmacy technician it is important to be familiar with laws and regulations that pertain to your industry. As part of our series of the laws and regulations that govern our industry, today we are going to look at the Orphan Drug Act of 1983.

Orphan Drug Act of 1983
The Orphan Drug Act was signed into law on January 4, 1983. The act provides incentives for pharmaceutical manufacturers to develop drugs, biotechnology products, and medical devices for the treatment of rare diseases and conditions affecting less than 200,000 people, termed orphan drugs.

Without this act, developing and marketing such a drug would be prohibitively expensive. The Orphan Drug Act of 1983 encourages the development of orphan drugs by providing tax incentives and granting manufacturers a time for exclusive licenses to market such drugs. Orphan products often receive expedited review and accelerated approval because they are for serious or life-threatening disease. Over 250 orphan drugs have been approved by the FDA for marketing.

There are many diseases and conditions, such as Huntington’s disease, myoclonus, ALS (Lou Gehrig’s disease), Tourette syndrome, and muscular dystrophy that affect such small numbers of individuals in the United States that they are considered rare; adequate drugs for many of such diseases and conditions have not been developed.

For example, Wilson disease is a rare progressive genetic disorder characterized by excess copper stored in various body tissues that can lead to organ dysfunction and premature death. The incidence is approximately 1 in 30,000. Early diagnosis and treatment may prevent serious long-term disability and life-threatening complications. Treatment is aimed at reducing the amount of copper that has accumulated in the body and maintaining normal copper levels thereafter. The research costs for developing treatments for such a rare disorder would be prohibitive to a pharmaceutical manufacturer without financial incentives, patent and legal protections, and accelerated approvals.

Sources:
http://www.law.umaryland.edu/marshall/crsreports/crsdocuments/RS20971.pdf
http://www.fda.gov/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/significantamendmentstothefdcact/orphandrugact/default.htm


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