Orphan Diseases & the FDA
An orphan disease is a disease that affects only a small number of people. When a disease does not affect a large number of people, there is less funding to find treatments for that condition. In 1983, Congress passed the Orphan Drug Act to address this concern. Drugs that treat an orphan disease are called orphan drugs. The FDA is responsible for implementing the Orphan Drug Act and the Office of Orphan Products Development handles the grant program.
What is an Orphan Disease?
According to "Orphan Products," an orphan disease is a rare disease or condition that affects less than 200,000 people in the United States. This includes conditions such as Lou Gehrig's disease, Tourette's and acromegaly, more commonly known as gigantism. Most, but not all, orphan diseases are caused by a genetic abnormality or defect. Due to the rarity of these diseases, it is often very difficult for patients to receive an accurate diagnosis.
The Orphan Drug Act
The Orphan Drug Act was passed in 1983. This act gave financial incentives to companies to research, develop and market orphan drugs for rare diseases. According to "The Orphan Drug Act: Implementation and Impact," pharmaceutical companies developing orphan drugs are eligible to receive grant money for clinical trials as well as up to fifty percent of the cost of human clinical trials. The company will also have exclusive marketing rights for seven years.
Effectiveness of the Orphan Drug Act
Since the implementation of the Orphan Drug Act, the number of orphan drugs approved by the FDA has increased substantially. According to "Orphan Products: Hope for People With Rare Diseases," in the ten years prior to the implementation of the Orphan Drug Act, only 10 new medications were approved by the FDA. Since 1983, over 250 new orphan drugs have been approved and are on the market for patients with rare diseases.
Grant Program
The FDA's Office of Orphan Products Development (OOPD) is responsible for reviewing all grant applications under the Orphan Drug Act. According to "FDA's Office of Orphan Products," approximately $14.2 million in grant money is available. The OOPD also gathers information on orphan drugs and new drugs still in the testing phase and distributes that to patients to help them plan their treatment and inform them of new clinical studies.
Downside of the Orphan Drug Act
The downside of the Orphan Drug Act is that it has created a monopoly for certain pharmaceutical companies. Because the Act gives the company exclusive marketing rights for seven years, there is no competition to regulate the pricing of the drugs. For example, according to "The Orphan Drug Act: Implementation and Impact," one orphan drug, Alglucerase, can cost up to $300,000 per year. Although in many cases the pricing is similar to non-orphan drugs, due to the exclusivity factor, patients do not have the option to purchase a generic version of the drug.
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