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Celebrating the Successes of the Orphan Drug Act

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Twenty-five years ago, former President Ronald Reagan signed into law the Orphan Drug Act (ODA) of 1983. No one could have imagined then that it would become one of the most important pieces of health care legislation today.  

ODA means hope for more than 25 million Americans who currently have one of 7,000 rare diseases or conditions.  More than 300 treatments have been approved by FDA in the 25 years since this groundbreaking legislation went into effect.  In the decade before the Act was passed, only 10 treatments had been developed for rare diseases.  

What Is an Orphan Drug?

The term "orphan drug" refers to a drug or biologic, such as a vaccine or blood product, that treats a rare disease or condition. A disease is rare if fewer than 200,000 people in the United States have it.

Orphan drugs may treat a new indication using an already approved drug or they may be completely new products. A drug or biologic becomes an orphan drug when the person studying it applies for orphan-drug status, that status is granted, and the drug is then approved by FDA.

But like every other drug, the orphan drug must go through the normal FDA marketing approval process and be evaluated for safety and effectiveness for its intended use. To date, more than 1,700 drugs and biologics have been designated as orphan drugs.

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How ODA Has Helped

Because discovering and developing a new drug can be expensive, and because rare diseases affect a relatively small number of people, there is generally little interest by drug companies to perform research or develop new products to treat those diseases. Companies also have faced further difficulties in recruiting a sufficient number of people for clinical studies to test potential treatments.

The law behind ODA offers a drug developer certain financial benefits and incentives from the government in exchange for researching, developing, and getting a drug approved for a rare disease or condition:

These benefits will help manufacturers recover the costs of developing a drug for small numbers of people. Since being signed in 1983, the Act has been amended by Congress several times—1984, 1985, 1988, 2007—to provide further incentives for treatment development.

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How FDA Has Helped

In 1982, FDA established the Office of Orphan Products Development (OOPD) to identify orphan products and to promote the development of those that demonstrate promise for diagnosing or treating rare diseases.

Serving as Director of that office from 1987 to 2006, Marlene Haffner, M.D., MPH, says, “The ODA happened at just the right time.  Science and biotechnology were in the right place at the right time to make breakthroughs, and the Orphan Drug Act has certainly grown the economy."

Currently directed by Timothy Coté, M.D., MPH, OOPD works to encourage the development of safe and effective treatments for rare diseases with 

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Humanitarian Use Devices

Medical devices are not eligible for orphan designation. But FDA has made it easier and less costly for manufacturers to bring to the market medical devices for rare diseases and conditions.

The Humanitarian Device Exemption (HDE) provisions of the Safe Medical Devices Act of 1990 allow a medical device to be approved as long as manufacturers show it is safe and has probable benefit to patients with an extremely rare condition—one that affects fewer than 4,000 Americans each year. These devices require only evidence that their probable health benefit is greater than the risk of use—a standard that is less costly to achieve than the level of safety and effectiveness required for regular devices.

To date, OOPD has designated 123 humanitarian use devices, and 43 of those were approved as HDEs. Two such devices include a stent to treat urinary tract obstruction in unborn babies, and a titanium rib for children born unable to survive without an adequate ribcage. Neither of these devices would be available outside this program.

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Toward a More Promising Future

ODA promises to identify and address even more challenges that might ultimately translate into hope for people with rare diseases or conditions.  Coté says that means continuing and expanding one of the federal government's most tangibly successful grants programs and increasing the number of promising compounds receiving orphan status designation for rare diseases.

"Looking forward to the next 25 years," he says, "we can envision fuller realization of the true spirit of the Orphan Drug Act."  

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For More Information

FDA Office of Orphan Products Development (OOPD)

NIH Office of Rare Diseases (ORD)

The Genetic and Rare Diseases Information Center

National Organization for Rare Diseases (NORD)

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Date Posted: February 8, 2008

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