The Most Expensive Prescription Drugs in the U.S.

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The High Cost of Good Health

One reason behind soaring healthcare costs are expensive pharmaceuticals. Thousands of people take drugs that cost from $50,000 to $100,000 per year, such as the cancer drugs Provenge and Avastin and the medication for multiple sclerosis, Lemtrade.

But a much smaller number of people have rare conditions that require lifesaving drugs whose prices are astronomical—up to $400,000 per year, prices that may reflect the cost of developing the medication.

Most of these medications are covered by insurance if doctors deem the drugs are necessary. Some pharmaceutical companies also offer patient assistance programs for people who can't afford the medications or the copays.

Click through the gallery to see some medications that cost as much per year as a large house.

Photo: Adrianna Williams/Getty Images

Soliris

This drug is used to treat paroxysmal nocturnal hemoglobinuria, a rare disorder that causes the breakdown of red blood cells and release of hemoglobin into the urine. The sudden, recurring attacks—which are often triggered by stress, exertion or infection—result in anemia.

Little is known about the incidence of paroxysmal nocturnal hemoglobinuria, which can affect people of any age, but it's believed to occur at a rate of about one to five per one million. The drug Soliris, made by Alexion Pharmaceuticals and approved by the Food and Drug Administration in 2007, stops the breakdown of cells. Soliris is a monoclonal antibody—a product engineered in the lab to mimic a natural substance. Studies show it leads to a dramatic improvement of symptoms and reduces the risks of complications.

Elaprase

Elaprase treats an inherited condition called Hunter syndrome, a metabolic disorder in which a key enzyme that breaks down complex sugars is missing or malfunctions. The incurable condition occurs in about one in 150,000 people, almost always males.

The disorder typically appears in childhood and impairs growth and mental development. Afflicted children also have a distinct thickening of facial features. A late-onset version of Hunter syndrome is usually milder but can delay growth and damage joints, vision and hearing. Elaprase is an injected medication that replaces the missing enzyme. Manufactured by Shire, it was approved in 2006 improves patients' ability to walk.

Naglazyme

This medication is for a rare connective tissue disorder called Maroteaux-Lamy syndrome, which is caused by a deficient enzyme that breaks down large sugar molecules called glycosaminoglycans.

The condition occurs in about one of every 200,000 to 600,000 people. The deficiency causes growth retardation in early childhood in addition to heart disease. Naglazyme is made by Biomarin and was approved in 2005. It helps break down and remove glycosaminoglycans. Studies show Naglazyme improves patients' endurance, growth and mobility.

Cinryze

Cinryze is used to treat hereditary angioedema, a rare genetic condition that runs in some families and causes life-threatening and painful swelling of the hands, feet, face, genitals, abdomen and throat. The condition occurs because a gene that controls a blood protein called C1 inhibitor is defective. C1 inhibitor helps the immune system function and blood to clot. But in people with HAE, C1 inhibitor levels are abnormally low or undetectable.

The condition affects about one in 10,000 to 50,000 people. If one parent has the condition, that person's children have a 50 percent chance of having HAE, although studies show the condition can also appear spontaneously. There have been few options for treating HAE. Cinryze, manufactured by ViroPharma, is an intravenous medication made from human blood. It was approved in 2008 to prevent attacks and limit swelling.

Folotyn

This medication is used to treat an aggressive type of cancer of the lymph system, peripheral T-cell lymphoma, which has spread throughout the body. The lymph system is comprised of white blood cells and T cells which fight viruses. There are many types of lymphoma, but T-cell lymphoma is rare, affecting about one to two people per 100,000.

Folotyn, made by Allos Therapeutics, was approved in 2009 and helps prolong survival. It's given to people who have exhausted all other options for treatment and whose cancer has recurred. The drug, an injection, is thought to kill cancer cells; however studies so far have not shown that it prolongs survival. Still, Folotyn was approved as part of the FDA's accelerated drug approval process to address the needs of people who have a poor prognosis.

Myozyme

This drug was designed to treat people with Pompe disease, a rare disorder in which a missing enzyme called acid alpha-glucosidase leads to a build-up of glycogen (a type of sugar) in the blood and eventual deterioration of the heart and skeletal muscles.

Although Pompe disease can range in severity, it's typically diagnosed in infancy. It affects about one in 40,000 newborns. Without the medication, Myozyme—the first drug approved to treat Pompe—most children would die by age 1. The medication, made by Sanofi, was approved in 2006. It's an enzyme replacement therapy that promotes normal heart function and improves muscle tone.

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