Drug companies spend millions of dollars developing and testing drugs before they’re ready for use by humans to treat medical conditions. Hence, the initial cost of these medications is high, but they generally drop in price as the drug company recoups the cost of development and testing.
However, some drugs, known as orphan drugs, are used to treat extremely rare diseases. Since so few people need these medications, there are fewer customers to share these enormous costs. These drugs are incredibly expensive, and remain so. Here are the most expensive drugs in the world, and what they treat.
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Developed by Alexion Pharmaceutidals, Soliris’ generic name is eculizumab. It costs $409,500 per year, and is used to treat two extremely rare medical conditions. Soliris was originally developed to treat paroxysmal nocturnal hemoglobinuria, a condition in which the body is missing the gene PIG-A, which causes the blood cells to break down far too quickly and leads to severe anemia and other medical issues. There are only about 8,000 documented cases of paroxysmal nocturnal hemoglobinuria in the United States. Recently, Soliris was approved for treating another rare condition, known as atypical hemolytic uremic syndrome, or aHUS, which is a kidney disease that can lead to death. There are about 300 cases of aHUS in the U.S.
About 2,000 people around the world, almost all males, suffer from an incurable condition known as mucopolysaccharidosis II (MPS II), or Hunter’s Syndrome. With this disease, the body fails to produce certain important enzymes, and the condition leads to brain deterioration, failing organs, a distorted appearance, and impaired physical abilities. The drug company Shire developed Elaprase (generic name idursulfase), which helps manage symptoms in these patients. The annual cost is about $375,000.
In the condition mucopolysaccharidosis VI (MPS VI), genetic abnormalities cause the body not to produce certain enzymes. Without these enzymes, the patient has severe swelling, often hearing and vision loss, and becomes disfigured. The drug Naglazyme (generic name galsulfase) can help treat this disorder for about $365,000 per year. The drug is produced by BioMarin Pharmaceutical Inc. Experts aren’t sure how many people suffer from MPS VI, but estimate this condition occurs in one out of every 250,000 to 600,000 births. Generic alternatives to Naglazyme are available.
Developed by Lev Pharmaceuticals, Cinryze (generic name c1 esterase inhibitor) was acquired by ViroPharma when they bought the company in 2008. This drug treats another genetic disorder, called hereditary angioedema, or HAE. This condition causes severe and recurrent episodes of swelling, especially in the face, limbs, abdomen, and air passageways. About 6,000 Americans suffer from HAE. At an annual cost of $350,000, Cinryze can help reduce the severity and frequency of HAE episodes.
ACTH (generic name adrenocorticotropic hormone) is a drug used to treat seizures in babies under the age of two. At a cost of $300,000 for two courses of treatment, the child usually has to be hospitalized in order to begin treatments. The drug encourages the child’s body to produce adrenal steroids (glucocorticoids), and therapy often results in quick and complete elimination of the seizures. Though the treatments are usually successful, doctors still don’t understand how it works. Though effective, side effects do exist, including skin problems, abnormal hairiness, irritability, Cushing’s syndrome, infections, and other serious conditions.
T-cell lymphoma is a non-Hodgkins lymphoma, or cancer of the blood. About 15 percent of all non-Hodgkins lymphoma cases in the U.S. are attributed to T-cell lymphoma. The cancer in the blood can spread throughout the body, including the lymph nodes, spleen, bone marrow, blood, and other organs. Folotyn (generic name pralatrexate) is usually given by injection at an average cost of $30,000 per month. The most dangerous side effect of Folotyn is kidney damage.
Pompe disease, a rare, often fatal genetic disease, renders the body unable to produce a necessary enzyme called acid alpha-glucosidase (or GAA). These enzymes help the body break down and use certain substances, such as sugars. Without the enzymes, these sugars accumulate all over the body, particularly in the heart and muscles. The condition can onset at most any time of life, from early childhood through senior adulthood. Myozyme is the most common treatment. A child’s dose of the drug costs about $100,000 per year, and an adult’s dose runs about $300,000 per year. Around the world, there are only about 900 cases of Pompe disease.
Unlike many of the drugs on this list, Arcalyst is used to treat an entire family of disorders. Cryopyrin-associated autoinflammatory syndrome (or CAPS) are three separate diseases, all related to a single missing gene. Each disease affects different organs of the body. Neonatal Onset Multisystem Inflammatory Disease (NOMID) is the worst of the three, causing multiple organs to swell. It also causes pain, stunted growth, and sometimes loss of hearing and/or vision.
Another of these disorders is Muckle-Wells syndrome, which causes fever, rash, red eyes, joint pain and severe headaches and usually results in severe hearing loss by the patient’s teenage years. You can get medicine to treat a few of these symptoms from canadianpharmacymeds.com. The third form, Familial cold autoinflammatory syndrome, causes a rash, fever, chills, nausea, severe thirst, headaches and joint pain. It is the mildest form with the least long-term damage. Arcalyst (generic name rilonacept) acts by blocking the proteins that cause the worst of the symptoms of these disorders. Though it isn’t a cure, it can help lessen the symptoms of these painful and damaging disorders. Arcalyst costs about $250,000 per year.
Though these drugs are terribly expensive, patients and families who need to treat these terrifying conditions are simply glad that there is medication available at all. Though few of them are a total cure, these drugs can lessen pain and other symptoms, giving the patient a far better quality of life than they would experience without these medications. Getting these orphan drugs approved for use in other medical conditions expands the patient base and gradually helps bring down the costs of these incredibly pricey medications.
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