In 2015, Glybera outpaced Soliris to become the most expensive drug in the world. The cost for this medication is over $1.2 million a year. Not approved in the United States, it is used in Europe to treat familial lipoprotein lipase deficiency, a condition affecting just 1,200 people in Europe and one million worldwide.
Its maker, UniQure, said in 2017 that it would not be renewing the drug's marketing authorization in Europe. Despite its high price tag, Glybera never caught on due to extremely limited patient demand.
Soliris can cost patients up to $700,000 a year, an effective drug used to treat paroxysmal nocturnal hemoglobinuria. Only 8,000 people in the world have the condition. The disease destroys red blood cells, causing patients to have infections, anemia, and blood clots.
In September 2017, Canadian authorities ordered manufacturer Alexion Pharmaceuticals to lower the price of Soliris, calling it excessive.
Elaprase is used to treat Hunter Syndrome, an extraordinarily rare condition affecting just 500 people in the United States that inhibits brain function and physical development. The annual cost for the drug is over $500,000.
Naglazyme is used to treat Maroteaux-Lamy Syndrome, a condition that affects connective tissues. Common symptoms include dwarfism, inhibited development, heart issues, and brain damage. The drug costs over $365,000.
Cinryze treats a condition called angioedema, a disease affecting one in 50,000 people in the United States. At a cost of $350,000 a year, the drug manages swelling of the hands, throat, and stomach.
Folotyn is a therapy for a rare form of cancer called T-cell lymphoma. It is considered a last resort treatment that lasts for six weeks. At a cost of over $320,000, it has not been shown to extend life for very long, so it is usually used in only extreme circumstances.
Acthar is prescribed to treat infantile spasms or seizures. This condition typically affects children between the ages of four and 11 months old, but Acthar is approved to treat children up to two years old. It's also used to treat a variety of conditions in adults, including multiple sclerosis. It costs over $200,000 a year.
A 2017 study in the Journal of the American Medical Associaton questioned the effectiveness of Acthar, suggesting it was no more effective than other less expensive treatments available.
Myozyme is a treatment for Pompe, a rare and sometimes fatal disease that attacks the heart and skeletal muscles. When it affects infants, mortality often strikes in the first year. Myozyme is a lysosomal glycogen-specific enzyme that in studies increased survivability and reduced patients reliance on ventilators. Its annual cost is between $100,000 and $300,000 a year. It's not currently available in the U.S.
The Most Expensive Prescription Drugs in the World
The business of developing and selling medications is a huge industry, with companies bringing in billions in revenue every year. Globally, sales of prescription drugs are expected to surpass $1.3 trillion in 2018. For some people, particularly those with rare diseases, the cost of these life-medications can be dear. Orphan drugs, medications designed to treat diseases experienced by only a few people, can easily cost well into six figures.
Some insurance companies will not cover all or a significant portion of these drugs, leaving families and affected individuals to handle the costs themselves. This is a significant financial burden for many and has caused a great deal of scrutiny towards the industry.
Some measures have been put in place to offset costs, such as non-profit organizations and prescription assistance programs. While these can be helpful for some, a large percentage of people do not qualify and have to pay out of pocket for these life-saving medications.