5 Most Expensive Medicines in the World

Pharmaceutical expenditures in 2014 exceeded USD 1 trillion for the first time, which was equal to 1.3% of global GDP and higher than the cumulative annual GDP of 15 countries. It is estimated that global pharmaceutical expenditures will continue to increase. IMS health project expenditures are expected to increase from USD 1 trillion in 2014 to USD 1.3 trillion in 2018. The world’s five most expensive medicines are as follows:

Why are prescription drug prices soaring?

The cost of R&D and marketing of some new drugs is considered to be one of the reasons why drug prices are soaring. For example, it is known that during the R&D of new drugs, drug developers need to pay certain R&D expenses, but consumers often forget that drug developers also want to recover the dozens, hundreds, and even thousands of times of research losses. Pharmaceutical companies are also vigorously promoting the development of drugs for rare diseases and designing personalized drugs for specific genes or proteins, which means much to pharmaceutical product developers and gives insurance companies few reasons to reject conducting underwriting. This is the reason why prescription drug prices have been rising continuously.

The world’s most expensive drugs in 2015–2016

The world’s five most expensive drugs in 2015–2016 are not Orkambi, or Harvoni of Gilead for treating HCV. In fact, a small number of drugs priced between USD 0.30 million and USD 0.36 million are not listed. However, two points shall be noted first.

Firstly, pharmaceutical drug developers may change pharmaceutical drug prices quarterly, meaning that the drug price ranking may change every three months.

Secondly, the treatment cost depends on the living environment: Harvoni is wholesaled at USD 1125/tablet in the U.S., but the price of the same drug may be as low as USD 10/tablet in overseas emerging markets. In other words, drug prices are slightly subjective, which shall be noted and before buying do not forget to check the summary of drugs

The world’s five most expensive drugs are as follows:

1. Glybera: Wholesale cost of USD 1.21 million per year

We believed that there would be the first drug priced at seven digits someday in the world. And now there has been a drug with such a price: Glybera, based on the EU’s approval of UniQure. Glybera is used to treat a kind of rare disease suffered by one in a million people, therefore Glybera will only have a market of 150-200 persons in the EU.

As a kind of gene therapy, Glybera copies and passes on one functional LPL gene to the skeletal muscle by using a kind of adeno-associated virus (AAV), and is used to treat a kind of extremely rare, inherited and metabolic disease–lipoprotein lipase deficiency (LPLD). LPLD is a rare disease with a morbidity of less than one or two millionths. LPLD patients are unable to process the fat particles in the blood and have the risk of acute and potentially life-threatening inflammation of the pancreas. In the clinical trial, Glybera could significantly reduce the morbidity of acute inflammation of the pancreas of LPLD patients after they received the first Glybera treatment.

2. Soliris: Wholesale cost of USD 0.70 million per year

Soliris researched and developed by Alexion Pharmaceuticals, Inc. has been the world’s most expensive drug over the years, therefore people would be surprised if it were not in the list of the world’s most expensive drugs. The consumption cost of Soliris in the U.S. last year approached USD 0.537 million, and the Patented Medicine Prices Review Board pointed out that the annual cost of Soliris in Canada last year could reach USD 0.70 million per person.

There are two indications of Soliris: paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), which are both ultra-rare diseases. The reason why Alexion can price Soliris so high is that there is no external competition, and more importantly, PNH patients can survive 10-15 years after being diagnosed, therefore, Soliris can become a long-term source of income for Alexion even though the number of patients is small. After having obtained an income of USD 2.2 billion in 2014, Alexion expected to double its income in 2018.

3. Naglazyme: Wholesale cost of USD 485,747 per year

According to the data of FiercePharma, Naglazyme researched and developed by BioMarin Pharmaceutical Inc. last year, whose annual wholesale cost approached USD 0.50 million, ranked third. The price of Naglazyme has been updated in 2015. The price of this drug may be determined based on the situation of dozens of patients every year, according to Evaluate Pharma. Naglazyme is used to treat mucopolysaccharidosis VI (MPS VI, a kind of rare inherited disease). MPS VI is a kind of severe life-threatening inherited disease. The patients congenitally lack a kind of enzyme, which affects the formation of carbohydrates in the body, thus leading to progressive cell, tissue, and organ system dysfunction.

4. Vimizim: Wholesale cost of USD 0.38 million per year

BioMarin Pharmaceutical Inc., an expert in rare diseases, had brought us the world’s most expensive drug Vimizim. As a kind of enzyme replacement therapy, Vimizim was the first drug approved by the FDA for treating Mucopolysaccharidosis IVA (Morquio A syndrome, MPS IVA). MPS IV (Morquio syndrome) has two subtypes the cause of IVA is galactose-6-sulfatase (GALNS) deficiency and the cause of IVB is a β-D-galactosidase deficiency.

The disease belongs to autosomal recessive inheritance and has the clinical characteristics of significant growth retardation, abnormal gait, and skeletal deformity which gradually becomes significant, with the longevity of most patients of 20–30 years old. There are currently about 800 patients with MorquioA syndrome in the U.S., and Vimizim is considered to have a market of about 3000 persons in developed countries. As predicted by an analyst, Vimizim can bring nearly USD 0.50 million in sales to BioMarin every year, despite of its relatively small market potential.

5. Elaprase: Wholesale cost of USD 0.375 million per year (based on an update in 2010)

Finally, the drug ranking fifth in the world’s most expensive drugs is Elaprase of Shire Plc, which is a kind of enzyme replacement therapy to treat Mucopolysaccharidosis II (MPS II) or Hunter syndrome. You might note that the final price of Elaprase is based on 2010, but there is no reason to believe that its price will fall thereafter because the Shire has no other competitor in the field of treating Hunter syndrome.

Patients with Hunter syndrome lack a kind of very important enzyme (2-sulfatase) that can help remove long chains of sugar molecules. If such molecules are not removed from the patient’s body, it will lead to gradual organ failure, especially for the heart, lungs, liver, and spleen. The morbidity of this disease is relatively low: the EU reported that one in 0.14 million-0.156 million newborns would suffer from this disease. Based on the above, the annual consumption higher than 6 digits will soon become the price standard of rare disease drugs.