Nabil Alhakamy

The Most Expensive Medicines Globally!

الخميس - 15 يونيو 2023

Thu - 15 Jun 2023

Dear reader, it is no secret that the global pharmaceutical market is a very large market, reaching billions of dollars annually. In this article, we will review the three most expensive medicines in the world and rank them according to the cost of treatment for one year for each medicine. The pricing of medicines has always been a controversial issue among many parties.

To start our article today, let us first ask a question before we review the most expensive medicines globally, which is why some medicines are very expensive. The pricing of drugs depends on many factors, the most important of which are the cost of research, development, and innovation, which may cost billions of dollars and may take many years just to approve the drug by the relevant authorities. Another factor is the size of the targeted pharmaceutical market. For example, suppose the number of patients with a certain disease around the world is about 10,000 compared to another disease where the number of patients around the world exceeds 10 million patients. In that case, it will greatly affect the drug price. Other factors include whether there is competition from other alternative drugs for the same disease, and we may discuss many other factors in a separate article in the future.

If it is a specialized drug, a gene therapy, or a biological drug, it will be one of the most expensive drugs today. Gene therapies, for example, are unique in that they only need to be taken once for life-threatening diseases and can result in complete healing.

Now, let us review the three most expensive medicines in the world, starting with “Zolgensma” (onasemnogene abeparvovec-xioi)," which ranks third as the most expensive drug in the world with a total cost of approximately $2.125 million per patient. It is a life-saving gene therapy drug that was approved by the US Food and Drug Administration (FDA) in 2019 to treat Spinal Muscular Atrophy (SMA). This rare genetic motor neuron disease causes breathing and swallowing problems in children. Most children with SMA do not survive their early childhood due to the inability to breathe. This drug is given as a one-time intravenous (IV) dose, as it works by replacing or correcting the defective or missing gene for motor neuron 1 (SMN1) cells.

The second most expensive drug in the world is "Zynteglo" (betibeglogene autotemcel or beti-cel), with a total cost of approximately $2.8 million per patient. It was approved by the US FDA in 2022 as a gene therapy treatment for adult and pediatric patients with Beta-Thalassemia. This genetic blood disorder is one of the main types of Thalassemia. It is classified as anemia, where red blood cell counts or hemoglobin levels fall below normal levels in the body. Patients need regular red blood cell transfusions in blood transfusion procedures, and their life depends on it if left untreated. The process is long; patients usually undergo transfusions every two to five weeks.

The most expensive drug in the world is "Skysona" (elivaldogene autotemcel or eli-cel), with a total cost that exceeds $3 million per patient. It was approved by the US FDA in 2022 as the first treatment for slowing the progression of neurological degeneration in patients aged 4 to 17 who suffer from early active cerebral adrenoleukodystrophy (CALD), also known as "brain adrenal gland degeneration." CALD is a rare and deadly genetic disease that primarily affects young patients, and the death rate due to this disease reaches half of the patients who do not receive treatment within five years of the onset of symptoms. "Skysona" is produced from the patient's stem cells and modified to contain a copy of the functional ALDP protein gene. The treatment is given in a single dose only.

Finally, access to medication is crucial for the well-being of people, and governments play a vital role in ensuring that they are available to everyone. It's heartening to see that the Saudi government is committed to this cause and is working towards providing the latest drugs to its citizens and residents for free, regardless of the cost or price. Such efforts will go a long way in improving the quality of life of people and helping them lead healthier and happier lives.