Did you know that Americans spend more money on healthcare than any other country in the world (both as a country and as individuals) but see fewer and diminishing healthcare returns? This includes a falling life expectancy. Most of the money spent in America on healthcare actually goes to health insurance companies, not the healthcare itself. Due to deregulation in the industry, much of this cost goes to protecting the patents on existing pharmaceuticals.

Depending on your insurance status, or which insurance you have if you live in the United States can cause you to spend hundreds to thousands on prescription medications. More common medications or purchasing a generic brand can reduce those costs for United States patients, but drugs that treat rarer conditions can often add up quickly. Here is a list of the most expensive pharmaceuticals in the world.

Why Does Medication Cost So Much?

There are a few different factors that can contribute to the cost of medications. Typically, the more expensive or rare the drug, the more pharmaceutical manufacturers feel justified in charging. At the most basic level, medication costs money to produce. Research, manufacturing, development, production, marketing, distribution, and patent costs are the first factors to consider for pharmaceutical companies. For medications that treat rare diseases or illnesses, these basic costs can be much higher due to the time it takes to research and produce the drug.

Another factor that can affect prescription drug pricing is government regulations or the lack of regulations. Some countries, like Japan, are very regulated in terms of how much healthcare and medications are legally allowed to cost. This creates a cost-containment system that prevents manufacturers from price-gouging patients.

In countries like the United States, very few or no-cost containment regulations exist, especially for rare drugs. There is virtually no counterbalance that can keep price gouging in check for patients. In recent years, in 2024, pharmaceutical companies saw more than $7.3 billion in profit. That means that after accounting for the other costs, they still made $7.3 billion.

Zolgensma

• Price: $2,125,000
• Used For: Treatment of SMA Type 1
• Prevalence of SMA Type 1: 1–2 per 100,000 people

Zolgensma is a one-time dose medication used to cure children under the age of 2 with spinal muscular atrophy. Patients diagnosed through genetic testing before symptom onset report a 100% achievement of all assessed milestones.  Zolgensma is said to be a cure for SMA if administered before symptom onset. If administered after symptoms occur, there is up to 7.5 years of sustained durability.

Zokinvy

• Price: $1,032,480/ Year ($1,414 per dose)
• Used For: Hutchinson-Gilford Progeria Syndrome (HGPS)
• Number of Patients: 1 in 4 million newborns

Zokinvy is used twice per day for one year to delay the symptoms of HGPS. HGPS causes young people to age extremely quickly, which leads to heart disease before the age of 15. Zokinvy delays the quick-aging process for several years.

Danyelza (naxitamab)

• Price: $977,664
• Treats: Neuroblastoma
• Number of Patients: 1 in 10 million people

Danyelza is a drug that causes a complete response or gets rid of the brain tumor 34%–45% of the time. This drug is an anti-GD2 monoclonal antibody that treats an extremely rare brain cancer that affects children the most. It is administered through IV a few times per week for every week of the treatment cycle (4 or 8 weeks). 

Luxturna

• Price: $850,000
• Treats: Retinal Dystrophy
• Number of Patients: 1 in every 3,000 persons

Luxturna is an intraocular injection (injection into the eye), used to treat genetic disorders which result in vision loss. This is a form of gene therapy that treats mutations in gene RPE65. It is administered by a highly specialized eye surgeon.

Ravicti

• Price: $794,000 Per Year ($5,016 Per Vial)
• Treats: Urea Cycle Disorders (UCDs)
• Number of reported cases of UCDs: 1 in 35,000 newborns

Ravicti is a treatment that controls ammonia. It is administered orally and is touted to be nearly odorless and tasteless. UCD is a disorder that causes high blood levels of ammonia. Along with this long-term management medication, the patient must also be on a low-protein diet.

Myalept (metreleptin)

• Price: $889,904-$1M
• Treats: Congenital Leptin Deficiency
• Number of reported cases of CLD: A Few Dozen Cases Ever Reported

This once daily, subcutaneous injection, is only available through a small number of specialized clinics nationally. This isn’t a cure for Congenital Leptin Deficiency, but it is used to improve the quality of life of patients with leptin deficiency. Leptin is a protein hormone that helps the body feel satiated. Leptin deficiency is a rare genetic condition that causes insatiable hunger, high or low percentage of body fat, low energy, infertility, absent or late puberty, and absence of libido.

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