Research and clinical trials can be very time-consuming, sometimes it can even last for decades before the drug reaches its patients, and many substances simply fail during this process. Adverse and side effects or economic reasons can put an end to the whole research process overnight. When the number of patients affected by a condition is relatively low, sometimes concerning only hundreds, the economic burden of research reflects on the treatment price- it reaches enormous numbers. This article is a list of the 10 most expensive treatments per patient in the world.
10. Spinraza (API Nursinersen)
Nusinersen (branded name Spinraza) treats a disease known as Spinal Muscular Atrophy (SMA). SMA occurs due to a mutation on a fifth chromosome (5q) characterized by progressive muscle atrophy that typically begins shortly after birth (although some forms of the disease have late-onset). The drug comes in 12mg vials as a solution for injection. It’s given directly into the spine by specially trained personnel. The first dose is administered after the diagnosis, followed by one vial at 2, 4, and 9 weeks after the first one. After that, a maintenance dose is administered every 4 months for as long as the patient benefits from it. The annual cost of treatment is $375,000 ($750,000 in the first year).
Manufacturer: Biogen, American multinational biotechnology company.
9. Lumizyme (API Alglucosidase alfa)
The drug is used for the treatment of Pompe disease (rare, inherited disorder). The disease is characterized by the buildup of glycogen throughout the body, which ultimately leads to impaired function of the affected organs (predominately muscles and liver). It affects one in every 40,000 persons in the US. The drug is given as an intravenous infusion, 20mg per kilogram of body mass, every two weeks. The drug comes in 50mg vials with a price of $870. Minimal annual cost of treatment is $520,000 (and can go up to $625,000).
Manufacturer: Sanofi Genzyme, MA, US.
8. Elaprase (API Idursulfase)
The drug is used for the treatment of a rare inherited disease that affects only males- Hunter syndrome. Due to the inability to breakdown specific metabolic byproducts, they build up throughout the body which causes walking and breathing difficulties. Without treatment, the symptoms become more severe over time. The dose depends on the patient’s body weight (0.5mg/kg), and the drug is given every week through slow intravenous infusion. The price per vial (6mg) is $4,215, and annual treatment for a child that weighs 35kg is $657,000.
Manufacturer: the Shire, a Biotechnology company from Ireland.
7. Brineura (API Cerliponase alfa)
Brineura is used to treat CLN2 disease (neuronal ceroid lipofuscinosis type 2 disease), a rare disorder that causes progressive brain damage.The drug is delivered every two weeks through a special device that runs from the outside of the skull to the region of infusion (inside the brain). The treatment lasts for as long as it remains beneficial for the patient.
6. Soliris (API Eculizumab)
Eculizumab (brand name Soliris) is a drug used for the treatment of a rare group of diseases that affect red blood cells. It comes in vials as a solution for intravenous infusion. The dosage depends on the stage of treatment (initial or maintenance) and the patient’s body mass, so it may be administered anywhere from once a week to once every three weeks.
5. Carbaglu (API Carglumic acid)
Carbaglu is used for the treatment of patients with elevated levels of ammonia in the blood. The indication area comes down to a handful of rare disorders, so the number of patients indicated for this kind of treatment is low. The dose ranges from 100 mg to 250 mg per kilogram of body mass, daily. The price for 5 tablets (each containing 200 mg of active substance) is around $1,040, and depending on the severity of the condition, annual treatment cost can build up to $790,000 (although, typically it’s closer to half of this price).
Manufacturer: Recordati, Italian pharmaceutical company.
4. Ravicti (API Glycerol Phenylbutyrate)
The drug is used to treat patients with urea cycle disorders (UCDs). Aside from precisely defined dosage and intake dynamics, patients who suffer UCDs need to adhere to a rigorous dietary regiment. Ravicti comes as an oral solution in 25mL vials, with a price of $5,016 per vial. The body surface area and the patient’s age determines the dosage, so the annual cost of treatment can reach up to $794,000.
3. Luxturna (API Voretigene neparvovec)
Luxturna treats adults and children with a specific kind of retinal dystrophy (mutations in gene RPE65). The drug is given as an intraocular injection after a few days of preparation (immunosuppressive medicines that reduce the chance of medicine being rejected by the patient’s body). Application of the drug is carried out in a highly controllable hospital setting by an experienced eye surgeon.
2. Zokinvy (API Ionafarnib)
Zokinvy is the first and only drug used to decrease the risk of mortality in Hutchinson-Gilford Progeria Syndrome. Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare disorder which affects approximately one in 20 million individuals. It results in young people aging way more quickly than the usual pace, which leads to the death of most patients before the age of 15 years from heart diseases. Before the FDA’s approval, the only treatment options possible were supportive care and therapies. Now, using the drug regularly can help to increase the lifespan of patients by up to several years.
1. Zolgensma (API Onasemnogene abeparvovec-xioi)
In May 2019, FDA approved new gene therapy for SMA, and with the price tag of $2,125,000, it’s the most expensive drug the world has ever seen. The drug comes in the form of suspension for intravenous infusion and targets children under the age of 2 with SMA, including those not showing the symptoms yet (the diagnosis is made through genetic testing). It’s the one-time treatment, and this is the argument that justifies such a high price- compared to the only rival on the market- Biogen’s Spinraza, Zolgensma provides a definitive cure for the disease. Having in mind the clinical research evidence collected so far, it might become a standard treatment for SMA in the future.