Mostly in these Doc Gumshoe pieces I try to keep my focus on what the physicians and researchers and pharmaceutical outfits are doing to try to treat, or at least manage, all these diseases, ailments, and conditions that affect our lives. But other issues keep thrusting themselves into the picture, especially the cost factor. In the most recent Doc Gumshoe pronouncement, I looked into a new proposed treatment for a disease of genetic origin that is responsible for about 20% of all instances of childhood blindness. The treatment is indeed promising. One little fact that I mentioned is that a one-time treatment for both eyes was estimated to cost about $850,000.
I also discussed the surgical treatment for Parkinsonism, which called for creating pluripotent stem cells from the patients’ own skin cells and then implanting these stem cells into the patients’ brains, and which would also be enormously expensive, at least until generating those stem cells became more routine. That is, of course, normal for any novel medical treatment. New drugs, new surgeries, new treatment options, will always be considerably more expensive than the established drugs and the commonly practiced procedures.
The most expensive drugs today in the US
Topping the list is Zolgensma (onasemnogene abeparvovec-xioi, from Novartis), at $2,125,000 for a one-time treatment. It is approved for spinal muscular atrophy, a previously incurable disease caused by a missing or defective gene labeled SMN1. A number of experts have suggested that despite its astronomical price, Zolgensma is a cost-effective treatment, because it can actually cure a once-incurable disease, thereby eliminating the costs of lifetime care. Children who have been treated with this drug show no further signs of the disease.
Myalept (metreleptin, from Aegerion, developed by AstraZeneca) comes in second at $855,678 per year, which in terms of price is well behind Zolgensma. However, the $855,678 sticker price is based on a monthly estimated cost of $71,306.48 per month, so a lifetime of treatment could mount up considerably. It is used to treat leptin deficiency in patients with generalized lipodystrophy, a condition of abnormal fat distribution in the body. Patients self-administer Myalept once a day. It is the only drug available to treat this condition.
Luxturna (voretigene neparvovec, from Spark Therapeutics) is number three on the list, at $850,000. Like Zolgensma, it is a form of gene therapy. Luxturna addresses an inherited form of retinal dystrophy, a condition that can cause complete blindness. Patients should require just one dose of Luxturna per eye, at $425,000 per vial.
Folotyn (pralatrexate, from Acrotech) stands at number four on the list, with an annual cost of $745,785. It is used to treat peripheral T-cell lymphoma, a rare form of blood cancer which is sometimes fatal. It is administered by health care professionals, and patients usually are given 45 vials of the drug annually.
Soliris (eculizumab, from Alexion) occupies the fifth slot on the list. The estimated annual cost is $678,392 per year. As we can tell from the name, it is a monoclonal antibody, and it binds and disables proteins in the blood that can destroy red blood cells in some persons. It is used to treat adults with paroxysmal nocturnal hemoglobinurea and also to treat hemolytic uremic syndrome and myasthenia gravis. Alexion is also planning to start a phase 2 study of Soliris as a treatment for COVID-19 very shortly.
Blincyto (blinatumomab, from Amgen) is in sixth place in the ranking of the most expensive drugs, at an annual list price of $672,968. It is used to treat a rare form of acute lymphoblastic leukemia, which is a cancer of the blood and bone marrow. The treatment is administered in cycles, starting with the induction phase, which aims at reducing the number of cancer cells. The succeeding treatment cycles foster the growth of new, healthy cells. Patients need different amounts of the medication during each phase, but will typically use about 168 vials per year at the current list price per vial of $4,005.76.
Ravicti (glycerol phenylbutyrate, from Horizon Therapeutics) occupies seventh place on the list, at an annual list price of $664,092. It is used to treat urea cycle disorders, which can result in dangerously high levels of ammonia in the blood. It is approved for anyone over two months of age.
Lumizyme (algoglucosidase alpha, from Sanofi/Gemzyme) comes in at eighth place in the ranking, at an annual list price of $643,243. It is a drug that replaces an enzyme, alpha glucosidase, which is missing or deficient in persons with Pompe disease. Pompe disease is a genetic disorder resulting in a build-up of a complex sugar (glycogen) in the body’s cells, and a significant reduction in life expectancy.
Actimmune (interferon gamma 1b, from Horizon Therapeutics) is in ninth place on the list, at an annual list price of $633,325. It is indicated for reducing the frequency and severity of serious infections associated with chronic granulomatous disease, and for delaying time to disease progression in patients with severe, malignant osteopetrosis. This is a rare bone disease in which the bones become harder, more brittle, and prone to fracture.
Takhzyro (lanadelumab flyo, from Takeda) is in tenth place on the list, at an annual list price of $591,035. It is used to treat hereditary angioedema, which causes recurrent episodes of severe swelling in the lim