Health Insurance Matters: 10 Most Expensive Drugs

By Harry P. Thal

One of the blogs I most enjoy is provided by goodrx.com. I’m sure you have seen their ads on television and other media. This company provides a free service and is a great resource for seeking the best prices for medication, for especially those with either no health insurance or with a high deductible or co-pay.

The cost of research, development, manufacturing and distribution of a drug is spread out over the millions of people with the condition who benefit from the medication. When the number of patients is small and limited, the costs are borne by a few.

Tori Marsh, MPH, is on the Research team at GoodRx and is the resident expert on drug pricing and savings. My thanks to Dr. Marsh for her insightful work.
Many drugs are expensive—but some drugs are crazy expensive. That’s what a new analysis by the GoodRx Research team reveals.

Every quarter, the GoodRx Research team tracks the most expensive medications filled at local pharmacies in the United States, and the same drugs continue to make the top of the list. Actimmune, Myalept, and Daraprim all cost over $40,000 for a typical monthly supply. However, these are the most expensive drugs filled at pharmacies and are self-administered by patients on a regular basis.

But these days, with the million-dollar drugs in the headlines, we wanted to also track the most expensive drugs, period—including the expensive infusions, cancer treatments, and the gene therapies that you can’t get from a pharmacy. We call these drugs “healthcare practitioner administered drugs,” and it may not be surprising—but they aren’t cheap.

Many of the most expensive drugs are taken for an extended period of time, and costs quickly add up. This analysis ranks drugs by their annual cost for a typical course of therapy. Prices are based on the drug’s list price, which is the price that the pharmaceutical company assigns as an official price to a drug, then adjusted for time to create a comparable benchmark.

The following are the 10 most expensive drugs, period, in the U.S., taking into account both drugs you can get at a pharmacy and healthcare practitioner-administered drugs. Most of these drugs are taken by relatively few people—but their outsized costs translate into higher premiums and more restrictive formularies for everyone.

1) Zolgensma is one of the newest drugs on the market, and the most expensive. With a list price of $2.1 million for a course of treatment, Zolgensma has caused an uproar in the prescription drug community with many blasting it for its astronomical price tag. However, many are citing it as a surprisingly cost-effective treatment, as it can cure a once-incurable disease.

Zolgensma is approved to treat spinal muscular atrophy, a rare childhood disorder that causes muscular erosion that can lead to lung infections and muscle weakness. Zolgensma is a one-time curative therapy, and many children who have been treated with the drug now show no signs of the disease.

So, how can anyone afford this life-saving drug? Unfortunately, cash-paying patients may be on the hook for the full price of the drug. Luckily, some insured patients may be able to get coverage through their plan, as the manufacturer has allowed certain insurers to pay for the drug in annual installments of $425,000 over five years.

2) Luxturna At $850,000 a year, Luxturna is the second most expensive drug on the list but is over $1 million cheaper than Zolgensma. Luxturna is a gene therapy that treats an inherited form of retinal dystrophy, a condition that causes vision loss and even complete blindness. Luxturna cannot be dispensed at a pharmacy as it requires a doctor to administer one vial of Luxturna into each eye. Patients should only need one dose of Luxturna ever, but the list price is steep—ringing up at $425,000 per vial. Luxturna cannot be dispensed at a pharmacy as it requires a doctor to administer one vial of Luxturna into each eye. Patients should only need one dose of Luxturna ever, but the list price is steep—ringing up at $425,000 per vial.

3 ) Myalept is the first medication on the list that is dispensed in a pharmacy. It is used to treat leptin deficiency in patients with generalized lipodystrophy, a condition of abnormal fat distribution in the body. Myalept is self-administered once a day, and patients typically use 14 vials per month at a list price of $4,633 per vial, bringing the yearly price tag to $778,314. Because Myalept is the only treatment available to control this rare condition, there are no other cost-saving options. Aegerion Pharmaceuticals offers a copay card to help commercially insured patients afford Myalept.

4) Folotyn is approved to treat peripheral T-cell lymphoma, a rare blood cancer that can cause death. Folotyn is administered by a healthcare professional, and patients are typically given 45 vials of the drug annually. With a list price of $5,524 per vial, the annual cost is $745,785. Despite its high price tag, Folotoyn’s effectiveness has been called into question. In fact, it has not yet been approved in the E.U. due to insufficient evidence of health gains.

5) Soliris Priced at $678,392 per year, Soliris is used to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), blood disorders that result in the destruction of red blood cells. The dosing regimen for Soliris can differ depending on the age of the patient and the condition being treated, but most patients need a maintenance dose of 1200 mg every 2 weeks. Soliris can only be administered by a healthcare practitioner. Because Soliris comes with a high risk of infection, the drug is part of a Risk Evaluation Mitigation Strategy (REMS) program. Medications with serious safety concerns require a REMS program, and healthcare providers who prescribe these drugs often need to undergo specific training to administer them.

6) Blincyto The sixth most expensive drug, Blincyto, has an annual list price of $641,533. Manufactured by Amgen, Blincyto is used to treat a rare form of acute lymphoblastic leukemia, a cancer of the blood and bone marrow. Blinctyo can only be administered by a healthcare practitioner, and dosing is done in cycles. The first cycle, known as the induction phase, is aimed at reducing the number of cancer cells, whereas cycles two through four, known as consolidation phases, help new healthy cells grow. Patients need different amounts of the medication during each phase, but will typically use about 168 vials per year. The current list price per vial is $3,819.

7) Ravicti Used to treat urea cycle disorders, Ravicti has an annual list price of $633,072. Urea cycle disorders are genetic conditions that result in high levels of ammonia in the blood. If left untreated, urea cycle disorders can lead to confusion, coma, or even death. Ravicti is dispensed in a pharmacy and doesn’t require administration from a healthcare practitioner. Patients are typically given 132 bottles annually, each bottle with a list price of $4,796. Horizon Therapeutics, the manufacturer, offers a way for some patients to save on Racivti. Through the Horizon Cares Patient Assistance Program, uninsured and low-income patients can get Ravicti for free.

8) Lumizyme is used to treat Pompe disease, an inherited disorder that causes a buildup of complex sugars in the body that affects the liver, heart, and muscles, which leads to muscle weakness and trouble breathing. Lumizyme has an annual list price of $630,630.
Patients must get Lumizyme from their doctor, and the drug is not available in pharmacies. Dosing for Lumizyme is based on a patient’s weight; patients are given 20 mg of Lumizyme per kg of body weight through an IV infusion. For example, a person weighing 75 kg will likely use 60 vials a month of Lumizyme, each vial with a list price of $809.

9) Actimmune After Ravicti, Actimmune is the second drug on this list manufactured by Horizon Therapeutics, and it has an annual list price of $575,540. Actimmune is approved to treat osteopetrosis and chronic granulomatous disease, rare disorders that cause the immune system to malfunction. Actimmune can be dispensed in a pharmacy, and patients typically use about 132 vials in a year, each vial with a list price of $4,360. Horizon Therapeutics offers a patient assistance program to help uninsured and low-income patients get Actimmune at no cost.

10) Takhzyro The 10th most expensive drug in the U.S., Takhzyro, is used to treat hereditary angioedema, a condition that causes swelling in the limbs, face, intestinal tract, and airways. Patients typically need two vials of Takhzyro per month. With a list price of $22,070 per vial, Takhzyro’s annual list price comes out to $573,820.

Harry P. Thal, MA, is a licensed insurance broker in California (0621106) and 27 other states. His offices are in Kernville. He is a member of the Society of Certified Senior Advisors and Past-President of the Kern Association of Health Underwriters. He may be reached at 760-376-2100, e-mail harrythal@aol.com or visit him on the web at www.harrythal.com.