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Specialty Meds: The Top 10 Most Expensive Drugs

Medically reviewed on Oct 29, 2017 by L. Anderson, PharmD.

Can You Afford Your Medications?

Prescription drugs are one of the most important -- if not the most important part -- of a patient's health care regimen. But drugs are expensive.

A medication can ease a simple headache, as with an affordable and generically available pain reliever like acetaminophen (Tylenol). A drug may be life-saving, say any one of the new biologic immunotherapy agents like pembrolizumab (Keytruda) that can target a melanoma skin cancer. Either way - in the U.S. - a patient should be able to afford the treatment they need.

But you have probably found yourself in a situation where the cost of treatment was just too high. Are your annual deductibles so out of reach - say $3000 per year or more - that it requires you to borrow money to pay your off your bills? Do the co-insurance bills from the doctor pile up on your table as you try to figure out how pay them? Did you skip filling that prescription last month because groceries were more important for your family?

What Determines The Cost of Drugs?

Why are prescription drugs so expensive in the US? How much a patient pays for medication is dependent upon many factors:

  • Whether or not a patient has insurance coverage for drugs
  • Whether a drug is paid for on the health insurance formulary
  • Any rebates or discount from the manufacturer, patient deductibles and copays
  • Any financial assistance they might be eligible to receive from either the government (Medicaid, Medicare), discounting coupons, or free samples from the doctor.
  • The price set by the manufacturer
  • Generic or biosimilar availability

Why Are Drugs So Expensive in the US?

Why are drugs expensive? How a drug's price is determined is complicated and for most of us remains a mystery. There is a lack of transparency in how drugs are priced by the pharmaceutical company. Add to that, complicated discounting and rebates to wholesalers and government entities, insurance formularies, and FDA drug laws that extend the patent life, and the confusion grows.

A 2016 report by America's Health Insurance Plans (AHIP) projected that drug spending in the US will grow to $590 billion by 2020, up from $337 billion in 2015. But is it's not the pain relievers, blood pressure pills, or simple antibiotics that's fueling this growth. The culprit is specialty drugs developed in recent years, like the new biologics for multiple sclerosis, rheumatoid arthritis, or as cancer treatments. Why is drug development so expensive? The complicated manufacturing processes involved in some of the most advanced therapies today play a part in high costs. But some expensive drugs are just repurposed generics, too.

AHIP reported that 225 new specialty-type drugs would come to market by 2021. Specialty drugs make up roughly 30% of all spending on drugs, but only account for 2% of all prescriptions. And close to half of these drugs cost over $100,000 per patient-per year. What costly drugs and medical conditions are burdening the American Health Care System in 2017?

Just How Expensive Are Drugs? A Look at Sovaldi

Looking at the monthly cost of a drug can give you an idea of the out-of-pocket cost in the short-term, but the bigger impact comes when you start looking at the cost of the medication over a required treatment period, or on an annual per patient-per year basis. AHIP estimated costs for 150 specialty medications based on their real-world use using commonly accepted doses based on medical condition, age, weight, clinical trial data, and average or yearly duration of use. Pricing is presented in a range of estimated low to high costs.

For example, one of the most controversial - and costly - drugs in recent history was when the hepatitis C virus (HCV) medication Sovaldi (sofosbuvir) from Gilead Sciences was approved in December 2013. Headlines proclaimed that the medication, which is actually a life-saving and curative drug for many HCV patients, would cost $1,000 per pill. However, drug expenses could run between $84,000 and $170,000 or more over the treatment course depending upon if a patient needs 12 or 24 weeks of treatment. Here's a look at the Top 10 most expensive drugs in the U.S. -- and on the planet.

10. Ilaris (canakinumab)

Autoimmune system disorders, such as rheumatoid arthritis, are notorious for high priced medications that are developed as biologics. Enbrel, Humira, and Xeljanz all fall in this group. Biosimilars are now coming to the market for some of these agents, but how much biosimilars might save the actual patient who is responsible for high copays waits to be seen.

Ilaris (canakinumab), approved in June 2009, is an injectable human anti-interleukin-1β monoclonal antibody from Novartis approved for:

The annual per-patient expenditure for Ilaris ranges from about $379,000 to $462,000, as published by AHIP in 2016.

9. Demser (metyrosine)

Demser, (metyrosine) from Valeant Pharmaceuticals, is used to decrease the amount of certain chemicals (catecholamines) in the body caused by a type of adrenal gland tumor (pheochromocytoma). Patients with pheochromocytoma produce excessive amounts of norepinephrine and epinephrine, which can dangerously elevate the blood pressure.

Demser may be used as a pretreatment before surgery or for the long-term treatment of symptoms associated with this disorder, such as in patients when surgery is contraindicated, or for chronic treatment of patients with malignant pheochromocytoma.

According to AHIP, Demser tends to run anywhere from $96,000 to $472,000 annually per patient depending upon if you are looking at costs from the heavily discounted Federal Supply Schedule (FSS) from the VA up to the highest prices found for the Average Wholesale Price (AWP).

8. Folotyn (pralatrexate)

Folotyn (pralatrexate), FDA-approved in 2009 from Allos Therapeutics, is used as a last chance medication for patients with relapsed or refractory peripheral T-cell lymphoma (PTCL), an aggressive form of non-Hogkins's lymphoma. PTLC is a relatively rare disease, occurring in less than 9,500 patients each year in the US. Folotyn is classified as a cytotoxic antimetabolite (folate antagonist) cancer agent given by intravenous injection.

As noted in AHIP's report, cost for Folotyn runs from a low of roughly $345,000 to a high of $541,000 per year, per patient.

According the manufacturer, clinical benefit such as improvement in progression-free survival or overall survival has not been demonstrated.

7. Soliris (eculizumab)

Soliris (eculizumab), from Alexion Pharmaceuticals is a specialty drug FDA-approved to treat a rare, chronic blood disease called atypical hemolytic uremic syndrome (aHUS), as well as to prevent the breakdown of red blood cells in people with paroxysmal nocturnal hemoglobinuria (PNH). Plus, in October 2017, it was approved to treat adults with the neuromuscular disorder generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive.

It is a monoclonal antibody, only available through the Soliris REMS program, and falls within treatments of genetic diseases - one of the most costly groups of diseases to treat.

It's annual price tag in AHIP's list can run anywhere from between $432,000 and $542,000 per patient. Soliris is often described as the most expensive drug in the world, but it's obvious when you look at other medications -- and their annualized cost -- this may not always be the case.

6. Actimmune (interferon gamma-1b)

Actimmune (interferon gamma-1b) from Horizon also falls in the very expensive group of genetic disease treatments. When used to treat severe, life-threatening osteopetrosis (the formation of abnormally dense bone, as opposed to osteoporosis) or to lower the occurrence of serious infections due to chronic granulomatous disease, the sticker shock per patient can be palpable, ranging from $244,000 to 572,000 annually per AHIP.

It's important to remember many specialty drugs may have manufacturer patient assistance programs, federal and/or state government programs, or consumer groups to help with cost assistance. However, eligibility can depend on numerous factors, including:

  • Income
  • Health insurance status
  • Government assistance for healthcare

5. Lumizyme (alglucosidase alfa)

Lumizyme (alglucosidase alfa) from Genzyme is a lysosomal glycogen-specific enzyme used to treat Pompe disease.

Lumizyme was approved for the Pompe disease indication in 2014. A specific enzyme is needed to break down glycogen, a stored form of sugar used for energy. Excess glycogen causes worsening muscle weakness (myopathy) and can affect the heart, skeletal muscles, liver and nervous system.

Lumizyme could have a price tag running anywhere from $524,000 to 626,000 per year. While this drug may seem like a bargain at about $800 per vial, each patient, on average, would need about 56 vials per month, according to AHIP.

4. Carbaglu (carglumic acid)

Carbaglu (carglumic acid) is an orally administered drug used to help treat and prevent build-up of dangerous ammonia (hyperammonemia) in the blood due to the lack of a needed liver enzyme.

Like Lumizyme, Carbaglu is extremely expensive, ranging from a minimum of $419,000 to a maximum of $790,000 per patient-per year as noted by AHIP. Of course, what is actually paid by insurance groups or the government may depend upon if any rebates are awarded if the drug is put on their formulary. And what the patient pays depends upon multiple factors that are often out of their hands.

You might ask if Carbaglu treatment goes on for an extended time? In clincial trials the effectiveness of Carbaglu in the treatment of hyperammonemia due to the enzyme deficiency was evaluated in 23 patients who received treatment for a median of 7.9 years, with a range 0.6 to 20.8 years. Long-term treatment costs can be exhorbitant for the patient and the health care system to absorb.

3. Brineura (cerliponase alfa)

Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, is a rare inherited disorder that primarily affects the nervous system. It's devastating, patients often require the use of a wheelchair by late childhood and typically do not survive past their teens

Batten disease typically begins in early childhood - between the ages of 2 and 4 - with symptoms such as language delay, seizures and difficulty coordinating movements. At the end of April 2017, the FDA approved Brineura (cerliponase alfa), from BioMarin Pharmaceutical, for this specific form of Batten disease. Brineura is an enzyme replacement therapy for tripeptidyl peptidase-1 (TPP1). It's the first FDA-approved treatment to slow the decline of walking ability in symptomatic children 3 years of age and older with CLN2.

This orphan drug for this rare disease joins others in the long list of drugs approaching a million dollars per year of treatment. Reports from Fortune state that Brineura could run close to $700,000 per year, although what a patient or their family pays is affected by multiple circumstances, including manufacturer support.

2. Ravicti (glycerol phenylbutyrate)

Ravicti (glycerol phenylbutyrate), from Horizon Pharma, is another drug to help prevent a build-up of toxic ammonia in the blood in people with urea cycle disorder. Ammonia is very toxic in the blood and tissues and can lead to permanent brain damage, coma, or death. Urea cycle disorders only affect about 2,100 people in the U.S. -- definitely an orphan drug for an orphan disease. Ravicti is given as an oral liquid.

The price tag for Ravicti, as noted by AHIP, could run up to $793,000 per patient-per year of treatment. In 2016, Horizon had net sales of Ravicti hovering in the $151 million range. Acquiring or developing orphan drugs, and attaching an extraordinary price tag, is the new business model for many pharmaceutical companies in the US, and because economic outcome studies are not required prior to FDA approval, the costs vs. benefits are often misaligned.

1. What is the Most Expensive Drug in the World?

The most expensive drug in the world is not even approved in the U.S. - and it probably never will ever be. Overall, it's commercial success has been a failure, and it may be due to its sky-high cost and uber-rarity of the disease.

The drug is an injection known as Glybera (alipogene tiparvovec) and it was the first approved gene therapy in the Western world. It's used to treat a very rare condition known as lipoprotein lipase deficiency. These patients have a defect in the gene for lipoprotein lipase, an enzyme responsible for breaking down fats. Patients with lipoprotein lipase deficiency have severe and life-threatening attacks of pancreatitis (inflammation of the pancreas) despite maintaining a low-fat diet.

The asking price of the world's most expensive pharmaceutical drug? Over $1.2 million per year of treatment. It was approved in the EU in 2012, but isn't seeing much use even for this rare condition. In fact, the manufacturer UniQure reported that they will not pursue marketing authorization in 2017 for renewal of Glybera in Europe. Plus, the company abandoned its plans to win an FDA approval after the agency asked for new clinical trials, per FiercePharma.

Finished: Specialty Meds: The Top 10 Most Expensive Drugs

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