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Specialty Drugs: 10 of the Most Expensive

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Oct 20, 2019.

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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, and it seems to be getting worse.

  • 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 one of the novel biologic immunotherapy checkpoint inhibitors like pembrolizumab (Keytruda) that can target a potentially deadly melanoma skin cancer.

Either way - a patient should be able to afford or have coverage for the treatments they need to stay alive.

But you have probably found yourself in a situation where the cost of treatment was just too high.

  • Are your annual medical deductibles so out of reach -- say $4000 per year or more -- that it requires you to borrow money to pay your off your bills?
  • Do the 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 you had to buy groceries for your family, and there was no other option?

What Determines The Cost of Drugs?

Why are prescription drugs so expensive in the US?

How much you pay for medication is dependent upon many factors:

  • Whether or not you have insurance coverage for drugs.
  • If a drug is paid for on the health insurance formulary.
  • Any rebates or discounts from the manufacturer
  • Your prescription deductibles and copays.
  • If it's a specialty drug, gene therapy, or biologic drug, considered some of the highest priced drugs today.
  • Any financial assistance you might be eligible to receive from either the government (Medicaid, Medicare), discount coupons, or free samples from your doctor.
  • The price set by the manufacturer and their interaction with pharmacy benefit managers (PBMs).
  • Generic or biosimilar availability.

Learn More: FAQs on Generics and Biosimilars

Why Are Drugs So Expensive in the US?

How a drug's price is determined is complicated and for most of us remains a true mystery. There is a lack of transparency in how drugs are priced by the pharmaceutical company. The true cost of any drug may be hard to determine until you're at the pharmacy or get a bill in the mail.

Add to that:

  • complicated discounting and rebates to wholesalers and government entities
  • constantly changing insurance formularies
  • FDA drug laws that extend the patent life of a drug before it can become a generic
  • the need to use a "specialty" pharmacy to determine if your medicine is covered by insurance

and the confusion grows.

A 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 just a few years ago.

But it's not the pain relievers, blood pressure pills, or 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.

Granted, many of these new medications are life-saving and important. But 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 are some examples of costly drugs and medical conditions that are burdening the American Health Care System?

Just How Expensive Are Drugs? A Look Back 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 by using commonly accepted doses. Pricing is presented in a range of estimated low to high costs.

For example, one of the most controversial -- and costly -- drugs in past history was when the hepatitis C virus (HCV) medication Sovaldi (sofosbuvir) from Gilead Sciences was first 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 $90,000 and $180,000 or more over the treatment course depending upon if a patient needed 12 or 24 weeks of treatment.

So that was just a start: here's a look at some of the most expensive drugs in the U.S. -- and on the planet. And remember, most of these drugs you cannot get in a regular pharmacy. Usually your doctor would administer it as an injection or you might have to receive them in specialty infusion clinic.

Ilaris (canakinumab)

Ilaris (canakinumab), approved in June 2009, is an injectable human anti-interleukin-1β monoclonal antibody from Novartis approved for various Periodic Fever Syndromes -- rare conditions often caused by mutations in certain genes.

  • People with a periodic fever syndrome have episodes of fever and inflammation without other causes such as infection or virus.
  • Ilaris is also approved to treat Sudden Juvenile Idiopathic Arthritis (SJIA) in children 2 years of age and older.

The annual per-patient expenditure for Ilaris when used for SJIA can range from $379,000 to $462,000, as published by AHIP.

Demser (metyrosine)

Patients with pheochromocytoma produce excessive amounts of norepinephrine and epinephrine, which can dangerously elevate the blood pressure.

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).

Demser may be used:

  • as a pretreatment before surgery or for the long-term treatment of symptoms associated with this disorder
  • 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).

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 U.S.
  • Folotyn is classified as a cytotoxic antimetabolite (folate antagonist) cancer agent and it's given by intravenous injection.

As noted in AHIP's report, cost for Folotyn runs from a low of roughly $345,000 to over $500,000 per year, per patient.

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

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)
  • to prevent the breakdown of red blood cells in people with paroxysmal nocturnal hemoglobinuria (PNH)
  • for adults with the neuromuscular disorder generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive
  • the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) 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 yearly price tag as noted by WellRx can run upwards of $$600,000 per patient, per year.

Soliris used to be considered the most expensive drug in the world, but there are others now that far exceed it.

Actimmune (interferon gamma-1b)

Actimmune (interferon gamma-1b) from Horizon also falls in the very expensive group of genetic disease treatments.

Actimmune is indicated:

  • to treat severe, life-threatening osteopetrosis (the formation of abnormally dense bone, as opposed to osteoporosis, which is brittle bone)
  • to lower the occurrence of serious infections due to chronic granulomatous disease.

The sticker shock per patient can be palpable, roughly $700,000 per year, based on reported prices from WellRx.

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:

  • Financial status, such as income
  • Health insurance status
  • Government assistance for healthcare.

Lumizyme (alglucosidase alfa)

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

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

While this drug may seem like a bargain at about $800 per vial, each patient, on average, would need about 56 vials per month, for a 70 kg (154 lb) person. Based on this, Lumizyme could have a price tag running about $650,000 per year.

Carbaglu (carglumic acid)

It's obvious having a rare disease where you are missing a needed enzyme can be very expensive.

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 required liver enzyme (known as N-acetylglutamate synthase, or NAGS). It works by preventing a build-up of nitrogen in the bloodstream that can quickly cause coma and death.

Like Lumizyme, Carbaglu is wildly expensive, running at about $200 per 200 mg tablet, as posted by WellRx. At a mid-point recommended maintenance dose (50 mg/kg/day), a 70 kg person would need roughly 3,500 mg per day, or 18 tablets per day ($3600 per day), which would run about $1.3 million per year.

But does Carbaglu treatment actually continue over a long period, say for years?

  • In clinical trials the effectiveness of Carbaglu in the treatment of hyperammonemia due to the enzyme deficiency was evaluated in only 23 patients, as it's such a rare disease. They received treatment for a median of 7.9 years, with a range from 7.2 months to over 20 years.

These long-term treatment costs can be exhorbitant for the patient and the health care system to absorb. Of course, what is actually paid by insurance groups or the government may depend upon if any rebates are awarded, or if the drug is actually covered. Patient assistant for drug costs may also be available, but what the patient actually pays depends upon multiple factors that are often out of their hands.

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 and patients often require the use of a wheelchair by late childhood. These children 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
  • difficulty coordinating movements.

At the end of April 2017, the FDA approved Brineura (cerliponase alfa), from BioMarin Pharmaceuticals, for this specific form of Batten disease.

An orphan drug for this rare disease, Brineura joins others in the long list of drugs approaching one million dollars per year of treatment. Orphan drugs are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.

Brineura is an enzyme replacement therapy for tripeptidyl peptidase-1 (TPP1). It's the first FDA-approved treatment to slow the decline of walking disability in symptomatic children 3 years of age and older with CLN2.

Reports from Fortune state that Brineura runs close to $700,000 per year, although what a patient or their family pays is affected by multiple circumstances, including manufacturer support. Orphan drugs are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.

Ravicti (glycerol phenylbutyrate)

Urea cycle disorder definitely classifies as an orphan disease as it only affects about 2,100 people in the U.S

Like Carbaglu, Ravicti (glycerol phenylbutyrate), from Horizon Pharma, is a treatment approved to help prevent a build-up of toxic ammonia in the blood in children from birth and older. Ammonia in the blood and tissues and can lead to permanent brain damage, coma, or death.

The price tag for Ravicti, given as an oral liquid, could run up to $1.22 million per patient-per year of treatment based on the maximum recommended dose. In the first half of 2019, Horizon had sales of Ravicti hovering in the $100 million range.

Acquiring or developing orphan drugs, and attaching an extraordinary price tag, is the new business model for many pharmaceutical companies in the U.S. Because economic outcome studies are not required prior to FDA approval, the costs vs. benefits are often misaligned. However, without these treatments, many patients might not have any other options.

What is the World's Most Expensive Drug?

So what is the world's most expensive drug now? That's up for debate. Many say it's Zolgensma (generic name: onasemnogene abeparvovec-xioi), a life-saving gene therapy approved in May 2019 to treat pediatric Spinal Muscular Atrophy (SMA).

  • SMA is a rare genetic motor neuron disease that results in problems with breathing, holding up your head, and swallowing. The majority of children with SMA do not survive past early childhood due to an inability to breathe.
  • At approval time, Zolgensma's total cost was $2.125 million, according to AveXis/Novartis, the pharma group tasked with commercialization. It's considered the world's most expensive single-dose drug. But over a lifetime, Biogen's SMA drug Spinraza (nusinersen) might actually cost much more.
  • Zolgensma is given as a one-time intravenous (IV) dose. It works by replacing the defective or missing survival motor neuron 1 (SMN1) gene, and is considered potentially curative.

Zolgensma now replaces Glybera (alipogene tiparvovec), previously the most expensive drug and the first approved gene therapy in the Western world. Glybera is no longer marketed.

  • It was approved in Europe, but is no longer available there, and it never reached FDA approval status in the U.S. It was priced at over $1.2 million per year of treatment
  • It was used to treat a very rare condition known as lipoprotein lipase deficiency that leads to severe and life-threatening attacks of pancreatitis (inflammation of the pancreas) despite maintaining a low-fat diet.
  • Glybera's commercial success was a failure, due to a mix of costly price tag and rarity of the disease it treats.

Finished: Specialty Drugs: 10 of the Most Expensive

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