Drug pricing is one of the hottest topics in the healthcare segment. Several arguments have been put forward by people, governments, healthcare companies, and other organizations in favor of and against expensive medicines. Some of the drugs are so expensive that they are nearly out of reach of the common people. However, most of the expensive drugs available in the market are taken by a relatively small patient pool and the majority of these drugs are mainly prescribed for rare genetic diseases

Taking the case of Zolgensma, which is one of the most expensive drugs in the US costs around USD 2.1 million in the US. The cost paid by the patient is worth justifying by the fact that it is a 1-time treatment for SMA. While the alternative for the same drug is needed to be taken on a regular basis, for a period of 10 years. 

Expensive medications have a direct impact on the monthly household budget and take a major chunk of the savings. In some cases when the healthcare insurance plans are not available it can have a devastating social and economic impact on the person and their families. It is observed that due to the high prices, sometimes people prefer to skip the prescription altogether. The expiry of patents and the availability of generic or biosimilar alternatives bring some relief to the patients.

Companies follow different drug pricing policies based on the factors such as the number of competitors, types of payers, the drug’s uniqueness, the drug’s effectiveness, and patent protection of drugs. A considerable amount of investment in research and development (R&D) activities also affects the prices of new drugs. In some countries such as India, the government fixes the prices of some essential drugs and their formulations with the “Essential Commodities Act”. While in the United States, the price of prescription drugs is relatively unregulated. The pharma companies are provided with the option to increase their drug prices beyond inflation rates and regardless of demand. 

Through this article, we have covered some of the most expensive drugs in the US healthcare market, some of which include – 


Zolgensma manufactured by Novartis is an adeno-associated virus vector-based gene therapy that is approved for the treatment of pediatric patients under the age of two who have Spinal Muscular Atrophy (SMA) and bi-allelic mutations in the survival of motor neuron 1 (SMN1) gene. Zolgensma is administered as a single, one-time dose to address the genetic root cause of the disorder and, in the end, to generate long-term effects; many patients who take the drug exhibit no indications of the disease in the future.

Since its FDA clearance in May 2019, Zolgensma has remained the most expensive prescription drug in the United States, with a list price of USD 2.1 million for a course of therapy. Paying for Zolgensma is difficult since insurance may not cover it or may have certain requirements for individuals to qualify for coverage. To encourage insurers to cover this gene therapy, AveXis has agreed to allow certain insurers to pay for the drug in USD 425,000 annual installments over a 5-year period. AveXis provides the OneGene program to help patients negotiate insurance restrictions.


Zokinvy (lonafarnib) by Eiger BioPharmaceuticals is a prescription medication used to treat persons with rare genetic disorders that cause premature and fast aging. Lonafarnib is intended for usage in adults and children over the age of 12 whose bodies have reached a particular size (a body surface area of 0.39 square meters). Zokinvy is a drug that is used to reduce the risk of death in persons who have Hutchinson-Gilford Progeria Syndrome (HGPS). Zokinvy is also used to treat specific kinds of “processing-deficient” Progeroid Laminopathies. This medication should not be used in patients who have non-HGPS Progeroid Syndromes or “processing-proficient” Progeroid Laminopathies.

The FDA approved this breakthrough drug in November 2020. It is the first medication for Hutchinson-Gilford progeria syndrome that has been approved by the FDA. Zokinvy costs around USD 22,500 for 30 of the 50 mg capsules and USD 33,600 for 30 of the 75 mg capsules. Dosing is determined on body surface area, however, for a patient taking four 50-mg capsules each day, the annual cost would be around USD 1.08 million. While there is no copay card for this drug, the Eiger ONECARE program can assist patients with insurance coverage and financial assistance alternatives.


Danyelza is another most expensive prescription drug in the US healthcare market. Danyelza is a humanized monoclonal antibody that targets the ganglioside GD2, which is seen in a variety of neuroectoderm-derived cancers and sarcomas. Danyelza is given to patients in an outpatient environment three times a week, and the treatment is repeated every four weeks. The FDA has granted the product Priority Review, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations to Danyelza.

In November 2020, Y-mAbs Therapeutics received FDA approval for Danyelza (naxitamab-gqgk) 40mg/10ml. Danyelza is indicated for the treatment of pediatric patients 1 year of age and older, as well as adult patients, with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”). However, the drug is not cheap. One vial of Danyelza intravenous (IV) solution (40 mg/10 mL) costs roughly USD 22,018. The cost of this medicine over a year may vary between USD 1.5 and USD 2 million, but most patients would not take it for that long. Y-mAbs provides Y-mAbs Connect, a program that can help patients manage insurance, as well as a patient aid program for qualifying uninsured individuals.


Metreleptin, sold under the brand name Myalept by Amyrt Pharma, is a synthetic counterpart of the hormone leptin that is used to treat various kinds of dyslipidemia. It has been approved in Japan for metabolic diseases, including lipodystrophy, and in the United States as a replacement treatment to address the problems of leptin insufficiency in individuals with congenital or acquired generalized lipodystrophy, in addition to the diet.

Myalept’s monthly supply price rose from USD 71,306 to USD 74,159. Patients self-administer Myalept once a day and generally utilize 14 vials each month, totaling USD 889,904 per year. There are no other cost-effective options because Myalept is the sole therapy available to control this rare disease. Aegerion Pharmaceuticals provides a copay card to assist patients with affording Myalept.


Luxturna (Spark Therapeutics) is an adeno-associated virus vector-based gene therapy recommended for the treatment of individuals with verified biallelic RPE65 mutation-associated Retinal Dystrophy. Patients must have viable retinal cells, as assessed by the treating physician (s). The FDA approved Luxturna in November 2017 to treat children and adult patients with an inherited form of vision loss that may result in blindness. 

Luxturna is among the most costly therapy on the list, costing USD 850,000 per year. Luxturna cannot be obtained from a pharmacy since it needs a doctor to inject one vial of the drug into each eye. Patients should only require one dosage of Luxturna at a time, but the advertised price is exorbitant USD 425,000 per vial. Spark Therapeutics Generation Patient Services can help patients negotiate insurance coverage and link them to additional financing options.


Folotyn (pralatrexate injection) manufactured by Allos Therapeutics is an antineoplastic (anti-cancer) medication used to treat metastatic T-cell lymphoma. Folotyn is used to treat relapsed T-cell Lymphoma or when previous drugs have failed to work. In a 7-week cycle, the recommended dose of Folotyn, 30 mg/m 2 is provided as an intravenous push over 3–5 minutes once weekly for six weeks, followed by one week of rest.

Folotyn was approved to treat Peripheral T-cell Lymphoma by the FDA in September 2009. Folotyn must be administered by a healthcare practitioner, and patients are normally given 45 vials of the medicine each year. As Folotyn dose is dependent on body surface area and is administered in cycles, the cost varies, but a yearly cost can be close to USD 900,000.


Brineura (cerliponase alfa) (BioMarin Pharmaceuticals) is the sole enzyme replacement therapy that helps in the treatment of CLN2 disease, a common type of Batten Disease. Brineura is approved to reduce the loss of capacity to walk or crawl in symptomatic CLN2 disease pediatric patients 3 years of age and older by the FDA in April 2017. 

Brineura costs almost USD 763,000 per year, based on a USD 29,335 kit price. In pediatric patients 3 years of age and older, the recommended dose of Brineura is 300 mg delivered once every other week through intraventricular infusion (infusion into the fluid of the brain) using a specific port.

Brineura, an orphan medicine for this uncommon condition, joins a lengthy list of treatments that cost more than USD 1 million per year of therapy. BioMarin Co-pay Assistance Programs for Brineura is available to qualifying patients.


Blinatumomab sold under the brand name Blincyto is a bispecific CD19-directed CD3 T-cell engager used to treat relapsed or refractory B-cell precursor Acute Lymphoblastic Leukemia (ALL) in Philadelphia chromosome-negative patients. Blincyto is developed by Amgen and got FDA approval in September 2016. In addition, in 2017 and 2018, the FDA expanded its approval for relapsed or refractory B-cell Precursor Acute Lymphoblastic Leukemia in adults and children and minimal residue disease positive B-cell Precursor Acute Lymphoblastic Leukemia treatment respectively.

Blincyto is on the list of the most expensive drugs, costing USD 712,672 per year on the market. Blincyto is only available through a healthcare professional, and it is given in cycles. The first cycle referred as the induction phase, is designed to reduce the number of cancer cells in the body. The consolidation stages, which last from cycles two to four, aid the growth of new, healthy cells. During each phase, patients will require varying amounts of medication, but on average, 168 vials will be used every year. In January 2021, the current list price of USD 4,242 per vial climbed by 5.89%.


Ravicti by Horizon Therapeutics is a prescription medication used to treat the symptoms of Urea Cycle Disorders. Ravicti can be taken alone or in conjunction with other drugs. Ravicti belongs to a class of medications known as Urea Cycle Disorder Treatment Agents. The FDA approved Ravicti in February 2013. In addition, in 2017 and 2018, the FDA extends its approval to the age range for Ravicti oral liquid to people with Urea Cycle Disorders two months of age and older, and newborns respectively.

Ravicti, which is administered as a liquid by mouth, costs around USD 700,000 per year of treatment. These expenses, however, will vary depending on the precise dose. The dosage will change if you switch from sodium phenylbutyrate to Ravicti. The Horizon Cares Patient Assistance Program offered by manufacturer Horizon Therapeutics allows some patients to save money on Ravicti.


Soliris is a first-in-class terminal complement inhibitor discovered, developed, and commercialized by Alexion. Soliris works by specifically suppressing the activation of particular complement system proteins (C5a and C5b), which play a role in the pathogenesis of multiple rare diseases. Soliris is used to treat the symptoms of Paroxysmal Nocturnal Hemoglobinuria, Hemolytic Uremic Syndrome, Myasthenia Gravis, and Neuromyelitis Optica Spectrum Disorder. Soliris can be taken alone or in combination with other drugs. Soliris belong to a class of drugs known as monoclonal antibodies. Soliris is associated with a high risk of infection. As a result, the medicine is included in a Risk Evaluation and Mitigation Strategy (REMS) program. Drugs such as Soliris raise major safety issues and require a REMS program.

The first approval for the Soliris came in 2007 when the FDA approved the drug for Paroxysmal Nocturnal Hemoglobinuria. In 2011, Alexion got FDA approval for Soliris for Atypical Hemolytic Uremic Syndrome. Furthermore, in 2017, the drug got approved for the treatment of patients with Generalized Myasthenia Gravis. In 2019, Soliris received its fourth approval by the FDA for Neuromyelitis Optica Spectrum Disorder treatment. Soliris is sold in single-dose vials of 300 mg/30 mL (10 mg/mL) for around USD 6820. Its annual cost ranges from USD 566,000 to USD 764,000 per patient, depending on the condition being treated and body weight (in patients under 18 years of age). Although there is no copay card for Soliris, the manufacturer Alexion’s OneSource program can assist patients with navigating health insurance.


Lumizyme (alglucosidase alfa) from Genzyme is a lysosomal glycogen-specific enzyme used to treat Pompe Disease. Excess glycogen in this disease causes worsening muscle weakness (myopathy) and can impair the heart, skeletal muscles, liver, and nervous system. This enzyme is required to degrade glycogen, a stored type of sugar utilized for energy. Lumizyme, which substitutes this enzyme, was authorized for the Pompe disease indication in 2014 by the FDA. While this drug may appear to be a steal at around USD 905 per vial, each patient would require an average of 56 vials each month for a 70 kg (154 lb) individual. Based on this, Lumizyme might cost around USD 51,000 per month or close to USD 612,000 per year. However, Lumizyme’s manufacturer Genzyme offers copay help to eligible patients.


Actimmune is a part of the drug regimen used to treat Chronic Granulomatous Disease, often known as CGD. CGD is a genetic condition that impairs some immune system cells and the body’s capacity to fight infections efficiently. It is often diagnosed in childhood. Antibiotics, antifungals, and Actimmune are frequently used to treat but not cure CGD. Actimmune is also used to treat severe malignant Osteopetrosis (SMO). SMO is a hereditary condition that interferes with normal bone growth and is often detected within the first few months of life.

Horizon’s Actimmune (interferon gamma-1b) is also among the most costly genetic disease treatment drugs. Actimmune is administered three times per week as a subcutaneous (under the skin) injection. For patients taking the 50 mcg/m2 dosage, the sticker shock can be real, costing more than USD 819,000 per year. 


As per various surveys conducted worldwide across different age groups, it is observed that the majority of the people think that the costs of prescription drugs are unaffordable and similarly a major proportion of the people said that they haven’t taken their medications as prescribed due to cost factors. However, considering the amount of money invested in the R&D activities of drug development, advertising, and marketing, the high pricing is the only way to recover the investment.