New champion for the most expensive drug in the US

It's widely acknowledged that medications for rare diseases and gene therapies come with a hefty price tag. In the United States, the FDA oversees drug approval, but there's no governmental oversight on pricing. After FDA approval, drug sponsors and manufacturers have the autonomy to propose and set the drug's price. Typically, pricing considerations revolve around factors like patient population, investment in drug development and approval, and anticipated financial gains, rather than the drug's effectiveness or its benefit-risk profile.

Before this month (March, 2024), the top 10 most expensive drugs in the US are:

Brand Name	Cost	Manufacturer	Indication	FDA approval date	Reason for high cost
Hemgenix	$3.5 million per one-time dose	CSL Behring	Hemophilia B	November 22, 2022	Gene therapy
Elevidys	$3.2 million per one-time dose	Sarepta Therapeutics	Duchenne Muscular Dystrophy (DMD)	June 22, 2023	Gene therapy
Skysona	$3 million per one-time dose	Bluebird Bio, Inc.	Cerebral Adrenoleukodystrophy (CALD)	September 16, 2022	Gene therapy
Zynteglo	$2.8 million per one-time dose	Bluebird Bio, Inc	Beta-thalassemia	September 16, 2022	Gene therapy
Zolgensma	$2.1 million per one-time dose	Novartis	Spinal Muscular Atrophy	May 24, 2019	Gene therapy
Myalept	$1.3 Million annually	Amryt Pharmaceuticals	Lipodystrophy/Leptin deficiency	February 24, 2014	Ultra rare disease
Danyelza	$1.2 million annually	Y-mAbs Therapeutics	Neuroblastoma	November 25, 2020	Ultra rare disease
Zokinvy	$1.2 million annually	Eiger Biopharmaceuticals	Progeria and Progeroid Laminopathies	November 20, 2020	Ultra rare disease
Kimmtrak	$1.1 million annually	Immunocore	Uveal Melanoma	January 25, 2022	Ultra rare disease
Luxturna	$850,000 per one-time dose	Spark Therapeutics	Biallelic RPE65-Mediated Inherited Retinal Disease	December 19, 2017	Gene therapy

In the past month, a new titleholder emerged for the most expensive drug in the US. Orchard Therapeutics, a subsidiary of Japan's Kyowa Kirin, now claims the top spot for producing the priciest medication.

Brand Name	Cost	Manufacturer	Indication	FDA approval date	Reason for high cost
Lenmeldy	$4.25 million per one-time treatment	Orchard Therapeutics, a unit of Kyowa Kirin	Metachromatic Leukodystrophy (MLD)	March 18, 2024	Gene therapy

There's an organization known as the Institute for Clinical and Economic Review (ICER), tasked with suggesting drug prices based on health economic assessments or Health Technology Assessments (HTAs). However, manufacturers often diverge from ICER's recommendations, frequently setting prices significantly higher. Take, for instance, a recent case where Merck, the drug's manufacturer, proposed a price nearly ten times higher than ICER's recommendation.

These exorbitant prices must ultimately be endorsed by health insurance companies or the Centers for Medicare & Medicaid Services (CMS). Consequently, it's the insurance companies or the Medicare & Medicaid program that bear the burden of these steep costs, as patients are unable to afford medications with such astronomical price tags.