Friday, March 29, 2024

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.

No comments:

Post a Comment