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Price comparison of most expensive drugs U.S. 2020

The number one most expensive drug, as of 2020, was Zolgensma or onasemnogene abeparvovec, a drug for the treatment of spinal muscular atrophy (SMA), with a list price of around 2.13 million U.S. dollars per year. Myalept was ranked the second expensive drug, used in the long-term treatment of urea cycle disorders. Myalept is marketed by Amryt Pharma. The most costly drugs worldwide based on wholesale acquisition cost (list price) are mostly orphan drugs for rare diseases.

What are orphan drugs?

Orphan drugs are pharmaceutical products for the treatment of so-called rare diseases. These are diseases with a very low prevalence within a population. Thus, it is not too appealing for companies to invest into R&D for drugs, which they potentially could sell only to a small number of patients. However, because persons afflicted by rare diseases should have the same right for care and treatment as persons with more common diseases, specific regulations were introduced over time. In 1983, the Orphan Drug Act was signed in the United States, for example.

The impact of the Orphan Drug Act

The Orphan Drug Act applies certain incentives for pharmaceutical companies to develop and market orphan drugs. This includes, for example, tax credits on clinical research, accelerated administrative procedures, and a 7-year market exclusivity after approval. These and other benefits significantly changed the level of interest in rare disease treatments. Given the fact that there are around 7,000 rare and ultra-rare diseases, drug manufacturers are increasingly entering this market with large potential. It becomes especially profitable when an already existing drug for common diseases also shows effects on a rare disease, or when an orphan drug emerges to be effective for other, more common diseases. In such cases, the orphan status of the drug helps companies to extend market exclusivity and add higher price tags, causing some critics to claim that the industry is gaming the system.

Most expensive drugs in the U.S. based on annual list price as of 2020

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Release date

August 2020



Survey time period

as of August 2020

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Statistics on "Orphan drugs"

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