It is projected that in 2028 Vertex will generate 94 percent of its total sales from orphan drugs. The development of orphan drugs are incentivized in the United States through the Orphan Drug Act of 1983.
Ophan drugs overview
Orphan drugs are pharmaceutical products designed to help treat, prevent and even diagnose extremely rare disorders and disease. A rare disease and disorder is defined as an illness that affects 1 in 2,000 people (in Europe) or one that affects less than 200,000 individuals (in the U.S.). The top orphan drug based on revenue in 2021 was Imbruvica. It is a drug used to treat certain types of cancers. It is projected that by 2028 the top orphan drug, by revenue, will be Darzalex.
The orphan drug market
The number of orphan drugs on the market in the U.S. had increased dramatically over the last two decades. There were over 370 drugs with an orphan distinction approved in the U.S. in 2023. Orphan drugs are typically significantly more expensive than non-orphan drugs. The average per patient cost of an orphan drug in the U.S. is about 4.5 times as expensive as a non-orphan drug. Despite the cost and the number of drugs available, the global orphan drug market has experienced varying degrees of growth. Data indicates that, while there will still be revenue growth in the future, the percentage of growth will begin to decline or remain stable.
Leading pharmaceutical companies worldwide by projected orphan drug sales share in 2028
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Evaluate. (April 23, 2024). Leading pharmaceutical companies worldwide by projected orphan drug sales share in 2028 [Graph]. In Statista. Retrieved November 10, 2024, from https://www-statista-com.ezproxy.canberra.edu.au/statistics/373373/top-companies-based-on-global-orphan-drug-sales-share/
Evaluate. "Leading pharmaceutical companies worldwide by projected orphan drug sales share in 2028." Chart. April 23, 2024. Statista. Accessed November 10, 2024. https://www-statista-com.ezproxy.canberra.edu.au/statistics/373373/top-companies-based-on-global-orphan-drug-sales-share/
Evaluate. (2024). Leading pharmaceutical companies worldwide by projected orphan drug sales share in 2028. Statista. Statista Inc.. Accessed: November 10, 2024. https://www-statista-com.ezproxy.canberra.edu.au/statistics/373373/top-companies-based-on-global-orphan-drug-sales-share/
Evaluate. "Leading Pharmaceutical Companies Worldwide by Projected Orphan Drug Sales Share in 2028." Statista, Statista Inc., 23 Apr 2024, https://www-statista-com.ezproxy.canberra.edu.au/statistics/373373/top-companies-based-on-global-orphan-drug-sales-share/
Evaluate, Leading pharmaceutical companies worldwide by projected orphan drug sales share in 2028 Statista, https://www-statista-com.ezproxy.canberra.edu.au/statistics/373373/top-companies-based-on-global-orphan-drug-sales-share/ (last visited November 10, 2024)
Leading pharmaceutical companies worldwide by projected orphan drug sales share in 2028 [Graph], Evaluate, April 23, 2024. [Online]. Available: https://www-statista-com.ezproxy.canberra.edu.au/statistics/373373/top-companies-based-on-global-orphan-drug-sales-share/