Prescription drugs

Orphan Drugs Are Gaining Ground in the Pharma Market

Orphan drugs are steadily expanding their footprint in the global prescription market, as both sales and market share continue to rise. Estimates for 2021 put worldwide orphan drug sales at nearly $150 billion, equivalent to 14 percent of total prescription drug sales. By 2026, sales are expected to reach $225 billion and the orphan share in the market is expected to climb to 18 percent, underscoring how treatments for rare diseases are moving further into the commercial mainstream.

According to Evaluate’s Orphan Drugs Report 2025, this growth is set to continue, with orphan medicines projected to represent around one-fifth of worldwide prescription drug sales by 2030, a share that the report says has roughly doubled over the last decade. Evaluate also points out that orphan drugs are still growing faster than non-orphan medicines, but that this gap is shrinking. A sign the segment is maturing, even as it continues to be a major contributor to the pharma industry’s revenue growth.

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This chart shows the estimated and projected orphan drug sales worldwide and share of total prescription drug market (2021-2026).

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Leading orphan drugs in development based on net present value 2025
Prescription drugs spending in the U.S. by payer 2010-2024
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Boehringer Ingelheim's top prescription drugs 2023-2024
Time between orphan drugs approval and patient access in Europe 2025, by country
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Projection of top orphan drugs by global revenue 2030
Share of new orphan drugs available in Europe in 2025, by country

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