Orphan drugs - Statistics & Facts
Key insights
- Projected revenue for orphan drugs in 2028
- 300 bn USD
Detailed statistics
Projection of global revenue for orphan prescription drugs 2016-2028
- Global growth for prescription orphan drugs in 2018
- 11.1%
Detailed statistics
Annual growth in global revenue for prescribed orphan drugs 2011-2024
- Average annual cost of orphan drugs per patient in 2018
- 150,854 USD
Detailed statistics
Annual average cost of orphan drugs per patient 2014-2018
Editor’s Picks Current statistics on this topic
Current statistics on this topic
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Rare diseases
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- Basic Statistic Prevalence of selected rare diseases worldwide 2017
- Basic Statistic Overview of rare diseases worldwide and in the U.S. as of 2016
- Premium Statistic Impact of medicines on life expectancy for select rare diseases worldwide as of 2017
- Basic Statistic Distribution of U.S. patients with rare factor deficiencies as of 2023, by gender
Rare diseases
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Basic Statistic
Prevalence of selected rare diseases worldwide 2017
Prevalence of selected rare diseases worldwide 2017
Prevalence rate of selected rare diseases worldwide as of 2017 (per 100,000 population)*
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Basic Statistic
Overview of rare diseases worldwide and in the U.S. as of 2016
Overview of rare diseases worldwide and in the U.S. as of 2016
Number of rare diseases, disease patients and treatments worldwide and in the U.S. as of 2016
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Premium Statistic
Impact of medicines on life expectancy for select rare diseases worldwide as of 2017
Impact of medicines on life expectancy for select rare diseases worldwide as of 2017
Life expectancy for individuals with select rare diseases before available treatment and now worldwide as of 2017
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Basic Statistic
Distribution of U.S. patients with rare factor deficiencies as of 2023, by gender
Distribution of U.S. patients with rare factor deficiencies as of 2023, by gender
Distribution of patients in the U.S. with rare factor deficiencies as of 2023, by gender*
Market overview
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- Basic Statistic Projection of global revenue for orphan prescription drugs 2016-2028
- Premium Statistic Price comparison of most expensive drugs U.S. 2023
- Basic Statistic Worldwide share of orphan drug sales by therapy category 2022 vs. 2028
- Basic Statistic Global rare disease diagnostics market size 2019 and 2030
- Basic Statistic Global rare disease diagnostics market size 2019 and 2030 by therapy area
Market overview
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Basic Statistic
Projection of global revenue for orphan prescription drugs 2016-2028
Projection of global revenue for orphan prescription drugs 2016-2028
Global prescription orphan drug sales from 2016 to 2028 (in billion U.S. dollars)
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Premium Statistic
Price comparison of most expensive drugs U.S. 2023
Price comparison of most expensive drugs U.S. 2023
Most expensive drugs in the United States as of 2023
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Basic Statistic
Worldwide share of orphan drug sales by therapy category 2022 vs. 2028
Worldwide share of orphan drug sales by therapy category 2022 vs. 2028
Share of worldwide orphan drug sales by top therapy category in 2022 and a forecast for 2028
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Basic Statistic
Global rare disease diagnostics market size 2019 and 2030
Global rare disease diagnostics market size 2019 and 2030
Global rare disease diagnostics market in 2019 and a forecast for 2030 (in billion U.S. dollars)
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Basic Statistic
Global rare disease diagnostics market size 2019 and 2030 by therapy area
Global rare disease diagnostics market size 2019 and 2030 by therapy area
Global rare disease diagnostics market size in 2019 and a forecast for 2030, by therapy area (in billion U.S. dollars)
Companies
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- Premium Statistic Top 50 pharmaceutical companies - Rx sales and R&D spending 2022
- Premium Statistic Top global companies by orphan drug revenues 2022
- Premium Statistic Top 10 companies by global revenue from orphan drugs 2028
- Premium Statistic Orphan drugs sales share among top pharma companies 2022 vs. 2028
Companies
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Premium Statistic
Top 50 pharmaceutical companies - Rx sales and R&D spending 2022
Top 50 pharmaceutical companies - Rx sales and R&D spending 2022
Leading 50 global pharmaceutical companies by prescription sales and R&D spending in 2022 (in billion U.S. dollars)
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Premium Statistic
Top global companies by orphan drug revenues 2022
Top global companies by orphan drug revenues 2022
Leading pharmaceutical companies by orphan drug revenue worldwide in 2022 (in billion U.S. dollars)
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Premium Statistic
Top 10 companies by global revenue from orphan drugs 2028
Top 10 companies by global revenue from orphan drugs 2028
Forecast of leading 10 pharmaceutical companies by global orphan drug revenue in 2028 (in billion U.S. dollars)
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Premium Statistic
Orphan drugs sales share among top pharma companies 2022 vs. 2028
Orphan drugs sales share among top pharma companies 2022 vs. 2028
Leading pharmaceutical companies worldwide by orphan drug sales share in 2028 compared to 2022
Products
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- Premium Statistic Projection of top orphan drugs by global revenue 2028
- Premium Statistic Number of orphan designations accepted in the U.S. 2003-2023
- Basic Statistic Oncology or orphan drug share in approved new active substances 2015-2021
- Premium Statistic FDA approved orphan drugs with non-orphan indications 1983-2018
- Basic Statistic Share of new orphan drugs available in Europe in 2023, by country
- Basic Statistic Time between orphan drugs approval and patient access in Europe 2023, by country
Products
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Premium Statistic
Projection of top orphan drugs by global revenue 2028
Projection of top orphan drugs by global revenue 2028
Projected leading 10 orphan drugs worldwide by revenue in 2028 (in billion U.S. dollars)
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Premium Statistic
Number of orphan designations accepted in the U.S. 2003-2023
Number of orphan designations accepted in the U.S. 2003-2023
Annual number of accepted orphan designations in the United States from 2003 to 2023
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Basic Statistic
Oncology or orphan drug share in approved new active substances 2015-2021
Oncology or orphan drug share in approved new active substances 2015-2021
Share of orphan or oncology drugs among approved new active substances (NAS) in select developed countries from 2015 to 2021
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Premium Statistic
FDA approved orphan drugs with non-orphan indications 1983-2018
FDA approved orphan drugs with non-orphan indications 1983-2018
Percentage of FDA-approved orphan drugs with additional non-orphan indications between 1983 and 2018
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Basic Statistic
Share of new orphan drugs available in Europe in 2023, by country
Share of new orphan drugs available in Europe in 2023, by country
Percentage of orphan drugs approved by the EMA available to patients in Europe as of 2023, by country
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Basic Statistic
Time between orphan drugs approval and patient access in Europe 2023, by country
Time between orphan drugs approval and patient access in Europe 2023, by country
Average time between marketing authorization and patient access for orphan drugs in Europe as of 2023, by country (in days)
Research and development
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- Premium Statistic Total global pharmaceutical R&D spending 2014-2028
- Premium Statistic Total rare diseases funding by the National Institutes for Health 2013-2024
- Basic Statistic Orphan and non-orphan drugs in the pipeline worldwide 2021
- Basic Statistic Leading orphan drugs in development based on net present value 2023
- Basic Statistic Orphan vs. non-orphan pipeline sales forecast worldwide 2023-2028
- Premium Statistic U.S. development Phase success rates for rare and common disease drugs 2011-2020
Research and development
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Premium Statistic
Total global pharmaceutical R&D spending 2014-2028
Total global pharmaceutical R&D spending 2014-2028
Total global spending on pharmaceutical research and development from 2014 to 2028 (in billion U.S. dollars)
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Premium Statistic
Total rare diseases funding by the National Institutes for Health 2013-2024
Total rare diseases funding by the National Institutes for Health 2013-2024
Total rare diseases funding by the National Institutes for Health (NIH) from FY 2013 to FY 2024 (in million U.S. dollars)
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Basic Statistic
Orphan and non-orphan drugs in the pipeline worldwide 2021
Orphan and non-orphan drugs in the pipeline worldwide 2021
Share of orphan and non-orphan drugs in the global drug pipeline in 2021
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Basic Statistic
Leading orphan drugs in development based on net present value 2023
Leading orphan drugs in development based on net present value 2023
Leading orphan drugs in development (phase III/filed) by NPV as of 2023 (in billion U.S. dollars)
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Basic Statistic
Orphan vs. non-orphan pipeline sales forecast worldwide 2023-2028
Orphan vs. non-orphan pipeline sales forecast worldwide 2023-2028
Pipeline sales forecast for orphan and non-orphan drugs worldwide from 2023 to 2028 (in billion U.S. dollars)
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Premium Statistic
U.S. development Phase success rates for rare and common disease drugs 2011-2020
U.S. development Phase success rates for rare and common disease drugs 2011-2020
Probability of success for new rare disease and high prevalence disease drugs in the U.S. by development phase from 2011 to 2020
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