
Matej Mikulic
Research expert covering health, pharma & medtech

Detailed statistics
Projection of global revenue for orphan prescription drugs 2016-2030

Detailed statistics
Annual growth in global revenue for prescribed orphan drugs 2011-2024

Detailed statistics
Annual average cost of orphan drugs per patient 2014-2018

Projection of global revenue for orphan prescription drugs 2016-2030
Global prescription orphan drug sales from 2016 to 2030 (in billion U.S. dollars)

Price comparison of most expensive drugs U.S. 2025
Most expensive drugs in the United States as of 2025 (in million U.S. dollars)

Worldwide share of orphan drug sales by therapy category 2030
Projected share of worldwide orphan drug sales by top therapy category in 2030

Global rare disease diagnostics market size 2024 and 2029
Global rare disease diagnostics market in 2024 and a forecast for 2029 (in billion U.S. dollars)

Major 50 pharmaceutical companies - Rx sales and R&D spending 2024
Leading 50 global pharmaceutical companies by prescription sales and R&D spending in 2024 (in billion U.S. dollars)

Top global companies by orphan drug revenues 2024
Leading pharmaceutical companies by orphan drug revenue worldwide in 2024 (in billion U.S. dollars)

Top 10 companies by global revenue from orphan drugs 2030
Forecast of leading 10 pharmaceutical companies by global orphan drug revenue in 2030 (in billion U.S. dollars)

Orphan drugs sales share among top pharma companies 2030 forecast
Leading pharmaceutical companies worldwide by projected orphan drug sales share in 2030

Projection of top orphan drugs by global revenue 2030
Projected leading 10 orphan drugs worldwide by revenue in 2030 (in billion U.S. dollars)

Number of orphan designations accepted in the U.S. 2003-2025
Annual number of accepted orphan designations in the United States from 2003 to 2025

Oncology or orphan drug share in approved new active substances 2015-2023
Share of orphan or oncology drugs among approved new active substances (NAS) in select developed countries from 2015 to 2023

Share of new orphan drugs available in Europe in 2025, by country
Percentage of new orphan drugs approved by the EMA available to patients in Europe as of 2025, by country

Time between orphan drug approval and patient access in Europe 2021-2024, by country
Average time between marketing authorization and patient access for orphan drugs in Europe from 2021 to 2024, by country (in days)

Total global pharmaceutical R&D spending 2016-2030
Total global spending on pharmaceutical research and development from 2016 to 2030 (in billion U.S. dollars)

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)

Leading orphan drugs in development based on net present value 2025
Leading orphan drugs in development (phase III/filed) by NPV as of 2025 (in billion U.S. dollars)

Orphan vs. non-orphan pipeline sales forecast worldwide 2025-2030
Pipeline sales forecast for orphan and non-orphan drugs worldwide from 2025 to 2030 (in billion U.S. dollars)

Share of orphan medicines in R&D pipeline worldwide 2023, by phase
Share of orphan medicines in the R&D pipeline worldwide as of 2023, by development phase

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

Prevalence of selected rare diseases worldwide 2017
Prevalence rate of selected rare diseases worldwide as of 2017 (per 100,000 population)*

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

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

Distribution of U.S. patients with rare factor deficiencies as of 2024, by gender
Distribution of patients in the U.S. with rare factor deficiencies as of 2024, by gender*
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