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Orphan drugs

Statistics report about the orphan drugs industry and market

This report presents key facts and figures about the global and U.S. orphan drugs market. Orphan drugs are medications which are used for so called rare diseases, a group of conditions which affects a very small group of a population. This report provides a closer look on top companies, leading products, research and development, etc.

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Table of contents

Market overview

4
  • Basic Statistic Projection of global revenue for orphan prescription drugs 2016-2030
  • Premium Statistic Price comparison of most expensive drugs U.S. 2025
  • Basic Statistic Worldwide share of orphan drug sales by therapy category 2030
  • Basic Statistic Global rare disease diagnostics market size 2024 and 2029

Companies

4
  • Premium Statistic Major 50 pharmaceutical companies - Rx sales and R&D spending 2024
  • Premium Statistic Top global companies by orphan drug revenues 2024
  • Premium Statistic Top 10 companies by global revenue from orphan drugs 2030
  • Premium Statistic Orphan drugs sales share among top pharma companies 2030 forecast

Products

5
  • Premium Statistic Projection of top orphan drugs by global revenue 2030
  • Premium Statistic Number of orphan designations accepted in the U.S. 2003-2025
  • Basic Statistic Oncology or orphan drug share in approved new active substances 2015-2023
  • Basic Statistic Share of new orphan drugs available in Europe in 2025, by country
  • Basic Statistic Time between orphan drug approval and patient access in Europe 2021-2024, by country

Research and development

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  • Premium Statistic Total global pharmaceutical R&D spending 2016-2030
  • Premium Statistic Total rare diseases funding by the National Institutes for Health 2013-2024
  • Basic Statistic Leading orphan drugs in development based on net present value 2025
  • Premium Statistic Orphan vs. non-orphan pipeline sales forecast worldwide 2025-2030
  • Basic Statistic Share of orphan medicines in R&D pipeline worldwide 2023, by phase
  • Premium Statistic U.S. development Phase success rates for rare and common disease drugs 2011-2020

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 2024, by gender
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