Orphan drugs

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Statista dossier about the orphan drugs industry and market

This dossier 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 document provides a closer look on top companies, top products, research and development, treatment costs, etc.

Table of contents

  • 1. Market overview
    • Projection of global revenue for orphan prescription drugs 2010-2024

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

    • Annual average cost of orphan drugs per patient 2014-2018

    • Price comparison of the 10 most expensive drugs as of 2018

    • Orphan vs. non-orphan drugs median cost per patient per year 2014-2018

  • 2. Companies
    • Top 50 pharmaceutical companies - Rx sales and R&D spending 2019

    • Top global companies by orphan drug revenues 2018

    • Top 10 companies by global revenue from orphan drugs 2024

    • Market share of top 20 companies for orphan drugs worldwide 2018 and 2024

  • 3. Products
    • Top global orphan drugs by revenue 2018

    • Projection of top 10 orphan drugs by global revenue 2024

    • Number of orphan designations accepted in the U.S. 2003-2018

    • Percentage of select orphan drug US sales by non/orphan use 2017

    • FDA approved orphan drugs with non-orphan indications 1983-2018

  • 4. Research and development
    • Total global pharmaceutical R&D spending 2010-2024

    • Leading orphan drugs in development based on net present value 2019

    • Global therapeutic pipeline for non-oncology orphan drug programs by company 2019

    • Time from orphan drug design to final exclusivity 2018, by current status

    • U.S. development Phase success rates for rare and common disease drugs 2006-2015

  • 5. Rare diseases
    • Overview of rare diseases worldwide and in the U.S. as of 2016

    • Impact of medicines on life expectancy for select rare diseases worldwide as of 2017

    • Distribution of U.S. patients with rare factor deficiencies as of 2019, by gender

    • U.S. rare disease patient feelings as of 2015

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