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. Rare diseases
    • Prevalence of selected rare diseases worldwide 2017

    • 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 2020, by gender

  • 2. Market overview
    • Projection of global revenue for orphan prescription drugs 2012-2026

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

    • Price comparison of most expensive drugs U.S. 2020

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

    • Global cell and gene therapy market share by therapy class 2019 & 2025

    • Global rare disease diagnostics market size 2018 vs. 2025 projection

  • 3. 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

    • US and European private VC-backed orphan biopharma company M&A values 2014-2019

  • 4. 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. 2003-2020

    • Oncology or orphan drug share in approved new active substances 2015-2019

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

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

  • 5. Research and development
    • Total global pharmaceutical R&D spending 2012-2026

    • Total rare diseases funding by the National Institutes for Health 2013-2020

    • Leading orphan drugs in development based on net present value 2020

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

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

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