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Global Orphan Drug Market

Global Orphan Drug Market

The treatment of rare diseases requires the use of specialized drugs. Patient populations for rare diseases are most often quite small, consequently the products treating them earn the designation orphan drugs. These drugs must undergo the research, development, and clinical trials that all drugs do, yet the investment into the development is great, while the promise for recouping financial costs is not guaranteed. This study covers the spectrum of orphan diseases and highlights drugs already marketed as well as those in the development pipeline for them. Regional highlights, market opportunities, companies to watch, and legislative timelines are provided. This snapshot captures the global orphan drug landscape as of June 2016.


  • Key Methodology
  • Executive Summary
    • Scope and Segmentation
    • Key Questions this Snapshot will Address
    • Key Takeaways-Orphan Drug Market
    • Executive Summary-Market Snapshot
  • Commercialization Analysis
    • 8 Big Market Themes for Orphan Drugs
    • A Vibrant Ecosystem
    • Cancer Therapies Remain the Largest Orphan Drug Segment
    • Global Overview and Analysis
      • Table Orphan Drug Market: Regional Highlights, Global, 2000-2016
    • Regional Highlights
    • Game-changing Companies
    • Companies to Watch
    • New Market Opportunities-Orphan Drugs
    • Key 2016 Predictions-Orphan Drug Market
    • Timeline of Orphan Drug Legislation
    • Disruptive Technologies and Advances in this Field
    • Overall Environmental Impact Factors
    • Orphan Drug Payer Environment
    • A Snapshot of Global Drug Price Control Methods
  • Conclusion and Take Home Points
    • Key Conclusions
    • The Last Word-3 Big Predictions
  • Appendix A-Orphan Diseases with FDA-approved Therapies
    • Orphan Diseases with FDA-approved Therapies
  • Appendix B-Pipeline Analyses for Select Rare Diseases
    • Top Indications by Activity and Disease Overview-Blood and Lymphatic System Diseases
      • Table Orphan Drug Market: Blood and Lymphatic System Diseases-Top Indications, US, 2016
    • SCD-Orphan Drugs on the Market
    • SCD-Orphan Drugs in Development
    • MDS-Orphan Drugs on the Market
    • MDS-Orphan Drugs in Development
    • TTP-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Cardiovascular Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Cardiovascular Diseases-Top Indications, US, 2016
    • PAH-Orphan Drugs on the Market
    • PAH-Orphan Drugs in Development
    • Calciphylaxis-Orphan Drugs in Development and on the Market
  • Pipeline Analyses for Select Rare Diseases-Lung Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Lung Diseases-Top Indications, US, 2016
    • CF-Orphan Drugs on the Market
    • CF-Orphan Drugs in Development
    • IPF-Orphan Drugs on the Market
    • IPF-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Congenital and Genetic Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Congenital and Genetic Diseases-Top Indications, US, 2016
    • Fragile X Syndrome-Orphan Drugs in Development
    • HIBM-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Connective Tissue/Skin Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Connective Tissue/Skin Diseases-Top Indications, US, 2016
    • SSc-Orphan Drugs in Development
    • EB-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Digestive/Nutritional Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Digestive/Nutritional Diseases-Top Indications, US, 2016
    • Ped CD-Orphan Drugs on the Market
    • Ped CD-Orphan Drugs in Development
    • Ped UC-Orphan Drugs on the Market
    • Ped UC-Orphan Drugs in Development
    • Pouchitis-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Endocrine Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Endocrine Diseases-Top Indications, US, 2016
    • T1D with Residual Beta-cell Function-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Exposure/Toxicity
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Exposure/Toxicity-Top Indications, US, 2016
    • ARS-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Eye Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Eye Diseases-Top Indications, US, 2016
    • RP-Orphan Drugs in Development
    • Stargardt Disease-Orphan Drugs in Development
    • Uveitis-Orphan Drugs on the Market
    • Uveitis-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Immunological/Inflammatory Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Immunological/Inflammatory Diseases-Top Indications, US, 2016
    • Behcet's Disease-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Infectious/Parasitic Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Infectious/Parasitic Diseases-Top Indications, US, 2016
    • CMV Infection-Orphan Drugs on the Market
    • CMV Infection-Orphan Drugs in Development
    • Malaria-Orphan Drugs on the Market
    • Malaria-Orphan Drugs in Development
    • Leishmaniasis-Orphan Drugs on the Market
    • Leishmaniasis-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Metabolic Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Metabolic Diseases-Top Indications, US, 2016
    • MPS-Orphan Drugs on the Market
    • MPS-Orphan Drugs in Development
    • Acromegaly-Orphan Drugs on the Market
    • Acromegaly-Orphan Drugs in Development
    • Urea Cycle Disorders-Orphan Drugs on the Market
    • Urea Cycle Disorders-Orphan Drugs in Development
    • NPC Disease-Orphan Drugs on the Market
    • NPC Disease-Orphan Drugs in Development
    • Pompe Disease-Orphan Drugs on the Market
    • Pompe Disease-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Musculoskeletal Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Musculoskeletal Diseases-Top Indications, US, 2016
    • DMD-Orphan Drugs in Development
    • SMA-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Neurologic Diseases
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Neurologic Diseases-Top Indications, US, 2015-2022
    • ALS-Orphan Drugs in Development
    • HD-Orphan Drugs on the Market
    • HD-Orphan Drugs in Development
    • FA-Orphan Drugs in Development
  • Pipeline Analyses for Select Rare Diseases-Rare Cancers
    • Top Indications by Activity and Disease Overview
      • Table Orphan Drug Market: Rare Cancers-Top Indications, US, 2015-2022
    • Pancreatic Cancer-Orphan Drugs on the Market
    • Pancreatic Cancer-Orphan Drugs in Development
    • Glioma-Orphan Drugs on the Market
    • Glioma-Orphan Drugs in Development
    • AML-Orphan Drugs on the Market
    • AML-Orphan Drugs in Development

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