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Increased Government Support, Product Launches, and Huge Unmet Needs Drive the Global Orphan Drugs Market

Published: January 2018


  • Code: MCP-7443
  • Pages: 436
  • Tables: 49
  • Companies: 140


The global market for Orphan Drugs is projected to reach US$225 billion by 2024, against the backdrop of rare diseases assuming new importance in public healthcare. Other major factors driving growth include recent developments in genomics and bioinformatics; continuous research focus on molecular genomic diagnosis of rare diseases; rising incidence of rare medical conditions, growing awareness and better diagnosis; and regulatory incentives to encourage the R&D and development of orphan drugs. The huge unmet needs and the untapped business opportunity are encouraging big pharmaceutical companies to focus on orphan drug development. The United States represents the largest and the fastest growing market worldwide, supported by financial incentives and seven year market exclusivity period offered to manufacturers by the US FDA. Additionally, the premium prices and full reimbursements commanded by orphan drugs drive growth in the market. Europe closely follows the U.S. due to the adoption of regulations similar to the US FDA. In Japan, introduction of the Orphan Drug Development Program enabled the entry of a number of orphan drug developers, spurring the number of orphan drug approvals. In Asia-Pacific, population growth and the increasing burden of rare genetic diseases, is encouraging the regulation of rare diseases and orphan drugs on par with the developed economies.  

    Summary of Findings

  • The Market Remains Challenged by the High Cost of Drug Development for Rare Diseases Due to Factors such as Developmental Challenges Resulting from Small Population Size, Difficulty in Enrolling Patients for Clinical Trials, and Unclear Clinical Endpoints
  • Strategies such as Financial Support and Incentives, Tax Credits and Extended Market Exclusivity to Help Orphan Drug Development Efforts
  • Growing Regulatory Flexibility and the Resulting Ability to Expand Indications for Orphan Drugs to Benefit Market Growth
  • Legislative Benefits for Orphan Drug Development Attract Major Pharmaceutical Companies
  • Innovations in Pre-Symptomatic Screening and Early Diagnosis to Accelerate the Pressure to Develop New Drugs to Treat Orphan Diseases

    Timeline for Analysis

  • Market Estimates and Forecasts for 2016-2024
  • Historic Review 2011-2015

    Geographic Markets Analyzed

  • US, Japan, Europe, and Rest of World

    Market Segmentation & Classification

  • Segments:
    • Biologics, and Non-Biologics


  • AbbVie, Inc.
  • Actelion Pharmaceuticals Ltd.
  • Alexion Pharmaceuticals, Inc.
  • Amgen, Inc.
  • AstraZeneca Plc
  • Bayer AG


  • Review of Industry/Market Structure
  • Analysis of Trends & Drivers
  • Insights on Macro Market Scenarios
  • Market SWOT Analysis
  • Latent Demand Forecasts & Projections
  • Comprehensive Geographic Market Analysis
  • Presentation Ready Facts & Statistical Data Findings
  • Coverage of Major/Niche Players, Market Shares & Competition
  • Coverage of major Company/Technology/Product/Financial Stories
  • Extensive Product/Service/End-Use/Technology Coverage Where Applicable

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