Global Markets for Orphan Drugs

Jun 2013| PHM038D| BCC Publishing

Report Highlights

The global market for orphan drugs reached nearly $86.1 billion in 2012. The market is expected to reach $112.1 billion in 2017 for a compound annual growth rate (CAGR) of 5.4%.

Report Includes

  • An overview of orphan drug criteria, the history of the Orphan Drug Act and its influence, advantages inherent in orphan drug designation, and future growth trends
  • Analyses of global market trends, with data from 2011 and 2012, and projections of compound annual growth rates (CAGRs) through 2017
  • Detailed discussion of successful orphan products
  • Detailed analysis of blockbuster orphan drugs
  • The rising influence of pharmacogenomics in the orphan drug market
  • A discussion of specific rare diseases for which orphan drugs exist
  • Analysis of the most promising therapeutic areas in the industry and leading orphan drugs in these areas
  • An outline of legislation and regulation, including public and private agencies involved in support and development
  • Technology trends analysis within the orphan drug market
  • Comprehensive company profiles of major players in the industry.


The scope of this study includes orphan drugs in pharmaceutical markets worldwide.  BCC analyzes each market and its applications, regulatory environment, technology, market projections and market shares.  Technological issues include the latest trends and developments.  The emerging market for orphan drugs includes countries such as India, China, Japan, Korea, Taiwan, Canada, Africa, Australia and New Zealand.

Analyst Credentials

Shalini Shahani Dewan focuses on pharmaceuticals and biotechnology and has been a BCC Research contributor since 2002 as both an analyst and project manager. She has explored a wide range of topics and companies, including working for Johnson & Johnson doing market surveillance. She has an undergraduate degree in pharmacy and master's degree in medicinal chemistry. She resides in the Bay Area.

Table of Contents & Pricing

All reports provided in PDF format. For shared licensing options (5+ Users), please call a representative at (+1) 781-489-7301 or contact us at
Title/Chapter NamePagesPrice Member Price
Full Report: Global Markets for Orphan Drugs182 $2,750 Free
Chapter- 1: INTRODUCTION3Free
Chapter- 2: EXECUTIVE SUMMARY3$250Free
Chapter- 3: OVERVIEW23$348Free
Chapter- 8: PATENT ANALYSIS7$106Free
Chapter- 9: CURRENT SITUATION4$60Free
Chapter- 10: COMPANY PROFILES21$317Free
Published - May-2010| Analyst - Syamala Ariyanchira| Code - PHM038C

Report Highlights

  • Worldwide, the orphan drug market reached $84.9 billion in 2009. The market is expected to grow at a compound annual growth rate (CAGR) of nearly 6% to reach $112.1 billion by 2014. The U.S. accounted for 51% of the market in 2009 and is expected to grow at a CAGR of 8.9% to reach $65.9 billion by 2014.
  • Biologic drugs accounted for a major share (64.3%) of the orphan drug market with sales of $54.6 billion in 2009, up from $51.4 billion in 2008.  The size of the biologic orphan drug market is projected to grow at a 6.9% CAGR to reach $76.2 billion by 2014.
  • Orphan drugs for the cancer sector generated the largest amount of revenues, $30.6 billion in 2009, accounting for 36% of the market.  Revenues for cancer-related orphan drugs are expected to grow at a CAGR of 10% to reach $49.7 billion in 2014.
Published - Sep-2007| Analyst - Syamala Ariyanchira| Code - PHM038B

Report Highlights

  • The global orphan drugs market reached $58.7 billion in 2006, growing by 8% from $54.5 billion in 2005. It is expected to grow at a compounded annual growth rate (CAGR) of 7% to reach $81.8 billion by 2011.
  • Regionally, U.S. revenues in the market grew to $32.5 billion accounting for 55% of the market in 2006. The U.S. market is expected to grow at a CAGR of 8% to reach $47.8 billion by 2011.
  • Biologic drugs account for over 60% of the orphan drug market with sales of $35.3 billion in 2006, up from $30.2 billion in 2005. The market size of biologic orphan drugs is projected to grow at a 9% CAGR to reach $53.4 billion by 2011.
Published - Feb-2004| Analyst - Lynn Gray| Code - PHM038A

Report Highlights

  • The total worldwide market for orphan drugs, including sales for nonorphan applications, exceeded $28 billion in 2003.
  • This market is expected to rise at an AAGR (average annual growth rate) of 9.1% to reach $43.6 billion in 2008.
  • As a percentage of overall global drug sales, orphans are expected to increase their share from 7.0% in 2003 to 7.5% in 2008, as growth of the orphan market outpaces that of the total market.
  • As of mid-2003, 221 orphan drugs had been developed for the treatment of rare diseases affecting about 11 million patients.
  • Some orphan products have sales in excess of $1 billion annually.

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