Global Markets for Orphan Drugs

May 2010| PHM038C| BCC Publishing

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.

The orphan drug industry has come a long way since its implementation, leaving many noticeable benchmarks along the way as the U.S. Orphan Drug Act (ODA), which was implemented in 1983, entered its 26th year in 2009. The purpose of this report is to analyze the orphan drug industry and provide market, technology, and revenue forecasts for the period 2009 to 2014. The report also analyzes the influence of the orphan drug industry in the development of the biotechnology industry.

This BCC report provides an in-depth look at orphan drugs that had sales of more than $200 million in 2009. Future growth prospects of these drugs as well as the therapeutic areas of these drugs in general have been analyzed based on the market dynamics and regulatory factors. Life stories of blockbuster orphan drugs are tracked and analyzed in detail, recognizing various factors that played key roles in their success. Regional analysis of the industry in terms of current and future trends of the orphan drug market in important regions has been presented. The report also provides revenues of leading orphan products, identifies key market players with significant market shares, analyzes current and future strategies of these companies that will have an impact in the market, and, finally, analyzes the rare diseases that are being addressed by the industry and the broad therapeutic areas that these diseases represent and their future growth trends.

This report also analyzes the current and possible future legislation affecting development and marketing of orphan drugs in the U.S. and other countries and examines the influence of the Orphan Drug Act in the development of the biotech industry. The impact of developments in pharmacogenomics in the orphan drug industry is also analyzed. The rising influence of ODA in business strategy development of pharmaceutical companies, which is driving the emergence of innovative business strategies and approaches, is also analyzed in detail. Issues such as the impact of market exclusivity in drug pricing, affordability, and healthcare insurance are also analyzed. Finally, technology trends analysis has been presented in the report, identifying how the most prevalent technologies in the market have changed over the past decade with the advent of biotech companies and future growth trends.
BCC published comprehensive reports on the orphan drug industry in 2004, and in 2007. The emerging trends identified in 2007 have become more established during the past 3 years. Moreover, new trends are emerging as big pharma realizes the failing of blockbuster business model. The role of biotechnology industry in the pharmaceutical market has become more prominent, which is reflected in the orphan drug sector as well.
The changing regulatory landscape of the industry and its effect in driving the growth is obvious and has been pointed out in the earlier BCC report, PHM038B, published in 2007. During the past 3 years, the role of regulators in deciding the future of the sector has become more prominent. Globally, awareness about rare diseases has increased, while the importance of ODA has received rising recognition. Following the example of the U.S. Food and Drug Administration’s (FDA’s) Orphan Drug Act, similar legislation — such as Singapore’s Orphan Drug Exemption to the Medicines Act and Japan’s Orphan Drug Amendment to the Pharmaceutical Affairs Law — has been developed by many other countriesThis has opened new markets for companies involved in orphan drug development. The number of orphan drugs that have become blockbusters has also gone up. In 2006, there were 19 orphan drugs with sales of more than $1 billion. In 2009, this increased to 27.
The change in the attitude of the pharmaceutical industry towards orphan drugs is becoming more prominent. Business deals involving big pharmaceutical companies and small companies with a pipeline of orphan drug designations have increased lately, indicating the rising importance of orphan-drug-based business strategies in the pharmaceutical industry. Such new trends are changing the landscape of the entire pharmaceutical industry. This report analyzes the current and emerging trends in the market. The report also analyzes affordability issues. Rising prices of orphan drugs and the reluctance of insurance agencies to cover orphan drugs that are priced extremely high are two issues being taken seriously by governments and patient support groups. Various business models are being suggested and being designed to tackle these issues. This report analyzes these rising business models in detail.   
This study provides insight on successful regulatory strategies that helped orphan drugs become blockbusters. Valuable insights on various business models that take advantage of ODA incentives are also provided. In addition, emerging regulatory strategies in key markets for improving the affordability of orphan drugs by patients are included.
The key therapeutic categories that are getting high attention from the developers and the rare indications within these therapeutic areas that have benefited from ODA are identified. Business strategies that helped big pharmaceutical companies as well as biotech companies to enter the market as well as maintain their market shares have been identified. These detailed analyses are targeted to help strategy developers and decision makers of biotech and pharmaceutical companies. It also gives insights into the incentives that have been attractive for different segments of the industry. This is especially suitable for legislators involved in government policy development programs. Technology trend analysis gives an overview of successful technologies, which are helpful for research managers in charge of project identification and development. 
This report contains:
  • An overview of orphan drug criteria, the history of the Orphan Drug Act and its influence and advantages inherent in orphan drug designation and future growth trends
  • Detailed discussion of successful orphan products
  • Global sales projections through 2014 for as many as 57 orphan drugs with sales exceeding $200 million
  • Key players in the market and their market shares
  • Detailed analysis of blockbuster orphan drugs
  • Analysis of the Orphan Drug Act and its influence in the growth of the biotech industry
  • 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 therapeutic 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
  • In-depth analysis of leading companies and their successful strategies within the orphan drug market segment.
The base year of this study is 2009. The market size reflects the global revenues of drugs with orphan drugs status in the United States that have generated more than $200 million in revenues in 2009. Drugs with market exclusivity active during the base year as well as those with expired market exclusivities in the U.S. market are included. The sales represent total revenues, which include the sales for rare diseases as well as other indications. This approach is important since the objective of this report is to identify the significance of ODA in the growth of the orphan drugs segment. Regulatory incentives provided by ODA have helped to promote clinical trials of existing drugs to identify their effectiveness for several rare indications in addition to the emergence of companies with exclusive orphan-drug-based business models.
Market figures are based on revenues at the manufacturer level and are projected at 2009 dollar values (i.e., inflation is not computed into the projection figures). Forecasts are mainly based on the projections of the marketers. Growth of competing drugs, patient population, patent and market exclusivity status, and life cycle management strategies adopted by marketers have also been considered for estimating the future growth trends.
The information and data to prepare this study were derived from regulatory agencies, trade literature, patient support groups, annual reports of companies, and industry-related databases. The analysis presented are based on an extensive survey of the pharmaceutical industry as well as a detailed examination of published literature and reports obtained from regulatory authorities, medical research institutions, and national and world health organizations. Key information from the literature was used to obtain expert opinion and insights on commercial potential and market sizes from industry professionals, academic researchers, and government agency spokespersons.
Syamala Ariyanchira is actively involved in technology assessment, strategic planning, competitor analysis, due diligence, and market analysis. She combines her technical background with business sector issues with ease. Her clients include investment firms, government agencies, and chemical and pharmaceutical companies. She holds a PhD from the Indian Institute of Science in Bangalore, India, and has more than 15 years of experience in the industry.
BCC offers an online information retrieval service. BCC’s home page, located at, enables readers to:
  • Examine BCC’s complete catalog of Market Research Reports and place direct orders
  • Subscribe to any of BCC’s many industry newsletters
  • Read announcements of recently published reports and newly launched newsletters
  • Register for BCC’s well-known conferences
  • Request additional information on any BCC product
  • Take advantage of special offers
The information developed in this report is intended to be as reliable as possible at the time of publication and of a professional nature. This information does not constitute managerial, legal, or accounting advice; nor should it serve as a corporate policy guide, laboratory manual, or an endorsement of any product, as much of the information is speculative in nature. The author assumes no responsibility for any loss or damage that might result from reliance on the reported information or its use.

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 Drugs205 $2,750 Free
Chapter- 1: INTRODUCTION5Free
Chapter- 2: SUMMARY2$250Free
Chapter- 3: OVERVIEW23$309Free
Chapter- 5: PRODUCTS46$617Free
Chapter- 6: APPLICATIONS66$885Free
Chapter- 9: COMPANY PROFILES12$161Free
Chapter- 10: GLOSSARY3$40Free
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.

Market Research Report

Single User License: $2750

Become a Member

Already a member? Login to access this report free.

Have a Question? Ask Us.

Why BCC Research.?

Every purchase provides access to:

  • Thousands of recently published reports from select publishers
  • Member Discounts of up to 50% on over 20,000 additional reports from all publishers
  • Our analysts and research concierge for all inquiries