Report Highlights
Report Includes
- 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; this report focuses on biologics
- 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.
- Analyses 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.
INTRODUCTION
STUDY OBJECTIVES
The orphan drug market is one of the fastest-growing segments in the pharmaceutical and biotechnology industry. BCC’s goal in conducting this study is to provide an overview of the current and future global market for orphan drugs. The key objective is to present a comprehensive analysis of current orphan drugs and those under development as important therapies and treatments for rare diseases.
This report explores present and future strategies and regulations within the orphan drug market, advances in the market, the setbacks, the current situation and the needs of the market. The market for each drug listed by its generic name/active ingredient and approved indication(s) is also analyzed in this report.
A detailed analysis of the structure of the orphan drug industry has been conducted. Revenues are broken down by region drug type (i.e., biological and nonbiological), active ingredient and area of medical application. Sales figures are estimated for the five-year period from 2012 through 2017.
Applications for orphan drugs are also discussed in the report, with an emphasis on the usage of the drugs in various disease segments. The report also covers significant patents and their allotments in each category.
REASONS FOR DOING THIS STUDY
Extensive research in the field of orphan drugs has resulted in therapeutic treatments for a number of rare diseases. Most of the big pharmaceutical companies have now begun to explore the advantages of orphan drugs as a major source of profit and a steady market.
R&D spending, competition and new technologies are increasing. Advances, new product launches and assistance from regulatory bodies are positively impacting the growth of the market for orphan drugs in the near term. This study examines these factors.
This study focuses on the manufacturers and users of orphan drugs and its market growth. Pharmaceutical biotechnical companies, research institutes and physicians will find this study to be of interest.
SCOPE OF THE STUDY
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.
METHODOLOGY
Both primary and secondary research methodologies were used in preparing this study. BCC conducted a comprehensive literature search, which included technical newsletters and journals and many other sources. Projections were based on estimates such as the current number of end users, potential end users, mergers and acquisitions and market trends.
INFORMATION SOURCES
Many companies were surveyed to obtain data for this study. Included were manufacturers and end users of orphan drugs within the therapeutic categories examined and various disease sector industries. Data were gathered from various industry sources. BCC spoke with officials within the industry, consulted newsletters, company literature, product literature and a host of technical articles, journals, indexes and abstracts. Exhaustive investigations of databases by key terminology were completed. In addition, data were compiled from current financial, trade and government sources.
BCC ONLINE SERVICES
BCC Research offers an online information retrieval service. Its home page, located at www.bccresearch.com, 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 upcoming reports and newly launched newsletters.
- Register for BCC’s well-known conferences.
- Request additional information about any BCC product.
- Take advantage of special offers.
To reach this important new information resource, go to www.bccresearch.com.
DISCLAIMER
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 from its use.
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.
Related Reports
Biologic Therapeutic Drugs: Technologies and Global Markets
The market for biologics was valued at $150 billion in 2011 and should reach $163 billion in 2012. Total market value is expected to reach $252 billion in 2017 after increasing at a five-year compound annual growth rate of 9%.
Global Markets for Diabetes Therapeutics and Diagnostics
The global market for diabetes therapeutics and diagnostics was valued at $110 billion in 2011 and should reach nearly $118.7 billion in 2012. Total market value is expected to reach nearly $157 billion in 2017 after increasing at a five-year compound annual growth rate (CAGR) of 5.7%.
Biosimilars: Global Markets
The global market for biosimilars reached $1.7 billion in 2010 and $2.5 billion in 2011. The market is expected to reach $3.6 billion by 2016, a compound annual growth rate (CAGR) of 7.7%.
Antibody Drugs: Technologies and Global Markets
The global market for therapeutic monoclonal antibodies (mAbs) was estimated at $44.6 billion in 2011. With the rollout of at least eight new therapeutic mAb products and expanded indications for existing products expected during the forecast period, the global mAb market is expected to rise at a compound annual growth rate (CAGR) of 5.3% to nearly $58 billion in 2016.
Recent Reports
Global Market for Contract Research Organization (CRO) Services
The global market for contract research organization (CRO) services is expected to grow from $111.8 billion in 2024 and is projected to reach $166.0 billion by the end of 2029, at a compound annual growth rate (CAGR) of 8.2% during the forecast period of 2024 to 2029.
Global Oncology Pharmaceuticals Market
The global market for oncology pharmaceuticals is estimated to increase from $196.4 billion in 2023 to reach $401.4 billion by 2029, at a compound annual growth rate (CAGR) of 12.7% from 2024 through 2029.
Excipients in Pharmaceuticals: Global Markets to 2029
The global market for pharmaceutical excipients was valued at $9.2 billion in 2023. It is expected to grow from $9.7 billion in 2024 to $12.4 billion by the end of 2029, at a compound annual growth rate (CAGR) of 5.1% from 2024 to 2029.
Antibody-Drug Conjugates: Technologies and Global Markets
The global market for antibody-drug conjugates is estimated to increase from $10.8 billion in 2023 to reach $47.0 billion by 2029, at a compound annual growth rate (CAGR) of 28.4% from 2024 through 2029.
Global Markets for Contract Pharmaceutical Manufacturing, Research and Packaging
The global market for contract pharmaceutical manufacturing, research and packaging is estimated to increase from $212.2 billion in 2023 to reach $310.2 billion by 2029, at a compound annual growth rate (CAGR) of 6.7% from 2024 through 2029.
Top Trending Reports
Global Artificial Intelligence (AI) Market: Investments vs Potential
The global market for artificial intelligence (AI) is estimated to increase from $148.8 billion in 2023 to reach $1.1 trillion by 2029, at a compound annual growth rate (CAGR) of 39.7% from 2024 through 2029.
Drug Discovery Technologies
The global market for drug discovery technologies is expected to grow from $111.9 billion in 2024 and is projected to reach $197.0 billion by the end of 2029, at a compound annual growth rate (CAGR) of 12.0% during the forecast period of 2024 to 2029.
Artificial Intelligence (AI) in Life Sciences Market
The global market for artificial intelligence (AI) in life sciences products was valued at $8.0 billion in 2023. The market is estimated to grow from $9.8 billion in 2024 to $33.5 billion by the end of 2029, at a compound annual growth rate (CAGR) of 27.9% from 2024 to 2029.
Synthetic Biology: Global Markets
The global market for synthetic biology products was valued at $15.4 billion in 2023. The market is projected to grow from $19.3 billion in 2024 to $61.6 billion by the end of 2029, at a compound annual growth rate (CAGR) of 26.1% from 2024 through 2029.
Gene Editing Therapeutics Market
The report provides an overview of current and future market potential of gene editing therapeutics and provides a detailed analysis of the drivers, restraints and opportunities in this market. It also include company profiles of key players, featuring detailed information regarding each company’s business segments, financials, product portfolios and recent developments.
Become A Member
BCC Research offers a comprehensive library of reports, granting members unlimited access to data, insights, and market intelligence for informed business decisions, while actively supporting members in their evolving journeys and prioritizing high-quality, relevant topics based on continuous engagement with the research community.
Find Out MoreCustom Consulting
BCC Research emphasizes the importance of organizations leveraging highly customized market insights aligned with specific strategic business objectives through direct engagement with primary sources and proprietary forecasting models for profitable decision-making in maximizing growth opportunities and minimizing risks.
Customize NowScorecard
The Venture Scorecard provides commercialization offices and decision makers with expert analysis, offering strategic insights crucial for aligning objectives with market realities at various stages of commercializing new products or evaluating investment opportunities, from opportunity assessment to growth planning.
Find Out MoreInnovation Spotlight
Our industry experts offer strategic guidance to maximize the market potential of commercialized products, patents, and IP by providing insights into market trends, competitive dynamics, and effective positioning, using the Innovation Spotlight service for enhanced exposure to thought leaders and the wider community.
Find Out More