Global Markets for Orphan Drugs

Feb 2024| PHM038G| BCC Publishing

Report Highlights

The global market for orphan drugs is expected to increase from $233.9 billion in 2023 to $415.2 billion by the end of 2028, with a compound annual growth rate (CAGR) of 12.2% during the forecast period of 2023-2028.

Report Includes

  • 33 data tables and 46 additional tables
  • Overview and an up-to-date analysis of the global markets for orphan drugs (ODs) for rare diseases treatment
  • Analyses of the global market trends, with historical market revenue data (sales figures) from 2021 to 2022, estimates for 2023, forecasts for 2024, and projections of compound annual growth rates (CAGRs) through 2028
  • Highlights of the current and future market potential of orphan drugs, along with a detailed analysis of the market drivers, restraints, and opportunities
  • Estimate of the actual market size and revenue forecast for the global orphan drugs market, and its corresponding market share analysis based on the product type, therapeutic area, and region
  • Analysis of market growth opportunities with a holistic review of Porter’s five forces model and PESTLE analyses considering the micro- and macroeconomic factors prevailing in the marketplace
  • Discussion of sustainability trends and factors in the orphan drugs market, with emphasis on consumer attitudes, ESG score analysis, future of ESG, case study, and the ESG practices followed
  • Analyses of the novel therapeutic applications of orphan drugs in rare and orphan diseases as well as examination of the regulatory framework, patents, and recent innovations in the industry
  • Review of the current status and trends in clinical trials for the development of drugs in rare diseases among the three registries from the U.S., EU and Japan
  • Discussion of successful orphan medicinal products, rising influence of pharmacogenomics in the ODs market, information on specific rare diseases for which orphan drugs exist
  • Assessment of the most promising therapeutic areas in the industry and leading orphan drugs in these areas
  • Overview of the industry structure for orphan drugs, pricing and reimbursement policies, COVID-19 impact, and market share analysis of the leading manufacturers of orphan drugs along with their SWOT analyses
  • Company profiles of major players within the industry, including AstraZeneca Plc, Eisai Co. Ltd., Bristol Myers Squibb and Pfizer Inc.

Report Scope

The scope of this study includes orphan drugs used in the global pharmaceutical markets. This report analyzes the therapeutic applications of orphan drugs in rare and orphan diseases as well as examines the regulatory framework, patents, and recent innovations in the industry. The report also analyzes each submarket and its applications, projections, and market shares. Technological issues include the latest trends and developments.

This report covers the regional segmentation included as North America, Europe, Asia-Pacific, and Rest of the World. This report also analyzes the regulatory framework for the clinical trials of orphan drugs in rare and orphan diseases. The report examines the trends and characteristics of clinical trials conducted on rare diseases from 1999-2018, using information from various sources. In addition, this report provides an overview of the global orphan drugs market and its competitive landscape. This report also examines acquisition strategies and collaborations, discusses the market’s strengths and weaknesses considering new technologies, and analyzes the industry’s growing competition and changing customer needs.

Frequently Asked Questions (FAQs)

The global orphan drugs market was valued at an estimated $233.9 billion in 2023 and is projected to reach $415.2 billion by 2028, at an estimated CAGR of 12.2% during the forecast period of 2023 to 2028.
  • Favorable Orphan Drugs Act and similar legislations
  • Technological advancements in genomics
  • Patent extensions
  • Premium pricing for orphan drugs
The report will provide information on orphan drugs along with their growth projections in the coming years. Recent developments of companies operating in this market space have been covered. Information about product pipelines, pricing analysis, clinical trials will help the intended audience formulate investment plans and business strategies. The report also identifies promising and emerging countries where the established players can plausibly expand their business.
The biological orphan drugs segment dominates the overall orphan drugs market and will continue to do so by 2028.
North America holds the highest share in the market.

Analyst Credentials

BCC Research Team possesses expertise and experience in life and physical science domains. They specialize in offering valuable business insights, including industry analysis, competitor intelligence, strategic and financial analysis, and opportunity assessment. The team has in-depth knowledge of various sectors, including healthcare, biotechnology, pharmaceuticals, IT, automation, advanced materials, and energy. They are proficient in qualitative and quantitative market intelligence providing clients with actionable insights. With a vast understanding of the competitive landscape, the team can support clients in making data-driven decisions to help them achieve a competitive edge in their respective markets.

Report Synopsis

Report Metrics Details
Base year considered 2022
Forecast period considered 2023-2028
Base year market size $210.0 billion
Market size forecast $415.2 billion
Growth rate CAGR of 12.2% for the forecast period of 2023-2028
Units considered $ Millions
Segments covered Product Type, Therapeutic Application, Region
Regions covered North America, Europe, Asia-Pacific, Rest of the World (RoW)
Countries covered U.S., Canada, Mexico, Spain, Italy, Netherlands, Turkey, Austria, Poland, Germany, UK, France, China, India, Japan, Australia, South Korea, Thailand, Singapore, Vietnam, Middle East and Africa
Key Market Drivers
  • Favorable Orphan Drug Act and Similar Legislation
  • Technological Advances in Genomics
  • Lack of Competition from Generic Drugs
  • Patent Extensions
  • Premium Pricing of Orphan Drugs
  • Innovations in Manufacturing Technologies
  • Increasing Collaborations and Licensing Agreements
Companies studied
  • ABBVIE INC.
  • ASTELLAS PHARMA INC.
  • ASTRAZENECA PLC
  • BRISTOL-MYERS SQUIBB CO.
  • EISAI CO. LTD.
  • ELI LILLY AND CO.
  • F. HOFFMANN-LA ROCHE LTD.
  • MERCK & CO. INC.
  • NOVARTIS AG
  • PFIZER INC.
  • SANOFI
  • TAKEDA PHARMACEUTICAL CO. LTD.

Table of Contents

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 info@bccresearch.com
Title/Chapter NamePagesMember Price
Full Report: Global Markets for Orphan Drugs189Free
Chapter- 1: Introduction4Free
Chapter- 2: Summary and Highlights3Free
Chapter- 3: Market Overview3Free
Chapter- 4: Market Dynamics35Free
Chapter- 5: Global Market for Orphan Drugs by Product Type7Free
Chapter- 6: Global Market for Orphan Drugs by Therapeutic Application12Free
Chapter- 7: Global Market for Orphan Drugs by Region15Free
Chapter- 8: ESG Outlook in the Orphan Drugs Market8Free
Chapter- 9: Orphan Drugs Exclusivity and Pricing Policies4Free
Chapter- 10: Clinical Trials on Drugs for Rare Diseases44Free
Chapter- 11: Competitive Landscape3Free
Chapter- 12: Company Profiles49Free
Chapter- 13: Appendix: Acronyms2Free
Published - Aug-2021| Analyst - BCC Publishing| Code - PHM038F

Report Highlights

The global orphan drugs market should reach $248.2 billion by 2026 from $190.8 billion in 2021 at a compound annual growth rate (CAGR) of 5.4% for the forecast period of 2021 to 2026.

Report Includes

  • 26 tables
  • An updated review of the global markets for orphan drugs (ODs) within the healthcare industry
  • Analyses of the global market trends, with data from 2020, estimates for 2021 and projections of compound annual growth rates (CAGRs) through 2026
  • Estimation of market size and revenue forecast for biological and non-biological orphan drugs, and corresponding market share analysis by product type, therapeutic category and geographic region
  • Emphasis on the regulation of clinical trials on orphan designated drugs by leading regulatory authorities including the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Pharmaceutical Affairs and Food Sanitation Council (PAFSC) of Japan
  • Review of the current status and trends in clinical trials for the development of drugs in rare diseases among the three registries from the U.S., EU and Japan
  • Discussion of successful orphan medicinal products, rising influence of pharmacogenomics in the ODs market, information on specific rare diseases for which orphan drugs exist
  • Assessment of the most promising therapeutic areas in the industry and leading orphan drugs in these areas
  • Highlights of the industry structure for orphan drugs, pricing and reimbursement policies, COVID-19 impact assessment and market share analysis of the top 20 manufacturers of orphan drugs
  • Descriptive company profiles of the market leading participants, including AbbVie Inc., Eli Lilly, Johnson & Johnson, Merck & Co., Pfizer Inc. and Sanofi-Aventis
Published - Sep-2015| Analyst - Shalini Shahani Dewan| Code - PHM038E

Report Highlights

The global orphan drug market totaled nearly $123 billion in 2014 and will continue to grow to reach nearly $191 billion by 2019, demonstrating a compound annual growth rate (CAGR) of 9.2% during the forecast period (2014 to 2019).

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 2013 and 2014, and projections of compound annual growth rates (CAGRs) through 2019.
  • 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.

Published - Jun-2013| Analyst - Shalini Shahani Dewan| Code - PHM038D

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.

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.

Global Markets for Orphan Drugs

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