Global Markets for Orphan Drugs

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

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.

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.

• 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 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.

• 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.