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Orphan Drugs Market Size, Share & COVID-19 Impact Analysis, By Therapy Area (Oncology, Hematology, Neurology, Endocrinology, Cardiovascular, Respiratory, Immunotherapy, and Others), By Drug Type (Biologics, and Non-Biologics), By Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Sales, and Others), and Regional Forecast, 2020-2027

Region : Global | Format: PDF | Report ID: FBI100088



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The global orphan drugs market size was valued at USD 151.00 billion in 2019 and is projected to reach USD 340.84 billion by 2027, exhibiting a CAGR of 10.5% during the forecast period.

According to the European Organization for Rare Diseases, these drugs are produced for diagnosis, prevention, and treatment of rare disorders. The oncology therapy area holds a considerably higher share in the global market due to emergence of new cancer-related diseases. Additionally, FDA approvals to a series of cancer-related orphan drugs are expected to result in increased commercialization of these drugs, especially the oncology therapy area, during the forecast period. The market is will derive growth from higher demand for immunomodulators, as they regularize or amplify the immune system. Rising prevalence of infectious diseases in developed as well as emerging nations and higher prices of medications are projected to fuel demand for immunomodulators, thus contributing to orphan drugs market growth during the forecast period.

COVID-19 Pandemic is Anticipated to Negatively Impact the Global Market

The impact of the COVID-19 pandemic on certain pharmaceuticals markets is expected to be more pronounced due to the postponement of the diagnosis and treatment of non-life-threatening diseases. This is especially true for individuals suffering from rare diseases as more financial and healthcare resources by various governments were directed towards the fight against the pandemic. For instance, a report by TransUnion Healthcare estimated decreases of between 32-60% of outpatient hospital visits in March 2020 for over 500 hospitals across the U.S. This is particularly expected to affect individuals suffering from rare diseases as the lack of diagnostic and consulting services by healthcare professionals will impact the timely prescription of orphan drugs. Hence, as of now, the 2019-20 coronavirus pandemic is expected to have a negative impact on the global market.


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Strong Product Launches to Drive the Market Growth

Globally, patients suffering from rare diseases are seeking efficient and potent treatment of these disorders. Increasing R&D initiatives and increasing advocacy by patients’ groups for effective treatment are propelling the introduction of newer and effective product offerings. These trends have led to the development and marketing of several blockbuster drugs for the treatment of several types of cancers, immunological diseases, and other rare diseases. For instance, in June 2020, Chiasma, Inc. announced that the company had received the FDA approval for their product offering of Mycapssa for the treatment of acromegaly, a rare disorder. Thus, the introduction of such sophisticated orphan therapeutics by established and emerging companies have been instrumental in boosting the adoption of these drugs, which, in turn, is expected to aid the growth of the global market during the forecast period.


Increasing R&D Initiatives to Drive the Global Market Growth

One of the critical driving factors prevailing in the global market is the increasing R&D investments by prominent players for the orphan drug development of novel product offerings. Since the awareness and understanding regarding rare diseases has increased, a number of key clinical stage biopharmaceutical companies and established market players have strong pipeline candidates for orphan drugs in various stages of clinical trials. This increasing foray into rare disorders’ therapeutics is owing to the reason that major pharmaceutical breakthroughs resulting in the blockbuster drugs developments are quite possible in rare disorders in comparison to the traditional pharmaceutical portfolios. Another strong driver for this is that the pharmaceutical companies are necessitated to conduct larger outcome studies for the traditional therapeutics for diseases such as diabetes and coronary artery disease (CAD) regulatory approvals in comparison to the rare diseases. This is projected to propel the market growth during the forecast period.

Growing Patient Population with Rare Diseases is Propelling Growth of the Market

Strong research and development initiatives for rare diseases’ therapeutics are expected to have a significant positive impact on the global market. According to an estimate by European Organisation for Rare Diseases (EURORDIS) in 2020, approximately more than 300 million people are living with one or more of over 6,000 identified rare diseases around the world. Furthermore, the study states that the rare disorders currently affect 3.5% - 5.9% of the global population. This has led to the generation of a sizeable patient population who have significantly unmet clinical needs which are extremely serious in terms of nature. Hence, growing investment in R&D and the increasing involvement of major and emerging players in the development of sophisticated and effective products is expected to occur in the forecast period.


High Cost Associated with Orphan Drugs to Limit Market Adoption

Despite, the increasing focus given to the development and marketing of rare diseases’ therapeutics globally, certain limitations are restricting the global market growth. One of the critical restraining factors is the high costs associated with these drugs. According to an estimate published by the University of California, Los Angeles (UCLA) in 2020, the cost of Spinraza, one of the most critical drugs ranged from US$ 50,000 for the first year of treatment and $375,000 per year after that. Such high cost of treatments has significantly limited the widespread adoption of these product offerings, especially in emerging countries. Such high costs of treatment are also a cause of concern for the developed countries where many of the drugs may not be adequately reimbursed, thus significantly increasing the patient’s out-of-pocket costs.


By Therapy Area Analysis

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Neurology Segment to Grow at Faster Pace during 2020-2027

On the basis of product, the market includes oncology, hematology, neurology, endocrinology, cardiovascular, respiratory, immunotherapy, and others. The oncology segment held a dominant orphan drugs market share in 2019. The dominance is due to the presence of several oncology drugs in the product development pipelines of key players, and also the presence of a large number of orphan drugs dedicated for the treatment of cancers. The hematology segment is projected to be the second most dominant segment owing to a number of new product launches and increasing positive regulatory approvals. The neurology segment is also anticipated to register a comparatively strong CAGR due to positive developments in the product offerings for key diseases such as multiple sclerosis.

However, the endocrinology, respiratory, and cardiovascular drugs segments are anticipated to register lower CAGRs in the forecast period. The immunotherapy segment is expected to register a very strong CAGR due to presence of strong products such as Keytruda, and also increased research initiatives.

By Drug Type Analysis

Biologics Segment Dominated the Market in 2019

Based on drug type, the market is segmented into biologics, and non-biologics. The overwhelming presence of product offerings classified as biologics are some of the major factors responsible for the dominance of the segment in 2019. According to an estimate by the U.S. Food and Drug Administration (U.S. FDA), the Orphan Products Development (OOPD) of the organization have led to the development and marketing of over 600 drugs and biologic products for rare disorders since 1983. The non-biologics segment accounted for a lower share of the global market and also registered a comparatively low CAGR.

By Distribution Channel Analysis

Online Sales to Register a Higher CAGR during the Forecast Period

On the basis of distribution channel, the market is segmented into hospital pharmacy, retail pharmacy online sales, online pharmacy, and others. The hospital pharmacy is expected to hold the highest share in the market during the forecast period. The primary cause of this dominance is because a significantly large number of drugs have to be administered at hospitals intravenously by trained healthcare professionals. The retail pharmacy segment is expected to grow at a comparatively low CAGR during the forecast period. The online pharmacy segment is expected to register a high CAGR especially due to the ease given to the consumers in terms of the purchase of drugs. Also, in the uncertain times of COVID-19, online pharmacy is expected to register a strong boost in terms of growth due to the increasing number of consumers switching to online pharmacies for the purchase of these drugs. 


North America Orphan Drugs Market Size, 2019 (USD Billion)

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In terms of the global market, the regions include North America, Europe, Asia Pacific, and the Rest of the World. In North America, the market size stood at USD 81.22 billion in 2019. The strong share of North America is attributable to the significant expenditure on orphan drugs, strong patient population and the presence of key market players leading to the development of sophisticated and innovative products. For instance, according to an estimate published by National Institutes of Health (NIH), in 2017, there were an estimated 7,000 rare diseases in the U.S., with approximately 25-30 million Americans living with a rare disease. This coupled with the presence of favorable reimbursement policies in the United States, is responsible for North America’s dominance in the global market.

The European market is anticipated to register a strong CAGR owing to the strong patient population in the region combined with the strong adoption of sophisticated rare diseases’ therapeutics. The market in Asia Pacific is expected to register the highest growth owing to the increasing healthcare expenditure, and increased awareness of rare diseases. The Rest of the World comprised of Latin America & the Middle East & Africa which accounted for a lower share of the global market owing to the under penetration of market.


Strong Portfolio has Propelled Bristol-Myers Squibb Company, and F. Hoffmann-La Roche Ltd.  to Lead the Global Market.

The market is monopolistic in nature (presence of many companies in the market), owing to the presence of players of various sizes and the diverse product development pipelines of these players. Currently, Bristol-Myers Squibb Company, and F. Hoffmann-La Roche Ltd. dominate the market, accounting for a significant share of the global market in 2019. This is primarily due to the strong sales and product portfolio of the oncology related drugs of these companies. However, several upcoming key players such as Biogen Inc., and BioMarin Pharmaceutical Inc. with their innovative product pipelines for various orphan diseases are emerging. Other prominent players include Amgen, Alexion, Novartis, AstraZeneca, DAIICHI SANKYO COMPANY, LIMITED and Agios Pharmaceuticals., who also have significant presence in the global market with novel drugs.


  • Amgen Inc. (Thousand Oaks, U.S.)

  • Bayer AG (Leverkusen, Germany)

  • F. Hoffmann-La Roche Ltd (Basel, Switzerland)

  • Alexion Pharmaceuticals Inc. (Boston, U.S.)

  • Novo Nordisk A/S (Bagsværd, Denmark)

  • Novartis AG (Basel, Switzerland)

  • Bristol-Myers Squibb Company (New York, U.S.)

  • AstraZeneca (Cambridge, U.K.)


  • GlaxoSmithKline plc (Brentford, U.K.)

  • Other Players


  • June 2020 – Chiasma, Inc. announced that the company had received the FDA approval for their product offering of Mycapssa for the treatment of acromegaly, a rare disorder.

  • June 2020 – Agios Pharmaceuticals, Inc. announced that the company had received the FDA Orphan Drug Designation for their pipeline candidate of Mitapivat for the treatment of Thalassemia.

  • May 2020 – AstraZeneca, and Daiichi Sankyo Company announced that the company had received the FDA Orphan Drug Designation for their product offering of Enhertu (trastuzumab deruxtecan) for the treatment of patients with gastric cancer, including gastroesophageal junction cancer.


An Infographic Representation of Orphan Drugs Market

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The orphan drugs market research report provides a detailed analysis of the market and focuses on key aspects such as prevalence of major orphan disease in key countries, pipeline review for these drugs, technological developments, and healthcare spending by key countries - 2019. Besides this, the report offers insights into the market trends and highlights key industry developments. In addition to the aforementioned factors, the report encompasses several factors that have contributed to the growth of the advanced market over the recent years.

Report Scope & Segmentation



Study Period


Base Year


Forecast Period


Historical Period



  Value (USD billion)


By Therapy Area

  • Oncology

  • Hematology

  • Neurology

  • Endocrinology

  • Cardiovascular

  • Respiratory

  • Immunotherapy

  • Others

By Drug Type

  • Biologics

  • Non-Biologics

By Distribution Channel

  • Hospital Pharmacy

  • Retail Pharmacy

  • Online Sales

  • Others

By Geography

  • North America (U.S. and Canada)

  • Europe (U.K., Germany, France, Italy, Spain, Scandinavia, and Rest of Europe)

  • Asia-Pacific (Japan, China, India, Australia, Southeast Asia, and Rest of Asia- Pacific)

  • Rest of the World

Frequently Asked Questions

Fortune Business Insights says that the global orphan drugs market size was USD 151.00 billion in 2019 and is projected to reach USD 340.84 billion by 2027.

In 2019, the North America market value stood at USD 81.22 billion.

Growing at a CAGR of 10.5%, the market will exhibit steady growth in the forecast period (2020-2027).

The oncology segment is expected to be the leading segment in this market during the forecast period.

Increasing prevalence of serious diseases such as cancers and increasing R&D of orphan drugs by market players are major factors driving the growth of the market.

Bristol-Myers Squibb Company, and F. Hoffmann-La Roche Ltd. are major players of the global market.

North America dominated the market share in 2019.

Launch of advanced product offerings by prominent market players, increasing R&D initiatives, and increased awareness of rare diseases, are expected to drive the adoption of products in the global market.

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