Orphan Drugs Global Market Report 2022 – By Therapy Area (Oncology, Blood, Central Nervous System, Endocrine, Cardiovascular, Respiratory, Immunomodulatory), By Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Sales), By Drug Type (Biological, Non-Biological), By Diesease Type (Oncology, Hematology, Neurology, Cardiovascular) – Market Size, Trends, And Global Forecast 2022-2026

Starting Price : $5000.00 | Pages : 150 | Published : February 2022 | SKU CODE : 7610 | Delivery Time: 2-3 business days | Format :


The orphan drugs market consists of sales of orphan drugs and related services that are used to treat rare diseases. An orphan drug is a pharmaceutical drug developed to treat patients suffering from rare diseases and would not make profit if developed commercially.

The main types of orphan drugs are biological and non-biological. The various therapy area include oncology, blood, central nervous system, endocrine, cardiovascular, respiratory and immunomodulatory use for the treatment of oncology, hematology, neurology, cardiovascular, and others. The study of blood and blood disorders is known as haematology. The drugs are distributed through hospital pharmacy, retail pharmacy and online sales.

The global orphan drugs market size is expected to grow from $140.40 billion in 2021 to $154.81 billion in 2022 at a compound annual growth rate (CAGR) of 10.3%. The growth in the market is mainly due to the companies resuming their operations and adapting to the new normal while recovering from the COVID-19 impact, which had earlier led to restrictive containment measures involving social distancing, remote working, and the closure of commercial activities that resulted in operational challenges. The orphan drugs market is expected to reach $229.71 billion in 2026 at a CAGR of 10.4%.

The rising prevalence of rare diseases is a key factor driving the growth of the orphan drugs market. Any disease that affects a small percentage of the population is a rare disease. Rare disease treatments often require a cold chain, a supply chain regulated by temperature that is not readily accessible in all countries. Orphan diseases or rare diseases occurs rarely among the people (i.e. 7 out of 10,000). However, globally, the prevalence of rare diseases is increasing in recent years. According to the study published in European Journal of Human Genetics in 2019, there are 300 million cases of rare diseases and approximately 4% of total population is affected by rare disease at any point in time. Thus, the rising prevalence of rare diseases is driving the growth of the orphan drugs market.

Lack of supportive government policies hinders the orphan drugs market. Due to the lack of relevant policies for orphan drug, certain drugs do not receive any special recognition or priorities for approval by regulatory authority. Medgenome Labs Ltd., global research partner in accelerating insights into complex genetic diseases, pointed out that companies manufacturing orphan drugs frequently drop out in foreign markets due to a lack of government funding. For example, orphan medical products (OMPs) in India, due to lack of proper regulations and clear guidelines, do not obtain tax cuts or exemptions from customs duties. Therefore, lack of supportive government policies limits the growth of the orphan drugs market.

Approval of biological orphan drugs for multiple indication act as a key trend driving the growth of the orphan drugs market. The biological drugs are used for treating rare diseases such as cancer with fewer side effects that have a high prevalence rate in the developed world. For instance, in order to launch the company’s biological orphan drug development program Cardax, Inc. announced that it has been engaged with biological orphan drug expert Frederick D. Sancilio, Ph.D. For the development of commercial products, the companies are focused on obtaining biological orphan drugs to increase their revenue.

In November 2019, Bristol-Myers Squibb, a biopharmaceutical company whose mission is to discover, develop, and deliver innovative medicines, acquired Celgene for an undisclosed amount. Through this acquisition, Celgene shareholders received for each share, 1 share of Bristol-Myers Squibb common stock, $50.00 in cash without interest and one tradeable Contingent Value Right (CVR), which will entitle the holder to receive a payment of $9.00 in cash if certain future regulatory milestones are achieved. Celgene, a biopharmaceutical company positioned to address the needs of the patients with serious diseases.

Major players in the orphan drugs market are Bristol-Myers Squibb Company, Celgene Corporation, F. Hoffmann-La Roche Ltd, Amgen, Biogen, Bayer, Novartis AG, GlaxoSmithKline plc, Johnson & Johnson, AbbVie Inc, Eli Lilly and Company, Alexion Pharmaceuticals, Shire, Novo Nordisk, AstraZeneca, Eisai, Daiichi Sankyo, Merck, Teva Pharmaceutical Industries, Actelion Pharmaceuticals Ltd., and Aegerion Pharmaceuticals.

North America was the largest region in the orphan drugs market in 2021. The Middle East is expected to be the fargest growing region in the forecast period. The regions covered in the orphan drugs market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the orphan drugs market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA.

The global orphan drugs market is segmented -

1) By Therapy Area: Oncology, Blood, Central Nervous System, Endocrine, Cardiovascular, Respiratory, Immunomodulatory

2) By Distribution Channel: Hospital Pharmacy, Retail Pharmacy, Online Sales

3) By Drug Type: Biological, Non-Biological

4) By Disease Type: Oncology, Hematology, Neurology, Cardiovascular, Others

    Table Of Contents

    1. Executive Summary

    2. Orphan Drugs Market Characteristics

    3. Orphan Drugs Market Trends And Strategies

    4. Impact Of COVID-19 On Orphan Drugs

    5. Orphan Drugs Market Size And Growth

    5.1. Global Orphan Drugs Historic Market, 2016-2021, $ Billion

    5.1.1. Drivers Of The Market

    5.1.2. Restraints On The Market

    5.2. Global Orphan Drugs Forecast Market, 2021-2026F, 2031F, $ Billion

    5.2.1. Drivers Of The Market

    5.2.2. Restraints On the Market

    6. Orphan Drugs Market Segmentation

    6.1. Global Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    Oncology

    Blood

    Central Nervous System

    Endocrine

    Cardiovascular

    Respiratory

    Immunomodulatory

    6.2. Global Orphan Drugs Market, Segmentation By Distribution Channel, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    Hospital Pharmacy

    Retail Pharmacy

    Online Sales

    6.3. Global Orphan Drugs Market, Segmentation By Drug Type, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    Biological

    Non-Biological

    6.4. Global Orphan Drugs Market, Segmentation By Diesease Type, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    Oncology

    Hematology

    Neurology

    Cardiovascular

    Others

    7. Orphan Drugs Market Regional And Country Analysis

    7.1. Global Orphan Drugs Market, Split By Region, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    7.2. Global Orphan Drugs Market, Split By Country, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    8. Asia-Pacific Orphan Drugs Market

    8.1. Asia-Pacific Orphan Drugs Market Overview

    Region Information, Impact Of COVID-19, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies

    8.2. Asia-Pacific Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    9. China Orphan Drugs Market

    9.1. China Orphan Drugs Market Overview

    9.2. China Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F,$ Billion

    10. India Orphan Drugs Market

    10.1. India Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    11. Japan Orphan Drugs Market

    11.1. Japan Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    12. Australia Orphan Drugs Market

    12.1. Australia Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    13. Indonesia Orphan Drugs Market

    13.1. Indonesia Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    14. South Korea Orphan Drugs Market

    14.1. South Korea Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    15. Western Europe Orphan Drugs Market

    15.1. Western Europe Orphan Drugs Market Overview

    15.2. Western Europe Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    16. UK Orphan Drugs Market

    16.1. UK Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    17. Germany Orphan Drugs Market

    17.1. Germany Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    18. France Orphan Drugs Market

    18.3. France Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    19. Eastern Europe Orphan Drugs Market

    19.1. Eastern Europe Orphan Drugs Market Overview

    19.2. Eastern Europe Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    20. Russia Orphan Drugs Market

    20.1. Russia Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    21. North America Orphan Drugs Market

    21.1. North America Orphan Drugs Market Overview

    21.2. North America Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    22. USA Orphan Drugs Market

    22.1. USA Orphan Drugs Market Overview

    22.2. USA Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    23. South America Orphan Drugs Market

    23.1. South America Orphan Drugs Market Overview

    23.2. South America Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    24. Brazil Orphan Drugs Market

    24.1. Brazil Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    25. Middle East Orphan Drugs Market

    25.1. Middle East Orphan Drugs Market Overview

    25.2. Middle East Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    26. Africa Orphan Drugs Market

    26.1. Africa Orphan Drugs Market Overview

    26.2. Africa Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

    27. Orphan Drugs Market Competitive Landscape And Company Profiles

    27.1. Orphan Drugs Market Competitive Landscape

    27.2. Orphan Drugs Market Company Profiles

    27.2.1. Bristol-Myers Squibb Company

    27.2.1.1. Overview

    27.2.1.2. Products and Services

    27.2.1.3. Strategy

    27.2.1.4. Financial Performance

    27.2.2. Celgene Corporation

    27.2.2.1. Overview

    27.2.2.2. Products and Services

    27.2.2.3. Strategy

    27.2.2.4. Financial Performance

    27.2.3. F. Hoffmann-La Roche Ltd

    27.2.3.1. Overview

    27.2.3.2. Products and Services

    27.2.3.3. Strategy

    27.2.3.4. Financial Performance

    27.2.4. Amgen

    27.2.4.1. Overview

    27.2.4.2. Products and Services

    27.2.4.3. Strategy

    27.2.4.4. Financial Performance

    27.2.5. Biogen

    27.2.5.1. Overview

    27.2.5.2. Products and Services

    27.2.5.3. Strategy

    27.2.5.4. Financial Performance

    28. Orphan Drugs Pipeline Analysis

    29. Key Mergers And Acquisitions In The Orphan Drugs Market

    30. Orphan Drugs Market Future Outlook and Potential Analysis

    31. Appendix

    31.1. Abbreviations

    31.2. Currencies

    31.3. Research Inquiries

    31.4. The Business Research Company

    31.5. Copyright And Disclaimer

List Of Tables

    Figure 1: Global Historic Market Growth, 2016-2021, $ Billion
  • Figure 2: Global Forecast Market Growth, 2021-2026F, 2031F, $ Billion
  • Figure 3: Global Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 4: Global Orphan Drugs Market, Segmentation By Distribution Channel, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 5: Global Orphan Drugs Market, Segmentation By Drug Type, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 6: Global Orphan Drugs Market, Segmentation By Diesease Type, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 7: Global Orphan Drugs Market, Split By Region, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 8: Global Orphan Drugs Market, Split By Country, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 9: Asia-Pacific, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 10: China, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 11: India, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 12: Japan, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 13: Australia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 14: Indonesia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 15: South Korea, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 16: Western Europe, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 17: UK, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 18: Germany, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 19: France, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 20: Eastern Europe, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 21: Russia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 22: North America, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 23: USA, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 24: South America, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 25: Brazil, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 26: Middle East, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 27: Africa, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Figure 28: Bristol-Myers Squibb Company Financial Performance
  • Figure 29: Celgene Corporation Financial Performance
  • Figure 30: F. Hoffmann-La Roche Ltd Financial Performance
  • Figure 31: Amgen Financial Performance
  • Figure 32: Biogen Financial Performance

List Of Figures

    Table 1: Global Historic Market Growth, 2016-2021, $ Billion
  • Table 2: Global Forecast Market Growth, 2021-2026F, 2031F, $ Billion
  • Table 3: Global Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 4: Global Orphan Drugs Market, Segmentation By Distribution Channel, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 5: Global Orphan Drugs Market, Segmentation By Drug Type, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 6: Global Orphan Drugs Market, Segmentation By Diesease Type, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 7: Global Orphan Drugs Market, Split By Region, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 8: Global Orphan Drugs Market, Split By Country, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 9: Asia-Pacific, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 10: China, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 11: India, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 12: Japan, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 13: Australia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 14: Indonesia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 15: South Korea, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 16: Western Europe, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 17: UK, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 18: Germany, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 19: France, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 20: Eastern Europe, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 21: Russia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 22: North America, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 23: USA, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 24: South America, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 25: Brazil, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 26: Middle East, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 27: Africa, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion
  • Table 28: Bristol-Myers Squibb Company Financial Performance
  • Table 29: Celgene Corporation Financial Performance
  • Table 30: F. Hoffmann-La Roche Ltd Financial Performance
  • Table 31: Amgen Financial Performance
  • Table 32: Biogen Financial Performance
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