Global Orphan Drugs Market Report 2024

Orphan Drugs Global Market Report 2023 – 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, Other Diesease Types) – Market Size, Trends, And Global Forecast 2023-2032

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Orphan drugs refer to drugs that are specifically designed to treat an uncommon medical problem. It might be characterised as medicines that address public health needs but are not produced by the pharmaceutical industry due to financial constraints. Since a chemical may be used to treat a common condition but not have been designed for another, more rare indication, the indications of a medicine may likewise be called "orphan."

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

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, Haematology, Neurology, Cardiovascular, Other Disease Types

The global orphan drugs market size grew from $154.81 billion in 2022 to $170.77 billion in 2023 at a compound annual growth rate (CAGR) of 10.3%. The Russia-Ukraine war disrupted the chances of global economic recovery from the COVID-19 pandemic, at least in the short term. The war between these two countries has led to economic sanctions on multiple countries, a surge in commodity prices, and supply chain disruptions, causing inflation across goods and services and affecting many markets across the globe. The market size of orphan drugs is expected to grow from $293.6 billion in 2027 at a CAGR of 14.5%.

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 rare. Rare disease treatments often require a cold chain, a supply chain regulated by the temperature that is not readily accessible in all countries. Orphan diseases or rare diseases occur rarely among people (i.e. 7 out of 10,000). However, globally, the prevalence of rare diseases is increasing in recent years. For instance, in January 2022, according to an article shared by the American Cancer Society, in USA, There are about 54,000 new instances of oropharyngeal or oral cancer each year. 11,230 people lose their lives to oropharyngeal or oral cancer. The average age of most people diagnosed with these cancers is 63, but they can occur in young people. Just over 20% (1 in 5) of cases occur in patients younger than 55. Thus, the rising prevalence of rare diseases is driving the growth of the orphan drugs market.

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.

The lack of supportive government policies hinders the orphan drugs market. Due to the lack of relevant policies for orphan drugs, certain drugs do not receive any special recognition or priority for approval by regulatory authorities. Medgenome Labs Ltd., the 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 a lack of proper regulations and clear guidelines, do not obtain tax cuts or exemptions from customs duties. Therefore, the 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, 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 October 2020, Retrophin Inc., a US-based biopharmaceutical company, acquired Orphan Technologies for $90 million. This agreement adds OT-58, a brand-new enzyme replacement medication for the treatment of classic homocystinuria that is now in Phase 1/2 research. The acquisition increases the number of possible first-in-class treatments for rare diseases in the pipeline. Orphan Technologies is a Switzerland-based clinical-stage biopharmaceutical company focused on the development of OT-58.

North America was the largest region in the global orphan drugs market in 2022. Middle East is expected to be the fastest growing region in the orphan drugs market report during the forecast period. The regions covered in the orphan drugs analysis report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, the 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, the UK, and the USA.

The orphan drugs market consists of sales of ivacaftor and alglucerase. Values in this market are factory gate values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organisations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the chain or as part of other products.

The orphan drugs market research report is one of a series of new reports from The Business Research Company that provides orphan drugs market statistics, including orphan drugs industry global market size, regional shares, competitors with an orphan drugs market share, detailed orphan drugs market segments, market trends and opportunities, and any further data you may need to thrive in the orphan drugs industry. This orphan drugs market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

    Table Of Contents

    1. Executive Summary

    2. Orphan Drugs Market Characteristics

    3. Orphan Drugs Market Trends And Strategies

    4. Orphan Drugs Market – Macro Economic Scenario

    4.1 COVID-19 Impact On Orphan Drugs Market

    4.2 Ukraine-Russia War Impact On Orphan Drugs Market

    4.3 Impact Of High Inflation On Orphan Drugs Market

    5. Orphan Drugs Market Size And Growth

    5.1. Global Orphan Drugs Historic Market, 2017-2022, $ Billion

    5.1.1. Drivers Of The Market

    5.1.2. Restraints On The Market

    5.2. Global Orphan Drugs Forecast Market, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ Billion

    Oncology

    Blood

    Central Nervous System

    Endocrine

    Cardiovascular

    Respiratory

    Immunomodulatory

    6.2. Global Orphan Drugs Market, Segmentation By Distribution Channel, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    Hospital Pharmacy

    Retail Pharmacy

    Online Sales

    6.3. Global Orphan Drugs Market, Segmentation By Drug Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    Biological

    Non-Biological

    6.4. Global Orphan Drugs Market, Segmentation By Diesease Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    Oncology

    Hematology

    Neurology

    Cardiovascular

    Other Diesease Types

    7. Orphan Drugs Market Regional And Country Analysis

    7.1. Global Orphan Drugs Market, Split By Region, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    7.2. Global Orphan Drugs Market, Split By Country, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F,$ Billion

    10. India Orphan Drugs Market

    10.1. India Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    11. Japan Orphan Drugs Market

    11.1. Japan Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    12. Australia Orphan Drugs Market

    12.1. Australia Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    13. Indonesia Orphan Drugs Market

    13.1. Indonesia Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    14. South Korea Orphan Drugs Market

    14.1. South Korea Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ Billion

    16. UK Orphan Drugs Market

    16.1. UK Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    17. Germany Orphan Drugs Market

    17.1. Germany Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion

    18. France Orphan Drugs Market

    18.3. France Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ Billion

    20. Russia Orphan Drugs Market

    20.1. Russia Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ Billion

    24. Brazil Orphan Drugs Market

    24.1. Brazil Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ 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, 2017-2022, 2022-2027F, 2032F, $ 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. Historic And Forecast Inflation Rates

    31.4. Research Inquiries

    31.5. The Business Research Company

    31.6. Copyright And Disclaimer

List Of Tables

    Table 1: Global Historic Market Growth, 2017-2022, $ Billion
  • Table 2: Global Forecast Market Growth, 2022-2027F, 2032F, $ Billion
  • Table 3: Global Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 4: Global Orphan Drugs Market, Segmentation By Distribution Channel, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 5: Global Orphan Drugs Market, Segmentation By Drug Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 6: Global Orphan Drugs Market, Segmentation By Diesease Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 7: Global Orphan Drugs Market, Split By Region, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 8: Global Orphan Drugs Market, Split By Country, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 9: Asia-Pacific, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 10: China, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 11: India, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 12: Japan, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 13: Australia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 14: Indonesia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 15: South Korea, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 16: Western Europe, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 17: UK, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 18: Germany, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 19: France, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 20: Eastern Europe, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 21: Russia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 22: North America, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 23: USA, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 24: South America, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 25: Brazil, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 26: Middle East, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Table 27: Africa, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ 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

List Of Figures

    Figure 1: Global Historic Market Growth, 2017-2022, $ Billion
  • Figure 2: Global Forecast Market Growth, 2022-2027F, 2032F, $ Billion
  • Figure 3: Global Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 4: Global Orphan Drugs Market, Segmentation By Distribution Channel, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 5: Global Orphan Drugs Market, Segmentation By Drug Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 6: Global Orphan Drugs Market, Segmentation By Diesease Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 7: Global Orphan Drugs Market, Split By Region, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 8: Global Orphan Drugs Market, Split By Country, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 9: Asia-Pacific, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 10: China, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 11: India, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 12: Japan, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 13: Australia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 14: Indonesia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 15: South Korea, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 16: Western Europe, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 17: UK, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 18: Germany, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 19: France, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 20: Eastern Europe, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 21: Russia, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 22: North America, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 23: USA, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 24: South America, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 25: Brazil, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 26: Middle East, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion
  • Figure 27: Africa, Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ 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
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