Orphan Diseases Market Research Report- Forecast To 2030

Orphan diseases Market information, by Type of Diseases (autoimmune disorders, genetic disorders, blood disorders, cancer, growth disorder, cardiovascular diseases, neurological disorders, respiratory disorders, digestive disorders, eye disorders), Type of Treatment (gene therapy, cell therapy, drug therapy , End user (hospital and clinics, research laboratory) -Forecast to 2030

ID: MRFR/Pharma/1907-HCR | 85 Pages | Author: Kinjoll Dey | October 2023         

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Orphan Drugs Market

The global Orphan Drugs Market size is projected to reach 328.5 Billion at a CAGR of 10.70% from 2022 to 2030.

By Type of Diseases autoimmune disorders genetic disorders blood disorders cancer growth disorder cardiovascular diseases neurological disorders respiratory disorders digestive disorders eye disorders
By Type of Treatment gene therapy cell therapy drug therapy
By End user hospital and clinics research laboratory
Key Players
Companies Profiled   Bristol-Myers Squibb Company (US)   Novartis AG (Switzerland)   CELGENE CORPORATION (US)   F. Hoffmann-La Roche Ltd (Switzerland)   Pfizer Inc. (US)   Alexion (US)   Sanofi (French)
Market Driving Forces   The rising prevalence of rare diseases   Favorable government policies.
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Orphan Diseases Market Overview 

The Orphan diseases market size is anticipated to reach USD 328.5 billion by 2030 at 10.70% of CAGR during the forecast timeframe 2022-2030. Orphan diseases are also popularly called rare diseases. The market for the orphan disease is growing rapidly in the forecast period. This disease affects a small percentage of the population across the World. Research says that most orphan diseases remain with the patient throughout life as it is genetic. The exact number of diseases are not found yet, but there are nearly more than 7000 various rare disorders and diseases are present across the World. 

The increasing patient population with a rare disease contributes to the global market throughout the review period. Moreover, the research and development initiatives are increasing day by day, fuelling the worldwide market growth. Also, the increasing adoption of orphan drugs is enhancing the market growth. 

According to the Orphan Diseases Market Trends report, the current market has some intended audiences who contribute to generating more scope and opportunities for the global market. These intended audiences are hospitals and clinics, medical research laboratories, orphan diseases drug manufacturers and suppliers, potential investors, government and independent regulatory authorities, market research and consulting service providers, and research and development (R&D) companies. 

COVID-19 Analysis 

In the health care sector, the workflow has been affected due to the arrival of the Covid-19 pandemic. At this time, many sub-domains of healthcare industries have shut their doors for a temporary period. 

Also, the global Orphan Diseases Market Value has decreased with the reduced access to healthcare specialists, limited operations in most industries, the decline in screening services, and inadequate funding. 

Market Dynamics 

  • Key Market Drivers 

The global Orphan Diseases Market growth is driven due to the increasing prevalence of rare diseases across the World. Another important factor that enhances market growth is favorable government policies.

The US food and Drug Administration (US FDA) has estimated that since 1983, the Orphan Products Development (OOPD) organization has resulted in over 600 drugs and biologic products development and marketing for rare disorders. These kinds of initiatives are growing day-by-day that is fuelling the growth of the market during the assessment period.

  • Market Challenges 

The Orphan Diseases Market might face challenges due to the high cost of the orphan drug and the stringent rules and regulations of the government relating to the orphan drug.

Moreover, the manufacturing chain and supply chain have been affected during the pandemic period, which has brought major challenges for the global market due to the lack of transportation, workforce, etc. 

  • Market Opportunities 

The growing novel indications for known orphan drugs are expected to bring lucrative opportunities for the global market players and manufacturers. 

Moreover, the market might go through significant opportunities due to the untapped emerging economies across the World. 

  • Market Restraints 

The global orphan diseases market growth might experience restraints due to the correct treatments, correct diagnosis and availability of healthcare facilities during the research period. 

Moreover, the lack of awareness might affect the growth of the market in the forecast period. In addition, the high cost associated with orphan drugs might hamper the growth of the global market. 

Cumulative Evaluation of The Market

While looking into the past records, the global Market has witnessed significant growth and is expected to continue to maintain the trend in the upcoming years.

As per the orphan diseases market analysis, the regional market players exist across regions like North America, Asia Pacific, and Europe, who contribute efficiently to enhance the market growth in the coming years. 

Orphan Diseases Market Segmentation

  • Orphan Diseases types of diseases Insights:

The global market has been classified as genetic disorders, oncological diseases, cardiovascular diseases, respiratory disorders, eye disorders, autoimmune disease, blood disorders, growth disorders, neurological disorders and others.

Further, the oncological disease is sub-divided into Pancreatic cancer, multiple myeloma, acute myeloid leukemia (AML), ovarian cancer, renal cell carcinoma, and others. In 2020, the oncological disease segment was anticipated to hold the largest orphan diseases market share due to the increasing awareness about cancer among the population, rise in prevalence of oncological disease, availability of oncological orphan drugs for all to treat cancer, and early screening of cancer. Further, this disease is estimated to maintain the dominating place throughout the research period. 

  • Orphan Diseases End-users Insights:

The global market has been segregated into a research laboratory, hospital and clinics, and others depending on the end-user segment.

Among these, the hospital and clinic segments are estimated to generate the largest orphan diseases market size during the forecast period. This segment is growing due to the rising number of drugs administered by professional healthcare workers at the hospital. 

  • Orphan Diseases drug type Insights:

On the basis of drug type segment, the global market has been divided into non-biologics and biologics. 

In 2019, the biologics segment was projected to dominate the global market by generating significant market share. On the contrary, the non-biologics segment is registered a low CAGR comparatively and generated a lower market share for the year.

  • Orphan Diseases treatment type Insights:

The global market has been segregated into drug therapy, cell therapy, gene therapy, and others in terms of treatment type segment. 

Regional Analysis 

Based on the regional segmentation, the global orphan diseases market has been spread across different geographical locations such as North America, Asia Pacific, South America, Europe, and the rest of the World. 

In the present global scenario, the North American region is estimated to dominate the global market by generating the largest market share throughout the research period. This region is expanding due to the presence of key players leading to the development of innovative and sophisticated products. Moreover, the strong patient population and the significant expenditure on orphan drugs are also contributing to the North American regional market growth. 

Apart from this region, the European market for Orphan Diseases is considered to be the second-largest growing market in the forecast period. This region has strong adoption of sophisticated rare disease therapeutics, which is fuelling the European market for Orphan Diseases. Moreover, the Asia Pacific market for Orphan Diseases is anticipated to expand at a significant rate throughout the research period due to the growing awareness of the rare disease and rising healthcare expenditure. Furthermore, the rest of the World, including the Middle East & Africa, and Latin America, is estimated to show steady growth during the forecast period because of the under penetration of the market for Orphan Diseases.

Competitive Intensity Within the Industry  

As per the Orphan Diseases Market Outlook report, the market players are the main contributors to the growth of the market for Orphan Diseases. These major market players include:

  • Bristol-Myers Squibb Company (US), 


  • Novartis AG (Switzerland), 

  • Pfizer Inc. (US), 

  • AbbVie Inc. (US),

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

  • Alexion (US), 

  • Vertex Pharmaceuticals Incorporated (US), 

  • Merck & Co., Inc (US),

  • Sanofi (French),

  • GlaxoSmithKline plc. (UK).

These market players are spread across all parts of the globe and focus on the research and developmental activities relating to Orphan Diseases. Moreover, they are also doing partnerships, collaborations, mergers and acquisitions to enhance the orphan diseases market growth. Likely, they are aiming to show their presence to the World in the upcoming years. 

Recent Market Developments 

  • In June 2020, the prominent market player Agios Pharmaceuticals, Inc. declared that the company had received the Orphan Drug Designation of FDA for their Mitapivat pipeline candidate to treat Thalassemia.

  • In June 2020, the major key player Chiasma, Inc. declared that it had received approval from FDA for its Mycapssa product to treat a rare disorder, i.e., acromegaly. 

Report Overview 

The global market report highlights the market overview, segmentation, regional classification, competitive analysis, recent developments and market dynamics. This report is a qualitative and quantitative analysis of the market. 

According to the global orphan diseases market forecast report, the current market has been segmented based on the types of disease, end-users, and treatment type. 

Key Industrial Segments 

Orphan Diseases types of diseases Outlook:

  • genetic disorders, 

  • cancer, 

  • cardiovascular diseases, 

  • respiratory disorders, 

  • eye disorders, 

  • autoimmune disorders, 

  • blood disorders, 

  • growth disorder, 

  • neurological disorders, 

  • digestive disorders, 

  • others.

Orphan Diseases drug types Outlook

  • non-biologics 

  • biologics

Orphan Diseases End-users Outlook

  • research laboratory, 

  • hospital and clinics, 

  • others.

Orphan Diseases treatment type Outlook

  • drug therapy, 

  • cell therapy, 

  • gene therapy, 

  • others. 

Report Scope:

Report Attribute/Metric Details
  Market Size   USD 328.5 Billion
  CAGR   10.70%
  Base Year   2021
  Forecast Period   2022-2030
  Historical Data   2020
  Forecast Units   Value (USD Billion)
  Report Coverage   Revenue Forecast, Competitive Landscape, Growth Factors, and Trends
  Segments Covered   Type of Diseases, Type of Treatment, End User
  Geographies Covered   North America, Europe, Asia-Pacific, and Rest of the World (RoW)
  Key Vendors   Bristol-Myers Squibb Company (US), Novartis AG (Switzerland), CELGENE CORPORATION (US), F. Hoffmann-La Roche Ltd (Switzerland), Pfizer Inc. (US), Alexion (US), Sanofi (French), Vertex Pharmaceuticals Incorporated (US), GlaxoSmithKline plc. (UK), Merck & Co., Inc (US), AbbVie Inc. (US)
  Key Market Opportunities

  • Growth in novel indications for known orphan drugs
  • Untapped emerging economies
  •   Key Market Drivers

  • The rising prevalence of rare diseases
  • Favorable government policies.

  • Frequently Asked Questions (FAQ) :

    The Orphan Diseases Market is projected grow at approximately 10.70% CAGR during the assessment period (2022-2030).

    Novartis AG (Switzerland), Bristol-Myers Squibb Company (US), CELGENE CORPORATION (US), Pfizer Inc. (US), F. Hoffmann-La Roche Ltd (Switzerland), Alexion (US), Vertex Pharmaceuticals Incorporated (US), Sanofi (French), GlaxoSmithKline plc. (UK), AbbVie Inc. (US), and Merck & Co., Inc. (US), are some of the major players operating in the Global Orphan Diseases Market.

    The valuation of the Orphan Diseases Market is estimated to increase to USD 328.5 BN by the end of 2030.

    Spurting growth in the Healthcare sector and growing awareness among populaces are the major tailwinds pushing the growth of the Orphan Diseases Market.

    North America holds the largest share in the Orphan Diseases Market, followed by Europe and the Asia Pacific, respectively.

    Orphan diseases market has been segmented on the basis of types of diseases which includes autoimmune disorders, genetic disorders, blood disorders, cancer, growth disorder, cardiovascular diseases, neurological disorders, respiratory disorders, digestive disorders, eye disorders and others.

    Key Questions Answered

    • Global Market Outlook
    • In-depth analysis of global and regional trends
    • Analyze and identify the major players in the market, their market share, key developments, etc.
    • To understand the capability of the major players based on products offered, financials, and strategies.
    • Identify disrupting products, companies, and trends.
    • To identify opportunities in the market.
    • Analyze the key challenges in the market.
    • Analyze the regional penetration of players, products, and services in the market.
    • Comparison of major players’ financial performance.
    • Evaluate strategies adopted by major players.
    • Recommendations
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