Orphan Drug Market Size, Share, Opportunities, And Trends By Disease Type (Oncologic Disease, Metabolic Disease, Hematologic and Immunologic Disease, Infectious Disease, Neurologic Disease, Others), By Product Type (Biological Products, Non-biological Products), And By Geography - Forecasts From 2025 to 2030

Orphan Drug Market Size:

The Orphan Drug Market is expected to grow from US$253.667 billion in 2025 to US$398.644 billion in 2030, at a CAGR of 9.46%.

During the forecast period, prescription sales in the orphan drug sector are projected to grow at twice the rate of the overall prescription market. However, various government authorities encourage to development and market of orphan drugs. The cost incurred during the research and development of orphan drugs is higher when compared with non-orphan drugs. The orphan drug market is classified into various segments by disease types, indication, and geographical regions. In the orphan drugs disease type segment, oncologic diseases occupied a significant share of the market and the segment is predicted to maintain the attained growth trend during the forecast period. This is due to the growing prevalence of different forms of rare cancers, such as leukemia, myeloma, angiosarcoma, and others prevalent in the patient population.

Orphan Drug Market Segmentation Analysis:

• By Disease Type

The global orphan drug market is segmented into various types of diseases orphan drug manufacturing companies target, namely oncologic diseases, metabolic diseases, hematologic and immunologic diseases, infectious diseases, neurologic diseases, and other RARE Diseases. The Oncologic segment of the orphan drug market was the most dominant sector due to a large number of rare cancer form cases found in patients. The oncology segment is expected to maintain the trend and will be the key segment during the period of the next five years.

• By Product Type

The global orphan drug market is segmented into two segments on basis of the origin of their components, biological or non-biological. The global orphan drug market is offering products that are driven by biological or non-biological sources. Biological products are costlier as extraction and purification take large investments and also sometimes sources may be rare which adds to the cost.

Orphan Drug Market Geographical Outlook:

The global orphan drug market is expanding globally to cure rare diseases and is thus expected to grow in all regions. North America is estimated to have a significant market share due to the high number of research and development going on in the region coupled with the high medical costs and skilled labor force available in the region.

Orphan Drug Market Competitive Insights:

The orphan drug market is competitive owing to the presence of well-diversified global players. Various pioneer companies in the orphan drug market are Bayer, Amryt Pharma Plc., Bristol-Myers Squibb Company, Novartis AG, Celgene Corporation, F. Hoffmann-La Roche AG, and Pfizer Inc. among others.

Orphan Drug Market Scope:

Report Metric	Details
Orphan Drug Market Size in 2025	US$253.667 billion
Orphan Drug Market Size in 2030	US$398.644 billion
Growth Rate	CAGR of 9.46%
Study Period	2020 to 2030
Historical Data	2020 to 2023
Base Year	2024
Forecast Period	2025 – 2030
Forecast Unit (Value)	USD Billion
Segmentation	Disease Type Product Type Geography
Geographical Segmentation	North America, South America, Europe, Middle East and Africa, Asia Pacific
List of Major Companies in Orphan Drug Market	Bayer Amryt Pharma PLC Bristol-Myers Squibb Company Novartis AG Celgene Corporation
Customization Scope	Free report customization with purchase

 

The Orphan Drug Market is analyzed into the following segments:

• By Disease Type

• • Oncologic Disease
  • Metabolic Disease
  • Hematologic and Immunologic Disease
  • Infectious Disease
  • Neurologic Disease
  • Others

• By Product Type

• • Biological Products
  • Non-biological Products

• By Geography

• • North America

• • • USA
    • Canada
    • Others

• • South America

• • • Brazil
    • Others

• • Europe

• • • Germany
    • France
    • United Kingdom
    • Others

• • Middle East and Africa

• • • Saudi Arabia
    • Israel
    • Others

• • Asia Pacific

• • • China
    • Japan
    • South Korea
    • India
    • Others

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1. INTRODUCTION

1.1. Market Overview

1.2. Market Definition

1.3. Scope of the Study

1.4. Currency

1.5. Assumptions

1.6. Base, and Forecast Years Timeline

2. RESEARCH METHODOLOGY  

2.1. Research Design

2.2. Secondary Sources

3. EXECUTIVE SUMMARY

4. MARKET DYNAMICS

4.1. Market Segmentation

4.2. Market Drivers

4.3. Market Restraints

4.4. Market Opportunities

4.5. Porter’s Five Force Analysis

4.5.1. Bargaining Power of Suppliers

4.5.2. Bargaining Power of Buyers

4.5.3. Threat of New Entrants

4.5.4. Threat of Substitutes

4.5.5. Competitive Rivalry in the Industry

4.6. Life Cycle Analysis - Regional Snapshot

4.7. Market Attractiveness

5. ORPHAN DRUG MARKET BY  DISEASE TYPE

5.1. Oncologic Disease

5.2. Metabolic Disease

5.3. Hematologic and Immunologic Disease

5.4. Infectious Disease

5.5. Neurologic Disease

5.6. Others

6. ORPHAN DRUG MARKET BY PRODUCT TYPE

6.1. Biological Products

6.2. Non-Biological Products

7. ORPHAN DRUG MARKET BY GEOGRAPHY

7.1. North America

7.1.1. USA

7.1.2. Canada

7.1.3. Others

7.2. South America

7.2.1. Brazil

7.2.2. Others

7.3. Europe

7.3.1. Germany

7.3.2. France

7.3.3. United Kingdom

7.3.4. Others

7.4. Middle East and Africa

7.4.1. Saudi Arabia

7.4.2. Israel

7.4.3. Others

7.5. Asia Pacific

7.5.1. China

7.5.2. Japan

7.5.3. South Korea

7.5.4. India

7.5.5. Others

8. COMPETITIVE INTELLIGENCE

8.1.  Investment Analysis

8.2.  Recent Investment and Deals

8.3. Strategies of Key Players

9. COMPANY PROFILES

9.1. Bayer

9.1.1. Company Overview

9.1.2. Financials

9.1.3. Products and Services

9.1.4. Recent Developments

9.2. Amryt Pharma PLC

9.2.1. Company Overview

9.2.2. Financials

9.2.3. Products and Services

9.2.4. Recent Developments

9.3. Brisol-Myers Squibb Company

9.3.1. Company Overview

9.3.2. Financials

9.3.3. Products and Services

9.3.4. Recent Developments

9.4.  Novartis AG

9.4.1. Company Overview

9.4.2. Financials

9.4.3. Products and Services

9.4.4. Recent Developments

9.5.  Celgene Corporation

9.5.1. Company Overview

9.5.2. Financials

9.5.3. Products and Services

9.5.4. Recent Developments

9.6. F.Hoffman-La Roche AG

9.6.1. Company Overview

9.6.2. Financials

9.6.3. Products and Services

9.6.4. Recent Developments

9.7. Pfizer Inc.

9.7.1. Company Overview

9.7.2. Financials

9.7.3. Products and Services

9.7.4. Recent Developments

9.8. Alexion

9.8.1. Company Overview

9.8.2. Financials

9.8.3. Products and Services

9.8.4. Recent Developments

9.9. Sanofi

9.9.1. Company Overview

9.9.2. Financials

9.9.3. Products and Services

9.9.4. Recent Developments

9.10. AbbVie, Inc.

9.10.1. Company Overview

9.10.2. Financials

9.10.3. Products and Services

9.10.4. Recent Developments

9.11. RegeneRX Biopharmaceuticals, Inc.

9.11.1. Company Overview

9.11.2. Financials

9.11.3. Products and Services

9.11.4. Recent Developments

9.12. Johnson & Johnson Services, Inc.

9.12.1. Company Overview

9.12.2. Financials

9.12.3. Products and Services

9.12.4. Recent Developments

9.13. Shire PLC

9.13.1. Company Overview

9.13.2. Financials

9.13.3. Products and Services

9.13.4. Recent Developments

9.14. Merck & Co. Inc.

9.14.1. Company Overview

9.14.2. Financials

9.14.3. Products and Services

9.14.4. Recent Developments

9.15. AstraZeneca

9.15.1. Company Overview

9.15.2. Financials

9.15.3. Products and Services

9.15.4. Recent Developments

LIST OF FIGURES

LIST OF TABLES

Bayer

Amryt Pharma PLC

Bristol-Myers Squibb Company

Novartis AG

Celgene Corporation

F.Hoffman-La Roche AG

Pfizer Inc.

Alexion

Sanofi 

AbbVie, Inc.

RegeneRX Biopharmaceuticals, Inc.

Johnson & Johnson Services, Inc.

Shire PLC

Merck & Co. Inc.

AstraZeneca