Worldwide Orphan Drugs Industry to 2030 - Use of Orphan Drugs for Novel Conditions/Indications Presents Opportunities


Dublin, June 15, 2022 (GLOBE NEWSWIRE) -- The "Orphan Drugs Market by Disease Type: Global Opportunity Analysis and Industry Forecast, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

The global orphan drugs market was valued at $140,000.0 million in 2020 and is projected to reach $435,686.3 million by 2030 registering a CAGR of 11.8% from 2021 to 2030.

The pharmaceutical agents administered to treat, diagnose, and prevent life-threatening diseases (rare diseases) are known as orphan drugs. Orphan drugs are used to treat rare (orphaned) diseases prevalent in a very small patient pool. Thus, the pharmaceutical industry takes little interest in their development. Moreover, their development requires high cost and the chances of returns on investment are comparatively less when compared to non-orphan drugs. These drugs treat different rare diseases, such as non-Hodgkin lymphoma, acute myeloid leukemia, cystic fibrosis, and Duchenne muscular dystrophy, graft vs. host disease, and others.

The main factor that drives the growth of the orphan drugs market includes increase in prevalence of rare diseases. In addition, favorable government policies, and availability of market exclusivity for orphan drugs developers further supplement the global orphan drugs market growth.

Conversely, limited patient pool for clinical trials and product marketing, and high treatment costs per patient are expected to obstruct the growth of the market during the forecast years. On the other hand, high growth potential in untapped emerging economies, and rise in novel indications for known orphan drugs are expected to offer profitable opportunities for the growth of the market during the forecast period. The global orphan drugs market is segmented on the basis of disease type, and region to provide a detailed assessment of the market.

By disease type, it is categorized into oncological diseases, metabolic diseases, hematologic & immunologic diseases, infectious diseases, neurological diseases, and other rare diseases such as transplantation diseases, gastrointestinal diseases, dermatologic diseases, perinatal & congenital abnormalities, and others. Oncological diseases segment is further divided into acute myeloid leukemia (AML), pancreatic cancer, ovarian cancer, multiple myeloma, renal cell carcinoma, and others.

In addition, the metabolic diseases segment is again classified into hunter syndrome, Fabry disease, Gaucher disease, hypoparathyroidism, and others. Furthermore, hematologic & immunologic diseases segment is segmented into hereditary angioedema (HAE), hemophilia, and others. Moreover, neurological diseases segment is further segmented into Duchenne muscular dystrophy (DMD), Alzheimer's disease, Huntington's disease, and others. Region wise, the market is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

The key market players profiled in the report include Abbvie Inc., Amgen Inc., Amryt Pharma Plc, Bristol-Myers Squibb Company, F. Hoffmann-La Roche AG, GlaxoSmithkline PLC, Johnson & Johnson (Janssen Global Services, LLC), Novartis AG (Sandoz), Pfizer, Inc., and Sanofi S.A.

Key Benefits

  • The study provides an in-depth analysis of the global orphan drugs market along with the current trends and future estimations to elucidate the imminent investment pockets
  • A comprehensive analysis of the factors that drive and restrict the market growth is provided in the report
  • Comprehensive quantitative analysis of the industry from 2020 to 2030 is provided to enable the stakeholders to capitalize on the prevailing market opportunities
  • Extensive analysis of the key segments of the industry helps to understand the application and products of orphan drugs used across the globe
  • Key market players and their strategies have been analyzed to understand the competitive outlook of the market

Key Topics Covered:

Chapter 1: Introduction

Chapter 2: Executive Summary

Chapter 3: Market Landscape
3.1. Market Definition and Scope
3.2. Key Findings
3.2.1. Top Investment Pockets
3.2.2. Top Winning Strategies
3.3. Porter'S Five Force Analysis
3.4. Top Player Positioning, 2020
3.5. Patent Analysis, 2017-2022
3.5.1. Patent Analysis, World
3.5.2. Patent Analysis, by Indication
3.6. Regulation
3.6.1. U. S.
3.6.2. Europe
3.6.3. Japan
3.7. Market Dynamics
3.7.1. Drivers
3.7.1.1. Increase in Prevalence of Rare Diseases
3.7.1.2. Favorable Government Policies
3.7.1.3. Availability of Exclusivity for Orphan Drugs
3.7.2. Restraints
3.7.2.1. Limited Patient Pool for Clinical Trials and Product Marketing
3.7.2.2. High Treatment Costs Per Patient
3.7.3. Opportunity
3.7.3.1. High Growth Potential in Untapped Emerging Economies
3.7.3.2. Use of Orphan Drugs for Novel Conditions/Indications
3.7.4. Impact Analysis
3.8. Covid-19 Impact Analysis on the Orphan Drugs Market

Chapter 4: Orphan Drugs Market, Disease Type
4.1. Overview
4.1.1. Market Size and Forecast
4.2. Oncological Diseases
4.2.1. Key Market Trends, Growth Factors, and Opportunities
4.2.2. Market Size and Forecast, by Region
4.2.3. Market Analysis, by Country
4.2.4. Market Size and Forecast, by Type
4.2.5. Acute Myeloid Leukemia (Aml)
4.2.5.1. Market Size and Forecast, by Region
4.2.5.2. Market Analysis, by Country
4.2.6. Pancreatic Cancer
4.2.6.1. Market Size and Forecast, by Region
4.2.6.2. Market Analysis, by Country
4.2.7. Ovarian Cancer
4.2.7.1. Market Size and Forecast, by Region
4.2.7.2. Market Analysis, by Country
4.2.8. Multiple Myeloma
4.2.8.1. Market Size and Forecast, by Region
4.2.8.2. Market Analysis, by Country
4.2.9. Renal Cell Carcinoma
4.2.9.1. Market Size and Forecast, by Region
4.2.9.2. Market Analysis, by Country
4.2.10. Others
4.2.10.1. Market Size and Forecast, by Region
4.2.10.2. Market Analysis, by Country
4.3. Metabolic Diseases
4.3.1. Key Market Trends, Growth Factors, and Opportunities
4.3.2. Market Size and Forecast, by Region
4.3.3. Market Analysis, by Country
4.3.4. Market Size and Forecast, by Type
4.3.5. Hunter Syndrome
4.3.5.1. Market Size and Forecast, by Region
4.3.5.2. Market Analysis, by Country
4.3.6. Fabry Disease
4.3.6.1. Market Size and Forecast, by Region
4.3.6.2. Market Analysis, by Country
4.3.7. Gaucher Disease
4.3.7.1. Market Size and Forecast, by Region
4.3.7.2. Market Analysis, by Country
4.3.8. Hypoparathyroidism
4.3.8.1. Market Size and Forecast, by Region
4.3.8.2. Market Analysis, by Country
4.3.9. Others
4.3.9.1. Market Size and Forecast, by Region
4.3.9.2. Market Analysis, by Country
4.4. Hematologic & Immunologic Diseases
4.4.1. Key Market Trends, Growth Factors, and Opportunities
4.4.2. Market Size and Forecast, by Region
4.4.3. Market Analysis, by Country
4.4.4. Market Size and Forecast, by Type
4.4.5. Hereditary Angioedema (Hae)
4.4.5.1. Market Size and Forecast, by Region
4.4.5.2. Market Analysis, by Country
4.4.6. Hemophilia
4.4.6.1. Market Size and Forecast, by Region
4.4.6.2. Market Analysis, by Country
4.4.7. Others
4.4.7.1. Market Size and Forecast, by Region
4.4.7.2. Market Analysis, by Country
4.5. Infectious Diseases
4.5.1. Key Market Trends, Growth Factors, and Opportunities
4.5.2. Market Size and Forecast, by Region
4.5.3. Market Analysis, by Country
4.6. Neurological Diseases
4.6.1. Key Market Trends, Growth Factors, and Opportunities
4.6.2. Market Size and Forecast, by Region
4.6.3. Market Analysis, by Country
4.6.4. Market Size and Forecast, by Type
4.6.5. Duchenne Muscular Dystrophy
4.6.5.1. Market Size and Forecast, by Region
4.6.5.2. Market Analysis, by Country
4.6.6. Alzheimer's Disease
4.6.6.1. Market Size and Forecast, by Region
4.6.6.2. Market Analysis, by Country
4.6.7. Huntington's Disease
4.6.7.1. Market Size and Forecast, by Region
4.6.7.2. Market Analysis, by Country
4.6.8. Others
4.6.8.1. Market Size and Forecast, by Region
4.6.8.2. Market Analysis, by Country
4.7. Other Rare Diseases
4.7.1. Key Market Trends, Growth Factors, and Opportunities
4.7.2. Market Size and Forecast, by Region
4.7.3. Market Analysis, by Country

Chapter 5: Orphan Drugs Market, by Region

Chapter 6: Company Profiles
6.1. Abbvie Inc.
6.1.1. Company Overview
6.1.2. Company Snapshot
6.1.3. Operating Business Segments
6.1.4. Product Portfolio
6.1.5. Business Performance
6.1.6. Key Strategic Moves and Developments
6.2. Amgen Inc.
6.2.1. Company Overview
6.2.2. Company Snapshot
6.2.3. Operating Business Segments
6.2.4. Product Portfolio
6.2.5. Business Performance
6.2.6. Key Strategic Moves and Developments
6.3. Amryt Pharma plc.
6.3.1. Company Overview
6.3.2. Company Snapshot
6.3.3. Operating Business Segments
6.3.4. Product Portfolio
6.3.5. Business Performance
6.3.6. Key Strategic Moves and Developments
6.4. Bristol-Myers Squibb Company
6.4.1. Company Overview
6.4.2. Company Snapshot
6.4.3. Operating Business Segments
6.4.4. Product Portfolio
6.4.5. Business Performance
6.4.6. Key Strategic Moves and Developments
6.5. F. Hoffmann-La Roche Ltd.
6.5.1. Company Overview
6.5.2. Company Snapshot
6.5.3. Operating Business Segments
6.5.4. Product Portfolio
6.5.5. Business Performance.
6.5.6. Key Strategic Moves and Developments
6.6. GlaxoSmithKline plc (Gsk)
6.6.1. Company Overview
6.6.2. Company Snapshot
6.6.3. Operating Business Segments
6.6.4. Product Portfolio
6.6.5. Business Performance
6.7. Johnson & Johnson (Janssen Global Services, LLC)
6.7.1. Company Overview
6.7.2. Company Snapshot
6.7.3. Operating Business Segments
6.7.4. Product Portfolio
6.7.5. Business Performance
6.7.6. Key Strategic Moves and Developments
6.8. Novartis International AG (Sandoz)
6.8.1. Company Overview
6.8.2. Company Snapshot
6.8.3. Operating Business Segments
6.8.4. Product Portfolio
6.8.5. Business Performance
6.8.6. Key Strategic Moves and Developments
6.9. Pfizer Inc.
6.9.1. Company Overview
6.9.2. Company Snapshot
6.9.3. Operating Business Segments
6.9.4. Product Portfolio
6.9.5. Business Performance
6.9.6. Key Strategic Moves and Developments
6.10. Sanofi S. A
6.10.1. Company Overview
6.10.2. Company Snapshot
6.10.3. Operating Business Segments
6.10.4. Product Portfolio
6.10.5. Business Performance
6.10.6. Key Strategic Moves and Developments

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Global Orphan Drugs Market

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