Global Rare Diseases Treatment Market is valued at approximately USD 119.6 billion in 2021 and is anticipated to grow with a healthy growth rate of more than 12.8% over the forecast period 2022-2029. Rare Diseases are diseases that affect a small percentage of people. It is coded as ICD-11 by the world health organization, ICD-11 consists of around 5500 rare diseases. Rare disease can also be termed as an orphan disease, a condition that lacks a market size large enough to attract funding and resources for the development of remedies, barring the government from giving favorable economic terms for developing and commercializing such treatments. According to the US Food and Drug Administration, a rare disease is one that affects less than two lakh persons in the country. Increasing approval and launch of new orphan drugs, increasing prevalence of rare diseases, government support and initiatives for the treatment of rare diseases, increasing research and development in the sector, and favorable government policies are the factors contributing to the growth of the market.
According to the National Institutes of Health, One in ten Americans (about 30 million people) have a rare condition, and there are approximately 7,000 identified rare diseases in the US. Additionally, In January 2022, Amgen announced the approval of LUMAKRAS® drug in Japan for the treatment of rare cancer, and in October 2021, Scemblix drug was approved by the Food and Drug Administration for patients with chronic-phase (CP)-CML as a third line of treatment. Increasing approval and launch of new orphan drugs and increasing prevalence of rare diseases are the major factors driving the growth of the market. Furthermore, Increasing initiatives by the government and increasing awareness of rare diseases are the factors creating major market opportunities in the forecast period. However, the high cost of Rare Diseases Treatment stifles market growth throughout the forecast period of 2022-2029.
The key regions considered for the Global Rare Diseases Treatment Market study includes Asia Pacific, North America, Europe, Latin America, and Rest of the World. North America dominated the market in terms of revenue, owing to the dominance of rare diseases, easy accessibility to products, new product approval, and increasing government support and investment in the region. Asia Pacific is expected to grow with the highest CAGR during the forecast period, owing to factors such as rising government initiatives, increasing prevalence of orphan diseases, geographic expansion of key players, and active participation of government.
Major market players included in this report are:
- F. Hoffmann-La Roche Ltd.
- Pfizer, Inc
- PTC Therapeutics
- AstraZeneca
- Novartis AG
- Novo Nordisk Pharmatech A/S
- Takeda Pharmaceutical Company Limited
- Bayer AG
- Merck & Co., Inc.
- Bristol Myers Squibb Company
Recent Developments in the Market:
- In July 2021, AstraZeneca acquired Alexion Pharmaceuticals, Inc. to enjoy a stronger scientific foothold in immunology and to continue to lead the way in the research and development of drugs for people with rare diseases through Alexion's cutting-edge complement-biology platform and a strong pipeline.
- In November 2022, The FDA received a new Biologics License Application (BLA) for PRX-102 (pegunigalsidasealfa) from ProtalixBiotherapeutics Inc. and Chiesi Global Rare Diseases for the treatment of adult Fabry disease patients.
Global Rare Diseases Treatment Market Report Scope:
- Historical Data 2019-2020-2021
- Base Year for Estimation 2021
- Forecast period 2022-2029
- Report Coverage Revenue forecast, Company Ranking, Competitive Landscape, Growth factors, and Trends
- Segments Covered Therapeutic Area, Route of Administration, Drug Type, Distribution Channel, Region
- Regional Scope North America; Europe; Asia Pacific; Latin America; Rest of the World
- Customization Scope Free report customization (equivalent up to 8 analyst's working hours) with purchase. Addition or alteration to country, regional & segment scope*
The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within countries involved in the study.
The report also caters detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, it also incorporates potential opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and Therapeutic Area offerings of key players. The detailed segments and sub-segment of the market are explained below:
By Therapeutic Area:
- Cancer
- Neurological Conditions
- Cardiovascular Conditions
- Musculoskeletal Conditions
- Hematologic Disorders
- Infectious Diseases
- Metabolic disorders
- Endocrine disorders
- Others
By Drug Type:
- Biologics
- Biosimilar
- Small Molecule
By Route of Administration:
By Distribution Channel:
- Hospital Pharmacy
- Specialty Pharmacy
- Online Pharmacy
By Region:
- North America
- U.S.
- Canada
- Europe
- UK
- Germany
- France
- Spain
- Italy
- ROE
- Asia Pacific
- China
- India
- Japan
- Australia
- South Korea
- RoAPAC
- Latin America
- Brazil
- Mexico
- Rest of the World
Table of Contents
Chapter 1. Executive Summary
- 1.1. Market Snapshot
- 1.2. Global & Segmental Market Estimates & Forecasts, 2019-2029 (USD Billion)
- 1.2.1. Rare Diseases Treatment Market, by Region, 2019-2029 (USD Billion)
- 1.2.2. Rare Diseases Treatment Market, by Therapeutic Area, 2019-2029 (USD Billion)
- 1.2.3. Rare Diseases Treatment Market, by Drug Type, 2019-2029 (USD Billion)
- 1.2.4. Rare Diseases Treatment Market, by Route of Administration, 2019-2029 (USD Billion)
- 1.2.5. Rare Diseases Treatment Market, by Distribution Channel, 2019-2029 (USD Billion)
- 1.3. Key Trends
- 1.4. Estimation Methodology
- 1.5. Research Assumption
Chapter 2. Global Rare Diseases Treatment Market Definition and Scope
- 2.1. Objective of the Study
- 2.2. Market Definition & Scope
- 2.2.1. Scope of the Study
- 2.2.2. Industry Evolution
- 2.3. Years Considered for the Study
- 2.4. Currency Conversion Rates
Chapter 3. Global Rare Diseases Treatment Market Dynamics
- 3.1. Rare Diseases Treatment Market Impact Analysis (2019-2029)
- 3.1.1. Market Drivers
- 3.1.1.1. Increasing approval and launch of new orphan drugs
- 3.1.1.2. Increasing prevalence of rare diseases
- 3.1.2. Market Challenges
- 3.1.2.1. High Cost of Rare Diseases Treatment
- 3.1.3. Market Opportunities
- 3.1.3.1. Increasing initiatives by the government
- 3.1.3.2. Increasing awareness of rare diseases
Chapter 4. Global Rare Diseases Treatment Market Industry Analysis
- 4.1. Porter's 5 Force Model
- 4.1.1. Bargaining Power of Suppliers
- 4.1.2. Bargaining Power of Buyers
- 4.1.3. Threat of New Entrants
- 4.1.4. Threat of Substitutes
- 4.1.5. Competitive Rivalry
- 4.2. Futuristic Approach to Porter's 5 Force Model (2019-2029)
- 4.3. PEST Analysis
- 4.3.1. Political
- 4.3.2. Economical
- 4.3.3. Social
- 4.3.4. Technological
- 4.4. Investment Adoption Model
- 4.5. Analyst Recommendation & Conclusion
- 4.6. Top investment opportunity
- 4.7. Top winning strategies
Chapter 5. Risk Assessment: COVID-19 Impact
- 5.1. Assessment of the overall impact of COVID-19 on the industry
- 5.2. Pre COVID-19 and post COVID-19 Market scenario
Chapter 6. Global Rare Diseases Treatment Market, by Therapeutic Area
- 6.1. Market Snapshot
- 6.2. Global Rare Diseases Treatment Market by Therapeutic Area, Performance - Potential Analysis
- 6.3. Global Rare Diseases Treatment Market Estimates & Forecasts by Therapeutic Area 2019-2029 (USD Billion)
- 6.4. Rare Diseases Treatment Market, Sub Segment Analysis
- 6.4.1. Cancer
- 6.4.2. Neurological Conditions
- 6.4.3. Cardiovascular Conditions
- 6.4.4. Musculoskeletal Conditions
- 6.4.5. Hematologic Disorders
- 6.4.6. Infectious Diseases
- 6.4.7. Metabolic disorders
- 6.4.8. Endocrine disorders
- 6.4.9. Others
Chapter 7. Global Rare Diseases Treatment Market, by Drug Type
- 7.1. Market Snapshot
- 7.2. Global Rare Diseases Treatment Market by Drug Type, Performance - Potential Analysis
- 7.3. Global Rare Diseases Treatment Market Estimates & Forecasts by Drug Type 2019-2029 (USD Billion)
- 7.4. Rare Diseases Treatment Market, Sub Segment Analysis
- 7.4.1. Biologics
- 7.4.2. Biosimilar
- 7.4.3. Small Molecule
Chapter 8. Global Rare Diseases Treatment Market, by Route of Administration
- 8.1. Market Snapshot
- 8.2. Global Rare Diseases Treatment Market by Route of Administration, Performance - Potential Analysis
- 8.3. Global Rare Diseases Treatment Market Estimates & Forecasts by Route of Administration 2019-2029 (USD Billion)
- 8.4. Rare Diseases Treatment Market, Sub Segment Analysis
- 8.4.1. Oral
- 8.4.2. Injectable
- 8.4.3. Others
Chapter 9. Global Rare Diseases Treatment Market, by Distribution Channel
- 9.1. Market Snapshot
- 9.2. Global Rare Diseases Treatment Market by Distribution Channel, Performance - Potential Analysis
- 9.3. Global Rare Diseases Treatment Market Estimates & Forecasts by Distribution Channel 2019-2029 (USD Billion)
- 9.4. Rare Diseases Treatment Market, Sub Segment Analysis
- 9.4.1. Hospital Pharmacy
- 9.4.2. Specialty Pharmacy
- 9.4.3. Online Pharmacy
Chapter 10. Global Rare Diseases Treatment Market, Regional Analysis
- 10.1. Rare Diseases Treatment Market, Regional Market Snapshot
- 10.2. North America Rare Diseases Treatment Market
- 10.2.1. U.S. Rare Diseases Treatment Market
- 10.2.1.1. Therapeutic Area breakdown estimates & forecasts, 2019-2029
- 10.2.1.2. Drug Type breakdown estimates & forecasts, 2019-2029
- 10.2.1.3. Route of Administration breakdown estimates & forecasts, 2019-2029
- 10.2.1.4. Distribution Channel breakdown estimates & forecasts, 2019-2029
- 10.2.2. Canada Rare Diseases Treatment Market
- 10.3. Europe Rare Diseases Treatment Market Snapshot
- 10.3.1. U.K. Rare Diseases Treatment Market
- 10.3.2. Germany Rare Diseases Treatment Market
- 10.3.3. France Rare Diseases Treatment Market
- 10.3.4. Spain Rare Diseases Treatment Market
- 10.3.5. Italy Rare Diseases Treatment Market
- 10.3.6. Rest of Europe Rare Diseases Treatment Market
- 10.4. Asia-Pacific Rare Diseases Treatment Market Snapshot
- 10.4.1. China Rare Diseases Treatment Market
- 10.4.2. India Rare Diseases Treatment Market
- 10.4.3. Japan Rare Diseases Treatment Market
- 10.4.4. Australia Rare Diseases Treatment Market
- 10.4.5. South Korea Rare Diseases Treatment Market
- 10.4.6. Rest of Asia Pacific Rare Diseases Treatment Market
- 10.5. Latin America Rare Diseases Treatment Market Snapshot
- 10.5.1. Brazil Rare Diseases Treatment Market
- 10.5.2. Mexico Rare Diseases Treatment Market
- 10.6. Rest of The World Rare Diseases Treatment Market
Chapter 11. Competitive Intelligence
- 11.1. Top Market Strategies
- 11.2. Company Profiles
- 11.2.1. F. Hoffmann-La Roche Ltd.
- 11.2.1.1. Key Information
- 11.2.1.2. Overview
- 11.2.1.3. Financial (Subject to Data Availability)
- 11.2.1.4. Product Summary
- 11.2.1.5. Recent Developments
- 11.2.2. Pfizer, Inc
- 11.2.3. PTC Therapeutics
- 11.2.4. AstraZeneca
- 11.2.5. Novartis AG
- 11.2.6. Novo Nordisk Pharmatech A/S
- 11.2.7. Takeda Pharmaceutical Company Limited
- 11.2.8. Bayer AG
- 11.2.9. Merck & Co., Inc.
- 11.2.10. Bristol Myers Squibb Company
Chapter 12. Research Process
- 12.1. Research Process
- 12.1.1. Data Mining
- 12.1.2. Analysis
- 12.1.3. Market Estimation
- 12.1.4. Validation
- 12.1.5. Publishing
- 12.2. Research Attributes
- 12.3. Research Assumption