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Global Rare Diseases Treatment Market to reach USD 313.5 billion by the end of 2029

Global Rare Diseases Treatment Market Size study & Forecast, by Therapeutic Area (Cancer, Neurological Conditions, Cardiovascular Conditions, Musculoskeletal Conditions, Hematologic Disorders, Infectious Diseases, Metabolic disorders, Endocrine disorders, Others) by Route of Administration (Oral, Injectable, Others), by Drug Type (Biologics, Biosimilar, Small Molecule), by Distribution Channel (Hospital Pharmacy, Specialty Pharmacy, Online Pharmacy) and Regional Analysis, 2022-2029

Product Code: HLSHS-85415180
Publish Date: 20-03-2023
Page: 200

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 player included in this report are:
F. Hoffmann-La Roche Ltd.
Pfizer, Inc
PTC Therapeutics
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:
Neurological Conditions
Cardiovascular Conditions
Musculoskeletal Conditions
Hematologic Disorders
Infectious Diseases
Metabolic disorders
Endocrine disorders

By Drug Type:
Small Molecule

By Route of Administration:

By Distribution Channel:
Hospital Pharmacy
Specialty Pharmacy
Online Pharmacy

By Region:
North America
Asia Pacific
South Korea
Latin America
Rest of the World

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 Increasing approval and launch of new orphan drugs Increasing prevalence of rare diseases
3.1.2. Market Challenges High Cost of Rare Diseases Treatment
3.1.3. Market Opportunities Increasing initiatives by the government 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 Therapeutic Area breakdown estimates & forecasts, 2019-2029 Drug Type breakdown estimates & forecasts, 2019-2029 Route of Administration breakdown estimates & forecasts, 2019-2029 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. Key Information Overview Financial (Subject to Data Availability) Product Summary 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

At Bizwit Research and Consultancy, we employ a thorough and iterative research methodology with the goal of minimizing discrepancies, ensuring the provision of highly accurate estimates and predictions over the forecast period. Our approach involves a combination of bottom-up and top-down strategies to effectively segment and estimate quantitative aspects of the market, utilizing our proprietary data & AI tools. Our Proprietary Tools allow us for the creation of customized models specific to the research objectives. This enables us to develop tailored statistical models and forecasting algorithms to estimate market trends, future growth, or consumer behavior. The customization enhances the accuracy and relevance of the research findings.
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Critical elements of methodology employed for all our studies include:
Data Collection:
To determine the appropriate methods of data collection based on the research objectives, we consider both primary and secondary sources. Primary data collection involves gathering information directly from various industry experts in core and related fields, original equipment manufacturers (OEMs), vendors, suppliers, technology developers, alliances, and organizations. These sources encompass all segments of the value chain within the specific industry. Through in-depth interviews, we engage with key industry participants, subject-matter experts, C-level executives of major market players, industry consultants, and other relevant experts. This allows us to obtain and validate critical qualitative and quantitative information while evaluating market prospects. AI and Big Data are instrumental in our primary research, providing us with powerful tools to collect, analyze, and derive insights from data efficiently. These technologies contribute to the advancement of research methodologies, enabling us to make data-driven decisions and uncover valuable findings.
In addition to primary sources, we extensively utilize secondary sources to enhance our research. These include directories, databases, journals focusing on related industries, company newsletters, and information portals such as Bloomberg, D&B Hoovers, and Factiva. These secondary sources enable us to identify and gather valuable information for our comprehensive, technical, market-oriented, and commercial study of the market. Additionally, we utilize AI algorithms to automate the collection of vast amounts of data from various sources such as surveys, social media platforms, online transactions, and web scraping. And employ Big Data technologies for storage and processing of large datasets, ensuring that no valuable information is missed during the data collection process.
Data Analysis:
Our team of experts carefully examine the gathered data using suitable statistical techniques and qualitative analysis methods. For quantitative analysis, we employ descriptive statistics, regression analysis, and other advanced statistical methods, depending on the characteristics of the data. This analysis may also incorporate the utilization of AI tools and big data analysis techniques to extract meaningful insights.
To ensure the accuracy and reliability of our findings, we extensively leverage data science techniques, which help us minimize discrepancies and uncertainties in our analysis. We employ Data Science to clean and preprocess the data, ensuring its quality and reliability. This involves handling missing data, removing outliers, standardizing variables, and transforming data into suitable formats for analysis. The application of data science techniques enhances our accuracy, efficiency, and depth of analysis, enabling us to stay competitive in dynamic market environments.
Market Size Estimation:
Our proprietary data tools play a crucial role in deriving our market estimates and forecasts. Each study involves the creation of a unique and customized model. The model incorporates the gathered information on market dynamics, technology landscape, application development, and pricing trends. AI techniques, such as machine learning and deep learning, aid us to analyze patterns within the data to identify correlations, trends, and relationships. By recognizing patterns in consumer behavior, purchasing habits, or market dynamics, our AI algorithms aid us in more precise estimations of market size. These factors are simultaneously analyzed within the model, allowing for a comprehensive assessment. To quantify their impact over the forecast period, correlation, regression, and time series analysis are employed.
To estimate and validate the market size, we employ both top-down and bottom-up approaches. The preference is given to a bottom-up approach, where key regional markets are analyzed as separate entities. This data is then integrated to obtain global estimates. This approach is crucial as it provides a deep understanding of the industry and helps minimize errors.
In our forecasting process, we consider various parameters such as economic tools, technological analysis, industry experience, and domain expertise. By taking all these factors into account, we strive to produce accurate and reliable market forecasts. When forecasting, we take into consideration several parameters, which include:
Market driving trends and favorable economic conditions
Restraints and challenges that are expected to be encountered during the forecast period.
Anticipated opportunities for growth and development
Technological advancements and projected developments in the market
Consumer spending trends and dynamics
Shifts in consumer preferences and behaviors.
The current state of raw materials and trends in supply versus pricing
Regulatory landscape and expected changes or developments.
The existing capacity in the market and any expected additions or expansions up to the end of the forecast period.
To assess the market impact of these parameters, we assign weights to each one and utilize weighted average analysis. This process allows us to quantify their influence on the market and derive an expected growth rate for the forecasted period. By considering these various factors and applying a weighted analysis approach, we strive to provide accurate and reliable market forecasts.
Insight Generation & Report Presentation:
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Next, we create a well-structured research report that effectively communicates the research findings, insights, and recommendations to the client. To enhance clarity and comprehension, we utilize visual aids such as charts, graphs, and tables. These visual elements are employed to present the data in an engaging and easily understandable format, ensuring that the information is accessible and visually appealing to the client. Our aim is to deliver a clear and concise report that conveys the research findings effectively and provides actionable recommendations to meet the client’s specific needs.

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