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Global Orphan Drugs Market to reach USD XX billion by the end of 2030.

Global Orphan Drugs Market Size study & Forecast, by Drug Type (Biological, Non-biological) by Top Selling Drugs (Revlimid, Darzalex, Rituxan, Tafinlar, Ninlaro, Other), by Disease Type (Oncology, Hematology, Neurology, Cardiovascular, Other Disease Types) and Regional Analysis, 2023-2030

Product Code: HLSDT-46332608
Publish Date: 27-06-2023
Page: 200

Global Orphan Drugs Market is valued approximately USD XX billion in 2022 and is anticipated to grow with a healthy growth rate of more than XX% over the forecast period 2023-2030. Orphan drugs, also known as orphan medicinal products, are pharmaceutical drugs developed to treat rare diseases or conditions. The prevalence of rare diseases among the world’s population has grown recently. Both emerging and industrialized nations have adopted policies to address this issue that encourage the discovery of medications for uncommon diseases and ensure that these medications are readily available. The Orphan Drugs market is expanding because of factors such as rising prevalence of rare diseases and favorable government policies.

According to the Genetic and Rare Diseases (GARD) Information Centre in May 2022, there are around 7,000 rare diseases, and one in ten Americans (nearly 30 million individuals) have one. Furthermore, according to statistics from “GlobalGenes” that was updated in September 2021, more than 400 million people globally are affected by uncommon diseases. Inherent genetic causes for 80% of uncommon disorders have been found. Furthermore, according to the study “Rare diseases: maintaining momentum” published in March 2022, drug companies invested USD 22.9 billion in total in research on rare disorders in 2021, a 28% increase from 2020. As a result, there are more prospects for new orphan medications due to rising prevalence and rising research and development spending on uncommon disease, which is ultimately driving the market growth. In addition, increasing research and development activities in the development of new therapies for rare diseases and strong product launches activities is creating a lucrative opportunity to the market. However, high per patient treatment cost and limited patient pool for clinical trials and product marketing stifles market growth throughout the forecast period of 2023-2030.

The key regions considered for the Global Orphan Drugs Market study includes Asia Pacific, North America, Europe, Latin America, and Middle East & Africa. North America dominated the market in 2022 owing to the dominance of branded products and the region’s increasing disease prevalence. Asia Pacific is expected to grow significantly during the forecast period, owing to factors such as rising target populations, an increase in the number of collaborations for product development, geographic expansion of key players, and active participation of government and nonprofit organizations in the market space.

Major market player included in this report are:
Alexion Pharmaceuticals Inc
Bristol-Myers Squibb Company (Celgene Corporation)
Novartis AG
Pfizer Inc.
F. Hoffmann-La Roche Ltd
Takeda Pharmaceutical Company Limited
Amgen Inc.
Sanofi S.A
Johnson & Johnson Pvt Ltd
AstraZeneca Plc

Recent Developments in the Market:
Ø In June 2022, The Food and medicine Administration (FDA) has designated ALX Oncology Holdings Inc.’s evorpacept, a next-generation CD47 blocker, as an orphan medicine for the treatment of acute myeloid leukaemia (AML).
Ø In March 2021, The Food and Drug Administration has granted orphan drug classification to Intellia Therapeutics’ CRISPR-edited T cell receptor (TCR) T-cell therapy for the treatment of acute myeloid leukaemia (AML).

Global Orphan Drugs Market Report Scope:
ü Historical Data – 2020 – 2021
ü Base Year for Estimation – 2022
ü Forecast period – 2023-2030
ü Report Coverage – Revenue forecast, Company Ranking, Competitive Landscape, Growth factors, and Trends
ü Segments Covered – Drug Type, Top Selling Drugs, Disease Type, Region
ü Regional Scope – North America; Europe; Asia Pacific; Latin America; Middle East & Africa
ü 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 Drug Typeofferings of key players. The detailed segments and sub-segment of the market are explained below:

By Drug Type:
Biological
Non-biological

By Top Selling Drugs:
Revlimid
Darzalex
Rituxan
Tafinlar
Ninlaro
Other

By Disease Type:
Oncology
Hematology
Neurology
Cardiovascular
Other Disease Types

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

Middle East & Africa
Saudi Arabia
South Africa
Rest of Middle East & Africa

Chapter 1. Executive Summary
1.1. Market Snapshot
1.2. Global & Segmental Market Estimates & Forecasts, 2020-2030 (USD Billion)
1.2.1. Orphan Drugs Market, by Region, 2020-2030 (USD Billion)
1.2.2. Orphan Drugs Market, by Drug Type, 2020-2030 (USD Billion)
1.2.3. Orphan Drugs Market, by Top Selling Drugs, 2020-2030 (USD Billion)
1.2.4. Orphan Drugs Market, by Disease Type, 2020-2030 (USD Billion)
1.3. Key Trends
1.4. Estimation Methodology
1.5. Research Assumption
Chapter 2. Global Orphan Drugs Market Definition and Scope
2.1. Objective of the Study
2.2. Market Definition & Scope
2.2.1. Industry Evolution
2.2.2. Scope of the Study
2.3. Years Considered for the Study
2.4. Currency Conversion Rates
Chapter 3. Global Orphan Drugs Market Dynamics
3.1. Orphan Drugs Market Impact Analysis (2020-2030)
3.1.1. Market Drivers
3.1.1.1. Rising Prevalence of Rare Diseases
3.1.1.2. Favorable Government Policies
3.1.2. Market Challenges
3.1.2.1. High Per Patient Treatment Cost
3.1.2.2. Limited Patient Pool for Clinical Trials and Product Marketing
3.1.3. Market Opportunities
3.1.3.1. Increasing research and development activities in the development of new therapies for rare diseases
3.1.3.2. Strong Product Launches activities
Chapter 4. Global Orphan Drugs 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. Porter’s 5 Force Impact Analysis
4.3. PEST Analysis
4.3.1. Political
4.3.2. Economical
4.3.3. Social
4.3.4. Technological
4.3.5. Environmental
4.3.6. Legal
4.4. Top investment opportunity
4.5. Top winning strategies
4.6. COVID-19 Impact Analysis
4.7. Disruptive Trends
4.8. Industry Expert Perspective
4.9. Analyst Recommendation & Conclusion
Chapter 5. Global Orphan Drugs Market, by Drug Type
5.1. Market Snapshot
5.2. Global Orphan Drugs Market by Drug Type, Performance – Potential Analysis
5.3. Global Orphan Drugs Market Estimates & Forecasts by Drug Type 2020-2030 (USD Billion)
5.4. Orphan Drugs Market, Sub Segment Analysis
5.4.1. Biological
5.4.2. Non-biological
Chapter 6. Global Orphan Drugs Market, by Top Selling Drugs
6.1. Market Snapshot
6.2. Global Orphan Drugs Market by Top Selling Drugs, Performance – Potential Analysis
6.3. Global Orphan Drugs Market Estimates & Forecasts by Top Selling Drugs 2020-2030 (USD Billion)
6.4. Orphan Drugs Market, Sub Segment Analysis
6.4.1. Revlimid
6.4.2. Darzalex
6.4.3. Rituxan
6.4.4. Tafinlar
6.4.5. Ninlaro
6.4.6. Other
Chapter 7. Global Orphan Drugs Market, by Disease Type
7.1. Market Snapshot
7.2. Global Orphan Drugs Market by Disease Type, Performance – Potential Analysis
7.3. Global Orphan Drugs Market Estimates & Forecasts by Disease Type 2020-2030 (USD Billion)
7.4. Orphan Drugs Market, Sub Segment Analysis
7.4.1. Oncology
7.4.2. Hematology
7.4.3. Neurology
7.4.4. Cardiovascular
7.4.5. Other Disease Types
Chapter 8. Global Orphan Drugs Market, Regional Analysis
8.1. Top Leading Countries
8.2. Top Emerging Countries
8.3. Orphan Drugs Market, Regional Market Snapshot
8.4. North America Orphan Drugs Market
8.4.1. U.S. Orphan Drugs Market
8.4.1.1. Drug Type breakdown estimates & forecasts, 2020-2030
8.4.1.2. Top Selling Drugs breakdown estimates & forecasts, 2020-2030
8.4.1.3. Disease Type breakdown estimates & forecasts, 2020-2030
8.4.2. Canada Orphan Drugs Market
8.5. Europe Orphan Drugs Market Snapshot
8.5.1. U.K. Orphan Drugs Market
8.5.2. Germany Orphan Drugs Market
8.5.3. France Orphan Drugs Market
8.5.4. Spain Orphan Drugs Market
8.5.5. Italy Orphan Drugs Market
8.5.6. Rest of Europe Orphan Drugs Market
8.6. Asia-Pacific Orphan Drugs Market Snapshot
8.6.1. China Orphan Drugs Market
8.6.2. India Orphan Drugs Market
8.6.3. Japan Orphan Drugs Market
8.6.4. Australia Orphan Drugs Market
8.6.5. South Korea Orphan Drugs Market
8.6.6. Rest of Asia Pacific Orphan Drugs Market
8.7. Latin America Orphan Drugs Market Snapshot
8.7.1. Brazil Orphan Drugs Market
8.7.2. Mexico Orphan Drugs Market
8.8. Middle East & Africa Orphan Drugs Market
8.8.1. Saudi Arabia Orphan Drugs Market
8.8.2. South Africa Orphan Drugs Market
8.8.3. Rest of Middle East & Africa Orphan Drugs Market

Chapter 9. Competitive Intelligence
9.1. Key Company SWOT Analysis
9.1.1. Company 1
9.1.2. Company 2
9.1.3. Company 3
9.2. Top Market Strategies
9.3. Company Profiles
9.3.1. Alexion Pharmaceuticals Inc
9.3.1.1. Key Information
9.3.1.2. Overview
9.3.1.3. Financial (Subject to Data Availability)
9.3.1.4. Product Summary
9.3.1.5. Recent Developments
9.3.2. Bristol-Myers Squibb Company (Celgene Corporation)
9.3.3. Novartis AG
9.3.4. Pfizer Inc.
9.3.5. F. Hoffmann-La Roche Ltd
9.3.6. Takeda Pharmaceutical Company Limited
9.3.7. Amgen Inc.
9.3.8. Sanofi S.A
9.3.9. Johnson & Johnson Pvt Ltd
9.3.10. AstraZeneca Plc
Chapter 10. Research Process
10.1. Research Process
10.1.1. Data Mining
10.1.2. Analysis
10.1.3. Market Estimation
10.1.4. Validation
10.1.5. Publishing
10.2. Research Attributes
10.3. 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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