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Report
Global Rare Disease Treatment Market Report and Forecast 2023-2031
Market Report I 2023-04-15 I 147 Pages I EMR Inc.
Global Rare Disease Treatment Market Report and Forecast 2023-2031
Global Rare Disease Treatment Market Outlook
The global rare diseases treatment market is expected to witness significant growth during the forecast period of 2023-2031. The increasing prevalence of rare diseases worldwide, coupled with the growing investment in research and development activities, is driving the growth of the market. The global rare diseases treatment market is likely to grow at a CAGR of 12.7 % during the forecast period.
Rare Disease Treatment Market: Introduction
The global rare diseases treatment market report by Expert Market Research provides an in-depth analysis of the market and its segments. Rare diseases are a group of diseases that affect a small percentage of the population. The lack of effective treatments for these diseases has been a major challenge for healthcare providers and researchers worldwide. The report covers the market for rare diseases treatment and provides a detailed analysis of the market size, growth, and trends.
The rare diseases treatment market is expected to witness significant advancements in the coming years. The development of novel therapies, such as gene therapy and cell therapy, and the increasing focus on personalized medicine are likely to drive the growth of the market. Additionally, the expansion of healthcare infrastructure in emerging economies and the increasing demand for advanced diagnostic tools and treatment options are expected to create lucrative opportunities for the market players.
Moreover, the growing adoption of telemedicine and digital health technologies may provide new avenues for the management and treatment of rare diseases, potentially improving patient outcomes and reducing healthcare costs. As a result, the global rare diseases treatment market is expected to continue its growth trajectory throughout the forecast period, offering numerous opportunities for key players and investors in the industry.
Rare Disease Epidemiology
The global epidemiology of rare diseases varies based on factors such as the type of disease, geographic location, and ethnicity. The lack of effective treatments and the complexity of rare diseases make them a major public health concern. The global prevalence of rare diseases is estimated to be approximately 6-8%, with over 7,000 rare diseases identified to date.
The rare diseases treatment market is highly fragmented, with numerous small and mid-sized companies operating in the market. The lack of effective treatments for rare diseases has led to the emergence of many companies focusing on the development of innovative therapies for these diseases.
Rare Disease Treatment Market Segmentations
The market can be categorised into therapeutic area, route of administration, phase type, patient type, drug type, distribution channel, and region.
Market Breakup by Therapeutic Area
- Oncological Disorders
- Neurological Disorders
- Cardiovascular Disorders
- Musculoskeletal Disorders
- Hematological Disorders
- Infectious Disorders
- Metabolic Disorders
- Endocrine Disorders
- Others
Market Breakup by Route of Administration
- Oral
- Injectables
- Others
Market Breakup by Phase Type
- Preclinical Phase
- Phase I
- Phase II
- Phase III
- Phase IV
Market Breakup by Patient Type
- Neonates
- Pediatrics
- Adults
Market Breakup by Drug Type
- Biologics
- Non-Biologics
- Others
Market Breakup by Distribution Channel
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
Market Breakup by Region
- North America
? United States of America
? Canada
- Europe
? United Kingdom
? Germany
? France
? Italy
? Others
- Asia Pacific
? China
? Japan
? India
? ASEAN
? Australia
? Others
- Latin America
? Brazil
? Argentina
? Mexico
? Others
- Middle East and Africa
? Saudi Arabia
? United Arab Emirates
? Nigeria
? South Africa
? Others
Rare Disease Treatment Market Scenario
North America dominates the rare diseases treatment market, followed by Europe and the Asia Pacific. The high prevalence of rare diseases in North America, coupled with the favourable reimbursement policies, is driving the growth of the market in the region. Europe is also a significant market for rare diseases treatment, with the presence of key players and increasing investment in research and development activities. The Asia Pacific region is expected to grow at the highest CAGR during the forecast period, owing to the increasing awareness about rare diseases and the rising healthcare expenditure in the region.
The market is highly fragmented, with numerous small and mid-sized companies operating in the market. The lack of effective treatments for rare diseases has led to the emergence of a large number of companies focusing on the development of innovative therapies for these diseases.
The rare disease treatment market can be categorized based on drug type, disease type, distribution channel, and region. The market for biological drugs is expected to grow significantly during the forecast period, driven by the increasing adoption of innovative therapies such as gene therapy and cell therapy.
The market for rare genetic disorders is expected to witness significant growth during the forecast period, driven by the high prevalence of these disorders worldwide. The market for oncology is also expected to grow significantly, driven by the increasing incidence of cancer worldwide.
The hospital pharmacies segment dominates the market, followed by retail pharmacies and online pharmacies. The high adoption of hospital pharmacies is driven by the availability of a wide range of drugs and the presence of trained healthcare professionals.
Key Players in the Global Rare Disease Treatment Market
The report gives an in-depth analysis of the key players involved in the rare disease treatment market, sponsors manufacturing the drugs, and putting them through trials to get FDA approvals. The companies included in the market are as follows:
- F. Hoffmann-La Roche Ltd
- Pfizer, Inc.
- PTC Therapeutics
- AstraZeneca
- Novartis AG
- Bristol-Myers Squibb Company
- Takeda Pharmaceutical Company Limited
- Bayer AG
- AbbVie Inc.
- Merck & Co., Inc.
- Teva Pharmaceutical Industries Ltd.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage - Key Segmentation
2 Research Methodology
3 Executive Summary
4 Rare Diseases Overview
4.1 Guidelines and Stages
4.2 Pathophysiology
4.3 Screening and Diagnosis
4.4 Treatment Pathway
5 Patient Profile
5.1 Patient Profile Overview
5.2 Patient Psychology and Emotional Impact Factors
5.3 Risk Assessment and Treatment Success Rate
6 Current Scenario Evaluation and Regulatory Framework
6.1 Emerging Therapies and Clinical Trials
6.2 Patent Landscape
6.2.1 Patent Overview
6.2.1.1 Patent Status and Expiry
6.2.1.2 Timelines from Drug Development to Commercial Launch
6.2.1.3 New Drug Application
6.2.1.3.1 Documentation and Approval Process
6.3 Cost of Treatment
6.4 Regulatory Framework
6.4.1 Regulatory Overview
6.4.1.1 US FDA
6.4.1.2 EU EMA
6.4.1.3 INDIA CDSCO
6.4.1.4 JAPAN PMDA
6.4.1.5 Others
7 Challenges and Unmet Needs
7.1 Treatment Pathway Challenges
7.2 Compliance and Drop-Out Analysis
7.3 Awareness and Prevention Gaps
8 Global Rare Diseases Treatment Market
8.1 Global Rare Diseases Treatment Market Overview
8.2 Global Rare Diseases Treatment Market Analysis
8.2.1 Market Overview
8.2.1.1 Global Rare Diseases Treatment Market Historical Value (2016-2022)
8.2.1.2 Global Rare Diseases Treatment Market Forecast Value (2023-2031)
8.2.2 Global Rare Diseases Treatment Market by Therapeutic Area
8.2.2.1 Market Overview
8.2.2.1.1 Oncological Disorders
8.2.2.1.2 Neurological Disorders
8.2.2.1.3 Cardiovascular Disorders
8.2.2.1.4 Musculoskeletal Disorders
8.2.2.1.5 Hematological Disorders
8.2.2.1.6 Infectious Disorders
8.2.2.1.7 Metabolic Disorders
8.2.2.1.8 Endocrine Disorders
8.2.2.1.9 Others
8.2.3 Global Rare Diseases Treatment Market by Route of Administration
8.2.3.1 Market Overview
8.2.3.1.1 Oral
8.2.3.1.2 Injectables
8.2.3.1.3 Others
8.2.4 Global Rare Diseases Treatment Market by Phase Type
8.2.4.1 Market Overview
8.2.4.1.1 Preclinical Phase
8.2.4.1.2 Phase I
8.2.4.1.3 Phase II
8.2.4.1.4 Phase III
8.2.4.1.5 Phase IV
8.2.5 Global Rare Diseases Treatment Market by Patient Type
8.2.5.1 Market Overview
8.2.5.1.1 Neonates
8.2.5.1.2 Pediatrics
8.2.5.1.3 Adults
8.2.6 Global Rare Diseases Treatment Market by Drug Type
8.2.6.1 Market Overview
8.2.6.1.1 Biologics
8.2.6.1.2 Non-Biologics
8.2.6.1.3 Others
8.2.7 Global Rare Diseases Treatment Market by Distribution Channel
8.2.7.1 Market Overview
8.2.7.1.1 Hospital Pharmacies
8.2.7.1.2 Retail Pharmacies
8.2.7.1.3 Online Pharmacies
8.2.8 Global Rare Diseases Treatment Market by Region
8.2.8.1 Market Overview
8.2.8.1.1 North America
8.2.8.1.2 Europe
8.2.8.1.3 Asia Pacific
8.2.8.1.4 Middle East and Africa
8.2.8.1.5 Latin America
9 North America Rare Disease Treatment Market
9.1 Market Share by Country
9.2 United States of America
9.2.1 Historical Trend (2016-2022)
9.2.2 Forecast Trend (2023-2031)
9.3 Canada
9.3.1 Historical Trend (2016-2022)
9.3.2 Forecast Trend (2023-2031)
10 Europe Rare Disease Treatment Market
10.1 Market Share by Country
10.2 United Kingdom
10.2.1 Historical Trend (2016-2022)
10.2.2 Forecast Trend (2023-2031)
10.3 Germany
10.3.1 Historical Trend (2016-2022)
10.3.2 Forecast Trend (2023-2031)
10.4 France
10.4.1 Historical Trend (2016-2022)
10.4.2 Forecast Trend (2023-2031)
10.5 Italy
10.5.1 Historical Trend (2016-2022)
10.5.2 Forecast Trend (2023-2031)
10.6 Others
11 Asia Pacific Rare Disease Treatment Market
11.1 Market Share by Country
11.2 China
11.2.1 Historical Trend (2016-2022)
11.2.2 Forecast Trend (2023-2031)
11.3 Japan
11.3.1 Historical Trend (2016-2022)
11.3.2 Forecast Trend (2023-2031)
11.4 India
11.4.1 Historical Trend (2016-2022)
11.4.2 Forecast Trend (2023-2031)
11.5 ASEAN
11.5.1 Historical Trend (2016-2022)
11.5.2 Forecast Trend (2023-2031)
11.6 Australia
11.6.1 Historical Trend (2016-2022)
11.6.2 Forecast Trend (2023-2031)
11.7 Others
12 Latin America Rare Disease Treatment Market
12.1 Market Share by Country
12.2 Brazil
12.2.1 Historical Trend (2016-2022)
12.2.2 Forecast Trend (2023-2031)
12.3 Argentina
12.3.1 Historical Trend (2016-2022)
12.3.2 Forecast Trend (2023-2031)
12.4 Mexico
12.4.1 Historical Trend (2016-2022)
12.4.2 Forecast Trend (2023-2031)
12.5 Others
13 Middle East and Africa Rare Disease Treatment Market
13.1 Market Share by Country
13.2 Saudi Arabia
13.2.1 Historical Trend (2016-2022)
13.2.2 Forecast Trend (2023-2031)
13.3 United Arab Emirates
13.3.1 Historical Trend (2016-2022)
13.3.2 Forecast Trend (2023-2031)
13.4 Nigeria
13.4.1 Historical Trend (2016-2022)
13.4.2 Forecast Trend (2023-2031)
13.5 South Africa
13.5.1 Historical Trend (2016-2022)
13.5.2 Forecast Trend (2023-2031)
13.6 Others
14 Global Rare Diseases Treatment Market Dynamics
14.1 Market Drivers and Constraints
14.2 SWOT Analysis
14.3 Porter's Five Forces Model
14.4 Key Demand Indicators
14.5 Key Price Indicators
14.6 Industry Events, Initiatives, and Trends
14.7 Value Chain Analysis
15 Supplier Landscape
15.1 F. Hoffmann-La Roche Ltd
15.1.1 Company Overview
15.1.2 Product Portfolio
15.1.3 Demographic Reach and Achievements
15.1.4 Mergers and Acquisitions
15.1.5 Certifications
15.2 Pfizer, Inc.
15.2.1 Company Overview
15.2.2 Product Portfolio
15.2.3 Demographic Reach and Achievements
15.2.4 Mergers and Acquisitions
15.2.5 Certifications
15.3 PTC Therapeutics
15.3.1 Company Overview
15.3.2 Product Portfolio
15.3.3 Demographic Reach and Achievements
15.3.4 Mergers and Acquisitions
15.3.5 Certifications
15.4 AstraZeneca
15.4.1 Company Overview
15.4.2 Product Portfolio
15.4.3 Demographic Reach and Achievements
15.4.4 Mergers and Acquisitions
15.4.5 Certifications
15.5 Novartis AG
15.5.1 Company Overview
15.5.2 Product Portfolio
15.5.3 Demographic Reach and Achievements
15.5.4 Mergers and Acquisitions
15.5.5 Certifications
15.6 Bristol-Myers Squibb Company
15.6.1 Company Overview
15.6.2 Product Portfolio
15.6.3 Demographic Reach and Achievements
15.6.4 Mergers and Acquisitions
15.6.5 Certifications
15.7 Takeda Pharmaceutical Company Limited
15.7.1 Company Overview
15.7.2 Product Portfolio
15.7.3 Demographic Reach and Achievements
15.7.4 Mergers and Acquisitions
15.7.5 Certifications
15.8 Bayer AG
15.8.1 Company Overview
15.8.2 Product Portfolio
15.8.3 Demographic Reach and Achievements
15.8.4 Mergers and Acquisitions
15.8.5 Certifications
15.9 AbbVie Inc.
15.9.1 Company Overview
15.9.2 Product Portfolio
15.9.3 Demographic Reach and Achievements
15.9.4 Mergers and Acquisitions
15.9.5 Certifications
15.10 Merck & Co., Inc.
15.10.1 Company Overview
15.10.2 Product Portfolio
15.10.3 Demographic Reach and Achievements
15.10.4 Mergers and Acquisitions
15.10.5 Certifications
15.11 Teva Pharmaceutical Industries Ltd.
15.11.1 Company Overview
15.11.2 Product Portfolio
15.11.3 Demographic Reach and Achievements
15.11.4 Mergers and Acquisitions
15.11.5 Certifications
16 Global Rare Diseases Treatment Market- Drug Distribution Model (Additional Insight)
16.1 Overview
16.2 Potential Distributors
16.3 Key Parameters for Distribution Partner Assessment
17 Payment Methods (Additional Insight)
17.1 Government Funded
17.2 Private Insurance
17.3 Out-of-Pocket
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