The rare disease clinical trials market size is projected to be valued at US$ 12,566.14 million in 2023 and is expected to rise to US$ 31,715.25 million by 2033. The sales of rare disease clinical trials are expected to record a significant CAGR of 9.7% during the forecast period.
Various Factors Propelling the Demand for Rare Disease Clinical Trials
The rare disease clinical trials market refers to the development of drugs and therapies for the treatment of rare diseases. These diseases, by definition, affect a small percentage of the population. Consequently, there is often limited research and development in this area.
Clinical trials for rare diseases can be challenging due to the small patient population, limited natural history data, and a lack of standardized endpoints. However, the market is growing due to increased funding and attention to the development of treatments for these diseases.
For example, The Orphan Drug Act was passed in the United States in 1983. The act was enacted to offer financial incentives to pharmaceutical companies to develop treatments for rare diseases.
The advancement in technology and genomics allows for more targeted treatments and personalized medicine. Additionally, there has been increased collaboration between patient advocacy groups, researchers, and the industry. This collaboration has helped to overcome some challenges of conducting clinical trials in rare diseases.
Overall, the market represents an important area of research and development. As it offers hope for patients with conditions that may have previously been considered untreatable.
| Attribute | Details |
|---|---|
| Rare Disease Clinical Trials Market Estimated Size (2023) | US$ 12,566.14 million |
| Rare Disease Clinical Trials Market CAGR (2023 to 2033) | 9.7% |
| Rare Disease Clinical Trials Market Forecasted Size (2033) | US$ 31,715.25 million |
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The rare disease clinical trials market was valued at US$ 11,455.0 million in 2022 with a substantial growth trajectory of 8.9% between the analysis period of 2018 to 2022.
Future Market Insights expects the rare disease clinical trials market to expand at a CAGR of 9.7% during the forecast period of 2023 to 2033. The growth can be attributed to the following reasons
Considering the above-mentioned factors, FMI opines, the rare disease clinical trials market is likely to witness a market value of US$ 31,715.25 million by the end of 2033.
Principal Consultant
In 2022, the oncology segment held a notable share of 33.9%. A few of the key factors fueling the segment growth are
For example, in January 2023, the USFDA approved atezolizumab (Tecentriq), an immunotherapy drug for advanced alveolar soft part sarcoma patients (ASPS).
During the forecast period, the infectious disease segment is expected to expand at an exponential rate of 10.6%. Acanthamoeba keratitis, Q fever, Marburg virus, and other rare infectious diseases are examples. As these rare diseases are fatal, researchers are hugely interested in developing treatments and vaccines for them. Contact lens wearers are likely to develop Acanthamoeba keratitis. Over 85% of people in the United States wear contact lenses. This increases the risk of disease and fuels the demand for treatment. These factors are assisting segment growth.
In 2022, Phase II had a notable share of 42.6%. Phase II studies are divided into two parts: the first part includes dose range exploration as well as efficacy studies, while the second part includes dose finalization. Over 100-300 participants are recruited for Phase II clinical trials. The phase II segment had the most registered clinical studies as of November 2022. The ClinicalTrial.gov portal had 72,522 studies registered as of November 2022. The high amount of Phase II clinical trials is driving the segment's growth.
The United States has a well-established regulatory framework for rare disease clinical trials, with the Orphan Drug Act of 1983. It provides incentives for the development of drugs for rare diseases. The FDA's Breakthrough Therapy Designation program has also facilitated the development of drugs for rare diseases. Additionally, the National Institutes of Health (NIH) has established a Rare Diseases Clinical Research Network (RDCRN) to facilitate the conduct of clinical trials for rare diseases.
Europe is a significant market for rare disease clinical trials, with countries like Germany, France, and the United Kingdom accounting for a significant share of the market. The European Medicines Agency (EMA) has been proactive in offering regulatory support for rare disease drug development. Therefore, the support from several agencies has increased the clinical trials in Europe.
Asia Pacific is a significantly growing market for rare disease clinical trials, with countries like China, Japan, and India emerging as key players. The region offers a substantial patient population and low trial costs compared to Western countries. Making it an attractive destination for colossal clinical trials. However, the regulatory environment in some countries can be challenging, and there is a need for great awareness and education about rare diseases.
Latin America is an emerging market for rare disease clinical trials, with countries like Brazil and Mexico. The region offers a diverse patient population and low trial costs compared to North America and Europe. However, there are challenges in terms of regulatory compliance and infrastructure, which can limit the growth of the market.
The Middle East and Africa region is a small but growing market for rare disease clinical trials. Countries like South Africa and Israel are emerging as key players due to their advanced healthcare infrastructure and supportive regulatory environment. However, there are challenges related to political instability, low awareness about rare diseases, and limited healthcare resources in some parts of the region.
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The rare disease clinical trials market is highly competitive, with several global players operating in the space. Some key players in the market include
In addition to these companies, there are many other small CROs and specialized service providers operating in the rare disease clinical trials market. As the market continues to grow, competition among these companies is expected to increase. These players are competing on factors such as service quality, expertise, and pricing.
| Attribute | Details |
|---|---|
| Growth Rate | CAGR of 9.7% from 2023 to 2033 |
| Base Year of Estimation | 2022 |
| Historical Data | 2018 to 2022 |
| Forecast Period | 2023 to 2033 |
| Quantitative Units | Revenue in US$ million and Volume in Units and F-CAGR from 2023 to 2033 |
| Report Coverage | Revenue Forecast, Volume Forecast, Company Ranking, Competitive Landscape, growth factors, Trends, and Pricing Analysis |
| Key Segments Covered | By Therapeutic Area, By Sponsor, By Phases, By Region |
| Regions Covered | North America; Latin America; Europe; East Asia; South Asia; The Middle East & Africa;Oceania |
| Key Countries Profiled | The United States, Canada, Brazil, Mexico, Germany, Italy, France, The United Kingdom, Spain, Russia, China, Japan, India, GCC Countries, Australia |
| Key Companies Profiled | Takeda Pharmaceutical Company; F. Hoffmann-La Roche Ltd.; Pfizer, Inc.; AstraZeneca; Novartis AG; LabCorp; IQVIA, Inc.; Charles River Laboratories; Icon PLC; Parexel International Corporation |
| Customization & Pricing | Available upon Request |
The market is valued at US$ 12.5 billion in 2023.
Pfizer, Inc., AstraZeneca, and Novartis AG are the top market players.
The market held a CAGR of 8.9% from 2018 to 2022.
Advancements in personalized medicine to boost market growth.
Phase II segment is likely to remain preferred through 2033.
1. Executive Summary | Rare Disease Clinical Trials Market
1.1. Global Market Outlook
1.2. Demand-side Trends
1.3. Supply-side Trends
1.4. Technology Roadmap Analysis
1.5. Analysis and Recommendations
2. Market Overview
2.1. Market Coverage / Taxonomy
2.2. Market Definition / Scope / Limitations
3. Market Background
3.1. Market Dynamics
3.1.1. Drivers
3.1.2. Restraints
3.1.3. Opportunity
3.1.4. Trends
3.2. Scenario Forecast
3.2.1. Demand in Optimistic Scenario
3.2.2. Demand in Likely Scenario
3.2.3. Demand in Conservative Scenario
3.3. Opportunity Map Analysis
3.4. Investment Feasibility Matrix
3.5. PESTLE and Porter’s Analysis
3.6. Regulatory Landscape
3.6.1. By Key Regions
3.6.2. By Key Countries
3.7. Regional Parent Market Outlook
4. Global Market Analysis 2018 to 2022 and Forecast, 2023 to 2033
4.1. Historical Market Size Value (US$ Million) Analysis, 2018 to 2022
4.2. Current and Future Market Size Value (US$ Million) Projections, 2023 to 2033
4.2.1. Y-o-Y Growth Trend Analysis
4.2.2. Absolute $ Opportunity Analysis
5. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Therapeutic Area
5.1. Introduction / Key Findings
5.2. Historical Market Size Value (US$ Million) Analysis By Therapeutic Area, 2018 to 2022
5.3. Current and Future Market Size Value (US$ Million) Analysis and Forecast By Therapeutic Area, 2023 to 2033
5.3.1. Oncology
5.3.2. Cardiovascular Disorders
5.3.3. Neurological Disorders
5.3.4. Infectious Disease
5.3.5. Genetic Disorders
5.3.6. Autoimmune And Inflammation
5.3.7. Hematologic Disorders
5.3.8. Musculoskeletal Disorders
5.3.9. Others
5.4. Y-o-Y Growth Trend Analysis By Therapeutic Area, 2018 to 2022
5.5. Absolute $ Opportunity Analysis By Therapeutic Area, 2023 to 2033
6. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Phase
6.1. Introduction / Key Findings
6.2. Historical Market Size Value (US$ Million) Analysis By Phase, 2018 to 2022
6.3. Current and Future Market Size Value (US$ Million) Analysis and Forecast By Phase, 2023 to 2033
6.3.1. Phase I
6.3.2. Phase II
6.3.3. Phase III
6.3.4. Phase IV
6.4. Y-o-Y Growth Trend Analysis By Phase, 2018 to 2022
6.5. Absolute $ Opportunity Analysis By Phase, 2023 to 2033
7. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Sponsor
7.1. Introduction / Key Findings
7.2. Historical Market Size Value (US$ Million) Analysis By Sponsor, 2018 to 2022
7.3. Current and Future Market Size Value (US$ Million) Analysis and Forecast By Sponsor, 2023 to 2033
7.3.1. Pharmaceutical & Biopharmaceutical Companies
7.3.2. Non-profit Organizations
7.4. Y-o-Y Growth Trend Analysis By Sponsor, 2018 to 2022
7.5. Absolute $ Opportunity Analysis By Sponsor, 2023 to 2033
8. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Region
8.1. Introduction
8.2. Historical Market Size Value (US$ Million) Analysis By Region, 2018 to 2022
8.3. Current Market Size Value (US$ Million) Analysis and Forecast By Region, 2023 to 2033
8.3.1. North America
8.3.2. Latin America
8.3.3. Europe
8.3.4. South Asia
8.3.5. East Asia
8.3.6. Oceania
8.3.7. MEA
8.4. Market Attractiveness Analysis By Region
9. North America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
9.1. Historical Market Size Value (US$ Million) Trend Analysis By Market Taxonomy, 2018 to 2022
9.2. Market Size Value (US$ Million) Forecast By Market Taxonomy, 2023 to 2033
9.2.1. By Country
9.2.1.1. USA
9.2.1.2. Canada
9.2.2. By Therapeutic Area
9.2.3. By Phase
9.2.4. By Sponsor
9.3. Market Attractiveness Analysis
9.3.1. By Country
9.3.2. By Therapeutic Area
9.3.3. By Phase
9.3.4. By Sponsor
9.4. Key Takeaways
10. Latin America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
10.1. Historical Market Size Value (US$ Million) Trend Analysis By Market Taxonomy, 2018 to 2022
10.2. Market Size Value (US$ Million) Forecast By Market Taxonomy, 2023 to 2033
10.2.1. By Country
10.2.1.1. Brazil
10.2.1.2. Mexico
10.2.1.3. Rest of Latin America
10.2.2. By Therapeutic Area
10.2.3. By Phase
10.2.4. By Sponsor
10.3. Market Attractiveness Analysis
10.3.1. By Country
10.3.2. By Therapeutic Area
10.3.3. By Phase
10.3.4. By Sponsor
10.4. Key Takeaways
11. Europe Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
11.1. Historical Market Size Value (US$ Million) Trend Analysis By Market Taxonomy, 2018 to 2022
11.2. Market Size Value (US$ Million) Forecast By Market Taxonomy, 2023 to 2033
11.2.1. By Country
11.2.1.1. Germany
11.2.1.2. United kingdom
11.2.1.3. France
11.2.1.4. Spain
11.2.1.5. Italy
11.2.1.6. Rest of Europe
11.2.2. By Therapeutic Area
11.2.3. By Phase
11.2.4. By Sponsor
11.3. Market Attractiveness Analysis
11.3.1. By Country
11.3.2. By Therapeutic Area
11.3.3. By Phase
11.3.4. By Sponsor
11.4. Key Takeaways
12. South Asia Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
12.1. Historical Market Size Value (US$ Million) Trend Analysis By Market Taxonomy, 2018 to 2022
12.2. Market Size Value (US$ Million) Forecast By Market Taxonomy, 2023 to 2033
12.2.1. By Country
12.2.1.1. India
12.2.1.2. Malaysia
12.2.1.3. Singapore
12.2.1.4. Thailand
12.2.1.5. Rest of South Asia
12.2.2. By Therapeutic Area
12.2.3. By Phase
12.2.4. By Sponsor
12.3. Market Attractiveness Analysis
12.3.1. By Country
12.3.2. By Therapeutic Area
12.3.3. By Phase
12.3.4. By Sponsor
12.4. Key Takeaways
13. East Asia Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
13.1. Historical Market Size Value (US$ Million) Trend Analysis By Market Taxonomy, 2018 to 2022
13.2. Market Size Value (US$ Million) Forecast By Market Taxonomy, 2023 to 2033
13.2.1. By Country
13.2.1.1. China
13.2.1.2. Japan
13.2.1.3. South Korea
13.2.2. By Therapeutic Area
13.2.3. By Phase
13.2.4. By Sponsor
13.3. Market Attractiveness Analysis
13.3.1. By Country
13.3.2. By Therapeutic Area
13.3.3. By Phase
13.3.4. By Sponsor
13.4. Key Takeaways
14. Oceania Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
14.1. Historical Market Size Value (US$ Million) Trend Analysis By Market Taxonomy, 2018 to 2022
14.2. Market Size Value (US$ Million) Forecast By Market Taxonomy, 2023 to 2033
14.2.1. By Country
14.2.1.1. Australia
14.2.1.2. New Zealand
14.2.2. By Therapeutic Area
14.2.3. By Phase
14.2.4. By Sponsor
14.3. Market Attractiveness Analysis
14.3.1. By Country
14.3.2. By Therapeutic Area
14.3.3. By Phase
14.3.4. By Sponsor
14.4. Key Takeaways
15. MEA Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
15.1. Historical Market Size Value (US$ Million) Trend Analysis By Market Taxonomy, 2018 to 2022
15.2. Market Size Value (US$ Million) Forecast By Market Taxonomy, 2023 to 2033
15.2.1. By Country
15.2.1.1. GCC Countries
15.2.1.2. South Africa
15.2.1.3. Israel
15.2.1.4. Rest of MEA
15.2.2. By Therapeutic Area
15.2.3. By Phase
15.2.4. By Sponsor
15.3. Market Attractiveness Analysis
15.3.1. By Country
15.3.2. By Therapeutic Area
15.3.3. By Phase
15.3.4. By Sponsor
15.4. Key Takeaways
16. Key Countries Market Analysis
16.1. USA
16.1.1. Pricing Analysis
16.1.2. Market Share Analysis, 2022
16.1.2.1. By Therapeutic Area
16.1.2.2. By Phase
16.1.2.3. By Sponsor
16.2. Canada
16.2.1. Pricing Analysis
16.2.2. Market Share Analysis, 2022
16.2.2.1. By Therapeutic Area
16.2.2.2. By Phase
16.2.2.3. By Sponsor
16.3. Brazil
16.3.1. Pricing Analysis
16.3.2. Market Share Analysis, 2022
16.3.2.1. By Therapeutic Area
16.3.2.2. By Phase
16.3.2.3. By Sponsor
16.4. Mexico
16.4.1. Pricing Analysis
16.4.2. Market Share Analysis, 2022
16.4.2.1. By Therapeutic Area
16.4.2.2. By Phase
16.4.2.3. By Sponsor
16.5. Germany
16.5.1. Pricing Analysis
16.5.2. Market Share Analysis, 2022
16.5.2.1. By Therapeutic Area
16.5.2.2. By Phase
16.5.2.3. By Sponsor
16.6. United kingdom
16.6.1. Pricing Analysis
16.6.2. Market Share Analysis, 2022
16.6.2.1. By Therapeutic Area
16.6.2.2. By Phase
16.6.2.3. By Sponsor
16.7. France
16.7.1. Pricing Analysis
16.7.2. Market Share Analysis, 2022
16.7.2.1. By Therapeutic Area
16.7.2.2. By Phase
16.7.2.3. By Sponsor
16.8. Spain
16.8.1. Pricing Analysis
16.8.2. Market Share Analysis, 2022
16.8.2.1. By Therapeutic Area
16.8.2.2. By Phase
16.8.2.3. By Sponsor
16.9. Italy
16.9.1. Pricing Analysis
16.9.2. Market Share Analysis, 2022
16.9.2.1. By Therapeutic Area
16.9.2.2. By Phase
16.9.2.3. By Sponsor
16.10. India
16.10.1. Pricing Analysis
16.10.2. Market Share Analysis, 2022
16.10.2.1. By Therapeutic Area
16.10.2.2. By Phase
16.10.2.3. By Sponsor
16.11. Malaysia
16.11.1. Pricing Analysis
16.11.2. Market Share Analysis, 2022
16.11.2.1. By Therapeutic Area
16.11.2.2. By Phase
16.11.2.3. By Sponsor
16.12. Singapore
16.12.1. Pricing Analysis
16.12.2. Market Share Analysis, 2022
16.12.2.1. By Therapeutic Area
16.12.2.2. By Phase
16.12.2.3. By Sponsor
16.13. Thailand
16.13.1. Pricing Analysis
16.13.2. Market Share Analysis, 2022
16.13.2.1. By Therapeutic Area
16.13.2.2. By Phase
16.13.2.3. By Sponsor
16.14. China
16.14.1. Pricing Analysis
16.14.2. Market Share Analysis, 2022
16.14.2.1. By Therapeutic Area
16.14.2.2. By Phase
16.14.2.3. By Sponsor
16.15. Japan
16.15.1. Pricing Analysis
16.15.2. Market Share Analysis, 2022
16.15.2.1. By Therapeutic Area
16.15.2.2. By Phase
16.15.2.3. By Sponsor
16.16. South Korea
16.16.1. Pricing Analysis
16.16.2. Market Share Analysis, 2022
16.16.2.1. By Therapeutic Area
16.16.2.2. By Phase
16.16.2.3. By Sponsor
16.17. Australia
16.17.1. Pricing Analysis
16.17.2. Market Share Analysis, 2022
16.17.2.1. By Therapeutic Area
16.17.2.2. By Phase
16.17.2.3. By Sponsor
16.18. New Zealand
16.18.1. Pricing Analysis
16.18.2. Market Share Analysis, 2022
16.18.2.1. By Therapeutic Area
16.18.2.2. By Phase
16.18.2.3. By Sponsor
16.19. GCC Countries
16.19.1. Pricing Analysis
16.19.2. Market Share Analysis, 2022
16.19.2.1. By Therapeutic Area
16.19.2.2. By Phase
16.19.2.3. By Sponsor
16.20. South Africa
16.20.1. Pricing Analysis
16.20.2. Market Share Analysis, 2022
16.20.2.1. By Therapeutic Area
16.20.2.2. By Phase
16.20.2.3. By Sponsor
16.21. Israel
16.21.1. Pricing Analysis
16.21.2. Market Share Analysis, 2022
16.21.2.1. By Therapeutic Area
16.21.2.2. By Phase
16.21.2.3. By Sponsor
17. Market Structure Analysis
17.1. Competition Dashboard
17.2. Competition Benchmarking
17.3. Market Share Analysis of Top Players
17.3.1. By Regional
17.3.2. By Therapeutic Area
17.3.3. By Phase
17.3.4. By Sponsor
18. Competition Analysis
18.1. Competition Deep Dive
18.1.1. Takeda Pharmaceutical Company
18.1.1.1. Overview
18.1.1.2. Product Portfolio
18.1.1.3. Profitability by Market Segments
18.1.1.4. Sales Footprint
18.1.1.5. Strategy Overview
18.1.1.5.1. Marketing Strategy
18.1.2. F. Hoffmann-La Roche Ltd.
18.1.2.1. Overview
18.1.2.2. Product Portfolio
18.1.2.3. Profitability by Market Segments
18.1.2.4. Sales Footprint
18.1.2.5. Strategy Overview
18.1.2.5.1. Marketing Strategy
18.1.3. Pfizer, Inc.
18.1.3.1. Overview
18.1.3.2. Product Portfolio
18.1.3.3. Profitability by Market Segments
18.1.3.4. Sales Footprint
18.1.3.5. Strategy Overview
18.1.3.5.1. Marketing Strategy
18.1.4. AstraZeneca
18.1.4.1. Overview
18.1.4.2. Product Portfolio
18.1.4.3. Profitability by Market Segments
18.1.4.4. Sales Footprint
18.1.4.5. Strategy Overview
18.1.4.5.1. Marketing Strategy
18.1.5. Novartis AG
18.1.5.1. Overview
18.1.5.2. Product Portfolio
18.1.5.3. Profitability by Market Segments
18.1.5.4. Sales Footprint
18.1.5.5. Strategy Overview
18.1.5.5.1. Marketing Strategy
18.1.6. LabCorp
18.1.6.1. Overview
18.1.6.2. Product Portfolio
18.1.6.3. Profitability by Market Segments
18.1.6.4. Sales Footprint
18.1.6.5. Strategy Overview
18.1.6.5.1. Marketing Strategy
18.1.7. IQVIA
18.1.7.1. Overview
18.1.7.2. Product Portfolio
18.1.7.3. Profitability by Market Segments
18.1.7.4. Sales Footprint
18.1.7.5. Strategy Overview
18.1.7.5.1. Marketing Strategy
18.1.8. Charles River Laboratories Inc.
18.1.8.1. Overview
18.1.8.2. Product Portfolio
18.1.8.3. Profitability by Market Segments
18.1.8.4. Sales Footprint
18.1.8.5. Strategy Overview
18.1.8.5.1. Marketing Strategy
18.1.9. Icon PLC
18.1.9.1. Overview
18.1.9.2. Product Portfolio
18.1.9.3. Profitability by Market Segments
18.1.9.4. Sales Footprint
18.1.9.5. Strategy Overview
18.1.9.5.1. Marketing Strategy
18.1.10. Parexel International Corporation
18.1.10.1. Overview
18.1.10.2. Product Portfolio
18.1.10.3. Profitability by Market Segments
18.1.10.4. Sales Footprint
18.1.10.5. Strategy Overview
18.1.10.5.1. Marketing Strategy
19. Assumptions & Acronyms Used
20. Research Methodology
Healthcare
November 2022
REP-GB-15802
250 pages
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Rare Disease Clinical Trials Market
