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PUBLISHER: Global Industry Analysts, Inc. | PRODUCT CODE: 1784072

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PUBLISHER: Global Industry Analysts, Inc. | PRODUCT CODE: 1784072

Rare Disease Clinical Trials

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Table of Contents

Global Rare Disease Clinical Trials Market to Reach US$19.7 Billion by 2030

The global market for Rare Disease Clinical Trials estimated at US$14.0 Billion in the year 2024, is expected to reach US$19.7 Billion by 2030, growing at a CAGR of 5.9% over the analysis period 2024-2030. Phase I, one of the segments analyzed in the report, is expected to record a 7.5% CAGR and reach US$8.6 Billion by the end of the analysis period. Growth in the Phase II segment is estimated at 4.3% CAGR over the analysis period.

The U.S. Market is Estimated at US$3.7 Billion While China is Forecast to Grow at 5.7% CAGR

The Rare Disease Clinical Trials market in the U.S. is estimated at US$3.7 Billion in the year 2024. China, the world's second largest economy, is forecast to reach a projected market size of US$3.2 Billion by the year 2030 trailing a CAGR of 5.7% over the analysis period 2024-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 5.7% and 4.8% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 4.5% CAGR.

Global Rare Disease Clinical Trials Market - Key Trends & Drivers Summarized

Why Are Rare Disease Clinical Trials Gaining Importance in Drug Development?

Rare disease clinical trials have become a focal point in drug development as pharmaceutical companies and regulatory agencies intensify efforts to address the unmet medical needs of patients suffering from rare and orphan diseases. With over 7,000 identified rare diseases affecting millions worldwide, the demand for effective therapies has grown exponentially. Traditional drug development models often overlook rare diseases due to limited patient populations and high research costs, but advancements in genomics, targeted therapies, and regulatory incentives have transformed the landscape. Governments and health organizations are providing grants, tax credits, and accelerated approval pathways to encourage rare disease drug development. The increasing adoption of adaptive trial designs, patient-centric approaches, and decentralized clinical trials has further improved the feasibility of conducting research on rare diseases. As the need for novel treatments rises, rare disease clinical trials are becoming an integral part of biopharmaceutical innovation.

How Are Emerging Technologies Improving Rare Disease Clinical Trials?

Technological advancements have significantly enhanced the efficiency and success rates of rare disease clinical trials. Artificial intelligence (AI) and machine learning are streamlining patient recruitment by identifying eligible participants based on genetic profiles and electronic health records (EHRs). The use of real-world data (RWD) and real-world evidence (RWE) is helping researchers gain insights into disease progression and treatment effectiveness, reducing the reliance on large patient cohorts. The adoption of virtual clinical trials and remote patient monitoring has improved trial accessibility, particularly for geographically dispersed rare disease patients. Biomarker-driven drug development and precision medicine approaches have also accelerated rare disease research, allowing for more targeted and effective treatment strategies. Additionally, gene therapy and CRISPR-based technologies are paving the way for groundbreaking rare disease treatments, reducing trial timelines and increasing the likelihood of successful outcomes.

What Market Trends Are Driving Rare Disease Clinical Trial Expansion?

The growing focus on orphan drug development and regulatory incentives has significantly boosted the rare disease clinical trial market. The FDA’s Orphan Drug Act and the European Medicines Agency’s (EMA) orphan designation program have incentivized pharmaceutical companies to invest in rare disease research by offering market exclusivity, reduced fees, and priority review programs. The rise of patient advocacy groups and increased patient engagement in trial designs have improved trial retention rates and recruitment efficiency. The expansion of gene and cell therapy research has led to a surge in clinical trials targeting rare genetic disorders, rare cancers, and neurodegenerative diseases. Additionally, strategic collaborations between pharmaceutical companies, biotech firms, and research institutions are driving innovation in rare disease drug development. The emergence of global rare disease registries and AI-powered clinical trial platforms is further enhancing trial design and data analysis, making rare disease research more viable and impactful.

What Are the Key Growth Drivers of the Rare Disease Clinical Trials Market?

The growth in the global rare disease clinical trials market is driven by several factors, including increasing regulatory support, advancements in genomic medicine, and the expansion of decentralized clinical trial models. The rising incidence of rare genetic disorders and the demand for personalized treatments have intensified the need for efficient and targeted clinical trials. The integration of AI-driven analytics, virtual patient monitoring, and real-world data is optimizing trial processes, reducing costs, and accelerating drug approvals. Additionally, biopharmaceutical companies are leveraging patient-centric trial methodologies to improve recruitment and retention rates, ensuring the success of rare disease studies. The growing investment in orphan drug development, coupled with collaborative efforts between pharmaceutical companies and patient advocacy groups, is further fueling market expansion. As rare disease research continues to evolve, innovative trial designs and cutting-edge biotechnologies are expected to drive breakthroughs in treatment development, improving outcomes for patients worldwide.

SCOPE OF STUDY:

The report analyzes the Rare Disease Clinical Trials market in terms of units by the following Segments, and Geographic Regions/Countries:

Segments:

Phase (Phase I, Phase II, Phase III, Phase IV); Therapeutic Area (Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases, Genetic Disorders, Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas); Sponsor (Pharma & Biotech Companies, Non-Profit Organizations, Other Sponsors)

Geographic Regions/Countries:

World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; and Rest of Europe); Asia-Pacific; Rest of World.

Select Competitors (Total 37 Featured) -

  • Alexion Pharmaceuticals
  • Alnylam Pharmaceuticals
  • Amgen Inc.
  • Arcturus Therapeutics
  • AstraZeneca
  • Biogen Inc.
  • Charles River Laboratories
  • Editas Medicine
  • F. Hoffmann-La Roche Ltd.
  • Fortrea
  • ICON plc
  • IQVIA Inc.
  • Labcorp Drug Development
  • Medpace
  • Novartis AG
  • Parexel International Corporation
  • Pfizer Inc.
  • Precision for Medicine
  • PTC Therapeutics
  • Sanofi
  • Scholar Rock
  • Takeda Pharmaceutical Company
  • Ultragenyx Pharmaceutical Inc.
  • United Therapeutics
  • Vertex Pharmaceuticals

AI INTEGRATIONS

We're transforming market and competitive intelligence with validated expert content and AI tools.

Instead of following the general norm of querying LLMs and Industry-specific SLMs, we built repositories of content curated from domain experts worldwide including video transcripts, blogs, search engines research, and massive amounts of enterprise, product/service, and market data.

TARIFF IMPACT FACTOR

Our new release incorporates impact of tariffs on geographical markets as we predict a shift in competitiveness of companies based on HQ country, manufacturing base, exports and imports (finished goods and OEM). This intricate and multifaceted market reality will impact competitors by increasing the Cost of Goods Sold (COGS), reducing profitability, reconfiguring supply chains, amongst other micro and macro market dynamics.

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Product Code: MCP32759

TABLE OF CONTENTS

I. METHODOLOGY

II. EXECUTIVE SUMMARY

  • 1. MARKET OVERVIEW
    • Influencer Market Insights
    • Tariff Impact on Global Supply Chain Patterns
    • Rare Disease Clinical Trials - Global Key Competitors Percentage Market Share in 2025 (E)
    • Competitive Market Presence - Strong/Active/Niche/Trivial for Players Worldwide in 2025 (E)
  • 2. FOCUS ON SELECT PLAYERS
  • 3. MARKET TRENDS & DRIVERS
    • Increased Orphan Drug Development Pipelines Propel Demand for Rare Disease Clinical Trial Infrastructure
    • Rising Patient Advocacy and Engagement Strengthens Feasibility and Recruitment in Rare Disease Trials
    • Advancements in Genomic Profiling and Target Identification Accelerate Trial Design and Execution
    • Regulatory Incentives for Orphan Drugs Spur Investment in Rare Disease Research and Clinical Studies
    • Shift Toward Decentralized and Virtual Trials Throws the Spotlight on Remote Patient Monitoring Technologies
    • Emergence of Adaptive Trial Designs Enhances Efficiency and Drives Broader Application in Small Populations
    • Partnerships Between CROs and Biotech Firms Streamline Trial Operations and Improve Data Quality
    • Use of AI in Site Selection and Trial Matching Optimizes Outcomes and Reduces Time-to-Market
    • Expansion of Global Patient Registries Improves Access to Trial Candidates and Rare Cohorts
    • Growth in Gene Therapy and mRNA-Based Platforms Supports Expansion of Trial Modalities
    • Increased Funding by Non-Profits and Government Agencies Strengthens Clinical Trial Ecosystems
    • Real-World Evidence Integration in Trial Protocols Enhances Regulatory Submissions and Market Access
    • Regulatory Flexibility in Trial Requirements for Rare Indications Supports Innovative Study Designs
    • Patient-Centric Trial Models Propel Use of At-Home Sampling and Mobile Health Monitoring
    • Rise in Basket and Umbrella Trials for Rare Genetic Disorders Expands Protocol Scalability
    • Technological Advancements in EDC and eCOA Platforms Improve Data Collection in Remote Trials
    • Biomarker-Driven Stratification Enhances Endpoint Precision and Drives Early Phase Trial Success
    • Collaborative Consortia Among Academia, Pharma, and Regulators Bolster Rare Disease Trial Networks
    • Growth in Pediatric Rare Disease Studies Requires Age-Specific Protocol Adaptations and Ethical Oversight
    • Globalization of Trial Sites in Low-Prevalence Regions Expands Trial Reach and Accelerates Enrollment
  • 4. GLOBAL MARKET PERSPECTIVE
    • TABLE 1: World Rare Disease Clinical Trials Market Analysis of Annual Sales in US$ Million for Years 2015 through 2030
    • TABLE 2: World Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 3: World 6-Year Perspective for Rare Disease Clinical Trials by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2025 & 2030
    • TABLE 4: World Recent Past, Current & Future Analysis for Phase I by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 5: World 6-Year Perspective for Phase I by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 6: World Recent Past, Current & Future Analysis for Phase II by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 7: World 6-Year Perspective for Phase II by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 8: World Recent Past, Current & Future Analysis for Phase III by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 9: World 6-Year Perspective for Phase III by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 10: World Recent Past, Current & Future Analysis for Phase IV by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 11: World 6-Year Perspective for Phase IV by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 12: World Recent Past, Current & Future Analysis for Autoimmune & Inflammation by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 13: World 6-Year Perspective for Autoimmune & Inflammation by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 14: World Recent Past, Current & Future Analysis for Hematologic Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 15: World 6-Year Perspective for Hematologic Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 16: World Recent Past, Current & Future Analysis for Musculoskeletal Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 17: World 6-Year Perspective for Musculoskeletal Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 18: World Recent Past, Current & Future Analysis for Other Therapeutic Areas by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 19: World 6-Year Perspective for Other Therapeutic Areas by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 20: World Recent Past, Current & Future Analysis for Oncology by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 21: World 6-Year Perspective for Oncology by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 22: World Recent Past, Current & Future Analysis for Cardiovascular Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 23: World 6-Year Perspective for Cardiovascular Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 24: World Recent Past, Current & Future Analysis for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 25: World 6-Year Perspective for Neurological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 26: World Recent Past, Current & Future Analysis for Infectious Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 27: World 6-Year Perspective for Infectious Diseases by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 28: World Recent Past, Current & Future Analysis for Genetic Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 29: World 6-Year Perspective for Genetic Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 30: World Recent Past, Current & Future Analysis for Pharma & Biotech Companies by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 31: World 6-Year Perspective for Pharma & Biotech Companies by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 32: World Recent Past, Current & Future Analysis for Non-Profit Organizations by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 33: World 6-Year Perspective for Non-Profit Organizations by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030
    • TABLE 34: World Recent Past, Current & Future Analysis for Other Sponsors by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 35: World 6-Year Perspective for Other Sponsors by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2025 & 2030

III. MARKET ANALYSIS

  • UNITED STATES
    • Rare Disease Clinical Trials Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United States for 2025 (E)
    • TABLE 36: USA Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 37: USA 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 38: USA Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 39: USA 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 40: USA Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 41: USA 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • CANADA
    • TABLE 42: Canada Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 43: Canada 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 44: Canada Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 45: Canada 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 46: Canada Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 47: Canada 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • JAPAN
    • Rare Disease Clinical Trials Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Japan for 2025 (E)
    • TABLE 48: Japan Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 49: Japan 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 50: Japan Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 51: Japan 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 52: Japan Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 53: Japan 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • CHINA
    • Rare Disease Clinical Trials Market Presence - Strong/Active/Niche/Trivial - Key Competitors in China for 2025 (E)
    • TABLE 54: China Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 55: China 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 56: China Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 57: China 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 58: China Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 59: China 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • EUROPE
    • Rare Disease Clinical Trials Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Europe for 2025 (E)
    • TABLE 60: Europe Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Million for Years 2024 through 2030 and % CAGR
    • TABLE 61: Europe 6-Year Perspective for Rare Disease Clinical Trials by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2025 & 2030
    • TABLE 62: Europe Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 63: Europe 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 64: Europe Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 65: Europe 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 66: Europe Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 67: Europe 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • FRANCE
    • Rare Disease Clinical Trials Market Presence - Strong/Active/Niche/Trivial - Key Competitors in France for 2025 (E)
    • TABLE 68: France Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 69: France 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 70: France Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 71: France 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 72: France Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 73: France 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • GERMANY
    • Rare Disease Clinical Trials Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Germany for 2025 (E)
    • TABLE 74: Germany Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 75: Germany 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 76: Germany Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 77: Germany 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 78: Germany Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 79: Germany 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • ITALY
    • TABLE 80: Italy Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 81: Italy 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 82: Italy Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 83: Italy 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 84: Italy Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 85: Italy 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • UNITED KINGDOM
    • Rare Disease Clinical Trials Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United Kingdom for 2025 (E)
    • TABLE 86: UK Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 87: UK 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 88: UK Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 89: UK 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 90: UK Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 91: UK 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • REST OF EUROPE
    • TABLE 92: Rest of Europe Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 93: Rest of Europe 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 94: Rest of Europe Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 95: Rest of Europe 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 96: Rest of Europe Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 97: Rest of Europe 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • ASIA-PACIFIC
    • Rare Disease Clinical Trials Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Asia-Pacific for 2025 (E)
    • TABLE 98: Asia-Pacific Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 99: Asia-Pacific 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 100: Asia-Pacific Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 101: Asia-Pacific 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 102: Asia-Pacific Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 103: Asia-Pacific 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030
  • REST OF WORLD
    • TABLE 104: Rest of World Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Phase - Phase I, Phase II, Phase III and Phase IV - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 105: Rest of World 6-Year Perspective for Rare Disease Clinical Trials by Phase - Percentage Breakdown of Value Sales for Phase I, Phase II, Phase III and Phase IV for the Years 2025 & 2030
    • TABLE 106: Rest of World Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Therapeutic Area - Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 107: Rest of World 6-Year Perspective for Rare Disease Clinical Trials by Therapeutic Area - Percentage Breakdown of Value Sales for Autoimmune & Inflammation, Hematologic Disorders, Musculoskeletal Disorders, Other Therapeutic Areas, Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases and Genetic Disorders for the Years 2025 & 2030
    • TABLE 108: Rest of World Recent Past, Current & Future Analysis for Rare Disease Clinical Trials by Sponsor - Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors - Independent Analysis of Annual Sales in US$ Million for the Years 2024 through 2030 and % CAGR
    • TABLE 109: Rest of World 6-Year Perspective for Rare Disease Clinical Trials by Sponsor - Percentage Breakdown of Value Sales for Pharma & Biotech Companies, Non-Profit Organizations and Other Sponsors for the Years 2025 & 2030

IV. COMPETITION

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