희귀질환 임상시험 시장 규모, 점유율, 동향 분석 보고서 : Phase별, 치료 영역별, 스폰서별, 지역별, 부문별 예측(2025-2030년)

Rare Disease Clinical Trials Market Growth & Trends:

The global rare disease clinical trials market size is expected to reach USD 19.02 billion by 2030, expanding at 6.8% CAGR from 2025 to 2030, according to a new report by Grand View Research, Inc. The main driving factors of the rare disease clinical trial industry are the high burden of rare diseases worldwide, which has led to rising demand for research. Furthermore, intermittent launches and a large number of products in the pipeline also propel growth. Government initiatives that encourage product development, such as the Orphan Drug Act, are further expected to assist industry growth.

According to the National Institutes of Health (NIH), there are more than 7,000 rare diseases worldwide. These diseases affect nearly 30 million Americans, or 1 in every 10 people. Eurodis states that 72% of rare diseases are genetic, while others are caused by infections, allergies, and environmental causes. The growing awareness among people regarding rare diseases and growing interest in developing potential treatments for treating rare diseases are expected to support market growth.

Pharmaceutical companies and non-profit organizations are actively providing funding for rare disease clinical research. For instance, in February 2022, the Health Research Board funded USD 1.1 million to support rare disease clinical trials. Similar initiatives in the future are expected to support the market during the forecast period.

Orphan drug sales have been steadily increasing in recent years. For instance, according to Evaluate Pharma, orphan drug sales accounted for USD 138 billion in 2020, and by 2022, they accounted for USD 172 billion. It is estimated that orphan drug sales will reach USD 268 billion by 2026. The rise in the sales of orphan drugs is expected to generate interest among researchers in developing new treatments in the area of rare diseases and thus support the market in the post-pandemic period.

Rare Disease Clinical Trials Market Report Highlights:

• The phase III segment dominated the market with the largest revenue share of 54.2% in 2024. The segment's growth is supported by the high cost of phase III rare disease clinical trials due to the requirement for a large number of volunteers
• The oncology segment dominated the market with the largest revenue share in 2024. The growing interest of researchers in the development of new potential treatments for treating rare cancers is supporting the growth of the segment
• The non-profit organizations segment is projected to grow at a significant CAGR over the forecast period owing to the significant funding offered by these organizations to support rare disease clinical research
• North America dominated the market and accounted for a revenue share of 42.6% in 2024. The high burden of rare diseases in North America and the presence of a large number of players in clinical trials are some of the key reasons for its high market share

Table of Contents

Chapter 1. Methodology and Scope

• 1.1. Market Segmentation & Scope
• 1.2. Segment Definitions
  • 1.2.1. Phase
  • 1.2.2. Therapeutic Area
  • 1.2.3. Sponsor
• 1.3. Estimates and Forecast Timeline
• 1.4. Research Methodology
• 1.5. Information Procurement
  • 1.5.1. Purchased Database
  • 1.5.2. GVR's Internal Database
  • 1.5.3. Secondary Sources
  • 1.5.4. Primary Research
• 1.6. Information Analysis
  • 1.6.1. Data Analysis Models
• 1.7. Market Formulation & Data Visualization
• 1.8. Model Details
  • 1.8.1. Commodity Flow Analysis
• 1.9. List of Secondary Sources
• 1.10. Objectives

Chapter 2. Executive Summary

• 2.1. Market Snapshot
• 2.2. Segment Snapshot
• 2.3. Competitive Landscape Snapshot

Chapter 3. Rare Disease Clinical Trials Market Variables, Trends, & Scope

• 3.1. Market Lineage Outlook
• 3.2. Market Dynamics
  • 3.2.1. Market Driver Analysis
  • 3.2.2. Market Restraint Analysis
• 3.3. Business Environment Analysis
  • 3.3.1. Industry Analysis - Porter's Five Forces Analysis
    • 3.3.1.1. Supplier Power
    • 3.3.1.2. Buyer Power
    • 3.3.1.3. Substitution Threat
    • 3.3.1.4. Threat of New Entrants
    • 3.3.1.5. Competitive Rivalry
  • 3.3.2. PESTLE Analysis

Chapter 4. Rare Disease Clinical Trials Market: Phase Business Analysis

• 4.1. Phase Market Share, 2024 & 2030
• 4.2. Phase Segment Dashboard
• 4.3. Market Size & Forecasts and Trend Analysis, by Phase, 2018 to 2030 (USD Billion)
• 4.4. Phase I
  • 4.4.1. Phase I Market, 2018 - 2030 (USD Billion)
• 4.5. Phase II
  • 4.5.1. Phase II Market, 2018 - 2030 (USD Billion)
• 4.6. Phase III
  • 4.6.1. Phase II Market, 2018 - 2030 (USD Billion)
• 4.7. Phase IV
  • 4.7.1. Phase IV Market, 2018 - 2030 (USD Billion)

Chapter 5. Rare Disease Clinical Trials Market: Therapeutic Area Business Analysis

• 5.1. Therapeutic Area Market Share, 2024 & 2030
• 5.2. Therapeutic Area Segment Dashboard
• 5.3. Market Size & Forecasts and Trend Analysis, by Therapeutic Area, 2018 to 2030 (USD Billion)
• 5.4. Oncology
  • 5.4.1. Oncology Market, 2018 - 2030 (USD Billion)
• 5.5. Cardiovascular Disorders
  • 5.5.1. Cardiovascular Disorders Market, 2018 - 2030 (USD Billion)
• 5.6. Neurological Disorders
  • 5.6.1. Neurological Disorders Market, 2018 - 2030 (USD Billion)
• 5.7. Infectious Disease
  • 5.7.1. Infectious Disease Market, 2018 - 2030 (USD Billion)
• 5.8. Genetic Disorders
  • 5.8.1. Genetic Disorders Market, 2018 - 2030 (USD Billion)
• 5.9. Autoimmune and Inflammation
  • 5.9.1. Autoimmune and Inflammation Market, 2018 - 2030 (USD Billion)
• 5.10. Hematologic Disorders
  • 5.10.1. Hematologic Disorders Market, 2018 - 2030 (USD Billion)
• 5.11. Musculoskeletal Disorders
  • 5.11.1. Musculoskeletal Disorders Market, 2018 - 2030 (USD Billion)
• 5.12. Others
  • 5.12.1. Others Market, 2018 - 2030 (USD Billion)

Chapter 6. Rare Disease Clinical Trials Market: Sponsor Business Analysis

• 6.1. Sponsor Market Share, 2024 & 2030
• 6.2. Sponsor Segment Dashboard
• 6.3. Market Size & Forecasts and Trend Analysis, by Sponsor, 2018 to 2030 (USD Billion)
• 6.4. Pharmaceutical & Biopharmaceutical Companies
  • 6.4.1. Pharmaceutical & Biopharmaceutical Companies Market, 2018 - 2030 (USD Billion)
• 6.5. Non-Profit Organizations
  • 6.5.1. Non-Profit Organizations Market, 2018 - 2030 (USD Billion)
• 6.6. Other
  • 6.6.1. Other Market, 2018 - 2030 (USD Billion)

Chapter 7. Rare Disease Clinical Trials Market: Regional Estimates & Trend Analysis

• 7.1. Regional Market Share Analysis, 2024 & 2030
• 7.2. Regional Market Dashboard
• 7.3. Market Size & Forecasts Trend Analysis, 2018 to 2030:
• 7.4. North America
  • 7.4.1. North America Rare Disease Clinical Trials Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.4.2. U.S.
    • 7.4.2.1. Key Country Dynamics
    • 7.4.2.2. Regulatory Framework
    • 7.4.2.3. Competitive Insights
    • 7.4.2.4. U.S. Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.4.3. Canada
    • 7.4.3.1. Key Country Dynamics
    • 7.4.3.2. Regulatory Framework
    • 7.4.3.3. Competitive Insights
    • 7.4.3.4. Canada Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.4.4. Mexico
    • 7.4.4.1. Key Country Dynamics
    • 7.4.4.2. Regulatory Framework
    • 7.4.4.3. Competitive Insights
    • 7.4.4.4. Mexico Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 7.5. Europe
  • 7.5.1. Europe Rare Disease Clinical Trials Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.5.2. UK
    • 7.5.2.1. Key Country Dynamics
    • 7.5.2.2. Regulatory Framework
    • 7.5.2.3. Competitive Insights
    • 7.5.2.4. UK Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.3. Germany
    • 7.5.3.1. Key Country Dynamics
    • 7.5.3.2. Regulatory Framework
    • 7.5.3.3. Competitive Insights
    • 7.5.3.4. Germany Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.4. France
    • 7.5.4.1. Key Country Dynamics
    • 7.5.4.2. Regulatory Framework
    • 7.5.4.3. Competitive Insights
    • 7.5.4.4. France Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.5. Italy
    • 7.5.5.1. Key Country Dynamics
    • 7.5.5.2. Regulatory Framework
    • 7.5.5.3. Competitive Insights
    • 7.5.5.4. Italy Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.6. Spain
    • 7.5.6.1. Key Country Dynamics
    • 7.5.6.2. Regulatory Framework
    • 7.5.6.3. Competitive Insights
    • 7.5.6.4. Spain Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.7. Denmark
    • 7.5.7.1. Key Country Dynamics
    • 7.5.7.2. Regulatory Framework
    • 7.5.7.3. Competitive Insights
    • 7.5.7.4. Denmark Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.8. Sweden
    • 7.5.8.1. Key Country Dynamics
    • 7.5.8.2. Regulatory Framework
    • 7.5.8.3. Competitive Insights
    • 7.5.8.4. Sweden Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.5.9. Norway
    • 7.5.9.1. Key Country Dynamics
    • 7.5.9.2. Regulatory Framework
    • 7.5.9.3. Competitive Insights
    • 7.5.9.4. Norway Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 7.6. Asia Pacific
  • 7.6.1. Asia Pacific Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.2. Japan
    • 7.6.2.1. Key Country Dynamics
    • 7.6.2.2. Regulatory Framework
    • 7.6.2.3. Competitive Insights
    • 7.6.2.4. Japan Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.3. China
    • 7.6.3.1. Key Country Dynamics
    • 7.6.3.2. Regulatory Framework
    • 7.6.3.3. Competitive Insights
    • 7.6.3.4. China Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.4. India
    • 7.6.4.1. Key Country Dynamics
    • 7.6.4.2. Regulatory Framework
    • 7.6.4.3. Competitive Insights
    • 7.6.4.4. India Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.5. South Korea
    • 7.6.5.1. Key Country Dynamics
    • 7.6.5.2. Regulatory Framework
    • 7.6.5.3. Competitive Insights
    • 7.6.5.4. South Korea Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.6. Australia
    • 7.6.6.1. Key Country Dynamics
    • 7.6.6.2. Regulatory Framework
    • 7.6.6.3. Competitive Insights
    • 7.6.6.4. Australia Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.6.7. Thailand
    • 7.6.7.1. Key Country Dynamics
    • 7.6.7.2. Regulatory Framework
    • 7.6.7.3. Competitive Insights
    • 7.6.7.4. Thailand Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 7.7. Latin America
  • 7.7.1. Latin America Rare Disease Clinical Trials Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.7.2. Brazil
    • 7.7.2.1. Key Country Dynamics
    • 7.7.2.2. Regulatory Framework
    • 7.7.2.3. Competitive Insights
    • 7.7.2.4. Brazil Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.7.3. Argentina
    • 7.7.3.1. Key Country Dynamics
    • 7.7.3.2. Regulatory Framework
    • 7.7.3.3. Competitive Insights
    • 7.7.3.4. Argentina Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 7.8. MEA
  • 7.8.1. MEA Rare Disease Clinical Trials Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 7.8.2. South Africa
    • 7.8.2.1. Key Country Dynamics
    • 7.8.2.2. Regulatory Framework
    • 7.8.2.3. Competitive Insights
    • 7.8.2.4. South Africa Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.8.3. Saudi Arabia
    • 7.8.3.1. Key Country Dynamics
    • 7.8.3.2. Regulatory Framework
    • 7.8.3.3. Competitive Insights
    • 7.8.3.4. Saudi Arabia Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.8.4. UAE
    • 7.8.4.1. Key Country Dynamics
    • 7.8.4.2. Regulatory Framework
    • 7.8.4.3. Competitive Insights
    • 7.8.4.4. UAE Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 7.8.5. Kuwait
    • 7.8.5.1. Key Country Dynamics
    • 7.8.5.2. Regulatory Framework
    • 7.8.5.3. Competitive Insights
    • 7.8.5.4. Kuwait Rare Disease Clinical Trials Market Estimates and Forecasts, 2018 - 2030 (USD Billion)

Chapter 8. Competitive Landscape

• 8.1. Participant Overview
• 8.2. Company Market Position Analysis
• 8.3. Company Categorization
• 8.4. Strategy Mapping
• 8.5. Company Profiles/Listing
  • 8.5.1. Takeda Pharmaceutical Company Limited
    • 8.5.1.1. Overview
    • 8.5.1.2. Financial Performance
    • 8.5.1.3. Product Benchmarking
    • 8.5.1.4. Strategic Initiatives
  • 8.5.2. F. Hoffmann-La Roche Ltd
    • 8.5.2.1. Overview
    • 8.5.2.2. Financial Performance
    • 8.5.2.3. Product Benchmarking
    • 8.5.2.4. Strategic Initiatives
  • 8.5.3. Pfizer Inc
    • 8.5.3.1. Overview
    • 8.5.3.2. Financial Performance
    • 8.5.3.3. Product Benchmarking
    • 8.5.3.4. Strategic Initiatives
  • 8.5.4. AstraZeneca
    • 8.5.4.1. Overview
    • 8.5.4.2. Financial Performance
    • 8.5.4.3. Product Benchmarking
    • 8.5.4.4. Strategic Initiatives
  • 8.5.5. Novartis AG
    • 8.5.5.1. Overview
    • 8.5.5.2. Financial Performance
    • 8.5.5.3. Product Benchmarking
    • 8.5.5.4. Strategic Initiatives
  • 8.5.6. Laboratory Corporation of America Holdings (LabCorp)
    • 8.5.6.1. Overview
    • 8.5.6.2. Financial Performance
    • 8.5.6.3. Product Benchmarking
    • 8.5.6.4. Strategic Initiatives
  • 8.5.7. IQVIA
    • 8.5.7.1. Overview
    • 8.5.7.2. Financial Performance
    • 8.5.7.3. Product Benchmarking
    • 8.5.7.4. Strategic Initiatives
  • 8.5.8. Charles River Laboratories
    • 8.5.8.1. Overview
    • 8.5.8.2. Financial Performance
    • 8.5.8.3. Product Benchmarking
    • 8.5.8.4. Strategic Initiatives
  • 8.5.9. ICON plc
    • 8.5.9.1. Overview
    • 8.5.9.2. Financial Performance
    • 8.5.9.3. Product Benchmarking
    • 8.5.9.4. Strategic Initiatives
  • 8.5.10. Parexel International (MA) Corporation
    • 8.5.10.1. Overview
    • 8.5.10.2. Financial Performance
    • 8.5.10.3. Product Benchmarking
    • 8.5.10.4. Strategic Initiatives