Global Rare Disease Clinical Trials Market Size & Outlook

The rare disease clinical trials industry worldwide is expected to reach a projected revenue of US$ 16,069.6 million by 2030. A compound annual growth rate of 9.8% is expected of the worldwide rare disease clinical trials industry from 2024 to 2030.
Revenue, 2023 (US$M)
$8,368.1
Forecast, 2030 (US$M)
$16,069.6
CAGR, 2024 - 2030
9.8%
Report Coverage
Worldwide

Global rare disease clinical trials market, 2018-2030 (US$M)

Global rare disease clinical trials market highlights

  • The global rare disease clinical trials market generated a revenue of USD 8,368.1 million in 2023 and is expected to reach USD 16,069.6 million by 2030.
  • The market is expected to grow at a CAGR (2024 - 2030) of 9.8% by 2030.
  • In terms of segment, phase ii accounted for a revenue of USD 3,570.0 million in 2023.
  • Phase III is the most lucrative phase segment registering the fastest growth during the forecast period.
  • In terms of region, North America was the largest revenue generating market in 2023.
  • Country-wise, Kuwait is expected to register the highest CAGR from 2024 to 2030.

Global data book summary

Market revenue in 2023USD 8,368.1 million
Market revenue in 2030USD 16,069.6 million
Growth rate9.8% (CAGR from 2023 to 2030)
Largest segmentPhase ii
Fastest growing segmentPhase III
Historical data covered2018 - 2022
Base year for estimation2023
Forecast period covered2024 - 2030
Quantitative unitsRevenue in USD million
Market segmentationPhase I, Phase II, Phase III, Phase IV

Other key industry trends

  • In terms of revenue, the North America accounted for 49.1% of the global rare disease clinical trials market in 2023.
  • By country, the U.S. is projected to lead the global market in terms of revenue in 2030.
  • By country, Kuwait is the fastest growing regional market and is projected to reach USD 17.8 million by 2030.

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Rare Disease Clinical Trials Market Companies

Name Profile # Employees HQ Website

Global rare disease clinical trials market outlook

The databook is designed to serve as a comprehensive guide to navigating this sector. The databook focuses on market statistics denoted in the form of revenue and y-o-y growth and CAGR across the globe and regions. A detailed competitive and opportunity analyses related to rare disease clinical trials market will help companies and investors design strategic landscapes.


Phase ii was the largest segment with a revenue share of 42.66% in 2023. Horizon Databook has segmented the Global rare disease clinical trials market based on phase i, phase ii, phase iii, phase iv covering the revenue growth of each sub-segment from 2018 to 2030.


  • Global Rare Disease Clinical Trials Phase Outlook (Revenue, USD Million, 2018-2030)
    • Phase I
    • Phase II
    • Phase III
    • Phase IV
    • Others
  • Global Rare Disease Clinical Trials Study Design Outlook (Revenue, USD Million, 2018-2030)
    • Interventional trials
    • Observational trials
    • Expanded access trials
  • Global Rare Disease Clinical Trials Therapeutic Area Outlook (Revenue, USD Million, 2018-2030)
    • Cardiovascular Disorders
    • Neurological Disorders
    • Infectious Disease
    • Genetic Disorders
    • Autoimmune and Inflammation
    • Hematologic Disorders
    • Musculoskeletal Disorders
    • Others

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Target buyers of Global rare disease clinical trials market databook

  • Our clientele includes a mix of rare disease clinical trials market companies, investment firms, advisory firms & academic institutions.
  • 30% of our revenue is generated working with investment firms and helping them identify viable opportunity areas.
  • Approximately 65% of our revenue is generated working with competitive intelligence & market intelligence teams of market participants (manufacturers, service providers, etc.).
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Horizon Databook provides a detailed overview of global-level data and insights on the Global rare disease clinical trials market , including forecasts for subscribers. This global databook contains high-level insights into Global rare disease clinical trials market from 2018 to 2030, including revenue numbers, major trends, and company profiles.

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Global rare disease clinical trials market size, by region, 2018-2030 (US$M)

Top 10 countries: Rare disease clinical trials market size, 2023 (US$M)

Global rare disease clinical trials market share, by phase, 2023 & 2030 (%, US$M)

Rare disease clinical trials market: Opportunity assessment by country

Global rare disease clinical trials market, by region, 2023 (US$M)

Global rare disease clinical trials market size, by region, 2018-2030 (US$M)

Phase 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 CAGR
Latin America 223.3 248.7 244.9 271.6 300.3 331.2 364.3 399.6 437.4 477.7 522.4 571.8 626.7 7.5%
Europe 1,564.1 1,750.7 1,732.2 1,930.1 2,144.3 2,375.8 2,625.6 2,894.0 3,182.3 3,492.6 3,837.1 4,220.3 4,647.1 7.9%
North America 2,789.6 3,103.5 3,052.3 3,380.4 3,733.0 4,111.3 4,516.2 4,948.0 5,408.0 5,899.7 6,442.7 7,043.4 7,709.2 7.4%
MEA 167.5 186.0 182.5 201.5 221.8 243.2 265.8 289.6 314.5 340.7 369.2 400.2 433.9 6.8%
Asia Pacific 837.7 942.6 937.7 1,050.3 1,173.1 1,306.7 1,451.7 1,608.7 1,778.3 1,962.1 2,167.2 2,396.3 2,652.7 8.4%
Overall 5,582.1 6,231.5 6,149.6 6,834.0 7,572.5 8,368.1 9,223.6 10,139.9 11,120.5 12,172.9 13,338.6 14,631.9 16,069.6 9.8%

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