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.).
  • The rest of the revenue is generated working with academic and research not-for-profit institutes. We do our bit of pro-bono by working with these institutions at subsidized rates.

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)

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