Global Rare Diseases Treatment Market Size & Outlook

The rare diseases treatment industry worldwide is expected to reach a projected revenue of US$ 374,391.0 million by 2030. A compound annual growth rate of 11.6% is expected of the worldwide rare diseases treatment industry from 2025 to 2030.
Revenue, 2024 (US$M)
$195,174.8
Forecast, 2030 (US$M)
$374,391.0
CAGR, 2025 - 2030
11.6%
Report Coverage
Worldwide

Global rare diseases treatment market, 2018-2030 (US$M)

Global rare diseases treatment market highlights

  • The global rare diseases treatment market generated a revenue of USD 195,174.8 million in 2024 and is expected to reach USD 374,391.0 million by 2030.
  • The market is expected to grow at a CAGR (2025 - 2030) of 11.6% by 2030.
  • In terms of segment, cancer accounted for a revenue of USD 60,824.3 million in 2024.
  • Musculoskeletal Conditions is the most lucrative therapeutic area segment registering the fastest growth during the forecast period.
  • In terms of region, North America was the largest revenue generating market in 2024.
  • Country-wise, India is expected to register the highest CAGR from 2025 to 2030.

Global data book summary

Market revenue in 2024USD 195,174.8 million
Market revenue in 2030USD 374,391.0 million
Growth rate11.6% (CAGR from 2025 to 2030)
Largest segmentCancer
Fastest growing segmentMusculoskeletal Conditions
Historical data covered2018 - 2023
Base year for estimation2024
Forecast period covered2025 - 2030
Quantitative unitsRevenue in USD million
Market segmentationCancer, Neurological Conditions, Cardiovascular Conditions, Musculoskeletal Conditions, Hematologic Disorders, Infectious Diseases, Metabolic Diseases, Endocrine Disorders

Other key industry trends

  • In terms of revenue, the North America accounted for 62.9% of the global rare diseases treatment market in 2024.
  • By country, the U.S. is projected to lead the global market in terms of revenue in 2030.
  • By country, India is the fastest growing regional market and is projected to reach USD 6,630.4 million by 2030.

No credit card required*

Horizon in a snapshot

  • 30K+ Global Market Reports
  • 120K+ Country Reports
  • 1.2M+ Market Statistics
  • 200K+ Company Profiles
  • Industry insights and more

Rare Diseases Treatment Market Companies

Name Profile # Employees HQ Website

Global rare diseases treatment 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 diseases treatment market will help companies and investors design strategic landscapes.


Cancer was the largest segment with a revenue share of 31.16% in 2024. Horizon Databook has segmented the Global rare diseases treatment market based on cancer, neurological conditions, cardiovascular conditions, musculoskeletal conditions, hematologic disorders, infectious diseases, metabolic diseases, endocrine disorders covering the revenue growth of each sub-segment from 2018 to 2030.


  • Global Rare Diseases Treatment Therapeutic Area Outlook (Revenue, USD Million, 2018-2030)
    • Cancer
    • Neurological Conditions
    • Cardiovascular Conditions
    • Musculoskeletal Conditions
    • Hematologic Disorders
    • Infectious Diseases
    • Metabolic Diseases
    • Endocrine Disorders
    • Others
  • Global Rare Diseases Treatment Route of Administration Outlook (Revenue, USD Million, 2018-2030)
    • Oral
    • Injectable
    • Others
  • Global Rare Diseases Treatment Drug Type Outlook (Revenue, USD Million, 2018-2030)
    • Biologics
    • Biosimilar
    • Small molecules
  • Global Rare Diseases Treatment Distribution Channel Outlook (Revenue, USD Million, 2018-2030)
    • Hospital Pharmacy
    • Specialty Pharmacy
    • Online Pharmacy

Reasons to subscribe to Global rare diseases treatment market databook:

  • Access to comprehensive data: Horizon Databook provides over 1 million market statistics and 20,000+ reports, offering extensive coverage across various industries and regions.
  • Informed decision making: Subscribers gain insights into market trends, customer preferences, and competitor strategies, empowering informed business decisions.
  • Cost-Effective solution: It's recognized as the world's most cost-effective market research database, offering high ROI through its vast repository of data and reports.
  • Customizable reports: Tailored reports and analytics allow companies to drill down into specific markets, demographics, or product segments, adapting to unique business needs.
  • Strategic advantage: By staying updated with the latest market intelligence, companies can stay ahead of competitors, anticipate industry shifts, and capitalize on emerging opportunities.

Target buyers of Global rare diseases treatment market databook

  • Our clientele includes a mix of rare diseases treatment 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 diseases treatment market , including forecasts for subscribers. This global databook contains high-level insights into Global rare diseases treatment market from 2018 to 2030, including revenue numbers, major trends, and company profiles.

Partial client list

  • logo

Global rare diseases treatment market report size, by regions, 2018-2030 (US$M)

Top 10 countries: Rare diseases treatment market report size, 2024 (US$M)

Global rare diseases treatment market report share, by therapeutic area, 2024 & 2030 (%, US$M)

Rare diseases treatment market: Opportunity assessment by country

Global rare diseases treatment market, by region, 2024 (US$M)

Global rare diseases treatment market report size, by regions, 2018-2030 (US$M)

Sign up - it's easy, and free!

Sign up and get instant basic access to databook, upgrade
when ready, or enjoy our free plan indefinitely.

Explore all plans

Included in Horizon account

  • 30K+ Global Market Reports
  • 120K+ Country Reports
  • 1.2M+ Market Statistics
  • 200K+ Company Profiles
  • Industry insights and more