Global Rare Disease Treatment Market Outlook, Trends And Future Opportunities (2023-2030)

Global Rare Disease Treatment Market is Forecasted to Hit US$ 341.29 Bn By 2030 | CAGR 11.6%

  • Date: 27 Sep, 2023

Global rare disease treatment market is expected to witness a growth rate of 11.6% to reach US$ 341.29 Bn market size by 2030.

Industry drivers and trends:

Rare diseases treatment space has seen high earning brands over the past few years which has garnered attention of researchers and pharmaceutical industry alike. This has resulted in increased research and development of treatments for rare diseases. Subsequently, there have been successful product launches in the recent past which has propelled global rare disease treatment market growth. For instance, on December 14, 2022, Takeda Biopharmaceuticals launched Cinryze, an injectable prescription drug for the treatment of hereditary angioedema (HAE) patients in India.

Furthermore increasing prevalence of rare diseases has created a need of better patient condition monitoring, minimally invasive treatment, and possibly early diagnosis of diseases. For instance, according to the National Institutes of Health (NIH), there are approximately 7,000 rare diseases affecting between 25 and 30 million Americans.

  • Leading and newer players are engaged in collaborations and partnerships which is expected to support global rare disease treatment market growth over the forecast period. For instance, in December 2022, AbCellera and Rallybio Corporation entered into a strategic alliance to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases.
  • Global rare disease treatment market is being led by blockbuster oncology drugs such as monoclonal antibodies which are novel therapies.
  • Moreover, some of the blockbuster oncology drugs have lost patent protection over the last few years and have had to face competition from biosimilars. For instance, Rituxan (Roche) has faced tough competition from biosimilars since 2018.

Key Takeaways:

  • North America region occupies highest market share in the global rare disease treatment market followed by Europe
  • Oncology segment is expected to hold majority of the market share over the forecast period in the therapy area segment
  • Specialty pharmacies segment is expected to show highest growth over the forecast period owing to better access to patients, and availability
  • Factors such as low competition, fast-tracked regulatory review process and lower-cost late-stage development are further expected to support global rare disease treatment market growth over the forecast period

Segments Covered in the global rare disease treatment market report:

The global rare disease treatment market is segmented based on treatment type, therapy area, route of administration, distribution channel, region as follows:

  • Treatment Type (Revenue, USD Billion; 2022–2030)
    • Biologics
    • Biosimilar
    • Others
  • Therapy Area (Revenue, USD Billion; 2022–2030)
    • Oncology
    • Neurological Diseases
    • Cardiovascular Diseases
    • Infectious Diseases
    • Musculoskeletal Diseases
    • Hematologic Diseases
    • Others
  • Route of Administration (Revenue, USD Billion; 2022–2030)
    • Oral
    • Injectable
    • Others
  • Distribution Channel (Revenue, USD Billion; 2022–2030)
    • Hospital Pharmacies
    • Specialty Pharmacies
    • Online Pharmacies
  • Region (Revenue, USD Billion; 2022–2030)
    • North America
      • U.S.
      • Canada
    • Europe
      • Germany
      • U.K.
      • France
      • Italy
      • Spain
      • Rest of Europe
    • Asia Pacific
      • China
      • Japan
      • South Korea
      • India
      • ASEAN
      • Rest of APAC
    • Latin America
      • Brazil
      • Mexico
      • Rest of LATAM
    • Middle East & Africa
      • Saudi Arabia
      • UAE
      • Rest of MEA


The term "Rare Disease Treatment market" refers to the segment of the healthcare industry that deals with developing, producing, and providing medical therapies and interventions for rare diseases. Rare diseases, also known as orphan diseases, are conditions that affect a relatively small number of people in the population. These diseases often have a low prevalence and can be challenging to diagnose and treat due to their rarity.

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