Rare diseases affect a small percentage of the population at any given time. Given the rarity of some of these diseases there is limited diagnostic as well treatment information available. According to Genetic and Rare Diseases Information Centre (GARD), there are more than 7,000 rare diseases. Generally, rare diseases are categorized as diseases having a prevalence of less than 5 in 10,000 people. As such, there is a huge disparity between diagnosed rare diseases and available treatment options. Rare diseases treatments are also called as orphan drugs and orphan diseases respectively.  

Market Drivers and Restraints:

• Increasing prevalence of rare diseases is expected to support growth of the global rare disease treatment market. For instance, according to Rare Genomics Institute, ~300 million people worldwide are affected by rare diseases.  
• Established global organizations as well as start-up’s have introduced novel therapies for the treatment of some of the rare diseases such as Hutchinson-Gilford progeria syndrome. For instance, in January 2021, Eiger Biopharmaceuticals launched Zokinvy (lonafarnib) which is the first and only treatment available for Progeria and Processing-Deficient Progeroid Laminopathies. 

However, there are certain limitations associated with orphan diseases treatment development such as limited knowledge, diagnostic capability, and limited patient pool for effective research and development. Also, high treatment costs also limit accessibility of the treatment. These factors are expected to restrain global rare disease treatment market growth up to certain extent.   

Global Rare Disease Treatment Market accounted for US$ 142.84 Bn in 2022 and is expected to witness a CAGR of 11.6% over the forecast period (2022-2030) to reach US$ 341.29 Bn by 2030

Competitive Landscape:

The global rare disease treatment market is fairly fragmented with multinational corporations and smaller companies operating in the industry. Leading companies operating in the market such as Celgene, Roche, and Novartis are offering advanced treatment options for rare diseases and are working on developing novel therapies for such diseases.

We have employed tools and techniques such as PEST analysis, SWOT analysis, PORTER’s analysis, BCG matrix to provide competitive and market intelligence.

Some of the prominent players operating in the market are:

• F. Hoffmann-La Roche Ltd.
• Pfizer, Inc.
• PTC Therapeutics
• AstraZeneca
• Novartis AG
• Takeda Pharmaceutical Company
• Bayer AG
• AbbVie Inc.
• Merck & Co. Inc.
• Bristol Myers Squibb

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