Focus on Rare Diseases is the Crucial Trend in the Pharmaceutical Industry – The Rare Disease Clinical Trails Market is to Record 9.7% CAGR by 2033 | FMI

Explore the thriving Rare Disease Clinical Trials Market Detailed Information about Each Segment, and Identify Key Trends, Drivers, and Challenges in the Market.

NEWARK, Del, Sept. 12, 2023 (GLOBE NEWSWIRE) -- The rare disease clinical trials market has been growing prominently over the past few years. The market is driven by increased funding from governments and pharmaceutical companies. Moreover, advances in technology and understanding of rare diseases are improving the performance of the market.

According to FMI, the rare disease clinical trials market size is projected to be valued at US$ 12,566.14 million in 2023 and is expected to rise to US$ 31,715.25 million by 2033. The sales of rare disease clinical trials are expected to expand at a significant CAGR of 9.7% during the forecast period. The report states that clinical trials for rare diseases have grown significantly, with numerous biopharmaceutical companies focusing on rare diseases.

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One of the key drivers of market growth is the Orphan Drug Act. It provides incentives to pharmaceutical companies to develop treatments for rare diseases. Additionally, the rise in funding for rare disease research from both private and public sources has also contributed to the growth of the market.

Another key trend in the market is the use of innovative technologies such as gene therapy and precision medicine in rare disease clinical trials. These technologies offer the potential for more targeted and effective treatments for rare diseases.

The rare disease clinical trials market still faces several challenges. These challenges included difficulties in recruiting patients for clinical trials, high costs of drug development, and regulatory hurdles. Nonetheless, with the growing demand for treatments for rare diseases, the market is expected to continue to grow in the coming years.

Key Takeaways from the Rare Disease Clinical Trails Market

  • In terms of phase, Phase III is expected to have a high CAGR of 10.3% during the forecast period. The high proportion of Phase III trials is because they are expensive and involve subsequent subjects. In phase III trials, long-term safety studies are conducted for registration and post-marketing commitments.
  • High prevalence of rare diseases and the presence of a robust healthcare system for diagnosis and treatment are anticipated to expand North America to 49.3%.
  • Pharmaceutical and biopharmaceutical companies recorded a strong revenue share of 58.5% in 2022. Pharmaceutical companies are actively involved in rare disease clinical trials through collaboration with other companies.
  • Asia Pacific region is expected to hold a swift rate of 10.6%. The region's expansion can be attributed to government initiatives to assist orphan disease patients. For example, the Indian government directed national and state governments in July 2022 to ensure the effective implementation of health policies developed to treat patients suffering from orphan diseases.
  • During the forecast period, the nonprofit organization segment is expected to secure a CAGR of 9.9%. Non-profit and other public organizations are actively involved in funding rare disease clinical research to support the development of potential treatments for rare diseases.

Rare Disease Clinical Trials Market Competitive Landscape:

The market for rare disease treatment is competitive, with several leading players. A few key players are dominating the market in terms of market share. Amgen Inc., AstraZeneca (Alexion Pharmaceuticals Inc.), Bristol-Myers Squibb Company, Biomarin Pharmaceuticals, and Bayer AG are among the companies currently dominating the market. Market participants readily implement a variety of initiatives, such as mergers, acquisitions, and product launches, to strengthen their market position.

Rare Disease Clinical Trials Market Latest Developments:

  • ProtalixBiotherapeutics Inc. and Chiesi Global Rare Diseases resubmitted the Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) in November 2022. The license was submitted for PRX-102 (pegunigalsidasealfa) which is used for the treatment of adult patients with Fabry disease.
  • The National Institutes of Health, the United States Food and Drug Administration, ten pharmaceutical companies, and five non-profit organizations joined forces in October 2022. The collaboration took place to accelerate the development of gene therapies for people suffering from rare diseases.

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Top 10 Players in the Rare Disease Clinical Trials Market:

  1. Takeda Pharmaceutical Company
  2. F. Hoffmann-La Roche Ltd.
  3. Pfizer, Inc.
  4. AstraZeneca
  5. Novartis AG
  6. LabCorp
  7. IQVIA, Inc.
  8. Charles River Laboratories
  9. Icon PLC
  10. Parexel International Corporation

Rare Disease Clinical Trials Market Segmentation

Therapeutic Area Outlook:

  • Oncology
  • Cardiovascular Disorders
  • Neurological Disorders
  • Infectious Disease
  • Genetic Disorders
  • Autoimmune And Inflammation
  • Hematologic Disorders
  • Musculoskeletal Disorders
  • Others

Phase Outlook:

  • Phase I
  • Phase II
  • Phase III
  • Phase IV

Sponsor Outlook:

  • Pharmaceutical & Biopharmaceutical Companies
  • Non-profit Organizations
  • Others

By Region:

  • North America
  • Latin America
  • Western Europe
  • Eastern Europe
  • Asia Pacific excluding Japan (APEJ)
  • Japan
  • The Middle East & Africa (MEA)


Sabyasachi Ghosh (Associate Vice President at Future Market Insights, Inc.) holds over 12 years of experience in the Healthcare, Medical Devices, and Pharmaceutical industries. His curious and analytical nature helped him shape his career as a researcher.

Identifying key challenges faced by clients and devising robust, hypothesis-based solutions to empower them with strategic decision-making capabilities come naturally to him. His primary expertise lies in areas such as Market Entry and Expansion Strategy, Feasibility Studies, Competitive Intelligence, and Strategic Transformation.

Holding a degree in Microbiology, Sabyasachi has authored numerous publications and has been cited in journals, including The Journal of mHealth, ITN Online, and Spinal Surgery News

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