Rare Disease Clinical Trials Market Size, Share, Trend and Global Analysis by Type, Application, Key Players, Region and Forecast 2023 To 2033

BySanket Badgujar
Market Research

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 upscaling 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 increasing clinical trials for rare diseases have grown significantly with numerous biopharmaceutical companies focusing on rare diseases.

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.

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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 due to the fact that 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.

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 and acquisitions and product launches, to strengthen their market position.

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.

Key Segments Covered

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