Rare Disease Clinical Trials Market

Rare Disease Clinical Trials Market

The rare disease clinical trials market size is projected to be valued at USD 12,566.14 million in 2023 and is expected to rise to USD 31,715.25 million by 2033. The sales of rare disease clinical trials are expected to record a significant CAGR of 9.7% during the forecast period.

Various Factors Propelling the Demand for Rare Disease Clinical Trials

The rare disease clinical trials market refers to the development of drugs and therapies for the treatment of rare diseases. These diseases, by definition, affect a small percentage of the population. Consequently, there is often limited research and development in this area.

Clinical trials for rare diseases can be challenging due to the small patient population, limited natural history data, and a lack of standardized endpoints. However, the market is growing due to increased funding and attention to the development of treatments for these diseases.

For example, The Orphan Drug Act was passed in the United States in 1983. The act was enacted to offer financial incentives to pharmaceutical companies to develop treatments for rare diseases.

The advancement in technology and genomics allows for more targeted treatments and personalized medicine. Additionally, there has been increased collaboration between patient advocacy groups, researchers, and the industry. This collaboration has helped to overcome some challenges of conducting clinical trials in rare diseases.

Overall, the market represents an important area of research and development. As it offers hope for patients with conditions that may have previously been considered untreatable.

Attribute	Details
Rare Disease Clinical Trials Market Estimated Size (2023)	USD 12,566.14 million
Rare Disease Clinical Trials Market CAGR (2023 to 2033)	9.7%
Rare Disease Clinical Trials Market Forecasted Size (2033)	USD 31,715.25 million

2018 to 2022 Rare Disease Clinical Trials Market Outlook Compared to 2023 to 2033 Forecast

The rare disease clinical trials market was valued at USD 11,455.0 million in 2022 with a substantial growth trajectory of 8.9% between the analysis period of 2018 to 2022.

Future Market Insights expects the rare disease clinical trials market to expand at a CAGR of 9.7% during the forecast period of 2023 to 2033. The growth can be attributed to the following reasons

• Increased Focus on Gene Therapies: Gene therapies are emerging as a promising treatment option for rare diseases caused by genetic mutations. Several gene therapies have been approved by regulatory authorities and some are in the pipeline.
• Growing Interest in RNA-targeted Therapies: These therapies aim to modulate the expression of specific genes. It is also gaining attention as a potential treatment for rare diseases. Several RNA-targeted therapies are currently in development for rare diseases such as spinal muscular atrophy and Huntington's disease.
• Increasing Collaboration between Stakeholders: Collaboration between patient advocacy groups, researchers, and industry in the rare disease clinical trials market. This collaboration can overcome some challenges of conducting clinical trials in rare diseases. Moreover, it can help to accelerate the development of new treatments.

Considering the above-mentioned factors, FMI opines, the rare disease clinical trials market is likely to witness a market value of USD 31,715.25 million by the end of 2033.

Investment Opportunities in the Market

• Increasing Use of Precision Medicine: Precision medicine involves tailoring treatments to the specific genetic and molecular characteristics of an individual patient. With advances in genomics and personalized medicine, there is a growing trend towards developing treatments that are targeted towards specific rare diseases.
• Growth in the Use of Adaptive Clinical Trial Designs: Adaptive clinical trials allow for changes to be made to the trial design based on interim data. This can help to improve the efficiency of rare disease clinical trials, as it allows for the identification of effective treatments quickly and efficiently.
• Emphasis on Patient-centricity: Patient advocacy groups and patient representatives are increasingly involved in the design and implementation of rare disease clinical trials. They ensure that patient perspectives are taken into account.
• Use of Real-world Evidence: Real-world evidence, such as data from electronic health records and patient registries, is increasingly being used to supplement clinical trial data in rare disease research. This can help to provide a complete picture of the safety and efficacy of treatments.
• Increasing Use of Virtual and Remote Clinical Trials: The COVID-19 pandemic has accelerated the trend toward virtual and remote clinical trials. It also allowed patients to participate in clinical trials from the comfort of their own homes. This has increased patient participation in rare disease clinical trials, which can be challenging due to the small patient population.

Category-wise Insights of Rare Disease Clinical Trials

Insights into the Therapeutic Domain

In 2022, the oncology segment held a notable share of 33.9%. A few of the key factors fueling the segment growth are

• The high amount of cancer rare disease drug approvals
• Increasing clinical trials for cancer treatment
• Researchers' growing interest in finding effective treatments for rare cancers.

For example, in January 2023, the USFDA approved atezolizumab (Tecentriq), an immunotherapy drug for advanced alveolar soft part sarcoma patients (ASPS).

During the forecast period, the infectious disease segment is expected to expand at an exponential rate of 10.6%. Acanthamoeba keratitis, Q fever, Marburg virus, and other rare infectious diseases are examples. As these rare diseases are fatal, researchers are hugely interested in developing treatments and vaccines for them.

Contact lens wearers are likely to develop Acanthamoeba keratitis. Over 85% of people in the United States wear contact lenses. This increases the risk of disease and fuels the demand for treatment. These factors are assisting segment growth.

High Amount of Clinical Studies is Driving the Phase II Segment

In 2022, Phase II had a notable share of 42.6%. Phase II studies are divided into two parts: the first part includes dose range exploration as well as efficacy studies, while the second part includes dose finalization. Over 100-300 participants are recruited for Phase II clinical trials.

The phase II segment had the most registered clinical studies as of November 2022. The ClinicalTrial.gov portal had 72,522 studies registered as of November 2022. The high amount of Phase II clinical trials is driving the segment's growth.

Regional Analysis

In 2022, North America had a Strong Revenue Share of 49.3%. Highly Developed Healthcare Infrastructure is Providing Significant Opportunity in the United States.

The United States has a well-established regulatory framework for rare disease clinical trials, with the Orphan Drug Act of 1983. It provides incentives for the development of drugs for rare diseases. The FDA's Breakthrough Therapy Designation program has also facilitated the development of drugs for rare diseases. Additionally, the National Institutes of Health (NIH) has established a Rare Diseases Clinical Research Network (RDCRN) to facilitate the conduct of clinical trials for rare diseases.

Europe Market Insight

Europe is a significant market for rare disease clinical trials, with countries like Germany, France, and the United Kingdom accounting for a significant share of the market. The European Medicines Agency (EMA) has been proactive in offering regulatory support for rare disease drug development. Therefore, the support from several agencies has increased the clinical trials in Europe.

Asia Pacific Region is Anticipated to Increase at a Robust Pace of 10.6%

Asia Pacific is a significantly growing market for rare disease clinical trials, with countries like China, Japan, and India emerging as key players. The region offers a substantial patient population and low trial costs compared to Western countries. Making it an attractive destination for colossal clinical trials. However, the regulatory environment in some countries can be challenging, and there is a need for great awareness and education about rare diseases.

Latin America

Latin America is an emerging market for rare disease clinical trials, with countries like Brazil and Mexico. The region offers a diverse patient population and low trial costs compared to North America and Europe. However, there are challenges in terms of regulatory compliance and infrastructure, which can limit the growth of the market.

The Middle East and Africa

The Middle East and Africa region is a small but growing market for rare disease clinical trials. Countries like South Africa and Israel are emerging as key players due to their advanced healthcare infrastructure and supportive regulatory environment. However, there are challenges related to political instability, low awareness about rare diseases, and limited healthcare resources in some parts of the region.

Competitive Landscape - Key Players Operating in the Market

The rare disease clinical trials market is highly competitive, with several global players operating in the space. Some key players in the market include

• IQVIA Holdings Inc.: A leading provider of clinical trial services, including patient recruitment and management, data management, and regulatory compliance.
• PRA Health Sciences Inc.: A global clinical research organization (CRO) that offers a range of services, including clinical trial management, data management, and regulatory affairs.
• PPD Inc.: A leading CRO that provides a range of clinical trial services, including site selection, patient recruitment, and project management.
• Syneos Health Inc.: A global CRO that offers a range of services, including clinical trial management, data management, and regulatory affairs.
• Charles River Laboratories International Inc.: A provider of preclinical and clinical development services, including toxicology testing and clinical trial management.
• Covance Inc.: A leading CRO that offers a range of clinical trial services, including site selection, patient recruitment, and data management.
• Parexel International Corporation: A global CRO that provides a range of clinical trial services, including project management, data management, and regulatory affairs.
• ICON plc: A provider of clinical trial services, including study design, patient recruitment, and data management.

In addition to these companies, there are many other small CROs and specialized service providers operating in the rare disease clinical trials market. As the market continues to grow, competition among these companies is expected to increase. These players are competing on factors such as service quality, expertise, and pricing.

Scope of the Report

Attribute	Details
Growth Rate	CAGR of 9.7% from 2023 to 2033
Base Year of Estimation	2022
Historical Data	2018 to 2022
Forecast Period	2023 to 2033
Quantitative Units	Revenue in USD million and Volume in Units and F-CAGR from 2023 to 2033
Report Coverage	Revenue Forecast, Volume Forecast, Company Ranking, Competitive Landscape, growth factors, Trends, and Pricing Analysis
Key Segments Covered	By Therapeutic Area, By Sponsor, By Phases, By Region
Regions Covered	North America; Latin America; Europe; East Asia; South Asia; The Middle East & Africa;Oceania
Key Countries Profiled	The United States, Canada, Brazil, Mexico, Germany, Italy, France, The United Kingdom, Spain, Russia, China, Japan, India, GCC Countries, Australia
Key Companies Profiled	Takeda Pharmaceutical Company; F. Hoffmann-La Roche Ltd.; Pfizer, Inc.; AstraZeneca; Novartis AG; LabCorp; IQVIA, Inc.; Charles River Laboratories; Icon PLC; Parexel International Corporation
Customization & Pricing	Available upon Request

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

By Region:

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

Frequently Asked Questions

How Big is the Rare Disease Clinical Trials Market?

The market is valued at USD 12.5 billion in 2023.

Who are the Top Market Players?

Pfizer, Inc., AstraZeneca, and Novartis AG are the top market players.

How was the Historical Performance of the Market?

The market held a CAGR of 8.9% from 2018 to 2022.

What Opportunities Await the Market Players?

Advancements in personalized medicine to boost market growth.

Which is the Leading Segment by Phase Outlook?

Phase II segment is likely to remain preferred through 2033.