The Global Rare Disease Clinical Trials Market Size is valued at 12.28 billion in 2023 and is predicted to reach 25.05 billion by the year 2031 at a 9.39% CAGR during the forecast period for 2024-2031.
The lack of medications available to treat rare diseases, advancements in personalized medicine, and cell and gene therapies, which are opening up new pathways for new treatments for rare diseases, are a few of the causes contributing to this expansion. Also, a rise in financing for clinical trials for rare diseases from pharmaceutical, biotech, and non-profit groups is driving market expansion.
The COVID-19 outbreak significantly impacted the capacity to perform clinical trials. The pandemic forced the suspension and postponement of numerous clinical trials for uncommon diseases and the delay of participant recruitment. Due to this, new medications and therapies created for the treatment of rare diseases took longer. However, by the second half of 2020, clinical studies of rare illnesses had resumed, and they have now begun. This is likely to sustain the industry throughout the post-pandemic phase.
The Rare Disease Clinical Trials market is segmented by therapeutic area, phase, and sponsor. Based on the therapeutic site, the market is segmented as Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Disease, Genetic Disorders, Autoimmune and Inflammation, Hematologic Disorders, Musculoskeletal Disorders, and Others. By phase the market is segmented into Phase I, II, III, and IV. By drug, the market is segmented as OTC and Rx. By sponsor, the market is segmented as Pharmaceutical & Biopharmaceutical Companies, Non-profit Organizations, and Others.
The majority belonged to Phase II. Phase II studies are carried out in two stages; the first stage combines efficacy and dose range exploration; the second stage finalises the dose. Between 100 to 300 people are enrolled in Phase II clinical trials. The phase II category had the most registered clinical studies as of November 2022. On the ClinicalTrial.gov portal, 72,522 research had been registered as of November 2022. The large number of Phase II clinical studies is assisting in the segment's expansion.
The largest share belonged to the oncology sector. Some of the main drivers of the segment's growth are the high number of cancer drug approvals for rare diseases, the rise in cancer clinical trials, and the increased interest among scientists in developing effective treatments for uncommon malignancies. As an illustration, the USFDA authorised the immunotherapy medication atezolizumab (Tecentriq) in January 2023 for use in patients with advanced alveolar soft part sarcoma (ASPS).
Due to advantageous reimbursement rules, increased spending on orphan pharmaceuticals for the treatment of rare diseases, and the existence of prominent market participants who encouraged the creation of novel products, North America accounted for the most significant revenue share. Moreover, the U.S. FDA can approve medications used to treat critical disorders quickly. The FDA's drug approval procedure is streamlined by the Accelerated Approval Rules, which permit a treatment used to treat a critical ailment to be approved using a surrogate end-point. All of these elements are anticipated to boost the North American market.
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Report Attribute |
Specifications |
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Market size value in 2023 |
USD 12.28 Bn |
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Revenue forecast in 2031 |
USD 25.50 Bn |
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Growth rate CAGR |
CAGR of 9.39% from 2024 to 2031 |
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Quantitative units |
Representation of revenue in US$ Billion, and CAGR from 2024 to 2031 |
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Historic Year |
2019 to 2023 |
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Forecast Year |
2023-2031 |
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Report coverage |
The forecast of revenue, the position of the company, the competitive market statistics, growth prospects, and trends |
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Segments covered |
Therapeutic Area, Phase, And Sponsor |
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Regional scope |
North America; Europe; Asia Pacific; Latin America; Middle East & Africa |
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Country scope |
U.S.; Canada; U.K.; Germany; China; India; Japan; Brazil; Mexico; The UK; France; Italy; Spain; China; Japan; India; South Korea; Southeast Asia; South Korea; Southeast Asia |
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Competitive Landscape |
Takeda Pharmaceutical Company; F. Hoffmann-La Roche Ltd.; Pfizer, Inc.; AstraZeneca; Novartis AG; LabCorp; IQVIA, Inc.; Charles River Laboratories; Icon PLC; Parexel International Corporation. |
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Customization scope |
Free customization report with the procurement of the report, Modifications to the regional and segment scope. Particular Geographic competitive landscape. |
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Pricing and available payment methods |
Explore pricing alternatives that are customized to your particular study requirements. |
Chapter 1. Methodology and Scope
1.1. Research Methodology
1.2. Research Scope & Assumptions
Chapter 2. Executive Summary
Chapter 3. Global Rare Disease Clinical Trials Market Snapshot
Chapter 4. Global Rare Disease Clinical Trials Market Variables, Trends & Scope
4.1. Market Segmentation & Scope
4.2. Drivers
4.3. Challenges
4.4. Trends
4.5. Investment and Funding Analysis
4.6. Industry Analysis – Porter’s Five Forces Analysis
4.7. Competitive Landscape & Market Share Analysis
4.8. Impact of Covid-19 Analysis
Chapter 5. Market Segmentation 1: by Therapeutic Area Estimates & Trend Analysis
5.1. by Therapeutic Area & Market Share, 2023 & 2031
5.2. Market Size (Value (US$ Mn)) & Forecasts and Trend Analyses, 2019 to 2031 for the following by Therapeutic Area:
5.2.1. Oncology
5.2.2. Cardiovascular Disorders
5.2.3. Neurological Disorders
5.2.4. Infectious Disease
5.2.5. Genetic Disorders
5.2.6. Autoimmune and Inflammation
5.2.7. Hematologic Disorders
5.2.8. Musculoskeletal Disorders
5.2.9. Others
Chapter 6. Market Segmentation 2: by Phase Estimates & Trend Analysis
6.1. by Phase & Market Share, 2023 & 2031
6.2. Market Size (Value (US$ Mn)) & Forecasts and Trend Analyses, 2019 to 2031 for the following by Phase:
6.2.1. Phase I
6.2.2. Phase II
6.2.3. Phase III
6.2.4. Phase IV
Chapter 7. Market Segmentation 3: by Sponsor Estimates & Trend Analysis
7.1. by Sponsor & Market Share, 2023 & 2031
7.2. Market Size (Value (US$ Mn)) & Forecasts and Trend Analyses, 2019 to 2031 for the following by Sponsor:
7.2.2. Non-Profit Organizations
7.2.3. Others
Chapter 8. Rare Disease Clinical Trials Market Segmentation 4: Regional Estimates & Trend Analysis
8.1. North America
8.1.1. North America Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Therapeutic Area, 2023-2031
8.1.2. North America Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Phase, 2023-2031
8.1.3. North America Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Sponsor, 2023-2031
8.1.4. North America Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by country, 2023-2031
8.2. Europe
8.2.1. Europe Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Therapeutic Area, 2023-2031
8.2.2. Europe Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Phase, 2023-2031
8.2.3. Europe Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Sponsor, 2023-2031
8.2.4. Europe Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by country, 2023-2031
8.3. Asia Pacific
8.3.1. Asia Pacific Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Therapeutic Area, 2023-2031
8.3.2. Asia Pacific Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Phase, 2023-2031
8.3.3. Asia-Pacific Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Sponsor, 2023-2031
8.3.4. Asia Pacific Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by country, 2023-2031
8.4. Latin America
8.4.1. Latin America Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Therapeutic Area, 2023-2031
8.4.2. Latin America Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Phase, 2023-2031
8.4.3. Latin America Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Sponsor, 2023-2031
8.4.4. Latin America Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by country, 2023-2031
8.5. Middle East & Africa
8.5.1. Middle East & Africa Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Therapeutic Area, 2023-2031
8.5.2. Middle East & Africa Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Phase, 2023-2031
8.5.3. Middle East & Africa Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by Sponsor, 2023-2031
8.5.4. Middle East & Africa Rare Disease Clinical Trials Market Revenue (US$ Million) Estimates and Forecasts by country, 2023-2031
Chapter 9. Competitive Landscape
9.1. Major Mergers and Acquisitions/Strategic Alliances
9.2. Company Profiles
9.2.1. Takeda Pharmaceutical Company;
9.2.2. F. Hoffmann-La Roche Ltd.;
9.2.3. Pfizer, Inc.;
9.2.4. AstraZeneca;
9.2.5. Novartis AG;
9.2.6. LabCorp;
9.2.7. IQVIA, Inc.;
9.2.8. Charles River Laboratories;
9.2.9. Icon PLC;
9.2.10. Parexel International Corporation
9.2.11. Other Prominent Players
Rare Disease Clinical Trials Market By Therapeutic area-
Rare Disease Clinical Trials Market By Phase-
Rare Disease Clinical Trials Market By Sponsor-
Rare Disease Clinical Trials Market By Region-
North America-
Europe-
Asia-Pacific-
Latin America-
Middle East & Africa-
InsightAce Analytic follows a standard and comprehensive market research methodology focused on offering the most accurate and precise market insights. The methods followed for all our market research studies include three significant steps – primary research, secondary research, and data modeling and analysis - to derive the current market size and forecast it over the forecast period. In this study, these three steps were used iteratively to generate valid data points (minimum deviation), which were cross-validated through multiple approaches mentioned below in the data modeling section.
Through secondary research methods, information on the market under study, its peer, and the parent market was collected. This information was then entered into data models. The resulted data points and insights were then validated by primary participants.
Based on additional insights from these primary participants, more directional efforts were put into doing secondary research and optimize data models. This process was repeated till all data models used in the study produced similar results (with minimum deviation). This way, this iterative process was able to generate the most accurate market numbers and qualitative insights.
Secondary research
The secondary research sources that are typically mentioned to include, but are not limited to:
The paid sources for secondary research like Factiva, OneSource, Hoovers, and Statista
Primary Research:
Primary research involves telephonic interviews, e-mail interactions, as well as face-to-face interviews for each market, category, segment, and subsegment across geographies
The contributors who typically take part in such a course include, but are not limited to:
Data Modeling and Analysis:
In the iterative process (mentioned above), data models received inputs from primary as well as secondary sources. But analysts working on these models were the key. They used their extensive knowledge and experience about industry and topic to make changes and fine-tuning these models as per the product/service under study.
The standard data models used while studying this market were the top-down and bottom-up approaches and the company shares analysis model. However, other methods were also used along with these – which were specific to the industry and product/service under study.
To know more about the research methodology used for this study, kindly contact us/click here.
