Rare Disease Clinical Trials Market Comprehensive Overview by Phase Sponsor and Therapeutic Segment with Market Drivers and Restraints

InsightAce Analytic Pvt. Ltd. announces the release of a market assessment report on the "Global Rare Disease Clinical Trials Market Size, Share & Trends Analysis Report By Therapeutic Area (Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Disease, Genetic Disorders, Autoimmune and Inflammation, Hematologic Disorders, Musculoskeletal Disorders), Phase (Phase I, II, III, and IV), And Sponsor (Pharmaceutical & Biopharmaceutical Companies, Non-profit Organizations)- Market Outlook And Industry Analysis 2031"

The global Rare Disease Clinical Trials market is estimated to reach over 25.05 billion by the year 2031, exhibiting a CAGR of 9.39% during the forecast period.

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The global market for rare disease treatments was notably impacted by the COVID-19 pandemic, which led to the widespread cancellation of elective procedures, including both therapeutic and diagnostic interventions. A review published in the Frontiers in Public Health journal in April 2021 highlighted the significant challenges faced by patients with rare diseases, concluding that routine clinical care, counseling, and therapies were largely inadequate during this period. The suspension of research initiatives and clinical trials for rare and genetic disorders further compounded difficulties for clinicians, researchers, and scientists in this field. However, following the easing of pandemic-related restrictions, the market has shown signs of recovery. It is projected to experience steady growth throughout the forecast period.

Moreover, the market expansion is being driven by increased research and development activities targeting innovative therapeutics, a rise in new drug approvals, the growing prevalence of rare diseases, supportive vaccination initiatives, and favorable government policies. These factors collectively contribute to the positive outlook and sustained growth of the market under analysis.

List of Prominent Players in the Rare Disease Clinical Trials Market:
• 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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Market Dynamics:
Drivers:
The expansion of the market is driven by ongoing treatment innovations, increasing product approvals, and strategic collaborations and acquisitions among leading companies. For example, in May 2022, the European Medicines Agency (EMA) approved Xenpozyme (olipudase alfa) for the treatment of non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), a rare and progressive genetic disorder. Xenpozyme represents the first ASMD-specific therapy authorized in the European Union, functioning by replacing the deficient or malfunctioning acid sphingomyelinase (ASM) enzyme. This enzyme replacement reduces intracellular fat accumulation and alleviates various symptoms associated with ASMD. Such regulatory approvals are expected to serve as significant catalysts for market growth during the forecast period.

Challenges:
Clinical trials for rare diseases, even at early phases I and II, often require multicenter and international collaboration to achieve sufficient patient enrollment. This complexity can challenge the coordination of study protocols, ethical reviews, liability arrangements, clinical service delivery, standards of care, and cultural considerations. Additionally, the rigorous demands placed on patients-such as administering additional medications, maintaining symptom diaries, and reporting adverse events-can exacerbate the existing disease burden, potentially impacting trial adherence and patient well-being.

Regional Trends:
North America is projected to retain a dominant share of the global rare disease treatment market, driven by the high prevalence of rare disorders such as Huntington's disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn's disease, cystic fibrosis, and Duchenne muscular dystrophy. The region benefits from heightened awareness and a robust healthcare infrastructure dedicated to the diagnosis and management of rare diseases. According to the Genetic and Rare Diseases (GARD) Information Center, approximately 7,000 rare diseases have been identified, with an estimated 30 million Americans (one in ten) affected. Similarly, the 2021 report from the Canadian Organization for Rare Disorders (CORD) estimates that one in twelve Canadians is impacted annually, with genetic mutations accounting for 80% of these cases. This significant disease burden underpins the anticipated strong market growth in the region over the coming years.

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Recent Developments:
• In January 2023, Genethon, an R&D company, began a crucial clinical trial for the use of gene therapy to treat Crigler-Najjar Syndrome. Crigler-Najjar syndrome is a rare genetic liver disease marked by abnormally elevated bilirubin levels in the blood. (hyperbilirubinemia).
• In November 2022, The Biologics License Application (BLA) for PRX-102 (pegunigalsidasealfa) for the treatment of adult patients with Fabry disease was re-submitted to the US Food and Drug Administration (FDA) by ProtalixBiotherapeutics Inc. and Chiesi Global Rare Diseases.

Segmentation of Rare Disease Clinical Trials Market-
By Therapeutic area-
• Oncology
• Cardiovascular Disorders
• Neurological Disorders
• Infectious Disease
• Genetic Disorders
• Autoimmune and Inflammation
• Hematologic Disorders
• Musculoskeletal Disorders
• Others
By Phase-
• Phase I
• Phase II
• Phase III
• Phase IV
By Sponsor-
• Pharmaceutical & Biopharmaceutical Companies
• Non-profit Organizations
• Others
By Region-
North America-
• The US
• Canada
• Mexico
Europe-
• Germany
• The UK
• France
• Italy
• Spain
• Rest of Europe
Asia-Pacific-
• China
• Japan
• India
• South Korea
• South East Asia
• Rest of Asia Pacific
Latin America-
• Brazil
• Argentina
• Rest of Latin America
Middle East & Africa-
• GCC Countries
• South Africa
• Rest of Middle East and Africa

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This release was published on openPR.