Rare Diseases Clinical Trials Market Size, Share, Opportunities, And Trends By Therapeutic Area (Oncology, Cardiovascular Disorders, Neurological Disorders, Infectious Diseases, Genetic Disorders, Autoimmune And Inflammation, Hematologic Disorder, Musculoskeletal Disorders, Others), By Phase (Phase I, Phase II, Phase III, Phase IV), By Sponsor (Pharmaceutical & Biopharmaceutical Companies, Non-profit Organizations, Others), And By Geography - Forecasts From 2024 To 2029

The rare diseases clinical trial market is projected to show steady growth during the forecast period.

A rare illness is defined as a health condition that affects a very small percentage of the population. A clinical trial serves as a research study aimed at identifying potential treatment options for uncommon medical conditions. By participating in clinical trials for rare diseases, patients can gain access to experimental therapies that have the potential to offer novel or improved approaches to managing their condition. According to Johnson and Johnson's February 2023 published article, in the United States, a rare disease is defined as a condition that affects fewer than 200,000 individuals, which is equivalent to 60 per 100,000 people. As per the same article, the European Union categorizes a disease as rare if it impacts no more than 50 per 100,000 individuals. Enhanced emphasis on gene therapies, assistance by the government for orphan diseases, and increased investment in research and development are driving the rare diseases clinical trial market growth.

Market Drivers:

• Growing emphasis on gene therapies boosts the rare diseases clinical trial market growth.

Gene therapy is an innovative medical strategy that seeks to address or hinder illnesses by introducing genetic material into the cells of a patient. For instance, according to AstraZeneca's press release in July 2023 Alexion, the rare disease division of AstraZeneca declared a conclusive agreement with Pfizer to procure a collection of preclinical gene therapy initiatives and facilitating technologies for rare diseases. This acquisition further bolsters Alexion's dedication to advancing groundbreaking and cutting-edge therapies for individuals suffering from rare diseases. The U.S Food and Drug Administration (FDA), in May 2023, approved Vyjuvek, a gene therapy based on the herpes simplex virus type 1 (HSV-1) vector, to be used in the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) who are 6 months of age and older and have mutations in the collagen type VII alpha 1 chain (COL7A1) gene.

• Assistance by the government for orphan diseases enhances the rare diseases trial market growth.

Government assistance such as tax incentives, funding, and exclusive market rights encourages companies to research and produce medications for uncommon illnesses, broadening the market with innovative treatment choices and drawing in additional financial backing. According to FDA August 2022 article, after receiving approval from the FDA, orphan drugs are eligible for seven years, where they have exclusive rights in the market. The provision enables companies to recover the initial investment and potentially generate profits that can be utilized for additional research purposes. The Office of Orphan Products Development (OOPD) under the FDA provides research grants to aid in the progress of potential orphan products. These grants offer monetary assistance for both preclinical and clinical research undertakings.

• Increased Investment in research and development propels the rare diseases trial market growth.

Increased funding for research and development is instrumental in advancing the rare disease clinical trial sector, as it helps identify new treatment possibilities, expands access, simplifies trial procedures, and reduces costs, ultimately attracting a more diverse group of participants. For instance, in July 2023, the National Organization for Rare Disorders (NORD) and the Autoimmune Polyglandular Syndrome (APS) Type 1 Foundation collaborated to grant an allocation of $50,000 for the investigation of the uncommon autoimmune condition. Autoimmune Polyglandular Syndrome (APS) Type 1 is a rare and intricate hereditary autoimmune disorder resulting from mutations in the AIRE gene. For instance, in July 2023 Medical Research Council (MRC) and the National Institute for Health and Care Research (NIHR) jointly invested £14 million for the establishment of a “UK Rare Disease Research” platform that would foster new & innovative treatments development to target rare disease for five years.

North America is anticipated to hold a considerable share

North America is projected to account for a significant share of the rare diseases clinical trial market due to the favorable number of initiatives in the region to fight rare diseases. For instance, the Food and Drug Administration’s “FDA Rare Disease 20203” theme was "Investigating the Intersections with Rare Diseases”, which focused on overcoming challenges in clinical trials with limited patient numbers, especially among children, and improving inclusivity. The FDA is dedicated to advancing the development of products for rare diseases and is actively promoting patient engagement by fostering meaningful connections.

Asia Pacific is expected to grow significantly.

The Asia Pacific region is projected to experience the most rapid growth in the upcoming forecast period, which is primarily due to the efforts made by governments to assist patients with rare diseases. According to the “National Policy for Rare Disease 2021”, financial assistance of up to Rs. 50 lakhs are available for patients afflicted with any category of Rare Diseases, enabling them to receive treatment at any of the Centers of Excellence (CoEs) specified in NPRD-2021. The action opens up a chance for manufacturers to provide top-notch orphan drugs to the government and generate income. According to the Press India Bureau's July 2023 release, the sum of Rs. 92.84 Crore (Rs. Ninety-Two Crore Eighty-Four Lakh Only) has been allocated for the healthcare needs of patients suffering from rare diseases in the designated Centers of Excellence under the National Policy for Rare Diseases (NPRD), 2021, for the fiscal year 2023-24.

Market Restraints:

• Development expenses restrain the rare diseases clinical trial market growth.

Generating new therapies is inherently expensive, and the limited market potential for rare diseases exacerbates the challenge of recouping these costs for companies. As a result, this can discourage potential investments and hinder the advancement of groundbreaking treatments.

Key Developments:

• In September 2023: Biogen purchased Reata Pharmaceuticals, along with its small molecule Friedreich’s ataxia drug. SKYCLARYS, the primary product of Reata Pharmaceuticals, received approval in the United States in 2023 for the treatment of Friedreich’s ataxia (FA), a rare neuromuscular disorder.
• In November 2023: AstraZeneca formed a collaborative alliance and reached a financial agreement with Cellectis to accelerate the objectives in the fields of cell therapy and genomic medicine.

Company Products:

• Adakveo (crizanlizumab): offered by Novartis is a pharmaceutical drug that was formerly employed to avert vaso-occlusive crises (VOCs) in individuals aged 16 years and above who suffer from sickle cell disease (SCD).
• Elecsys total PSA: Offered by Roche the test tool is designed to quantitatively measure total prostate-specific antigen (tPSA) in human serum and plasma, including both free and complex forms. It is recommended for use alongside digital rectal examination (DRE) to assist in identifying prostate cancer in males aged 50 years and above. A prostate biopsy is necessary to confirm the diagnosis of prostate cancer.

Segmentation

• By Therapeutic Area
  • Oncology
  • Cardiovascular Disorders
  • Neurological Disorders
  • Infectious Diseases
  • Genetic Disorders
  • Autoimmune and Inflammation
  • Hematologic Disorder
  • Musculoskeletal Disorders
  • Others
• By Phase
  • Phase I
  • Phase II
  • Phase III
  • Phase IV
• By Sponsor
  • Pharmaceutical & Biopharmaceutical Companies
  • Non-profit Organizations
  • Others
• By Geography
  • North America
    • United States
    • Canada
    • Mexico
  • South America
    • Brazil
    • Argentina
    • Others
  • Europe
    • United Kingdom
    • Germany
    • France
    • Italy
    • Spain
    • Others
  • Middle East and Africa
    • Saudi Arabia
    • UAE
    • Others
  • Asia Pacific
    • Japan
    • China
    • India
    • South Korea
    • Taiwan
    • Thailand
    • Indonesia
    • Others