Overview


According to FutureWise analysis the market for Rare Disease Treatment in 2023 is US$ 152.77 billion, and is expected to reach US$ 406.71 billion by 2031 at a CAGR of 13.00%.

CRISPR gene-editing technology is being used to generate tailored medicines for rare diseases. CRISPR is integrated into the adeno-associated virus to distribute the medicine systemically (AAV). Low patient numbers, a lack of understanding of pathology and progression, and a lack of established objectives are all obstacles to conducting efficient and successful drug development trials for rare illnesses. As a result, various regulatory authorities are working to create regulatory guidelines for rare illness drug approval in order to ensure patient safety and efficacy. The FDA in the United States is concentrating on the development of gene therapy techniques for the treatment of uncommon diseases. The National Institutes of Health (NIH) and the Food and Drug Administration (FDA) oversee training and guidelines to improve the quality and commercialization of NIH-funded orphan medications for rare diseases. In addition, the National Institutes of Health (NIH) has started a number of larger programmes to help with medicine delivery for uncommon diseases. The value of the rare illness treatment market has risen as a result of increased focus on research and development of novel therapeutic medications. In addition, as product development continues, technologically driven new technologies for diagnosing uncommon genetic illnesses are becoming available. Furthermore, favourable government policies aimed at raising awareness about the treatment of rare diseases are leading to an increase in the use of effective medicines for uncommon disorders.

FutureWise Market Research has instantiated a report that provides an intricate analysis of Rare Disease Treatment Market trends that shall affect the overall market growth. Furthermore, it includes detailed information on the graph of profitability, SWOT analysis, market share and regional proliferation of this business. Moreover, the report offers insights on the current stature of prominent market players in the competitive landscape analysis of this market.

According to the research study conducted by FutureWise research analysts, the Rare Disease Treatment Market is anticipated to attain substantial growth by the end of the forecast period. The report explains that this business is predicted to register a noteworthy growth rate over the forecast period. This report provides crucial information pertaining to the total valuation that is presently held by this industry and it also lists the segmentation of the market along with the growth opportunities present across this business vertical.

  • Novartis AG
  • Pfizer Inc.
  • F. Hoffmann-La Roche Ltd.
  • Celgene Corporation
  • AbbVie Inc.
  • Johnson and Johnson Services, Inc.
  • Shire
  • Alexion
  • Novo Nordisk A/S
  • Sanofi
  • Bayer AG
  • Amgen, Inc.
  • Allergan plc.
  • Eisai Co. Ltd.
  • Bristol-Myers Squibb Company
  • MerckCo., Inc.
  • Vertex Pharmaceuticals
  • Baxter
  • BioMarin
  • Teva Pharmaceutical Industries Ltd.

(Note: The list of the major players will be updated with the latest market scenario and trends)

By Therapeutic Area

  • Hematology
    • Genetic Diseases
    • Neurological Diseases
  • Oncology
    • Infectious Diseases
    • Inflammatory Diseases
    • Metabolic Diseases
    • Pulmonary Diseases
    • Kidney Diseases
    • Musculoskeletal Diseases
    • Endocrine Diseases
    • Skin Disorders

By Drug Type

  • Originator Drugs (Patented Molecules)
  • Generic Drugs

By Drug Class

  • Biologics
  • Organic Compounds

By Mode of Administration

  • Injectable
  • Oral
  • Retail Pharmacies (includes topical preparations)

By Distribution Channels

  • Hospital Pharmacies
  • Specialty Pharmacies
  • Retail Pharmacies

By Region

  • North America
  • Europe
  • Asia-Pacific
  • Latin America
  • Middle East and Africa

  • Tier 1 players- established companies in the market with a major market share
  • Tier 2 players
  • Emerging players which are growing rapidly
  • New Entrants

  • Growth prospects
  • SWOT analysis
  • Key trends
  • Key data-points affecting market growth

  • To provide with an exhaustive analysis on the Rare Disease Treatment Market By Therapeutic Area, By Drug Type, By Drug Class, By Mode of Administration, By Distribution Channels and By Region.
  • To cater comprehensive information on factors impacting market growth (drivers, restraints, opportunities, and industry-specific restraints)
  • To evaluate and forecast micro-markets and the overall market
  • To predict the market size, in key regions— North America, Europe, Asia Pacific, Latin America and Middle East and Africa.
  • To record and evaluate competitive landscape mapping- product launches, technological advancements, mergers and expansions

  • We have a flexible delivery model and you can suggest changes in the scope/table of content as per your requirement
  • The customization Mobility Care offered are free of charge with purchase of any license of the report
  • You can directly share your requirements/changes to the current table of content to: sales@futurewiseresearch.com

Table of Contents


  • 1.   Market Introduction
    •   1. Objectives of the Study
        2. Market Definition
        3. Market Scope
         3.1. Years Considered for the Study
         3.2. Market Covered
        4. Currency
        5. Limitations
        6. Stakeholders
  • 2.   Research Methodology
    •   1. Research Data
         1.1. Secondary Data
          1.1.1. Key Data from Secondary Sources
         1.2. Primary Data
          1.2.1. Key Data from Primary Sources
        2. Market Size Estimation
        3. Market Breakdown and Data Triangulation
        4. Assumptions for the Study
  • 3.   Executive Summary
    •   1. Market Outlook
        2. Segment Outlook
        3. Competitive Insights
  • 4.   Rare Disease Treatment Market Variables, Trends and Scope
    •   1. Market Lineage Outlook
        2. Penetration and Growth Prospect Mapping
        3. Industry Value Chain Analysis
        4. Cost Analysis Breakdown
        5. Technology Overview
        6. Regulatory Framework
         6.1. Reimbursement Framework
         6.2. Standards and Compliances

  • 5.   Market Overview
    •   1. Market Dynamics
         1.1. Market Driver Analysis
          1.1.1. Increasing focus of Rare Disease Treatment Market Companies on Brand Protection
          1.1.2. Untapped Opportunities in Emerging Regions
         1.2. Market Restraint Analysis
          1.2.1. High Cost Associated with Implementation of Predictive Analysis
         1.3. Industry Challenges
          1.3.1. Presence of Ambiguous Regulatory Framework
  • 6.   Rare Disease Treatment Market Analysis Tools
    •   1. Industry Analysis - Porter’s
         1.1. Supplier Power
         1.2. Buyer Power
         1.3. Substitution Threat
         1.4. Threat from New Entrants
         1.5. Competitive Rivalry
        2. Pestel Analysis
         2.1. Political Landscape
         2.2. Environmental Landscape
         2.3. Social Landscape
         2.4. Technology Landscape
         2.5. Legal Landscape
        3. Major Deals And Strategic Alliances Analysis
         3.1. Joint Ventures
         3.2. Mergers and Acquisitions
         3.3. Licensing and Partnership
         3.4. Technology Collaborations
         3.5. Strategic Divestments
        4. Market Entry Strategies
        5. Case Studies
  • 7.   Rare Disease Treatment Market, By Therapeutic Area Historical Analysis and Forecast 2023-2031 (USD Million)
    •   1. Hematology
         1.1. Genetic Diseases
         1.2. Neurological Diseases
        2. Oncology
         2.1. Infectious Diseases
         2.2. Inflammatory Diseases
         2.3. Metabolic Diseases
         2.4. Pulmonary Diseases
         2.5. Kidney Diseases
         2.6. Musculoskeletal Diseases
         2.7. Endocrine Diseases
         2.8. Skin Disorders

  • 8.   Rare Disease Treatment Market, By Drug Type Historical Analysis and Forecast 2023-2031 (USD Million)
    •   1. Originator Drugs (Patented Molecules)
        2. Generic Drugs

  • 9.   Rare Disease Treatment Market, By Drug Class Historical Analysis and Forecast 2023-2031 (USD Million)
    •   1. Biologics
        2. Organic Compounds

  • 10.   Rare Disease Treatment Market, By Mode of Administrations Historical Analysis and Forecast 2023-2031 (USD Million)
    •   1. Injectable
        2. Oral
        3. Retail Pharmacies (includes topical preparations)

  • 11.   Rare Disease Treatment Market, By Distribution Channels Historical Analysis and Forecast 2023-2031 (USD Million)
    •   1. Hospital Pharmacies
        2. Specialty Pharmacies
        3. Retail Pharmacies

  • 12.   North America Rare Disease Treatment Market Analysis 2017-2022 and Forecast 2023-2031 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2017-2022
         2.1. U.S.A
         2.2. Canada
         2.3. Mexico
        3. Market Size (USD Million) Forecast for North America 2023-2031

  • 13.   Latin America Rare Disease Treatment Market Analysis 2017-2022 and Forecast 2023-2031 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2017-2022
         2.1. Brazil
         2.2. Venezuela
         2.3. Argentina
         2.4. Rest of Latin America
        3. Market Size (USD Million) Forecast for Latin America 2023-2031

  • 14.   Europe Rare Disease Treatment Market Analysis 2017-2022 and Forecast 2023-2031 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2017-2022
         2.1. Germany
         2.2. U.K
         2.3. France
         2.4. Italy
         2.5. Spain
         2.6. Russia
         2.7. Poland
         2.8. Rest of Europe
        3. Market Size (USD Million) Forecast for Europe 2023-2031

  • 15.   Asia Pacific Rare Disease Treatment Market Analysis 2017-2022 and Forecast 2023-2031 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2017-2022
         2.1. Japan
         2.2. China
         2.3. India
         2.4. Australia and New Zealand
         2.5. ASEAN
         2.6. Rest of Asia Pacific
        3. Market Size (USD Million) Forecast for Asia Pacific 2023-2031

  • 16.   Middle East and Africa Rare Disease Treatment Market Analysis 2017-2022 and Forecast 2023-2031 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2017-2022
         2.1. GCC
         2.2. Israel
         2.3. South Africa
         2.4. Rest of MEA
        3. Market Size (USD Million) Forecast for MEA 22023-2031

  • 17.   Market Share Analysis and Competitive Landscape
    •   1. Global Landscape - Key Players, Revenue and Presence
        2. Global Share Analysis - Key Players (Tier 1, Tier 2, Tier 3)
        3. Global Emerging Companies
        4. North America - Market Share Analysis and Key Regional Players
        5. Europe - Market Share Analysis and Key Regional Players
        6. Asia Pacific - Market Share Analysis and Key Regional Players
        7. Global Key Player - Growth Matrix
  • 18.   Company Profiles (Competition Dashboard, Competitors Deep Dive, Products Offered and Financial Layouts)
    •       1. Novartis AG
               1.1. Company Overview
               1.2. Product Portfolio
               1.3. SWOT Analysis
               1.4. Financial Overview
               1.5. Strategic Overview
           2. Pfizer Inc.
               2.1. Company Overview
               2.2. Product Portfolio
               2.3. SWOT Analysis
               2.4. Financial Overview
               2.5. Strategic Overview
            3. F. Hoffmann-La Roche Ltd.
               3.1. Company Overview
               3.2. Product Portfolio
               3.3. SWOT Analysis
               3.4. Financial Overview
               3.5. Strategic Overview
           4. Celgene Corporation
               4.1. Company Overview
               4.2. Product Portfolio
               4.3. SWOT Analysis
               4.4. Financial Overview
               4.5. Strategic Overview
            5. AbbVie Inc.
               5.1. Company Overview
               5.2. Product Portfolio
               5.3. SWOT Analysis
               5.4. Financial Overview
               5.5. Strategic Overview          
            6. Johnson & Johnson Services, Inc.
               6.1. Company Overview
               6.2. Product Portfolio
               6.3. SWOT Analysis
               6.4. Financial Overview
               6.5. Strategic Overview
            7. Shire
               7.1. Company Overview
               7.2. Product Portfolio
               7.3. SWOT Analysis
               7.4. Financial Overview
               7.5. Strategic Overview
            8. Alexion
               8.1. Company Overview
               8.2. Product Portfolio
               8.3. SWOT Analysis
               8.4. Financial Overview
               8.5. Strategic Overview
            9. Novo Nordisk A/S
               9.1. Company Overview
               9.2. Product Portfolio
               9.3. SWOT Analysis
               9.4. Financial Overview
               9.5. Strategic Overview          
           10. Sanofi
               10.1. Company Overview
               10.2. Product Portfolio
               10.3. SWOT Analysis
               10.4. Financial Overview
               10.5. Strategic Overview
           11. Bayer AG
               11.1. Company Overview
               11.2. Product Portfolio
               11.3. SWOT Analysis
               11.4. Financial Overview
               11.5. Strategic Overview          
           12. Amgen, Inc.
               12.1. Company Overview
               12.2. Product Portfolio
               12.3. SWOT Analysis
               12.4. Financial Overview
               12.5. Strategic Overview
           13. Allergan plc.
               13.1. Company Overview
               13.2. Product Portfolio
               13.3. SWOT Analysis
               13.4. Financial Overview
               13.5. Strategic Overview
           14. Eisai Co. Ltd.
               14.1. Company Overview
               14.2. Product Portfolio
               14.3. SWOT Analysis
               14.4. Financial Overview
               14.5. Strategic Overview
           15. Bristol-Myers Squibb Company
               15.1. Company Overview
               15.2. Product Portfolio
               15.3. SWOT Analysis
               15.4. Financial Overview
               15.5. Strategic Overview
           16. Merck & Co., Inc.
               16.1. Company Overview
               16.2. Product Portfolio
               16.3. SWOT Analysis
               16.4. Financial Overview
               16.5. Strategic Overview
           17. Vertex Pharmaceuticals
               17.1. Company Overview
               17.2. Product Portfolio
               17.3. SWOT Analysis
               17.4. Financial Overview
               17.5. Strategic Overview
           18. Baxter
                18.1. Company Overview
                18.2. Product Portfolio
                18.3. SWOT Analysis
                18.4. Financial Overview
                18.5. Strategic Overview
           19. BioMarin
               19.1. Company Overview
               19.2. Product Portfolio
               19.3. SWOT Analysis
               19.4. Financial Overview
               19.5. Strategic Overview
           20. Teva Pharmaceutical Industries Ltd.
               20.1. Company Overview
               20.2. Product Portfolio
               20.3. SWOT Analysis
               20.4. Financial Overview
               20.5. Strategic Overview

  • 19.   Pre and Post COVID-19 Impact
    • 1. Positive influence on the healthcare industry
      2. The financial disruption of the manufacturing sector
      3. Impact of COVID-19 on emerging companies
      4. Significant mandates in the healthcare regulations initiated by administrations
      5. The overall economic slowdown of the developing and developed nations

  • 20.   FutureWise SME Key Takeaway Points for Client
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