Rare Disease Treatment Market - Growth, Trends, Covid-19 Impact, and Forecasts (2022 – 2027)

The Rare Disease Treatment Market is segmented by Drug Type (Biologics and Non-biologics), Therapeutic Area (Genetic Diseases, Neurological Diseases, Oncology, Infectious Diseases, Cardiovascular Diseases and Others), Mode of Administration (Oral, Injection and Others) and Geography (North America, Europe, Asia-Pacific, Middle East and Africa and South America). The report offers the value (in USD million) for the above segments.

Market Snapshot

Rare Disease Treatment Market Overview
Study Period: 2019-2027
Base Year: 2021
Fastest Growing Market: Asia-Pacific
Largest Market: North America
CAGR: 8.6 %

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Market Overview

The Rare Disease Treatment Market is projected to register a CAGR of 8.6% during the forecast period (2022 to 2027).

The healthcare system has witnessed enormous challenges due to the COVID-19 pandemic. All outpatient treatments were postponed or restricted during the COVID-19 pandemic to ​reduce the risk of viral transmission, as most chronic therapies were regarded as non-urgent. A review article titled “Research and Management of Rare Diseases in the COVID-19 Pandemic Era: Challenges and Countermeasures,” published in the journal of Frontiers in Public Health in April 2021, studied the challenges faced by patients affected by rare diseases. The article concluded that regular clinical services, counseling, and therapies were extremely inadequate. Also, the clinicians, researchers, and scientists working on rare genetic diseases face extra challenges due to the pandemic since research projects and clinical trials for rare and genetic diseases were stalled during the COVID-19 pandemic.

The definition of rare diseases has distinct in different parts of the world. The United States Food and Drug Administration defines a rare disease as a disease or condition that affects less than 200,000 people in the United States. The European Commission Public Health states that a rare disease affects no more than one person in 2000 in the European Union. According to Health Canada, the rare disease affects less than five persons in 10,000 in Canada.

An article in the journal of Diseases in November 2020 titled “Precision Medicine in Rare Diseases” discussed using precision or personalized medicine to treat rare diseases. The study indicated that more than 90% of rare diseases do not have an effective treatment method. This inadequacy in the therapies for rare diseases necessitates novel treatment methods, which is expected to propel the growth of the rare disease treatment market in the forecast period.

Various governments around the world have been formulating several initiatives for the treatment of rare diseases. For instance, the Indian government Ministry of Health and Family Welfare’s National Policy for Rare Diseases, 2021 aimed at lowering the incidence and prevalence of rare diseases based on an integrated and comprehensive preventive strategy encompassing awareness generation, premarital, post-marital, pre-conception, and post-conception screening and counseling programs to prevent births of children with rare diseases, and within the constraints on resources and competing health care priorities, enable access to affordable health care to patients of rare diseases which are amenable to one-time treatment or relatively low-cost therapy.

The increase in the necessity of rare disease treatment worldwide is expected to propel the growth of the rare disease treatment market.​ However, a lack of awareness regarding rare disease treatment and the high cost of treatment is expected to hinder the market growth during the study period.

Scope of the Report

As per the scope of this report, rare diseases are diseases that affect only a small population compared to the general population. Rare diseases are region specific in that a rare disease could be rare in one region while it is common in another. Rare diseases may be chronic or incurable, though many short-term medical conditions are also rare diseases. The Rare Disease Treatment Market is segmented by Drug Type (Biologics and Non-biologics), Therapeutic Area (Genetic Diseases, Neurological Diseases, Oncology, Infectious Diseases, Cardiovascular Diseases, and Others), Mode of Administration (Oral, Injection, and Others), and Geography (North America, Europe, Asia-Pacific, Middle-East and Africa, and South America). The Market report also covers the estimated market sizes and trends of 17 countries across major regions globally. The report offers values (in USD million) for the above segments.

By Drug Type
Therapeutic Area
Genetic Diseases
Neurological Diseases
Infectious Diseases
Cardiovascular Diseases
Mode of Administration
North America
United States
United Kingdom
Rest of Europe
South Korea
Rest of Asia-Pacific
Middle-East and Africa
South Africa
Rest of Middle-East and Africa
South America
Rest of South America

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Key Market Trends

The Biologics Segment is Expected to Hold a Major Market Share in the Rare disease treatment Market.

Biologics occupy a major segment in the rare disease treatment market. Over the years, various market players have invested in researching and developing new biologics for the treatment of rare diseases. For example, CHIESI Farmaceutici S.p.A. in February 2020, Chiesi launched Global Rare Diseases, a new business unit segment focused on the research, development, and commercialization of treatments and patient support services for rare and ultra-rare disorders. In May 2020, Chiesi Global Rare Diseases, together with Protalix BioTherapeutics, announced the submission of a biologics license application to the US Food and Drug Administration for Pegunigalsidase Alfa for the treatment of Fabry disease. Upon approval of the biologics license application, Protalix BioTherapeutics will be eligible for a breakthrough payment from Chiesi to assist in the next phase III study of the drug. Increasing investment in treating rare diseases such as Fabry disease is expected to fuel the growth of the market under study.​

An article titled “Biological Treatments in Inflammatory Bowel Disease: A Complex Mix of Mechanisms and Actions,” published in the MDPI journal of Biologics in August 2021, studied the main biological treatments currently available for inflammatory bowel disease treatment due to the extremely important current role of biologics. The study showed that various biologic therapies such as anti-TNF-α therapy, anti-integrin therapy, anti-cytokine therapy, and other new biologics are widely used to treat rare inflammatory bowel disease. The study indicated that further studies are needed to better understand the action mechanism of the biological drugs, which will help understand how to improve the efficacy and safety of the treatment methods.​

Another article presented in the Orphanet Journal of Rare Diseases titled “Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group,” published in July 2021, studied the list of biologics approved by the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA) and China’s National Medical Products Administration (NMPA), which are useful against the rare diseases. The goal of the list was to further accelerate interactions among patient organizations, health care providers, industry, and government agencies to improve standards of care for rare diseases by promoting access to treatments.

The increasing research on the study of biologics for rare disease treatment is expected to boost the growth of the studied market over the forecast period.

Projected Number of Type 1 Diabetes Cases, Global, 2010-2050

North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period (2022 – 2027).

The rise in the adoption of highly advanced techniques and systems in manufacturing rare disease treatments and the technological advancements made in immunohistochemistry studies is expected to boost the market's growth in the North American region. The high awareness among the population on the availability of innovative rare disease treatment models in the market contributes to the high market growth in the region.

The increasing number of rare disease cases in the North American region contributes to the growth of the rare disease treatment market. The large population affected by rare diseases and the increasing need for rare disease treatments for easy diagnosis and treatment of these diseases are expected to fuel the growth of the rare disease treatment market. The US Food and Drug Administration's Rare Disease Team aims to facilitate, support, and accelerate the development of drugs and biologics to benefit patients with rare disorders. On Rare Disease Day 2020, the Food and Drug Administration reported that about 7,000 rare diseases affect an estimated 30 million people in the United States. A large number of rare diseases in the region is likely to fuel the growth of the rare disease treatment market over the forecast period.

Rising healthcare expenditure in the United States is also expected to drive demand for the rare disease treatment market in the region. According to the 2020 National Health Expenditure data from the U.S. Centers for Health Care and Medicaid Services, health care spending increased by as much as 9.7% to $4.1 trillion in 2020 and accounted for approximately 19.7% of gross domestic product. The expenditure is projected to reach USD 6.2 trillion by 2028. The rise in healthcare expenditure in the United States region is likely to fuel the growth of the rare disease treatment market during the forecast period of the study.

Thus the increasing necessity for rare disease treatment coupled with increasing investment in the health care department is known to propel the market growth in this region.

Rare Disease Treatment Market Forecast

Competitive Landscape

The Rare disease treatment Market is competitive and consists of several major players. In terms of market share, a few major players dominate the market. Some companies currently dominating the market are Amgen, Inc., Alexion Pharmaceuticals, Inc., Bristol-Myers Squibb Company, Biomarin Pharmaceuticals, Bayer AG, and others. Market players readily adopt multiple initiatives such as mergers and acquisitions and product launches to strengthen their position in the market.

Recent Development

  • In May 2022, the Center for Drug Evaluation and Research (CDER) of the U.S. Food and Drug Administration (FDA) launched a new program for expediting the development of new treatment options for rare diseases, the Accelerating Rare disease Cures (ARC) Program.
  • In April 2022, National Organisation for Rare Diseases Inc. announced a grant funding of USD 100,000 for the research in rare diseases. The National Organization for Rare Disorders (NORD) announced three new requests for proposal for grant funding related to rare diseases, Autoimmune Polyglandular Syndrome Type 1 (APS-1), Levy-Yeboa Syndrome (LYS), and Megacystis Microcolon Intestinal Hypoperistalsis Syndrome (MMIHS).

Table of Contents


    1. 1.1 Study Assumptions and market definition

    2. 1.2 Scope of the Study




    1. 4.1 Market Overview

    2. 4.2 Market Drivers

      1. 4.2.1 Increase in Number of Rare Disease Cases

      2. 4.2.2 Rising R&D Activities for Novel Therapeutics and Drugs and Increase in Number of New Drug Launches and Favourable Government Policies

    3. 4.3 Market Restraints

      1. 4.3.1 Lack of Awareness Regarding Rare Disease Treatment

      2. 4.3.2 High Cost of Treatment

    4. 4.4 Porter's Five Forces Analysis

      1. 4.4.1 Threat of New Entrants

      2. 4.4.2 Bargaining Power of Buyers/Consumers

      3. 4.4.3 Bargaining Power of Suppliers

      4. 4.4.4 Threat of Substitute Products

      5. 4.4.5 Intensity of Competitive Rivalry

  5. 5. MARKET SEGMENTATION (Market Size by Value - in USD Million)

    1. 5.1 By Drug Type

      1. 5.1.1 Biologics

      2. 5.1.2 Non-biologics

    2. 5.2 Therapeutic Area

      1. 5.2.1 Genetic Diseases

      2. 5.2.2 Neurological Diseases

      3. 5.2.3 Oncology

      4. 5.2.4 Infectious Diseases

      5. 5.2.5 Cardiovascular Diseases

      6. 5.2.6 Others

    3. 5.3 Mode of Administration

      1. 5.3.1 Oral

      2. 5.3.2 Injection

      3. 5.3.3 Others

    4. 5.4 Geography

      1. 5.4.1 North America

        1. United States

        2. Canada

        3. Mexico

      2. 5.4.2 Europe

        1. Germany

        2. United Kingdom

        3. France

        4. Italy

        5. Spain

        6. Rest of Europe

      3. 5.4.3 Asia-Pacific

        1. China

        2. Japan

        3. India

        4. Australia

        5. South Korea

        6. Rest of Asia-Pacific

      4. 5.4.4 Middle-East and Africa

        1. GCC

        2. South Africa

        3. Rest of Middle-East and Africa

      5. 5.4.5 South America

        1. Brazil

        2. Argentina

        3. Rest of South America


    1. 6.1 Company Profiles

      1. 6.1.1 AbbVie Inc.

      2. 6.1.2 Alexion Pharmaceuticals, Inc.

      3. 6.1.3 Amgen, Inc.

      4. 6.1.4 Baxter

      5. 6.1.5 Bayer AG

      6. 6.1.6 Biomarin Pharmaceuticals

      7. 6.1.7 Bristol-Myers Squibb Company

      8. 6.1.8 Eisai Co. Ltd.

      9. 6.1.9 Eli Lilly and Company

      10. 6.1.10 F. Hoffmann-La Roche Ltd.

      11. 6.1.11 Novartis AG

      12. 6.1.12 Pfizer Inc

      13. 6.1.13 Sanofi

      14. 6.1.14 Teva Pharmaceuticals

      15. 6.1.15 Vertex Pharmaceuticals

    2. *List Not Exhaustive

**Competitive Landscape covers- Business Overview, Financials, Products and Strategies and Recent Developments

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Frequently Asked Questions

The Global Rare Disease Treatment Market market is studied from 2019 - 2027.

The Global Rare Disease Treatment Market is growing at a CAGR of 8.6% over the next 5 years.

Asia-Pacific is growing at the highest CAGR over 2021- 2026.

North America holds highest share in 2021.

Alexion Pharmaceuticals , Amgen Inc. , Biomarin Pharmaceuticals , Bayer, Bristol Myer Squibb are the major companies operating in Global Rare Disease Treatment Market.

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