Rare Diseases Treatment Market - Global Industry Analysis (2019 - 2021), Growth Trends, and Market Forecast (2022 - 2029)

Rare Diseases Treatment Market - Global Industry Analysis (2019 - 2021), Growth Trends, and Market Forecast (2022 - 2029)

The global rare diseases treatment market is headed toward sustained growth during the projection period of 2022 to 2029. Orphan drug development has emerged as the foremost growth enabler across the market, setting the stage for innovation-driven expansion. World over, the incidence of rare diseases and genetic disorders has been increasing, propelling healthcare decision-makers to invest in vigorous drug discovery endeavors. This factor, coupled with government support for medical innovation, is set to generate lucrative outcomes in the market.

According to a recent report by Fairfield Market Research, the rare diseases treatment market is poised to attain a valuation of US$346.5 Bn by the end of 2029. The comprehensive study delves into the emerging trends influencing the demand for rare disease treatment methods, predicting that the market will register a CAGR of 10.4% during the forecast period.

Evolving trends in drug development such as advanced biologics, targeted therapies, and innovative clinical trials are set to create a lucrative environment for novel drug discovery initiatives. Moreover, the creation of promising public health policies, progressive regulatory approaches, and campaigns directed at enhancing public awareness are some of the secondary factors that are expected to expand the market.

Key Trends Across Global Rare Diseases Treatment Market

In 2019, the oncology segment dominated the rare diseases treatment market. By 2029, this segment will continue to be the dominating segment, with a contribution of ~48%

Based on patient type, the pediatrics segment is set to register a robust growth rate of 10.7% during the study period

North America will reach a market valuation of US$177.8 Bn by 2029, cementing its position as the most significant market

Investments in Oncology & Biologics Create Optimistic Outlook for Market Expansion

Based on therapeutic area, the rare diseases treatment market is segmented into cardiovascular, neurology, oncology, hematology, and respiratory. Among these, the oncology segment is expected to continue its dominance in the market due to the rigorous pursuit of advanced cancer treatments. Notably, the rare neurology drugs pipeline is particularly limited for a wide range of related disorders, offering limited competition opportunities to potential investors. On the other hand, as per treatment type, biologics are predicted to command a lion’s share across the market. This is owing to the intense experimentation and exploration of gene therapies for patients with rare diseases. All through the study period, it is anticipated that the development of supportive regulations and standardized policies will positively impact M&A and revenue generation in the market.

North America Maintains Global Dominance, Asia Pacific a Rising Regional Player

During the forecast period, North America is slated to sustain its dominance in the global rare diseases treatment market. This can be attributed to this region’s sophisticated healthcare infrastructure, promising government funding for medical innovation and drug discovery, and the presence of prominent industry leaders in the US, and Canada. Meanwhile, the Asia Pacific is rising as a formidable regional contender due to progress in orphan drug development and the higher prevalence of rare diseases in this region.

Key Market Players

Major players in the rare diseases treatment market are AstraZeneca, Novartis, Vertex Pharmaceuticals, Johnson & Johnson, Sanofi, Bristol Myers Squibb, Pfizer, Roche, Abbvie, and Takeda.

Please note: The PDF e-mail from publisher version of this report is for a global site license.


1. Executive Summary
1.1. Global Rare Diseases Treatment Market Outlook, 2019 - 2029 (US$ Billion)
1.2. Global Rare Diseases Treatment Market Incremental Opportunity, 2022 - 2029 (US$ Billion)
1.3. Key Industry Trends
2. Market Overview
2.1. Market Definitions and Segmentations
2.2. Market Dynamics
2.2.1. Market Drivers
2.2.2. Market Threats
2.2.3. Market Opportunities
2.3. Regulatory Framework
2.4. Covid 19 Impact Analysis
3. Global Rare Diseases Treatment Market Outlook, 2019 - 2029
3.1. Global Rare Diseases Treatment Market Outlook, By Therapeutic Area, 2019 - 2029
3.1.1. Key Highlights
3.1.2. Global Rare Diseases Treatment Market Outlook, by Therapeutic Area, Value (US$ Billion), 2019 - 2029
3.1.2.1. Oncology
3.1.2.2. Hematology
3.1.2.3. Neurology
3.1.2.4. Respiratory
3.1.2.5. Cardiovascular
3.1.2.6. Other
3.1.3. Global Rare Diseases Treatment Market Share and BPS Analysis, by Therapeutic Area, Value, 2022 and 2029
3.1.4. Global Rare Diseases Treatment Market Attractiveness Analysis, by Therapeutic Area, Value, 2019 - 2029
3.2. Global Rare Diseases Treatment Market Outlook, By Type, 2019 - 2029
3.2.1. Key Highlights
3.2.2. Global Rare Diseases Treatment Market Outlook, by Type, Value (US$ Billion), 2019 - 2029
3.2.2.1. Biologics
3.2.2.2. Non-Biologics
3.2.3. Global Rare Diseases Treatment Market Share and BPS Analysis, by Type, Value, 2022 and 2029
3.2.4. Global Rare Diseases Treatment Market Attractiveness Analysis, by Type, Value, 2019 - 2029
3.3. Global Rare Diseases Treatment Market Outlook, By Patient Type, 2019 - 2029
3.3.1. Key Highlights
3.3.2. Global Rare Diseases Treatment Market Outlook, By Patient Type, Value (US$ Billion), 2019 - 2029
3.3.2.1. Adults
3.3.2.2. Paediatrics
3.3.2.3. Geriatrics
3.3.3. Global Rare Diseases Treatment Market Share and BPS Analysis, By Patient Type, Value, 2022 and 2029
3.3.4. Global Rare Diseases Treatment Market Attractiveness Analysis, By Patient Type, Value, 2019 - 2029
3.4. Global Rare Diseases Treatment Market Outlook, By Region, 2019 - 2029
3.4.1. Key Highlights
3.4.2. Global Rare Diseases Treatment Market Outlook, by Region, Value (US$ Billion), 2019 - 2029
3.4.2.1. North America
3.4.2.2. Europe
3.4.2.3. Asia Pacific
3.4.2.4. Rest of the World
3.4.3. Global Rare Diseases Treatment Market Share and BPS Analysis, by Region, Value, 2022 and 2029
3.4.4. Global Rare Diseases Treatment Market Attractiveness Analysis, by Region, Value, 2019 - 2029
4. North America Rare Diseases Treatment Market Outlook, 2019 - 2029
4.1. North America Rare Diseases Treatment Market Outlook, By Therapeutic Area, 2019 - 2029
4.1.1. Key Highlights
4.1.2. North America Rare Diseases Treatment Market Outlook, by Therapeutic Area, Value (US$ Billion), 2019 - 2029
4.1.2.1. Oncology
4.1.2.2. Hematology
4.1.2.3. Neurology
4.1.2.4. Respiratory
4.1.2.5. Cardiovascular
4.1.2.6. Other
4.1.3. North America Rare Diseases Treatment Market Share and BPS Analysis, by Therapeutic Area, Value, 2022 and 2029
4.1.4. North America Rare Diseases Treatment Market Attractiveness Analysis, by Therapeutic Area, Value, 2019 - 2029
4.2. North America Rare Diseases Treatment Market Outlook, By Type, 2019 - 2029
4.2.1. Key Highlights
4.2.2. North America Rare Diseases Treatment Market Outlook, by Type, Value (US$ Billion), 2019 - 2029
4.2.2.1. Biologics
4.2.2.2. Non-Biologics
4.2.3. North America Rare Diseases Treatment Market Share and BPS Analysis, by Type, Value, 2022 and 2029
4.2.4. North America Rare Diseases Treatment Market Attractiveness Analysis, by Type, Value, 2019 - 2029
4.3. North America Rare Diseases Treatment Market Outlook, By Patient Type, 2019 - 2029
4.3.1. Key Highlights
4.3.2. North America Rare Diseases Treatment Market Outlook, By Patient Type, Value (US$ Billion), 2019 - 2029
4.3.2.1. Adults
4.3.2.2. Paediatrics
4.3.2.3. Geriatrics
4.3.3. North America Rare Diseases Treatment Market Share and BPS Analysis, By Patient Type, Value, 2022 and 2029
4.3.4. North America Rare Diseases Treatment Market Attractiveness Analysis, By Patient Type, Value, 2019 - 2029
4.4. North America Rare Diseases Treatment Market Outlook, By Country, 2019 - 2029
4.4.1. Key Highlights
4.4.2. North America Rare Diseases Treatment Market Outlook, by Country, Value (US$ Billion), 2019 - 2029
4.4.2.1. United States
4.4.2.2. Canada
4.4.3. North America Rare Diseases Treatment Market Share and BPS Analysis, by Country, Value, 2022 and 2029
4.4.4. North America Rare Diseases Treatment Market Attractiveness Analysis, by Country, Value, 2019 - 2029
5. Europe Rare Diseases Treatment Market Outlook, 2019 - 2029
5.1. Europe Rare Diseases Treatment Market Outlook, By Therapeutic Area, 2019 - 2029
5.1.1. Key Highlights
5.1.2. Europe Rare Diseases Treatment Market Outlook, by Therapeutic Area, Value (US$ Billion), 2019 - 2029
5.1.2.1. Oncology
5.1.2.2. Hematology
5.1.2.3. Neurology
5.1.2.4. Respiratory
5.1.2.5. Cardiovascular
5.1.2.6. Other
5.1.3. Europe Rare Diseases Treatment Market Share and BPS Analysis, by Therapeutic Area, Value, 2022 and 2029
5.1.4. Europe Rare Diseases Treatment Market Attractiveness Analysis, by Therapeutic Area, Value, 2019 - 2029
5.2. Europe Rare Diseases Treatment Market Outlook, By Type, 2019 - 2029
5.2.1. Key Highlights
5.2.2. Europe Rare Diseases Treatment Market Outlook, by Type, Value (US$ Billion), 2019 - 2029
5.2.2.1. Biologics
5.2.2.2. Non-Biologics
5.2.3. Europe Rare Diseases Treatment Market Share and BPS Analysis, by Type, Value, 2022 and 2029
5.2.4. Europe Rare Diseases Treatment Market Attractiveness Analysis, by Type, Value, 2019 - 2029
5.3. Europe Rare Diseases Treatment Market Outlook, By Patient Type, 2019 - 2029
5.3.1. Key Highlights
5.3.2. Europe Rare Diseases Treatment Market Outlook, By Patient Type, Value (US$ Billion), 2019 - 2029
5.3.2.1. Adults
5.3.2.2. Paediatrics
5.3.2.3. Geriatrics
5.3.3. Europe Rare Diseases Treatment Market Share and BPS Analysis, By Patient Type, Value, 2022 and 2029
5.3.4. Europe Rare Diseases Treatment Market Attractiveness Analysis, By Patient Type, Value, 2019 - 2029
5.4. Europe Rare Diseases Treatment Market Outlook, By Country, 2019 - 2029
5.4.1. Key Highlights
5.4.2. Europe Rare Diseases Treatment Market Outlook, by Country, Value (US$ Billion), 2019 - 2029
5.4.2.1. Germany
5.4.2.2. France
5.4.2.3. United Kingdom
5.4.2.4. Italy
5.4.2.5. Spain
5.4.2.6. Rest of Europe
5.4.3. Europe Rare Diseases Treatment Market Share and BPS Analysis, by Country, Value, 2022 and 2029
5.4.4. Europe Rare Diseases Treatment Market Attractiveness Analysis, by Country, Value, 2019 - 2029
6. Asia Pacific Rare Diseases Treatment Market Outlook, 2019 - 2029
6.1. Asia Pacific Rare Diseases Treatment Market Outlook, By Therapeutic Area, 2019 - 2029
6.1.1. Key Highlights
6.1.2. Asia Pacific Rare Diseases Treatment Market Outlook, by Therapeutic Area, Value (US$ Billion), 2019 - 2029
6.1.2.1. Oncology
6.1.2.2. Hematology
6.1.2.3. Neurology
6.1.2.4. Respiratory
6.1.2.5. Cardiovascular
6.1.2.6. Other
6.1.3. Asia Pacific Rare Diseases Treatment Market Share and BPS Analysis, by Therapeutic Area, Value, 2022 and 2029
6.1.4. Asia Pacific Rare Diseases Treatment Market Attractiveness Analysis, by Therapeutic Area, Value, 2019 - 2029
6.2. Asia Pacific Rare Diseases Treatment Market Outlook, By Type, 2019 - 2029
6.2.1. Key Highlights
6.2.2. Asia Pacific Rare Diseases Treatment Market Outlook, by Type, Value (US$ Billion), 2019 - 2029
6.2.2.1. Biologics
6.2.2.2. Non-Biologics
6.2.3. Asia Pacific Rare Diseases Treatment Market Share and BPS Analysis, by Type, Value, 2022 and 2029
6.2.4. Asia Pacific Rare Diseases Treatment Market Attractiveness Analysis, by Type, Value, 2019 - 2029
6.3. Asia Pacific Rare Diseases Treatment Market Outlook, By Patient Type, 2019 - 2029
6.3.1. Key Highlights
6.3.2. Asia Pacific Rare Diseases Treatment Market Outlook, By Patient Type, Value (US$ Billion), 2019 - 2029
6.3.2.1. Adults
6.3.2.2. Paediatrics
6.3.2.3. Geriatrics
6.3.3. Asia Pacific Rare Diseases Treatment Market Share and BPS Analysis, By Patient Type, Value, 2022 and 2029
6.3.4. Asia Pacific Rare Diseases Treatment Market Attractiveness Analysis, By Patient Type, Value, 2019 - 2029
6.4. Asia Pacific Rare Diseases Treatment Market Outlook, By Country, 2019 - 2029
6.4.1. Key Highlights
6.4.2. Asia Pacific Rare Diseases Treatment Market Outlook, by Country, Value (US$ Billion), 2019 - 2029
6.4.2.1. Japan
6.4.2.2. China
6.4.2.3. India
6.4.2.4. Australia & New Zealand
6.4.2.5. Rest of Asia Pacific
6.4.3. Asia Pacific Rare Diseases Treatment Market Share and BPS Analysis, by Country, Value, 2022 and 2029
6.4.4. Asia Pacific Rare Diseases Treatment Market Attractiveness Analysis, by Country, Value, 2019 - 2029
7. Rest of the World Rare Diseases Treatment Market Outlook, 2019 - 2029
7.1. Rest of the World Rare Diseases Treatment Market Outlook, By Therapeutic Area, 2019 - 2029
7.1.1. Key Highlights
7.1.2. Rest of the World Rare Diseases Treatment Market Outlook, by Therapeutic Area, Value (US$ Billion), 2019 - 2029
7.1.2.1. Oncology
7.1.2.2. Hematology
7.1.2.3. Neurology
7.1.2.4. Respiratory
7.1.2.5. Cardiovascular
7.1.2.6. Other
7.1.3. Rest of the World Rare Diseases Treatment Market Share and BPS Analysis, by Therapeutic Area, Value, 2022 and 2029
7.1.4. Rest of the World Rare Diseases Treatment Market Attractiveness Analysis, by Therapeutic Area, Value, 2019 - 2029
7.2. Rest of the World Rare Diseases Treatment Market Outlook, By Type, 2019 - 2029
7.2.1. Key Highlights
7.2.2. Rest of the World Rare Diseases Treatment Market Outlook, by Type, Value (US$ Billion), 2019 - 2029
7.2.2.1. Biologics
7.2.2.2. Non-Biologics
7.2.3. Rest of the World Rare Diseases Treatment Market Share and BPS Analysis, by Type, Value, 2022 and 2029
7.2.4. Rest of the World Rare Diseases Treatment Market Attractiveness Analysis, by Type, Value, 2019 - 2029
7.3. Rest of the World Rare Diseases Treatment Market Outlook, By Patient Type, 2019 - 2029
7.3.1. Key Highlights
7.3.2. Rest of the World Rare Diseases Treatment Market Outlook, By Patient Type, Value (US$ Billion), 2019 - 2029
7.3.2.1. Adults
7.3.2.2. Paediatrics
7.3.2.3. Geriatrics
7.3.3. Rest of the World Rare Diseases Treatment Market Share and BPS Analysis, By Patient Type, Value, 2022 and 2029
7.3.4. Rest of the World Rare Diseases Treatment Market Attractiveness Analysis, By Patient Type, Value, 2019 - 2029
7.4. Rest of the World Rare Diseases Treatment Market Outlook, By Sub-Region, 2019 - 2029
7.4.1. Key Highlights
7.4.2. Rest of the World Rare Diseases Treatment Market Outlook, by Sub-Region, Value (US$ Billion), 2019 - 2029
7.4.2.1. Latin America
7.4.2.2. Middle East & Arica
7.4.3. Rest of the World Rare Diseases Treatment Market Share and BPS Analysis, by Sub-Region, Value, 2022 and 2029
7.4.4. Rest of the World Rare Diseases Treatment Market Attractiveness Analysis, by Sub-Region, Value, 2019 - 2029
8. Competitive Landscape
8.1. Market Share Analysis, 2021
8.2. Company Profiles
8.2.1. Bristol Myers Squibb
8.2.1.1. Company overview
8.2.1.2. Financial performance
8.2.1.3. Product Portfolio
8.2.1.4. Recent Developments
8.2.2. Bayer AG
8.2.3. BioMarin Pharmaceutical, Inc.
8.2.4. F. Hoffman La Roche Ltd
8.2.5. Grifols SA
8.2.6. Sanofi
8.2.7. Takeda Pharmaceutical Company Limited
8.2.8. Medexus Pharmaceuticals, Inc.
8.2.9. NovoNordisk A/S
8.2.10. Pfizer
9. Appendix
9.1. Research Methodology
9.2. Report Specific Research Approach

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