USD 134.91 billion
Rare Disease Treatment Market Size, Share, Growth Analysis, By Route of Administration(Oral, Injectable, and Others), By Drug(Biologics, Biosimilar, and Small Molecule), By Region - Industry Forecast 2025-2032
Report ID:
SQMIG35E2037 |
Region:
Global |
Published Date: March, 2024
Pages:
219
|Tables:
63
|Figures:
65
Rare Disease Treatment Market Insights
Rare Disease Treatment Market size was valued at USD 152.18 Billion in 2023 and is poised to grow from USD 171.66 Billion in 2024 to USD 449.93 Billion by 2032, growing at a CAGR of 12.8% during the forecast period (2025-2032).
The rare disease treatment market encompasses a critical facet of the pharmaceutical industry dedicated to addressing medical conditions that affect a limited number of individuals. These disorders, often referred to as orphan diseases, collectively impact a relatively small population, making them a challenge for research and development due to their scarcity and complexity. Despite their individual rarity, the combined prevalence of rare diseases is significant, affecting millions worldwide.
The market's growth is driven by various factors, including increasing awareness and advocacy efforts, advancements in genetic and molecular research, and the development of innovative therapeutic approaches. The emergence of precision medicine, enabled by breakthroughs in genomics, has opened new avenues for targeted therapies tailored to the specific genetic makeup of patients with rare diseases. Additionally, supportive regulatory initiatives such as orphan drug designations and incentives have incentivized pharmaceutical companies to invest in rare disease drug development.
Collaboration between academia, research institutions, and pharmaceutical companies is also a pivotal driver of the market. Public-private partnerships and collaborations between patient advocacy groups and industry players have led to accelerated research, clinical trials, and drug approvals for rare diseases. However, the high cost of development, limited patient populations for clinical trials, and challenges in diagnosis and patient recruitment remain significant hurdles in this market. Nonetheless, the rare disease treatment market continues to expand with the promise of delivering life-changing therapies to those who need them the most, reaffirming the commitment to improving the lives of individuals affected by these often-neglected conditions.
US Rare Disease Treatment Market is poised to grow at a sustainable CAGR for the next forecast year.
Market snapshot - 2024-2031
Global Market Size
Largest Segment
Injectable
Fastest Growth
Injectable
Growth Rate
12.8% CAGR
Global Rare Disease Treatment Market ($ Bn)
Country Share for North America Region (%)
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Rare Disease Treatment Market Segmental Analysis
The global rare diseases treatment market is segmented into Therapeutic Area, Route of Administration, Drug, Distribution Channel, and Region. Based on Therapeutic Area the market is segmented as, Cancer, Neurological Conditions, Cardiovascular Conditions, Musculoskeletal Conditions, Hematologic Disorders, Infectious Diseases, Metabolic disorders, Endocrine disorders, others. Based on Route of Administration the market is segmented as Oral, Injectable, Others. Based on Drug the market is segmented as, Biologics, Biosimilar, and Small Molecule. Based on Distribution Channel the market is segmented as, Hospital Pharmacy, Specialty Pharmacy, Online Pharmacy .Based on Regional the market is segmented as, North America, Europe, Asia Pacific, and LAMEA.
Rare Disease Treatment Market Analysis By Route Of Administration
The injectable segment held a dominant position in the market, contributing to more than 50.0% of the total revenue in 2021. This trend is anticipated to continue with significant growth projected in the forecast period. The surge in this segment's prominence can be attributed to the introduction of novel injectable therapies for addressing rare diseases. An illustrative example is the approval of Sarepta Therapeutics' AMONDYS 45 (casimersen injection) by the U.S. FDA in February 2021, designed for the treatment of Duchenne muscular dystrophy (DMD).
The global market is further categorized based on the route of administration, including oral, injectable, and other methods. An instance of the market's evolution comes from Pfizer Inc., which secured U.S. FDA approval in July 2022 for Xalkori (crizotinib) to treat pediatric and adult patients with ALK-positive inflammatory myofibroblastic tumor (IMT). The oral administration of Xalkori, with a recommended dose of 250 mg taken orally twice daily, underscores the growth potential of the oral segment. These recent advancements in both injectable and oral therapies underline the market's trajectory toward offering more diverse and effective treatment options for various rare diseases.
Rare Disease Treatment Market Analysis By Drug
In 2022, biologics asserted their dominance in the market, capturing a substantial revenue share exceeding 55.0%. The advancements achieved in biotechnology and research methodologies have significantly facilitated the creation of novel biologic drugs. The potential of these biological therapies to precisely target disease factors and transform the treatment landscape for numerous rare diseases is expected to be a driving force behind the segment's growth. A notable example is the approval granted by the U.S. FDA in 2019 for Zolgensma, a gene therapy designed to combat spinal muscular atrophy.
Within the global market, the drug type category exhibits segmentation into biologics, biosimilars, and small molecules. The emergence of biosimilars as a viable treatment option for rare diseases marked a significant milestone, offering patients new avenues of care. The expiration of patents for orphan biologics, coupled with regulatory provisions that support the development of biosimilars, is anticipated to foster increased market competition. This competitive landscape holds the potential to drive down drug prices, benefiting patients undergoing treatment for various rare conditions. Reports indicate that approximately 11% of orphan biologic manufacturers have biosimilar candidates in their development pipelines, signifying the progressive expansion of this segment. However, while the growth prospects are promising, challenges such as substantial R&D costs and a relatively small patient population could impact the segment's trajectory.
Global Rare disease treatment market By Route of Administration
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Rare Disease Treatment Market Regional Insights
With a commanding revenue share of over 60.0% in 2022, North America emerged as the dominant player in the rare disease treatment market. This can be attributed to a combination of factors including the high prevalence of rare diseases, a robust healthcare infrastructure, and the consistent approval of novel treatment products. A significant milestone was reached in October 2021 when AstraZeneca secured orphan drug designation from the U.S. FDA for Tezepelumab, intended for the treatment of eosinophilic esophagitis (EoE). The presence of accessible treatment options can contribute to enhanced patient compliance, consequently broadening the consumer base and driving revenue growth within the market.
On the other hand, the Asia Pacific region is poised to exhibit the most rapid growth during the forecast period. This trend is influenced by the proactive measures taken by governments to extend support to individuals affected by orphan diseases. For instance, in July 2022, the Indian government directed both national and state bodies to diligently implement health policies aimed at the care of patients with orphan diseases. Such governmental initiatives offer a significant opening for manufacturers to provide high-quality orphan medications to the government, thereby fostering revenue generation. This regional outlook underscores the potential for significant expansion within the rare disease treatment market.
Global Rare disease treatment market By Geography
- Largest
- Fastest
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Rare Disease Treatment Market Dynamics
Rare Disease Treatment Market Drivers
Increasing Research and Development:
- The growing investment in research and development activities to develop innovative treatments for rare diseases is a major driver. Advancements in biotechnology, genomics, and personalized medicine are enabling the discovery of novel therapeutic approaches.
Supportive Regulatory Environment:
- Favorable regulatory policies, such as orphan drug designations and fast-track approvals, encourage pharmaceutical companies to develop treatments for rare diseases. These incentives streamline the development and commercialization process.
Rare Disease Treatment Market Restraints
High Development Costs:
- The high cost of research, clinical trials, and development of treatments for rare diseases is a significant restraint. The limited patient population makes it challenging to recover these costs, leading to high drug prices.
Limited Patient Pool:
- The small patient population for each rare disease limits the market size for these treatments. Pharmaceutical companies face challenges in achieving economies of scale, resulting in higher costs per patient.
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Rare Disease Treatment Market Competitive Landscape
The competitive landscape of the rare disease treatment market is characterized by a mix of established pharmaceutical companies and emerging biotechnology firms. Key players in the market are actively focusing on developing innovative therapies and expanding their product portfolios to address the unmet medical needs of patients with rare diseases. These companies are investing heavily in research and development to bring novel treatments to the market, leveraging advancements in biotechnology, genomics, and precision medicine. Additionally, partnerships, collaborations, and licensing agreements with academic institutions and research organizations are common strategies adopted to accelerate the development and commercialization of rare disease treatments. The market is witnessing a surge in strategic acquisitions and mergers aimed at consolidating resources, expertise, and pipelines to expedite drug development. With the growing emphasis on orphan drug designations and favorable regulatory incentives, the competitive landscape is expected to remain dynamic, with companies vying to secure a strong market position by delivering effective and targeted therapies for a wide range of rare diseases.
Rare Disease Treatment Market Top Player’s Company Profile
- Novartis AG
- Pfizer Inc.
- Roche Holding AG
- Sanofi SA
- Johnson & Johnson
- AbbVie Inc.
- Vertex Pharmaceuticals Incorporated
- Amgen Inc.
- AstraZeneca PLC
- Bristol Myers Squibb Company
Rare Disease Treatment Market Recent Developments
- In January 2023, Novartis announced the approval of sebelipase alfa for the treatment of patients with lipoprotein lipase deficiency (LPLD). Sebelipase alfa is a gene therapy that is designed to replace the missing or defective lipoprotein lipase enzyme.
- In February 2023, Roche announced the approval of risdiplam for the treatment of spinal muscular atrophy (SMA). Risdiplam is a small molecule that is designed to increase the production of the SMN protein, which is essential for motor neuron function.
- In March 2023, Pfizer announced the approval of bimekizumab for the treatment of atopic dermatitis. Bimekizumab is a biologic that is designed to block the activity of the IL-17A and IL-17F cytokines, which are involved in the inflammation that is associated with atopic dermatitis.
Rare Disease Treatment Key Market Trends
- Advancements in Precision Medicine: The field of precision medicine is rapidly evolving, enabling the development of targeted therapies for specific genetic mutations or molecular abnormalities responsible for rare diseases. This approach enhances treatment efficacy and reduces adverse effects.
Rare Disease Treatment Market SkyQuest Analysis
SkyQuest’s ABIRAW (Advanced Business Intelligence, Research & Analysis Wing) is our Business Information Component types team that Collects, Collates, Correlates, and Analyzes the Data collected by means of Primary Exploratory Research backed by robust Secondary Desk research.
According to our Global Rare Disease Treatment Market analysis, the rare disease treatment market is characterized by its focus on addressing medical conditions affecting a small population. Geographically, North America dominated the market in 2021, accounting for over 60.0% of the revenue share due to its advanced healthcare infrastructure, high disease burden, and new product approvals. Meanwhile, the Asia Pacific region is projected to exhibit the fastest growth, attributed to government initiatives supporting orphan disease patients.
Segment-wise, the market is categorized by drug type into biologics, biosimilars, and small molecules. Biologics led with a revenue share of over 55.0% in 2021, driven by advancements in biotechnology and their high target specificity. The injectable route of administration held the largest share, with recent launches of novel injectables contributing to growth. The growth of this market is further fueled by a robust product pipeline, increased drug approvals, and the rising demand for rare disease treatments.
| Report Metric | Details |
|---|---|
| Market size value in 2023 | USD 134.91 billion |
| Market size value in 2031 | USD 398.87 billion |
| Growth Rate | 12.8% |
| Forecast period | 2024-2031 |
| Forecast Unit (Value) | USD Billion |
| Segments covered |
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| Regions covered | North America (US, Canada), Europe (Germany, France, United Kingdom, Italy, Spain, Rest of Europe), Asia Pacific (China, India, Japan, Rest of Asia-Pacific), Latin America (Brazil, Rest of Latin America), Middle East & Africa (South Africa, GCC Countries, Rest of MEA) |
| Companies covered |
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| Customization scope | Free report customization with purchase. Customization includes:-
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Table Of Content
Executive Summary
Market overview
- Exhibit: Executive Summary – Chart on Market Overview
- Exhibit: Executive Summary – Data Table on Market Overview
- Exhibit: Executive Summary – Chart on Rare Disease Treatment Market Characteristics
- Exhibit: Executive Summary – Chart on Market by Geography
- Exhibit: Executive Summary – Chart on Market Segmentation
- Exhibit: Executive Summary – Chart on Incremental Growth
- Exhibit: Executive Summary – Data Table on Incremental Growth
- Exhibit: Executive Summary – Chart on Vendor Market Positioning
Parent Market Analysis
Market overview
Market size
- Market Dynamics
- Exhibit: Impact analysis of DROC, 2021
- Drivers
- Opportunities
- Restraints
- Challenges
- Exhibit: Impact analysis of DROC, 2021
- SWOT Analysis
KEY MARKET INSIGHTS
- Technology Analysis
- (Exhibit: Data Table: Name of technology and details)
- Pricing Analysis
- (Exhibit: Data Table: Name of technology and pricing details)
- Supply Chain Analysis
- (Exhibit: Detailed Supply Chain Presentation)
- Value Chain Analysis
- (Exhibit: Detailed Value Chain Presentation)
- Ecosystem Of the Market
- Exhibit: Parent Market Ecosystem Market Analysis
- Exhibit: Market Characteristics of Parent Market
- IP Analysis
- (Exhibit: Data Table: Name of product/technology, patents filed, inventor/company name, acquiring firm)
- Trade Analysis
- (Exhibit: Data Table: Import and Export data details)
- Startup Analysis
- (Exhibit: Data Table: Emerging startups details)
- Raw Material Analysis
- (Exhibit: Data Table: Mapping of key raw materials)
- Innovation Matrix
- (Exhibit: Positioning Matrix: Mapping of new and existing technologies)
- Pipeline product Analysis
- (Exhibit: Data Table: Name of companies and pipeline products, regional mapping)
- Macroeconomic Indicators
COVID IMPACT
- Introduction
- Impact On Economy—scenario Assessment
- Exhibit: Data on GDP - Year-over-year growth 2016-2022 (%)
- Revised Market Size
- Exhibit: Data Table on Rare Disease Treatment Market size and forecast 2021-2027 ($ million)
- Impact Of COVID On Key Segments
- Exhibit: Data Table on Segment Market size and forecast 2021-2027 ($ million)
- COVID Strategies By Company
- Exhibit: Analysis on key strategies adopted by companies
MARKET DYNAMICS & OUTLOOK
- Market Dynamics
- Exhibit: Impact analysis of DROC, 2021
- Drivers
- Opportunities
- Restraints
- Challenges
- Exhibit: Impact analysis of DROC, 2021
- Regulatory Landscape
- Exhibit: Data Table on regulation from different region
- SWOT Analysis
- Porters Analysis
- Competitive rivalry
- Exhibit: Competitive rivalry Impact of key factors, 2021
- Threat of substitute products
- Exhibit: Threat of Substitute Products Impact of key factors, 2021
- Bargaining power of buyers
- Exhibit: buyers bargaining power Impact of key factors, 2021
- Threat of new entrants
- Exhibit: Threat of new entrants Impact of key factors, 2021
- Bargaining power of suppliers
- Exhibit: Threat of suppliers bargaining power Impact of key factors, 2021
- Competitive rivalry
- Skyquest special insights on future disruptions
- Political Impact
- Economic impact
- Social Impact
- Technical Impact
- Environmental Impact
- Legal Impact
Market Size by Region
- Chart on Market share by geography 2021-2027 (%)
- Data Table on Market share by geography 2021-2027(%)
- North America
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- USA
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Canada
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Europe
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- Germany
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Spain
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- France
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- UK
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Rest of Europe
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Asia Pacific
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- China
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- India
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Japan
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- South Korea
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Rest of Asia Pacific
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Latin America
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- Brazil
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Rest of South America
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Middle East & Africa (MEA)
- Chart on Market share by country 2021-2027 (%)
- Data Table on Market share by country 2021-2027(%)
- GCC Countries
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- South Africa
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
- Rest of MEA
- Exhibit: Chart on Market share 2021-2027 (%)
- Exhibit: Market size and forecast 2021-2027 ($ million)
KEY COMPANY PROFILES
- Competitive Landscape
- Total number of companies covered
- Exhibit: companies covered in the report, 2021
- Top companies market positioning
- Exhibit: company positioning matrix, 2021
- Top companies market Share
- Exhibit: Pie chart analysis on company market share, 2021(%)
- Total number of companies covered
Methodology
For the Rare Disease Treatment Market, our research methodology involved a mixture of primary and secondary data sources. Key steps involved in the research process are listed below:
1. Information Procurement: This stage involved the procurement of Market data or related information via primary and secondary sources. The various secondary sources used included various company websites, annual reports, trade databases, and paid databases such as Hoover's, Bloomberg Business, Factiva, and Avention. Our team did 45 primary interactions Globally which included several stakeholders such as manufacturers, customers, key opinion leaders, etc. Overall, information procurement was one of the most extensive stages in our research process.
2. Information Analysis: This step involved triangulation of data through bottom-up and top-down approaches to estimate and validate the total size and future estimate of the Rare Disease Treatment Market.
3. Report Formulation: The final step entailed the placement of data points in appropriate Market spaces in an attempt to deduce viable conclusions.
4. Validation & Publishing: Validation is the most important step in the process. Validation & re-validation via an intricately designed process helped us finalize data points to be used for final calculations. The final Market estimates and forecasts were then aligned and sent to our panel of industry experts for validation of data. Once the validation was done the report was sent to our Quality Assurance team to ensure adherence to style guides, consistency & design.
Analyst Support
Customization Options
With the given market data, our dedicated team of analysts can offer you the following customization options are available for the Rare Disease Treatment Market:
Product Analysis: Product matrix, which offers a detailed comparison of the product portfolio of companies.
Regional Analysis: Further analysis of the Rare Disease Treatment Market for additional countries.
Competitive Analysis: Detailed analysis and profiling of additional Market players & comparative analysis of competitive products.
Go to Market Strategy: Find the high-growth channels to invest your marketing efforts and increase your customer base.
Innovation Mapping: Identify racial solutions and innovation, connected to deep ecosystems of innovators, start-ups, academics, and strategic partners.
Category Intelligence: Customized intelligence that is relevant to their supply Markets will enable them to make smarter sourcing decisions and improve their category management.
Public Company Transcript Analysis: To improve the investment performance by generating new alpha and making better-informed decisions.
Social Media Listening: To analyze the conversations and trends happening not just around your brand, but around your industry as a whole, and use those insights to make better Marketing decisions.
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