FEATURED COMPANIES
- Bristol-Myers Squibb Co.
- F. Hoffmann-La Roche Ltd.
- Novartis AG
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Global Orphan Drugs Market: About this market
The orphan drugs market analysis considers sales from both biologics and non-biologics. Our analysis also considers the sales of orphan drugs in Asia, Europe, North America, and ROW. In 2018, the biologics segment had a significant market share, and this trend is expected to continue over the forecast period. Factors such as high efficacy will play a significant role in the biologics segment to maintain its market position. Also, our global orphan drugs market report looks at factors such as robust pipeline and recent drug approvals, rising incidence of the rare disease, availability of incentives for orphan drug development. However, uncertainties in the delay in diagnosis, risks associated with the clinical development of novel orphan drugs and challenges associated with pricing and reimbursements for orphan drugs may hamper the growth of the orphan drugs industry over the forecast period.
Global Orphan Drugs Market: Overview
Rising incidence of rare diseases
Orphan drugs are specifically used to treat rare diseases, and the prevalence of such health conditions has a direct influence on the sales of orphan drugs. Rare diseases include inherited genetic disorders, rare types of cancer, tropical infectious diseases, and neurodegenerative diseases. The prevalence and distribution of rare diseases are skewed. Although some rare diseases are inherited, others are a product of harmful environmental factors. This will lead to the expansion of the global orphan drugs market at a CAGR of almost 12% during the forecast period.
The emergence of combination therapies and novel targets
The potential advances in several areas such as immunotherapies and CGT are rekindling interest and enabling progress in the development of orphan drugs. These advances have been made in target selection, and combination therapy approaches for effectively reversing the immunosuppressive mechanisms, thereby enhancing the durability of the therapeutics. This development is expected to have a positive impact on the overall market growth.
Competitive Landscape
With the presence of several major players, the global orphan drugs market is moderately fragmented. This robust vendor analysis is designed to help clients improve their market position, and in line with this, this report provides a detailed analysis of several leading orphan drugs manufacturers, that include Bristol-Myers Squibb Co., F. Hoffmann-La Roche Ltd., Johnson & Johnson Services Inc., Novartis AG, Takeda Pharmaceutical Co. Ltd.
Also, the orphan drugs market analysis report includes information on upcoming trends and challenges that will influence market growth. This is to help companies strategize and leverage on all forthcoming growth opportunities.
The orphan drugs market analysis considers sales from both biologics and non-biologics. Our analysis also considers the sales of orphan drugs in Asia, Europe, North America, and ROW. In 2018, the biologics segment had a significant market share, and this trend is expected to continue over the forecast period. Factors such as high efficacy will play a significant role in the biologics segment to maintain its market position. Also, our global orphan drugs market report looks at factors such as robust pipeline and recent drug approvals, rising incidence of the rare disease, availability of incentives for orphan drug development. However, uncertainties in the delay in diagnosis, risks associated with the clinical development of novel orphan drugs and challenges associated with pricing and reimbursements for orphan drugs may hamper the growth of the orphan drugs industry over the forecast period.
Global Orphan Drugs Market: Overview
Rising incidence of rare diseases
Orphan drugs are specifically used to treat rare diseases, and the prevalence of such health conditions has a direct influence on the sales of orphan drugs. Rare diseases include inherited genetic disorders, rare types of cancer, tropical infectious diseases, and neurodegenerative diseases. The prevalence and distribution of rare diseases are skewed. Although some rare diseases are inherited, others are a product of harmful environmental factors. This will lead to the expansion of the global orphan drugs market at a CAGR of almost 12% during the forecast period.
The emergence of combination therapies and novel targets
The potential advances in several areas such as immunotherapies and CGT are rekindling interest and enabling progress in the development of orphan drugs. These advances have been made in target selection, and combination therapy approaches for effectively reversing the immunosuppressive mechanisms, thereby enhancing the durability of the therapeutics. This development is expected to have a positive impact on the overall market growth.
Competitive Landscape
With the presence of several major players, the global orphan drugs market is moderately fragmented. This robust vendor analysis is designed to help clients improve their market position, and in line with this, this report provides a detailed analysis of several leading orphan drugs manufacturers, that include Bristol-Myers Squibb Co., F. Hoffmann-La Roche Ltd., Johnson & Johnson Services Inc., Novartis AG, Takeda Pharmaceutical Co. Ltd.
Also, the orphan drugs market analysis report includes information on upcoming trends and challenges that will influence market growth. This is to help companies strategize and leverage on all forthcoming growth opportunities.
Note: Product cover images may vary from those shown
FEATURED COMPANIES
- Bristol-Myers Squibb Co.
- F. Hoffmann-La Roche Ltd.
- Novartis AG
PART 01: EXECUTIVE SUMMARY
PART 02: SCOPE OF THE REPORT
PART 07: MARKET SEGMENTATION BY PRODUCT
PART 09: GEOGRAPHIC LANDSCAPE
PART 11: DRIVERS AND CHALLENGES
Exhibit 01: Recent mega-mergers in pharmaceuticals
Exhibit 02: Global pharmaceuticals market
Exhibit 03: Segments of global pharmaceuticals market
Exhibit 04: Market characteristics
Exhibit 05: Market segments
Exhibit 06: Market definition - Inclusions and exclusions checklist
Exhibit 07: Market size 2018
Exhibit 08: Global market: Size and forecast 2018-2023 ($ billions)
Exhibit 09: Global market: Year-over-year growth 2019-2023 (%)
Exhibit 10: Some of the first-in-class orphan drugs approved by the US FDA during 2017-2018
Exhibit 11: Five forces analysis 2018
Exhibit 12: Five forces analysis 2023
Exhibit 13: Bargaining power of buyers
Exhibit 14: Bargaining power of suppliers
Exhibit 15: Threat of new entrants
Exhibit 16: Threat of substitutes
Exhibit 17: Threat of rivalry
Exhibit 18: Market condition - Five forces 2018
Exhibit 19: Some of the late-stage orphan drugs
Exhibit 20: Product - Market share 2018-2023 (%)
Exhibit 21: Comparison by product
Exhibit 22: Biologics - Market size and forecast 2018-2023 ($ billions)
Exhibit 23: Number of new or expanded indications approved for novel orphan biologics to the number of rare and non-rare diseases from the US FDA January 2013-June 2019
Exhibit 24: Promising regenerative orphan drugs pricing plans
Exhibit 25: Biologics - Year-over-year growth 2019-2023 (%)
Exhibit 26: Non-biologics - Market size and forecast 2018-2023 ($ billions)
Exhibit 27: Non-biologics - Year-over-year growth 2019-2023 (%)
Exhibit 28: Market opportunity by product
Exhibit 29: Customer landscape
Exhibit 30: Market share by geography 2018-2023 (%)
Exhibit 31: Geographic comparison
Exhibit 32: North America - Market size and forecast 2018-2023 ($ billions)
Exhibit 33: Number of orphan drug approvals in US 2014-2018
Exhibit 34: US FDA orphan drug marketing approvals in major therapeutic areas 2008-2017
Exhibit 35: List of expensive orphan drugs launched in US
Exhibit 36: Leading vendors and their product sales in US 2018.
Exhibit 37: Status of BTD, PR, and ODD for novel drugs 2013-2017
Exhibit 38: RMAT designation requests by product type 2018
Exhibit 39: North America - Year-over-year growth 2019-2023 (%)
Exhibit 40: Europe - Market size and forecast 2018-2023 ($ billions)
Exhibit 41: Orphan drug approvals in Europe during 2010-2018
Exhibit 42: Applications for ODD in EU 2005-2018
Exhibit 43: Europe - Year-over-year growth 2019-2023 (%)
Exhibit 44: Asia - Market size and forecast 2018-2023 ($ billions)
Exhibit 45: Orphan drugs eligible for reimbursable under China's state health insurance schemes
Exhibit 46: Asia - Year-over-year growth 2019-2023 (%)
Exhibit 47: ROW - Market size and forecast 2018-2023 ($ billions)
Exhibit 48: ROW - Year-over-year growth 2019-2023 (%)
Exhibit 49: Key leading countries
Exhibit 50: Market opportunity
Exhibit 51: List of promising drugs approved in US, Japan, and EU 2017-2019
Exhibit 52: Number of orphan drugs approved in US, EU, and Japan 2017 and 2018
Exhibit 53: Percentage of orphan and non-orphan drugs approved by the US FDA 2018
Exhibit 54: Incidence rate of rare diseases per year in US
Exhibit 55: Prevalence of rare diseases 2018 (millions)
Exhibit 56: Orphan drugs regulatory framework and incentives in key leading regions or countries
Exhibit 57: Impact of drivers and challenges
Exhibit 58: Several major M&A in the market
Exhibit 59: Expansion of combination therapies research areas of ICI approved drugs
Exhibit 60: Ongoing immunotherapy combinations studies with co-inhibitory molecules
Exhibit 61: Ongoing immunotherapy combinations studies with co-stimulatory molecules
Exhibit 62: Vendor landscape
Exhibit 63: Landscape disruption
Exhibit 64: Vendors covered
Exhibit 65: Vendor classification
Exhibit 66: Market positioning of vendors
Exhibit 67: Bristol-Myers Squibb Co. - Vendor overview
Exhibit 68: Bristol-Myers Squibb Co. - Business segments
Exhibit 69: Bristol-Myers Squibb Co. - Organizational developments
Exhibit 70: Bristol-Myers Squibb Co. - Geographic focus
Exhibit 71: Bristol-Myers Squibb Co. - Key offerings
Exhibit 72: Bristol-Myers Squibb Co. - Key customers
Exhibit 73: F. Hoffmann-La Roche Ltd. - Vendor overview
Exhibit 74: F. Hoffmann-La Roche Ltd. - Business segments
Exhibit 75: F. Hoffmann-La Roche Ltd. - Organizational developments
Exhibit 76: F. Hoffmann-La Roche Ltd. - Geographic focus
Exhibit 77: F. Hoffmann-La Roche Ltd. - Segment focus
Exhibit 78: F. Hoffmann-La Roche Ltd. - Key offerings
Exhibit 79: F. Hoffmann-La Roche Ltd. - Key customers
Exhibit 80: Johnson & Johnson Services Inc. - Vendor overview
Exhibit 81: Johnson & Johnson Services Inc. - Business segments
Exhibit 82: Johnson & Johnson Services Inc. - Organizational developments
Exhibit 83: Johnson & Johnson Services Inc. - Geographic focus
Exhibit 84: Johnson & Johnson Services Inc. - Segment focus
Exhibit 85: Johnson & Johnson Services Inc. - Key offerings
Exhibit 86: Johnson & Johnson Services Inc. - Key customers
Exhibit 87: Novartis AG - Vendor overview
Exhibit 88: Novartis AG - Business segments
Exhibit 89: Novartis AG - Organizational developments
Exhibit 90: Novartis AG - Geographic focus
Exhibit 91: Novartis AG - Segment focus
Exhibit 92: Novartis AG - Key offerings
Exhibit 93: Novartis AG - Key customers
Exhibit 94: Takeda Pharmaceutical Co. Ltd. - Vendor overview
Exhibit 95: Takeda Pharmaceutical Co. Ltd. - Business segments
Exhibit 96: Takeda Pharmaceutical Co. Ltd. - Organizational developments
Exhibit 97: Takeda Pharmaceutical Co. Ltd. - Geographic focus
Exhibit 98: Takeda Pharmaceutical Co. Ltd. - Key offerings
Exhibit 99: Takeda Pharmaceutical Co. Ltd. - Key customers
Exhibit 100: Validation techniques employed for market sizing
Exhibit 101: Definition of market positioning of vendors
PART 02: SCOPE OF THE REPORT
- Preface
- Currency conversion rates for US$
- Market ecosystem
- Market characteristics
- Market segmentation analysis
- Market definition
- Market sizing 2018
- Market size and forecast 2018-2023
- Bargaining power of buyers
- Bargaining power of suppliers
- Threat of new entrants
- Threat of substitutes
- Threat of rivalry
- Market condition
PART 07: MARKET SEGMENTATION BY PRODUCT
- Market segmentation by product
- Comparison by product
- Biologics - Market size and forecast 2018-2023
- Non-biologics - Market size and forecast 2018-2023
- Market opportunity by product
PART 09: GEOGRAPHIC LANDSCAPE
- Geographic segmentation
- Geographic comparison
- North America - Market size and forecast 2018-2023
- Europe - Market size and forecast 2018-2023
- Asia - Market size and forecast 2018-2023
- ROW - Market size and forecast 2018-2023
- Key leading countries
- Market opportunity
PART 11: DRIVERS AND CHALLENGES
- Market drivers
- Market challenges
- Strategic focus on alliances, partnerships, and M&A
- Emergence of combination therapies and novel targets
- Rising demand for genetic tests and protein biomarkers
- Overview
- Landscape disruption
- Competitive scenario
- Vendors covered
- Vendor classification
- Market positioning of vendors
- Bristol-Myers Squibb Co.
- F. Hoffmann-La Roche Ltd.
- Johnson & Johnson Services Inc.
- Novartis AG
- Takeda Pharmaceutical Co. Ltd.
- Research methodology
- List of abbreviations
- Definition of market positioning of vendors
Exhibit 01: Recent mega-mergers in pharmaceuticals
Exhibit 02: Global pharmaceuticals market
Exhibit 03: Segments of global pharmaceuticals market
Exhibit 04: Market characteristics
Exhibit 05: Market segments
Exhibit 06: Market definition - Inclusions and exclusions checklist
Exhibit 07: Market size 2018
Exhibit 08: Global market: Size and forecast 2018-2023 ($ billions)
Exhibit 09: Global market: Year-over-year growth 2019-2023 (%)
Exhibit 10: Some of the first-in-class orphan drugs approved by the US FDA during 2017-2018
Exhibit 11: Five forces analysis 2018
Exhibit 12: Five forces analysis 2023
Exhibit 13: Bargaining power of buyers
Exhibit 14: Bargaining power of suppliers
Exhibit 15: Threat of new entrants
Exhibit 16: Threat of substitutes
Exhibit 17: Threat of rivalry
Exhibit 18: Market condition - Five forces 2018
Exhibit 19: Some of the late-stage orphan drugs
Exhibit 20: Product - Market share 2018-2023 (%)
Exhibit 21: Comparison by product
Exhibit 22: Biologics - Market size and forecast 2018-2023 ($ billions)
Exhibit 23: Number of new or expanded indications approved for novel orphan biologics to the number of rare and non-rare diseases from the US FDA January 2013-June 2019
Exhibit 24: Promising regenerative orphan drugs pricing plans
Exhibit 25: Biologics - Year-over-year growth 2019-2023 (%)
Exhibit 26: Non-biologics - Market size and forecast 2018-2023 ($ billions)
Exhibit 27: Non-biologics - Year-over-year growth 2019-2023 (%)
Exhibit 28: Market opportunity by product
Exhibit 29: Customer landscape
Exhibit 30: Market share by geography 2018-2023 (%)
Exhibit 31: Geographic comparison
Exhibit 32: North America - Market size and forecast 2018-2023 ($ billions)
Exhibit 33: Number of orphan drug approvals in US 2014-2018
Exhibit 34: US FDA orphan drug marketing approvals in major therapeutic areas 2008-2017
Exhibit 35: List of expensive orphan drugs launched in US
Exhibit 36: Leading vendors and their product sales in US 2018.
Exhibit 37: Status of BTD, PR, and ODD for novel drugs 2013-2017
Exhibit 38: RMAT designation requests by product type 2018
Exhibit 39: North America - Year-over-year growth 2019-2023 (%)
Exhibit 40: Europe - Market size and forecast 2018-2023 ($ billions)
Exhibit 41: Orphan drug approvals in Europe during 2010-2018
Exhibit 42: Applications for ODD in EU 2005-2018
Exhibit 43: Europe - Year-over-year growth 2019-2023 (%)
Exhibit 44: Asia - Market size and forecast 2018-2023 ($ billions)
Exhibit 45: Orphan drugs eligible for reimbursable under China's state health insurance schemes
Exhibit 46: Asia - Year-over-year growth 2019-2023 (%)
Exhibit 47: ROW - Market size and forecast 2018-2023 ($ billions)
Exhibit 48: ROW - Year-over-year growth 2019-2023 (%)
Exhibit 49: Key leading countries
Exhibit 50: Market opportunity
Exhibit 51: List of promising drugs approved in US, Japan, and EU 2017-2019
Exhibit 52: Number of orphan drugs approved in US, EU, and Japan 2017 and 2018
Exhibit 53: Percentage of orphan and non-orphan drugs approved by the US FDA 2018
Exhibit 54: Incidence rate of rare diseases per year in US
Exhibit 55: Prevalence of rare diseases 2018 (millions)
Exhibit 56: Orphan drugs regulatory framework and incentives in key leading regions or countries
Exhibit 57: Impact of drivers and challenges
Exhibit 58: Several major M&A in the market
Exhibit 59: Expansion of combination therapies research areas of ICI approved drugs
Exhibit 60: Ongoing immunotherapy combinations studies with co-inhibitory molecules
Exhibit 61: Ongoing immunotherapy combinations studies with co-stimulatory molecules
Exhibit 62: Vendor landscape
Exhibit 63: Landscape disruption
Exhibit 64: Vendors covered
Exhibit 65: Vendor classification
Exhibit 66: Market positioning of vendors
Exhibit 67: Bristol-Myers Squibb Co. - Vendor overview
Exhibit 68: Bristol-Myers Squibb Co. - Business segments
Exhibit 69: Bristol-Myers Squibb Co. - Organizational developments
Exhibit 70: Bristol-Myers Squibb Co. - Geographic focus
Exhibit 71: Bristol-Myers Squibb Co. - Key offerings
Exhibit 72: Bristol-Myers Squibb Co. - Key customers
Exhibit 73: F. Hoffmann-La Roche Ltd. - Vendor overview
Exhibit 74: F. Hoffmann-La Roche Ltd. - Business segments
Exhibit 75: F. Hoffmann-La Roche Ltd. - Organizational developments
Exhibit 76: F. Hoffmann-La Roche Ltd. - Geographic focus
Exhibit 77: F. Hoffmann-La Roche Ltd. - Segment focus
Exhibit 78: F. Hoffmann-La Roche Ltd. - Key offerings
Exhibit 79: F. Hoffmann-La Roche Ltd. - Key customers
Exhibit 80: Johnson & Johnson Services Inc. - Vendor overview
Exhibit 81: Johnson & Johnson Services Inc. - Business segments
Exhibit 82: Johnson & Johnson Services Inc. - Organizational developments
Exhibit 83: Johnson & Johnson Services Inc. - Geographic focus
Exhibit 84: Johnson & Johnson Services Inc. - Segment focus
Exhibit 85: Johnson & Johnson Services Inc. - Key offerings
Exhibit 86: Johnson & Johnson Services Inc. - Key customers
Exhibit 87: Novartis AG - Vendor overview
Exhibit 88: Novartis AG - Business segments
Exhibit 89: Novartis AG - Organizational developments
Exhibit 90: Novartis AG - Geographic focus
Exhibit 91: Novartis AG - Segment focus
Exhibit 92: Novartis AG - Key offerings
Exhibit 93: Novartis AG - Key customers
Exhibit 94: Takeda Pharmaceutical Co. Ltd. - Vendor overview
Exhibit 95: Takeda Pharmaceutical Co. Ltd. - Business segments
Exhibit 96: Takeda Pharmaceutical Co. Ltd. - Organizational developments
Exhibit 97: Takeda Pharmaceutical Co. Ltd. - Geographic focus
Exhibit 98: Takeda Pharmaceutical Co. Ltd. - Key offerings
Exhibit 99: Takeda Pharmaceutical Co. Ltd. - Key customers
Exhibit 100: Validation techniques employed for market sizing
Exhibit 101: Definition of market positioning of vendors
Note: Product cover images may vary from those shown
FEATURED COMPANIES
- Bristol-Myers Squibb Co.
- F. Hoffmann-La Roche Ltd.
- Novartis AG
The following companies are recognised as the key players in the global orphan drugs market: Bristol-Myers Squibb Co., F. Hoffmann-La Roche Ltd., Johnson & Johnson Services Inc., Novartis AG, Takeda Pharmaceutical Co. Ltd.
Commenting on the report, an analyst from the research team said: “The latest trend gaining momentum in the market is the emergence of combination therapies and novel targets.”
According to the report, one of the major drivers for this market is the rising incidence of rare diseases.
Further, the report states that one of the major factors hindering the growth of this market is risks associated with the clinical development of novel orphan drugs.
The study was conducted using an objective combination of primary and secondary information, including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.
Commenting on the report, an analyst from the research team said: “The latest trend gaining momentum in the market is the emergence of combination therapies and novel targets.”
According to the report, one of the major drivers for this market is the rising incidence of rare diseases.
Further, the report states that one of the major factors hindering the growth of this market is risks associated with the clinical development of novel orphan drugs.
The study was conducted using an objective combination of primary and secondary information, including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.
Note: Product cover images may vary from those shown
- Bristol-Myers Squibb Co.
- F. Hoffmann-La Roche Ltd.
- Johnson & Johnson Services Inc.
- Novartis AG
- Takeda Pharmaceutical Co. Ltd.
Note: Product cover images may vary from those shown
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