Chapter 2 Summary and Highlights
- Market Outlook
- Market Summary
Chapter 3 Market Overview
- Market and Technology Background
- Global Definitions of Rare Diseases by Country/Region
- U.S.
- EU
- Taiwan
- Japan
- Australia
- Singapore
- South Korea
- Switzerland
- Brazil
Chapter 4 Market Dynamics
- Overview
- Drivers
- Favorable Orphan Drug Act and Similar Legislation
- Technological Advances in Genomics
- Lack of Competition from Generic Drugs
- Patent Extensions
- Premium Pricing of Orphan Drugs
- Innovations in Manufacturing Technologies
- Increasing Collaborations and Licensing Agreements
- Restraints
- Lack of Trained Professionals
- Vulnerable Target Groups
- Regulatory Challenges
- Opportunities
- Huge Product Pipeline of New Orphan Drugs
- Regulatory Landscape
- U.S.
- Europe
- Japan
- Taiwan
- South Korea
Chapter 5 Global Market for Orphan Drugs by Product Type
- Biological Orphan Drugs
- Non-biological Orphan Drugs
Chapter 6 Global Market for Orphan Drugs by Therapeutic Application
- Oncology
- Blood Disorders
- Central Nervous System
- Respiratory System
- Immunomodulators
- Cardiovascular Therapies
- Endocrine System
- Musculoskeletal System
- Others
Chapter 7 Global Market for Orphan Drugs by Region
- Global Market Share Analysis
- North America
- Europe
- Asia-Pacific
- Rest of the World (RoW)
Chapter 8 ESG Outlook in the Orphan Drugs Market
- Introduction to ESG
- Importance of ESG in the Orphan Drug Industry
- ESG Pillars in the Orphan Drugs Industry
- ESG Ratings and Metrics: Understanding the Data
- ESG practices in the Orphan Drug Industry
- Concluding Remarks
Chapter 9 Orphan Drugs Exclusivity and Pricing Policies
- Reimbursed Price of ODs: Current Strategies and Potential Improvements
- Comprehensive Value Assessment
- Early Dialogues
- Innovative Reimbursement Approaches
- Societal Participation in Producing ODs
Chapter 10 Clinical Trials on Drugs for Rare Diseases
- List of Clinical Trials on Drugs for Rare Diseases
Chapter 11 Competitive Landscape
- Top 10 Selling Orphan Drugs Globally
- Market Share Analysis
Chapter 12 Company Profiles
- Abbvie Inc.
- Astellas Pharma Inc.
- Astrazeneca Plc
- Bristol Myers Squibb
- Eisai Co. Ltd.
- Eli Lilly And Co.
- F. Hoffmann-La Roche Ltd.
- Merck & Co. Inc.
- Novartis Ag
- Pfizer Inc.
- Sanofi S.A.
- Takeda Pharmaceutical Co. Ltd.
Chapter 13 Appendix: Acronyms
List of Tables
Summary Table: Global Market for Orphan Drugs, by Product Type, Through 2028
Table 1: Prevalence of Rare Diseases
Table 2: Market Dynamics: Impact Analysis
Table 3: Orphan-Designation Application Documents
Table 4: Summary of Orphan Designation in Europe
Table 5: Overview of Organizations and Responsibilities in Japan
Table 6: Comparison of the Regulation of Rare Diseases and Orphan Drugs Worldwide
Table 7: Global Market for Orphan Drugs, by Product Type, Through 2028
Table 8: Global Market for Biological Orphan Drugs, by Region, Through 2028
Table 9: Global Market for Non-biological Orphan Drugs, by Region, Through 2028
Table 10: Global Market for Oncology Orphan Drugs, by Region, Through 2028
Table 11: Global Market for Blood Disorders Orphan Drugs, by Region, Through 2028
Table 12: Global Market for Central Nervous System Orphan Drugs, by Region, Through 2028
Table 13: Global Market for Respiratory System Orphan Drugs, by Region, Through 2028
Table 14: Global Market for Immunomodulator Therapies Orphan Drugs, by Region, Through 2028
Table 15: Global Market for Cardiovascular Therapies Orphan Drugs, by Region, Through 2028
Table 16: Global Market for Endocrine System Orphan Drugs, by Region, Through 2028
Table 17: Global Market for Musculoskeletal System Orphan Drugs, by Region, Through 2028
Table 18: Global Market for Other Therapeutic Applications Orphan Drugs, by Region, Through 2028
Table 19: Global Market Shares of Orphan Drugs, by Region, 2023
Table 20: North American Market for Orphan Drugs, by Product Type, Through 2028
Table 21: North American Market for Orphan Drugs, by Therapeutic Application, Through 2028
Table 22: European Market for Orphan Drugs, by Product Type, Through 2028
Table 23: European Market for Orphan Drugs, by Therapeutic Application, Through 2028
Table 24: Asia-Pacific Market for Orphan Drugs, by Product Type, Through 2028
Table 25: Asia-Pacific Market for Orphan Drugs, by Therapeutic Application, Through 2028
Table 26: Rest of the World Market for Orphan Drugs, by Product Type, Through 2028
Table 27: Rest of the World Market for Orphan Drugs, by Therapeutic Application, Through 2028
Table 28: ESG Ratings and Metrics
Table 29: Environmental Performance in the Orphan Drug Market
Table 30: Social Performance in the Orphan Drugs Market
Table 31: Governance Performance in the Orphan Drugs Market
Table 32: ESG Rankings for Major Orphan Drugs Companies, 2023*
Table 33: Clinical Trials in Drugs for Rare Diseases
Table 34: Global Market Ranking of the Top 10 Selling Orphan Drugs, 2022
Table 35: Global by Company Market Shares of Orphan Drugs, 2022
Table 36: AbbVie Inc.: Company Snapshot
Table 37: AbbVie Inc.: Financial Performance, 2022
Table 38: AbbVie Inc.: News, 2020-2022
Table 39: AbbVie Inc.: Products
Table 40: Astellas Pharma Inc.: Company Snapshot
Table 41: Astellas Pharma Inc.: Financial Performance, 2023
Table 42: Astellas Pharma Inc.: News, 2023
Table 43: Astellas Pharma Inc.: Products
Table 44: AstraZeneca Plc.: Company Snapshot
Table 45: AstraZeneca Plc: Financial Performance, 2022
Table 46: AstraZeneca Plc: News, 2022 and 2023
Table 47: AstraZeneca Plc: Products
Table 48: Bristol Myers Squibb: Company Snapshot
Table 49: Bristol Myers Squibb: Financial Performance, 2022
Table 50: Bristol Myers Squibb: News, 2021-2023
Table 51: Bristol Myers Squibb: Products
Table 52: Eisai Co. Ltd.: Company Snapshot
Table 53: Eisai Co. Ltd.: Financial Performance, 2022
Table 54: Eisai Co. Ltd.: News, 2021 and 2022
Table 55: Eisai Co. Ltd.: Products
Table 56: Eli Lilly and Co.: Company Snapshot
Table 57: Eli Lilly and Co.: Financial Performance, 2022
Table 58: Eli Lilly and Co.: Products
Table 59: F. Hofmann-La Roche Ltd.: Company Snapshot
Table 60: F. Hofmann-La Roche Ltd.: Financial Performance, 2022
Table 61: F. Hofmann-La Roche Ltd.: Products
Table 62: Merck & Co. Inc.: Company Snapshot
Table 63: Merck & Co. Inc.: Financial Performance, 2022
Table 64: Merck & Co. Inc.: Products
Table 65: Novartis AG: Company Snapshot
Table 66: Novartis AG: Financial Performance, 2022
Table 67: Novartis AG: News, 2020-2022
Table 68: Novartis AG: Products
Table 69: Pfizer Inc.: Company Snapshot
Table 70: Pfizer Inc.: Financial Performance, 2022
Table 71: Sanofi S.A.: Company Snapshot
Table 72: Sanofi S.A.: Financial Performance, 2022
Table 73: Sanofi S.A.: Products
Table 74: Takeda Pharmaceutical Co. Ltd.: Company Snapshot
Table 75: Takeda Pharmaceutical Co. Ltd.: Financial Performance, 2022
Table 76: Takeda Pharmaceutical Co. Ltd.: News, 2023
Table 77: Takeda Pharmaceutical Co. Ltd.: Product Portfolio
Table 78: Acronyms Used in This Report
List of Figures
Summary Figure: Global Market for Orphan Drugs, by Product Type, 2020-2028
Figure 1: Orphan Drugs Market Dynamics
Figure 2: Orphan Designation Process, Japan
Figure 3: Orphan Designation Consultation and Evaluation, Japan
Figure 4: Global Market for Orphan Drugs, by Product Type, 2020-2028
Figure 5: Global Market for Biological Orphan Drugs, by Region, 2020-2028
Figure 6: Global Market for Non-biological Orphan Drugs, by Region, 2020-2028
Figure 7: Global Market Shares of Orphan Drugs, by Therapeutic Application, 2023
Figure 8: Global Market for Oncology Orphan Drugs, by Region, 2020-2028
Figure 9: Global Market for Blood Disorders Orphan Drugs, by Region, 2020-2028
Figure 10: Global Market for Central Nervous System Orphan Drugs, by Region, 2020-2028
Figure 11: Global Market for Respiratory System Orphan Drugs, by Region, 2020-2028
Figure 12: Global Market for Immunomodulator Therapies Orphan Drugs, by Region, 2020-2028
Figure 13: Global Market for Cardiovascular Therapies Orphan Drugs, by Region, 2020-2028
Figure 14: Global Market for Endocrine System Orphan Drugs, by Region, 2020-2028
Figure 15: Global Market for Musculoskeletal System Orphan Drugs, by Region, 2020-2028
Figure 16: Global Market for Other Therapeutic Applications Orphan Drugs, by Region, 2020-2028
Figure 17: Global Market Shares of Orphan Drugs, by Region, 2023
Figure 18: North American Market for Orphan Drugs, by Product Type, 2020-2028
Figure 19: North American Market for Orphan Drugs, by Therapeutic Application, 2020-2028
Figure 20: European Market for Orphan Drugs, by Product Type, 2020-2028
Figure 21: European Market for Orphan Drugs, by Therapeutic Application, 2020-2028
Figure 22: Asia-Pacific Market for Orphan Drugs, by Product Type, 2020-2028
Figure 23: Asia-Pacific Market for Orphan Drugs, by Therapeutic Application, 2020-2028
Figure 24: Rest of the World Market for Orphan Drugs, by Product Type, 2020-2028
Figure 25: Rest of the World Market for Orphan Drugs, by Therapeutic Application, 2020-2028
Figure 26: Conceptual Framework for Analyzing Current Strategies for Pricing Definition
Figure 27: Global Company Market Shares of Orphan Drugs, 2022
Figure 28: AbbVie Inc.: Financial Performance, 2021 and 2022
Figure 29: AbbVie Inc.: Revenue Share, by Segment, 2022
Figure 30: AbbVie Inc.: Revenue Share, by Region, 2022
Figure 31: Astellas Pharma Inc.: Financial Performance, 2022 and 2023
Figure 32: Astellas Pharma Inc.: Revenue Share, by Segment, 2023
Figure 33: Astellas Pharma Inc.: Revenue Share, by Region/Country, 2023
Figure 34: AstraZeneca Plc: Financial Performance, 2021 and 2022
Figure 35: AstraZeneca Plc: Revenue Share, by Segment, 2022
Figure 36: AstraZeneca Plc: Revenue Share, by Region, 2022
Figure 37: Bristol Myers Squibb: Financial Performance, 2021 and 2022
Figure 38: Bristol Myers Squibb: Revenue Share, by Region, 2022
Figure 39: Eisai Co. Ltd.: Financial Performance, 2021 and 2022
Figure 40: Eisai Co. Ltd.: Revenue Share, by Segment, 2022
Figure 41: Eli Lilly and Co.: Financial Performance, 2021 and 2022
Figure 42: Eli Lilly and Co.: Revenue Share, by Segment, 2022
Figure 43: Eli Lilly and Co.: Revenue Share, by Region, 2022
Figure 44: F. Hofmann-La Roche Ltd.: Financial Performance, 2021 and 2022
Figure 45: F. Hofmann-La Roche Ltd.: Revenue Share, by Business Segment, 2022
Figure 46: Merck & Co. Inc.: Financial Performance, 2021 and 2022
Figure 47: Merck & Co. Inc.: Revenue Share, by Business Unit, 2022
Figure 48: Merck & Co. Inc.: Revenue Share, by Region, 2022
Figure 49: Novartis AG: Financial Performance, 2021 and 2022
Figure 50: Novartis AG: Revenue Share, by Segment, 2022
Figure 51: Novartis AG: Revenue Share, by Region, 2022
Figure 52: Pfizer Inc.: Financial Performance, 2021 and 2022
Figure 53: Pfizer Inc.: Revenue Share, by Segment, 2022
Figure 54: Pfizer Inc.: Revenue Share, by Region, 2022
Figure 55: Sanofi S.A.: Financial Performance, 2021 and 2022
Figure 56: Sanofi S.A.: Revenue Share, by Segment, 2022
Figure 57: Sanofi S.A.: Revenue Share, by Region, 2022
Figure 58: Takeda Pharmaceutical Co. Ltd.: Financial Performance, 2021 and 2022
Figure 59: Takeda Pharmaceutical Co. Ltd.: Revenue Share, by Business Unit, 2022
Figure 60: Takeda Pharmaceutical Co. Ltd.: Revenue Share, by Region/Country, 2022
