Global Markets for Orphan Drugs
Global Markets for Orphan Drugs
Abstract
Report Scope:
The scope of this study includes orphan drugs used in the global pharmaceutical markets. This report analyzes the therapeutic applications of orphan drugs in rare and orphan diseases as well as examines the regulatory framework, patents, and recent innovations in the industry. The report also analyzes each submarket and its applications, projections and market shares. Technological issues include the latest trends and developments.
This report covers the U.S. and European markets, and emerging global markets such as India, Japan, China, Singapore, Taiwan, Korea, Canada, Brazil, Africa, Australia and New Zealand.
This report also analyzes the regulatory framework for the clinical trials of orphan drugs in rare and orphan diseases. The report examines the trends and characteristics of clinical trials conducted on rare diseases from 1999-2018, using information from various sources. In addition, this report provides an overview of the global orphan drugs market and its competitive landscape.
Report Includes:
- 26 tables
- An updated review of the global markets for orphan drugs (ODs) within the healthcare industry
- Analyses of the global market trends, with data from 2020, estimates for 2021 and projections of compound annual growth rates (CAGRs) through 2026
- Estimation of market size and revenue forecast for biological and non-biological orphan drugs, and corresponding market share analysis by product type, therapeutic category and geographic region
- Emphasis on the regulation of clinical trials on orphan designated drugs by leading regulatory authorities including the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Pharmaceutical Affairs and Food Sanitation Council (PAFSC) of Japan
- Review of the current status and trends in clinical trials for the development of drugs in rare diseases among the three registries from the U.S., EU and Japan
- Discussion of successful orphan medicinal products, rising influence of pharmacogenomics in the ODs market, information on specific rare diseases for which orphan drugs exist
- Assessment of the most promising therapeutic areas in the industry and leading orphan drugs in these areas
- Highlights of the industry structure for orphan drugs, pricing and reimbursement policies, COVID-19 impact assessment and market share analysis of the top 20 manufacturers of orphan drugs
- Descriptive company profiles of the market leading participants, including AbbVie Inc., Eli Lilly, Johnson & Johnson, Merck & Co., Pfizer Inc. and Sanofi-Aventis
Summary:
Orphan drugs have paved theway for the treatment of the many unmet clinical and therapeuticneeds of patientswith orphan and rare diseases such as Gaucher’s disease, Pompe disease, cystic fibrosis, Hunter syndrome, tuberous sclerosis complex and chronic myelogenous leukemia. These diseases, which have different globaldefinitions depending on theirprevalence in the general population, have gained significant attention since the approval of the 1983 OrphanDrug Act (ODA) in the U.S. and the formation of organizations such as the National Organization forRare Diseases (NORD) in 1983 and the EuropeanOrganization forRare Diseases (EURORDIS) in 1997. These developments have spurred
pharmaceutical and biotech companies such as Roche,Novartis, GlaxoSmithKline, Johnson&Johnson, Bristol-Myers Squibb,Merck& Co, Pfizer, Shire and others to invest in and develop orphan and rare disease therapies and drugs.
The orphan drug market has emerged as a niche business of the pharmaceutical industry. This market’s primary growth factors include incentives for orphan drug sponsors, tax breaks,market exclusivity, grants programs for clinical studies, funds for the development of orphan products andmultiple indication approvals. The unveiling of the human genetic code,which created a betterunderstanding of humanmetabolismand the role of genetics in the manifestation of disease, also played a significant role in the development of the orphan drugmarket.
By region, the U.S. dominates the global orphan drugmarket due to growth factors such as advanced healthcare infrastructure and the U.S. Food and Drug Administration (FDA)-sponsored grant programs for the development of potential therapeutic compounds through itsOffice ofOrphan Products Development (OOPD). The Europeanmarket,with itswell-established orphan drug legal framework, also is expanding.Growth factors include multiple post-market safety and efficacy clinical trials and the commission of potentialorphan drugs through patient networks such as the EuropeanOrganization for Rare Diseases (EURORDIS).
The Asian pharmaceutical market offers potentialas countries such as Japan, Taiwan and South Korea have implemented legal legislations akin to the U.S.OrphanDrug Act. The main growth factors in this emerging regional market are patient awareness programs, global connectivity through rare disease patient organizations and the availability of orphan drugs through joint collaborations.
The global orphan drug market reached REDACTED in 2020. This market should reach REDACTED and REDACTED in 2021 and 2026, respectively, achieving aCAGRof REDACTED during the forecast period. Growth factors include the increased involvement of pharmaceutical companies in developing orphan drugs, the growing numberof newapprovals and the increased global availability of these drugs.
The biological orphan drug market reached REDACTED in 2020. This market is expected to reach REDACTED and REDACTED by 2021 and 2026, respectively, achieving a CAGRof REDACTED during the forecast period.Multiple orphan approvals for pre-existing drugs should boost this growth. The non-biological orphan drugmarket reached REDACTED in 2020. This market should reach REDACTED by 2026, growing at a CAGRof REDACTED during the forec ast period.
Content
Table of Contents
Chapter 1 Introduction
Study Goals and Objectives
Reasons for Doing This Study
What's New in This Update
Scope of Report
Information Sources
Methodology
Geographic Breakdown
Analyst's Credentials
BCC Custom Research
Related BCC Research Reports
Chapter 2 Summary and Highlights
Chapter 3 Definitions and Background
Types of Orphan Drugs
Biological Orphan Drugs
Chemical or Non-biological Orphan Drugs
Global Definitions of Rare Diseases by Country/Region
U.S.
EU
Taiwan
Japan
Australia
Chapter 4 FDA Regulation of Clinical Trials in Orphan-Designated Drugs
Regulation of Drugs and Biologics
Accelerated Approvals
Fast-track Designation
Breakthrough Designation
Priority Review
Controlled Substances Act
Special Protocol Assessments
New Surveillance and Safety Requirements
Regulation of Clinical Trials in Orphan-Designated Drugs
Content and Format of a Sponsor's Request
Providing Written Recommendations
Refusal to Provide Written Recommendations
Orphan Drug Designation
Orphan Drug Exclusive Approval
Chapter 5 European Regulation of Clinical Trials in Orphan-Designated Drugs
European Orphan Regulation
Legal Framework
Applying for Orphan Designation
Application Challenges and Maintenance
Demonstrating Significant Benefit
Timelines
Activities After Orphan Designation: Annual Reports
Activities During Marketing Authorization Application
Maintenance of the Orphan Drug Status
Chapter 6 Asian Regulation of Clinical Trials in Orphan-Designated Drugs
Orphan Drug Designation System in Japan
Designation Criteria
Orphan Drug/Medical Device Designation Procedure
Designation Consultation
Regulation of Rare Diseases and Orphan Drugs in Taiwan
Regulation of Rare Diseases and Orphan Drugs in South Korea
Chapter 7 Trends in Clinical Trials for Drug Development in Rare Diseases
NCT, EUCTR and JPRN Registry Characteristics
The Relationship Among Clinical Trials, Diseases and Drugs
Characteristics of the Three Registries and Disease-Drug Relationships
Chapter 8 Global Orphan Drug Market Analysis
Market Overview
Market Revenue
Market Shares
Market by Region
Market Overview
Market Revenue
Market Share
Biological Orphan Drugs
Market by Region
Non-biological Orphan Drugs
Market by Region
Global Market by Product Type
Global Orphan Drug Sales by Therapeutic Category
Chapter 9 Industry Structure and Current Trends
Impact of COVID-19 on the Orphan Drug Market
Factors Boosting the Orphan Drug Market
Orphan Drug Act and Similar Legislation
Technological Advances and Genetic Codes
Generic Competition
Patent Expirations
Premium Pricing
Innovations in Manufacturing Technologies
Collaborations and Licensing Agreements
Challenges
Lack of Trained Professionals
Vulnerable Target Groups
Multiple Usages
Regulatory Challenge
Chapter 10 Orphan Drug 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 11 Company Profiles
ABBVIE INC.
ASTELLAS PHARMA INC.
ASTRAZENECA PLC
BRISTOL-MYERS SQUIBB
EISAI CO., LTD.
ELI LILLY AND CO.
F. HOFFMANN-LA ROCHE LTD.
GILEAD SCIENCES INC.
JOHNSON & JOHNSON
MERCK & CO.
NOVARTIS AG
PFIZER INC.
SANOFI-AVENTIS
TAKEDA PHARMACEUTICAL CO., LTD.
TEVA PHARMACEUTICAL INDUSTRIES LTD.
Chapter 12 Clinical Trials in Drugs for Rare Diseases
List of Clinical Trials in Drugs for Rare Diseases
Chapter 13 Appendix: References and Acronyms
References
Acronyms
List of Tables
Summary Table : Global Market for Orphan Drugs, by Product Type, Through 2026
Table 1 : Prevalence of Rare Diseases
Table 2 : Orphan-Designation Application Documents
Table 3 : Summary of Orphan Designation in Europe
Table 4 : Overview of Organizations and Responsibilities in Japan
Table 5 : Comparison of the Regulation of Rare Diseases and Orphan Drugs Worldwide
Table 6 : Characteristics of the NCT, EUCTR and JPRN Trials
Table 7 : Number of Trials in NCT, EUCTR and JPRN Trials, by Recruitment Status
Table 8 : Number of Trials in NCT, EUCTR and JPRN Trials, by Gender
Table 9 : Number of Trials in NCT, EUCTR and JPRN Trials, by Phase
Table 10 : Number of Trials in NCT, EUCTR and JPRN Trials, by Country
Table 11 : Top 20 Most Studied Rare Diseases, by Number of Trials
Table 12 : Top 20 Most Studied Rare Diseases, by Number of Diseases
Table 13 : Global Market for Orphan Drugs, by Product Type, Through 2026
Table 14 : Global Market for Orphan Drugs Share, by Product Type, 2020
Table 15 : Growth in Orphan Designations, by Region/Country, 2010-2020
Table 16 : Global Market for Orphan Drugs, by Region, Through 2026
Table 17 : Global Market Share of Orphan Drugs, by Region, 2020
Table 18 : Global Market for Biological Orphan Drugs, by Region, Through 2026
Table 19 : Global Market for Non-biological Orphan Drugs, by Region, Through 2026
Table 20 : Global Orphan Drug Sales, by Product Type, Through 2026
Table 21 : Global Orphan Drug Sales, by Therapeutic Category, Through 2026
Table 22 : Market Shares of Top 20 Companies, 2020
Table 23 : Orphan Drug Approvals and Exclusivity, 2020
Table 24 : Clinical Trials in Drugs for Rare Diseases
Table 25 : Acronyms Used in This report
List of Figures
Summary Figure : Global Market for Orphan Drugs, by Product Type, 2020-2026
Figure 1 : Orphan Designation Process, Japan
Figure 2 : Orphan Designation Consultation and Evaluation, Japan
Figure 3 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Trials
Figure 4 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Diseases
Figure 5 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Drugs
Figure 6 : Global Market Share of Orphan Drugs, by Product Type, 2020
Figure 7 : Global Market Share of Orphan Drugs, by Region, 2020
Figure 8 : Cumulative Change from Baseline in Total Market and Orphan Drug New Therapy Starts During COVID-19
Figure 9 : Conceptual Framework for Analyzing Current Strategies for Pricing Definition
The scope of this study includes orphan drugs used in the global pharmaceutical markets. This report analyzes the therapeutic applications of orphan drugs in rare and orphan diseases as well as examines the regulatory framework, patents, and recent innovations in the industry. The report also analyzes each submarket and its applications, projections and market shares. Technological issues include the latest trends and developments.
This report covers the U.S. and European markets, and emerging global markets such as India, Japan, China, Singapore, Taiwan, Korea, Canada, Brazil, Africa, Australia and New Zealand.
This report also analyzes the regulatory framework for the clinical trials of orphan drugs in rare and orphan diseases. The report examines the trends and characteristics of clinical trials conducted on rare diseases from 1999-2018, using information from various sources. In addition, this report provides an overview of the global orphan drugs market and its competitive landscape.
Report Includes:
- 26 tables
- An updated review of the global markets for orphan drugs (ODs) within the healthcare industry
- Analyses of the global market trends, with data from 2020, estimates for 2021 and projections of compound annual growth rates (CAGRs) through 2026
- Estimation of market size and revenue forecast for biological and non-biological orphan drugs, and corresponding market share analysis by product type, therapeutic category and geographic region
- Emphasis on the regulation of clinical trials on orphan designated drugs by leading regulatory authorities including the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Pharmaceutical Affairs and Food Sanitation Council (PAFSC) of Japan
- Review of the current status and trends in clinical trials for the development of drugs in rare diseases among the three registries from the U.S., EU and Japan
- Discussion of successful orphan medicinal products, rising influence of pharmacogenomics in the ODs market, information on specific rare diseases for which orphan drugs exist
- Assessment of the most promising therapeutic areas in the industry and leading orphan drugs in these areas
- Highlights of the industry structure for orphan drugs, pricing and reimbursement policies, COVID-19 impact assessment and market share analysis of the top 20 manufacturers of orphan drugs
- Descriptive company profiles of the market leading participants, including AbbVie Inc., Eli Lilly, Johnson & Johnson, Merck & Co., Pfizer Inc. and Sanofi-Aventis
Summary:
Orphan drugs have paved theway for the treatment of the many unmet clinical and therapeuticneeds of patientswith orphan and rare diseases such as Gaucher’s disease, Pompe disease, cystic fibrosis, Hunter syndrome, tuberous sclerosis complex and chronic myelogenous leukemia. These diseases, which have different globaldefinitions depending on theirprevalence in the general population, have gained significant attention since the approval of the 1983 OrphanDrug Act (ODA) in the U.S. and the formation of organizations such as the National Organization forRare Diseases (NORD) in 1983 and the EuropeanOrganization forRare Diseases (EURORDIS) in 1997. These developments have spurred
pharmaceutical and biotech companies such as Roche,Novartis, GlaxoSmithKline, Johnson&Johnson, Bristol-Myers Squibb,Merck& Co, Pfizer, Shire and others to invest in and develop orphan and rare disease therapies and drugs.
The orphan drug market has emerged as a niche business of the pharmaceutical industry. This market’s primary growth factors include incentives for orphan drug sponsors, tax breaks,market exclusivity, grants programs for clinical studies, funds for the development of orphan products andmultiple indication approvals. The unveiling of the human genetic code,which created a betterunderstanding of humanmetabolismand the role of genetics in the manifestation of disease, also played a significant role in the development of the orphan drugmarket.
By region, the U.S. dominates the global orphan drugmarket due to growth factors such as advanced healthcare infrastructure and the U.S. Food and Drug Administration (FDA)-sponsored grant programs for the development of potential therapeutic compounds through itsOffice ofOrphan Products Development (OOPD). The Europeanmarket,with itswell-established orphan drug legal framework, also is expanding.Growth factors include multiple post-market safety and efficacy clinical trials and the commission of potentialorphan drugs through patient networks such as the EuropeanOrganization for Rare Diseases (EURORDIS).
The Asian pharmaceutical market offers potentialas countries such as Japan, Taiwan and South Korea have implemented legal legislations akin to the U.S.OrphanDrug Act. The main growth factors in this emerging regional market are patient awareness programs, global connectivity through rare disease patient organizations and the availability of orphan drugs through joint collaborations.
The global orphan drug market reached REDACTED in 2020. This market should reach REDACTED and REDACTED in 2021 and 2026, respectively, achieving aCAGRof REDACTED during the forecast period. Growth factors include the increased involvement of pharmaceutical companies in developing orphan drugs, the growing numberof newapprovals and the increased global availability of these drugs.
The biological orphan drug market reached REDACTED in 2020. This market is expected to reach REDACTED and REDACTED by 2021 and 2026, respectively, achieving a CAGRof REDACTED during the forecast period.Multiple orphan approvals for pre-existing drugs should boost this growth. The non-biological orphan drugmarket reached REDACTED in 2020. This market should reach REDACTED by 2026, growing at a CAGRof REDACTED during the forec ast period.
Chapter 1 Introduction
Study Goals and Objectives
Reasons for Doing This Study
What's New in This Update
Scope of Report
Information Sources
Methodology
Geographic Breakdown
Analyst's Credentials
BCC Custom Research
Related BCC Research Reports
Chapter 2 Summary and Highlights
Chapter 3 Definitions and Background
Types of Orphan Drugs
Biological Orphan Drugs
Chemical or Non-biological Orphan Drugs
Global Definitions of Rare Diseases by Country/Region
U.S.
EU
Taiwan
Japan
Australia
Chapter 4 FDA Regulation of Clinical Trials in Orphan-Designated Drugs
Regulation of Drugs and Biologics
Accelerated Approvals
Fast-track Designation
Breakthrough Designation
Priority Review
Controlled Substances Act
Special Protocol Assessments
New Surveillance and Safety Requirements
Regulation of Clinical Trials in Orphan-Designated Drugs
Content and Format of a Sponsor's Request
Providing Written Recommendations
Refusal to Provide Written Recommendations
Orphan Drug Designation
Orphan Drug Exclusive Approval
Chapter 5 European Regulation of Clinical Trials in Orphan-Designated Drugs
European Orphan Regulation
Legal Framework
Applying for Orphan Designation
Application Challenges and Maintenance
Demonstrating Significant Benefit
Timelines
Activities After Orphan Designation: Annual Reports
Activities During Marketing Authorization Application
Maintenance of the Orphan Drug Status
Chapter 6 Asian Regulation of Clinical Trials in Orphan-Designated Drugs
Orphan Drug Designation System in Japan
Designation Criteria
Orphan Drug/Medical Device Designation Procedure
Designation Consultation
Regulation of Rare Diseases and Orphan Drugs in Taiwan
Regulation of Rare Diseases and Orphan Drugs in South Korea
Chapter 7 Trends in Clinical Trials for Drug Development in Rare Diseases
NCT, EUCTR and JPRN Registry Characteristics
The Relationship Among Clinical Trials, Diseases and Drugs
Characteristics of the Three Registries and Disease-Drug Relationships
Chapter 8 Global Orphan Drug Market Analysis
Market Overview
Market Revenue
Market Shares
Market by Region
Market Overview
Market Revenue
Market Share
Biological Orphan Drugs
Market by Region
Non-biological Orphan Drugs
Market by Region
Global Market by Product Type
Global Orphan Drug Sales by Therapeutic Category
Chapter 9 Industry Structure and Current Trends
Impact of COVID-19 on the Orphan Drug Market
Factors Boosting the Orphan Drug Market
Orphan Drug Act and Similar Legislation
Technological Advances and Genetic Codes
Generic Competition
Patent Expirations
Premium Pricing
Innovations in Manufacturing Technologies
Collaborations and Licensing Agreements
Challenges
Lack of Trained Professionals
Vulnerable Target Groups
Multiple Usages
Regulatory Challenge
Chapter 10 Orphan Drug 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 11 Company Profiles
ABBVIE INC.
ASTELLAS PHARMA INC.
ASTRAZENECA PLC
BRISTOL-MYERS SQUIBB
EISAI CO., LTD.
ELI LILLY AND CO.
F. HOFFMANN-LA ROCHE LTD.
GILEAD SCIENCES INC.
JOHNSON & JOHNSON
MERCK & CO.
NOVARTIS AG
PFIZER INC.
SANOFI-AVENTIS
TAKEDA PHARMACEUTICAL CO., LTD.
TEVA PHARMACEUTICAL INDUSTRIES LTD.
Chapter 12 Clinical Trials in Drugs for Rare Diseases
List of Clinical Trials in Drugs for Rare Diseases
Chapter 13 Appendix: References and Acronyms
References
Acronyms
List of Tables
Summary Table : Global Market for Orphan Drugs, by Product Type, Through 2026
Table 1 : Prevalence of Rare Diseases
Table 2 : Orphan-Designation Application Documents
Table 3 : Summary of Orphan Designation in Europe
Table 4 : Overview of Organizations and Responsibilities in Japan
Table 5 : Comparison of the Regulation of Rare Diseases and Orphan Drugs Worldwide
Table 6 : Characteristics of the NCT, EUCTR and JPRN Trials
Table 7 : Number of Trials in NCT, EUCTR and JPRN Trials, by Recruitment Status
Table 8 : Number of Trials in NCT, EUCTR and JPRN Trials, by Gender
Table 9 : Number of Trials in NCT, EUCTR and JPRN Trials, by Phase
Table 10 : Number of Trials in NCT, EUCTR and JPRN Trials, by Country
Table 11 : Top 20 Most Studied Rare Diseases, by Number of Trials
Table 12 : Top 20 Most Studied Rare Diseases, by Number of Diseases
Table 13 : Global Market for Orphan Drugs, by Product Type, Through 2026
Table 14 : Global Market for Orphan Drugs Share, by Product Type, 2020
Table 15 : Growth in Orphan Designations, by Region/Country, 2010-2020
Table 16 : Global Market for Orphan Drugs, by Region, Through 2026
Table 17 : Global Market Share of Orphan Drugs, by Region, 2020
Table 18 : Global Market for Biological Orphan Drugs, by Region, Through 2026
Table 19 : Global Market for Non-biological Orphan Drugs, by Region, Through 2026
Table 20 : Global Orphan Drug Sales, by Product Type, Through 2026
Table 21 : Global Orphan Drug Sales, by Therapeutic Category, Through 2026
Table 22 : Market Shares of Top 20 Companies, 2020
Table 23 : Orphan Drug Approvals and Exclusivity, 2020
Table 24 : Clinical Trials in Drugs for Rare Diseases
Table 25 : Acronyms Used in This report
List of Figures
Summary Figure : Global Market for Orphan Drugs, by Product Type, 2020-2026
Figure 1 : Orphan Designation Process, Japan
Figure 2 : Orphan Designation Consultation and Evaluation, Japan
Figure 3 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Trials
Figure 4 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Diseases
Figure 5 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Drugs
Figure 6 : Global Market Share of Orphan Drugs, by Product Type, 2020
Figure 7 : Global Market Share of Orphan Drugs, by Region, 2020
Figure 8 : Cumulative Change from Baseline in Total Market and Orphan Drug New Therapy Starts During COVID-19
Figure 9 : Conceptual Framework for Analyzing Current Strategies for Pricing Definition
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