Report Scope:
This report analyzes the regulatory framework for the clinical trials of orphan drugs in rare and orphan diseases. The report also analyzes the trends and characteristics of clinical trials conducted on rare diseases from 1999-2018, using information from various sources.
This report covers an overview of the global orphan drugs market and its competitive landscape.
The Report Includes:
- An overview of the drug development in rare diseases with a special focus on clinical trial and regulatory landscape
- A detailed description of different kinds of orphan drugs and a comparative assessment of biological and chemical or non-biological orphan drugs
- Discussion on Orphan Drug Act; insights into a general framework for the regulation of drugs and biologics, and regulations for clinical trials of orphan designated drugs
- Highlights of technological advances and innovations in genetic manufacturing technologies and information on terms such as generic competition, patent expirations, premium pricing and information on orphan drug designation applications
Table of Contents
Chapter 1 Introduction
- Study Goals and Objectives
- Reasons for Doing this Study
- Scope of Report
- Information Sources
- Methodology
- Analyst's Credentials
- Related Reports
Chapter 2 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 3 FDA Regulation of Clinical Trials on Orphan Designated Drugs
- General Framework of the 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 on Orphan Designated Drugs
- Content and Format of a Request for Written Recommendations
- Providing Written Recommendations
- Refusal to Provide Written Recommendations
- Orphan Drug Designation
- Orphan Drug Exclusive Approval
Chapter 4 European Regulation of Clinical Trials on Orphan Designated Drugs
- European Orphan Regulation
- Legal Framework
- Applying for Orphan Designation
- Orphan Drug 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 5 Asian Regulation of Clinical Trials on 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 6 Trends in Clinical Trials for Drug Development in Rare Diseases
- Characteristics of the Three Registries
- The Relationship Among Clinical Trials, Diseases and Drugs
- Characteristics of the Three Registries and Disease-Drug Relationships
Chapter 7 Competitive Landscape and Global Markets
- Global Orphan Drug Sales by Therapeutic Category
- Orphan Drug Designation Counts by Region and Year
Chapter 8 Current Trends
- 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 9 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 Pharmaceuticals Ltd.
- Teva Pharmaceutical Industries Ltd.
Chapter 10 Clinical Trials on Drugs for Rare Diseases
- List of Clinical Trials on Drugs for Rare Diseases
Chapter 11 Appendix
- References
List of Tables
Table 1: Rare Diseases and their Prevalence
Table 2: List of Documents Included in the Orphan Designation Application
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: Top 20 Global Orphan Drug Sales, Through 2025
Table 14: Global Orphan Drug Sales, by Therapeutic Category, 2019-2025
Table 15: Growth in Orphan Designations, by Region
Table 16: Clinical Trials on Drugs for Rare Diseases
List of Figures
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 Top 10 Companies, 2019
Figure 7: Global Market Shares of Top 10 Companies, 2025
Executive Summary
The orphan drug industry is gaining importance as a significant and rewarding market in the pharmaceutical and biotech sectors. Technological and scientific innovations in specific drug discovery pathways as well as major developments in genetics are cultivating growth in this industry.
The publisher's objective in conducting this report is to provide detailed information on drug development in rare diseases. This report provides a comprehensive analysis and examines the future direction of these drugs as an important means for the treatment of orphan and rare diseases.
Companies Mentioned
- 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 Pharmaceuticals Ltd.
- Teva Pharmaceutical Industries Ltd.
