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.
Report Includes:
- An overview of the drug development in rare diseases with special focus on clinical trial and regulatory landscape
- Detailed description of different kinds of orphan drugs and a comparative assessment on biological and chemical or non-biological orphan drugs
- Discussion on Orphan Drug Act; insights into general framework for the regulation of drugs and biologics, and regulations for clinical trials of orphan designated drugs
- H-ighlights 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
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. BCC Research'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.
Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape
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 BCC Research 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: A
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
Global Antioxidant Vitamins Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 100
The global Antioxidant Vitamins market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030.
North American market for Antioxidant Vitamins is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030.
Asia-Pacific market for Antioxidant Vitamins is estimated to increase from $ million in 2023 to reach $ millio......
Global Clavulanate Potassium Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 86
The global Clavulanate Potassium market was valued at US$ 446.6 million in 2023 and is anticipated to reach US$ 637.2 million by 2030, witnessing a CAGR of 5.2% during The forecast period 2024-2030.
North American market for Clavulanate Potassium is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030.
Asia-Pacific market for Clavulanate Potassium is estimated to increase from $ million in 2023......
Global Amoxicillin Sodium Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 84
The global Amoxicillin Sodium market was valued at US$ 460.9 million in 2023 and is anticipated to reach US$ 735.1 million by 2030, witnessing a CAGR of 7.0% during The forecast period 2024-2030.
North American market for Amoxicillin Sodium is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030.
Asia-Pacific market for Amoxicillin Sodium is estimated to increase from $ million in 2023 to reach......
Global Melatonin Supplements Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 106
The global Melatonin Supplements market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030.
North American market for Melatonin Supplements is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030.
Asia-Pacific market for Melatonin Supplements is estimated to increase from $ million in 2023 to reach $ mil......
Global Sleeping Pills (Prescription Drugs) Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 93
The global Sleeping Pills (Prescription Drugs) market was valued at US$ 1022.3 million in 2023 and is anticipated to reach US$ 1437.8 million by 2030, witnessing a CAGR of 4.9% during The forecast period 2024-2030.
The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during The next six years. The pharmaceutical market includes chemical drugs and biological drugs. For biologics is expected to 381 billion USD in 2022. In comparison, The chemical......
Global Sleeping Pills Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 94
The global Sleeping Pills market was valued at US$ 2042.3 million in 2023 and is anticipated to reach US$ 2979.5 million by 2030, witnessing a CAGR of 3.8% during The forecast period 2024-2030.
The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during The next six years. The pharmaceutical market includes chemical drugs and biological drugs. For biologics is expected to 381 billion USD in 2022. In comparison, The chemical drug market is estim......
Global Zinc Oral Drops Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 85
The global Zinc Oral Drops market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030.
North American market for Zinc Oral Drops is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030.
Asia-Pacific market for Zinc Oral Drops is estimated to increase from $ million in 2023 to reach $ million by 2030, at a......
Global Zinc Drops Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 85
The global Zinc Drops market was valued at US$ million in 2023 and is anticipated to reach US$ million by 2030, witnessing a CAGR of % during The forecast period 2024-2030.
North American market for Zinc Drops is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % during The forecast period of 2024 through 2030.
Asia-Pacific market for Zinc Drops is estimated to increase from $ million in 2023 to reach $ million by 2030, at a CAGR of % duri......
Global Anti-Infectives Market Research Report 2024
Published: 10-Jan-2024 Price: US 2900 Onwards Pages: 78
The global Anti-Infectives market was valued at US$ 22700 million in 2023 and is anticipated to reach US$ 27480 million by 2030, witnessing a CAGR of 2.3% during The forecast period 2024-2030.
The global pharmaceutical market is 1475 billion USD in 2022, growing at a CAGR of 5% during The next six years. The pharmaceutical market includes chemical drugs and biological drugs. For biologics is expected to 381 billion USD in 2022. In comparison, The chemical drug market is estima......
SERVICES
Value for Money
We believe in "optimum utilization of available budget and resources". While servicing our clients' (your) market research requirements, we keep the same approach in focus to help you get the best value for your $$s.
Ever Growing Inventory
Ranging from the smallest feasible / required data (datasheets, data facts, SWOT analysis, company profiles, etc) to full research reports that help you make decisions, our inventory is updated almost on a daily basis with the latest industry reports from domain experts that track more than 5000 niche sectors.
One Stop Solution
Need a custom research report on medical devices market? Require all available business intelligence on 3D printing industry? Exploring F&B sector of a particular country/region? RnRMarketResearch.com is your one-stop-solution to all market intelligence needs. We not only offer custom research and consulting services, we also "bundle" reports to meet your needs and help you fetch the data analysis you require for your business.
Dedicated Client Engagement
Not limited to only "finding" relevant reports for you, our client engagement team dedicates its efforts to understand your "business need" and accordingly maps available research data to help you move forward. Call "your" client engagement executive any time of your day and get your questions answered in order to make the correct business decision.
Saving Time and Efforts
Simply share your research requirement details with us and let us do all the hard work to find required intelligence for you. When you add up our "one stop solution" and "dedicated client engagement" services mentioned above, you obviously know the time and effort saving you do by working with us.
Payment Flexibility
Working with Fortune 500 organizations, we understand the importance of being flexible for payments. Share your payment terms with us and we will surely match up to them to ensure you get access to required business intelligence data without having to wait for the payment to be done.
Post-Purchase Research Support
Have questions after reading a report / datasheet bought through us? Not sure about the methodology used for data available in the research? Talk to us / Share your questions with us and if required, we will connect you with the analyst(s)/author(s) of the report(s) and ensure you get satisfactory answers for the same. Need more data / analysis / report(s) on the topic of your research/project? The RnRMarketResearch.com team is here for you 24X7 to support you with your post-purchase requirements.
Subscription Offers & Packages (Get in touch with us for more details -
[email protected] / +1 888 391 5441 )
- Ad Hoc
-
- Pay - as - you - go / Bucket Subscriptions
- Fixed Cost for #of reports
- Customize / Personalize as per your needs
