Global Rare Diseases Drug Development, Clinical Trials and the Regulatory Landscape Report 2021 with Analysis of Trends and Characteristics of Clinical Trials from 1999-2018

Dublin, Jan. 12, 2021 (GLOBE NEWSWIRE) -- The "Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape" report has been added to's offering.

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.

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.

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 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
  • 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

Key Topics Covered:

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

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