Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape

Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape

  • BCC Research
  • January 2021
  • Pharmaceutical
  • 107 pages

Report Description

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Report Highlights

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.

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

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.

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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

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

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