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Market Research Report
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1025504

Global Markets for Orphan Drugs

Published: | BCC Research | 128 Pages | Delivery time: 1-2 business days

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Global Markets for Orphan Drugs
Published: July 23, 2021
BCC Research
Content info: 128 Pages
Delivery time: 1-2 business days
  • Description
  • Table of Contents
  • List of Tables

Highlights:

The global orphan drugs market should reach $248.2 billion by 2026 from $190.8 billion in 2021 at a compound annual growth rate (CAGR) of 5.4% for the forecast period of 2021 to 2026.

The biological orphan drugs segment of the global orphan drugs market is expected to grow from $141.8 billion in 2021 to $184.0 billion in 2026 at a CAGR of 5.3% for the forecast period of 2021 to 2026.

The non-biological orphan drugs segment of the global orphan drugs market is expected to grow from $48.9 billion in 2021 to $64.2 billion in 2026 at a CAGR of 5.6% for the forecast period of 2021 to 2026.

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
Product Code: PHM038F

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