+353-1-416-8900REST OF WORLD
+44-20-3973-8888REST OF WORLD
1-917-300-0470EAST COAST U.S
1-800-526-8630U.S. (TOLL FREE)

Global Markets for Orphan Drugs

  • PDF Icon

    Report

  • 128 Pages
  • July 2021
  • Region: Global
  • BCC Research
  • ID: 3644022
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
Frequently Asked Questions about the Global Orphan Drugs Market

What is the estimated value of the Global Orphan Drugs Market?

The Global Orphan Drugs Market was estimated to be valued at $190.8 Billion in 2021.

What is the growth rate of the Global Orphan Drugs Market?

The growth rate of the Global Orphan Drugs Market is 5.4%, with an estimated value of $248.2 Billion by 2026.

What is the forecasted size of the Global Orphan Drugs Market?

The Global Orphan Drugs Market is estimated to be worth $248.2 Billion by 2026.

Who are the key companies in the Global Orphan Drugs Market?

Key companies in the Global Orphan Drugs Market include ABBVIE INC., ASTELLAS PHARMA INC., ASTRAZENECA PLC, BRISTOL, MYERS SQUIBB, EISAI CO., LTD., ELI LILLY AND CO., F. HOFFMANN and GILEAD SCIENCES INC..

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

Executive Summary

Orphan drugs have paved the way for the treatment of the many unmet clinical and therapeutic needs of patients with orphan and rare diseases such as Gaucher’s disease, Pompe disease, cystic fibrosis, Hunter syndrome, tuberous sclerosis complex and chronic myelogenous leukemia. These diseases, which have different global definitions depending on their prevalence in the general population, have gained significant attention since the approval of the 1983 Orphan Drug Act (ODA) in the U.S. and the formation of organizations such as the National Organization for Rare Diseases (NORD) in 1983 and the European Organization for Rare Diseases (EURORDIS) in 1997. These developments have spurred pharmaceutical and biotech companies such as Roche, Novartis, GlaxoSmithKline, Johnson & Johnson, Bristol-Myers Squibb, Merck & Co, Pfizer, Shire and others to invest in and develop orphan and rare disease therapies and drugs.


The orphan drug market has emerged as a niche business of the pharmaceutical industry. This market’s primary growth factors include incentives for orphan drug sponsors, tax breaks, market exclusivity, grants programs for clinical studies, funds for the development of orphan products and multiple indication approvals. The unveiling of the human genetic code, which created a better understanding of human metabolism and the role of genetics in the manifestation of disease, also played a significant role in the development of the orphan drug market.


By region, the U.S. dominates the global orphan drug market due to growth factors such as advanced healthcare infrastructure and the U.S. Food and Drug Administration (FDA)-sponsored grant programs for the development of potential therapeutic compounds through its Office of Orphan Products Development (OOPD). The European market, with its well-established orphan drug legal framework, also is expanding. Growth factors include multiple post-market safety and efficacy clinical trials and the commission of potential orphan drugs through patient networks such as the European Organization for Rare Diseases (EURORDIS).


The Asian pharmaceutical market offers potential as countries such as Japan, Taiwan and South Korea have implemented legal legislations akin to the U.S.Orphan Drug Act. The main growth factors in this emerging regional market are patient awareness programs, global connectivity through rare disease patient organizations and the availability of orphan drugs through joint collaborations.


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-AVENT