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
What is the estimated value of the Global Orphan Drugs Market?
What is the growth rate of the Global Orphan Drugs Market?
What is the forecasted size of the Global Orphan Drugs Market?
Who are the key companies in the Global Orphan Drugs Market?
| Report Attribute | Details |
|---|---|
| No. of Pages | 128 |
| Published | July 2021 |
| Forecast Period | 2021 - 2026 |
| Estimated Market Value ( USD | $ 190.8 Billion |
| Forecasted Market Value ( USD | $ 248.2 Billion |
| Compound Annual Growth Rate | 5.4% |
| Regions Covered | Global |
| No. of Companies Mentioned | 13 |
Table of Contents
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
