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 regional segmentation included as North America, Europe, Asia-Pacific, and Rest of the World. 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. This report also examines acquisition strategies and collaborations, discusses the market’s strengths and weaknesses considering new technologies, and analyzes the industry’s growing competition and changing customer needs.
Report Includes
- 33 data tables and 46 additional tables
- Overview and an up-to-date analysis of the global markets for orphan drugs (ODs) for rare diseases treatment
- Analyses of the global market trends, with historical market revenue data (sales figures) from 2021 to 2022, estimates for 2023, forecasts for 2024, and projections of compound annual growth rates (CAGRs) through 2028
- Highlights of the current and future market potential of orphan drugs, along with a detailed analysis of the market drivers, restraints, and opportunities
- Estimate of the actual market size and revenue forecast for the global orphan drugs market, and its corresponding market share analysis based on the product type, therapeutic area, and region
- Analysis of market growth opportunities with a holistic review of Porter’s five forces model and PESTLE analyses considering the micro
- and macroeconomic factors prevailing in the marketplace
- Discussion of sustainability trends and factors in the orphan drugs market, with emphasis on consumer attitudes, ESG score analysis, future of ESG, case study, and the ESG practices followed
- Analyses of the novel therapeutic applications of orphan drugs in rare and orphan diseases as well as examination of the regulatory framework, patents, and recent innovations in the industry
- 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
- Overview of the industry structure for orphan drugs, pricing and reimbursement policies, COVID-19 impact, and market share analysis of the leading manufacturers of orphan drugs along with their SWOT analyses
- Company profiles of major players within the industry, including AstraZeneca Plc, Eisai Co. Ltd., Bristol Myers Squibb and Pfizer Inc.
Table of Contents
Chapter 1 Introduction
Chapter 2 Summary and Highlights
Chapter 3 Market Overview
Chapter 4 Market Dynamics
Chapter 5 Global Market for Orphan Drugs by Product Type
Chapter 6 Global Market for Orphan Drugs by Therapeutic Application
Chapter 7 Global Market for Orphan Drugs by Region
Chapter 8 ESG Outlook in the Orphan Drugs Market
Chapter 9 Orphan Drugs Exclusivity and Pricing Policies
Chapter 10 Clinical Trials on Drugs for Rare Diseases
Chapter 11 Competitive Landscape
Chapter 12 Company Profiles
Chapter 13 Appendix: Acronyms
List of Tables
List of Figures
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., and Pfizer to invest in and develop orphan and rare disease therapies and drugs.In contrast to developing drugs for prevalent disorders, creating medications for orphan diseases can be more difficult. Pharmaceutical companies face several fundamental challenges when developing orphan drugs such as a lack of standard comparator drugs, a lack of clear understanding of pathophysiology or natural history, a lack of scientific evidence, and a lack of validated preclinical models. The small patient pool presents challenges that frequently lead to inadequate patient participation and impact the evaluation of clinical trial outcomes in patients with rare diseases. These patients frequently exhibit a great deal of variability in their clinical presentation and histories, with factors such as age, disease severity, and progression influencing reported outcomes.
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 sequencing 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, North America 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, is also 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 Asia-Pacific 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.
The global orphan drugs market was valued at an estimated $233.9 billion in 2023. This market is projected to reach $415.2 billion by the end of 2028, growing at a CAGR of 12.2% during the forecast period. Growth factors include the increased involvement of pharmaceutical companies in developing orphan drugs, the growing number of new approvals and the increased global availability of these drugs.
Companies Mentioned
- Abbvie Inc.
- Astellas Pharma Inc.
- Astrazeneca Plc
- Bristol Myers Squibb
- Eisai Co. Ltd.
- Eli Lilly And Co.
- F. Hoffmann-La Roche Ltd.
- Merck & Co. Inc.
- Novartis Ag
- Pfizer Inc.
- Sanofi S.A.
- Takeda Pharmaceutical Co. Ltd.
Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 189 |
| Published | January 2024 |
| Forecast Period | 2023 - 2028 |
| Estimated Market Value ( USD | $ 233.9 Billion |
| Forecasted Market Value ( USD | $ 415.2 Billion |
| Compound Annual Growth Rate | 12.2% |
| Regions Covered | Global |
| No. of Companies Mentioned | 12 |
