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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
Summary:
Orphan drugs have paved theway for the treatment of the many unmet clinical and therapeuticneeds of patientswith 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 globaldefinitions depending on theirprevalence in the general population, have gained significant attention since the approval of the 1983 OrphanDrug Act (ODA) in the U.S. and the formation of organizations such as the National Organization forRare Diseases (NORD) in 1983 and the EuropeanOrganization forRare 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 andmultiple indication approvals. The unveiling of the human genetic code,which created a betterunderstanding of humanmetabolismand the role of genetics in the manifestation of disease, also played a significant role in the development of the orphan drugmarket.
By region, the U.S. dominates the global orphan drugmarket 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 itsOffice ofOrphan Products Development (OOPD). The Europeanmarket,with itswell-established orphan drug legal framework, also is expanding.Growth factors include multiple post-market safety and efficacy clinical trials and the commission of potentialorphan drugs through patient networks such as the EuropeanOrganization for Rare Diseases (EURORDIS).
The Asian pharmaceutical market offers potentialas countries such as Japan, Taiwan and South Korea have implemented legal legislations akin to the U.S.OrphanDrug 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 drug market reached REDACTED in 2020. This market should reach REDACTED and REDACTED in 2021 and 2026, respectively, achieving aCAGRof REDACTED during the forecast period. Growth factors include the increased involvement of pharmaceutical companies in developing orphan drugs, the growing numberof newapprovals and the increased global availability of these drugs.
The biological orphan drug market reached REDACTED in 2020. This market is expected to reach REDACTED and REDACTED by 2021 and 2026, respectively, achieving a CAGRof REDACTED during the forecast period.Multiple orphan approvals for pre-existing drugs should boost this growth. The non-biological orphan drugmarket reached REDACTED in 2020. This market should reach REDACTED by 2026, growing at a CAGRof REDACTED during the forec ast period.