Global Markets for Orphan Drugs

Global Markets for Orphan Drugs



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 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 microand 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.


Chapter 1 Introduction
Study Goals and Objectives
Reasons for Doing This Study
Scope of Report
What's New in this Update?
Methodology
Information Sources
Geographic Breakdown
Segmentation Breakdown
Chapter 2 Summary and Highlights
Market Outlook
Market Summary
Chapter 3 Market Overview
Market and Technology Background
Global Definitions of Rare Diseases by Country/Region
U.S.
EU
Taiwan
Japan
Australia
Singapore
South Korea
Switzerland
Brazil
Chapter 4 Market Dynamics
Overview
Drivers
Favorable Orphan Drug Act and Similar Legislation
Technological Advances in Genomics
Lack of Competition from Generic Drugs
Patent Extensions
Premium Pricing of Orphan Drugs
Innovations in Manufacturing Technologies
Increasing Collaborations and Licensing Agreements
Restraints
Lack of Trained Professionals
Vulnerable Target Groups
Regulatory Challenges
Opportunities
Huge Product Pipeline of New Orphan Drugs
Regulatory Landscape
U.S.
Europe
Japan
Taiwan
South Korea
Chapter 5 Global Market for Orphan Drugs by Product Type
Biological Orphan Drugs
Non-biological Orphan Drugs
Chapter 6 Global Market for Orphan Drugs by Therapeutic Application
Oncology
Blood Disorders
Central Nervous System
Respiratory System
Immunomodulators
Cardiovascular Therapies
Endocrine System
Musculoskeletal System
Others
Chapter 7 Global Market for Orphan Drugs by Region
Global Market Share Analysis
North America
Europe
Asia-Pacific
Rest of the World (RoW)
Chapter 8 ESG Outlook in the Orphan Drugs Market
Introduction to ESG
Importance of ESG in the Orphan Drug Industry
ESG Pillars in the Orphan Drugs Industry
ESG Ratings and Metrics: Understanding the Data
ESG practices in the Orphan Drug Industry
Concluding Remarks from BCC Research
Chapter 9 Orphan Drugs 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 10 Clinical Trials on Drugs for Rare Diseases
List of Clinical Trials on Drugs for Rare Diseases
Chapter 11 Competitive Landscape
Top 10 Selling Orphan Drugs Globally
Market Share Analysis
Chapter 12 Company Profiles
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.
Chapter 13 Appendix: Acronyms
Acronyms

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