[email protected] +44 20 8123 2220 (UK) +1 732 587 5005 (US) Contact Us | FAQ |

Global Markets for Orphan Drugs

January 2024 | 189 pages | ID: G3527CFD3F4AEN
BCC Research

US$ 5,500.00

E-mail Delivery (PDF)

Download PDF Leaflet

Accepted cards
Wire Transfer
Checkout Later
Need Help? Ask a Question
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 micro%li%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.
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

LIST OF TABLES

Summary Table : Global Market for Orphan Drugs, by Product Type, Through 2028
Table 1 : Prevalence of Rare Diseases
Table 2 : Market Dynamics: Impact Analysis
Table 3 : Orphan-Designation Application Documents
Table 4 : Summary of Orphan Designation in Europe
Table 5 : Overview of Organizations and Responsibilities in Japan
Table 6 : Comparison of the Regulation of Rare Diseases and Orphan Drugs Worldwide
Table 7 : Global Market for Orphan Drugs, by Product Type, Through 2028
Table 8 : Global Market for Biological Orphan Drugs, by Region, Through 2028
Table 9 : Global Market for Non-biological Orphan Drugs, by Region, Through 2028
Table 10 : Global Market for Oncology Orphan Drugs, by Region, Through 2028
Table 11 : Global Market for Blood Disorders Orphan Drugs, by Region, Through 2028
Table 12 : Global Market for Central Nervous System Orphan Drugs, by Region, Through 2028
Table 13 : Global Market for Respiratory System Orphan Drugs, by Region, Through 2028
Table 14 : Global Market for Immunomodulator Therapies Orphan Drugs, by Region, Through 2028
Table 15 : Global Market for Cardiovascular Therapies Orphan Drugs, by Region, Through 2028
Table 16 : Global Market for Endocrine System Orphan Drugs, by Region, Through 2028
Table 17 : Global Market for Musculoskeletal System Orphan Drugs, by Region, Through 2028
Table 18 : Global Market for Other Therapeutic Applications Orphan Drugs, by Region, Through 2028
Table 19 : Global Market Shares of Orphan Drugs, by Region, 2023
Table 20 : North American Market for Orphan Drugs, by Product Type, Through 2028
Table 21 : North American Market for Orphan Drugs, by Therapeutic Application, Through 2028
Table 22 : European Market for Orphan Drugs, by Product Type, Through 2028
Table 23 : European Market for Orphan Drugs, by Therapeutic Application, Through 2028
Table 24 : Asia-Pacific Market for Orphan Drugs, by Product Type, Through 2028
Table 25 : Asia-Pacific Market for Orphan Drugs, by Therapeutic Application, Through 2028
Table 26 : Rest of the World Market for Orphan Drugs, by Product Type, Through 2028
Table 27 : Rest of the World Market for Orphan Drugs, by Therapeutic Application, Through 2028
Table 28 : ESG Ratings and Metrics
Table 29 : Environmental Performance in the Orphan Drug Market
Table 30 : Social Performance in the Orphan Drugs Market
Table 31 : Governance Performance in the Orphan Drugs Market
Table 32 : ESG Rankings for Major Orphan Drugs Companies, 2023*
Table 33 : Clinical Trials in Drugs for Rare Diseases
Table 34 : Global Market Ranking of the Top 10 Selling Orphan Drugs, 2022
Table 35 : Global by Company Market Shares of Orphan Drugs, 2022
Table 36 : AbbVie Inc.: Company Snapshot
Table 37 : AbbVie Inc.: Financial Performance, 2022
Table 38 : AbbVie Inc.: News, 2020-2022
Table 39 : AbbVie Inc.: Products
Table 40 : Astellas Pharma Inc.: Company Snapshot
Table 41 : Astellas Pharma Inc.: Financial Performance, 2023
Table 42 : Astellas Pharma Inc.: News, 2023
Table 43 : Astellas Pharma Inc.: Products
Table 44 : AstraZeneca Plc.: Company Snapshot
Table 45 : AstraZeneca Plc: Financial Performance, 2022
Table 46 : AstraZeneca Plc: News, 2022 and 2023
Table 47 : AstraZeneca Plc: Products
Table 48 : Bristol Myers Squibb: Company Snapshot
Table 49 : Bristol Myers Squibb: Financial Performance, 2022
Table 50 : Bristol Myers Squibb: News, 2021-2023
Table 51 : Bristol Myers Squibb: Products
Table 52 : Eisai Co. Ltd.: Company Snapshot
Table 53 : Eisai Co. Ltd.: Financial Performance, 2022
Table 54 : Eisai Co. Ltd.: News, 2021 and 2022
Table 55 : Eisai Co. Ltd.: Products
Table 56 : Eli Lilly and Co.: Company Snapshot
Table 57 : Eli Lilly and Co.: Financial Performance, 2022
Table 58 : Eli Lilly and Co.: Products
Table 59 : F. Hofmann-La Roche Ltd.: Company Snapshot
Table 60 : F. Hofmann-La Roche Ltd.: Financial Performance, 2022
Table 61 : F. Hofmann-La Roche Ltd.: Products
Table 62 : Merck & Co. Inc.: Company Snapshot
Table 63 : Merck & Co. Inc.: Financial Performance, 2022
Table 64 : Merck & Co. Inc.: Products
Table 65 : Novartis AG: Company Snapshot
Table 66 : Novartis AG: Financial Performance, 2022
Table 67 : Novartis AG: News, 2020-2022
Table 68 : Novartis AG: Products
Table 69 : Pfizer Inc.: Company Snapshot
Table 70 : Pfizer Inc.: Financial Performance, 2022
Table 71 : Sanofi S.A.: Company Snapshot
Table 72 : Sanofi S.A.: Financial Performance, 2022
Table 73 : Sanofi S.A.: Products
Table 74 : Takeda Pharmaceutical Co. Ltd.: Company Snapshot
Table 75 : Takeda Pharmaceutical Co. Ltd.: Financial Performance, 2022
Table 76 : Takeda Pharmaceutical Co. Ltd.: News, 2023
Table 77 : Takeda Pharmaceutical Co. Ltd.: Product Portfolio
Table 78 : Acronyms Used in This Report

LIST OF FIGURES

Summary Figure : Global Market for Orphan Drugs, by Product Type, 2020-2028
Figure 1 : Orphan Drugs Market Dynamics
Figure 2 : Orphan Designation Process, Japan
Figure 3 : Orphan Designation Consultation and Evaluation, Japan
Figure 4 : Global Market for Orphan Drugs, by Product Type, 2020-2028
Figure 5 : Global Market for Biological Orphan Drugs, by Region, 2020-2028
Figure 6 : Global Market for Non-biological Orphan Drugs, by Region, 2020-2028
Figure 7 : Global Market Shares of Orphan Drugs, by Therapeutic Application, 2023
Figure 8 : Global Market for Oncology Orphan Drugs, by Region, 2020-2028
Figure 9 : Global Market for Blood Disorders Orphan Drugs, by Region, 2020-2028
Figure 10 : Global Market for Central Nervous System Orphan Drugs, by Region, 2020-2028
Figure 11 : Global Market for Respiratory System Orphan Drugs, by Region, 2020-2028
Figure 12 : Global Market for Immunomodulator Therapies Orphan Drugs, by Region, 2020-2028
Figure 13 : Global Market for Cardiovascular Therapies Orphan Drugs, by Region, 2020-2028
Figure 14 : Global Market for Endocrine System Orphan Drugs, by Region, 2020-2028
Figure 15 : Global Market for Musculoskeletal System Orphan Drugs, by Region, 2020-2028
Figure 16 : Global Market for Other Therapeutic Applications Orphan Drugs, by Region, 2020-2028
Figure 17 : Global Market Shares of Orphan Drugs, by Region, 2023
Figure 18 : North American Market for Orphan Drugs, by Product Type, 2020-2028
Figure 19 : North American Market for Orphan Drugs, by Therapeutic Application, 2020-2028
Figure 20 : European Market for Orphan Drugs, by Product Type, 2020-2028
Figure 21 : European Market for Orphan Drugs, by Therapeutic Application, 2020-2028
Figure 22 : Asia-Pacific Market for Orphan Drugs, by Product Type, 2020-2028
Figure 23 : Asia-Pacific Market for Orphan Drugs, by Therapeutic Application, 2020-2028
Figure 24 : Rest of the World Market for Orphan Drugs, by Product Type, 2020-2028
Figure 25 : Rest of the World Market for Orphan Drugs, by Therapeutic Application, 2020-2028
Figure 26 : Conceptual Framework for Analyzing Current Strategies for Pricing Definition
Figure 27 : Global Company Market Shares of Orphan Drugs, 2022
Figure 28 : AbbVie Inc.: Financial Performance, 2021 and 2022
Figure 29 : AbbVie Inc.: Revenue Share, by Segment, 2022
Figure 30 : AbbVie Inc.: Revenue Share, by Region, 2022
Figure 31 : Astellas Pharma Inc.: Financial Performance, 2022 and 2023
Figure 32 : Astellas Pharma Inc.: Revenue Share, by Segment, 2023
Figure 33 : Astellas Pharma Inc.: Revenue Share, by Region/Country, 2023
Figure 34 : AstraZeneca Plc: Financial Performance, 2021 and 2022
Figure 35 : AstraZeneca Plc: Revenue Share, by Segment, 2022
Figure 36 : AstraZeneca Plc: Revenue Share, by Region, 2022
Figure 37 : Bristol Myers Squibb: Financial Performance, 2021 and 2022
Figure 38 : Bristol Myers Squibb: Revenue Share, by Region, 2022
Figure 39 : Eisai Co. Ltd.: Financial Performance, 2021 and 2022
Figure 40 : Eisai Co. Ltd.: Revenue Share, by Segment, 2022
Figure 41 : Eli Lilly and Co.: Financial Performance, 2021 and 2022
Figure 42 : Eli Lilly and Co.: Revenue Share, by Segment, 2022
Figure 43 : Eli Lilly and Co.: Revenue Share, by Region, 2022
Figure 44 : F. Hofmann-La Roche Ltd.: Financial Performance, 2021 and 2022
Figure 45 : F. Hofmann-La Roche Ltd.: Revenue Share, by Business Segment, 2022
Figure 46 : Merck & Co. Inc.: Financial Performance, 2021 and 2022
Figure 47 : Merck & Co. Inc.: Revenue Share, by Business Unit, 2022
Figure 48 : Merck & Co. Inc.: Revenue Share, by Region, 2022
Figure 49 : Novartis AG: Financial Performance, 2021 and 2022
Figure 50 : Novartis AG: Revenue Share, by Segment, 2022
Figure 51 : Novartis AG: Revenue Share, by Region, 2022
Figure 52 : Pfizer Inc.: Financial Performance, 2021 and 2022
Figure 53 : Pfizer Inc.: Revenue Share, by Segment, 2022
Figure 54 : Pfizer Inc.: Revenue Share, by Region, 2022
Figure 55 : Sanofi S.A.: Financial Performance, 2021 and 2022
Figure 56 : Sanofi S.A.: Revenue Share, by Segment, 2022
Figure 57 : Sanofi S.A.: Revenue Share, by Region, 2022
Figure 58 : Takeda Pharmaceutical Co. Ltd.: Financial Performance, 2021 and 2022
Figure 59 : Takeda Pharmaceutical Co. Ltd.: Revenue Share, by Business Unit, 2022
Figure 60 : Takeda Pharmaceutical Co. Ltd.: Revenue Share, by Region/Country, 2022


More Publications