全球孤儿药市场

全球孤儿药市场规模预计将从 2023 年的 2,339 亿美元成长到 2028年终的4,152 亿美元，预测期内复合年增长率为 12.2%。

生物製品产业预计将从 2023 年的 1,721 亿美元成长到年终的3,087 亿美元，复合年增长率为 12.4%。此外，非生物药品产业预计将从2023年的619亿美元成长到年终的1,065亿美元，复合年增长率为11.5%。

本报告调查了全球罕见疾病药物市场，提供了市场和技术背景、相关法律和政策资讯、市场影响因素分析、市场规模趋势和预测、各个细分市场和地区的详细分析，总结了ESG展望、定价/报销环境、临床试验趋势、竞争形势以及主要企业概况。

目录

第一章简介

第 2 章摘要/亮点

• 市场展望
• 市场摘要

第三章市场概况

• 市场与技术背景
• 按国家/地区分類的罕见疾病定义
• 美国
• EU
• 台湾
• 日本
• 澳洲
• 新加坡
• 韩国
• 瑞士
• 巴西

第四章市场动态

• 概述
• 促进因素
• 优惠孤儿药法案和类似法律
• 基因组学的技术进步
• 缺乏学名药竞争
• 专利延期
• 孤儿药的溢价
• 製造技术创新
• 合作/授权合约的增加
• 抑制因素
• 缺乏训练有素的专业人员
• 弱势目标群体
• 监管挑战
• 机会
• 庞大的新孤儿药产品平臺
• 监管状况
• 美国
• 欧洲
• 日本
• 台湾
• 韩国

第五章全球孤儿药市场：依产品类型

• 生物孤儿药
• 非生物孤儿药

第六章全球孤儿药市场：依治疗应用分类

• 瘤
• 血液疾病
• 中枢神经系统
• 呼吸系统
• 免疫调节剂
• 心血管治疗
• 内分泌系统
• 肌肉骨骼系统
• 其他的

第七章 全球孤儿药市场：按地区

• 全球市场占有率分析
• 北美洲
• 欧洲
• 亚太地区
• 其他地区

第八章 孤儿药市场的ESG前景

• ESG：简介
• ESG 在孤儿药产业的重要性
• ESG 是孤儿药产业的支柱
• ESG 评级和指标：了解资料
• 孤儿药产业的 ESG 实践
• BCC的结语

第九章 孤儿药的专营权与定价政策

• 孤儿药报销价格：当前策略和潜在改进
• 整体价值评估
• 早期对话
• 创新报销方式
• 社会参与孤儿药生产

第十章 孤儿药临床试验

第十一章竞争形势

• 全球孤儿药销售前十名
• 市场占有率分析

第十二章 公司简介

• 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 SA
• TAKEDA PHARMACEUTICAL CO. LTD.

第 13 章附录：首字母缩写词

Highlights:

The global market for orphan drugs is expected to increase from $233.9 billion in 2023 to $415.2 billion by the end of 2028, with a compound annual growth rate (CAGR) of 12.2% during the forecast period of 2023-2028.

Biological orphan drugs market for orphan drugs is expected to increase from $172.1 billion in 2023 to $308.7 billion by the end of 2028, with a CAGR of 12.4% during the forecast period of 2023-2028.

Non-biological orphan drugs market for orphan drugs is expected to increase from $61.9 billion in 2023 to $106.5 billion by the end of 2028, with a CAGR of 11.5% during the forecast period of 2023-2028.

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- 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

• 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