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孤儿药

市场调查报告书

商品编码

1663112

孤儿药市场规模、份额和成长分析（按治疗类型、适应症、药物类型、销售额、分销管道和地区）- 2025-2032 年行业预测

Orphan Drug Market Size, Share, and Growth Analysis, By Therapy Type (Oncology, Hematology), By Indication Type (Non-Hodgkin Lymphoma, Acute Myeloid Leukemia), By Drug Type, By Sale, By Distribution Channel, By Region - Industry Forecast 2025-2032

出版日期: 2025年02月19日 | 出版商:

SkyQuest | 英文 211 Pages | 商品交期: 3-5个工作天内

价格

简介目录

预计到 2023 年孤儿药市场规模将达到 1,957.6 亿美元，并从 2024 年的 2,188.6 亿美元成长到 2032 年的 5,342.0 亿美元，预测期内（2025-2032 年）的复合年增长率为 11.8%。

孤儿药市场正在获得显着的发展，特别是在 2019 年《孤儿药法案》出台之后，该法案旨在鼓励开发罕见疾病的治疗方法。世界卫生组织将罕见疾病定义为每 10,000 人中发病率不到 6.5-10 人的疾病。从历史上看，这些药物的商业化道路漫长而昂贵，从分子发现到治疗应用的成功率仅 10%。目前，全球共有超过378种孤儿药核准，其中美国占临床试验的30%以上，尤其在肿瘤领域。光是在美国就有约7,000种罕见疾病影响着2,500万至3,000万人，且每年都会发现约250种新疾病，对罕见疾病药物的需求不断增加，市场可望进一步发展。

北美洲
- 美国
- 加拿大
欧洲
- 德国
- 西班牙
- 法国
- 英国
- 义大利
- 其他欧洲国家地区
亚太地区
- 中国
- 印度
- 日本
- 韩国
- 其他亚太地区
拉丁美洲
- 巴西
- 其他拉丁美洲国家
中东和非洲
- 海湾合作委员会国家
- 南非
- 其他中东和非洲地区

竞争资讯

前 5 家公司对比
主要企业市场定位（2024年）
主要市场参与者所采用的策略
近期市场趋势
公司市场占有率分析（2024 年）
主要企业简介
- 公司详细信息
- 产品系列分析
- 公司分部份额分析
- 收益与前一年同期比较对比（2022-2024 年）

主要企业简介

Pfizer Inc.（United States）
AbbVie Inc.（United States）
Novartis AG（Switzerland）
Amgen Inc.（United States）
GSK plc（United Kingdom）
Johnson & Johnson（United States）
Sanofi（France）
F. Hoffmann-La Roche Ltd（Switzerland）
Takeda Pharmaceutical Company Limited（Japan）
Bristol-Myers Squibb Company（United States）
AstraZeneca plc（United Kingdom）
Biogen Inc.（United States）
BioMarin Pharmaceutical Inc.（United States）
Swedish Orphan Biovitrum AB（Sobi）（Sweden）
Amicus Therapeutics（United States）
Ultragenyx Pharmaceutical Inc.（United States）
PTC Therapeutics（United States）
Apellis Pharmaceuticals（United States）
Kamada Ltd.（Israel）

结论和建议

简介目录

Product Code: SQMIG30A2005

Orphan Drug Market size was valued at USD 195.76 billion in 2023 and is poised to grow from USD 218.86 billion in 2024 to USD 534.2 billion by 2032, growing at a CAGR of 11.8% during the forecast period (2025-2032).

The orphan drug market has gained significant traction, particularly following the Orphan Drug Act of 2019, which aims to spur the development of treatments for rare diseases, defined by the WHO as those affecting less than 6.5 to 10 out of 10,000 individuals. Historically, the path to commercialization of these drugs is lengthy and costly, with a mere 10% success rate from molecule discovery to therapeutic application. Currently, over 378 orphan drugs have been approved, with the U.S. leading in clinical trials, particularly in oncology, which accounts for over 30% of these efforts. With approximately 7,000 rare diseases impacting 25-30 million people in the U.S. alone-and around 250 new conditions identified annually-the demand for orphan drugs continues to rise, poised for further market evolution.

Top-down and bottom-up approaches were used to estimate and validate the size of the Orphan Drug market and to estimate the size of various other dependent submarkets. The research methodology used to estimate the market size includes the following details: The key players in the market were identified through secondary research, and their market shares in the respective regions were determined through primary and secondary research. This entire procedure includes the study of the annual and financial reports of the top market players and extensive interviews for key insights from industry leaders such as CEOs, VPs, directors, and marketing executives. All percentage shares split, and breakdowns were determined using secondary sources and verified through Primary sources. All possible parameters that affect the markets covered in this research study have been accounted for, viewed in extensive detail, verified through primary research, and analyzed to get the final quantitative and qualitative data.

Orphan Drug Market Segments Analysis

Global Orphan Drug Market is segmented by Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel and region. Based on Therapy Type, the market is segmented into Oncology, Hematology, Neurology, Endocrinology, Cardiovascular, Respiratory, Immunotherapy, Infectious Diseases and Others. Based on Indication Type, the market is segmented into Non-Hodgkin Lymphoma, Acute Myeloid Leukemia, Cystic Fibrosis, Glioma, Pancreatic Cancer, Ovarian Cancer, Multiple Myeloma, Duchenne Muscular Dystrophy, Renal Cell Carcinoma, Graft Vs Host Disease and Others. Based on Drug Type, the market is segmented into Biologics and Non-biologics. Based on Sale, the market is segmented into Generics and Prescribed. Based on Distribution Channel, the market is segmented into Hospital Pharmacy, Retail Pharmacy, Online Pharmacy and Others. Based on region, the market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

Driver of the Orphan Drug Market

The Orphan Drug market is being significantly driven by advancements in technology, particularly in genomics and gene sequencing, which have facilitated research and development for rare diseases. These innovations have paved the way for novel treatment approaches, including gene therapy and antisense therapy, allowing for targeted interventions that were once deemed unfeasible. Consequently, the combination of these technological breakthroughs has enhanced the capacity for diagnosing and treating uncommon genetic disorders, making them more manageable and attractive to pharmaceutical companies. As a result, the landscape for orphan medications is evolving, ushering in a new era of possibilities for both patients and developers.

Restraints in the Orphan Drug Market

The orphan drug market faces significant constraints primarily due to the inability of many orphan medications to meet established health technology assessment standards, especially regarding cost-effectiveness. These challenges, exacerbated by the high cost of these drugs and insufficient data supporting their value, can limit patient access significantly. The high price tags associated with these treatments often lead to scrutiny from health payers and regulators who seek value for money, making it difficult for healthcare providers to justify their use. Consequently, the combination of cost and data limitations creates substantial barriers within the orphan drug market.

Market Trends of the Orphan Drug Market

The Orphan Drug Market is experiencing a notable upward trend due to the strategic adoption of auxiliary or surrogate endpoints in clinical trials, which allows companies to demonstrate treatment effectiveness with less stringent criteria. This paradigm shift not only streamlines the research and development process but also significantly lowers time and cost investment in bringing orphan drugs to market. Consequently, pharmaceutical companies are increasingly incentivized to develop therapies for rare diseases, further propelling market growth. As regulatory bodies continue to support these innovations, the orphan drug sector is poised for continued expansion, addressing unmet medical needs while driving profitability for developers.

Introduction

Objectives of the Study
Scope of the Report
Definitions

Research Methodology

Information Procurement
Secondary & Primary Data Methods
Market Size Estimation
Market Assumptions & Limitations

Executive Summary

Global Market Outlook
Supply & Demand Trend Analysis
Segmental Opportunity Analysis

Market Dynamics & Outlook

Market Overview
Market Size
Market Dynamics
- Drivers & Opportunities
- Restraints & Challenges
Porters Analysis
- Competitive rivalry
- Threat of substitute
- Bargaining power of buyers
- Threat of new entrants
- Bargaining power of suppliers

Key Market Insights

Key Success Factors
Degree of Competition
Top Investment Pockets
Market Ecosystem
Market Attractiveness Index, 2024
PESTEL Analysis
Macro-Economic Indicators
Value Chain Analysis
Pricing Analysis

Global Orphan Drug Market Size by Therapy Type & CAGR (2025-2032)

Market Overview
Oncology
Hematology
Neurology
Endocrinology
Cardiovascular
Respiratory
Immunotherapy
Infectious Diseases
Others

Global Orphan Drug Market Size by Indication Type & CAGR (2025-2032)

Market Overview
Non-Hodgkin Lymphoma
Acute Myeloid Leukemia
Cystic Fibrosis
Glioma
Pancreatic Cancer
Ovarian Cancer
Multiple Myeloma
Duchenne Muscular Dystrophy
Renal Cell Carcinoma
Graft Vs Host Disease
Others

Global Orphan Drug Market Size by Drug Type & CAGR (2025-2032)

Market Overview
Biologics
Non-biologics

Global Orphan Drug Market Size by Sale & CAGR (2025-2032)

Market Overview
Generics
Prescribed

Global Orphan Drug Market Size by Distribution Channel & CAGR (2025-2032)

Market Overview
Hospital Pharmacy
Retail Pharmacy
Online Pharmacy
Others

Global Orphan Drug Market Size & CAGR (2025-2032)

North America (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
- US
- Canada
Europe (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
- Germany
- Spain
- France
- UK
- Italy
- Rest of Europe
Asia Pacific (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
- China
- India
- Japan
- South Korea
- Rest of Asia-Pacific
Latin America (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
- Brazil
- Rest of Latin America
Middle East & Africa (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
- GCC Countries
- South Africa
- Rest of Middle East & Africa

Competitive Intelligence

Top 5 Player Comparison
Market Positioning of Key Players, 2024
Strategies Adopted by Key Market Players
Recent Developments in the Market
Company Market Share Analysis, 2024
Company Profiles of All Key Players
- Company Details
- Product Portfolio Analysis
- Company's Segmental Share Analysis
- Revenue Y-O-Y Comparison (2022-2024)

Key Company Profiles

Pfizer Inc. (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
AbbVie Inc. (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Novartis AG (Switzerland)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Amgen Inc. (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
GSK plc (United Kingdom)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Johnson & Johnson (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Sanofi (France)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
F. Hoffmann-La Roche Ltd (Switzerland)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Takeda Pharmaceutical Company Limited (Japan)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Bristol-Myers Squibb Company (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
AstraZeneca plc (United Kingdom)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Biogen Inc. (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
BioMarin Pharmaceutical Inc. (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Swedish Orphan Biovitrum AB (Sobi) (Sweden)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Amicus Therapeutics (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Ultragenyx Pharmaceutical Inc. (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
PTC Therapeutics (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Apellis Pharmaceuticals (United States)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments
Kamada Ltd. (Israel)
- Company Overview
- Business Segment Overview
- Financial Updates
- Key Developments