美国的罕见疾病用医药品(孤儿药)市场:各治疗领域，各药物类型，各流通管道，各地区，机会，预测，2018年～2032年

美国孤儿药市场规模预计将从2024年的903.9亿美元成长到2032年的1973.1亿美元，在2025-2032年的预测期内，复合年增长率为10.25%。由于罕见疾病发病率的上升和诊断技术的进步，美国孤儿药市场正在经历强劲增长。市场独占权和税收抵免等政府激励措施正在推动医药创新。此外，肿瘤学仍然是最大的细分市场，人们对罕见癌症治疗的日益关注加速了市场的扩张。

例如，2024年9月11日，致力于开发罕见疾病治疗方法的细胞代谢和PK激活领域的领跑者Agios Pharmaceuticals, Inc.宣布，用于治疗骨髓增生异常综合征(MDS)的创新型丙酮酸激酶(PK)激活剂tebapibat (AG-946)已获得美国食品药品监督管理局(FDA)的孤儿药管理局(FDA)。

本报告提供美国的罕见疾病用医药品(孤儿药)市场相关调查，提供市场概要，以及各治疗领域，各药物类型，各流通管道，各地区趋势，及加入此市场的主要企业简介等资讯。

目录

第1章 计划的范围和定义

第2章 调查手法

第3章 美国的关税的影响

第4章 摘要整理

第5章 美国的孤儿药市场预测，2018年～2032年

• 市场规模分析与预测
• 市场占有率分析与预测
  • 各治疗领域
    • 肿瘤学
    • 血液学
    • 神经学
    • 内分泌学
    • 心血管系统
    • 呼吸系统
    • 免疫疗法
    • 感染疾病
    • 其他
  • 各药物类型
    • 生物製药
    • 非生物製药
  • 各流通管道
    • 医院药局
    • 零售药局和药妆店
    • 线上药局
  • 各地区
    • 西北部
    • 西南部
    • 西部
    • 东南部
    • 中西部
  • 各企业市场占有率分析(前五名公司以及其他- 各金额，2024年)
• 2024年的市场地图分析

第6章 需求供给分析

第7章 价值链分析

第8章 波特的五力分析

第9章 大环境分析

第10章 价格分析

第11章 市场动态

• 市场促进因素
• 市场课题

第12章 市场趋势与发展

第13章 法规结构和革新

• 法规当局的认证
• 临床试验

第14章 专利的形势

第15章 案例研究

第16章 竞争情形

• 前五名市场领导的竞争矩阵
• 前五名参与企业的SWOT分析
• 前10名市场主要企业的形势
  • Novartis AG
  • Amgen, Inc.
  • Bristol-Myers Squibb Company
  • AstraZeneca plc
  • Pfizer Inc.
  • Novo Nordisk A/S
  • Sanofi S.A.
  • Johnson & Johnson Services, Inc.
  • F. Hoffmann-La Roche Ltd
  • Vertex Pharmaceuticals Incorporated

第17章 策略性建议

第18章 关于调查公司·免责声明

United States orphan drugs market is projected to witness a CAGR of 10.25% during the forecast period 2025-2032, growing from USD 90.39 billion in 2024 to USD 197.31 billion in 2032. The U.S. orphan drugs market is witnessing strong growth due to rising rare disease prevalence and enhanced diagnostics. Government incentives like market exclusivity and tax credits are encouraging pharmaceutical innovation. Additionally, oncology remains the largest segment, with increasing focus on rare cancer therapies accelerating market expansion.

For instance, on September 11, 2024, Agios Pharmaceuticals, Inc., a frontrunner in cellular metabolism and PK activation dedicated to developing therapies for rare diseases, announced that the U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to its innovative pyruvate kinase (PK) activator, tebapivat (AG-946), intended for the treatment of myelodysplastic syndromes (MDS).

Rising Prevalence of Rare Diseases Fueling Demand for Orphan Drugs

The rising incidence of rare diseases in the United States has markedly increased the demand for orphan drugs. The National Institutes of Health (NIH) reports that around 7,000 rare diseases impact more than 30 million Americans. Many of these conditions lack adequate treatment options, leading to a significant unmet need for specialized therapeutics. Orphan drugs are designed specifically for these diseases, and their market has expanded due to heightened awareness, earlier diagnoses, and advancements in genetic testing. Additionally, the U.S. Food and Drug Administration (FDA) has introduced various incentives, including tax credits, market exclusivity, and accelerated approval processes, to promote the development of orphan drugs. These measures have led to a rise in research and development investments from pharmaceutical companies. For instance, in February 2024, Amgen received FDA approval for BLINCYTO (blinatumomab), an orphan drug for the treatment of minimal residual disease (MRD) in acute lymphoblastic leukemia (ALL) patients. This approval not only adds to Amgen's oncology pipeline but also highlights the industry's growing commitment to addressing rare disease burdens.

Government Incentives and Regulatory Support Accelerate Orphan Drug Approvals

The proactive regulatory framework established by the U.S. government significantly drives the orphan drug market. The Orphan Drug Act of 1983 provided essential regulatory and financial incentives, including seven years of market exclusivity, research grants, and tax credits for clinical trials. These advantages motivate biotech and pharmaceutical companies to pursue the development of treatments for rare diseases, even with limited patient populations. The FDA's Office of Orphan Products Development (OOPD) is instrumental in accelerating these therapies' review and approval processes through initiatives such as Fast Track, Breakthrough Therapy, and Priority Review designations. In recent years, this supportive environment has resulted in a notable increase in orphan drug designations and approvals. A notable example is when Acadia Pharmaceuticals Inc. had declared in the U.S. in March 2023, that the U.S. Food and Drug Administration (FDA) had approved DAYBU (trofinetide) to treat Rett syndrome in both adult and pediatric patients aged two years and older. DAYBUE is the sole medication authorized to treat Rett syndrome. The approval underlines how streamlined pathways and strong regulatory backing are helping more orphan drugs reach patients faster, boosting innovation and accessibility in the U.S. market.

Oncology Segment Dominates with Increasing Development of Rare Cancer Therapies

Oncology represents the largest U.S. orphan drugs market segment among various therapeutic areas. The increasing prevalence of rare cancers, including soft tissue sarcomas, gliomas, and specific blood malignancies, has notably heightened the demand for specialized treatment options. Pharmaceutical firms are sharpening their focus on developing targeted therapies and immuno-oncology medications that provide enhanced effectiveness and reduced side effects compared to traditional treatments. The robust presence of biotechnology companies, investments in precision medicine, and a growing pipeline of clinical trials in the U.S. further bolstered this sector's growth. Additionally, the orphan drug designation for oncology products frequently results in expedited regulatory approvals, lower development expenses, and prolonged market exclusivity, rendering it a highly profitable domain. For instance, in August 2023, Bristol Myers Squibb received FDA approval for REBLOZYL (luspatercept-aamt) as a first-line treatment for anemia in adults with lower-risk myelodysplastic syndromes, which includes certain rare cancers. This approval under the Orphan Drug Act highlights the market's strong momentum in rare oncology therapeutics.

Future Market Scenario (2025-2032F)

The orphan drugs market in the United States is set for significant expansion, driven by a growing awareness of rare diseases, favorable regulatory frameworks, and vigorous research and development efforts by pharmaceutical firms. Innovations in genomics and personalized medicine facilitate the discovery of new therapeutic targets, while expedited FDA approval processes are shortening the time required for orphan-designated drugs to reach the market. Additionally, the increasing presence of patient advocacy organizations and advancements in diagnostics are enhancing access to therapies for rare diseases. The prevalence of biologics and heightened investments in gene and cell therapies will likely transform the treatment landscape. With major industry players actively working on innovative pipeline projects, the market is expected to maintain its momentum, providing renewed hope for underrepresented patient populations across diverse therapeutic fields.

Key Players Landscape and Outlook

The key players in the market are significantly investing in the development of orphan drugs and are utilizing strategies such as mergers, acquisitions, partnerships, and new product launches to improve their services and competitiveness. Such efforts will propel significant growth in the market, allowing large-cap industry players to increase their presence and, therefore, find new opportunities in this market.

For instance, Rapid Commercialization Partners (RCP) has joined forces with Orphan Now to assist companies in accelerating the development of orphan drugs. This collaboration seeks to facilitate market entry, thereby providing quicker access to essential treatments for patients globally. The initiative improves the efficiency of commercialization, offering advantages to developers of drugs for rare diseases.

Table of Contents

1. Project Scope and Definitions

2. Research Methodology

3. Impact of U.S. Tariffs

4. Executive Summary

5. United States Orphan Drugs Market Outlook, 2018-2032F

• 5.1. Market Size Analysis & Forecast
  • 5.1.1. By Value
• 5.2. Market Share Analysis & Forecast
  • 5.2.1. By Therapy Area
    • 5.2.1.1. Oncology
    • 5.2.1.2. Hematology
    • 5.2.1.3. Neurology
    • 5.2.1.4. Endocrinology
    • 5.2.1.5. Cardiovascular
    • 5.2.1.6. Respiratory
    • 5.2.1.7. Immunotherapy
    • 5.2.1.8. Infectious Diseases
    • 5.2.1.9. Others
  • 5.2.2. By Drug Type
    • 5.2.2.1. Biologics
    • 5.2.2.2. Non-Biologics
  • 5.2.3. By Distribution Channel
    • 5.2.3.1. Hospital Pharmacies
    • 5.2.3.2. Retail Pharmacies and Drug Stores
    • 5.2.3.3. Online Pharmacies
  • 5.2.4. By Region
    • 5.2.4.1. Northwest
    • 5.2.4.2. Southwest
    • 5.2.4.3. West
    • 5.2.4.4. Southeast
    • 5.2.4.5. Midwest
  • 5.2.5. By Company Market Share Analysis (Top 5 Companies and Others - By Value, 2024)
• 5.3. Market Map Analysis, 2024
  • 5.3.1. By Therapy Area
  • 5.3.2. By Drug Type
  • 5.3.3. By Distribution Channel
  • 5.3.4. By Region

6. Demand Supply Analysis

7. Value Chain Analysis

8. Porter's Five Forces Analysis

9. PESTLE Analysis

10. Pricing Analysis

11. Market Dynamics

• 11.1. Market Drivers
• 11.2. Market Challenges

12. Market Trends and Developments

13. Regulatory Framework and Innovation

• 13.1. Regulatory Approvals
• 13.2. Clinical Trials

14. Patent Landscape

15. Case Studies

16. Competitive Landscape

• 16.1. Competition Matrix of Top 5 Market Leaders
• 16.2. SWOT Analysis for Top 5 Players
• 16.3. Key Players Landscape for Top 10 Market Players
  • 16.3.1. Novartis AG
    • 16.3.1.1. Company Details
    • 16.3.1.2. Key Management Personnel
    • 16.3.1.3. Products and Services
    • 16.3.1.4. Financials (As Reported)
    • 16.3.1.5. Key Market Focus and Geographical Presence
    • 16.3.1.6. Recent Developments/Collaborations/Partnerships/Mergers and Acquisitions
  • 16.3.2. Amgen, Inc.
  • 16.3.3. Bristol-Myers Squibb Company
  • 16.3.4. AstraZeneca plc
  • 16.3.5. Pfizer Inc.
  • 16.3.6. Novo Nordisk A/S
  • 16.3.7. Sanofi S.A.
  • 16.3.8. Johnson & Johnson Services, Inc.
  • 16.3.9. F. Hoffmann-La Roche Ltd
  • 16.3.10. Vertex Pharmaceuticals Incorporated

Companies mentioned above DO NOT hold any order as per market share and can be changed as per information available during research work.

17. Strategic Recommendations

18. About Us and Disclaimer

List of Tables

• Table 1. Competition Matrix of Top 5 Market Leaders
• Table 2. Mergers & Acquisitions/ Joint Ventures (If Applicable)
• Table 3. About Us - Regions and Countries Where We Have Executed Client Projects

List of Figures

• Figure 1. United States Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 2. United States Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 3. United States Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 4. United States Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 5. United States Orphan Drugs Market Share (%), By Region, 2018-2032F
• Figure 6. By Therapy Area Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 7. By Drug Type Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 8. By Distribution Channel Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 9. By Region Map-Market Size (USD Billion) & Growth Rate (%), 2024