美國的罕見疾病用醫藥品(孤兒藥)市場:各治療領域，各藥物類型，各流通管道，各地區，機會，預測，2018年～2032年

美國孤兒藥市場規模預計將從2024年的903.9億美元成長到2032年的1973.1億美元，在2025-2032年的預測期內，複合年增長率為10.25%。由於罕見疾病發病率的上升和診斷技術的進步，美國孤兒藥市場正在經歷強勁增長。市場獨佔權和稅收抵免等政府激勵措施正在推動醫藥創新。此外，腫瘤學仍然是最大的細分市場，人們對罕見癌症治療的日益關注加速了市場的擴張。

例如，2024年9月11日，致力於開發罕見疾病治療方法的細胞代謝和PK激活領域的領跑者Agios Pharmaceuticals, Inc.宣布，用於治療骨髓增生異常綜合徵(MDS)的創新型丙酮酸激酶(PK)激活劑tebapibat (AG-946)已獲得美國食品藥品監督管理局(FDA)的孤兒藥管理局(FDA)。

本報告提供美國的罕見疾病用醫藥品(孤兒藥)市場相關調查，提供市場概要，以及各治療領域，各藥物類型，各流通管道，各地區趨勢，及加入此市場的主要企業簡介等資訊。

目錄

第1章 計劃的範圍和定義

第2章 調查手法

第3章 美國的關稅的影響

第4章 摘要整理

第5章 美國的孤兒藥市場預測，2018年～2032年

• 市場規模分析與預測
• 市場佔有率分析與預測
  • 各治療領域
    • 腫瘤學
    • 血液學
    • 神經學
    • 內分泌學
    • 心血管系統
    • 呼吸系統
    • 免疫療法
    • 感染疾病
    • 其他
  • 各藥物類型
    • 生物製藥
    • 非生物製藥
  • 各流通管道
    • 醫院藥局
    • 零售藥局和藥妝店
    • 線上藥局
  • 各地區
    • 西北部
    • 西南部
    • 西部
    • 東南部
    • 中西部
  • 各企業市場佔有率分析(前五名公司以及其他- 各金額，2024年)
• 2024年的市場地圖分析

第6章 需求供給分析

第7章 價值鏈分析

第8章 波特的五力分析

第9章 大環境分析

第10章 價格分析

第11章 市場動態

• 市場促進因素
• 市場課題

第12章 市場趨勢與發展

第13章 法規結構和革新

• 法規當局的認證
• 臨床試驗

第14章 專利的形勢

第15章 案例研究

第16章 競爭情形

• 前五名市場領導的競爭矩陣
• 前五名參與企業的SWOT分析
• 前10名市場主要企業的形勢
  • 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 策略性建議

第18章 關於調查公司·免責聲明

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