全球罕见疾病治疗市场规模、份额、成长分析、按给药途径、按药物 - 产业预测 (2024-2031)

2022年全球罕见疾病治疗市场规模估值为1349.1亿美元，从2023年的1521.8亿美元增长到2031年的3988.7亿美元，在预测期内（2024-2031年）预计将保持12.8%的复合年增长率。

罕见疾病治疗市场是製药业务的重要组成部分，其治疗的疾病只影响少数人。这些疾病通常被称为罕见疾病，因为它们在人口中所占比例相对较小，而且其稀有性和复杂性给研究和开发带来了障碍。罕见疾病的累积盛行率是巨大的，儘管它们很罕见，但它们影响着全世界数百万人。

推动市场扩张的原因有很多，包括支持和意识的增强、遗传和分子生物学研究的改进以及新型治疗技术的创造。由于基因组学的进步，精准医学的出现，为针对患有罕见疾病的个体的独特遗传特征的标靶药物提供了新的机会。此外，孤儿药认定、优惠待遇等监管支援措施也鼓励製药业者参与罕见疾病药物的研发。

推动该行业的另一个重要因素是学术机构、研究中心和製药公司之间的合作。由于患者权益倡导者和製药公司之间的官民合作关係合作和产业合作，罕见疾病的研究正在迅速进展。然而，该领域仍有许多障碍需要克服，包括开发成本高、可供临床试验的患者数量少、患者招募和诊断困难等。儘管如此，罕见疾病治疗市场正在不断增长，希望为最需要的人带来改变生活的治疗方法，改变那些有时被忽视的疾病影响的人的生活。这再次表明我们致力于改善我们的业务。

目录

执行摘要

• 市场概况
• 命运之轮

调查方法

• 资讯采购
• 次要和主要资料来源
• 市场规模估算
• 市场假设与限制

母市场分析

• 市场概况
• 市场规模
• 市场动态
  • 促进因素
  • 机会
  • 抑制因素
  • 任务

主要市场考察

• 技术分析
• 价格分析
• 供应链分析
• 价值链分析
• 市场生态系统
• 智慧财产权分析
• 贸易分析
• Start-Ups分析
• 原料分析
• 创新矩阵
• 研发线产品分析
• 总体经济指标
• 主要投资分析
• 关键成功因素
• 竞争程度

市场动态及展望

• 市场动态
  • 促进因素
  • 机会
  • 抑制因素
  • 任务
• 监管环境
• 波特的分析
• 对未来中断的特殊考虑

罕见疾病治疗的全球市场：依给药途径分类

• 市场概况
• 口服
• 注射
• 其他的

全球罕见疾病治疗市场：依药物分类

• 市场概况
• 生技药品
• 生物相似药
• 低分子

全球罕见疾病治疗市场规模：按地区划分

• 市场概况
• 北美洲
  • 美国
  • 加拿大
• 欧洲
  • 德国
  • 西班牙
  • 法国
  • 英国
  • 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
  • 其他拉丁美洲地区
• 中东和非洲 (MEA)
  • 海湾合作委员会国家
  • 南非
  • 其他中东/非洲地区

竞争格局

• 前5名企业对比
• 主要企业市场定位（2021年）
• 主要市场参与者所采取的策略
• 关键成功策略
• 近期市集活动
• 主要企业市场占有率（2021年）

主要企业简介

• Novartis AG
• Pfizer Inc.
• Roche Holding AG
• Sanofi SA
• Johnson & Johnson
• AbbVie Inc.
• Vertex Pharmaceuticals Incorporated
• Amgen Inc.
• AstraZeneca PLC
• Bristol Myers Squibb Company

The Global Rare Disease Treatment Market size was valued at USD 134.91 billion in 2022 and is poised to grow from USD 152.18 billion in 2023 to USD 398.87 billion by 2031, growing at a CAGR of 12.8 % in the forecast period (2024-2031).

The market for treatments for rare diseases includes an important segment of the pharmaceutical business that deals with illnesses that only a small number of people have. Since they affect a comparatively small population as a whole, these illnesses-often referred to as orphan diseases-present a barrier for research and development because of their rarity and complexity. The cumulative prevalence of rare diseases is enormous, affecting millions of people globally, despite their separate rarities.

Numerous reasons, such as growing advocacy and awareness, improvements in genetic and molecular research, and the creation of novel therapeutic techniques, are propelling the market's expansion. Thanks to advances in genomics, precision medicine has emerged, providing new opportunities for targeted medicines catered to the unique genetic profiles of individuals suffering from uncommon diseases. Additionally, pharmaceutical companies have been encouraged to participate in the development of drugs for rare diseases by supportive regulatory initiatives such orphan drug designations and incentives.

An other important factor driving the industry is cooperation among academic institutions, research centers, and pharmaceutical businesses. Research on rare diseases has advanced faster thanks to public-private partnerships and industry alliances between patient advocacy groups and pharmaceutical companies. But there are still a lot of obstacles in this sector to overcome, including the high cost of development, the small number of patients available for clinical trials, and difficulties with patient recruitment and diagnosis. In spite of this, the market for rare disease treatments is growing with the hope of providing life-altering treatments to those who require them most, reiterating the dedication to enhancing the lives of those impacted by these sometimes disregarded illnesses.

Top-down and bottom-up approaches were used to estimate and validate the size of the Global Rare Disease Treatment 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.

Global Rare Disease Treatment Market Segmental Analysis

The therapeutic area, route of administration, drug, distribution channel, and region segments make up the global market for treatments for rare diseases. The market is divided into the following therapeutic areas: cancer, hematologic disorders, infectious diseases, metabolic disorders, endocrine disorders, neurological conditions, cardiovascular conditions, musculoskeletal conditions, and others. The market is divided into oral, injectable, and other segments based on the route of administration. The market is divided into three segments based on drugs: biologics, biosimilars, and small molecules. The market is divided into three segments based on the distribution channel: online pharmacy, specialty pharmacy, and hospital pharmacy.The market is divided into four regions: North America, Europe, Asia Pacific, and LAMEA.

Drivers of the Global Rare Disease Treatment Market

One important factor is the rising funding for research and development initiatives aimed at creating novel medicines for uncommon illnesses. Developments in personalized medicine, genetics, and biotechnology are opening up new avenues for medicinal development.

Restraints in the Global Rare Disease Treatment Market

One major obstacle is the high expense of research, clinical trials, and drug development for uncommon disorders. The small patient population makes it difficult to recoup these expenses, which drives up the cost of medications.

Market Trends of the Global Rare Disease Treatment Market

Developments in Precision Medicine: This rapidly developing discipline is making it possible to design tailored medicines for particular genetic mutations or molecular abnormalities that cause uncommon diseases. This method lessens side effects while increasing treatment efficacy.

Table of Contents

Executive Summary

• Market Overview
• Wheel of Fortune

Research Methodology

• Information Procurement
• Secondary & Primary Data Sources
• Market Size Estimation
• Market Assumptions & Limitations

Parent Market Analysis

• Market Overview
• Market Size
• Market Dynamics
  • Drivers
  • Opportunities
  • Restraints
  • Challenges

Key Market Insights

• Technology Analysis
• Pricing Analysis
• Supply Chain Analysis
• Value Chain Analysis
• Ecosystem of the Market
• IP Analysis
• Trade Analysis
• Startup Analysis
• Raw Material Analysis
• Innovation Matrix
• Pipeline Product Analysis
• Macroeconomic Indicators
• Top Investment Analysis
• Key Success Factor
• Degree of Competition

Market Dynamics & Outlook

• Market Dynamics
  • Drivers
  • Opportunities
  • Restraints
  • Challenges
• Regulatory Landscape
• Porters Analysis
  • Competitive rivalry
  • Threat of Substitute Products
  • Bargaining Power of Buyers
  • Threat of New Entrants
  • Bargaining Power of Suppliers
• Skyquest Special Insights on Future Disruptions
  • Political Impact
  • Economic Impact
  • Social Impact
  • Technical Impact
  • Environmental Impact
  • Legal Impact

Global Rare Disease Treatment Market by Route of Administration

• Market Overview
• Oral
• Injectable
• and Others

Global Rare Disease Treatment Market by Drug

• Market Overview
• Biologics
• Biosimilar
• and Small Molecule

Global Rare Disease Treatment Market Size by Region

• Market Overview
• North America
  • USA
  • Canada
• Europe
  • Germany
  • Spain
  • France
  • UK
  • Rest of Europe
• Asia Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Latin America
  • Brazil
  • Rest of Latin America
• Middle East & Africa (MEA)
  • GCC Countries
  • South Africa
  • Rest of MEA

Competitive Landscape

• Top 5 Player Comparison
• Market Positioning of Key Players, 2021
• Strategies Adopted by Key Market Players
• Top Winning Strategies
  • By Development
  • By Company
  • By Year
• Recent Activities in the Market
• Key Companies Market Share (%), 2021

Key Company Profiles

• Novartis AG
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Pfizer Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Roche Holding AG
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Sanofi SA
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Johnson & Johnson
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• AbbVie Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Vertex Pharmaceuticals Incorporated
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Amgen Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• AstraZeneca PLC
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Bristol Myers Squibb Company
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments