全球罕見疾病治療市場規模、佔有率、成長分析、按給藥途徑、按藥物 - 產業預測 (2024-2031)

2022年全球罕見疾病治療市場規模估值為1349.1億美元，從2023年的1521.8億美元成長到2031年的3988.7億美元，在預測期內（2024-2031年）預計將保持12.8%的複合年成長率。

罕見疾病治療市場是製藥業務的重要組成部分，其治療的疾病只影響少數人。這些疾病通常被稱為罕見疾病，因為它們在人口中所佔比例相對較小，而且其稀有性和複雜性給研究和開發帶來了障礙。罕見疾病的累積盛行率是巨大的，儘管它們很罕見，但它們影響著全世界數百萬人。

推動市場擴張的原因有很多，包括支持和意識的增強、遺傳和分子生物學研究的改進以及新型治療技術的創造。由於基因組學的進步，精準醫學的出現，為針對患有罕見疾病的個體的獨特遺傳特徵的標靶藥物提供了新的機會。此外，孤兒藥認定、優惠待遇等監管支援措施也鼓勵製藥業者參與罕見疾病藥物的研發。

推動該行業的另一個重要因素是學術機構、研究中心和製藥公司之間的合作。由於患者權益倡導者和製藥公司之間的官民合作關係合作和產業合作，罕見疾病的研究正在迅速進展。然而，該領域仍有許多障礙需要克服，包括開發成本高、可供臨床試驗的患者數量少、患者招募和診斷困難等。儘管如此，罕見疾病治療市場正在不斷成長，希望為最需要的人帶來改變生活的治療方法，改變那些有時被忽視的疾病影響的人的生活。這再次表明我們致力於改善我們的業務。

目錄

執行摘要

• 市場概況
• 命運之輪

調查方法

• 資訊採購
• 次要和主要資料來源
• 市場規模估算
• 市場假設與限制

母市場分析

• 市場概況
• 市場規模
• 市場動態
  • 促進因素
  • 機會
  • 抑制因素
  • 任務

主要市場考察

• 技術分析
• 價格分析
• 供應鏈分析
• 價值鏈分析
• 市場生態系統
• 智慧財產權分析
• 貿易分析
• Start-Ups分析
• 原料分析
• 創新矩陣
• 研發線產品分析
• 總體經濟指標
• 主要投資分析
• 關鍵成功因素
• 競爭程度

市場動態及展望

• 市場動態
  • 促進因素
  • 機會
  • 抑制因素
  • 任務
• 監管環境
• 波特的分析
• 對未來中斷的特殊考慮

罕見疾病治療的全球市場：依給藥途徑分類

• 市場概況
• 口服
• 注射
• 其他

全球罕見疾病治療市場：依藥物分類

• 市場概況
• 生技藥品
• 生物相似藥
• 低分子

全球罕見疾病治療市場規模：按地區分類

• 市場概況
• 北美洲
  • 美國
  • 加拿大
• 歐洲
  • 德國
  • 西班牙
  • 法國
  • 英國
  • 其他歐洲國家地區
• 亞太地區
  • 中國
  • 印度
  • 日本
  • 韓國
  • 其他亞太地區
• 拉丁美洲
  • 巴西
  • 其他拉丁美洲地區
• 中東和非洲 (MEA)
  • 海灣合作理事會國家
  • 南非
  • 其他中東/非洲地區

競爭格局

• 前5名企業對比
• 主要企業市場定位（2021年）
• 主要市場參與者所採取的策略
• 關鍵成功策略
• 近期市集活動
• 主要企業市場佔有率（2021年）

主要企業簡介

• 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