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1748722

罕見疾病治療市場－全球產業規模、佔有率、趨勢、機會及預測，依治療領域、給藥途徑、藥物類型、配銷通路、地區及競爭情況分類，2020-2030 年預測

Rare Disease Therapeutics Market - Global Industry Size, Share, Trends, Opportunity & Forecast, Segmented By Therapeutic Area, By Route of Administration, By Drug Type, By Distribution Channel, By Region & Competition, 2020-2030F
出版日期: | 出版商: TechSci Research | 英文 185 Pages | 商品交期: 2-3個工作天內

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簡介目錄

2024 年全球罕見疾病治療市場價值為 1,210.5 億美元，預計到 2030 年將達到 2,196.6 億美元，複合年成長率為 10.40%。該市場專注於治療罕見疾病或孤兒病，這些疾病單一患者僅影響少數人群，但集體患病則影響全球數百萬人。儘管這些疾病的發生率低，但由於治療選擇有限且未滿足的需求很高，因此代表著嚴重的醫療保健問題。市場成長得益於基因研究的進步，這使得基因療法和精準醫療等先進療法得以開發。美國《孤兒藥法案》以及全球類似框架等監管激勵措施鼓勵製藥公司投資該領域。此外，透過患者權益倡導和醫療保健組織提高意識，已將罕見疾病列為優先事項，刺激了資金投入和創新。這些因素共同創造了有利於治療進步的環境，並改善了罕見疾病患者的預後。

市場概覽
預測期 2026-2030
2024年市場規模 1210.5億美元
2030年市場規模 2196.6億美元
2025-2030 年複合年成長率 10.40%
成長最快的領域 血液系統疾病
最大的市場 北美洲

關鍵市場促進因素

罕見疾病患者數量不斷增加

主要市場挑戰

準確診斷

主要市場趨勢

不斷成長的產品創新

目錄

第 1 章：產品概述

第2章：研究方法

第3章：執行摘要

第4章：顧客之聲

第5章：罕見疾病治療市場展望

  • 市場規模和預測
    • 按價值
  • 市場佔有率和預測
    • 依治療領域（血液疾病、癌症、傳染病、心血管疾病、代謝性疾病、內分泌疾病、肌肉骨骼疾病、其他）
    • 依給藥途徑（注射、口服、其他）
    • 依藥物類型（生物製劑、生物相似藥、小分子）
    • 依配銷通路（專科藥房、醫院藥房、網路藥房）
    • 按地區
    • 按公司分類（2024）
  • 市場地圖

第6章：北美罕見疾病治療市場展望

  • 市場規模和預測
  • 市場佔有率和預測
  • 北美：國家分析
    • 美國
    • 加拿大
    • 墨西哥

第7章：歐洲罕見疾病治療市場展望

  • 市場規模和預測
  • 市場佔有率和預測
  • 歐洲：國家分析
    • 德國
    • 英國
    • 義大利
    • 法國
    • 西班牙

第8章：亞太罕見疾病治療市場展望

  • 市場規模和預測
  • 市場佔有率和預測
  • 亞太地區：國家分析
    • 中國
    • 印度
    • 日本
    • 韓國
    • 澳洲

第9章：南美洲罕見疾病治療市場展望

  • 市場規模和預測
  • 市場佔有率和預測
  • 南美洲：國家分析
    • 巴西
    • 阿根廷
    • 哥倫比亞

第10章：中東和非洲罕見疾病治療市場展望

  • 市場規模和預測
  • 市場佔有率和預測
  • MEA：國家分析
    • 南非
    • 沙烏地阿拉伯
    • 阿拉伯聯合大公國

第 11 章：市場動態

  • 驅動程式
  • 挑戰

第 12 章：市場趨勢與發展

  • 最新動態
  • 產品發布
  • 併購

第 13 章：全球罕見疾病治療市場：SWOT 分析

第 14 章：競爭格局

  • Novartis AG
  • AstraZeneca PLC
  • Pfizer Inc.
  • Sanofi SA
  • AbbVie Inc
  • Bristol-Myers Squibb Co
  • Bayer AG
  • F Hoffmann-La Roche Ltd.
  • Amgen Inc
  • Eisai Co Ltd
  • Novo Nordisk A/S

第 15 章：策略建議

第16章調查會社について,免責事項

簡介目錄
Product Code: 7609

The Global Rare Disease Therapeutics Market was valued at USD 121.05 Billion in 2024 and is projected to reach USD 219.66 Billion by 2030, growing at a CAGR of 10.40%. This market focuses on treatments for rare or orphan diseases, which individually affect small populations but collectively impact millions worldwide. Despite their low prevalence, these diseases represent a significant healthcare concern due to limited treatment options and high unmet needs. The market's growth is driven by progress in genetic research, which has enabled the development of advanced therapies such as gene therapy and precision medicine. Regulatory incentives like the Orphan Drug Act in the U.S. and similar frameworks globally have encouraged pharmaceutical companies to invest in this sector. Additionally, increasing awareness through patient advocacy and healthcare organizations has elevated rare diseases as a priority, spurring funding and innovation. Together, these factors are fostering a supportive environment for therapeutic advancements and improving outcomes for individuals affected by rare diseases.

Market Overview
Forecast Period2026-2030
Market Size 2024USD 121.05 Billion
Market Size 2030USD 219.66 Billion
CAGR 2025-203010.40%
Fastest Growing SegmentHematologic diseases
Largest MarketNorth America

Key Market Drivers

Rising Number of Patients with Rare Diseases

The growing recognition of rare diseases, which number around 7,000 globally, is a significant driver for the market. Conditions such as cystic fibrosis, cat eye syndrome, and numerous rare cancers and metabolic disorders often lack effective treatments. According to the Indian Organization for Rare Diseases, a vast majority of these conditions still do not have targeted therapies. Similarly, the World Economic Forum reported in 2020 that approved treatments are available for only about 5% of known rare diseases, underscoring an enormous unmet medical need. In Europe, rare diseases affect approximately 6% to 8% of the population, amounting to nearly 30 million people. However, the individual rarity of each condition leads to limited research investment, late diagnoses, and inadequate treatment infrastructure. This "rarity paradox" highlights the mismatch between collective prevalence and resource allocation, emphasizing the need for policy reform, cross-border research collaboration, and incentivized innovation to address therapeutic gaps for this large patient population.

Key Market Challenges

Accurate Diagnosis

Accurate diagnosis remains a major barrier in rare disease management. For many patients, securing a definitive diagnosis can take up to five years due to the subtle and often non-specific nature of symptoms. Physicians may struggle to identify rare conditions, particularly those they have never encountered. In such cases, comprehensive genetic testing may be required to detect disease-related mutations. However, interpreting complex genetic data poses challenges, especially in the absence of specialists. To improve diagnostic accuracy, patients benefit most from referrals to clinical geneticists or healthcare providers with experience in managing rare diseases. Delays in diagnosis hinder timely treatment initiation and reduce eligibility for clinical trials, making early identification critical for improving patient outcomes and accelerating research.

Key Market Trends

Growing Product Innovation

The growing demand for rare disease treatments has spurred significant product innovation. Leading pharmaceutical companies are accelerating the development and global rollout of new therapies. For example, in February 2023, Sanofi announced plans to launch two orphan drugs in India: Nexviazyme (avalglucosidase alfa) for Pompe disease and Xenpozyme (olipudase alfa) for Niemann-Pick disease (ASMD). Both drugs are already approved in markets such as the U.S., EU, UK, Japan, and Australia, and have received waivers for Phase III and IV trials in India. This trend highlights a broader industry shift toward expanding access to specialized treatments in emerging markets, supported by regulatory flexibility and increased investment in research. As more therapies secure global approvals, innovation continues to reshape the landscape, offering hope for patients with previously untreatable conditions.

Key Market Players

  • Novartis AG
  • AstraZeneca PLC
  • Pfizer Inc.
  • Sanofi SA
  • AbbVie Inc
  • Bristol-Myers Squibb Co
  • Bayer AG
  • F Hoffmann-La Roche Ltd.
  • Amgen Inc
  • Eisai Co Ltd
  • Novo Nordisk A/S

Report Scope:

In this report, the Global Rare Disease Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Rare Disease Therapeutics Market, By Therapeutic Area:

  • Hematologic Diseases
  • Cancer
  • Infectious Diseases
  • Cardiovascular Diseases
  • Metabolic Diseases
  • Endocrine Diseases
  • Musculoskeletal Diseases
  • Others

Rare Disease Therapeutics Market, By Route of Administration:

  • Injectable
  • Oral
  • Others

Rare Disease Therapeutics Market, By Drug Type:

  • Biologics
  • Biosimilar
  • Small Molecules

Rare Disease Therapeutics Market, By Distribution Channel:

  • Specialty Pharmacies
  • Hospital Pharmacies
  • Online Pharmacies

Rare Disease Therapeutics Market, By Region:

  • North America
    • United States
    • Canada
    • Mexico
  • Europe
    • France
    • United Kingdom
    • Italy
    • Germany
    • Spain
  • Asia-Pacific
    • China
    • India
    • Japan
    • Australia
    • South Korea
  • South America
    • Brazil
    • Argentina
    • Colombia
  • Middle East & Africa
    • South Africa
    • Saudi Arabia
    • UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Rare Disease Therapeutics Market.

Available Customizations:

Global Rare Disease Therapeutics market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

  • Detailed analysis and profiling of additional market players (up to five).

Table of Contents

1. Product Overview

  • 1.1. Market Definition
  • 1.2. Scope of the Market
    • 1.2.1. Markets Covered
    • 1.2.2. Years Considered for Study
    • 1.2.3. Key Market Segmentations

2. Research Methodology

  • 2.1. Objective of the Study
  • 2.2. Baseline Methodology
  • 2.3. Key Industry Partners
  • 2.4. Major Association and Secondary Sources
  • 2.5. Forecasting Methodology
  • 2.6. Data Triangulation & Validation
  • 2.7. Assumptions and Limitations

3. Executive Summary

  • 3.1. Overview of the Market
  • 3.2. Overview of Key Market Segmentations
  • 3.3. Overview of Key Market Players
  • 3.4. Overview of Key Regions/Countries
  • 3.5. Overview of Market Drivers, Challenges, Trends

4. Voice of Customer

5. Rare Disease Therapeutics Market Outlook

  • 5.1. Market Size & Forecast
    • 5.1.1. By Value
  • 5.2. Market Share & Forecast
    • 5.2.1. By Therapeutic Area (Hematologic Diseases, Cancer, Infectious Diseases, Cardiovascular Diseases, Metabolic Diseases, Endocrine Diseases, Musculoskeletal Diseases, Others)
    • 5.2.2. By Route of Administration (Injectable, Oral, Others)
    • 5.2.3. By Drug Type (Biologics, Biosimilar, Small Molecules)
    • 5.2.4. By Distribution Channel (Specialty Pharmacies, Hospital Pharmacies, Online Pharmacies)
    • 5.2.5. By Region
    • 5.2.6. By Company (2024)
  • 5.3. Market Map

6. North America Rare Disease Therapeutics Market Outlook

  • 6.1. Market Size & Forecast
    • 6.1.1. By Value
  • 6.2. Market Share & Forecast
    • 6.2.1. By Therapeutic Area
    • 6.2.2. By Route of Administration
    • 6.2.3. By Drug Type
    • 6.2.4. By Distribution Channel
    • 6.2.5. By Country
  • 6.3. North America: Country Analysis
    • 6.3.1. United States Rare Disease Therapeutics Market Outlook
      • 6.3.1.1. Market Size & Forecast
        • 6.3.1.1.1. By Value
      • 6.3.1.2. Market Share & Forecast
        • 6.3.1.2.1. By Therapeutic Area
        • 6.3.1.2.2. By Route of Administration
        • 6.3.1.2.3. By Drug Type
        • 6.3.1.2.4. By Distribution Channel
    • 6.3.2. Canada Rare Disease Therapeutics Market Outlook
      • 6.3.2.1. Market Size & Forecast
        • 6.3.2.1.1. By Value
      • 6.3.2.2. Market Share & Forecast
        • 6.3.2.2.1. By Therapeutic Area
        • 6.3.2.2.2. By Route of Administration
        • 6.3.2.2.3. By Drug Type
        • 6.3.2.2.4. By Distribution Channel
    • 6.3.3. Mexico Rare Disease Therapeutics Market Outlook
      • 6.3.3.1. Market Size & Forecast
        • 6.3.3.1.1. By Value
      • 6.3.3.2. Market Share & Forecast
        • 6.3.3.2.1. By Therapeutic Area
        • 6.3.3.2.2. By Route of Administration
        • 6.3.3.2.3. By Drug Type
        • 6.3.3.2.4. By Distribution Channel

7. Europe Rare Disease Therapeutics Market Outlook

  • 7.1. Market Size & Forecast
    • 7.1.1. By Value
  • 7.2. Market Share & Forecast
    • 7.2.1. By Therapeutic Area
    • 7.2.2. By Route of Administration
    • 7.2.3. By Drug Type
    • 7.2.4. By Distribution Channel
    • 7.2.5. By Country
  • 7.3. Europe: Country Analysis
    • 7.3.1. Germany Rare Disease Therapeutics Market Outlook
      • 7.3.1.1. Market Size & Forecast
        • 7.3.1.1.1. By Value
      • 7.3.1.2. Market Share & Forecast
        • 7.3.1.2.1. By Therapeutic Area
        • 7.3.1.2.2. By Route of Administration
        • 7.3.1.2.3. By Drug Type
        • 7.3.1.2.4. By Distribution Channel
    • 7.3.2. United Kingdom Rare Disease Therapeutics Market Outlook
      • 7.3.2.1. Market Size & Forecast
        • 7.3.2.1.1. By Value
      • 7.3.2.2. Market Share & Forecast
        • 7.3.2.2.1. By Therapeutic Area
        • 7.3.2.2.2. By Route of Administration
        • 7.3.2.2.3. By Drug Type
        • 7.3.2.2.4. By Distribution Channel
    • 7.3.3. Italy Rare Disease Therapeutics Market Outlook
      • 7.3.3.1. Market Size & Forecast
        • 7.3.3.1.1. By Value
      • 7.3.3.2. Market Share & Forecast
        • 7.3.3.2.1. By Therapeutic Area
        • 7.3.3.2.2. By Route of Administration
        • 7.3.3.2.3. By Drug Type
        • 7.3.3.2.4. By Distribution Channel
    • 7.3.4. France Rare Disease Therapeutics Market Outlook
      • 7.3.4.1. Market Size & Forecast
        • 7.3.4.1.1. By Value
      • 7.3.4.2. Market Share & Forecast
        • 7.3.4.2.1. By Therapeutic Area
        • 7.3.4.2.2. By Route of Administration
        • 7.3.4.2.3. By Drug Type
        • 7.3.4.2.4. By Distribution Channel
    • 7.3.5. Spain Rare Disease Therapeutics Market Outlook
      • 7.3.5.1. Market Size & Forecast
        • 7.3.5.1.1. By Value
      • 7.3.5.2. Market Share & Forecast
        • 7.3.5.2.1. By Therapeutic Area
        • 7.3.5.2.2. By Route of Administration
        • 7.3.5.2.3. By Drug Type
        • 7.3.5.2.4. By Distribution Channel

8. Asia-Pacific Rare Disease Therapeutics Market Outlook

  • 8.1. Market Size & Forecast
    • 8.1.1. By Value
  • 8.2. Market Share & Forecast
    • 8.2.1. By Therapeutic Area
    • 8.2.2. By Route of Administration
    • 8.2.3. By Drug Type
    • 8.2.4. By Distribution Channel
    • 8.2.5. By Country
  • 8.3. Asia-Pacific: Country Analysis
    • 8.3.1. China Rare Disease Therapeutics Market Outlook
      • 8.3.1.1. Market Size & Forecast
        • 8.3.1.1.1. By Value
      • 8.3.1.2. Market Share & Forecast
        • 8.3.1.2.1. By Therapeutic Area
        • 8.3.1.2.2. By Route of Administration
        • 8.3.1.2.3. By Drug Type
        • 8.3.1.2.4. By Distribution Channel
    • 8.3.2. India Rare Disease Therapeutics Market Outlook
      • 8.3.2.1. Market Size & Forecast
        • 8.3.2.1.1. By Value
      • 8.3.2.2. Market Share & Forecast
        • 8.3.2.2.1. By Therapeutic Area
        • 8.3.2.2.2. By Route of Administration
        • 8.3.2.2.3. By Drug Type
        • 8.3.2.2.4. By Distribution Channel
    • 8.3.3. Japan Rare Disease Therapeutics Market Outlook
      • 8.3.3.1. Market Size & Forecast
        • 8.3.3.1.1. By Value
      • 8.3.3.2. Market Share & Forecast
        • 8.3.3.2.1. By Therapeutic Area
        • 8.3.3.2.2. By Route of Administration
        • 8.3.3.2.3. By Drug Type
        • 8.3.3.2.4. By Distribution Channel
    • 8.3.4. South Korea Rare Disease Therapeutics Market Outlook
      • 8.3.4.1. Market Size & Forecast
        • 8.3.4.1.1. By Value
      • 8.3.4.2. Market Share & Forecast
        • 8.3.4.2.1. By Therapeutic Area
        • 8.3.4.2.2. By Route of Administration
        • 8.3.4.2.3. By Drug Type
        • 8.3.4.2.4. By Distribution Channel
    • 8.3.5. Australia Rare Disease Therapeutics Market Outlook
      • 8.3.5.1. Market Size & Forecast
        • 8.3.5.1.1. By Value
      • 8.3.5.2. Market Share & Forecast
        • 8.3.5.2.1. By Therapeutic Area
        • 8.3.5.2.2. By Route of Administration
        • 8.3.5.2.3. By Drug Type
        • 8.3.5.2.4. By Distribution Channel

9. South America Rare Disease Therapeutics Market Outlook

  • 9.1. Market Size & Forecast
    • 9.1.1. By Value
  • 9.2. Market Share & Forecast
    • 9.2.1. By Therapeutic Area
    • 9.2.2. By Route of Administration
    • 9.2.3. By Drug Type
    • 9.2.4. By Distribution Channel
    • 9.2.5. By Country
  • 9.3. South America: Country Analysis
    • 9.3.1. Brazil Rare Disease Therapeutics Market Outlook
      • 9.3.1.1. Market Size & Forecast
        • 9.3.1.1.1. By Value
      • 9.3.1.2. Market Share & Forecast
        • 9.3.1.2.1. By Therapeutic Area
        • 9.3.1.2.2. By Route of Administration
        • 9.3.1.2.3. By Drug Type
        • 9.3.1.2.4. By Distribution Channel
    • 9.3.2. Argentina Rare Disease Therapeutics Market Outlook
      • 9.3.2.1. Market Size & Forecast
        • 9.3.2.1.1. By Value
      • 9.3.2.2. Market Share & Forecast
        • 9.3.2.2.1. By Therapeutic Area
        • 9.3.2.2.2. By Route of Administration
        • 9.3.2.2.3. By Drug Type
        • 9.3.2.2.4. By Distribution Channel
    • 9.3.3. Colombia Rare Disease Therapeutics Market Outlook
      • 9.3.3.1. Market Size & Forecast
        • 9.3.3.1.1. By Value
      • 9.3.3.2. Market Share & Forecast
        • 9.3.3.2.1. By Therapeutic Area
        • 9.3.3.2.2. By Route of Administration
        • 9.3.3.2.3. By Drug Type
        • 9.3.3.2.4. By Distribution Channel

10. Middle East and Africa Rare Disease Therapeutics Market Outlook

  • 10.1. Market Size & Forecast
    • 10.1.1. By Value
  • 10.2. Market Share & Forecast
    • 10.2.1. By Therapeutic Area
    • 10.2.2. By Route of Administration
    • 10.2.3. By Drug Type
    • 10.2.4. By Distribution Channel
    • 10.2.5. By Country
  • 10.3. MEA: Country Analysis
    • 10.3.1. South Africa Rare Disease Therapeutics Market Outlook
      • 10.3.1.1. Market Size & Forecast
        • 10.3.1.1.1. By Value
      • 10.3.1.2. Market Share & Forecast
        • 10.3.1.2.1. By Therapeutic Area
        • 10.3.1.2.2. By Route of Administration
        • 10.3.1.2.3. By Drug Type
        • 10.3.1.2.4. By Distribution Channel
    • 10.3.2. Saudi Arabia Rare Disease Therapeutics Market Outlook
      • 10.3.2.1. Market Size & Forecast
        • 10.3.2.1.1. By Value
      • 10.3.2.2. Market Share & Forecast
        • 10.3.2.2.1. By Therapeutic Area
        • 10.3.2.2.2. By Route of Administration
        • 10.3.2.2.3. By Drug Type
        • 10.3.2.2.4. By Distribution Channel
    • 10.3.3. UAE Rare Disease Therapeutics Market Outlook
      • 10.3.3.1. Market Size & Forecast
        • 10.3.3.1.1. By Value
      • 10.3.3.2. Market Share & Forecast
        • 10.3.3.2.1. By Therapeutic Area
        • 10.3.3.2.2. By Route of Administration
        • 10.3.3.2.3. By Drug Type
        • 10.3.3.2.4. By Distribution Channel

11. Market Dynamics

  • 11.1. Drivers
  • 11.2. Challenges

12. Market Trends & Developments

  • 12.1. Recent Developments
  • 12.2. Product Launches
  • 12.3. Mergers & Acquisitions

13. Global Rare Disease Therapeutics Market: SWOT Analysis

14. Competitive Landscape

  • 14.1. Novartis AG
    • 14.1.1. Business Overview
    • 14.1.2. Product & Service Offerings
    • 14.1.3. Recent Developments
    • 14.1.4. Financials (If Listed)
    • 14.1.5. Key Personnel
    • 14.1.6. SWOT Analysis
  • 14.2. AstraZeneca PLC
  • 14.3. Pfizer Inc.
  • 14.4. Sanofi SA
  • 14.5. AbbVie Inc
  • 14.6. Bristol-Myers Squibb Co
  • 14.7. Bayer AG
  • 14.8. F Hoffmann-La Roche Ltd.
  • 14.9. Amgen Inc
  • 14.10.Eisai Co Ltd
  • 14.11.Novo Nordisk A/S

15. Strategic Recommendations

16. About Us & Disclaimer