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罕見疾病治療市場規模、佔有率、趨勢分析報告：依治療領域、給藥途徑、藥物類型、通路、地區、細分市場預測，2025-2030年

Rare Diseases Treatment Market Size, Share & Trends Analysis Report By Therapeutic Area (Cancer, Musculoskeletal Conditions), By Route Of Administration, By Drug Type, By Distribution Channel, By Region, And Segment Forecasts, 2025 - 2030
出版日期: | 出版商: Grand View Research | 英文 150 Pages | 商品交期: 2-10個工作天內

價格

罕見疾病治療市場成長與趨勢：

Grand View Research, Inc.即將完成的一份新報告顯示，預計2025年至2030年全球罕見疾病治療市場將以11.6%的複合年成長率成長，到2030年將達到3743.9億美元。

罕見疾病的盛行率不斷上升，促進了該行業的成長。根據 NCATS（國家轉化科學促進中心）估計，到 2022 年，美國將有超過 3,000 萬人受到罕見疾病的影響。罕見疾病患者數量的增加預計將在預測期內增加孤兒藥的銷售和需求。

政府機構支持性法規的存在，例如《孤兒藥法案》，透過為製藥公司開發的潛在候選藥物提供孤兒藥指定來協助產品開發，預計將支持孤兒藥的成長。該舉措將支持罕見疾病治療領域的研究和開發。

2020年4月，美國罕見疾病組織（NORD）針對受COVID-19大流行影響的罕見疾病患者啟動了「COVID-19危重症救助」計畫。根據該計劃，NORD 每年提供高達 1,000 美元的財政支持，以滿足罕見疾病患者及其看護者的醫療和非醫療需求。因此，這些計劃的可用性預計將在預測期內推動罕見疾病治療的採用。

此外，公司也採取聯盟和夥伴關係等舉措，在國際市場上開發、製造和商業化產品。例如，2020年8月，Sarepta Therapeutics, Inc.開始與佛羅裡達大學進行研究合作，開發新型基因藥物，用於治療包括Duchenne氏肌肉失養症（DMD）在內的各種罕見疾病患者。

然而，缺乏對罕見疾病的準確和早期診斷往往是罕見疾病患者面臨的挑戰。此外，罕見疾病治療臨床試驗的合格和招募合格患者也可能限制產業成長。不僅很難找到符合條件的患者，而且尋找治療這些患者的醫生也很複雜，導致新產品核准和市場推出的延遲。

罕見疾病治療市場報告亮點

  • 從治療領域來看，多發性骨髓瘤、急性淋巴性白血病、慢性骨髓性白血病和食道癌等癌症適應症的盛行率很高，並且由於藥物、腫瘤學的廣泛使用，罕見疾病療法被用於治療這些疾病的患者。
  • 從給藥途徑來看，注射劑在2024 年將佔最大佔有率，達到65.5%，由於這種給藥途徑在罕見疾病患者中具有較高的安全性、有效性和耐受性，預計將出現顯著成長。
  • 從藥物類型來看，生物製藥佔據市場主導地位，2024 年佔有率最大，為 58.1%。這是由於支持性報銷政策的存在以及生物製藥作為疾病修正治療在罕見疾病治療中的高效能。
  • 從分銷管道來看，由於政府支持專科藥局提供罕見疾病藥物的舉措，專科藥局將在 2024 年佔據市場最大佔有率，佔有率為 70.5%。例如，2022 年 7 月，利用審查和認證委員會 (URAC) 授予 Maxor Specialty Pharmacy 罕見疾病藥房卓越中心稱號，以表彰其為患者提供高品質的罕見疾病治療。
  • 預計亞太地區在預測期內將出現良好的成長。該地區的成長得益於新產品的進入該地區。據疾病挑戰基金會稱，截至2021年終，中國已核准87種新的罕見疾病治療藥物，治療47種罕見疾病。

目錄

第1章調查方法和範圍

第 2 章執行摘要

第3章 罕見疾病治療市場的變數、趨勢和範圍

  • 市場體系展望
    • 母市場展望
    • 相關/附隨市場展望
  • 市場動態
    • 市場促進因素分析
    • 市場限制因素分析
  • 罕見疾病治療市場分析工具
    • 產業分析-波特五力分析
    • PESTEL分析

第4章 罕見疾病治療市場：治療領域預估及趨勢分析

  • 細分儀表板
  • 罕見疾病治療市場：治療區域波動分析
  • 罕見疾病治療市場：依治療領域分類的前景
  • 2018-2030年市場規模/預測與趨勢分析
  • 癌症
  • 神經系統疾病
  • 心血管疾病
  • 肌肉骨骼疾病
  • 血液疾病
  • 感染疾病
  • 代謝紊亂
  • 內分泌疾病
  • 其他

第5章 罕見疾病治療市場：醫藥預測與趨勢分析

  • 細分儀表板
  • 罕見疾病治療市場：藥品波動分析
  • 罕見疾病治療市場：依藥物分類的前景
    • 2018-2030年市場規模/預測與趨勢分析
  • 生物製藥
  • 生物相似藥
  • 小分子

第6章 罕見疾病治療市場：給藥途徑預估及趨勢分析

  • 細分儀表板
  • 罕見疾病治療市場：給藥途徑變化分析
  • 罕見疾病治療市場：依給藥途徑分類的前景
    • 2018-2030年市場規模/預測與趨勢分析
  • 口服
  • 可注射的
  • 其他

第7章 罕見疾病治療市場：通路估算及趨勢分析

  • 細分儀表板
  • 罕見疾病治療市場：通路波動分析
  • 罕見疾病治療市場：按分銷管道分類的前景
    • 2018-2030年市場規模/預測與趨勢分析
  • 醫院藥房
  • 專業藥房
  • 網路藥房

第8章 罕見疾病治療市場：區域估算及趨勢分析

  • 區域儀表板
  • 2018-2030年市場規模、預測趨勢分析：
  • 北美洲
    • 美國
    • 加拿大
    • 墨西哥
  • 歐洲
    • 英國
    • 德國
    • 法國
    • 義大利
    • 西班牙
    • 挪威
    • 瑞典
    • 丹麥
  • 亞太地區
    • 日本
    • 中國
    • 印度
    • 澳洲
    • 韓國
    • 泰國
  • 拉丁美洲
    • 巴西
    • 阿根廷
  • 中東/非洲
    • 南非
    • 沙烏地阿拉伯
    • 阿拉伯聯合大公國
    • 科威特

第9章 競爭格局

  • 公司分類
  • 主要市場參與企業的最新趨勢和影響分析
  • 2024 年企業市場佔有率分析
  • 公司簡介
    • F. Hoffmann-La Roche Ltd.
    • Pfizer, Inc.
    • PTC Therapeutics Inc.
    • AstraZeneca
    • Novartis AG
    • Takeda Pharmaceutical Company Ltd
    • Bayer AG
    • AbbVie Inc.
    • Merck & Co. Inc.
    • Bristol Myers Squibb Company
Product Code: GVR-4-68039-965-6

Rare Diseases Treatment Market Growth & Trends:

The global rare disease treatment market size is estimated to reach USD 374.39 billion by 2030, expanding at a CAGR of 11.6% from 2025 to 2030, according to a new report by Grand View Research, Inc. Increasing prevalence of rare diseases contributes to industry growth. According to the National Center for Advancing Translational Sciences (NCATS), over 30 million of the U.S. population is estimated to be affected by rare diseases in 2022. Increasing prevalence of people living with orphan diseases is anticipated to increase the sales and demand for orphan medicines over the forecast period.

The presence of supportive regulations by government authorities, such as the Orphan Drug Act that support product development by offering the orphan drug designation to potential drug candidates developed by pharmaceutical companies, is expected to support industry growth. This initiative boosts the research and development in the field of orphan disease treatment.

In April 2020, the National Organization for Rare Disorders (NORD) launched the "COVID-19 critical relief" program for patients with rare diseases affected by the COVID-19 pandemic. Under this program, the NORD provided monetary assistance of up to USD 1,000 annually to orphan disease patients and their caregivers for fulfilling their medical and non-medical needs. Thus, the availability of such programs is anticipated to boost the adoption of rare disease treatment over the forecast period.

Moreover, companies have undertaken initiatives such as collaborations and partnerships for the development, manufacturing, and commercialization of products in the international market. For instance, in August 2020, Sarepta Therapeutics, Inc. entered into a collaboration with the University of Florida for the development of novel genetic medicines for the treatment of patients with various orphan diseases, including Duchenne Muscular Dystrophy (DMD).

However, a lack of accurate and early diagnosis of rare diseases can often present a challenge for orphan disease patients, and in some cases, an accurate diagnosis is not obtained for as long as five years. In addition, patient eligibility and recruitment of eligible patients for clinical trials for rare disease treatment may restrain the industry growth. Not only finding the target population is difficult but finding physicians who treat these patients is also complex, thus leading to delays in the new product approval and launch in the market.

Rare Diseases Treatment Market Report Highlights:

  • By therapeutic area, cancer segment dominated the market with the largest market share of 28.2% in 2024 due to high prevalence of cancer indications such as multiple myeloma, acute lymphoblastic leukemia, chronic myeloid leukemia, and esophageal cancer and wide availability of orphan drugs for the treatment of patients with these conditions
  • By route of administration, injectable segment dominated the market with the largest share of 65.5% in 2024 and is expected to witness significant growth owing to high safety, efficacy, and tolerability associated with this route of administration among rare disease patients
  • By drug type, the biologics dominated the market with the largest share of 58.1% in 2024 due to the presence of supportive reimbursement policies and the higher efficacy of biologics as disease-modifying therapies for the treatment of rare diseases
  • By distribution channel, the specialty pharmacy dominated the market with the largest share of 70.5% in 2024 due to government initiatives to support specialty pharmacies that provide orphan drugs. For instance, in July 2022, Utilization Review Accreditation Commission (URAC) awarded Maxor Specialty Pharmacy with the rare diseases pharmacy center of excellence for providing high-quality orphan drugs to patients
  • Asia Pacific is expected to witness lucrative growth during the forecast period. The growth of the region can be attributed to the entry of new products into the region. According to Illness Challenged Foundation, China had approved new 87 orphan drugs for 47 rare diseases by the end of 2021

Table of Contents

Chapter 1. Methodology and Scope

  • 1.1. Market Segmentation and Scope
  • 1.2. Segment Definitions
    • 1.2.1. Therapeutic Area
    • 1.2.2. Drug
    • 1.2.3. Route of Administration
    • 1.2.4. Distribution Channel
    • 1.2.5. Regional Scope
    • 1.2.6. Estimates and forecasts timeline
  • 1.3. Research Methodology
  • 1.4. Information Procurement
    • 1.4.1. Purchased database
    • 1.4.2. GVR's internal database
    • 1.4.3. Secondary sources
    • 1.4.4. Primary research
    • 1.4.5. Details of primary research
  • 1.5. Information or Data Analysis
    • 1.5.1. Data analysis models
  • 1.6. Market Formulation & Validation
  • 1.7. Model Details
    • 1.7.1. Commodity flow analysis (Model 1)
    • 1.7.2. Approach 1: Commodity flow approach
    • 1.7.3. Volume price analysis (Model 2)
    • 1.7.4. Approach 2: Volume price analysis
  • 1.8. List of Secondary Sources
  • 1.9. List of Primary Sources
  • 1.10. Objectives

Chapter 2. Executive Summary

  • 2.1. Market Outlook
  • 2.2. Segment Outlook
    • 2.2.1. Therapeutic Area Outlook
    • 2.2.2. Drug Outlook
    • 2.2.3. Route of Administration Outlook
    • 2.2.4. Distribution Channel Outlook
  • 2.3. Regional outlook
  • 2.4. Competitive Insights

Chapter 3. Rare Disease Treatment Market Variables, Trends & Scope

  • 3.1. Market Lineage Outlook
    • 3.1.1. Parent Market Outlook
    • 3.1.2. Related/ancillary market outlook
  • 3.2. Market Dynamics
    • 3.2.1. Market Driver Analysis
      • 3.2.1.1. Rising investment in Research and Development
      • 3.2.1.2. Favorable Regulatory Frameworks
      • 3.2.1.3. Advances in Precision Medicine
    • 3.2.2. Market Restraint Analysis
      • 3.2.2.1. High Treatment Costs
  • 3.3. Rare Disease Treatment Market Analysis Tools
    • 3.3.1. Industry Analysis - Porter's
      • 3.3.1.1. Bargaining power of suppliers
      • 3.3.1.2. Bargaining power of buyers
      • 3.3.1.3. Threat of substitutes
      • 3.3.1.4. Threat of new entrants
      • 3.3.1.5. Competitive rivalry
    • 3.3.2. PESTEL Analysis
      • 3.3.2.1. Political landscape
      • 3.3.2.2. Economic landscape
      • 3.3.2.3. Social landscape
      • 3.3.2.4. Technological landscape
      • 3.3.2.5. Environmental landscape
      • 3.3.2.6. Legal landscape

Chapter 4. Rare Disease Treatment Market: Therapeutic Area Estimates & Trend Analysis

  • 4.1. Segment Dashboard
  • 4.2. Rare Disease Treatment Market: Therapeutic Area Movement Analysis
  • 4.3. Rare Disease Treatment Market by Therapeutic Area Outlook (USD Billion)
  • 4.4. Market Size & Forecasts and Trend Analyses, 2018 to 2030 for the following
  • 4.5. Cancer
    • 4.5.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 4.6. Neurological Conditions
    • 4.6.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 4.7. Cardiovascular Conditions
    • 4.7.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 4.8. Musculoskeletal Conditions
    • 4.8.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 4.9. Hematologic Disorders
    • 4.9.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 4.10. Infectious Diseases
    • 4.10.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 4.11. Metabolic Disorders
    • 4.11.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 4.12. Endocrine Disorders
    • 4.12.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 4.13. Others
    • 4.13.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)

Chapter 5. Rare Disease Treatment Market: Drug Estimates & Trend Analysis

  • 5.1. Segment Dashboard
  • 5.2. Rare Disease Treatment Market: Drug Movement Analysis
  • 5.3. Rare Disease Treatment Market by Drug Outlook (USD Billion)
    • 5.3.1. Market Size & Forecasts and Trend Analyses, 2018 to 2030 for the following
  • 5.4. Biologics
    • 5.4.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 5.5. Biosimilar
    • 5.5.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 5.6. Small molecules
    • 5.6.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)

Chapter 6. Rare Disease Treatment Market: Route of Administration Estimates & Trend Analysis

  • 6.1. Segment Dashboard
  • 6.2. Rare Disease Treatment Market: Route of Administration Movement Analysis
  • 6.3. Rare Disease Treatment Market by Route of Administration Outlook (USD Billion)
    • 6.3.1. Market Size & Forecasts and Trend Analyses, 2018 to 2030 for the following
  • 6.4. Oral
    • 6.4.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 6.5. Injectable
    • 6.5.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 6.6. Others
    • 6.6.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)

Chapter 7. Rare Disease Treatment Market: Distribution Channel Estimates & Trend Analysis

  • 7.1. Segment Dashboard
  • 7.2. Rare Disease Treatment Market: Distribution Channel Movement Analysis
  • 7.3. Rare Disease Treatment Market by Distribution Channel Outlook (USD Billion)
    • 7.3.1. Market Size & Forecasts and Trend Analyses, 2018 to 2030 for the following
  • 7.4. Hospital Pharmacy
    • 7.4.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 7.5. Specialty Pharmacy
    • 7.5.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)
  • 7.6. Online Pharmacy
    • 7.6.1. Market Estimates And Forecast, 2018 - 2030 (USD Billion)

Chapter 8. Rare Disease Treatment Market: Regional Estimates & Trend Analysis

  • 8.1. Regional Dashboard
  • 8.2. Market Size, & Forecasts Trend Analysis, 2018 to 2030:
  • 8.3. North America
    • 8.3.1. U.S.
      • 8.3.1.1. Key country dynamics
      • 8.3.1.2. Regulatory framework/ reimbursement structure
      • 8.3.1.3. Competitive scenario
      • 8.3.1.4. U.S. market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.3.2. Canada
      • 8.3.2.1. Key country dynamics
      • 8.3.2.2. Regulatory framework/ reimbursement structure
      • 8.3.2.3. Competitive scenario
      • 8.3.2.4. Canada market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.3.3. Mexico
      • 8.3.3.1. Key country dynamics
      • 8.3.3.2. Regulatory framework/ reimbursement structure
      • 8.3.3.3. Competitive scenario
      • 8.3.3.4. Mexico market estimates and forecasts 2018 to 2030 (USD Billion)
  • 8.4. Europe
    • 8.4.1. UK
      • 8.4.1.1. Key country dynamics
      • 8.4.1.2. Regulatory framework/ reimbursement structure
      • 8.4.1.3. Competitive scenario
      • 8.4.1.4. UK market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.4.2. Germany
      • 8.4.2.1. Key country dynamics
      • 8.4.2.2. Regulatory framework/ reimbursement structure
      • 8.4.2.3. Competitive scenario
      • 8.4.2.4. Germany market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.4.3. France
      • 8.4.3.1. Key country dynamics
      • 8.4.3.2. Regulatory framework/ reimbursement structure
      • 8.4.3.3. Competitive scenario
      • 8.4.3.4. France market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.4.4. Italy
      • 8.4.4.1. Key country dynamics
      • 8.4.4.2. Regulatory framework/ reimbursement structure
      • 8.4.4.3. Competitive scenario
      • 8.4.4.4. Italy market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.4.5. Spain
      • 8.4.5.1. Key country dynamics
      • 8.4.5.2. Regulatory framework/ reimbursement structure
      • 8.4.5.3. Competitive scenario
      • 8.4.5.4. Spain market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.4.6. Norway
      • 8.4.6.1. Key country dynamics
      • 8.4.6.2. Regulatory framework/ reimbursement structure
      • 8.4.6.3. Competitive scenario
      • 8.4.6.4. Norway market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.4.7. Sweden
      • 8.4.7.1. Key country dynamics
      • 8.4.7.2. Regulatory framework/ reimbursement structure
      • 8.4.7.3. Competitive scenario
      • 8.4.7.4. Sweden market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.4.8. Denmark
      • 8.4.8.1. Key country dynamics
      • 8.4.8.2. Regulatory framework/ reimbursement structure
      • 8.4.8.3. Competitive scenario
      • 8.4.8.4. Denmark market estimates and forecasts 2018 to 2030 (USD Billion)
  • 8.5. Asia Pacific
    • 8.5.1. Japan
      • 8.5.1.1. Key country dynamics
      • 8.5.1.2. Regulatory framework/ reimbursement structure
      • 8.5.1.3. Competitive scenario
      • 8.5.1.4. Japan market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.5.2. China
      • 8.5.2.1. Key country dynamics
      • 8.5.2.2. Regulatory framework/ reimbursement structure
      • 8.5.2.3. Competitive scenario
      • 8.5.2.4. China market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.5.3. India
      • 8.5.3.1. Key country dynamics
      • 8.5.3.2. Regulatory framework/ reimbursement structure
      • 8.5.3.3. Competitive scenario
      • 8.5.3.4. India market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.5.4. Australia
      • 8.5.4.1. Key country dynamics
      • 8.5.4.2. Regulatory framework/ reimbursement structure
      • 8.5.4.3. Competitive scenario
      • 8.5.4.4. Australia market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.5.5. South Korea
      • 8.5.5.1. Key country dynamics
      • 8.5.5.2. Regulatory framework/ reimbursement structure
      • 8.5.5.3. Competitive scenario
      • 8.5.5.4. South Korea market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.5.6. Thailand
      • 8.5.6.1. Key country dynamics
      • 8.5.6.2. Regulatory framework/ reimbursement structure
      • 8.5.6.3. Competitive scenario
      • 8.5.6.4. Thailand market estimates and forecasts 2018 to 2030 (USD Billion)
  • 8.6. Latin America
    • 8.6.1. Brazil
      • 8.6.1.1. Key country dynamics
      • 8.6.1.2. Regulatory framework/ reimbursement structure
      • 8.6.1.3. Competitive scenario
      • 8.6.1.4. Brazil market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.6.2. Argentina
      • 8.6.2.1. Key country dynamics
      • 8.6.2.2. Regulatory framework/ reimbursement structure
      • 8.6.2.3. Competitive scenario
      • 8.6.2.4. Argentina market estimates and forecasts 2018 to 2030 (USD Billion)
  • 8.7. MEA
    • 8.7.1. South Africa
      • 8.7.1.1. Key country dynamics
      • 8.7.1.2. Regulatory framework/ reimbursement structure
      • 8.7.1.3. Competitive scenario
      • 8.7.1.4. South Africa market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.7.2. Saudi Arabia
      • 8.7.2.1. Key country dynamics
      • 8.7.2.2. Regulatory framework/ reimbursement structure
      • 8.7.2.3. Competitive scenario
      • 8.7.2.4. Saudi Arabia market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.7.3. UAE
      • 8.7.3.1. Key country dynamics
      • 8.7.3.2. Regulatory framework/ reimbursement structure
      • 8.7.3.3. Competitive scenario
      • 8.7.3.4. UAE market estimates and forecasts 2018 to 2030 (USD Billion)
    • 8.7.4. Kuwait
      • 8.7.4.1. Key country dynamics
      • 8.7.4.2. Regulatory framework/ reimbursement structure
      • 8.7.4.3. Competitive scenario
      • 8.7.4.4. Kuwait market estimates and forecasts 2018 to 2030 (USD Billion)

Chapter 9. Competitive Landscape

  • 9.1. Company Categorization
  • 9.2. Recent Developments & Impact Analysis by Key Market Participants
  • 9.3. Company Market Share Analysis, 2024
  • 9.4. Company overview
    • 9.4.1. F. Hoffmann-La Roche Ltd.
      • 9.4.1.1. Company Overview
      • 9.4.1.2. Financial performance
      • 9.4.1.3. Product benchmarking
      • 9.4.1.4. Strategic initiatives
    • 9.4.2. Pfizer, Inc.
      • 9.4.2.1. Company Overview
      • 9.4.2.2. Financial performance
      • 9.4.2.3. Product benchmarking
      • 9.4.2.4. Strategic initiatives
    • 9.4.3. PTC Therapeutics Inc.
      • 9.4.3.1. Company Overview
      • 9.4.3.2. Financial performance
      • 9.4.3.3. Product benchmarking
      • 9.4.3.4. Strategic initiatives
    • 9.4.4. AstraZeneca
      • 9.4.4.1. Company Overview
      • 9.4.4.2. Financial performance
      • 9.4.4.3. Product benchmarking
      • 9.4.4.4. Strategic initiatives
    • 9.4.5. Novartis AG
      • 9.4.5.1. Company Overview
      • 9.4.5.2. Financial performance
      • 9.4.5.3. Product benchmarking
      • 9.4.5.4. Strategic initiatives
    • 9.4.6. Takeda Pharmaceutical Company Ltd
      • 9.4.6.1. Company Overview
      • 9.4.6.2. Financial performance
      • 9.4.6.3. Product benchmarking
      • 9.4.6.4. Strategic initiatives
    • 9.4.7. Bayer AG
      • 9.4.7.1. Company Overview
      • 9.4.7.2. Financial performance
      • 9.4.7.3. Product benchmarking
      • 9.4.7.4. Strategic initiatives
    • 9.4.8. AbbVie Inc.
      • 9.4.8.1. Company Overview
      • 9.4.8.2. Financial performance
      • 9.4.8.3. Product benchmarking
      • 9.4.8.4. Strategic initiatives
    • 9.4.9. Merck & Co. Inc.
      • 9.4.9.1. Company Overview
      • 9.4.9.2. Financial performance
      • 9.4.9.3. Product benchmarking
      • 9.4.9.4. Strategic initiatives
    • 9.4.10. Bristol Myers Squibb Company
      • 9.4.10.1. Company Overview
      • 9.4.10.2. Financial performance
      • 9.4.10.3. Product benchmarking
      • 9.4.10.4. Strategic initiatives

List of Tables

  • Table 1. List of secondary Sources
  • Table 2. List of abbreviations
  • Table 3. Global rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 4. Global rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 5. Global rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 6. Global rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 7. North America rare disease treatment, by region, 2018 - 2030 (USD Billion)
  • Table 8. North America rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 9. North America rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 10. North America rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 11. North America rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 12. U.S. rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 13. U.S. rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 14. U.S. rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 15. U.S. rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 16. Canada rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 17. Canada rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 18. Canada rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 19. Canada rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 20. Mexico rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 21. Mexico rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 22. Mexico rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 23. Mexico rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 24. Europe rare disease treatment, by country, 2018 - 2030 (USD Billion)
  • Table 25. Europe rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 26. Europe rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 27. Europe rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 28. Europe rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 29. UK rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 30. UK rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 31. UK rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 32. UK rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 33. Germany rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 34. Germany rare disease treatment, by drug 2018 - 2030 (USD Billion)
  • Table 35. Germany rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 36. Germany rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 37. France rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 38. France rare disease treatment, by drug 2018 - 2030 (USD Billion)
  • Table 39. France rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 40. France rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 41. Italy rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 42. Italy rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 43. Italy rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 44. Italy rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 45. Spain rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 46. Spain rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 47. Spain rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 48. Spain rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 49. Denmark rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 50. Denmark rare disease treatment, by drug 2018 - 2030 (USD Billion)
  • Table 51. Denmark rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 52. Denmark rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 53. Norway rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 54. Norway rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 55. Norway rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 56. Norway rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 57. Sweden rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 58. Sweden rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 59. Sweden rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 60. Sweden rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 61. Asia Pacific rare disease treatment, by country, 2018 - 2030 (USD Billion)
  • Table 62. Asia Pacific rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 63. Asia Pacific rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 64. Asia Pacific rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 65. Asia Pacific rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 66. Japan rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 67. Japan rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 68. Japan rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 69. Japan rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 70. China rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 71. China rare disease treatment, by drug 2018 - 2030 (USD Billion)
  • Table 72. China rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 73. China rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 74. India rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 75. India rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 76. India rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 77. India rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 78. Australia rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 79. Australia rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 80. Australia rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 81. Australia rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 82. South Korea rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 83. South Korea rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 84. South Korea rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 85. South Korea rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 86. Thailand rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 87. Thailand rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 88. Thailand rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 89. Thailand rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 90. Latin America rare disease treatment, by country, 2018 - 2030 (USD Billion)
  • Table 91. Latin America rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 92. Latin America rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 93. Latin America rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 94. Latin America rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 95. Brazil rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 96. Brazil rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 97. Brazil rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 98. Brazil rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 99. Argentina rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 100. Argentina rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 101. Argentina rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 102. Argentina rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 103. Middle East & Africa rare disease treatment, by country, 2018 - 2030 (USD Billion)
  • Table 104. Middle East & Africa rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 105. Middle East & Africa rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 106. Middle East & Africa rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 107. Middle East & Africa rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 108. South Africa rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 109. South Africa rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 110. South Africa rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 111. South Africa rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 112. Saudi Arabia rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 113. Saudi Arabia rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 114. Saudi Arabia rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 115. Saudi Arabia rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 116. UAE rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 117. UAE rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 118. UAE rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 119. UAE rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)
  • Table 120. Kuwait rare disease treatment, by therapeutic area, 2018 - 2030 (USD Billion)
  • Table 121. Kuwait rare disease treatment, by drug, 2018 - 2030 (USD Billion)
  • Table 122. Kuwait rare disease treatment, by route of administration, 2018 - 2030 (USD Billion)
  • Table 123. Kuwait rare disease treatment, by distribution channel, 2018 - 2030 (USD Billion)

List of Figures

  • Fig. 1 Information procurement
  • Fig. 2 Primary research pattern
  • Fig. 3 Market research approaches
  • Fig. 4 Value chain-based sizing & forecasting
  • Fig. 5 Market formulation & validation
  • Fig. 6 Rare disease treatment market segmentation
  • Fig. 7 Market driver analysis (current & future impact)
  • Fig. 8 Market restraint analysis (current & future impact)
  • Fig. 9 SWOT analysis, by factor (political & legal, economic and technological)
  • Fig. 10 Porter's five forces analysis
  • Fig. 11 Rare disease treatment market: Therapeutic area outlook and key takeaways
  • Fig. 12 Cancer market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 13 Neurological Conditions market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 14 Cardiovascular Conditions market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 15 Musculoskeletal Conditions market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 16 Hematologic Disorders market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 17 Infectious Diseases market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 18 Metabolic Disorders market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 19 Endocrine Disorders market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 20 Others market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 21 Rare disease treatment market: Drug outlook and key takeaways
  • Fig. 22 Rare disease treatment market: Drug movement analysis
  • Fig. 23 Biologics market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 24 Biosimilar market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 25 Small molecules market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 26 Rare Disease Treatment Market: Regional outlook and key takeaways
  • Fig. 27 Breast cancer liquid biopsy: Regional movement analysis
  • Fig. 28 Rare disease treatment market: Route of administration outlook and key takeaways
  • Fig. 29 Rare disease treatment market: Route of administration movement analysis
  • Fig. 30 Oral market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 31 Injectable market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 32 Others market estimates and forecast, 2018 - 2030 (USD Billion)
  • Fig. 33 Rare disease treatment market: Distribution channel outlook and key takeaways
  • Fig. 34 Rare disease treatment market: Distribution channel movement analysis
  • Fig. 35 Rare disease treatment market: regional key takeaways (USD billion)
  • Fig. 36 Rare disease treatment market: regional outlook, 2023 & 2030, USD billion
  • Fig. 37 North America rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 38 US rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 39 Canada rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 40 Mexico rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 41 Europe rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 42 UK rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 43 Germany rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 44 France rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 45 Spain rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 46 Italy rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 47 Norway rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 48 Denmark rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 49 Sweden rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 50 Asia Pacific rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 51 China rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 52 Japan rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 53 India rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 54 South Korea rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 55 Australia rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 56 Thailand rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 57 Latin America rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 58 Brazil rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 59 Argentina rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 60 Middle East and Africa rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 61 South Africa rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 62 Saudi Arabia rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 63 UAE rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 64 Kuwait rare disease treatment market estimates and forecasts, 2018 - 2030 (USD billion)
  • Fig. 65 List of key emerging company's/technology disruptors/innovators