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市场调查报告书
商品编码
1630952

罕见疾病治疗市场规模、份额、趋势分析报告：依治疗领域、给药途径、药物类型、通路、地区、细分市场预测，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个工作天内

价格

罕见疾病治疗市场成长与趋势：

Grand View Research, Inc.即将完成的一份新报告显示，预计2025年至2030年全球罕见疾病治疗市场将以11.6%的复合年增长率增长，到2030年将达到3743.9亿美元。

罕见疾病的盛行率不断上升，促进了该行业的成长。根据 NCATS（国家转化科学促进中心）估计，到 2022 年，美国将有超过 3,000 万人受到罕见疾病的影响。罕见疾病患者数量的增加预计将在预测期内增加孤儿药的销售和需求。

政府机构支持性法规的存在，例如《孤儿药法案》，透过为製药公司开发的潜在候选药物提供孤儿药指定来协助产品开发，预计将支持孤儿药的成长。该倡议将支持罕见疾病治疗领域的研究和开发。

2020年4月，美国罕见疾病组织（NORD）针对受COVID-19大流行影响的罕见疾病患者启动了「COVID-19危重症救助」计画。根据该计划，NORD 每年提供高达 1,000 美元的财政支持，以满足罕见疾病患者及其看护者的医疗和非医疗需求。因此，这些计划的可用性预计将在预测期内推动罕见疾病治疗的采用。

此外，公司也采取联盟和伙伴关係等倡议，在国际市场上开发、製造和商业化产品。例如，2020年8月，Sarepta Therapeutics, Inc.开始与佛罗里达大学进行研究合作，开发新型基因药物，用于治疗包括Duchenne氏肌肉失养症（DMD）在内的各种罕见疾病患者。

然而，缺乏对罕见疾病的准确和早期诊断往往是罕见疾病患者面临的挑战。此外，罕见疾病治疗临床试验的合格和招募合格患者也可能限制产业成长。不仅很难找到符合条件的患者，而且寻找治疗这些患者的医生也很复杂，导致新产品核准和市场推出的延迟。

罕见疾病治疗市场报告亮点

  • 从治疗领域来看，多发性骨髓瘤、急性淋巴性白血病、慢性骨髓性白血病和食道癌等癌症适应症的盛行率很高，并且由于药物、肿瘤学的广泛使用，罕见疾病疗法被用于治疗这些疾病的患者。
  • 从给药途径来看，注射剂在2024 年将占最大份额，达到65.5%，由于这种给药途径在罕见疾病患者中具有较高的安全性、有效性和耐受性，预计将出现显着增长。
  • 从药物类型来看，生物製药占据市场主导地位，2024 年份额最大，为 58.1%。这是由于支持性报销政策的存在以及生物製药作为疾病修正治疗在罕见疾病治疗中的高效能。
  • 从分销管道来看，由于政府支持专科药局提供罕见疾病药物的倡议，专科药局将在 2024 年占据市场最大份额，份额为 70.5%。例如，2022 年 7 月，利用审查和认证委员会 (URAC) 授予 Maxor Specialty Pharmacy 罕见疾病药房卓越中心称号，以表彰其为患者提供高品质的罕见疾病治疗。
  • 预计亚太地区在预测期内将出现良好的成长。该地区的成长得益于新产品的进入该地区。据疾病挑战基金会称，截至2021年终，中国已核准87种新的罕见疾病治疗药物，治疗47种罕见疾病。

目录

第一章调查方法和范围

第 2 章执行摘要

第三章 罕见疾病治疗市场的变数、趋势和范围

  • 市场体系展望
    • 母市场展望
    • 相关/附随市场展望
  • 市场动态
    • 市场驱动因素分析
    • 市场限制因素分析
  • 罕见疾病治疗市场分析工具
    • 产业分析-波特五力分析
    • PESTEL分析

第四章 罕见疾病治疗市场：治疗领域预估及趋势分析

  • 细分仪表板
  • 罕见疾病治疗市场：治疗区域波动分析
  • 罕见疾病治疗市场：依治疗领域分類的前景
  • 2018-2030年市场规模/预测与趋势分析
  • 癌症
  • 神经系统疾病
  • 心血管疾病
  • 肌肉骨骼疾病
  • 血液疾病
  • 感染疾病
  • 代谢紊乱
  • 内分泌疾病
  • 其他的

第五章 罕见疾病治疗市场：医药预测与趋势分析

  • 细分仪表板
  • 罕见疾病治疗市场：药品波动分析
  • 罕见疾病治疗市场：依药物分類的前景
    • 2018-2030年市场规模/预测与趋势分析
  • 生物製药
  • 生物相似药
  • 小分子

第六章 罕见疾病治疗市场：给药途径预估及趋势分析

  • 细分仪表板
  • 罕见疾病治疗市场：给药途径变化分析
  • 罕见疾病治疗市场：依给药途径分類的前景
    • 2018-2030年市场规模/预测与趋势分析
  • 口服
  • 可注射的
  • 其他的

第七章 罕见疾病治疗市场：通路估算及趋势分析

  • 细分仪表板
  • 罕见疾病治疗市场：通路波动分析
  • 罕见疾病治疗市场：按分销管道分類的前景
    • 2018-2030年市场规模/预测与趋势分析
  • 医院药房
  • 专业药房
  • 网路药房

第八章 罕见疾病治疗市场：区域估算及趋势分析

  • 区域仪表板
  • 2018-2030年市场规模、预测趋势分析：
  • 北美洲
    • 美国
    • 加拿大
    • 墨西哥
  • 欧洲
    • 英国
    • 德国
    • 法国
    • 义大利
    • 西班牙
    • 挪威
    • 瑞典
    • 丹麦
  • 亚太地区
    • 日本
    • 中国
    • 印度
    • 澳洲
    • 韩国
    • 泰国
  • 拉丁美洲
    • 巴西
    • 阿根廷
  • 中东/非洲
    • 南非
    • 沙乌地阿拉伯
    • 阿拉伯聯合大公国
    • 科威特

第9章 竞争格局

  • 公司分类
  • 主要市场参与企业的最新趋势和影响分析
  • 2024 年企业市场占有率分析
  • 公司简介
    • 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