日本孤儿药市场报告（按药物类型、疾病类型、阶段、畅销药物、配销通路和地区划分）2025-2033

2024年，日本孤儿药市场规模达140.34亿美元。展望未来， IMARC Group预计到2033年，该市场规模将达到326.87亿美元，2025-2033年期间的复合年增长率（CAGR）为9.8%。缺乏治疗的罕见疾病数量不断增加，以及医疗保健可近性的改善，是推动市场发展的关键因素。

孤儿药是用于诊断、预防和治疗罕见疾病的药物。这些药物旨在满足特定的医疗保健需求，由于仅供少数患者使用，市场通常有限。它们已被证明可有效治疗多种疾病，包括但不限于肿瘤、代谢、血液、免疫、感染和神经系统疾病。此外，许多罕见疾病，如淋巴瘤、白血病、囊性纤维化、神经胶质瘤、胰臟癌、卵巢癌、多发性骨髓瘤和肾细胞癌，通常具有危及生命、慢性、进行性、退化性及致残性。这些疾病需要针对其独特症状的专门治疗方案。因此，孤儿药在日本各地的医院中被广泛使用。

日本孤儿药市场趋势：

在日本市场，多种因素正在推动市场成长。其中一个重要驱动因素是癌症和罕见遗传疾病的发生率不断上升。製药商正在积极开发创新孤儿药，为患者提供个人化治疗方案，以满足日益增长的需求，从而对区域市场产生了积极影响。此外，大众对孤儿药优势的认识不断提高，也促进了市场的成长。此外，新製药公司的涌现以及政府为遏制传染病传播而推出的优惠政策，也促进了这一积极的成长轨迹。除了这些因素之外，各种产品创新，例如生物孤儿药的研发，也成为额外的成长催化剂。这些药物能够治疗癌症等疾病，并逆转干细胞的损伤，从而显着提升了对此类药物的需求，这也是另一个重要的成长诱因。此外，医疗基础设施的改善以及对研发活动的大力投入预计将进一步推动市场扩张。

日本孤儿药市场细分：

药物类型洞察：

• 生物
• 非生物

疾病类型洞察：

• 肿瘤学
• 血液学
• 神经病学
• 心血管
• 其他的

阶段洞察：

• 第一阶段
• 第二阶段
• 第三阶段
• 第四阶段

畅销药品洞察：

• 来那度胺
• 利妥昔单抗
• 克帕松
• 奥狄沃
• 可瑞达
• 依鲁替尼
• 艾沃尼克斯
• 森西帕尔
• 索立利
• 其他的

配销通路洞察：

• 医院药房
• 零售药局
• 网上商店
• 其他的

竞争格局：

市场研究报告也对市场竞争格局进行了全面分析。报告涵盖了市场结构、关键参与者定位、最佳制胜策略、竞争指标和公司评估象限等竞争分析。此外，报告还提供了所有主要公司的详细资料。

本报告回答的关键问题：

• 日本孤儿药市场目前表现如何？未来几年将如何表现？
• COVID-19 对日本孤儿药市场有何影响？
• 日本孤儿药市场如何依药品类型划分？
• 日本孤儿药市场如何依疾病类型划分？
• 日本孤儿药市场以阶段划分是怎样的？
• 根据最畅销的药物，日本孤儿药市场是如何分類的？
• 日本孤儿药市场依照配销通路的分布是怎样的？
• 日本孤儿药市场的价值链分为哪些阶段？
• 日本孤儿药的关键驱动因素和挑战是什么？
• 日本孤儿药市场的结构是怎么样的？主要参与者是谁？
• 日本孤儿药市场的竞争程度如何？

本报告回答的关键问题：

• 日本孤儿药市场目前表现如何？未来几年将如何表现？
• COVID-19 对日本孤儿药市场有何影响？
• 日本孤儿药市场如何依药品类型划分？
• 日本孤儿药市场如何依疾病类型划分？
• 日本孤儿药市场以阶段划分是怎样的？
• 根据最畅销的药物，日本孤儿药市场是如何分類的？
• 日本孤儿药市场依照配销通路的分布是怎样的？
• 日本孤儿药市场的价值链分为哪些阶段？
• 日本孤儿药的关键驱动因素和挑战是什么？
• 日本孤儿药市场的结构是怎么样的？主要参与者是谁？
• 日本孤儿药市场的竞争程度如何？

目录

第一章：前言

第二章：范围与方法

• 研究目标
• 利害关係人
• 资料来源
  • 主要来源
  • 次要来源
• 市场评估
  • 自下而上的方法
  • 自上而下的方法
• 预测方法

第三章：执行摘要

第四章：日本孤儿药市场－简介

• 概述
• 市场动态
• 产业趋势
• 竞争情报

第五章：日本孤儿药市场概况

• 历史与当前市场趋势（2019-2024）
• 市场预测（2025-2033）

第六章：日本孤儿药市场-细分：依药物类型

• 生物
  • 概述
• 非生物
  • 概述

第七章：日本孤儿药市场-细分：依疾病类型

• 肿瘤学
  • 概述
• 血液学
  • 概述
• 神经病学
  • 概述
• 心血管
  • 概述
• 其他的

第 8 章：日本孤儿药市场 - 细分：依阶段

• 第一阶段
  • 概述
• 第二阶段
  • 概述
• 第三阶段
  • 概述
• 第四阶段
  • 概述

第九章：日本孤儿药市场-细分：按畅销药物

• 来那度胺
  • 概述
• 利妥昔单抗
  • 概述
• 克帕松
  • 概述
• 奥狄沃
  • 概述
• 可瑞达
  • 概述
• 依鲁替尼
  • 概述
• 艾沃尼克斯
  • 概述
• 森西帕尔
  • 概述
• 索立利
  • 概述
• 其他的

第十章：日本孤儿药市场-细分：按配销通路

• 医院药房
  • 概述
• 零售药局
  • 概述
• 网上商店
  • 概述
• 其他的

第 11 章：日本孤儿药市场 - 竞争格局

• 概述
• 市场结构
• 市场参与者定位
• 最佳获胜策略
• 竞争仪錶板
• 公司评估象限

第 12 章：关键参与者简介

• Company A
• Business Overview
• Product Portfolio
• Business Strategies
• SWOT Analysis
• Major News and Events
• Company B
• Business Overview
• Product Portfolio
• Business Strategies
• SWOT Analysis
• Major News and Events
• Company C
• Business Overview
• Product Portfolio
• Business Strategies
• SWOT Analysis
• Major News and Events
• Company D
• Business Overview
• Product Portfolio
• Business Strategies
• SWOT Analysis
• Major News and Events
• Company E
• Business Overview
• Product Portfolio
• Business Strategies
• SWOT Analysis
• Major News and Events

第十三章：日本孤儿药市场-产业分析

• 驱动因素、限制因素和机会
  • 概述
  • 驱动程式
  • 限制
  • 机会
• 波特五力分析
  • 概述
  • 买家的议价能力
  • 供应商的议价能力
  • 竞争程度
  • 新进入者的威胁
  • 替代品的威胁
• 价值链分析

第 14 章：附录

Japan orphan drugs market size reached USD 14,034 Million in 2024. Looking forward, IMARC Group expects the market to reach USD 32,687 Million by 2033, exhibiting a growth rate (CAGR) of 9.8% during 2025-2033. The rising number of rare diseases lacking treatment, along with improving access to healthcare, represent some of the key factors driving the market.

Orphan drugs are pharmaceutical products designed for the diagnosis, prevention, and treatment of rare medical conditions. These medications are developed to address specific healthcare needs and typically have a limited market because they are intended for a small number of patients. They prove effective against a wide range of medical conditions, including but not limited to oncological, metabolic, hematologic, immunologic, infectious, and neurological diseases. In addition to this, many of these conditions, such as lymphoma, leukemia, cystic fibrosis, glioma, pancreatic cancer, ovarian cancer, multiple myeloma, and renal cell carcinoma, are often life-threatening, chronic, progressive, degenerative, and disabling. They necessitate specialized treatment options that are tailored to address their unique symptoms. Consequently, orphan drugs are widely utilized in hospitals across Japan.

Japan Orphan Drugs Market Trends:

In the context of the Japan market, several factors are fueling market growth. One significant driver is the increasing prevalence of cancer and rare genetic disorders. Pharmaceutical manufacturers are actively developing innovative orphan drugs to offer personalized therapy options to patients in response to this growing demand, thereby positively influencing the regional market. Furthermore, the market is benefiting from heightened public awareness of the advantages of orphan drugs. Besides this, the emergence of new pharmaceutical companies in the market and the implementation of favorable government policies aimed at curbing the spread of contagious diseases are also contributing to this positive growth trajectory. In addition to these factors, various product innovations, such as the creation of biological orphan drugs, are serving as additional growth catalysts. These drugs have the capacity to treat diseases like cancer and reverse the damage inflicted on stem cells, significantly boosting their demand, which is acting as another significant growth-inducing factor. Moreover, improvements in healthcare infrastructure and a substantial focus on research and development (R&D) activities are expected to further propel the market's expansion.

Japan Orphan Drugs Market Segmentation:

Drug Type Insights:

• Biological
• Non-Biological

Disease Type Insights:

• Oncology
• Hematology
• Neurology
• Cardiovascular
• Others

Phase Insights:

• Phase I
• Phase II
• Phase III
• Phase IV

Top Selling Drugs Insights:

• Revlimid
• Rituxan
• Copaxone
• Opdivo
• Keytruda
• Imbruvica
• Avonex
• Sensipar
• Soliris
• Others

Distribution Channel Insights:

• Hospital Pharmacies
• Retail Pharmacies
• Online Stores
• Others

Competitive Landscape:

The market research report has also provided a comprehensive analysis of the competitive landscape in the market. Competitive analysis such as market structure, key player positioning, top winning strategies, competitive dashboard, and company evaluation quadrant has been covered in the report. Also, detailed profiles of all major companies have been provided.

Key Questions Answered in This Report:

• How has the Japan orphan drugs market performed so far and how will it perform in the coming years?
• What has been the impact of COVID-19 on the Japan orphan drugs market?
• What is the breakup of the Japan orphan drugs market on the basis of drug type?
• What is the breakup of the Japan orphan drugs market on the basis of disease type?
• What is the breakup of the Japan orphan drugs market on the basis of phase?
• What is the breakup of the Japan orphan drugs market on the basis of top selling drugs?
• What is the breakup of the Japan orphan drugs market on the basis of distribution channel?
• What are the various stages in the value chain of the Japan orphan drugs market?
• What are the key driving factors and challenges in the Japan orphan drugs?
• What is the structure of the Japan orphan drugs market and who are the key players?
• What is the degree of competition in the Japan orphan drugs market?

Table of Contents

1 Preface

2 Scope and Methodology

• 2.1 Objectives of the Study
• 2.2 Stakeholders
• 2.3 Data Sources
  • 2.3.1 Primary Sources
  • 2.3.2 Secondary Sources
• 2.4 Market Estimation
  • 2.4.1 Bottom-Up Approach
  • 2.4.2 Top-Down Approach
• 2.5 Forecasting Methodology

3 Executive Summary

4 Japan Orphan Drugs Market - Introduction

• 4.1 Overview
• 4.2 Market Dynamics
• 4.3 Industry Trends
• 4.4 Competitive Intelligence

5 Japan Orphan Drugs Market Landscape

• 5.1 Historical and Current Market Trends (2019-2024)
• 5.2 Market Forecast (2025-2033)

6 Japan Orphan Drugs Market - Breakup by Drug Type

• 6.1 Biological
  • 6.1.1 Overview
  • 6.1.2 Historical and Current Market Trends (2019-2024)
  • 6.1.3 Market Forecast (2025-2033)
• 6.2 Non-Biological
  • 6.2.1 Overview
  • 6.2.2 Historical and Current Market Trends (2019-2024)
  • 6.2.3 Market Forecast (2025-2033)

7 Japan Orphan Drugs Market - Breakup by Disease Type

• 7.1 Oncology
  • 7.1.1 Overview
  • 7.1.2 Historical and Current Market Trends (2019-2024)
  • 7.1.3 Market Forecast (2025-2033)
• 7.2 Hematology
  • 7.2.1 Overview
  • 7.2.2 Historical and Current Market Trends (2019-2024)
  • 7.2.3 Market Forecast (2025-2033)
• 7.3 Neurology
  • 7.3.1 Overview
  • 7.3.2 Historical and Current Market Trends (2019-2024)
  • 7.3.3 Market Forecast (2025-2033)
• 7.4 Cardiovascular
  • 7.4.1 Overview
  • 7.4.2 Historical and Current Market Trends (2019-2024)
  • 7.4.3 Market Forecast (2025-2033)
• 7.5 Others
  • 7.5.1 Historical and Current Market Trends (2019-2024)
  • 7.5.2 Market Forecast (2025-2033)

8 Japan Orphan Drugs Market - Breakup by Phase

• 8.1 Phase I
  • 8.1.1 Overview
  • 8.1.2 Historical and Current Market Trends (2019-2024)
  • 8.1.3 Market Forecast (2025-2033)
• 8.2 Phase II
  • 8.2.1 Overview
  • 8.2.2 Historical and Current Market Trends (2019-2024)
  • 8.2.3 Market Forecast (2025-2033)
• 8.3 Phase III
  • 8.3.1 Overview
  • 8.3.2 Historical and Current Market Trends (2019-2024)
  • 8.3.3 Market Forecast (2025-2033)
• 8.4 Phase IV
  • 8.4.1 Overview
  • 8.4.2 Historical and Current Market Trends (2019-2024)
  • 8.4.3 Market Forecast (2025-2033)

9 Japan Orphan Drugs Market - Breakup by Top Selling Drugs

• 9.1 Revlimid
  • 9.1.1 Overview
  • 9.1.2 Historical and Current Market Trends (2019-2024)
  • 9.1.3 Market Forecast (2025-2033)
• 9.2 Rituxan
  • 9.2.1 Overview
  • 9.2.2 Historical and Current Market Trends (2019-2024)
  • 9.2.3 Market Forecast (2025-2033)
• 9.3 Copaxone
  • 9.3.1 Overview
  • 9.3.2 Historical and Current Market Trends (2019-2024)
  • 9.3.3 Market Forecast (2025-2033)
• 9.4 Opdivo
  • 9.4.1 Overview
  • 9.4.2 Historical and Current Market Trends (2019-2024)
  • 9.4.3 Market Forecast (2025-2033)
• 9.5 Keytruda
  • 9.5.1 Overview
  • 9.5.2 Historical and Current Market Trends (2019-2024)
  • 9.5.3 Market Forecast (2025-2033)
• 9.6 Imbruvica
  • 9.6.1 Overview
  • 9.6.2 Historical and Current Market Trends (2019-2024)
  • 9.6.3 Market Forecast (2025-2033)
• 9.7 Avonex
  • 9.7.1 Overview
  • 9.7.2 Historical and Current Market Trends (2019-2024)
  • 9.7.3 Market Forecast (2025-2033)
• 9.8 Sensipar
  • 9.8.1 Overview
  • 9.8.2 Historical and Current Market Trends (2019-2024)
  • 9.8.3 Market Forecast (2025-2033)
• 9.9 Soliris
  • 9.9.1 Overview
  • 9.9.2 Historical and Current Market Trends (2019-2024)
  • 9.9.3 Market Forecast (2025-2033)
• 9.10 Others
  • 9.10.1 Historical and Current Market Trends (2019-2024)
  • 9.10.2 Market Forecast (2025-2033)

10 Japan Orphan Drugs Market - Breakup by Distribution Channel

• 10.1 Hospital Pharmacies
  • 10.1.1 Overview
  • 10.1.2 Historical and Current Market Trends (2019-2024)
  • 10.1.3 Market Forecast (2025-2033)
• 10.2 Retail Pharmacies
  • 10.2.1 Overview
  • 10.2.2 Historical and Current Market Trends (2019-2024)
  • 10.2.3 Market Forecast (2025-2033)
• 10.3 Online Stores
  • 10.3.1 Overview
  • 10.3.2 Historical and Current Market Trends (2019-2024)
  • 10.3.3 Market Forecast (2025-2033)
• 10.4 Others
  • 10.4.1 Historical and Current Market Trends (2019-2024)
  • 10.4.2 Market Forecast (2025-2033)

11 Japan Orphan Drugs Market - Competitive Landscape

• 11.1 Overview
• 11.2 Market Structure
• 11.3 Market Player Positioning
• 11.4 Top Winning Strategies
• 11.5 Competitive Dashboard
• 11.6 Company Evaluation Quadrant

12 Profiles of Key Players

• 12.1 Company A
  • 12.1.1 Business Overview
  • 12.1.2 Product Portfolio
  • 12.1.3 Business Strategies
  • 12.1.4 SWOT Analysis
  • 12.1.5 Major News and Events
• 12.2 Company B
  • 12.2.1 Business Overview
  • 12.2.2 Product Portfolio
  • 12.2.3 Business Strategies
  • 12.2.4 SWOT Analysis
  • 12.2.5 Major News and Events
• 12.3 Company C
  • 12.3.1 Business Overview
  • 12.3.2 Product Portfolio
  • 12.3.3 Business Strategies
  • 12.3.4 SWOT Analysis
  • 12.3.5 Major News and Events
• 12.4 Company D
  • 12.4.1 Business Overview
  • 12.4.2 Product Portfolio
  • 12.4.3 Business Strategies
  • 12.4.4 SWOT Analysis
  • 12.4.5 Major News and Events
• 12.5 Company E
  • 12.5.1 Business Overview
  • 12.5.2 Product Portfolio
  • 12.5.3 Business Strategies
  • 12.5.4 SWOT Analysis
  • 12.5.5 Major News and Events

13 Japan Orphan Drugs Market - Industry Analysis

• 13.1 Drivers, Restraints, and Opportunities
  • 13.1.1 Overview
  • 13.1.2 Drivers
  • 13.1.3 Restraints
  • 13.1.4 Opportunities
• 13.2 Porters Five Forces Analysis
  • 13.2.1 Overview
  • 13.2.2 Bargaining Power of Buyers
  • 13.2.3 Bargaining Power of Suppliers
  • 13.2.4 Degree of Competition
  • 13.2.5 Threat of New Entrants
  • 13.2.6 Threat of Substitutes
• 13.3 Value Chain Analysis

14 Appendix