日本の希少疾病用医薬品市場レポート：薬剤タイプ、疾患タイプ、フェーズ、売れ筋医薬品、流通チャネル、地域別、2025年～2033年

日本の希少疾病用医薬品市場規模は2024年に140億3,400万米ドルに達しました。今後、IMARC Groupは、同市場が2033年までに326億8,700万米ドルに達し、2025年から2033年にかけて9.8%の成長率（CAGR）を示すと予測しています。治療が必要な希少疾患の増加とヘルスケアへのアクセス向上が、市場を牽引する主な要因のひとつです。

本レポートで扱う主な質問

• 日本の希少疾病用医薬品市場はこれまでどのように推移し、今後どのように推移するのか？
• COVID-19が日本の希少疾病用医薬品市場に与えた影響は？
• 日本の希少疾病用医薬品市場の薬剤タイプ別区分は？
• 日本の希少疾病用医薬品市場の疾患タイプ別区分は？
• 日本の希少疾病用医薬品市場のフェーズ別区分は？
• 日本の希少疾病用医薬品市場を売れ筋医薬品別に分類すると？
• 日本の希少疾病用医薬品市場の流通チャネル別区分は？
• 日本の希少疾病用医薬品市場のバリューチェーンにおける様々なステージとは？
• 日本の希少疾病用医薬品の主要な促進要因と課題は何か？
• 日本の希少疾病用医薬品市場の構造と主要プレーヤーは？
• 日本の希少疾病用医薬品市場における競合の程度は？

目次

第1章 序文

第2章 調査範囲と調査手法

• 調査の目的
• ステークホルダー
• データソース
• 市場推定
• 調査手法

第3章 エグゼクティブサマリー

第4章 日本の希少疾病用医薬品市場-イントロダクション

• 概要
• 市場力学
• 業界動向
• 競合情報

第5章 日本の希少疾病用医薬品市場情勢

• 過去および現在の市場動向（2019～2024年）
• 市場予測（2025～2033年）

第6章 日本の希少疾病用医薬品市場- 薬物タイプ別の内訳

• 生物学的
• 非生物学的

第7章 日本の希少疾病用医薬品市場- 疾患タイプ別の内訳

• 腫瘍学
• 血液学
• 神経学
• 心血管系
• その他

第8章 日本の希少疾病用医薬品市場- フェーズ別の内訳

• フェーズI
• フェーズII
• フェーズIII
• フェーズIV

第9章 日本の希少疾病用医薬品市場- 売れ筋医薬品別の内訳

• レブラミド
• リツキサン
• コパキソン
• オプジーボ
• キイトルーダ
• イブルビカ
• アボネックス
• センシパー
• ソリリス
• その他

第10章 日本の希少疾病用医薬品市場- 流通チャネル別の内訳

• 病院薬局
• 小売薬局
• オンラインストア
• その他

第11章 日本の希少疾病用医薬品市場- 競合情勢

• 概要
• 市場構造
• 市場企業のポジショニング
• 主要成功戦略
• 競合ダッシュボード
• 企業評価象限

第12章 主要企業のプロファイル

第13章 日本の希少疾病用医薬品市場- 業界分析

• 促進要因・抑制要因・機会
• ポーターのファイブフォース分析
• バリューチェーン分析

第14章 付録

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