Japan Orphan Drugs Market Report by Drug Type, Disease Type, Phase, Top Selling Drug, Distribution Channel, and Region 2026-2034

Description

Japan orphan drugs market size reached USD 15,409.3 Million in 2025. Looking forward, IMARC Group expects the market to reach USD 34,333.9 Million by 2034, exhibiting a growth rate (CAGR) of 9.31% during 2026-2034. 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

Regional Insights:

Kanto Region
Kansai/Kinki Region
Central/ Chubu Region
Kyushu-Okinawa Region
Tohoku Region
Chugoku Region
Hokkaido Region
Shikoku Region
The report has also provided a comprehensive analysis of all the major regional markets, which include Kanto Region, Kansai/Kinki Region, Central/ Chubu Region, Kyushu-Okinawa Region, Tohoku Region, Chugoku Region, Hokkaido Region, and Shikoku Region.

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

1.What is the size of the orphan drugs market in Japan?
2.What is the expected growth rate of the Japan orphan drugs market during 2026-2034?
3.What are the key factors driving the Japan orphan drugs market?

Product Code: SR112026A18562

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 (2020-2025)
5.2 Market Forecast (2026-2034)

6 Japan Orphan Drugs Market - Breakup by Drug Type

6.1 Biological
- 6.1.1 Overview
- 6.1.2 Historical and Current Market Trends (2020-2025)
- 6.1.3 Market Forecast (2026-2034)
6.2 Non-Biological
- 6.2.1 Overview
- 6.2.2 Historical and Current Market Trends (2020-2025)
- 6.2.3 Market Forecast (2026-2034)

7 Japan Orphan Drugs Market - Breakup by Disease Type

7.1 Oncology
- 7.1.1 Overview
- 7.1.2 Historical and Current Market Trends (2020-2025)
- 7.1.3 Market Forecast (2026-2034)
7.2 Hematology
- 7.2.1 Overview
- 7.2.2 Historical and Current Market Trends (2020-2025)
- 7.2.3 Market Forecast (2026-2034)
7.3 Neurology
- 7.3.1 Overview
- 7.3.2 Historical and Current Market Trends (2020-2025)
- 7.3.3 Market Forecast (2026-2034)
7.4 Cardiovascular
- 7.4.1 Overview
- 7.4.2 Historical and Current Market Trends (2020-2025)
- 7.4.3 Market Forecast (2026-2034)
7.5 Others
- 7.5.1 Historical and Current Market Trends (2020-2025)
- 7.5.2 Market Forecast (2026-2034)

8 Japan Orphan Drugs Market - Breakup by Phase

8.1 Phase I
- 8.1.1 Overview
- 8.1.2 Historical and Current Market Trends (2020-2025)
- 8.1.3 Market Forecast (2026-2034)
8.2 Phase II
- 8.2.1 Overview
- 8.2.2 Historical and Current Market Trends (2020-2025)
- 8.2.3 Market Forecast (2026-2034)
8.3 Phase III
- 8.3.1 Overview
- 8.3.2 Historical and Current Market Trends (2020-2025)
- 8.3.3 Market Forecast (2026-2034)
8.4 Phase IV
- 8.4.1 Overview
- 8.4.2 Historical and Current Market Trends (2020-2025)
- 8.4.3 Market Forecast (2026-2034)

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 (2020-2025)
- 9.1.3 Market Forecast (2026-2034)
9.2 Rituxan
- 9.2.1 Overview
- 9.2.2 Historical and Current Market Trends (2020-2025)
- 9.2.3 Market Forecast (2026-2034)
9.3 Copaxone
- 9.3.1 Overview
- 9.3.2 Historical and Current Market Trends (2020-2025)
- 9.3.3 Market Forecast (2026-2034)
9.4 Opdivo
- 9.4.1 Overview
- 9.4.2 Historical and Current Market Trends (2020-2025)
- 9.4.3 Market Forecast (2026-2034)
9.5 Keytruda
- 9.5.1 Overview
- 9.5.2 Historical and Current Market Trends (2020-2025)
- 9.5.3 Market Forecast (2026-2034)
9.6 Imbruvica
- 9.6.1 Overview
- 9.6.2 Historical and Current Market Trends (2020-2025)
- 9.6.3 Market Forecast (2026-2034)
9.7 Avonex
- 9.7.1 Overview
- 9.7.2 Historical and Current Market Trends (2020-2025)
- 9.7.3 Market Forecast (2026-2034)
9.8 Sensipar
- 9.8.1 Overview
- 9.8.2 Historical and Current Market Trends (2020-2025)
- 9.8.3 Market Forecast (2026-2034)
9.9 Soliris
- 9.9.1 Overview
- 9.9.2 Historical and Current Market Trends (2020-2025)
- 9.9.3 Market Forecast (2026-2034)
9.10 Others
- 9.10.1 Historical and Current Market Trends (2020-2025)
- 9.10.2 Market Forecast (2026-2034)

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 (2020-2025)
- 10.1.3 Market Forecast (2026-2034)
10.2 Retail Pharmacies
- 10.2.1 Overview
- 10.2.2 Historical and Current Market Trends (2020-2025)
- 10.2.3 Market Forecast (2026-2034)
10.3 Online Stores
- 10.3.1 Overview
- 10.3.2 Historical and Current Market Trends (2020-2025)
- 10.3.3 Market Forecast (2026-2034)
10.4 Others
- 10.4.1 Historical and Current Market Trends (2020-2025)
- 10.4.2 Market Forecast (2026-2034)

11 Japan Orphan Drugs Market - Breakup by Region

11.1 Kanto Region
- 11.1.1 Overview
- 11.1.2 Historical and Current Market Trends (2020-2025)
- 11.1.3 Market Breakup by Drug Type
- 11.1.4 Market Breakup by Disease Type
- 11.1.5 Market Breakup by Phase
- 11.1.6 Market Breakup by Top Selling Drugs
- 11.1.7 Market Breakup by Distribution Channel
- 11.1.8 Key Players
- 11.1.9 Market Forecast (2026-2034)
11.2 Kansai/Kinki Region
- 11.2.1 Overview
- 11.2.2 Historical and Current Market Trends (2020-2025)
- 11.2.3 Market Breakup by Drug Type
- 11.2.4 Market Breakup by Disease Type
- 11.2.5 Market Breakup by Phase
- 11.2.6 Market Breakup by Top Selling Drugs
- 11.2.7 Market Breakup by Distribution Channel
- 11.2.8 Key Players
- 11.2.9 Market Forecast (2026-2034)
11.3 Central/ Chubu Region
- 11.3.1 Overview
- 11.3.2 Historical and Current Market Trends (2020-2025)
- 11.3.3 Market Breakup by Drug Type
- 11.3.4 Market Breakup by Disease Type
- 11.3.5 Market Breakup by Phase
- 11.3.6 Market Breakup by Top Selling Drugs
- 11.3.7 Market Breakup by Distribution Channel
- 11.3.8 Key Players
- 11.3.9 Market Forecast (2026-2034)
11.4 Kyushu-Okinawa Region
- 11.4.1 Overview
- 11.4.2 Historical and Current Market Trends (2020-2025)
- 11.4.3 Market Breakup by Drug Type
- 11.4.4 Market Breakup by Disease Type
- 11.4.5 Market Breakup by Phase
- 11.4.6 Market Breakup by Top Selling Drugs
- 11.4.7 Market Breakup by Distribution Channel
- 11.4.8 Key Players
- 11.4.9 Market Forecast (2026-2034)
11.5 Tohoku Region
- 11.5.1 Overview
- 11.5.2 Historical and Current Market Trends (2020-2025)
- 11.5.3 Market Breakup by Drug Type
- 11.5.4 Market Breakup by Disease Type
- 11.5.5 Market Breakup by Phase
- 11.5.6 Market Breakup by Top Selling Drugs
- 11.5.7 Market Breakup by Distribution Channel
- 11.5.8 Key Players
- 11.5.9 Market Forecast (2026-2034)
11.6 Chugoku Region
- 11.6.1 Overview
- 11.6.2 Historical and Current Market Trends (2020-2025)
- 11.6.3 Market Breakup by Drug Type
- 11.6.4 Market Breakup by Disease Type
- 11.6.5 Market Breakup by Phase
- 11.6.6 Market Breakup by Top Selling Drugs
- 11.6.7 Market Breakup by Distribution Channel
- 11.6.8 Key Players
- 11.6.9 Market Forecast (2026-2034)
11.7 Hokkaido Region
- 11.7.1 Overview
- 11.7.2 Historical and Current Market Trends (2020-2025)
- 11.7.3 Market Breakup by Drug Type
- 11.7.4 Market Breakup by Disease Type
- 11.7.5 Market Breakup by Phase
- 11.7.6 Market Breakup by Top Selling Drugs
- 11.7.7 Market Breakup by Distribution Channel
- 11.7.8 Key Players
- 11.7.9 Market Forecast (2026-2034)
11.8 Shikoku Region
- 11.8.1 Overview
- 11.8.2 Historical and Current Market Trends (2020-2025)
- 11.8.3 Market Breakup by Drug Type
- 11.8.4 Market Breakup by Disease Type
- 11.8.5 Market Breakup by Phase
- 11.8.6 Market Breakup by Top Selling Drugs
- 11.8.7 Market Breakup by Distribution Channel
- 11.8.8 Key Players
- 11.8.9 Market Forecast (2026-2034)

12 Japan Orphan Drugs Market - Competitive Landscape

12.1 Overview
12.2 Market Structure
12.3 Market Player Positioning
12.4 Top Winning Strategies
12.5 Competitive Dashboard
12.6 Company Evaluation Quadrant

13 Profiles of Key Players

13.1 Company A
- 13.1.1 Business Overview
- 13.1.2 Product Portfolio
- 13.1.3 Business Strategies
- 13.1.4 SWOT Analysis
- 13.1.5 Major News and Events
13.2 Company B
- 13.2.1 Business Overview
- 13.2.2 Product Portfolio
- 13.2.3 Business Strategies
- 13.2.4 SWOT Analysis
- 13.2.5 Major News and Events
13.3 Company C
- 13.3.1 Business Overview
- 13.3.2 Product Portfolio
- 13.3.3 Business Strategies
- 13.3.4 SWOT Analysis
- 13.3.5 Major News and Events
13.4 Company D
- 13.4.1 Business Overview
- 13.4.2 Product Portfolio
- 13.4.3 Business Strategies
- 13.4.4 SWOT Analysis
- 13.4.5 Major News and Events
13.5 Company E
- 13.5.1 Business Overview
- 13.5.2 Product Portfolio
- 13.5.3 Business Strategies
- 13.5.4 SWOT Analysis
- 13.5.5 Major News and Events

14 Japan Orphan Drugs Market - Industry Analysis

14.1 Drivers, Restraints, and Opportunities
- 14.1.1 Overview
- 14.1.2 Drivers
- 14.1.3 Restraints
- 14.1.4 Opportunities
14.2 Porters Five Forces Analysis
- 14.2.1 Overview
- 14.2.2 Bargaining Power of Buyers
- 14.2.3 Bargaining Power of Suppliers
- 14.2.4 Degree of Competition
- 14.2.5 Threat of New Entrants
- 14.2.6 Threat of Substitutes
14.3 Value Chain Analysis