Japan Orphan Drugs Market Report by Drug Type, Disease Type, Phase, Top Selling Drug, Distribution Channel, and Region 2025-2033

Description

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?

Product Code: SR112025A18562

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