Japan Rare Diseases Treatment Market Size, Share, and COVID-19 Impact Analysis, By Therapeutic Area (Cancer, Musculoskeletal Conditions, and Others), By Route of Administration (Oral, Injectable, and Others), and Japan Rare Diseases Treatment Market Insights, Industry Trend, Forecasts to 2035

Japan Rare Diseases Treatment Market Size Insights Forecasts to 2035

• The Japan Rare Diseases Treatment Market Size Was Estimated at USD 5276.15 Million in 2024
• The Market Size is Expected to Grow at a CAGR of Around 12.55% from 2025 to 2035
• The Japan Rare Diseases Treatment Market Size is Expected to Reach USD 19367.70 Million by 2035

According to a Research Report Published by Spherical Insights & Consulting, the Japan Rare Diseases Treatment Market Size is anticipated to reach USD 19367.70 million by 2035, growing at a CAGR of 12.55% from 2025 to 2035. The Japan rare disease treatment market is driven by various factors, including advancements in genetic research and diagnostic techniques, increasing government support, the development of targeted therapies, and growing collaboration between pharmaceutical companies and research institutes.

Market Overview

Rare disease refers to medical conditions that affect a small percentage of the population. Rare diseases include muscular dystrophy, Fabry disease, Gaucher disease, cystic fibrosis, and certain types of cancer. These are chronic, progressive, and life-threatening. The Japanese rare disease treatment focuses on research, development, and distribution of therapies and medications used to diagnose, manage, and cure rare diseases. The Japan rare disease treatment market is experiencing steady growth due to various major reasons, such as developments in genetic research and diagnostic technologies, which have enhanced the knowledge and identification of rare diseases, enabling more accurate diagnosis. Additionally, developments in patient-centred strategies, personalized medicine, biotechnology, and medication delivery technologies are anticipated to boost market expansion. The rising demand for targeted therapies presents an opportunity for the development of targeted treatments for specific rare genetic disorders.

Report Coverage

This research report categorizes the market for the Japan rare diseases treatment market based on various segments and regions, and forecasts revenue growth and analyzes trends in each submarket. The report analyses the key growth drivers, opportunities, and challenges influencing the Japan rare diseases treatment market. Recent market developments and competitive strategies such as expansion, product launch, development, partnership, merger, and acquisition, have been included to draw the competitive landscape in the market. The report strategically identifies and profiles the key market players and analyses their core competencies in each sub-segment of the Japan rare diseases treatment market.

Driving Factors

The government support for the research and development of rare disease treatment is one of the key driving factors for this market. The people are becoming more aware of the rare disease and their diagnostics, which further boosts the market growth. Additionally, advancements in technology and awareness campaigns play an important role in market expansion.

Restraining Factors

The rare disease treatments are extremely expensive due to high research and development costs and a limited patient population. Additionally, strict regulatory requirements further limit the market expansion.

Market Segmentation

The Japan rare disease treatment market share is classified into therapeutic area and route of administration.

• The cancer segment held the dominant share in 2024 and is expected to grow at a significant CAGR during the forecast period.

The Japan rare disease treatment market is segmented by therapeutic area into cancer, musculoskeletal conditions, and others. Among these, the cancer segment held the dominant share in 2024 and is expected to grow at a significant CAGR during the forecast period. This is due to the high prevalence and recurrence rate of rare cancer indications. Additionally, rare cancers, such as hepatoblastoma, thymic carcinoma, and essential thrombocythemia, require possible therapy.

• The injectable segment held the highest share in 2024 and is expected to grow at a substantial CAGR during the forecast period.

The Japan rare diseases treatment market is segmented by route of administration into oral, injectable, and others. Among these, the injectable segment held the highest share in 2024 and is expected to grow at a substantial CAGR during the forecast period. This is due to its effectiveness and efficacy in delivering medication straight to the targeted tissues or circulation. Additionally, they can deliver complex biological therapies.

Competitive Analysis:

The report offers the appropriate analysis of the key organizations/companies involved within the Japan rare diseases treatment market, along with a comparative evaluation primarily based on their product offering, business overviews, geographic presence, enterprise strategies, segment market share, and SWOT analysis. The report also provides an elaborative analysis focusing on the current news and developments of the companies, which includes product development, innovations, joint ventures, partnerships, mergers & acquisitions, strategic alliances, and others. This allows for the evaluation of the overall competition within the market.

List of Key Companies

• Takeda Pharmaceutical Company Ltd.
• Nobelpharma Co., Ltd.
• Nippon Shinyaku Co., Ltd.
• BioMarin Pharmaceutical Japan
• Amicus Therapeutics K.K.
• Novartis Pharma K.K.
• Other

Recent Developments

• In September 2024, Nippon Shinyaku and Japan’s NCNP received FDA Rare Pediatric Disease Designation for NS-050/NCNP-03, targeting exon 50 Skipping in DMD

Key Target Audience

• Market Players
• Investors
• End-users
• Government Authorities 
• Consulting and Research Firm
• Venture capitalists
• Value-Added Resellers (VARs)

Market Segment

This study forecasts revenue at Japan, regional, and country levels from 2020 to 2035. Spherical Insights has segmented the Japan rare diseases treatment market based on the below-mentioned segments:

Japan Rare Diseases Treatment Market, By Therapeutic Area

• Cancer
• Musculoskeletal Conditions
• Others

Japan Rare Diseases Treatment Market, By Route of Administration

• Oral
• Injectable
• Others