Orphan Drugs WG

This WG looks into issues pertaining to the development and application review of orphan drugs, and makes proposals for supportive measures to facilitate the orphan drug development.
（Activity period: From December, 2011）

Activities

Cooperative relationships between MHLW/PMDA and EC/EMA

• Set up the cooperative pilot program arrangement (Pilot Terms of Reference: ToR[146KB]) of orphan medicines under the cooperative relationships between MHLW/PMDA and EC/EMA.

(Note) MHLW/PMDA and EC/EMA have concluded a confidentiality arrangement (Confidentiality Arrangement) to exchange views on pharmaceutical regulatory systems.

• Based on ToR, exchanged opinions and strengthened mutual understanding concerning orphan drug designation systems.

 

EC: European Commission
EMA: European Medicines Agency
 

The activities conducted over the last five years.

Presentations

• Regulatory approach to promote orphan drug development in Japan[1.00MB]

The 7th Joint Conference of Taiwan Pharmacy, Taipei, November 2025

• Navigating regulatory pathways for rare disease drugs in Japan [1.06MB]

ENDPOINTS Events on-Demand Session (November 19^th, 2025) (external link)

• Health Equity: Tomorrow’s Normal in Rare Diseases[970KB]

22th DIA Japan Annual Meeting 2025, Tokyo, Japan, October 2025

• Orphan Drug Development Update: Issues and Measures for Global Cooperation Regulatory approach to promote orphan drug development in Japan[969KB]

20th DIA Japan Annual Meeting 2023, Tokyo, Japan, November 2023

• Regulatory approach to promote orphan drug development in Japan[1.24MB]

DIA China 2023 Annual Meeting, Suzhou, China (Web), June 2023

• Development of Drugs for Rare Diseases; Regulatory Perspective[514KB]

19th DIA Japan Annual Meeting 2022, Tokyo, Japan, October 2022

• Regulatory approach to promote orphan drug development in Japan[1.42MB]

9th Joint Conference of Taiwan and Japan on Medical Products Regulation (Web), October 2021

Publications

2025

• Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval

Anne R. Pariser, Violeta Stoyanova-Beninska, Oxana Iliach, Reda Jundi, Kerry Jo Lee ,  Hanako Morikawa, Samantha Parker, Caroline Pothet , Marco Rizzi, Julienne Vaillancourt, Ana Hidalgo-Simon
Drug Discov. Today.  2025; 30(12)

2021

• Orphan drug designation and development in Japan: 25 years of experience and assessment

Ken Sakushima, Hiroshi Takeda, Yoko Aoi
Nat Rev Drug Discov. 2021; 20(12): 893-4

Related Notification

• Guidance and Guidelines for Orphan Drugs.

Related links

• Overview of Orphan Drug Designation
• Ministry of Health, Labour and Welfare (MHLW)
• National Institute of Biomedical Innovation, Health and Nutrition (NIBN)

(Note) The MHLW and NIBN websites are written in Japanese. An English translation is available via the language selection option at the top of each website.