Orphan Drugs WG

About this 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.

Established

December, 2011

Members

Office of New Drug I to V
Office of Cellular and Tissue-based Products
Office of Vaccines and Blood Products
Office of Review Management
Office of Regulatory Science Coordination

Activities

• 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.
• Jointly held the following workshop on 10 March 2014;
  Joint EMA/FDA/MHLW-PMDA orphan medicinal product workshop

 

EC: European Commission
EMA: European Medicines Agency
FDA: US Food and Drug Administration

Related Notification

• Partial Revision of “Designation of Orphan Drugs etc.”[155KB]

January 16, 2024
PSB/PED Notification No. 0116-1・PSB/MDED Notification No. 0116-1
 

• Questions and Answers (Q&A) for Designation of Orphan Drugs etc.[104KB]

January 16, 2024
Administrative Notice

 

Past Presentations (Last 5 years)

• 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

 

• Clinical trials and regulatory supports for innovative drug development in Japan[1.29MB]

4th India-Japan Medical Products Regulation symposium 2019, Tokyo, Japan, February 2020

 

• Current situation and future perspectives of review and scientific consultation about orphan drugs[1.18MB]

16th DIA Japan Annual Meeting 2019, Tokyo, Japan, November 2019

 

Past Presentation (Related to PMDA-sponsored/co-sponsored workshop)

• Incentives and Regulatory Considerations in Orphan Drug/Medical Device Development - Situation in Japan -[363KB]

Joint EMA/FDA/MHLW-PMDA orphan medicinal product workshop, UK, March 2014

 

Related links

• Overview of Orphan Drug/Medical Device Designation System (Ministry of Health, Labour and Welfare (MHLW) website)
• Orphan Products Development Support Program (National Institute of Biomedical Innovation, Health and Nutrition (NIBIOHN) website)