Overview
The Orphan Drug Designation is to promote research and development of drugs for rare diseases, for which sufficient research and development is difficult in Japan due to the small number of patients, despite high unmet medical needs. When you get this designation based on the criteria, you can obtain special support for designated drugs throughout the research and development phase.
Designation criteria
To be designated as an orphan drug, it needs to meet criteria below.
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Number of subjects (any of the following is satisfied):
- Less than 50,000 in Japan
- The target disease is one of the designated intractable diseases (so-called “Nan-byo”)
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Medical needs:
- For serious diseases with high medical needs
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Possibility of development:
- Having organizations and plans that enable development in Japan
To understand more designation criteria, please see the following notifications and administrative notice.
- Partial Revision of “Designation of Orphan Drugs etc.” [155KB]
- Questions and Answers (Q&A) for Designation of Orphan Drugs etc.[104KB]
Incentives
Orphan drugs designated are eligible for the following incentives.
Financial incentives:
- Financial subsidies for up to 50% of expenses for drug development (NIBN)
- Tax credit during the NIBN subsidy period (NIBN)
- Marketing application fee reduction (PMDA)
- Consultation fee reduction (PMDA)
- Additional 10-20% premium drug pricing (MHLW)
Regulatory incentives:
- Priority review (PMDA)
- Priority consultation (PMDA)
- Extension of marketing exclusivity period so-called “re-examination period” (MHLW)
For more details, please see the following link.
Navigating regulatory pathways for rare disease drugs in Japan [1.06MB]
ENDPOINTS Events on-Demand Session (November 19th, 2025)
Orphan Drug Designation Procedure
An overview of the orphan drug designation process is shown in the figure below (including the designation consultation and application phases).
Applicants are encouraged to first review the section entitled “About preliminary preparations of designation consultation” at the link below.
https://www.mhlw.go.jp/stf/seisakunitsuite/bunya/0000068484.html
(Note) The website is written in Japanese. An English translation is available via the “Language” option next to the globe icon at the top of the website.
Once you have the information, please complete the application form at the same link and submit your request for designation consultation to below contact point.
When you are oversea’s company and wish to have designation consultation, MHLW request you to prepare interpreter for smooth communication or to submit its consultation request through your Japanese representative.
Contact point for applicants who wish to obtain an orphan drug designation
Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, MHLW
E-mail: orphan_drug[at]mhlw.go.jp
(Note) To block unsolicited emails being sent to MHLW, we request you to replace [at] with @ , when you send an email.
Related content
Organization of the orphan drug designation
The responsibilities of regulatory authorities involved are as follows.
Ministry of Health, Labour and Welfare (MHLW)
- Consultation on eligibility for orphan drug designation
- Review of eligibility for orphan drug designation
- Final decision of orphan drug designation
- Payment for the management expense grant of NIBN
Pharmaceuticals and Medical Devices Agency (PMDA)
- Preliminary evaluation of eligibility for orphan drug designation
- Priority scientific consultation for clinical trials and dossiers for marketing authorization of orphan drugs
- Priority scientific review of orphan drugs for marketing authorization
National Institute of Biomedical Innovation, Health and Nutrition (NIBN)
- Subsidy payment to the applicant
- Accreditation of research expenses used by the applicant for tax credits
- Guidance and advice on development support
Related links
- Ministry of Health, Labour and Welfare (MHLW)
- National Institute of Biomedical Innovation, Health and Nutrition (NIBN)
(Note) The above websites are written in Japanese. An English translation is available at the top of each website.