Program Outline

R&D efforts for drugs and medical devices are time- and resource-intensive. In particular, many R&D programs aimed at developing therapies for intractable diseases do not make sufficient progress because, despite their high unmet medical needs, the small populations of patients make it difficult for manufacturers to recoup their financial investments.

To address this problem, a government-led support initiative was launched in 1993. This initiative provides special support for R&D of pharmaceutical and medical product candidates that have been granted orphan status by the Minister of Health, Labour and Welfare (MHLW Minister). The Act for Partial Amendment to the Pharmaceutical Affairs Act, etc. enacted in 2013 (Act No. 84/2013) introduced a new legal term, “regenerative medicine (cellular and tissue-based) products,” to medical and pharmaceutical regulations. At the same time, a new initiative has been started concerning “regenerative medicine (cellular and tissue-based) products for orphan diseases.”

This program aims to deliver new, safe, and effective orphan drugs, orphan medical devices, and regenerative medicine (cellular and tissue-based) products for orphan diseases into clinical practice at the earliest opportunity. To achieve this goal, NIBIOHN offers financial support for the development of orphan-designated products, and also provides appropriate guidance and advice for obtaining marketing authorization.

References: Criteria for Orphan Designation by the MHLW Minister (Pharmaceutical and Food Safety Bureau Notification No. 0401-11)

(1) Number of patients

The number of patients who may use the drugs, medical device or regenerative medicine should be less than 50,000 in Japan.

(2) Medical needs

The drugs, medical devices or regenerative medicine should be indicated for the treatment of serious diseases, including difficult-to-treat diseases. In addition, they must be drugs, medical devices or regenerative medicine for which there are high medical needs satisfying one of the following criteria.

• There is no appropriate alternative drug/medical device/regenerative medicine or treatment
• High efficacy or safety is expected compared with existing products

(3) Possibility of development

There should be a theoretical rationale for the use of the product for the target disease, and the development plan should be appropriate.

Subsidy Granting

NIBIOHN grants financial assistance to the developers of pharmaceutical and medical product candidates that have been designated by the MHLW Minister as “orphan drugs,” “orphan medical devices,” or “regenerative medicine (cellular and tissue-based) products for orphan diseases.” The direct expenses of the R&D cost required for development and authorization of such products are eligible for subsidization.

Orphan developers who receive grants must refund part of the profits accruing from the sales of the commercialized orphan products.

Products Eligible for Subsidization

Product candidates designated by the MHLW Minister as “orphan drugs,” “orphan medical devices,” or “regenerative medicine (cellular and tissue-based) products for orphan diseases.”

Subsidy Period

From the time of orphan designation to the end of the fiscal year in which the company files an application for marketing authorization, with a maximum of 3 years.

Expenses Eligible for Coverage

Honoraria, travels, equipment, consumables, printing and binding, communication and transportation, rents and leases, meetings, labor services, and subcontracting fee.
Note: Consumption taxes are not covered, except for travel cost.

Subsidy Ceiling

Maximum of 50% of the R&D cost

Refunds

Refund amount = (sales - JPY 100 million)/100
Note: No refunds are necessary if annual sales are not greater than JPY 100 million. Refunds will not exceed the total amount of subsidization.

Guidance and Advice

NIBIOHN accepts consultation requests by the developers of candidates for “orphan drugs,” “orphan medical devices,” and “regenerative medicine (cellular and tissue-based) products for orphan diseases.” Its staff members provide guidance and advice concerning the interpretation of the designation criteria stipulated by the Pharmaceuticals and Medical Devices Act, subsidization requirements, and other matters related to orphan development. In addition, program officers with expertise and experience in pharmaceutical and medical product development provide advice and consultation both in and out of the NIBIOHN office.

Consultation Topics

Matters related to the development of orphan drugs, orphan medical devices, and regenerative medicine (cellular and tissue-based) products for orphan diseases.

R&D Expenses Applicable to Corporate Tax Credits

Based on requests by grantees, NIBIOHN makes assessments according to the Special Taxation Measures Act the direct expenses required during the subsidy period for R&D projects on orphan drugs, orphan medical devices, and regenerative medicine (cellular and tissue-based) products for orphan diseases. The developers can receive a tax credit for the subsidy period based on the outcome of these assessments.

Items and Period Covered

R&D cost spent in the last closed fiscal year in which the pharmaceutical or medical product candidates were approved for subsidization

Tax-Deductible Expenses

20% × [R&D cost − subsidy amount]

Period to File an Assessment Request

Within 1 month of the account closing date of the subsidized company

Application Guides and Forms

Please note that the Guides and forms available through the links below are in Japanese only.

Subsidy Application Guide and Forms 【Last Update : April 1, 2022】

• Subsidy Application Guide
• Subsidy Application Form No. 1
• Subsidy Application Form No. 1-1
• Subsidy Application Form No. 3
• Subsidy Application Form No. 5
• Subsidy Application Form No. 5-1 to 5-11（Spending　Book）
• Subsidy Application Form No. 8
• Subsidy Application Form No. 10
• Checklist

Assessment Request Guide and Forms 【Last Update : January 24, 2022】

• Assessment Request Guide
• Assessment Request Form

References

• Guide for Orphan Product Development 【Last　Update : April 10, 2021】
• List of Designated Orphan Drugs (English Version)*
• List of Designated Orphan Medical Devices (English Version)*
• List of Regenerative Medicine (cellular and tissue-based) products for Orphan diseases (English Version)*

*Update is stopped. 【Last Update : July 31,2018】

For the latest information (Japanese version), please follow the link provided below.
https://www.nibiohn.go.jp/nibio/part/promote/orphan_support/index.html#disclosure

Contact Point Information

Department of Research Support
National Institutes of Biomedical Innovation, Health and Nutrition
7-6-8 Saito-Asagi, Ibaraki-city, Osaka, 567-0085
TEL: +81-72-641-9804　FAX: +81-72-641-9831
E-mail: kisho-ph[at]nibiohn.go.jp