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Rare Disease And Orphan Drug Regulation In Japan

January 26, 2013

The Orphan Druganaut Blog continues its review of international rare disease and orphan drug regulations with a look at Japan. Last week we looked at Australia.

Intractable & Rare Diseases Research is a peer-reviewed journal of the International Research and Cooperation Association for Bio & Socio-Sciences Advancement that is published quarterly. A fact-filled and fascinating article on the status of rare diseases and orphan drugs in Asian countries can be found in this journal :

Peipei Song et al. “Rare Diseases, Orphan Drugs, And Their Regulation In Asia: Current Status and Future Perspectives”, Intractable & Rare Diseases Research. 2012; 1(1):3-9.

Below are a few categories for comparing rare disease and orphan drug definitions, legislation, and regulations for Japan vs. the United States :

Definition of Rare Diseases

– US: Prevalence of < 0.75% (effects < 200,000)

– Japan: Prevalence of < 0.40% (effects < 50,000)

Population Size

– US: 25-30 Million

– Japan: No information available


– US: Orphan Drug Act (1983) & Rare Disease Act of 2002

– Japan: Revised orphan drug regulations (Amendment of the Pharmaceutical Affairs Act & Drug Fund for Adverse Reaction Relief & Research Promotion Act – 1993)

Marketing Exclusivity

– US: 7 years

– Japan: 10 years (6 years for non-orphan drugs)

Tax Credits

– US: Up to 50% for clinical expenses

– Japan: up to 50% of expenses for clinical & non-clinical research; 15% tax credits, up to 14% corporate tax reduction

Fast Track Approval

– US: Yes

– Japan: Yes.

Additional statistics about rare diseases and orphan drugs in Japan :

– Almost 50% of orphan drugs on japanese market originated from EU or US

– 56 of 130 (approximately 43%) designated diseases are subject to reimbursement of medical expenses, with 30% paid by insurance companies, and the rest paid by national &prefectures governments.


Intractactable Disease Information Center

Japan’s Ministry of Health, Labour, & Welfare: Overview of Orphan Drug/Medical device Designation System

OrphaNet article on “Orphan Drugs in Japan”

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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