orphan drugs policy in Japan

A progress report on rare diseases and orphan drugs policy in Japan

An article published in Expert Opinion Orphan Drugs reports on the current and future policy initiatives on rare diseases in Japan. According to the authors, these initiatives have been an element of the Japanese national health system dated as early as 1972. The authors believe that due to the extensive support from the government, rare disease policy in Japan has witnessed “considerable progress over the past 40 years” which encompasses the orphan drug legislation of 1993.

This legislation not only encouraged research and development of orphan drugs, but also brought changes to the pricing and reimbursement systems. The authors contend that this legislation “facilitated access to orphan drugs, specific research programs to promote research on and development of orphan drugs, and a government-supported information centre to promote the understanding of rare diseases”.

In the article the authors contrast pricing and reimbursement strategies in EU and US, with the Japanese National Health Insurance (NHI) system. Unlike the EU and US, the NHI in Japan “negotiates prices with pharmaceutical companies once a drug is approved for use, allowing a selling price of cost plus 10% for orphan drugs”. Japan has designated130 diseases as intractable or rare diseases, out of which 56 can receive reimbursement of medical expenses. The reimbursement structure allows “30% of expenses paid by insurance companies and the rest paid by national and prefectural governments”.

The authors describe several research programmes that are supported by the Japanese government such as the project establishing ‘Bases for Early and Exploratory Clinical Trials in Specific Research Areas’, launched in 2011 “to promote the development of innovative orphan drugs and medical devices from Japan”. The authors also highlight another project ‘Enhanced International Information Exchange’ launched this year which will aid in “publicis(ing) the results of limited research on rare diseases and orphan drugs to a broader international audience”.

Finally, the authors illustrate the example of Japan Intractable Disease Information Center – a collaborative effort of the MHLW and the Japan Intractable Diseases Research Foundation – to dessiminate information about rare diseases in Japan. The authors highlight that this website gets over 15 million hits per year and is a testament to its in spreading knowledge about rare diseases in Japan
Consult the abstract

 source: orphanews