History of strategy for rare / Intractable diseases

In general, intractable diseases are defined as diseases which are either difficult to treat or are simply impossible to do so. Our nation’s history in relation to intractable diseases, and in particular, countermeasures against them, has followed a completely different path compared to that of other nations. In 1965, the devastating SMON (Subacute Myelo-Optico-Neuropathy) had a substantial impact on our society as a whole. With the cause of this disease uncovered by a research group from the Ministry of Health and Welfare through an urgent mass epidemiological survey in 1972, an intractable disease guideline was formulated as part of the government effort initiated in opposition against this “intractable disease.” This guideline would come to set a precedent for Japan’s approach to diseases of this nature. The guideline is based on three pillars: 1) the development of medical facilities; 2) the need for research projects; and, 3) the mitigation of self-pay medical costs for the people. Measures were quickly decided and enacted upon to adhere to these three pillars, including providing a substantial amount of funding and resources to the various studies and research as well as the foundation of a research project for the treatment of specified diseases, which would provide financial assistance to specific patients for a certain range of diseases.

By and large, the development of drugs designed for the treatment of intractable diseases with unknown causes has proven to be extremely difficult. Furthermore, due to the small number of patients and limited market for the drugs, pharmaceutical companies find it challenging to proceed in the development of new drugs. Consequently, our government has proceeded to undertake a comprehensive approach in developing countermeasures against intractable diseases. This internationally unprecedented political approach has contributed substantially to the identification of causes of some of these diseases and to the development of treatment of these diseases.


Some limitations of this approach included: 1) the large number of diseases where research has yet to be conducted; 2) feelings of inequality by patients who are unable to receive financial assistance for their medical costs; and, 3) the lack of finances required to sustain a stable financial support system for scientific research and medical costs. Thus, there was a need to fundamentally reassess the program. Hence, in 2015 a new medical law pertaining intractable diseases, aptly named “The Law Regarding Patients with Intractable Diseases”, was legislated.

Actions such as increasing the range of diseases eligible for financial support and utilizing the nation’s consumption tax as a source of fund for medical financial support programs have led to a new stage for the government’s countermeasures against intractable diseases.

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