PHP33 - UNDERSTANDING THE JAPANESE POLICY INITIATIVES AND THE PATIENT CHARACTERISTICS OF RARE, INTRACTABLE DISEASES

Abstract

To evaluate various health care policy initiatives for the rare disease and intractable disease population and understand the patient characteristics with intractable disease within Japan. Key government web-pages, municipal office level sources have been target-searched utilizing key terms such as "intractable disease” and "medical expense” in Japanese language. Data were synthesized and further verified with published literature including the governments' e-statistics data and insurance information sources, which encompasses real-world evidence of the economic burden associated with respective disease conditions. The number of diseases categorized as “intractable disease” has drastically increased over the past 4 years from 56 to 331 and the initiative budget has been growing year by year. In Japan, policies such as the “Medical expense assistance for intractable diseases” are implemented and applicable for patients across all age groups. In fiscal year 2017, the majority of patients receiving medical assistance were 60 years of age or older and ulcerative colitis (n=50,220) and Parkinson disease (n=120,799) had the highest number of patients. This study also revealed that public benefit programs are available for caregivers who have children with rare malignancies or chronic diseases that require long-term care. Growth hormone deficiency (n=13,891) and cretinism (n=6,015) had the most number of cases. Amid these efforts, other searches have revealed the economic burden outside of treatment cost, which is 100% out-of-pocket for the patient/family, such as accommodation and transportation is still considered substantial given the nature of long-term disease management. The national initiatives for rare disease are in place to improve access to high-cost treatments, despite the Japanese government's persistent focus on growing medical expenditure. As the literature were scant with respect to costs/burden outside of government subsidies, further investigation of other miscellaneous medical expenditures is warranted to better understand the economic burden from the patient perspective.