Application of the New Process for Unapproved Drug Use: Dilemma of Universal Health Care Coverage in Japan

Patient-proposed health services (PPHS; kanjya mouside ryouyou in Japanese), a new Japanese government program that allows patients to use publicly insured and uninsured therapies—a so-called mixed medical care service that is prohibited in principal— was introduced in April 2016. Under PPHS, a patient can petition the Ministry of Health, Labour, and Welfare (MHLW) to assess an unapproved treatment by submitting documents from a Medical Service Act– certificated core clinical research hospital, where a study protocol is prepared after a review of the proposed treatment from the following points of view: (1) no possible participation in the existing system, such as ongoing clinical trials or Advanced Medical Care B; (2) the safety and efficacy of the treatment; and (3) the possibility of future approval of the treatment. The distinctive feature of this system is that the required care is performed as clinical research to accelerate the approval of the treatment. PPHS is a unique system designed to support patient wishes regarding the earliest possible advanced medical care without disrupting Japan’s universal health care coverage.