Abstract
There are so many social systems and pharmaceutical jurisprudences those enable early access to drugs for unmet medical needs in the world; breakthrough therapy designation and expanded access program in the United States; early access medicines scheme and compassionate use program in the UK; fixed-term conditional approval, humane exceptional use of unapproved drugs, and patient-requested cure system in Japan etc. Patients with life-threatening and serious disease or long-suffering intractable orphan disease aspire unapproved novel drug candidates even if those efficacy/safety evidence are lacking. We, clinician, have to pay attention to those desire. Furthermore, considering the fact that Japanese universal health care's sustainability will face a crisis in 2025 when baby boomer will be elderly aged over 75 or over, industry-government-academia with patients must continue to devise an appropriate early access program in Japan.
