Study on Compassionate Use Program-Survey of Expanded Access Clinical Trials Conducted between 2016 and 2022 in Japan

Abstract

Compassionate use is a system that allows a patient with a fatal disease access to an unapproved investigational drug in the clinical study phase as an exception in cases where no alternative therapy exists for the disease. In Japan, this system is referred to as an “expanded access clinical trial”. The system was established in January 2016 and has only been implemented for 6 years since then. A detailed investigation regarding actual status has thus yet to be conducted. On the basis of available data, the present study investigated the statuses of implementation for expanded access clinical trials conducted between January 2016 and the end of May 2022 in Japan. Collection of data from the 6 years revealed that a total of 36 expanded access clinical trials (for 31 drugs) had been conducted, making up 1.53% of pivotal clinical trials. Anticancer drugs were the most common drugs studied (24 of 36 trials). The present study shows that expanded access clinical trials were successfully implemented in fields of high social and patient demands during the 6 years, but do not appear to have been aggressively implemented.