PSY1-4 - First-in-Human Trials: Regulator's Perspective in Japan

Abstract

ABSTRACT Sponsors of clinical trials or those conducting clinical trials on their own must submit the clinical trial protocol to the MHLW beforehand in accordance with Article 80-2, Paragraph 2 of Pharmaceutical Affairs Law. Those submitting protocols for the first time for the candidate drug in the clinical trial must submit the notification at least 31 days before the scheduled date of contract with the medical institution conducting the clinical trial or the scheduled date of obtaining the investigational drug, or the scheduled date of the initiation of the clinical trial, and shall not conclude the clinical trial contract or receive the investigational drug from the supplier of the drug concerned, or conduct the clinical trial on their own until after a period of 30 days has passed from the date of the acceptance of the clinical protocol notification by Pharmaceuticals and Medical Devices Agency (PMDA). In such cases, the Minister shall be able to have the necessary examinations of clinical trial protocols carried out by PMDA, in order not to jeopardize public health and hygiene (Article 80-2, Paragraph 3, Article 80-3 and MHLW Ordinance No. 77). Regarding the drugs for gene therapy or the drugs that are expected to be biological products, some points to be considered are separately specified. In Japan, about 500–600 clinical trial notifications are submitted every year, of which ∼120 are of initial notification of First-in-Japanese study (FIJ). I hereby introduce current situation and recent trend of FIH/FIJ studies actually experienced at PMDA, mainly of anticancer drugs as examples, with some brief comments on points to consider. As another issue to be noted, PMDA launched the Pharmaceutical Affairs Consultation on R&D Strategy, ‘Yakuji-Senryaku Sodan’, on 1 July 2011, mainly for universities, research institutions and venture capitals that possess promising ‘seed’ research or technologies, in order to achieve realization of innovative drugs and medical devices originating from Japan. The aim is to provide guidance and advice based on the tests needed from the final phase of the selection of candidate drugs or medical devices to early development stage (POC study) and the protocol designing etc. Some more information on this recently started consultation is also presented. In addition, ‘Guidance on Safety of First-in-Human study for the candidate drug’ (draft), for which public comments were invited from 11 May to 11 July 2011, is introduced.