New scheme related to new drug review and approval

Recommendations

• Addition of "Priority review system" and legislation of "Conditional early approval system": According to the promulgation of the Amended Pharmaceuticals and Medical Devices Act on Dec. 4, 2019, “Senkuteki Iyakuhin (Pioneering drugs)” (legislation of "Sakigake designation system"), "Specific use drugs, etc." designation system, and "Conditional early approval system" were implemented on Sep 1^st, 2020. For their operations, preparation of related laws based on the actual state and improvement in the relevant organisational structure and efficient implementation are required.
• Efficient implementation are required for the orphan drug designation and for the regulatory requirement to promote development of pediatric medicinal products. In addition, we propose improvement of the requirement for Japanese data (the need for Japanese Phase 1 studies in case of participation in international clinical studies).
• There is a room for further improvement in the approval review scheme for innovative new drugs in Japan compared to the U.S. and Europe. For example, in order for innovative new drugs to be approved in Japan without delay from the U.S. and Europe, a scheme similar to the Real Time Oncology Review in FDA, which is already in place in the U.S. and starts the review of new drug approval based on key results from a confirmatory clinical trial, should be introduced in Japan. We propose that a scheme similar to the Real Time Oncology Review, be introduced in Japan to shorten the period between a formal application and an approval.
• The PACMP system was established prior to TECHNICAL AND REGULATORY CONSIDERATIONS FOR PHARMACEUTICAL PRODUCT LIFECYCLE MANAGEMENT (ICH-Q12), but there appears to have been little progress in utilizing this system since its implementation on 1 August 2021 due to various factors. The relevant notifications and regulations should be reviewed to make the system more user-friendly.
• The following are proposed based upon the discussion in the MHLW panel: The review system of procedures for change control for the manufacturing process (including specifications and test methods, etc) and GMP compliance inspection system (inspection based on the risk of the manufacturing site, etc), and acceptance of the Western pharmacopoeia (USP/EP) as the official Pharmacopeia equivalent to the JP.