Joint General Meeting of the Drug Evaluation Committee and Pharmaceutical Affairs Committee for FY 2025

Mr. Daisaku Sato, Deputy Director-General, Pharmaceuticals Bureau, Ministry of Health, Labour and Welfare (MHLW)

The lecture focused on recent trends in pharmaceutical administration, particularly the revision of the Pharmaceuticals and Medical Devices Control Law. The revision of the Pharmaceutical Affairs Act was submitted on February 12, 2025, focusing on strengthening governance, ensuring a stable supply (ensuring the system on the corporate side), screening and industry promotion, and strengthening the functions of pharmacies. It was voted on by the House of Representatives Health, Labor and Welfare Committee and is scheduled to be enacted during this Diet session.

He also touched on trends in drug development not only in Japan but also in the world, and expressed his opinion that it is necessary to promote the creation of innovation through collaboration between academia, venture companies, and pharmaceutical companies and to form an open innovation community, given the current situation where venture companies are leading drug discovery and development. He also stated the need for the formation of an open innovation community. He also explained the revision of the Pharmaceutical Affairs Law, including the review of the conditional approval system, utilization of real-world data, the drug discovery ecosystem support project, and periodic GMP surveys as matters related to the revision of the Pharmaceutical Affairs Law.

Director, Medical Devices Evaluation and Management Division, Pharmaceuticals Bureau, Ministry of Health, Labour and Welfare (MHLW)

In the "DASH for SaMD2 - Past Initiatives" session, the progress of the four pillars of DASH for SaMD2 was reported. The "Early Identification of Budding Seeds and Publication of Review Concepts" included the status of video distribution via YouTube, "Promotion of Practical Application Based on SaMD Characteristics" included the organization and publication of the concept of two-step approval and the status of publication of approved cases, "Strengthening the System for Early Commercialization" included the status of strengthening PMDA's review system and establishing a new consultation category, and "Japan's Initial SaMD2" included the status of "Japan's Initial SaMD2" and the status of the establishment of a new consultation category. The "Support for International Development of Japan's First SaMD" section reported on the status of research on overseas regulatory approval and marketing systems for SaMD and the promotion of English translations of review reports and other documents to facilitate their acceptance in reference countries.

In the "Update on SaMD-related Data," the number of approvals for programmed medical devices by year was presented, and the steady increase in the number of approvals and the number of consultations received were reported.

In "SaMD-related Budget," he reported on the overall budgets of the Ministry of Health, Labour and Welfare (MHLW) and the Ministry of Economy, Trade and Industry (METI) and the Japan Agency for Medical Research and Development (AMED) projects related to SaMD.

Mr. Kiyoto Nakai, Drug Evaluation and Management Division, Pharmaceuticals Bureau, Ministry of Health, Labour and Welfare

In addition to the content of legal revisions such as the designation of orphan drugs and the obligatory effort to formulate plans for pediatric drug development as a response to drug lag/loss, the report also included the omission of Japanese PI studies prior to international joint clinical trials for products developed overseas, the abolition of the requirement for Japanese data for follow-on biotech products, partial changes to the definition of pioneering drugs, the conditional approval system, and the review system for drug manufacturing, etc. The revision of the conditional approval system was explained in detail in a wide range of areas, including the omission of Japanese PI studies prior to international joint clinical trials for products developed overseas.

Regarding the revision of the conditional approval system, the basic concept of Japanese data was documented, a provision was made to revoke conditional approval, and the scope of operation was expanded to include approval when clinical usefulness can be reasonably predicted. The committee commented that the scope of operation has been expanded to include the use of real-world data. Regarding the GCP renovation, he expressed his hope that the clinical trials in Japan will be activated and drug loss will be improved through the promotion of the clinical trial ecosystem.

Ms. Yumiko Nomura, Director, Drug Safety Division, Pharmaceutical Affairs Bureau, MHLW

First, regarding the system reform, an image of the operation of the RMP after the legislation was presented. It was mentioned that it is necessary to speed up the non-routine safety monitoring activities so that they can be handled smoothly, that how long risk minimization activities should be continued will be an issue in the future, and that the use of real world data (RWD) is important to evaluate effectiveness in terms of outcomes rather than processes. The importance of utilizing real-world data (RWD) to evaluate effectiveness in terms of processes and outcomes was also discussed.

He also explained that, from the viewpoint of strengthening governance, the three roles of a manufacturer and distributor have been legally mandated to order changes and to establish a quality assurance manager and a safety manager.

From the examples of recent safety measures, he reiterated the necessity of fostering a safety culture and the need for safety measures to include crisis management in addition to risk management.

Mr. Manabu Hasegawa, Director, Research and Development Policy Division, Medical Policy Bureau, Ministry of Health, Labour and Welfare

He explained about the Clinical Research Act, the Act for Ensuring the Safety of Regenerative Medicine, etc., and genome medicine.
Regarding the Clinical Research Act, he explained the outline of the Act, the scope of the subject, and the concept of the review of the legal system. It was indicated that the review of the legal system will resolve the issues of the Clinical Research Act, which had been a problem up to now, and that the law will even stipulate penalties.

Regarding the Law for Ensuring the Safety of Regenerative Medicine, etc., as a system to promote the practical application of regenerative medicine, the Law for Ensuring the Safety of Regenerative Medicine, etc. is applied to general medical treatment and clinical research to provide safe and prompt regenerative medicine, and the Pharmaceutical Affairs Law is applied to manufacturing and marketing to supply many products more quickly, and with regard to target technologies, the revised law The scope of application has been expanded, and it has been indicated that impartial on-site inspections, etc. will be conducted to the establishers of the Authorized Committee for Regenerative Medicine, etc., which examines plans for the provision of regenerative medicine, etc. when such plans are submitted.

Mr. Takashi Yasukawa, Executive Officer, Pharmaceuticals and Medical Devices Agency

At first, he explained about the establishment of the Pharmaceutical Affairs Consultation Center for Pediatric and Orphan Drugs, etc., and stated that the designation of orphan drugs in FY2024 is remarkably expanding. He also touched on the promotion of pediatric and orphan drug development and drug price evaluation, emphasizing the importance of the relationship between innovation evaluation and regulatory approval in drug price system reform.

Next, regarding the handling of Japanese data, he explained that a working group of the Project Team for the Creation of Cross-sectional Criteria has been established and that the necessity of Japanese data is being sorted out. The planning and implementation of post-marketing drug use-results surveys were also discussed, and it was agreed that there are cases in which such surveys can be conducted at an appropriate time after marketing, rather than before approval, and a Q&A has been issued.

Regarding the dissemination of information to overseas, it was suggested that it is necessary to disseminate information on the Japanese system to overseas in order to eliminate the drug lag/loss. It was explained that information dissemination to overseas academic societies and venture companies is underway and that PMDA's Washington Office has started consultation services.