Topics The 13th Annual Conference of the Regulatory Science Society of Japan" was held

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The "13th Annual Meeting of the Regulatory Science Society of Japan" was held at the Academic Center for Science and Technology (Chiyoda-ku, Tokyo) on September 8-9, 2023, under the theme of "Progress and Harmony Required in Regulatory Science.

Introduction

The Society for Regulatory Science was established in August 2010 with the founding philosophy of bringing together people in the medical field, universities and research institutions, industry, and regulatory authorities on an equal footing to openly discuss research results and ideas related to regulatory science for pharmaceuticals and medical devices, and to promote the academic progress and dissemination of such science. The Society was established in August 2010. Thirteen years have passed since its establishment, and the concept of regulatory science is becoming widely accepted.

In 2023, the conference was held on September 8-9, 2023, with lively discussions in each section under the theme of "Progress and Harmonization Required for Regulatory Science".

The conference consisted of a plenary lecture, 3 special lectures, 2 symposia, 12 symposia, and 27 general presentations (15 oral and 27 poster presentations).

Symposium

First, Mr. Shinichiro Iida, a research fellow at JPMA The Office of Pharmaceutical Industry Research, gave a presentation on the status and characteristics of unapproved drugs in Japan based on the results of a comparative analysis of items approved in Japan, the U.S., and Europe from 2010 to the end of 2020 as "Drug Lag / Drug Losses. The survey results showed that the number of unapproved drugs in Japan has been on the rise since the latter half of 2010, and that one of the challenges to solving the problem of unapproved drugs in Japan is to attract emerging biopharmaceutical companies conducting clinical trials outside of Japan in Europe, the United States, and Asia to develop drugs in Japan. He presented the importance of improving pharmaceutical regulations, improving the environment for conducting clinical trials, and creating an attractive domestic market as key points for solving this issue.

Next, Yuji Kashitani, Chairperson of the JPMA Regulatory Affairs Committee, presented "Proposals for Drug Loss/Drug Lag from a Corporate Perspective" based on the results of a survey conducted in May 2023 among domestic and foreign companies belonging to the JPMA Regulatory Affairs Committee regarding items that have not yet been developed in Japan. and issues that need to be addressed. The key issues identified in the survey results included ensuring profitability and business feasibility, developing a system to conduct clinical trials when the number of patients is small, a system to promote smooth participation in international joint clinical trials, and a flexible system to accept overseas data when applying for approval.

Next, Dr. Noboru Yamamoto of the National Cancer Center Hospital introduced actual cases of drug loss in the field of anti-malignant tumors from the viewpoint of a clinician in his presentation entitled "Current Status and Efforts for Drug Loss from the Viewpoint of Medical Practice. In addition, as an effort to improve drug loss from the medical front, he introduced activities to attract overseas emerging biotech companies developing anti-cancer drugs to conduct clinical trials in Japan.

Finally, Mr. Yuji Matsukura, Assistant Director, Drug Evaluation and Management Division, Pharmaceutical Affairs Bureau, Ministry of Health, Labour and Welfare (MHLW), gave a presentation titled "MHLW's Efforts to Improve Access to Drugs" and explained the current discussions at the "Study Group on the Drug Regulation System to Enhance Drug Discovery and Ensure Stable Supply," with the aim to eliminate drug lag and drug loss. The current status of issues and responses to pharmaceutical regulations currently being discussed by the "Study Group on the Ideal Pharmaceutical Regulations to Enhance Drug Discovery Capability and Ensure Stable Supply, etc." was presented. The main topics of discussion were the early designation of drugs for rare diseases, the promotion of pediatric drug development, the necessity of Japanese Phase I studies when participating in international joint clinical trials, and the dissemination of information on Japan's pharmaceutical affairs system to the rest of the world.

First, Mr. Yoshio Nakayama, a member of the Regulatory Affairs Committee of the JPMA, presented the results of the "Second Survey on Regulatory Treatment of Approval, Change Procedures, and GMP Compliance Surveillance" conducted by the JPMA and a discussion of the pharmaceutical affairs systems in Japan, the U.S., and Europe, including the differences in approval requirements in Japan, the U.S., and Europe, and the differences between Japan and the U.S. The results of JPMA's questionnaire survey on the differences in approval items between Japan, the U.S., and Europe; the European and U.S. systems for notification of medium-risk and low-risk changes, which do not exist in Japan; and the status of implementation of GMP surveys in Japan, the U.S., and Europe were also presented. Based on the results of the survey, issues regarding approval items, the change procedure system, and the GMP investigation system in Japan were raised.

Next, Mr. Masatsugu Kobayashi, Chairperson of the JPMA Regulatory Affairs Committee's Pharmaceutical Affairs System Subcommittee, gave a presentation on "The Aim of Approval Matters, Change Management, and GMP Surveillance", in which he explained that, with the globalization of the pharmaceutical manufacturing and supply environment, the JPMA's vision for a common pharmaceutical affairs system (GMP) based on international consistency should be pursued, given that quality is the same in each country. The Pharmaceutical Affairs Committee of the Pharmaceutical Manufacturers Association of Japan (PMAJ) proposed a common pharmaceutical affairs system (approval items, change procedures, and GMP investigations) based on international consistency. The committee also proposed specific measures for approval items, change procedures, and GMP investigations to achieve this goal.

Next, Prof. Osamu Hiruta of Kumamoto University of Health Sciences introduced the differences between GMP surveys in Japan, the U.S., and Europe as "the ideal form of GMP survey," noting that in Japan, GMP surveys are a requirement for marketing authorization and target product items, while in the U.S. and Europe they target quality systems at manufacturing sites. He also presented the challenges of the certificate of compliance system introduced in 2021 and the need for a GMP investigation system based on international harmonization.

Lastly, Mr. Yasuhiro Kishioka, Examiner, Regenerative Medicine Products Examination Department, Pharmaceuticals and Medical Devices Agency (PMDA), presented "ICMRA PQ KMS Initiatives - Focusing on the Joint Examination Pilot" in cooperation with the International Coalition of Medicines Regulatory Authorities (ICMRA). He introduced the progress of the Pharmaceutical Quality Knowledge Management System (PQ KMS) led by the International Coalition of Medicines Regulatory Authorities (ICMRA). The two joint review pilot programs are underway and the review of the Post-Approval Change Management Protocol (PACMP) for additional manufacturing sites for biopharmaceuticals has been completed, and the experience will be used for future pilot programs.

General Abstracts＜Poster

The JPMA Regulatory Affairs Committee made poster presentations on the following six topics

The first part, "General Face-to-Face Advice," included the number of consultations, the companies' views on Drug Evaluation Committee consultations, and their opinions on general face-to-face advice, while the second part reported the results of the survey on "individual cases. In the second part, the results of the survey on "individual cases" were reported.

Due to the impact of the new corona infection (COVID-19), face-to-face consultations were mainly conducted via the web, but many companies expressed the opinion that it was beneficial. Although there was evidence of a high level of satisfaction with face-to-face advice, requests for more flexible views and alternative proposals from PMDA and matters related to communication with PMDA personnel (supplementary explanation of background, etc.) were raised as opinions for further increasing the level of satisfaction. Regarding the pre-evaluation consultation, it was reported that, as in the previous survey, interest was high, but the number of consultations was low, and many respondents requested clarification of the benefits.

The results of the questionnaire survey on the lag in applications for 67 of the 69 drugs containing new active ingredients approved in FY2020-FY2021, excluding the unapproved drugs and off-label drug study meeting items, and the survey on 35 new drugs that received approval (additional indications or additional dosage and administration) related to pediatric indications during the same period. The results were reported.

The types of application lag indicated that simultaneous application and US/European/American prior application accounted for 42% and 40% of the total, respectively, and that new drugs approved for pediatric indications are most frequently approved in the US/European/American prior application. It was noted that Japan's participation in Phase III global clinical trials and simultaneous filing have become established as a major development strategy option, and that a factor for the West's leading position in pediatric indication approvals is the need to improve the clinical environment in Japan.

This presentation received the 13th Best Poster Award.

The results of a questionnaire survey of 117 new drugs approved by 64 member companies of the JPMA Regulatory Affairs Committee out of 162 new drugs approved between January and December 2022 were reported. The median time from application to approval was 10.9 months for regular review items and 7.3 months for non-regular review items.

The percentage of initial interviews conducted decreased for the third consecutive year, and it was reported that efficacy and effectiveness were the most frequently discussed topics in the initial interviews this year. Regarding satisfaction with the screening process, it was reported that several positive comments were received on improvements based on the results of previous surveys (provision of cell phones to staff, omission of seals, electronic gateways, etc.). On the other hand, there were expectations for further improvements in "efficiency" (e.g., review of specialized consultations), "transparency" (e.g., timing of issuing inquiries, operational process and discussion of the Guidelines for the Promotion of Optimal Use), and "review of systems and notifications.

The 64 member companies of the JPMA Regulatory Affairs Committee reported on the latest trends and changes in the global development of projects in development or under submission as of the end of March 2022. As in recent years, the report showed that anti-cancer drugs accounted for a high percentage of the development disease areas, with global development items accounting for more than 80% of the total. As for development strategies, the status of utilization of real-world data, the status of Phase I studies involving Japanese in global development, and corporate attitudes toward pediatric drug development were reported, and requests for hurdles and incentives for starting pediatric drug development were presented.

Among the various pharmaceutical affairs procedures or pharmaceutical affairs responses to each prefecture, the results of a questionnaire survey conducted in January 2023 among member companies of the JPMA Regulatory Affairs Committee's Pharmaceutical Affairs System Subcommittee on matters related to pharmaceutical affairs responses as a manufacturer and distributor, GMP compliance surveys, and pharmaceutical affairs responses as a wholesaler and distributor, etc., were introduced. The results of the survey were presented. It was revealed that there are differences among prefectures (procedures that need to be submitted at the counter, whether or not a seal is required, fee payment methods, and procedural documents) even for the same procedures. In addition, it was found that the handling of GMP conformity survey procedures and the description on the license certificate in the wholesale sample wholesaler license procedures differ from prefecture to prefecture, and that these prefectural differences may be a need for standardized procedures for pharmaceutical companies, which should be pursued to further reduce the burden on both the government and companies, or to improve efficiency. The committee also discussed the need for further pursuit of ways to reduce the burden or improve the efficiency of procedures for both the government and the companies.

The results of the questionnaire survey conducted in November 2022 among member companies of the Regulatory Affairs Committee of the JPMA regarding the Standard Confirmation Certificate System, which was introduced with the enforcement of the revised Pharmaceuticals and Medical Devices Act in August 2021, were compared with the results of the 2021 questionnaire survey reported at the 2022 Regulatory Science Conference. The results were compared with the results of the 2021 survey reported at the 2022 Regulatory Science Society meeting. The results of the survey showed that while there has been no progress in the use of this system in the two years since its introduction, and measures need to be taken to promote its use, there are still many requests for further development of this system, such as expansion of the scope of application, as the benefits of using this system cannot be found from the standpoint of manufacturers and distributors. Based on the verification of the contribution of this system to the efficiency of GMP inspections, it was suggested that it is necessary to optimize resources related to the GMP inspection system and further rationalize GMP inspections as a whole.

Concluding Remarks

In the "Expert Panel on Comprehensive Measures to Achieve Prompt and Stable Supply of Pharmaceuticals," which began in 2022, the following issues were discussed: elimination of the drug loss/drug lag problem, promotion of pediatric drug development, the ideal form of pharmaceutical affairs review, etc., related to manufacturing methods of pharmaceutical products, and the ideal form of real world data utilization in the pharmaceutical affairs system, It has been pointed out that it is necessary to examine how pharmaceutical regulations should be regulated, and the "Study Group on Regulations for Strengthening Drug Discovery and Securing Stable Supply of Drugs" has been established and is currently examining the issue.

At the 2023 Annual Meeting, these issues were taken up in a symposium, and numerous active discussions were held from various perspectives. Through these activities, it is expected that the promotion of regulatory science and further collaboration between industry, academia, and government will progress further, and that regulatory science will develop further and the activities of the society will become more active.

( Yutaka Takeuchi, Masato Komuro, Akinobu Nakanishi, Sonoko Yamauchi, Regulatory Affairs Committee)

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