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.

In recent years, outpatient chemotherapy has become the mainstream of cancer treatment, and the role played by community pharmacies is becoming increasingly important. In this research, we surveyed the contents of the websites of base hospitals for cancer treatment in order to understand the current status of information that can be easily obtained at any pharmacy as a way of implementing hospital-pharmacy collaboration in outpatient chemotherapy. We surveyed the availability of 20 items of information on hospital-pharmacy collaboration in outpatient chemotherapy on the websites of 51 Prefectural Base Hospitals for cancer treatment and 275 regional base hospitals for cancer treatment as of the end of February 2021. We selected 5 from 20 items as main evaluation, and calculated correlation coefficients from rate of hospitals providing information in Prefectural and Regional Base hospitals. In the result, there was a strong correlation between the content of information provision between the two groups. We conducted a Wilcoxon rank-sum test based on rate of hospitals providing information at Prefectural and Regional Base hospitals in the same prefecture. There was significant correspondence in three items: “Route of administration”, “Information on supportive care (e.g., antiemetics, hydration)”, and “Information on concomitant therapy (e.g., radiation)”. In this research, it was founded that hospitals have a certain unified view on the items that are important to provide information to community pharmacies. On the other hand, there is no significant correspondence in the status of information provision between prefectures and regional base hospitals within the same prefecture. These results suggest that community pharmacists need to respond individually to each hospital, which may inhibit hospital-pharmacy collaboration.

In recent years, recalls of generic drugs which might affect the reliability in terms of health and supply have been reported. The objective of this study was to clarify the current status of drug recalls by investigating the number of drug recalls over time, and to examine issues for improving the reliability of generic drugs and promoting their use. The study covered prescription drugs recalled in the fiscal years of 2018-2022 (until September 2022). Recall information was categorized by drug class, drug type, dosage forms, therapeutic categories, reasons for recall, and the manufacturer/distributor, and analyzed by fiscal year. In 2018-2021, the total number of recalls increased each year (111 recalls in 2018, 141 in 2019, 271 in 2020 and 507 in 2021). There were 64 recalls in the first half of 2022. The percentage of recalls of generic drugs increased from 30.6%, 41.1%, 74.2%, to 91.1%. As the reasons of generic drug recalls, deviations from the approved specifications in stability monitoring, deviation from the approval document increased. There were also reasons that occurred only with generic drugs. In order to reduce the numbers of recalls and reestablish the reliability of generic drugs, there is a need to reestablish a quality control system and re-educate legal compliance on the generic drug industry.

For a long time, methodological limitations have been pointed out as for the estimation of drug costs in Japan, which relies on the results of the National Medical Expenditure and Survey of the Insurance-based Medical Care Activities. This study, from a different perspective, aims to grasp the trend of drug costs in Japan and hereby answer drug-related policy questions by combining monthly sales data on all prescription drugs from IQVIA Solutions Japan K. K. from FY2012 to FY2021 with data from Iyaku-Joho-Kenkyujo, Inc. using the YJ code. The results answered several questions. The depiction of the market sizes of brand-name drugs, long-listed drugs, and generics showed that one of the Japan’s recent policy goals, shifting from long-listed drugs to on brand-name drugs, was considerably achieved, as well as more usage of generics. Next, the effects of the re-pricing of antiviral drugs and anti PD-1 antibody drugs, various re-pricing and the normal price-revisions, the price maintenance for new drugs, and the market size of the essential drugs from the point of view of stable delivery. Third, simulation analyses in the absence of the regular price-revisions and various re-pricing. Overall, the result suggested that these various re-pricing mechanisms contributed to controlling overall drug costs, which is in line with the 2016 “The Basic Policy for Fundamental Reform of the Drug Pricing System”.

A cost-effectiveness evaluation system has been introduced in April 2019 to adjust the prices of new health technologies in Japan. At the same time, the specialist development program for health economics evaluation at Keio University has been launched for fourth year. Following the 1^st report in FY2021 on this journal from the required class of this program, Special Lecture on Health Economic Evaluation, we report on two drugs of H1 categories which the cost-effectiveness analysis results had already been published. In FY2022, the students who have learned about basic knowledge of health economics mainly participated, and workshop for Mock pre-consultation meeting was held to discuss cost effectiveness analytical framework, which divided into pharmaceutical industries and authorities. In the mock meeting, both parties discussed the analytical framework from their perspectives, and after the mock meeting both parties discussed, and then agreed on the analytical framework. Finally, we discussed and considered the differences between the agreed framework and the analytical framework implemented by the official analysis, and raised issues such as the difficulty of selecting comparative technologies and whether it is possible to evaluate based on a single standard. In addition, we discussed common issues and critical point of the entire system after the class. So we report both deliverables regarding summary of these issues and discussions from student’s perspective.

A sub-forum was held on February 6, 2023 as a study session of the SaMD Forum. The themes were “Issues Related to Medical Reimbursement in SaMD” and “Issues Related to the Utilization of Medical Data”. This paper provides an extensive review of the two topics, not limiting itself to the discussions that took place on the day of the forum. In the former discussion, industry made recommendations for evaluation based on issues specific to SaMD. The latter discussion focused on current issues regarding specific methods of providing medical data with appropriate protection of personal information. Based on these discussions, specific evaluation methods for SaMD are currently being discussed at the Central Social Insurance Medical Council (Chuikyo) of the Ministry of Health, Labour and Welfare (MHLW). In addition, a research group of the MHLW is preparing guidelines on appropriate methods of providing medical data. It is hoped that discussions on how to balance innovation and regulation will continue in the future.

Recently, the development of SaMD (Software as a medical device) has been actively carried out along with the progress of AI utilization. On the other hand, the use of medical information which is essential for the development of SaMD requires measures based on various laws and guidelines. The need for measures to facilitate utilization medical information is indicated in “The implementation plan for regulatory reform”. This paper outlines the challenges and initiatives related to medical data utilization in Japan to date.

With the development of AI technology, research on its use as SaMD is progressing rapidly. Amid this development, it is necessary to use medical information as learning data. Secondary use of data is an important perspective throughout R & D and after social implementation. Three points need to be considered: (ⅰ) Personal Information Protection Law; (ⅱ) Ethical Guidelines for Life and Medical Sciences; and (ⅲ) Pharmaceuticals and Medical Devices Law. With regard to the Personal Information Protection Law, the provision of pseudonymized information is being developed. However, practical procedures and standards have not yet been established. In addition, the Ethical Guidelines face issues such as inconsistencies with relevant laws and regulations. We believe the Pharmaceutical Affairs Agency Law still leaves some questions unanswered with regard to the specific methods of reliability assurance used during the approval process. Nevertheless, the government has recently been acting swiftly to address this issue, and the industry has high expectations for the future. Talking about prospects, the government has released new systems such as two-step approval, and we are hopeful that the use of real world data (RWD) will pave the way for things that have been difficult to put into practical use or build evidence for insurance applications. As an industry, we would like to work toward a virtuous cycle of SaMD development and social implementation by accumulating instances of such social deployment and real-world cases that lead to positive patient outcomes, and by fostering public understanding.

Various kinds of medical information are required in large quantities for AI development. In this manuscript, we presented data provision cases of Kagawa University Hospital (KUH) which is a typical regional national university hospital in Japan, and addressed the issues we recognize in the current situation. Most important issue is the conflict with personal information protection. Even if anonymization is applied, there are still constraints. We have introduced the KUH’s workflow for extracting medical information from electronic medical records (EMR). The purposes for extracting data from EMR are for clinical practice, research, and education. Extraction for research purposes requires approval from the ethical committee. When obtaining ethical committee approval, preparation such as informed consent forms related to opt-in/opt-out options is necessary, and specifics such as data storage and recipients need to be identified. The research plan specifies the extraction criteria based on the planned study. The medical information department follows the extraction criteria for extraction requests, extracts the data, and preserves the records of the extracted data. We have several cases of data provision for Software as a Medical Device (SaMD) and/or AI development. Data can be provided either from the hospital’s core EMR system or directly from department systems. In the latter case, there is a possibility that there won’t be a record of the data provision in the hospital’s management department. Data collection for registry studies is also useful for SaMD and AI development. We efficiently conduct registry studies using a regional medical network called Kagawa Medical Information eXchange R (K-MIX R). Such methods need to be considered for studies that require a large amount of data.

The Medical Device Single Audit Program (MDSAP) is a framework wherein third-party auditing organizations, deemed appropriate by MDSAP participating countries, conduct Quality Management System (QMS) audits on medical device manufacturers, with the results being utilized by each respective country. Rooted in a framework discussed in the arena of international regulatory harmonization, the MDSAP, under the leadership of Japan, the United States, Canada, Australia, and Brazil, has been implemented in society since 2017, following pilot demonstrations. However, its approach differs from the international regulatory harmonization framework for pharmaceuticals, which are also medical products, and can be difficult to comprehend for those unfamiliar with international regulations concerning medical devices. Therefore, this paper aims to clarify the characteristics of the MDSAP by contrasting its efforts with the case in the pharmaceutical field. Moreover, this paper revisits the history of the MDSAP, from its inception to its current state, while simultaneously providing an overview of the MDSAP.

The Ministry of Health, Labor and Welfare (MHLW) has designated Ephedrine, Codeine (limited to antitussive and expectorant drugs), Bromovalerylurea, Pseudoephedrine and Methylephedrine (limited to liquid preparations for antitussive and expectorant drugs) as pharmaceutical ingredients that may be abused according to the Act on Securing Quality Efficacy and Safety of Products Including Pharmaceuticals and Medical Devices. However, it has been pointed out that the number of cases of abuse of OTC drugs is increasing, and that the ease of obtaining them and the legality of using them are pointed out as factors for the increasing number of abuses. While there are various indications about the abuse of OTC drugs, MHLW has expanded the scope of regulated ingredients that may be abused that the limitations on designated OTC drugs shall be removed, and is working to strengthen measures to prevent abuse.