The essential functions of pharmacies in Japan involve supporting self-care and self-medication related to the maintenance and promotion of community residents’ health. This study surveyed the Tokyo Metropolitan Government’s ‘Community Pharmacy Functional Information Report’ and the ‘Tokyo Metropolitan Government’s Independent Published Item Report’ published in 2020, to understand the characteristics of pharmacies that promote self-care and self-medication and the factors that support them. All pharmacies in Tokyo were divided into three groups : health support pharmacies, pharmacies with pharmacists who had completed training for health support pharmacies (pharmacies that had completed training), and pharmacies that did not fall into either category (general pharmacies), and pharmacy functions were compared. The percentage of pharmacies that could provide various consultation services was significantly higher in health support pharmacies and pharmacies that had completed training than that in general pharmacies. This suggests that the presence of pharmacists who have completed the health support pharmacy training program is one of the factors that enhanced the consultation system. Additionally, pharmacies that promote self-care and self-medication have large stockpiles of both prescription and non-prescription drugs, indicating that they have a system in place to provide appropriate drugs to their users.

 In 2010, the Ministry of Health, Labour and Welfare launched a committee, “Evaluation Committee on Unapproved or Off-label Drugs with High Medical Needs” to accept requests for development of unapproved or off-label drugs in Japan and encourage pharmaceutical companies to develop them. Of the requests submitted so far, about one-fifth have been judged as drugs without high medical needs, which resulted in no action. The purpose of this study was to clarify the eligibility requirements for drugs judged as those with high medical need (“necessary standard”) and also to highlight the important role of the committee. Of the fourth request, 102 drugs that had already been evaluated by August 30, 2023, were selected as study subject (1). Of these, 91 drugs submitted under the category of unapproved drugs and off-label drugs were selected as study subject (2). Information of explanatory variables was extracted from the request form, and logistic regression analysis was conducted with the necessary standard as a response variable. Presence of randomized controlled trial data, requests submitted by relevant academic societies, and requests submitted for the second or more times in the study subject (1), and degree of recommendation in overseas guidelines in the study subject (2) showed significant associations with the necessary standard. When submitting requests to the committee, it is recommended to collaborate with academic societies and carefully check the existence of literature with a high evidence level. The committee is expected to continuously play a crucial role in enabling patients to quickly access insurance-covered medications by utilizing existing scientific data.

Introduction : Adverse events in robot-assisted surgery (RAS) are multifaceted and influenced by both technical and human factors. This study aims to provide insights into post-market clinical study design for therapeutic devices in RAS by comparing early-stage research design on two generations of da Vinci and Versius, focusing on prospective studies, adverse event analysis, and levels of evidence. Methods : A comparative analysis was conducted using data from clinical studies and reports involving da Vinci and Versius in the post-market early stage. We compared the ratios of prospective studies, evaluated the inclusion of adverse event analysis, and assessed the level of evidence using a critical appraisal tool. Results : The study revealed a significant increase in prospective studies (22.7％ vs. 76.9％, p＝0.0002) and adverse event analysis (0％ vs. 15.4％, p＝0.0040) for Versius compared to da Vinci. In addition to analyzing adverse events by severity, two studies on Versius provide a detailed analysis of the association between devices and/or user error. Versius achieved Level 3 evidence earlier and in more studies than da Vinci. Discussion : The findings suggest that research designs in RAS have improved in prospective studies, adverse event analysis, and evidence levels. Evaluating the impact of technical and human factors is crucial in RAS for improving patient safety and ensuring the timely provision of information on the proper use and improvements of medical devices. Conclusion : The research design of post-market clinical studies in RAS reflects a shift towards higher-quality evidence, enhancing safety and effectiveness. Evaluating prospective studies in the post-market early stage of RAS, considering human factors, may lead to improved therapeutic efficacy and safety.

Background : There is a globally growing movement to promote the utilization of real-world data (RWD) in pharmaceutical regulatory approval. In Japan, there has been little progress in discussions regarding their utilization in new drug approvals. Objectives : This study aimed to clarify the current situation regarding the utilization of medical information databases in new drug approvals by the FDA, to promote discussions regarding their utilization in Japan. Method : We identified FDA-approved products that may have utilized RWD, reviewed the review reports for these products, and identified the background factors of RWD utilization, such as RWD type, role in submission packages, data format type, and whether or not confounding adjustment was applied. We also categorized the contribution level of RWD to the approval decision and evaluated the contribution for each background factor. Results : We identified 27 products with RWD utilization in FDA submissions in 2019-2021. There were many patterns of RWD utilization for effectiveness, and the degree of contribution to approval varied among background factors. Medical information databases were utilized in 5 products, with contributions to approval found in 1 product. Conclusion : The low contribution of medical information databases might be derived from the insufficient preliminary investigation at the planning stage due to the lack of experience in RWD utilization. To create real world evidence from medical information databases, it is essential to fully understand the information generated and accumulated in daily medical practice, select an appropriate database that meets the objectives, and apply an appropriate design and statistical method.

 Various regulatory incentives are established in many countries to encourage drug development for unprofitable diseases, such as rare diseases or neglected tropical diseases. One example is the Priority Review Voucher (PRV) program in the United States, which grants transferable priority review rights to companies that successfully develop drugs for pediatric rare diseases or neglected tropical diseases. Since 2007, at least 53 PRVs have been issued, with an average sale price of approximately 100 million USD. Because the policy research on PRV have not been conducted in Japan, this study newly defined “Transferable Regulatory Privilege” (TRP) as a transferable regulatory incentive optimized for the Japanese regulatory system and estimated its economic value. Considering the Japanese regulatory system, we hypothesized four transferable regulatory privileges : (1) priority review, (2) patent extension, (3) premium pricing, and (4) mitigation of price reduction. The economic value of each option was calculated as the incremental net present value (NPV) before and after applying TRP to blockbusters. The NPV of the base scenario was 77.6 billion yen. The economic values of the options were : (1) 6.6 billion yen per 6-month review acceleration, (2) 930 million yen per 6-month patent extension, (3) 3.7 billion yen per 5％ premium pricing, and (4) 5.6 billion yen per 1％ price reduction waiver. Considering the minimum value necessary to incentivize companies and balance between stakeholder benefits and costs, option (1) was suggested as the best choice. This study evaluated the economic value and feasibility of TRP optimized for Japan for the first time.

 AstraZeneca participated in the Japanese Pharmacopeia (JP) and United States Pharmacopeia (USP) Prospective Bilateral Harmonization Pilot Program for drug substance and drug product monographs as part of efforts toward international harmonization of pharmacopoeial standards. AstraZeneca is creating monographs for “Dapagliflozin Propylene Glycolate Hydrate” and “Dapagliflozin Propylene Glycolate Tablets” with the aim of international harmonization of pharmacopoeial standards. The aim of this initiative is to reduce various burdens and improve the efficiency of global supply chains, and it examined the benefits and challenges for companies. Specifically, AstraZeneca responded to technical and regulatory challenges by establishing new identification tests, introducing streamlined descriptions, unifying general test methods, and examining system suitability. This pilot program served as a model case showing the potential for expanding international harmonization of pharmacopoeial standards and provided a valuable opportunity for companies to directly participate in international harmonization activities. Through this initiative, AstraZeneca hopes to ensure quality of pharmaceuticals and stable product supply, and to serve as a pioneer in international cooperation and harmonization of pharmacopoeial standards.

 Pharmaceutical excipients are essential components of drug formulations, playing crucial roles in improving their stability, solubility, manufacturability, and ease of administration. However, the globalization of pharmaceutical supply chains has introduced new challenges in quality control. In particular, recent cases of diethylene glycol (DEG) and ethylene glycol (EG) adulteration in pharmaceutical excipients have emerged as significant public health threats worldwide. Various regulatory measures have been implemented to address these issues, including specific purity tests in individual pharmacopoeias and the World Health Organization’s proposed two-level approach for DEG/EG testing. To ensure both quality assurance and a stable supply of pharmaceutical excipients, strengthening international cooperation among regulatory authorities, pharmaceutical companies, and excipient manufacturers is critical. In this review, we discuss the regulatory framework for pharmaceutical excipients, with a focus on the Japanese Pharmacopoeia, and efforts toward international harmonization through the Pharmacopoeial Discussion Group.

 Due to the globalization of the supply chain of pharmaceutical substances and products, the importance of harmonization of pharmacopoeias is increasing. Therefore, the Japanese Pharmacopoeia (JP) and the United States Pharmacopeia (USP) bilaterally work on harmonization of “Dapagliflozin Propylene Glycol Hydrate” and “Dapagliflozin Propylene Glycol Tablets” monographs. In the current JP drafts, the main points that have been harmonized are (1) describing concentration (instead of weight) in the test procedure, (2) application of＜2.00＞chromatography, and (3) no Ultraviolet-visible spectroscopy in the identification. The main points that can’t be harmonized are the content specifications of the tablet, dissolution, and system suitability. It is believed that the completion of this project and the harmonization of the monographs between the JP and USP pharmacopoeias will reduce the burden on stakeholders and contribute to the supply of high-quality medicines.

 In recent years, there has been concern about the increase in “drug loss” : drugs that are approved in Europe and the United States but are not developed in Japan. Factors contributing to this include a lack of understanding of Japan’s drug discovery environment and pharmaceutical affairs system. Meanwhile, in Europe and the US, guidance and concept papers on the development of drugs using innovative technologies are published early on, leading to exchanges of opinions with industry and the promotion of development. In the Fifth Medium-Term Plan, the Pharmaceuticals and Medical Devices Agency (PMDA) advocates the implementation of consultations and reviews that accurately respond to innovation, such as dissemination of Early Consideration and development of clinical evaluation guidelines based on the latest scientific knowledge. Early Consideration is a reference information for practical application of innovation and promotion of development of innovative drugs, although it is at the stage where information is not collected sufficiently. It shows the view of the reviewers regarding the direction of development at that time. The aim is to provide consultation services and approval reviews for new pharmaceuticals that appropriately respond to innovation by compiling and publishing Early Consideration on issues such as the practical application of innovative technologies and the development and evaluation of pharmaceuticals, and by formulating new clinical evaluation guidelines and revising existing ones based on the latest scientific knowledge. This article introduces the background and outline of eight Early Consideration documents published by the PMDA by January 2025.