The utilization of disease registry is expected to conduct clinical trials/researches effectively, accelerate drug development, and deliver drugs on market speedy. MHLW and AMED started clinical innovation network (CIN), which includes six projects to prepare the high-quality registry in Japan. The status of registry utilization was surveyed in Japan, US, and Canada and made comparison between in Japan and US. US has 53 registries excluding 5 survey registries and Japan has 154 registries. 28 registries were matched both US and Japan based on diseases. The quality of registry has still improvement points for using those in new drug application in Japan. That in US was unclear based on the information of website. The scale of registries was various. The case of including registry data in new drug submission was not quite sufficient among countries. No registry based randomized clinical trial (Registry Based RCT) in Japan was conducted. On the other hand, five Registry Based RCTs were conducted by the State, or the hospital network in US. In conclusion, the utilization of registry in both countries is under development. Pharmaceutical industry, academia, and the government should collaborate to implement registry data to deliver innovative drugs to patients as soon as possible.

Establishing a prescription refill system in clinical sites is being considered in Japan, which would involve setting long-term prescriptions as these are the base of such a system. The purpose of this paper is to clarify the status of long-term prescriptions by investigating the current frequency of medical visits in patients with hypertension who are usually prescribed medication for a relatively long period of time. Furthermore, we investigated the burden on patients to clarify expectations for the refill system from the perspective of patients. The participants in the present study were 172 patients whose only disease was hypertension that was well-controlled with 1-2 antihypertension drugs, and who visited health insurance pharmacy chain stores over six days in May 2017. We collected data on the current frequency of medical visits, patients’ desired frequency of medical visits, and personal management of blood pressure. The results revealed that 79.7% (n=137) of patients visited a medical facility once monthly. Of the total number of patients, one fourth wished to reduce this frequency, and over half of these responded that the reason was that their “blood pressure was stable.” Additionally, 90.7% (n=156) of patients owned a blood pressure meter, but only 32.0% (n=55) measured their blood pressure daily. The results of a comparative analysis of different ages with respect to medication adherence showed that significantly fewer people aged 66 and over forgot to take their medication. In sum, there were few long-term prescriptions subject to refills among patients with stable hypertension. In order to decrease the burden on patients and implement an appropriate long-term prescription system, the pharmacist is expected to explain the importance of medication adherence to the patient and support at-home blood pressure monitoring and self-management.

EFPIA Japan and PhRMA Japan conducted a joint questionnaire survey for development of regenerative medical products. In November 2014, regenerative medical products were newly defined by the Pharmaceutical and Medical Device Act, and conditional/time-limited approval system was also legislated in Japan leading the world. This new legislation, however, does not seem to direct foreign-affiliated pharmaceutical companies to proactively engage into development activities in Japan. For that reason, this joint survey was planned to understand the present situation of introduction for regenerative medical products from Headquarters and also to find issues and/or concerns on development in Japan. Twenty nine out of thirty three EFPIA Japan or PhRMA Japan member companies responded on this survey (88% on response rate). Fourteen companies (48%) had regenerative medical products in Headquarters. Total number of under development or launched products were 31, and 12 out of them were introduced or planned to introduce into Japan. The detailed investigation revealed a large development gap especially for the products which had already begun clinical trial in Headquarters. It implied a drug lag in future. Regarding issues and/or concerns to introduce the products into Japan, CMC and quality related matters were the most frequent, followed by domestic regulation like Cartagena law and uncertainty of business case.

Genomic data that are obtained from clinical studies could be used in various ways, such as, optimization of patient therapy by the identification of genomic biomarker underlying variability in drug response, and the identification of new drug targets by a better understanding of pharmacological and pathological mechanisms. There was no harmonised International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guideline on genomic sampling and data management from clinical studies at that time. Thus, “ICH E18: Guideline on genomic sampling and management of genomic data” has been developed to foster interactions amongst stakeholders and to encourage genomic research in clinical studies. In this article, we present an overview of the ICH E18 guideline and a development process of this guideline from standpoint as regulatory members in the Expert Working Group.

The Project for the Promotion of Progressive Medicine, Medical Devices, and Regenerative Medicine by the Ministry of Health, Labour and Welfare was established in 2012. One of the projects that was adopted, “evaluation procedures for the efficacy and safety of pharmaceuticals that use pharmacogenomics,” conducted a non-clinical study and early-phase clinical trials on investigational drugs, and evaluated the effects of genetic polymorphisms of drug-metabolizing enzymes and transporters primarily involved in those trials. This work was conducted at the Graduate School of Pharmaceutical Sciences and Faculty of Pharmaceutical Sciences, Tohoku University, in cooperation with the National Institute of Health Sciences and Pharmaceuticals and Medical Devices Agency. As a main achievement of this project, we compiled the current scientific information on genetic polymorphisms, which would be evaluated during these non-clinical studies and phase I clinical drug trials in Japanese subjects/patients, obtained through this project and scientific papers. This points to consider document focuses on the genetic polymorphisms of drug metabolizing enzymes (cytochrome P450 enzymes among others) and transporters with reported functional significance and allele frequencies of 1% or higher in the Japanese population, and outlines in vitro procedures for the evaluation of their functional effects on investigational drugs. This document also provides examples of genetic polymorphisms and somatic mutations of proteins involving drug efficacy or adverse drug reactions. We believe that this information will be helpful to accelerate future research and continued discussions on drug development using pharmacogenomic information (e. g., dose adjustment and avoidance of adverse reactions) in Japan and possibly other East Asian countries. In addition to introduce this document, related guidelines/guidances of ICH, EMA and FDA were outlined.

ICH E18 was enacted to provide harmonised principles of genomic sampling and management of genomic data in clinical studies, and to encourage genomic research within clinical studies. In Japan, it was issued in January 2018. Comprehensive genomic sampling is encouraged during all clinical drug development stages in the guidelines, of which widely usage such as investigation of biomarker assay methodology, causality with disease, drug sensitivities, and/or safety assessment, should be addressed in the informed consent. Furthermore, this guideline is hopefully enhancing worldwide genomic data integration. However, some genomic data is legally defined as privacy information in Amended Act on the Protection of Personal Information by Japanese Government. Therefore, clinical study sponsors need to provide much further detailed information regarding the genomic sampling and analysis for study participants in clinical studies in Japan. For providing solution those issues, the Japan Pharmaceutical Manufacturers Association published update of the “Points to Consider for Pharmacogenomics in Clinical Studies of Pharmaceuticals (Preliminary version)” published in 2008 and renamed as “Points to consider to collect genomic samples in clinical studies of Pharmaceuticals” in 2018. In this article, the major revisions would like to be outlined, including of deletion of previous categories of genomic analysis, updates of anonymization procedures, and recommends of informed consents documentation.