With a recent accumulation of numerous genome data, personalized medicine using genetic information begins to be realistic. In particular, cancer chemotherapy has entered the era of the molecular targeted therapy that selectively prevents the growth of cancer cells expressing driver oncogenes as represented by a mutated EGFR gene in lung cancer. In Japan, “companion diagnostics (CoDx)”, which are in vitro diagnostic drugs to maximize the benefits of molecular targeted drugs, were newly defined in 2013 and several CoDx have been already approved. This review provides an overview of CoDx in relation to the molecular targeted therapeutics against cancer.

As the pharmaceutical company’s promotional activities sometimes communicate false or misleading information ultimately detrimental to the health of consumers/patients, the establishment of a third-party regulatory system for new drug promotional activities is necessary. This study explores the possibilities for such a system in Japan, by considering, as an example, the US Food and Drug Administration’s (FDA) existing regulatory system for the pharmaceutical company’s promotional activities. In this study, the current status of drug advertising violations in the USA was analyzed, based on a review of warning letters and untitled letters issued by the FDA. At the FDA, expert examiners in every medical field and specialty effectively review enormous quantities of specialized promotional materials. To counter violations in settings where surveillance is difficult, e. g. clinics and meetings, the FDA targets on-site health care practitioners through its Bad Ad Program, which incorporates both educational and violation-reporting capabilities. As violations in American electronic media have recently been on the rise, the monitoring of information on social media, e. g. Facebook, will become increasingly important.

Background: The Hokkaido University Graduate School of Medicine has been carrying out a project to create cell therapy products for stroke. The aim of this project includes establishing guidelines and initiating personal exchanges between academia and Pharmaceuticals and Medical Devices Agency (PMDA). It is worthwhile for the future development of medicinal products by academia to widely share among academics the knowledge and experiences gained during a project. Objectives: The objective of mid-term review is to elucidate the challenges facing human cell therapy product development in academia and identify potential improvement measures. Methods: The track records of the project, including meeting minutes, were reviewed. Challenges and improvement measures were identified through group discussion from the viewpoints of both the regulatory authority and academia. Results: As a result of our review of the project, we identified periodic review by program officers and consultation with PMDA as good points, insufficient understanding of regulation and obscure roll assignment in the development team as project delaying factors, defining roles and making appropriate assignments as improvement measures to be implemented, and the utilization of information and experience from other clinical trials as a recommendation for the future. Conclusions: In order to accelerate the development of medicinal products, it is important for academia to take full advantage of both information from their clinical trial experience and knowledge concerning project management accumulated in the organization.

Continuous Manufacturing (CM) is a manufacturing process in which materials and/or mixture are continuously charged into the process and products can be taken out without interruption. It can produce the required amount of products with desired quality whenever you want. Although many benefits are expected such as human error reduction and/or the realization of production control as to demand, any regulatory views on the CM have not been shown yet. In order to facilitate the smooth introduction of the CM in Japan, it is necessary to address issues of the CM together with industries, regulators and academia and to share our knowledge.

Nanotechnology is a leading tool for manufacturing highly functional drug formulations. The formulation techniques using nanotechnology include liposome, polymer nanoparticle, iron nanoparticle, nanoemulsion such as lipid microsphere, micelles, vaccine adjuvant, and nanocrystal technology. Some drug products using these techniques have been approved in Japan. Most of them have been used for the low-molecular-weight active substances. However, these techniques are recently used as the carriers for nucleic acids or proteins. Internationally, there has been a progress in nanomaterial measurement methods, development of reference materials, consensus-based documentary standards for nanotechnology. These activities are also important issues for us. Furthermore, we are involved in the development of new measurement methods for quality attributes which reflect the product performance of nanomedicines. We introduce our research to develop the evaluation method for bending modulus of liposomes using atomic force microscopy.

Nucleic acid drugs have recently attracted considerable attention as a novel category of pharmaceutical agents following the development of small molecular therapeutics and antibody-based drugs. While to date no more than five nucleic acid drugs have been approved for use around the world, the number of clinical trials of nucleic acid drugs being carried out currently stands at between 100 and 200, and this vigor is unlikely to abate in the near future. However, there are currently no guidelines that specifically address nucleic acid drugs. Consequently, examining the regulation of product quality, non-clinical safety, and clinical features is important for the promotion of nucleic acid drug research and development. This paper outlines the current status of research involving nucleic acid drugs and discusses a number of points related to the challenges in regulatory science pertaining to this drug class from the particular standpoint of quality control, including issues associated with synthesis, purification, analysis, chemical modification, and the management of higher-order structures.

Biologics are important in the pharmaceutical market worldwide, and future growth including biosimilars is expected. On the other hand, it has characteristics different from low molecule drugs in manufacturing development, manufacturing facilities, etc., and there are many problems unique to biologics. Also, some of these issues reflect the trend of global drug development trends, and some are specific to Japan. In this article, from the standpoint of industry, we state opinions regarding manufacturing, development and regulatory matters on biologics to in Japan. Viewpoints include biomanufacturing infrastructure, adaptation to expedited programs on drug development and human resource development for biomanufacturing.

In October 2015, the International Council for Harmonisation (ICH), formerly the International Conference on Harmonisation that was set up in 1990, established the ICH association, a legal entity under Swiss law, aiming for further expansion of ICH membership and global outreach for implementation of ICH guidelines. Given its successful track record of delivering more than 80 harmonized guidelines, development of harmonized guidelines continues to be the central role of the ICH. At the same time, almost all major topics for ICH guidelines have been covered, and with the growing diversity of the ICH membership their delivery is considered to become more challenging in the future. This article summarizes a process for new topic proposal and selection, and discusses the issues and future challenges surrounding new ICH topics by touching on the recent trends from the ICH Osaka meeting in November 2016. In that meeting, for the first time the ICH adopted a “strategic theme” to handle multiple guideline developments in a comprehensive manner. Along with planned “strategic themes,” new topic discussions are expected to arise in the future ICH. Considering this situation as a new opportunity, we believe that 1) facilitating dialogue between various stakeholders and 2) well-planned identification of, and training for advanced technical experts would be important in order to develop and implement effective ICH guidelines which facilitate delivery of new medicines for patients around the world. International harmonization starts with people; we believe this concept would trigger further enhancement of multiple platforms across the stakeholders which may lead to more ICH guidelines originating from Japan.