Writing in the September 2025 edition of DIA’s Global Forum magazine, Seiko Usami, Shinichi Noda, Daisuke Koga, and Naoyuki Yasuda of the Pharmaceuticals and Medical Devices Agency (PMDA), Japan’s regulatory body, outline the benefits for global pharma of including Japan in their drug development activities.
Japan is one of the world’s largest pharmaceutical markets. It also offers multiple advantages from a regulatory perspective.
The Regulatory Appeal of Developing Pharmaceuticals in Japan and for Japan
Japan’s pharmaceutical regulatory framework has been developed by the Pharmaceuticals and Medical Devices Agency (PMDA) in alignment with the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guidelines and is harmonized with regulations in Europe and the US. Japan offers several key regulatory advantages to drug developers, including the world’s fastest regulatory reviews, support through extensive consultation services, and a highly predictable process from application to approval and pricing. In addition, universal health coverage guarantees access to medicines for all Japanese citizens nationwide.
Regulatory Authorities in Japan Are Keen on the Development of Innovative Medicines for Early Patient Access
Promotion of Multiregional Clinical Trials (MRCTs)
A 2023 regulatory notification clarified that additional phase 1 trials in Japanese subjects are not required if safety and tolerability in Japanese participants can be adequately explained based on existing data, and if safety is clinically acceptable and manageable. Safety of the investigational drug is assessed comprehensively, considering safety information, such as the safety profile of the investigational product and the potential influence of ethnic factors. The judgment will be based on an assessment of whether Japanese participants in the MRCT may be exposed to any greater risk than non-Japanese participants, and whether their safety in the proposed dosing regimen is both clinically acceptable and manageable. The PMDA has traditionally made case-by-case decisions based on scientific discussions without necessarily always requiring Japanese data. This 2023 notification explicitly clarified the agency’s position. The PMDA promotes MRCTs, and also expects that, when drug developers formulate their development strategies, they will consider including Japanese participants in MRCTs in order to address the lack of Japanese data in the early stages of development.
Proactive Operation of the Orphan Drug Designation System
PMDA revised the criteria for the orphan drug designation system in January 2024. Designations are now actively granted at the early stages of development and for products addressing high unmet medical needs, such as those targeting intractable and serious diseases or pediatric populations. As a result, the number of orphan-designated products reached 86 in fiscal year 2024, more than doubling from 36 in the previous year. An additional notification issued in the same year outlined the conditions under which marketing authorization applications may be submitted without clinical trial data from Japanese patients, which may have also contributed to the increase in orphan-designated products. These conditions were established to minimize the risk of Japanese patients losing access to medicines, particularly for rare diseases, when confirmatory clinical trials have been conducted exclusively overseas.
Promotion of Development and Introduction of Pediatric and Orphan Drugs
To promote the development and introduction of pediatric and orphan drugs, PMDA established the Consultation Center for Pediatric and Orphan Drugs Development in July 2024 to provide the regulatory consultation support necessary for development of these products. Consultation fees at this center are partially or fully subsidized by government grants. This initiative aims to encourage developers to create pediatric development plans, strengthen the PMDA’s organizational capacity and consultation environment, expedite and expand the orphan drug designation process, and accelerate the development of “publicly solicited products” as discussed by the Study Group on Unapproved and Off-label Drugs of High Medical Need.
