apan 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.

Publication of PMDA’s Perspectives as Early Considerations

To facilitate efficient and prompt dialogue between developers and PMDA, the agency has been issuing “Early Consideration” documents since 2024 that provide key discussion points related to the practical application of innovative technologies in drug development and evaluation as an early-stage reference, to promote pharmaceutical innovation and development.

Amendment of the Pharmaceuticals and Medical Devices Act in May 2025

The scope of the Conditional Approval System was expanded in 2025 to enable earlier patient access to pharmaceuticals with particularly high medical need, such as treatments for rare or serious diseases. Under this system, a pharmaceutical product may be approved if its clinical usefulness is reasonably predicted during the exploratory trial phase, with provisions in place to revoke approval. At the same time, confirmatory studies of efficacy and safety after approval will be required as a condition of marketing authorization. If these evaluations fail to confirm the expected efficacy and safety, the authorization may be revoked.

PMDA’s Reach Extends Beyond Japan

To ensure that start ups based in Europe and the US fully understand Japan’s pharmaceutical regulations and the PMDA’s support system without facing language barriers, PMDA has expanded its outreach beyond Japan.

In 2024, the PMDA established two overseas offices. The Asia Office, opened in Bangkok in July 2024, promotes regulatory harmonization with Asian countries and supports the improvement of regulatory environments to facilitate smooth clinical development across the region. Japan is recognized as a “reference country” by many Asian nations, and the number of countries recognizing it continues to increase steadily each year.

The Washington D.C. Office, established in November 2024, aims primarily to strengthen collaboration with the US Food and Drug Administration (FDA) and promote pharmaceutical development in Japan by overseas start ups. In addition to its role as an information portal on Japan’s pharmaceutical regulations, the office launched comprehensive consultation services in English during US East Coast Time hours in March 2025. As of June 2025, it had received eight consultation requests. This initiative is expected to foster an accurate understanding of Japan’s regulatory system and encourage early inclusion of Japan in new drug development programs. The Washington office further collaborates with key organizations involved in business expansion and clinical trials in Japan, such as the Japan External Trade Organization (JETRO), the National Cancer Center Japan (NCC), and the Japan Agency for Medical Research and Development (AMED), thereby extending support beyond regulatory matters.

While the roles of the Asia Office and the Washington DC Office differ, the PMDA envisions future collaboration between these overseas offices to build an ecosystem in which innovative drugs developed in the US gain approval in Japan and are subsequently disseminated more rapidly throughout Asia.

In addition to formal consultation services provided by the Washington D.C. Office and the PMDA headquarters, the PMDA offers informal opportunities for stakeholders to ask questions about Japanese pharmaceutical regulations. At the 2025 DIA Europe Annual Meeting and 2025 DIA Global Annual Meeting, the PMDA secures private consultation rooms staffed by reviewers for on-site consultations. These sessions welcome straightforward questions about Japan’s pharmaceutical regulations, procedures, and the practical flow of formal consultations, as well as general inquiries about systems such as Orphan Drug Designation. This unique initiative plays an important role in ensuring that global pharmaceutical developers gain an accurate understanding of Japan’s regulatory framework and promotes drug development in Japan.

To effectively disseminate information about Japan’s pharmaceutical regulations and the PMDA’s activities globally, the PMDA has also revamped its newsletter, PMDA Updates. The refreshed newsletter now targets a broader range of stakeholders, including domestic and international industry players and start ups, in addition to regulatory authorities, and aims to provide clear and accessible information on the PMDA’s activities and Japan’s regulatory environment.

Toward True Global Execution of Pharmaceutical Development

Asia is a major market in global pharmaceutical development. To expand into the region, obtaining approval in Japan is important, as the country aligns with internationally harmonized regulatory standards and serves as both a gateway and a “reference country” for other Asian nations.

Emerging Biopharmaceutical Companies (EBPs), which have become key players in recent pharmaceutical development, tend to focus primarily on the US market. Studies indicate that EBPs, unlike larger pharmaceutical companies, often conduct clinical trials in single countries (see Exhibit 57 of this report). However, strategies that begin global development outside the US, such as in Japan or other parts of Asia only after gaining US approval, often result in duplicative efforts, including additional clinical trials and regulatory tasks. This prolongs the overall development timeline from the start of US development to product launch overseas (e.g., in Japan). PMDA highly recommends that developers design study and development plans from an early stage that include non-US regions (such as Japan) for efficient global development. Initiating early consultations with the PMDA enables developers to leverage regulatory support in Japan and facilitates efficient development. Accordingly, companies considering expansion into Japan and Asia are strongly encouraged to engage in early discussions with PMDA.

Moving Forward

After reading this article and learning about the large Japanese market, the highly predictable process from application to approval and pricing, and the open approach of its regulatory authorities, some readers may wish to engage in discussions with PMDA regarding the inclusion of Japan for marketing authorization applications for their products. The authors recommend first contacting the Washington DC Office, which provides English-language support during US East Coast Time hours.

PMDA will continue to explain Japan’s regulatory system and discuss challenges related to drug development targeting Japan through diverse opportunities. Moreover, free individual consultations about Japan’s pharmaceutical regulations will be held at various medical and pharmaceutical conference venues. We hope that awareness of these PMDA initiatives grows, encouraging developers to incorporate Japan into their development strategies and leading to an increase in applications submitted in Japan.