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Japan’s clinical trial transformation: Building a global future

Japan is known for strong scientific expertise and high-quality data, and is home to the world’s fourth largest pharmaceutical market.1 However, its clinical trial ecosystem has long presented challenges. Factors, such as complex operational requirements, lengthy review times and misalignment with global standards, among others, have led to relatively low participation in global clinical trials. 

Japan has also been struggling against drug lag and drug loss, in which new drugs that have been approved in other countries have experienced developmental delay or have not yet been developed domestically. This problem is particularly pronounced in paediatric and orphan diseases, since the relevant populations are relatively small.

In recent years, Japan has begun working to counteract drug lag and loss, and modernise its approach to clinical trials.Through a variety of reforms, it aims to establish a globally aligned, efficient and sustainable clinical trial ecosystem that supports simultaneous global development and delivers innovative therapies to patients without delay. Here, we explore some of these changes and why they are making Japan a more attractive choice for clinical trials.

MRCT reform

Multi-regional clinical trials (MRCTs), studies that are conducted across several countries or regions under a single protocol, are a central mechanism of global drug development. As part of its efforts to combat drug loss, Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has prioritised creating an environment for MRCTs that aligns more closely with global regulatory expectations. 

A key element of this alignment is a notification published in December of 2023, which addressed the previously maintained requirement that, for an MRCT to take place in Japan, it must be preceded by a Phase I trial consisting of Japanese participants.2 Under the revised guidance, the need for a study involving Japanese participants prior to an MRCT can be assessed on a case-by-case basis using criteria such as the safety profile of the study drug and the likelihood that ethnic factors will affect its safety and efficacy.3 If these risks are low, an early trial with Japanese participants may not be necessary, lowering a major barrier to conducting MRCTs in Japan.

Accelerated pathways

To encourage drug development and provide faster market access, Japan has implemented and continued investing in several accelerated approval pathways. One of these is the Sakigake Designation System, which promotes innovative new medical products for the treatment of severe illness, and functions similarly to the U.S. Food and Drug Administration’s Breakthrough Therapy Designation and the European Medicines Agency’s Priority Medicines scheme.4 It supports development through measures such as prioritised consultation with the PMDA, evaluation of documents prior to application and regulatory review within 6 months, compared to 9 months for priority review or 12 months for standard review.

Another accelerated pathway is the Conditional Early Approval System, which was established to put highly effective drugs for serious disease into practical use as early as possible.5 Under this system, drugs demonstrating a certain degree of safety and efficacy may receive conditional approval without confirmatory trials, although safety and efficacy must later be reexamined in the post-market setting. To qualify, a drug must demonstrate superiority over existing treatments for a serious disease, and confirmatory clinical trials must be difficult or impractical to conduct due to factors such as a limited number of patients.

Extensive consultation systems

A further key element supporting Japan’s clinical trial ecosystem is the PMDA’s extensive consultation framework. This framework helps to de-risk and streamline clinical trials by giving sponsors the opportunity to receive guidance and advice on clinical trials, and data for regulatory submissions.6 Recent efforts have increased the availability of consultations for key groups. 

In July of 2024, the Consultation Center for Pediatric and Orphan Drug Development was established to promote drug development in paediatric and orphan diseases through scientific guidance, as these areas are particularly impacted by drug loss.7 That same year, the PMDA began establishing overseas offices in locations including Thailand and the US to disseminate regulatory information and support startups based outside Japan.7,8 For sponsors unfamiliar with the Japanese regulatory landscape, these initiatives facilitate better preparation for late-stage clinical trials and help create more predictable review timelines.

Benefits of a renewed ecosystem

Efforts to improve the clinical trial ecosystem have numerous benefits for many different stakeholders. For global sponsors, the reduced need for Japanese-specific early phase data lowers barriers to entry. In general, MRCT-friendly policies are increasing alignment with global drug development timelines and practices, making the implementation of such trials much smoother. 

For the Japanese health system, in addition to reducing drug loss, these changes lead to easier participation in overseas-led global trials, expanding access to innovative molecules. For Japanese sponsors, they provide the opportunity to scale internationally by using MRCTs as a global launch platform. Furthermore, the regulatory flexibility introduced through these reforms reduces the cost burden for smaller Japanese biotechs, allowing them to flourish.

A promising future

Japan’s focus on repositioning has begun to build local industry momentum. Its participation in MRCTs has continued to rise, and exceeded 60% of all trials in Japan starting in 2021, confirming a more globally compatible environment.9 In 2025, 24 drugs with new active ingredients had been approved as of September, indicating a healthy inflow of new drug product submissions.10 With global and domestic sponsors alike currently pursuing high-value programs in Japan, the country has become an increasingly attractive destination for clinical trials.

References

  1. Pharmaceuticals and Medical Devices Agency. “Why Japan Should Be on Your Map for Medical Innovation.” PMDA Updates, Summer 2025. https://www.pmda.go.jp/files/000276746.pdf
  2. Ministry of Health, Labour and Welfare. “Basic principles for conducting phase 1 studies in Japanese prior to initiating multi-regional clinical trials including Japan for drugs in which early clinical development is preceding outside Japan.” Notification No. 1225-2. 25 Dec., 2023. https://www.pmda.go.jp/files/000266727.pdf
  3. Ministry of Health, Labour and Welfare. “Q&A for basic principles for conducting phase 1 studies in Japanese prior to initiating multi-regional clinical trials including Japan for drugs in which early clinical development is preceding outside Japan.” 25 Dec., 2023. https://www.pmda.go.jp/files/000266728.pdf
  4. Pharmaceuticals and Medical Devices Agency. “Strategy of SAKIGAKE by MHLW.” https://www.pmda.go.jp/english/review-services/reviews/advanced-efforts/0001.html. Accessed 18 May 2026.
  5. Inoue, S. “Regulatory Update from MHLW/PMDA.” 5th Joint Conference of Taiwan and Japan on Medical Products Regulation. 1 Dec., 2017. https://www.pmda.go.jp/files/000221880.pdf
  6. Pharmaceuticals and Medical Devices Agency. “Consultations.” https://www.pmda.go.jp/english/review-services/consultations/0002.html. Accessed 18 May 2026.
  7. Kohno, Yoichi, et al. “Expediting Drug Development in Japan: A PMDA Perspective.” Clinical Pharmacology & Therapeutics, vol. 118, no. 6, Dec. 2025, pp. 1262–64. https://doi.org/10.1002/cpt.70022.
  8. Otsubo, Yasuto. “PMDA Initiatives to Enhance Drug Development via Multi-Regional Clinical Trials.” Translational and Clinical Pharmacology, vol. 32, no. 4, 2024, p. 173. DOI.org (Crossref), https://doi.org/10.12793/tcp.2024.32.e16.
  9. “Regulatory Process Overview – ACTUAL RESULTS.” Japan CRO Association, https://www.jcroa.or.jp/english/actual-results/. Accessed 18 May 2026.
  10. Pharmaceuticals and Medical Devices Agency. “List of Approved Drugs April 2004 to September 2025.” https://www.pmda.go.jp/files/000279952.pdf. Accessed 18 May 2026.
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