Release of Clinical Trial Guidance to Facilitate the Speedy and Accurate Approval and Development of Medical Devices in Japan

1. What was released

On November 17, 2017, Japan’s Ministry of Health, Labour and Welfare (MHLW) issued an Administrative Notice releasing an appendix titled Clinical Trial Guidance to Facilitate the Speedy and Accurate Approval and Development of Medical Devices (dated March 2017). The notice asks stakeholders to consult the guidance (and reference materials) when developing medical devices.
Reference: Clinical Trial Guidance (PMDA PDF 000237132)

PMDA also lists this document on its English Regulatory Information (Medical Devices) page as a key notice for medical device approval/certification review.
Reference: PMDA Regulatory Information – Medical Devices

2. Why the guidance was needed

The notice highlights a central challenge in device regulation: medical devices are frequently upgraded or improved, so developers must assess whether clinical trials are needed based on the device’s features and then consider appropriate trial design (including target population size).

In the guidance preface, the lifecycle perspective is emphasized: earlier marketing (including for serious diseases with unmet needs) may be promoted even with limited pre-marketing clinical data, provided that benefit–risk balance is maintained through consistent pre- and post-marketing actions under a risk management plan, and that real-world evidence such as registries and use-results surveys is effectively used.

3. How the guidance is positioned (what it is—and is not)

The guidance is described as a practical consolidation of “currently acceptable concepts” on clinical trial necessity and common matters across device fields, prepared with reference to existing clinical trial-related notifications. It is not binding and may be revised as technology and knowledge evolve; clinical evaluation should remain flexible and scientifically rational for each device.

4. Core framework: determining whether a clinical trial is necessary

4.1 Basic principle

The guidance reiterates that clinical trials should be conducted when clinical efficacy and safety cannot be evaluated sufficiently using performance testing, non-clinical studies (e.g., animal studies), or existing literature. It references the long-standing MHLW “fundamental notification” that defined the scope of cases where device clinical study data is necessary, and frames clinical trial necessity as a case-by-case, comprehensive determination based on device characteristics, comparability to approved devices, and non-clinical evidence.

4.2 Key preparatory investigations

The table of contents shows the guidance expects structured prior investigation, including the development concept, clinical positioning, differences from approved similar devices, and conceptual requirements, before concluding whether trials are needed.

4.3 Handling foreign clinical trial results and “trial-unnecessary” cases

The guidance explicitly includes sections on example cases where clinical trials may not be necessary and on how to handle results from clinical trials in foreign countries, reflecting Japan’s interest in efficient global development strategies where appropriate.

5. Designing clinical trials efficiently: design and sample size concepts

The guidance dedicates an entire section to basic concepts for:

• clinical trial design, and
• sample size,
  recognizing that device trial designs are not uniform across technologies and that feasibility matters due to smaller production/distribution volumes compared with drugs.

6. Linking pre- and post-marketing evidence: four practical “handling” scenarios

A notable feature is a section on pre- and post-marketing actions, organized into practical scenarios, including:

• a conditional early approval concept for innovative medical devices (noted as the situation as of July 2017),
• handling clinical trials to evaluate compatibility with Japan’s medical environment,
• handling improved devices where incremental clinical value is small and serious risk is unlikely, and
• evaluation of diagnostic devices that measure physiological parameters as potential reference information for diagnosis.

For regulatory teams, this structure signals an expectation that development strategies should be end-to-end: determine what must be proven pre-market, define how uncertainty will be managed through proper-use measures, and plan what evidence will be generated post-market to confirm performance and safety in real-world use.

7. Shared terminology: what counts as a clinical trial in this context

The guidance defines “clinical trial” as a clinical study conducted in accordance with GCP (in Japan and/or abroad) for obtaining marketing approval, distinct from clinical evaluation reports based on literature, and it defines pivotal clinical trial terminology used for evidence planning.

8. Practical checklist for applicants (derived from the guidance’s intent)

• Start with a structured necessity assessment: clearly articulate device novelty, clinical positioning, and comparability; map what can be concluded from non-clinical and literature evidence before proposing a trial.
• Design trials proportionate to the question: align design and sample size with the specific objective, recognizing feasibility constraints common in device development.
• Plan the lifecycle evidence package: integrate pre-market evidence with post-market measures (risk management planning, proper-use controls, real-world data capture) when appropriate to support timely access while protecting patients.
• Use PMDA’s published regulatory index to triangulate applicable notices alongside this guidance when planning an approval pathway. :contentReference[oaicite:15]{index=15}