Notification of necessity for Japanese data when applying for drug approval (dated October 23, 2024)

On October 23, 2024, a notification was issued outlining the necessity of Japanese data when applying for drug approval.

The relevant notification is “Basic principles regarding Japanese data when confirmatory clinical trials for drugs used for rare diseases, etc. are conducted only overseas” (In Japanese. Notice from the Director of the Pharmaceuticals Review Division).

This notification is a response to the discussions of the “Committee on the Form of Pharmaceutical Regulation for Strengthening Drug Discovery and Ensuring Stable Supply, etc.”, which was held to advance discussions related to pharmaceutical regulations with the aim of eliminating drug lag/drug loss and strengthening drug discovery. (Click here for the report of the Committee in Japanese)

Contents of the Notification
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Contents of the Notification

This notification follows the report of the Committee.

First, at the beginning, it is stated that “in the approval review of pharmaceutical products in Japan, it is fundamental to evaluate efficacy and safety in Japanese people in the Japanese medical environment based on the results of international clinical trials or domestic trials in which Japan participates,” and that data on Japanese people is expected.

However, the following three cases are listed as cases in which it may be possible to “submit an approval application” without Japanese data.

When clinical trials that will be the main subject of evaluation have already been properly conducted overseas.
When it is difficult to conduct additional clinical trials due to an extremely small number of patients, etc. (As with conditional approval, cases in which the evaluation takes time or clinical trials due to acute diseases are also taken into consideration.)
When, based on the efficacy and safety information obtained, it is expected that the benefits in Japanese people will outweigh the risks overall.

If these conditions are met, a drug approval application without Japanese data may be approved, but it is stated that “considering the perspective of ensuring access to the drug to patients as much as possible and preparing for appropriate use in actual clinical practice in Japan, clinical trials (including expanded clinical trials) should be conducted in parallel with the drug approval application to collect as much information as possible on administration to Japanese patients.” In other words, it is indicated that drug approval applications may be submitted, but that clinical trials must be conducted in Japan at the same time.

In addition, it is indicated that conditional approval may be applied if clinical trial results are required in Japanese people. (In this case, although priority review will be given, the uncertainty regarding drug prices will increase.)

Contents of the Notification

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