Module 5 Presentations

18/07/2024

Waiver of Ph 1 PK study in Japanese before global study

Key Excerpt from the new guideline:

Regardless of conducting a phase 1 study in Japanese, it is important to assess the differences in PK and/or PD between Japanese and non-Japanese through measures such as collecting PK and/or PD data in Japanese in MRCTs prior to marketing authorization applications”.

Distribution to third parties is not allowed

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New Efforts to promote development of Orphan Drugs

Deregulation of ODD requirements

- Provided all conditions are met, It is now possible to obtain ODD in Japan with ONLY Ph1 data (Japanese data not required). Previously Ph 2 data was required.

- Relaxation of some eligibility criteria

- MHLW now accepts that products would be considered to meet criteria of medical needs also when existing drugs are not sufficient (Previously ODD could be considered only if no alternative product is available in JP, or significantly high efficacy/safety is expected vs existing product) - Patients subsets based on appropriate medical or pharmacological considerations, could be accepted if they target areas where limited progress despite high unmet needs.

Distribution to third parties is not allowed

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