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Orphan drug designation and development in Japan: 25 years of experience and assessment

  1. Ken Sakushima

    1. Orphan Drug Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.

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  2. Hiroshi Takeda

    1. Orphan Drug Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.

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  3. Yoko Aoi

    1. Orphan Drug Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.

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The Orphan Product Development Support Program was launched in Japan in 1993 to promote the development of therapies for rare diseases by providing various incentives for developers, such as financial subsidies for research, market exclusivity and prioritized scientific consultation/review. Eligibility for such incentives is determined by criteria for orphan drug designation linked to the number of affected patients in Japan and the extent of the unmet medical need, as well as a theoretical rationale for the use of the product for the target disease and an appropriate development plan (see Supplementary Box 1 for details). This initiative is managed by the collaborative work of the Ministry of Health, Labour, and Welfare (MHLW), the Pharmaceuticals and Medical Devices Agency (PMDA), and the National Institutes of Biomedical Innovation, Health and Nutrition (NIBIOHN). Furthermore, the Orphan Drug Working Group in the PMDA has been committed to supportive measures to facilitate orphan drug development.

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Nature Reviews Drug Discovery 20, 893-894 (2021)

doi: https://doi.org/10.1038/d41573-021-00045-3

Acknowledgements

The views of this article are personal opinions of the authors and do not necessarily reflect the official view of the PMDA.

Updates & Corrections

  • Clarification 15 September 2021: The supplementary file has been updated to clarify the characteristics of the data used to create Figure 2.

  • Correction 03 November 2021: Minor inconsistencies and errors related to the number of orphan drug designations and approvals have been corrected in the main text and Figure 1, and the explanation of the analysis in the Supplementary information has been modified accordingly. The corrections do not affect the trends shown in Figure 1 or the conclusions drawn in the text.

Supplementary Information

  1. Supplementary information

Competing Interests

The authors declare no competing interests.

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