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Regulatory Frameworks for Gene and Cell Therapies in Japan

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Regulatory Aspects of Gene Therapy and Cell Therapy Products

Part of the book series: Advances in Experimental Medicine and Biology ((ASGCT,volume 871))

Abstract

The regulations for the human use of advanced therapy medical products such as gene and cell therapy products have evolved in accordance with advance of clinical experience, scientific knowledge, and social acceptance to these technologies. In Japan, two laws, the Pharmaceuticals and Medical Devices (PMD) Act and the Act on the Safety of Regenerative Medicine (ASRM), were enacted in November 2014. The PMD Act defines regenerative medical products for the first time and introduces a system for the conditional and time-limited marketing authorization of regenerative medical products. Under ASRM, the responsibilities of medical institutions to ensure the safety and provide transparency of such medical technologies are described. Amendments to accompanying guidelines for these two Acts are currently in preparation. It is expected that the new legislative frameworks will promote the timely development of new products and technologies, to bring safe and effective regenerative medicines to Japanese patients.

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Abbreviations

ASRM:

Act on the Safety of Regenerative Medicine

ATMP:

Advanced therapy medical products

CT:

Cell therapy

DNA:

Deoxyribonucleic acid

GCP:

Good clinical practice

GCT:

Gene and cell therapy

GL:

Guideline

GT:

Gene therapy

HSC:

Health Science Council

ICH:

International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use

iPS:

Induced pluripotent stem

JSRM:

Japanese Society for Regenerative Medicine

MA:

Marketing authorization

MAA:

Marketing authorization application

MAH:

Marketing authorization holder

MHLW:

Ministry of Health, Labour and Welfare

NIHS:

National Institute of Health Sciences

OCTP:

Office of Cellular and Tissue-based Products

PAL:

Pharmaceutical Affairs Law

PASFC:

Pharmaceutical Affairs and Food Sanitation Council

PMD Act:

Pharmaceuticals and Medical Devices Act (revised PAL)

PMDA:

Pharmaceuticals and Medical Devices Agency

RNA:

Ribonucleic acid

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Acknowledgment

We thank Dr. Jun-ichi Sawada for helpful suggestions in preparing this chapter.

Author information

Authors and Affiliations

  1. Office of Cellular and Tissue-Based Products (OCTP), Pharmaceuticals and Medical Devices Agency (PMDA), 3-3-2, Kasumigaseki, Chiyoda-ku, Tokyo, 100-0013, Japan

    Daisuke Maeda Ph.D., Teruhide Yamaguchi Ph.D., Kazuhiro Takekita M.S. & Daisaku Sato Ph.D.

  2. Office of New Drug V, Pharmaceuticals and Medical Devices Agency (PMDA), 3-3-2, Kasumigaseki, Chiyoda-ku, Tokyo, 100-0013, Japan

    Takami Ishizuka Ph.D.

  3. Division of Pharmaceutical Affairs Consultation, Kansai Branch, Pharmaceuticals and Medical Devices Agency (PMDA), 3-1, Ofuka-cho, Kita-ku, Osaka, 530-0011, Japan

    Masakazu Hirata M.D., Ph.D.

Authors
  1. Daisuke Maeda Ph.D.
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  2. Teruhide Yamaguchi Ph.D.
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  3. Takami Ishizuka Ph.D.
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  4. Masakazu Hirata M.D., Ph.D.
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  5. Kazuhiro Takekita M.S.
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  6. Daisaku Sato Ph.D.
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Corresponding author

Correspondence to Daisuke Maeda Ph.D. .

Editor information

Editors and Affiliations

  1. Cell Biology and Neurosciences, Istituto Superiore di Sanità, Rome, Roma, Italy

    Maria Cristina Galli

  2. Center for Biologics Evaluation and Research/Office of Cellular, Tissue, and Gene Therapies, Pharmacology/Toxicology Branch, US Food and Drug Administration, Silver Spring, Maryland, USA

    Mercedes Serabian

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© 2015 American Society of Gene and Cell Therapy

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Maeda, D., Yamaguchi, T., Ishizuka, T., Hirata, M., Takekita, K., Sato, D. (2015). Regulatory Frameworks for Gene and Cell Therapies in Japan. In: Galli, M., Serabian, M. (eds) Regulatory Aspects of Gene Therapy and Cell Therapy Products. Advances in Experimental Medicine and Biology(), vol 871. Springer, Cham. https://doi.org/10.1007/978-3-319-18618-4_8

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