title: Regulatory Frameworks for Gene and Cell Therapies in Japan.
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.
authors.affiliation: Office of Cellular and Tissue-Based Products (OCTP), Pharmaceuticals and Medical Devices Agency (PMDA), 3-3-2, Kasumigaseki, Chiyoda-ku, Tokyo, 100-0013, Japan. maeda-daisuke@pmda.go.jp.
authors.forename: Daisuke
authors.initials: D
authors.lastname: Maeda
authors.affiliation: Office of Cellular and Tissue-Based Products (OCTP), Pharmaceuticals and Medical Devices Agency (PMDA), 3-3-2, Kasumigaseki, Chiyoda-ku, Tokyo, 100-0013, Japan. yamaguchi-teruhide@pmda.go.jp.
authors.forename: Teruhide
authors.initials: T
authors.lastname: Yamaguchi
authors.affiliation: Office of New Drug V, Pharmaceuticals and Medical Devices Agency (PMDA), 3-3-2, Kasumigaseki, Chiyoda-ku, Tokyo, 100-0013, Japan. ishizuka-takami@pmda.go.jp.
authors.forename: Takami
authors.initials: T
authors.lastname: Ishizuka
authors.affiliation: Division of Pharmaceutical Affairs Consultation, Kansai Branch, Pharmaceuticals and Medical Devices Agency (PMDA), 3-1, Ofuka-cho, Kita-ku, Osaka, 530-0011, Japan. hirata-masakazu@pmda.go.jp.
authors.forename: Masakazu
authors.initials: M
authors.lastname: Hirata
authors.affiliation: Office of Cellular and Tissue-Based Products (OCTP), Pharmaceuticals and Medical Devices Agency (PMDA), 3-3-2, Kasumigaseki, Chiyoda-ku, Tokyo, 100-0013, Japan. takekita-kazuhiro@pmda.go.jp.
authors.forename: Kazuhiro
authors.initials: K
authors.lastname: Takekita
authors.affiliation: Office of Cellular and Tissue-Based Products (OCTP), Pharmaceuticals and Medical Devices Agency (PMDA), 3-3-2, Kasumigaseki, Chiyoda-ku, Tokyo, 100-0013, Japan. sato-daisauku@pmda.go.jp.
authors.forename: Daisaku
authors.initials: D
authors.lastname: Sato
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000941:ethics* / Q000379:methods; D002986:Clinical Trials as Topic; D004335:Drug and Narcotic Control / Q000331:legislation & jurisprudence*; D015316:Genetic Therapy / Q000941:ethics / Q000331:legislation & jurisprudence*; D006801:Humans; D007564:Japan; D040541:Marketing / Q000331:legislation & jurisprudence*; D061214:Patient Safety / Q000331:legislation & jurisprudence; D017410:Practice Guidelines as Topic; D011786:Quality Control; D044968:Regenerative Medicine / Q000941:ethics / Q000331:legislation & jurisprudence*; D012107:Research Design; D057170:Translational Research, Biomedical / Q000941:ethics / Q000331:legislation & jurisprudence*
keywords: Act on the safety of regenerative medicine; Cell therapy; Gene therapy; Guidelines; Japan; Pharmaceuticals and medical devices act