日本基因与细胞疗法的监管框架

Regulatory Frameworks for Gene and Cell Therapies in Japan.

作者信息

Maeda Daisuke, Yamaguchi Teruhide, Ishizuka Takami, Hirata Masakazu, Takekita Kazuhiro, Sato Daisaku

机构信息

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

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

出版信息

Adv Exp Med Biol. 2015;871:147-62. doi: 10.1007/978-3-319-18618-4_8.

DOI:10.1007/978-3-319-18618-4_8

PMID:26374217

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.

摘要

基因和细胞治疗产品等先进治疗用医疗产品的人类使用法规，已随着临床经验、科学知识的进步以及社会对这些技术的接受程度而不断演变。在日本，2014年11月颁布了两部法律，即《药品和医疗器械法》（PMD Act）和《再生医学安全法》（ASRM）。《药品和医疗器械法》首次对再生医疗产品进行了定义，并引入了再生医疗产品有条件和有时限的上市许可制度。根据《再生医学安全法》，描述了医疗机构确保此类医疗技术安全并提供透明度的责任。目前正在准备对这两部法律的配套指南进行修订。预计新的立法框架将促进新产品和新技术的及时开发，为日本患者带来安全有效的再生药物。