太少,太快:日本在再生医学放松监管方面的实验
David Cyranoski1, Douglas Sipp2, Shreyashee Mallik3
1Institute for the Advanced Study of Human Biology (ASHBi), Kyoto 606-8303, Japan.
Cell stem cell
|July 7, 2023
概括
日本2013年对细胞和基因疗法的条件批准途径显示了潜在的问题. 分析显示安全性和有效性数据存在问题,质疑该途径的有效性.
科学领域:
- 生物医学科学 生物医学科学
- 监管科学 监管科学
- 卫生政策 卫生政策
背景情况:
- 日本在2013年为先进疗法引入了有条件批准途径.
- 这一途径旨在加速获得新型细胞和基因疗法.
研究的目的:
- 评估通过日本的有条件途径批准的产品的安全性和有效性数据的质量.
- 评估这一监管框架的整体成功和可靠性.
主要方法:
- 对细胞和基因治疗产品的监管提交数据的分析.
- 对四种有条件批准的产品提出的安全性和有效性证据的审查.
主要成果:
- 在提交的安全数据中发现了重大缺陷.
- 发现证据表明,支持产品批准的疗效数据不足.
- 对审批过程的稳定性提出了担忧.
结论:
- 目前的条件批准途径可能无法充分确保已批准的细胞和基因疗法的安全性和有效性.
- 进一步审查数据要求和市场后监测是合理的.
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