日本再生医学的有条件/限时早期批准制度：以自体骨骼肌成肌细胞片的兴衰为例
Japan's Conditional/Time-Limited Early Approval System in Regenerative Medicine: A Case Study of Rise and Falls of Autologous Skeletal Myoblast Sheets.
作者信息
Hakariya Hayase, Ozaki Akihiko, Kaneda Yudai, Tanimoto Tetsuya
机构信息
Interfaculty Institute of Biochemistry, University of Tuebingen, Tuebingen, Germany.
Institute for Pharmaceutical and Social Health Sciences, Ise, Japan.
出版信息
Clin Pharmacol Ther. 2025 May;117(5):1171-1174. doi: 10.1002/cpt.3562. Epub 2025 Jan 14.
Japan's conditional/time-limited early approval program, initiated in 2014, aimed to advance regenerative medicine by expediting market access. However, the withdrawal of autologous skeletal myoblast sheets (Heartsheet) due to ineffectiveness raises concerns about the balance between rapid approval and scientific integrity. While the program seeks to boost innovation, it risks endorsing costly, unclear treatments under national health care. This case highlights the need to refine regulatory approaches, ensuring clinical efficacy and fiscal responsibility in regenerative therapies.
日本于2014年启动的有条件/限时早期批准计划旨在通过加快市场准入来推动再生医学发展。然而，自体骨骼肌成肌细胞片（心肌片）因无效而撤出引发了人们对快速批准与科学完整性之间平衡的担忧。虽然该计划旨在促进创新，但在国家医疗保健体系下，它有批准成本高昂且疗效不明的治疗方法的风险。这一案例凸显了完善监管方法的必要性，以确保再生疗法的临床疗效和财政责任。
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