Japan's Conditional/Time-Limited Early Approval System in Regenerative Medicine: A Case Study of Rise and Falls of Autologous Skeletal Myoblast Sheets.

IF 6.3 2区医学 Q1 PHARMACOLOGY & PHARMACY

Clinical Pharmacology & Therapeutics Pub Date : 2025-01-14 DOI:10.1002/cpt.3562

Hayase Hakariya, Akihiko Ozaki, Yudai Kaneda, Tetsuya Tanimoto

引用次数: 0

Abstract

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.

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日本再生医学有条件/有时限的早期审批制度：自体骨骼肌细胞片的兴衰案例研究。

日本有条件/有时限的早期批准计划于2014年启动，旨在通过加快市场准入来推进再生医学。然而，自体骨骼肌母细胞片（Heartsheet）由于无效而退出，引起了对快速批准和科学完整性之间平衡的担忧。虽然该计划寻求促进创新，但它有可能支持国家医疗保健中昂贵且不明确的治疗方法。该病例强调需要完善监管方法，确保再生疗法的临床疗效和财政责任。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Clinical Pharmacology & Therapeutics 医学-药学

CiteScore

12.70

自引率

7.50%

发文量

290

审稿时长

2 months

期刊介绍： Clinical Pharmacology & Therapeutics (CPT) is the authoritative cross-disciplinary journal in experimental and clinical medicine devoted to publishing advances in the nature, action, efficacy, and evaluation of therapeutics. CPT welcomes original Articles in the emerging areas of translational, predictive and personalized medicine; new therapeutic modalities including gene and cell therapies; pharmacogenomics, proteomics and metabolomics; bioinformation and applied systems biology complementing areas of pharmacokinetics and pharmacodynamics, human investigation and clinical trials, pharmacovigilence, pharmacoepidemiology, pharmacometrics, and population pharmacology.