{"title":"Japan's Conditional/Time-Limited Early Approval System in Regenerative Medicine: A Case Study of Rise and Falls of Autologous Skeletal Myoblast Sheets","authors":"Hayase Hakariya, Akihiko Ozaki, Yudai Kaneda, Tetsuya Tanimoto","doi":"10.1002/cpt.3562","DOIUrl":null,"url":null,"abstract":"<p>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.</p>","PeriodicalId":153,"journal":{"name":"Clinical Pharmacology & Therapeutics","volume":"117 5","pages":"1171-1174"},"PeriodicalIF":5.5000,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/cpt.3562","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Clinical Pharmacology & Therapeutics","FirstCategoryId":"3","ListUrlMain":"https://ascpt.onlinelibrary.wiley.com/doi/10.1002/cpt.3562","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
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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.
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.
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