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

Hayase Hakariya; Akihiko Ozaki; Yudai Kaneda; Tetsuya Tanimoto

doi:10.1002/cpt.3562

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

Clin Pharmacol Ther. 2025 Jan 14. doi: 10.1002/cpt.3562. Online ahead of print.

Authors

Hayase Hakariya^{1

2}, Akihiko Ozaki³, Yudai Kaneda⁴, Tetsuya Tanimoto⁵

Affiliations

¹ Interfaculty Institute of Biochemistry, University of Tuebingen, Tuebingen, Germany.
² Institute for Pharmaceutical and Social Health Sciences, Ise, Japan.
³ Department of Breast and Thyroid Surgery, Jyoban Hospital of Tokiwa Foundation, Iwaki, Fukushima, Japan.
⁴ School of Medicine, Hokkaido University, Sapporo, Hokkaido, Japan.
⁵ Navitas Clinic, Tokyo, Japan.

PMID: 39807768
DOI: 10.1002/cpt.3562

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.