New Japanese Initiatives on Stem Cell Therapies

Abstract

Two laws aiming to provide a new legal framework to promote regenerative medicine, while ensuring the efficacy and safety of the treatments, came into effect in Japan on November 25, 2014. The scope of these laws is briefly described here.

Challenges of Running a GMP Facility for Regenerative Medicine in a Public Hospital

“…Indeed, one of the main and critical obstacles is the final costs of these products. Several facilitating strategies have been recently developed in different European and non-European countries to facilitate access to the market (such as the adaptive framework for the approval of regenerative medical products under the Pharmaceuticals, Medical Devices and Other Therapeutic Products Act in Japan, the conditional marketing authorization in Europe, and the US FDA-accelerated approval in the USA) [31][32][33][34][35]. However, these approaches have no yet direct effect on academic facilities that still rely on the acquisition of public and private funds on a competitive basis.…”

Current status of ex vivo gene therapy for hematological disorders: a review of clinical trials in Japan around the world

“…Under the traditional approval process, long-term data collection and evaluation in clinical trials for regenerative medical products were needed. New separate approval process, however, will make it possible for the regenerative, including ex vivo gene therapy medical product obtain conditional, time-limited approval, if an exploratory clinical trial predicts likely clinical benefit [87]. This new act would accelerate the development of ex vivo gene therapy in Japan.…”

Ethical Issues for Clinical Studies That use Human Embryonic Stem Cells: The 2014 Revisions to the Japanese Guidelines

“…Although we cannot describe them in detail here, the bioethical requirements for the destruction of human fertilized embryos (use of spare embryos (Article 7 [8]), uncompensated donation (Article 5 [8])) are the same stringent requirements as those previously required for use in basic research. To ensure the efficacy and safety needed to use hESCs in medical treatment (including clinical studies and trials), compliance with Bthe Act on the Safety of Regenerative Medicine^and Bthe Pharmaceuticals, Medical Devices and Other Therapeutic Products Act^is essential [10][11][12]. The Act on the Safety of Regenerative Medicine classifies regenerative medical techniques into three categories depending on their potential risk to human health.…”

“…hESCs are classified as Class I, which is the highest risk category. This category, which also includes iPSCs and genetically modified cells, requires a unique set of review procedures for the research plan and facility [12]. In Japan, these two laws, together with the guidelines, constitute the legal framework for hESCs, from their derivation to their use for medical purposes.…”

“…During the revision process, the Expert Panel on Bioethics in the Cabinet Office of Japan reviewed the ethical issues concerning the guidelines, and finally approved the revision. Laws aiming to commercially promote regenerative medicine-BThe Act on the Safety of Regenerative Medicineâ nd BThe Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act^-also apply to the handling of hESCs for medical purposes (including clinical studies and trials) [10][11][12].…”