The regulation of allogeneic human cells and tissue products as biomaterials

Joint Graduate School of Tokyo Women's Medical University and Waseda University, 8-1 Kawada-cho, Shinjuku-ku, Tokyo 162-866, Japan. Electronic address: .
Biomaterials (Impact Factor: 8.56). 04/2013; 34(13):3165-73. DOI: 10.1016/j.biomaterials.2013.01.048
Source: PubMed


The current definition of biomaterials differs vastly from it of just a decade ago. According to advancing technologies, it encompasses unpredictable materials such as engineered human cells and tissue. These biomaterials also have to be approved to use in health care business by regulatory authority, which are defined as drug, medical device, or biologics in the regulation. This Leading Opinion Paper addresses the regulatory issues of engineered human cells and tissue products using allogeneic cells that should have a great possibility to develop therapeutics for life-threating diseases or orphan diseases. Six allogeneic human cells and tissue products derived from neonatal or infant fibroblasts and/or keratinocytes were approved as medical devices or biologics in the United States as well as a hematopoietic cell product. For five of the seven products, well-controlled comparative clinical trials were conducted as pre-approval evaluation followed by post-approval evaluation. Although these products avoid a sterilization process usually used for medical devices, no serious malfunction that would lead to class 1 recall was reported. This article would provide insight for development of the engineered human cells and tissue.

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    • "clinical trials with a small number of subjects, although the other products had been evaluated using comparative clinical trials with control treatment. Autologous hCTPs would need postmarketoriented evaluation rather than premarket-oriented evaluation [6]. In allogeneic hCTPs, six somatic cell therapy products were submitted for market authorization in the USA, four were as a PMA, one as an IDE, and one as a BLA. "
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    ABSTRACT: The regulation of human cell therapy products is a key factor in their development and use to treat human diseases. In that regard, there is a recognized need for a global effort to develop a set of common principles that may serve to facilitate a convergence of regulatory approaches to ensure the smooth and efficient evaluation of products. This conference, with experts from regulatory agencies, industry, and academia, contributed to the process of developing such a document. Elements that could form a minimum consensus package of requirements for evaluating human cell therapy products were the overall focus of the conference. The important regulatory considerations that are unique to human cell therapy products were highlighted. Sessions addressed specific points that are different from those of traditional biological/biotechnological protein products. Panel discussions complemented the presentations. The conference concluded that most of the current regulatory framework is appropriate for cell therapy, but there are some areas where the application of the requirements for traditional biologicals is inappropriate. In addition, it was agreed that there is a need for international consensus on core regulatory elements, and that one of the major international organizations should take the lead in formulating such a consensus document. Copyright © 2015.
    Full-text · Article · Aug 2015 · Biologicals
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    • "However, few studies have been conducted to elucidate the similarity or difference of the definition, category, premarket approval evaluation by regulatory authorities, and adverse events of human cells and tissue products. Recently, we have published the regulation of allogeneic human cells and tissue products [16] that are seven products on the market in the US only, and five of seven products had evaluated using comparative clinical trials with control treatment. Otherwise, there is no such detailed study of autologous human cells and tissue products. "
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    ABSTRACT: Human cells and tissue products belong to a relatively new class of medical products. Therefore, limited information is available on the classification and premarket evaluation of human cells and tissue products in the United States (US), the European Union (EU), and Japan. In this study, the definition, legislation, and approval system of these products were surveyed. A total of nine autologous human cells and tissue products approved until October 2013 were collected. The definitions of human cells and tissue products were compatible among the US, the EU and Japan. The products were classified as human cells, tissue, and cellular and tissue-based products (HCT/Ps) in the US, advanced therapy medicinal products (ATMPs) in the EU, and cell/tissue-engineered products in Japan. These products were categorized as biologics and medical device in the US and Japan, and drug in the EU. The issuance of new guidance induced regulatory impact for manufacturer, especially in the US. These products are subjected to the accelerated approval of biological product, the humanitarian device exemption approval, the premarket application approval, the biologics license application approval, and new drug application approval with specific targeting of postapproval registry or surveillance. Of nine autologous human cells and tissue products, four products had been evaluated using clinical experiences or open clinical trials with small subjects, although the rests of products had been evaluated using comparative clinical trials with control treatment. Our survey suggests that autologous human cells and tissue products would need postmarket-oriented evaluation rather than premarket-oriented evaluation for doctors and patients.
    Full-text · Article · Jun 2015
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    • "). While most products on the market are composed of skin or cartilage bioprocessed with somatic cells [1] [2], R&D into next-generation products derived from stem cells, such as mesenchymal or embryonic stem cells, has been steadily promoted. Recently, a clinical study into iPSC therapy has been approved by the Ministry of Health, Labour and Welfare in Japan (http://www.rikenibri "
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    ABSTRACT: Substantial progress made in the areas of stem cell research and regenerative medicine has provided a number of innovative methods to repair or regenerate defective tissues and organs. Although previous studies regarding regenerative medicine, especially those involving induced pluripotent stem cells, have been actively promoted in the past decade, there remain some challenges that need to be addressed in order to enable clinical applications. Designed for use in clinical applications, cell sheet engineering has been developed as a unique, scaffold-free method of cell processing utilizing temperature-responsive cell culture vessels. Clinical studies using cell sheets have shown positive outcomes and will be translated into clinical practice in the near future. However, several challenges stand in the way of the industrialization of cell sheet products and the widespread acceptance of regenerative medicine based on cell sheet engineering. This review describes current strategies geared towards the realization of the regenerative medicine approach.
    Full-text · Article · Jul 2014 · Biotechnology Journal
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