Chemotherapy: Open Access
Open Access

Abstract

Diverse Approval Systems for Autologous Human Cells and Tissue Products

Yano K, Watanabe N and Yamato M

Human cells and tissue products are newly categorized products which are containing, consisting of, or derived from cells or tissue that are intended for implantation, transplantation, infusion, or transfer into human recipient. The human cells and tissue products are divided as autologous or allogeneic based on their origin. Since another product fabricated with patient?s own limbal epithelial stem cells, was approved as conditional approval for therapeutic indication which is the treatment of adult patients with moderate to severe limbal stem cell deficiency in the European Union, we reviewed the European Public Assessment Report. Of the ten autologous human cells and tissue products, the five products were evaluated using the clinical data with clinical experiences as well as retrospective observatory study or registry, or open clinical trials with small subjects, although for the rest of the products comparative clinical trials with control treatment were performed. In general, autologous human cells and tissue products would need post market-oriented evaluation rather than premarket-oriented evaluation for patients because some of these indications are for orphan diseases or life-threatening disease. However, the other products intended for repair of cartilaginous defects of knee, treatment of prostate cancer, or improvement of the appearance of nasolabial fold winkles were evaluated using comparative clinical data. As for the approval pathways, expedited or normal, both approval pathways are available in the US, the EU, and Japan. The expedited pathways include accelerated approval and humanitarian device exception in the US, conditional market authorization and market authorization under exceptional circumstances in the EU, and a conditional/time-limited approval system in Japan. Our additional review suggests that autologous human cells and tissue products approved using the expedited approval pathway would need post market-oriented evaluation rather than premarket-oriented evaluation.