Regulatory issues in cell-based therapy for clinical purposes

Abstract

Rapid development in the fields of cellular and molecular biology, biotechnology, and bioengineering medicine has brought new, highly innovative treatments and medicinal products, some of which contain viable cells and tissues associated with scaffolds and devices. These new cell-based therapy approaches in regenerative medicine have great potential for use in the treatment of a number of diseases that at present cannot be managed effectively. Given the unique challenges associated with the development of human cell-based medicinal products, great care is required in the development of procedures, practices, and regulation. In cell therapy, appropriate methodologies in the areas of production, reproducibility, maintenance, and delivery are essential for accurate definition and reliable assurance of the suitability and quality of the final products. Recently, the official European Community agencies (EMA) and the relevant authority in the USA (FDA) have made significant efforts to establish regulatory guidance for use in the application of the cell-based therapies for human patients. The guidelines surrounding cell-based therapy take into account the current legislation, but focus less on the heterogeneity and requirements of individual human cell-based products, including specific combination products and applications. When considering guidelines and regulation, a risk assessment approach is an effective method of identifying priority areas for the development of human cell-based medicinal products. Additionally, effective design and thorough validation of the manufacturing process in line with existing Good Manufacturing Practices (GMPs) and quality control regimes and a program that ensures the traceability and biovigilance of the final products are also all essential elements to consider.