Science-based regulatory considerations for regenerative medicine cellular products

Abstract Regenerative medicine is a rapidly expanding field given its potential to treat highly complex health conditions and rare diseases. However, the application of the US Food and Drug Administration (FDA) regulatory framework to these innovative products can be challenging given their highly variable and complex nature. Nevertheless, the rapid development of cellular products and their fast-paced innovation requires FDA to develop new tools, standards, and approaches with which to adequately assess their safety, efficacy, quality, and performance. In addition to FDA’s advancement of regulatory science, product developers bear the onus of generating high-quality objective data at key stages throughout the product life cycle that can be effectively and efficiently reviewed. To spur collaboration between FDA and product developers, this review paper provides a discussion about how regulatory science can be leveraged to improve the demonstration of product identity and potency testing for regenerative medicine cellular products.