Navigating the Manufacturing Process and Ensuring the Quality of Regenerative Medicine Therapies - A Workshop
Regenerative medicine holds the potential to create living, functional cells and tissues which can be used to repair or replace those that have suffered irreparable damage due to disease, age, traumatic injury, or congenital defects. The field of regenerative medicine is broad and includes research and development of gene and cell therapies, tissue engineering, and non-biologic constructs. Although regenerative medicine has the potential to result in health and economic benefits, this relatively new field faces unique regulatory and manufacturing challenges in the development of novel therapies that are both safe and effective. Some of these issues arise because regenerative medicine therapies produce and rely on living cells and tissues, which are inherently variable, even within a single type of cell. The reliance of regenerative medicine products on living cells and tissues adds a fundamental complexity to the manufacturing and scale-up process that is not found in the manufacturing of most non-biologic products.
Since the variety of cells and tissues used in regenerative medicine is vast and the characteristics of cells can differ between in vitro and in vivo environments, defining and assessing quality is challenging. Additionally, it can be difficult to accurately measure or test for defined quality attributes of cells because these attributes can be impacted by a cell’s maturity and its environment. Discussion of techniques used to identify, produce, and measure quality in regenerative medicine products may help inform the design of regulatory and manufacturing standards as well as the development of new technologies to assess quality and potency throughout the manufacturing process.
Building on a previous workshop that explored the state of the science and challenges of and opportunities for regenerative cellular therapies, the Forum on Regenerative Medicine decided to focus this public workshop on exploring what measurements, characteristics, and technologies may be important in the development of new products and therapies. A better understanding of which characteristics define a source cell or tissue can support the development of a quality product. Likewise, a deep understanding of the critical characteristics of the final regenerative medicine product can help ensure its consistency, safety, and potency.
On June 26, 2017, the Forum hosted a public workshop to examine and discuss challenges, opportunities, and best practices associated with defining and measuring the quality of cell and tissue products and raw materials in the research and manufacturing of regenerative medicine therapies. The goal of the workshop was to learn from existing examples of manufacturing of early generation regenerative medicine products and to address how progress could be made in identifying and measuring critical quality attributes as well as designing and adhering to standards to help in the navigation of the scale-up process from a research laboratory to the manufacturing environment.