Loughborough University, UK
Title: Regulation and development in cell-based therapeutics: The need for comparability and Implications for new business models
Nick Medcalf is a Professor of Regenerative Medicine Manufacture at Loughborough University, UK. He is Director of the EPSRC Centre for Innovative Manufacturing in Regenerative Medicine from 2014. Regulatory science’ i.e. industry-facing, challenge-led research aimed at providing the confidence in process design necessary to satisfy regulatory constraints for advanced therapeutic products, such as cell- and tissue-based products. Thomas Heathman is a PhD student at Loughborough University, UK. He is interested to develop a microcarrier based stirred culture system for scalable expansion of allogeneic bone marrow derived human mesenchymal stem cells to treat disorders of the skeletal system. We will be taking a quality engineering approach to build a comprehensive knowledge base of how various process parameters such as reactor & impeller dimensions, microcarrier type and culture media will affect the product critical-to-quality attributes. From this enhanced process understanding we will develop a control strategy to ensure acceptable overall product quality throughout operational scale up which is critical for regulatory approval and successful commercialisation.
The development and delivery of novel cell-based therapeutic products requires ongoing and improved methods for ensuring consistency of quality, safety and efficacy. Issues that must be addressed for cost-effective, regulatory-compliant supply include the design of suitable control systems, validation of transit arrangements and management of overall costs, especially for indications that are of moderate, rather than life-saving value-release. The introduction of automation is often cited as a solution to these challenges but investment choices for process improvement using automation must takes place within a whole-operation context if effective automation is to take place. This presentation examines the current options for production and supply for new cell therapy businesses and highlights the strengths and weaknesses of these in the light of supply-chain robustness, ease of clinical introduction and cost of manufacture. Some conclusions are presented about the likely direction of new business models and the technology required to enable these to be used while satisfying issues of process comparability.