A summary of how to develop a cell therapy from concept to market authorization

Cell therapies offer new opportunities for the treatment of disease and injury but considerable infrastructure and a complex range of activities and expertise are required to translate a promising cell therapy product into clinical use. Suppliers, developers, regulators, and care givers need to interact cooperatively in order to bring therapies to patients in the most efficient, safe, and economically viable way and GE Healthcare is working with the Karolinska University Hospital to investigate more effective routes for bringing potential cell therapies to market. This paper provides a representative summary of the key stages of planning, conducting, and reporting on a clinical trial and the regulatory requirements that must be fulfilled before gaining marketing authorization and approval for use in patients. The information provided is not exhaustive and is intended for guidance only. It is derived from the status of requirements in the European Union (EU) as of December 2015; equivalent United States (US) regulations are summarized, and contact information for national competent authorities in other countries is provided. More detail on each of the stages can be found in the Web based Cell Therapy Guide* that was developed as part of the Karolinska University Hospital and GE Healthcare collaboration.