There has been a surge in clinical development of Cell and Gene Therapy Products (Advanced Therapies), both in the EU and USA. Many start-up companies, and now even large biopharmaceutical companies, are planning or have already entered into manufacturing these genetically engineered viruses and/or human cell products for clinical studies.
While the ground rules for Good Manufacturing Practices (GMPs) and Quality of recombinant protein and monoclonal antibody manufacturing and control are well established, Advanced Therapy products are still under development. Their manufacturing and control presents unique GMP and Quality challenges, such as the heightened concern about the safety/variability of the starting materials, the quality of the raw materials added during processing, the need to protect against adventitious agent contamination throughout the entire manufacturing cycle, the limited analytical methods available to characterize these advanced therapy products, and the reality that the administered clinical medicinal product is a complex living organism. In addition, the expedited pace of clinical studies for these Advanced Therapies places enormous pressure on the need to also rapidly develop and enhance the manufacturing process control systems.
Patients in these clinical development programs need to be protected by common sense GMPs and Quality principles. The available EMA/FDA regulatory authority guidance documents for Advanced Therapy products will be thoroughly examined. In addition, the core, risk-based principles presented in PDA’s 2018 Technical Report No. 81: Cell-Based Therapy Control Strategy, will be discussed.