Giulia Detela, and Anthony Lodge. "Manufacturing process development of ATMPs within a regulatory framework for EU clinical trial & marketing authorisation applications." Cell Gene Therapy Insights 2, no.4 (April 2016): 425-452. https://doi.org/10.18609/cgti.2016.056.
Abstract:
The quality attributes of advanced therapy medicinal products (ATMPs) that correlate with safety and efficacy in patients are determined not only by manufacturing process inputs such as starting and raw materials, but also by how the manufacturing process itself is designed and controlled. To ensure regulatory compliance, the manufacturing process should therefore be developed based on thorough characterization of the ATMP during all stages of process and analytical development; this ensures that the critical quality attributes that correlate with safety and efficacy are identified and that their specifications can be met during routine manufacturing. In the European Union, the regulatory approval of ATMPs for use in patients requires that data demonstrating their quality, safety and efficacy are submitted in dossiers to regulatory agencies for review. Indeed, such dossiers have a specific format that, in the case of quality data in particular, is informative for the manufacturing process development strategy. This manuscript describes how dossier requirements can be implemented into the design of industrialized ATMP manufacturing processes and fulfilled to enable effective regulatory submissions.