Advanced Therapies: Innovations in CMC Conference

Control strategies are developed based on product data, prior knowledge, and risk assessments, and ensure delivery of safe and efficacious product throughout development and the commercial life of the product. For ATMPs development of risk-based control strategies may be beneficial, where relevant information from clinical and non-clinical domains in addition to CMC information is used to define the boundaries for CQAs. Incorporation of product characterization, bioassay, assay matrixing, non-clinical models, biomarkers, surrogate model and structure activity characterization as early in development as possible can all further elucidate criticality of CQAs for risk-based control strategies. Development of such risk-based control strategies can be used to set specifications for both release and stability, identify critical in-process controls and also improve readiness to support comparability studies evaluating CMC changes. New approaches and case studies supportive of risk-based control strategies are discussed in this session.