CAR-T Cell Therapies Data

•  Novartis and Kite collected several data to secure FDA approval for their CAR-T products, KYMRIAH and YESCARTA.
• The overall remission rate (ORR) is one type of data that was collected using digital technologies.
• This data type include "complete remission (CR) and complete remission with incomplete blood count recovery (CRi)."
• For KYMRIAH, the impact was measured by collecting the duration of complete remission.
• The efficacy was also determined by deriving the proportion of patients with "CR and minimal residue disease (MRD)."
• Other real world data that were collected include the rate of survival of the patients, rate of the completeness of the responses.
• For YESCARTA, the collected data include the chain of custody (COC) or the chain of identity (COI) information during the commercial manufacturing processes.
• The approval also entails data collected on the existence of the "replication competent retrovirus (RCR) and insertional mutagenesis."
• Both CAR-T products above used regulatory technologies to collect data to secure approval.
• The prevalence of side effects is also one type of data being monitored.
• As a condition for marketing approval, the "long-term safety and efficacy" data of these products also need to be collected.
• When manufacturing CAR-T products, the relevant data needs to be collected as well to ensure compliance to global regulatory requirements.
• Information on material handling and patient scheduling across the therapeutic process also need to be collected and submitted for approval.
• Other data that need to be collected after implementing process improvements include the cost of goods levels, drug prices levels, margins, and the number of compatibility testing needed.
• In terms of regulatory compliance, "donor screening and testing, traceability and labeling, patient confidentiality, and apheresis requirements" data also need to be captured.
• In transplant centers, the prevalence of cytokine-release syndrome need to be tracked prior to FDA approval or subsequent reimbursement.
• The rate of success for T-cell manufacture also need to be collected.
• A comprehensive postmarket tracking will also need to be implemented to gather data on potential benefits and risks prior to approval.