CAR-T Cell Therapies Data
Determine information on real world data collected using digital technologies to receive approval and reimbursement for CAR-T cell therapies. The insights will be used to build a case study and secure approval and reimbursement for some cell therapies.
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.
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