Wonder

Research Outline

Prepared for Ron R. | Delivered January 1, 2020

CAR-T Cell Therapies Data

Goals

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.

Early Findings

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. 