ICER Releases Scoping Document for CAR-T Therapies


The Institute for Clinical and Economic Review (ICER) has released a scoping document that will compare the clinical effectiveness and value of multiple chimeric antigen receptor (CAR)-T cell treatments under review by the FDA.


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CTL-019 (tisagenlecleucel-t, Novartis) and KTE-C19 (axicabtagene ciloleucal, Kite Pharma) are designed to target the CD19 antigen expressed on B-cells of various leukemias. CTL-019 recently received unanimous approval from the FDA’s Oncologic Drugs Advisory Committee to treat children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). KTE-C19 is indicated for B-cell non-Hodgkin lymphoma (NHL). Both CAR-T therapies are associated with adverse events, most notably cytokine release syndrome.

The ICER scoping document was developed with input from multiple patient advocacy organizations, specialty societies, hematologists, oncologists, payers, and pharmaceutical manufacturers. The document calls to question the durability of CAR-T therapy effects, management of adverse events, and the costs of treatment. An upcoming analysis will use the National Comprehensive Cancer Network (NCCN) Guidelines to choose comparative treatments for CAR-T in relapsed or refractory B-cell ALL. Salvage chemotherapy treatments of second-line treatments recommended by NCCN will be compared with CAR-T therapy in B-cell NHL. The primary outcome of interest is overall survival.

ICER intends to utilize a decision analytic model to measure the cost-effectiveness of CTL-019 and KTL-C19, relative to the chosen comparative treatments that have clinical and economic data available. Pediatric and young adult patients with relapsed or refractory B-cell ALL will be included in the CTL-019 analysis and adults (aged 18 years or older) with relapsed or refractory aggressive B-cell lymphoma will be included in the KTE-C19 analysis.

ICER will assess each intervention in regards to the proportion of responders over a 3-month period after transfusion. Life years and quality-adjusted life years gained will be evaluated, along with infusion costs, treatment acquisition, administration, monitoring, adverse events, and other health care utilization.

The scoping document is open for public comment through August 29. ICER plans to use input to inform a subsequent draft of the document to be posted in September.—Zachary Bessette