CAR T-cell therapy is a precision cancer treatment wherein each treatment dose is individually manufactured for the patient using their own T-cells, a type of white blood cell known as a lymphocyte. CAR T-cell therapy is a rapidly emerging adoptive cell transfer immunotherapy for select patients with relapsed or refractory cancers. Treatment protocols vary, but may be summarized in five steps:
- lymphocyte harvesting from the patient with cancer;
- creation of cancer-targeting lymphocytes in vitro using various immune modulators;
- selection of lymphocytes with reactivity to cancer antigens using enzyme-linked immuno-assay;
- depletion of the patient's remaining lymphocytes using immunosuppressive agents;
- transfusion of the cancer-targeting lymphocytes back into the patient with cancer-this transfusion represents one treatment.
In 2017, the Food and Drug Administration (FDA) approved tisagenlecleucel (Kymriah®) and axicabtagene ciloleucel (Yescarta®) CAR T-cell therapies for the treatment of certain relapsed or refractory leukemias and lymphomas. The two FDA-approved therapies include boxed warnings (calling attention to serious and life-threatening risks) and safety monitoring through an FDA Risk Evaluation and Mitigation Strategy (REMS). To date, few Medicare patients have been studied and follow up has been limited. The majority of patients who received CAR T-cell therapy also experienced adverse events including cytokine release syndrome and neurologic effects. Initial studies were also confined to the inpatient hospital setting.
On August 22, 2018, we are convening a Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) meeting to review the available evidence on this topic.