OBJECTIVES: The most common adverse effects (AE) of chimeric antigen receptor (CAR) T-cell therapy include cytokine release syndrome, neurologic events, neutropenia and anaemia. Although most adverse events can be managed, they can be serious and life threatening. Furthermore, the occurrence of mild or severe adverse effects during hospitalisation requires additional treatment and prolonged hospital length of stay, significantly increasing costs to the NHS. This paper aims to assess the cost of adverse events in patients treated with CAR T-cell therapy for blood cancers.

METHODS: Data from a UK multi-centre Phase I clinical trial of CAR T-cell therapy in adults (>16) will be used to identify the toxicities associated with CAR T-cell therapy. The micro-costing approach will be applied to enable a transparent, detailed and comprehensive cost analysis. Data from the trial will be used to estimate the cost per patient. Resource use will be collected prospectively and calculated by multiplying the quantity of resource use with the unit cost. Unit costs will be taken from standard national published sources. The time horizon will be 24 months.

RESULTS: Cost justification for CAR-T is a seemingly controversial topic and the high-costs associated with CAR T-cell therapy wanes enthusiasm for payers. Improving current understanding of the actual cost of adverse events associated with CAR T-cell therapy, will help clarify its weight on the overall costs and impact on the affordability of innovative therapies.

CONCLUSIONS: This research will add to knowledge by identifying (1) the cost of adverse events of autologous CAR T-cell therapy in patients treated with blood cancer in the UK context and (2) demonstrate the cost implications of adverse events to the NHS.