How should we evaluate the cost-effectiveness of CAR T-cell therapies?

摘要

Recent approvals of pioneering chimeric antigen receptors (CAR) T-cell therapies, axicabtagene ciloleucel (Yescarta?) and tisagenlecleucel (Kymriah?), have resulted in considerable interest in the budgetary impact, clinical effectiveness and cost-effectiveness of these technologies. Traditional approaches of appraising new technologies in the UK would mean several promising highly specialised technologies such as CAR T-cell therapies would not gain approval. This article provides insights into the opportunity costs and challenges associated with CAR T-cell therapies, and describes the reimbursement models developed to ensure market access for CAR T-cell therapies. In 2017, NICE's evaluation guide to the Process and Methods of Technology Appraisal (TA) proposed a threshold of ￡100,000-￡300,000 per QALY for the evaluation of highly specialised technologies (HSTs) [1]. The proposal was accompanied with a budget of ￡20 million per year [2], for each technology evaluated under the technology appraisal (TA) and highly specialised technologies (HST) guidance. The budget is set to signal the need for commercial discussions between the company and NHS England to keep prices low.