OBJECTIVES: Cost-effectiveness analysis is routinely used as part of health technology appraisal (HTA) processes to inform decisions around approval and reimbursement. Despite similar techniques used for economic analysis by HTA agencies in England, Canada and Australia, different decisions are often made. This study reviewed the similarities and differences that influenced HTA appraisal decisions for axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor T-cell (CAR-T) therapy, in diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) in England, Canda and Australia.

METHODS: HTA appraisals for axi-cel, which was first approved by the FDA in 2017, were identified from the websites of National Institute of Health and Care Excellence (NICE, England), Canada’s Drug Agency (CDA, Canada), and the Medical Service Advisory Committee (MSAC, Australia). Factors which might influence reimbursement were assessed, including the portrayed burden, unmet need, clinical evidence, and interpretation of the economic data.

RESULTS: Axi-cel was recommended for use in patients with DLBCL in England, Canada and Australia. For patients with FL it was recommended in Canada but not England; the appraisal is ongoing in Australia. Data presented for these submissions demonstrated the potential benefit of treatment with axi-cel, although each committee discussed uncertainties in the evidence, particularly around long-term outcomes. Uncertainties in the generalisability of axi-cel clinical trial data led to inclusion in the Cancer Drugs Fund (CDF) for DLBCL in England to enable further data collection. In Canada, for FL, uncertainties in long-term survival data resulted in a reimbursement condition of an 82–95% price reduction.

CONCLUSIONS: The variation in access to axi-cel is typically due to the attitude of the HTA agency to uncertainty in the presented data. Ability to demonstrate long-term benefit was seen as a driver of uncertainty across the markets, which led to reimbursement restrictions and ongoing data collection.