Chimeric antigen receptor (CAR) T-cell therapy is a novel cell therapy for treating hematological cancers including multiply relapsed large B-cell lymphoma in adults. Although for some patients it can produce long-term remission who would have otherwise run out of treatment options, it is very expensive, costing $373,000 U.S. dollars per patient. There are also other additional hospital costs related to management of severe adverse events. The first two Health Canada approved CAR T-cell therapy products were reviewed by the Canadian Agency for Drugs and Technologies in Health (CADTH) and recommended for funding. Because CAR T-cell therapies have not been administered before in Canadian hospitals, there are certain logistical concerns with implementation that are unique.
The first aim of this thesis is to describe processes of developing CAR T-cell therapy and administering it to patients, as well as describe the challenges to implementation and considerations for long-term sustainability of cell and gene therapy in Canada. The second aim is to evaluate the cost-effectiveness of axicabtagene ciloleucel, a CAR T-cell therapy for treating adult lymphoma patients, compared to salvage chemotherapy.
A qualitative interview study was conducted with 13 CAR T-cell therapy stakeholders including scientists, clinicians, and policy-makers in Canada. Questions were asked related to CAR T-cell therapy development, treating patients, challenges to implementation, and suggestions for logistical planning at the healthcare system level.
A partitioned survival model was developed in TreeAge Pro Software from a Canadian public payer perspective. Patients with large B-cell lymphomas were modeled and their health outcomes from being treated with axicabtagene ciloleucel or salvage chemotherapy were extrapolated over a lifetime time horizon. The cost-effectiveness was evaluated by the incremental cost-effectiveness ratio.
The results from the qualitative interviews were summarized into 4 main themes: novel, patient characteristics and experiences, processes from “bench to bedside”, and future state of CAR T-cell therapy in Canada, including both challenges and recommendations to ensure sustainability.
The ICER generated for the cost-effectiveness analysis was $170,380 per QALY. At a willingness-to-pay threshold of $150,000 per QALY, CAR T-cell therapy is not cost-effective. There is some uncertainty in the long-term efficacy of CAR T-cell therapy and in the cost of CAR T-cell therapy.
Valuable perspectives from CAR T-cell therapy stakeholders on the current and future state of CAR T-cell therapy were highlighted from a Canadian perspective. In addition, a reduction in price of CAR T-cell therapy and reduced uncertainty through collection of long-term health outcomes can improve the cost-effectiveness to ensure value-for-money.||en