1.1 Axicabtagene ciloleucel therapy is recommended for use within the Cancer Drugs Fund as an option for treating relapsed or refractory diffuse large B‑cell lymphoma or primary mediastinal large B‑cell lymphoma in adults after 2 or more systemic therapies, only if the conditions in the managed access agreement are followed.
1.2 This recommendation is not intended to affect both treatment in preparation for and treatment with axicabtagene ciloleucel that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.
Why the committee made these recommendations
There is no standard treatment for relapsed or refractory diffuse large B‑cell lymphoma or primary mediastinal large B‑cell lymphoma after 2 or more systemic therapies. Best supportive care is used and usually includes salvage chemotherapy. Axicabtagene ciloleucel is a chimeric antigen receptor (CAR) T‑cell therapy. It contains the patient's own T cells that have been modified to attach to and kill cancer cells.
Evidence from a small, single-arm study suggests that people having axicabtagene ciloleucel have clinically meaningful overall and progression-free survival and good response rates. However, the evidence is uncertain because there is limited follow‑up and no direct data comparing axicabtagene ciloleucel with salvage chemotherapy. Limitations in the available data mean that the exact size of the benefit of axicabtagene ciloleucel compared with salvage chemotherapy is unknown. There is also not enough evidence to determine the costs of treating side effects.
Axicabtagene ciloleucel meets NICE's criteria to be considered a life-extending treatment at the end of life. The most plausible cost-effectiveness estimates for axicabtagene ciloleucel compared with salvage chemotherapy are uncertain because survival data on axicabtagene ciloleucel are immature. However, the range of cost-effectiveness estimates shows that axicabtagene ciloleucel has plausible potential to be cost effective, and collecting further data on progression-free survival, overall survival and immunoglobulin usage will reduce the uncertainty in the evidence. Therefore, axicabtagene ciloleucel is recommended for use as an option within the Cancer Drugs Fund.