Axicabtagene ciloleucel is recommended for use within the Cancer Drugs Fund as an option for treating diffuse large B‑cell lymphoma in adults when an autologous stem cell transplant is suitable if it:
has relapsed within 12 months after first-line chemoimmunotherapy or
is refractory to first-line chemoimmunotherapy.
It is recommended only if the conditions in the managed access agreement for axicabtagene ciloleucel are followed.
This recommendation is not intended to affect 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
Standard care for relapsed or refractory diffuse large B‑cell lymphoma after first-line chemoimmunotherapy is chemotherapy followed by high-dose chemotherapy and an autologous stem cell transplant. Axicabtagene ciloleucel is a chimeric antigen receptor (CAR) T‑cell therapy. It uses the person's own immune system cells (T‑cells) that have been modified to attach to and kill cancer cells. It would be used as an alternative to standard care.
Clinical trial evidence suggests that, when an autologous stem cell transplant is suitable, axicabtagene ciloleucel increases how long people live compared with standard care. But it is uncertain by how much because the trial is still ongoing. Some people in the trial who had standard care went on to have a CAR T‑cell therapy. This is not standard care in the NHS, so adjusting the data to reflect this also adds uncertainty. Also, another treatment that is used during CAR T‑cell therapy in the NHS was not used in the trial. This adds uncertainty because it is not clear if the results from the trial fully reflect what would happen when using axicabtagene ciloleucel in the NHS.
Axicabtagene ciloleucel meets NICE's criteria to be considered a life-extending treatment at the end of life. The cost-effectiveness estimates are within the range NICE normally considers an acceptable use of NHS resources for end of life treatments. But, because of the uncertainty, an acceptable cost-effectiveness estimate would need to be at the lower end of the range. The cost-effectiveness estimate for axicabtagene ciloleucel is above the lower end of the range, so it cannot be recommended for routine use in the NHS.
Axicabtagene ciloleucel has the potential to be cost effective, but more evidence is needed to reduce the uncertainties. Evidence from the trial and from NHS practice could help address these. So, axicabtagene ciloleucel is recommended for use in the Cancer Drugs Fund.