1.1 Tisagenlecleucel therapy is recommended for use within the Cancer Drugs Fund as an option for treating relapsed or refractory B‑cell acute lymphoblastic leukaemia in people aged up to 25 years, 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 tisagenlecleucel 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. For young people aged under 18 years, this decision should be made jointly by the clinician and the young person, or the young person's parents or carers.
Why the committee made these recommendations
Current treatment for relapsed or refractory acute lymphoblastic leukaemia is usually blinatumomab or salvage chemotherapy. Tisagenlecleucel 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.
Clinical trial evidence suggests that compared with current treatment, people having tisagenlecleucel may live for longer, or have more time before their disease relapses. However, the evidence is uncertain and it is not known whether tisagenlecleucel can cure acute lymphoblastic leukaemia.
There is also not enough evidence to determine the costs of treating side effects and whether people will need a subsequent stem cell transplant.
The most plausible cost‑effectiveness estimates for tisagenlecleucel are higher than what NICE normally considers acceptable. The life expectancy of people with relapsed or refractory acute lymphoblastic leukaemia is uncertain. It does not meet both of NICE's criteria to be a life-extending treatment at the end of life. Therefore tisagenlecleucel cannot be recommended for routine use in the NHS.
Collecting more data on overall survival, subsequent stem cell transplant rates, and immunoglobulin usage will reduce the uncertainty in the clinical‑ and cost‑effectiveness evidence. Therefore tisagenlecleucel is recommended for use in the Cancer Drugs Fund.