Tisagenlecleucel is recommended, within its marketing authorisation, as an option for people 25 years and under for treating B‑cell acute lymphoblastic leukaemia that is:
relapsed after a transplant, or
relapsed for a second or later time, or
refractory.
It is only recommended if the company provides it according to the commercial arrangement.
Why the committee made these recommendations
This evaluation reviews the evidence for tisagenlecleucel for treating relapsed or refractory B‑cell acute lymphoblastic leukaemia (NICE technology appraisal guidance 554). It also reviews new evidence collected as part of the managed access agreement, which includes evidence from a clinical trial and from people having treatment in the NHS in England.
Usual treatment for B‑cell acute lymphoblastic leukaemia that is refractory, relapsed after a transplant, or relapsed for a second or later time in people 25 years and under includes blinatumomab and chemotherapy.
There are no clinical trials directly comparing tisagenlecleucel with usual treatments. But an indirect comparison suggests that people having tisagenlecleucel live longer than people having blinatumomab or chemotherapy.
When considering the condition's severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates are within the range that NICE considers an acceptable use of NHS resources. So, tisagenlecleucel is recommended for routine use in the NHS.