NICE has approved tisagenlecleucel (Kymriah) therapy for the treatment of relapsed or refractory B‑cell acute lymphoblastic leukaemia in people aged up to 25 years, within the Cancer Drug Fund.
The decision is based on clinical evidence from three single-arm studies: the international, multicentre, phase 2 ELIANA study (n=97), the US phase 2 ENSIGN study (n=73) and the single-centre phase 1/2a B2101J study (n=66). Results from the individual studies showed that tisagenlecleucel was effective in inducing remission and improving survival for patients with B‑cell acute lymphoblastic leukaemia.
In the ELIANA study, the overall remission rate was 81% (95% CI 71-89) and 12-month survival was 76% (95% CI 63-86).
In the ENSIGN trial, the overall remission rate was 69% (95% CI 53-82). Median overall survival was 23.8 months (95% CI 9-not reached) and 12-month survival was 63% (95% CI 46-76).
In the B2101J study, overall remission rate was 93%, while overall survival was not recorded, survival at 12 months was 79% (95% CI 69-91).
However, there is no clinical evidence directly comparing tisagenlecleucel with blinatumomab or salvage chemotherapy because all three trials were single‑arm studies. The NICE appraisal committee concluded that the lack of comparative data made the assessment of comparative effectiveness and cost‑effectiveness challenging.
Having concluded that tisagenlecleucel could not be recommended for routine use, the committee acknowledged that tisagenlecleucel is an innovative treatment and has the potential to meet the criteria for routine use if the uncertainty around efficacy and cost-effectiveness could be addressed.
More long-term survival data and further data on the rate of subsequent allogeneic stem cell transplants would allow for a more robust cost-effectiveness estimate, the committee said, and hence agreed to including the treatment in the Cancer Drugs Fund.