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New HTA Decisions in England
February 2019
Drug name
KYMRIAH® (tisagenlecleucel)
Company
Novartis Pharma
Decision date
21/12/2018
Therapeutic area
Blood and immune system conditions
Therapeutic sub area
Blood and bone marrow cancers
Decision
Recommended (CDF)
Indication
An option for treating relapsed or refractory B‑cell acute lymphoblastic leukaemia in people aged up to 25 years
Decision Detail
Only if the conditions in the managed access agreement are followed
Summary
The main evidence for clinical-effectiveness of tisagenlecleucel was presented by 3 single-arm studies; ELIANA, ENSIGN and B2101J. Individual study results showed that tisagenlecleucel was effective in inducing remission and improving survival for patients with B‑cell acute lymphoblastic leukaemia. It was noted by the committee, however, that the lack of clinical evidence directly comparing tisagenlecleucel with blinatumomab or salvage chemotherapy made assessment of comparative effectiveness more challenging. Additionally, there is no robust evidence to suggest that tisagenlecleucel has a curative effect, given that survival estimates beyond 30 months were highly uncertain. The most plausible cost-effectiveness estimates for tisagenlecleucel are higher than what NICE normally considers acceptable. It was concluded that clinical uncertainties could be addressed through further data collection and this would allow a more accurate reflection of costs and benefits associated with tisagenlecleucel in clinical practice. Tisagenlecleucal can be recommended for use in the Cancer Drugs Fund whilst further data on overall survival, subsequent stem cell transplant rates and immunoglobulin usage are obtained.