NICE has approved tisagenlecleucel therapy for use within the Cancer Drugs Fund as an option for treating relapsed or refractory diffuse large B-cell lymphoma in adults after two or more systemic therapies.
The decision relies on clinical evidence from the phase 2, open-label, single-arm JULIET study and a small observational study published in 2017.
The clinical evidence for tisagenlecleucel came from a phase 2, open-label single-arm study (JULIET) and a small observational study (Schuster 2017). Both studies included patients who could not have autologous stem cell transplant or whose disease had not responded to it. The company presented results from 111 patients from JULIET and 14 patients from the observational study.
At the December 2017 data-cut, the overall response rate (ORR) in JULIET was 51.6% (95% CI 41%-62%) and the complete response rate (CRR) was 39.8% (CI not reported). Median overall survival (OS) was 11.7 months.
In the small observational study, ORR was 50% (95% CI 23%-77%) and CRR was 43% (95% CI 18%-71%). Median OS was 22.2 months and median progression-free survival was 3.2 months.
While NICE noted that the survival data from JULIET is immature and the observational study cohort was small, it said the results are promising. It also remarked that there was currently no evidence on the effectiveness of tisagenlecleucel directly compared with that of salvage chemotherapy.
Because of the uncertainty around long-term benefits and the resulting uncertainty regarding cost-effectiveness, NICE took the decision not to recommend tisagenlecleucel for the routine treatment. However, tisagenlecleucel is recommended for use in the Cancer Drugs Fund.