Blinatumomab (Blincyto) has failed to secure NICE approval as a treatment option in adults with Philadelphia chromosome-negative CD19-positive B-precursor acute lymphoblastic leukaemia (ALL) in first or second complete remission with minimal residual disease (MRD) of at least 0.1%.
In a draft appraisal document released this week, NICE says its appraisal committee was unable to recommend blinatumomab for this indication because of a number of uncertainties.
The clinical evidence for blinatumomab in this setting came from the single-arm BLAST (n=116) and MT103-202 (n=20) studies. Both studies included patients with ALL in complete haematological remission with MRD. NICE remarked on the small cohort in MT103-202 and noted that the overall survival (OS) data in BLAST was immature. The committee was concerned that the single-arm design of the studies meant that the results were potentially biased. It noted that there was no evidence on the effectiveness of blinatumomab directly compared with salvage chemotherapy. The committee concluded that blinatumomab is clinically effective, but immature survival data and the lack of direct comparative data meant the size of the benefit is unclear.
Additional information is being requested from the manufacturer including a revised cost-effectiveness analysis reflecting the current treatment pathway and comparing blinatumomab with standard care, as well as the latest available evidence on survival outcomes after haematopoietic stem cell transplantation (HSCT) and the latest trial data cut.
The draft decision is now open for public consultation. The closing data for receipt of comments is 25 March 2019. The appraisal committee will meet again on 3 April 2019 to review the additional information provided by the manufacturer and the comments received during the consultation process.