Primary Risk Drivers
Below is a snapshot of domains that materially influence the MARA Rating.
Clinical effectiveness
The clinical trial evidence from the E1910 trial demonstrates that blinatumomab with chemotherapy significantly improves overall survival and relapse-free survival compared to chemotherapy alone, with a hazard ratio of 0.44 for overall survival and 0.53 for relapse-free survival. This indicates a clear clinical advantage over the standard of care.
Cost effectiveness
The cost-effectiveness estimates for blinatumomab with chemotherapy are around £30,000 per QALY gained, which is within the acceptable range for Healthcare resources. This suggests that the therapy is marginally cost-effective, supported by the significant survival benefits observed.
Quality of life
The trial did not collect direct HRQoL data, and the utility values used in the economic model were derived from other studies. While the committee acknowledged the potential for improved quality of life, the lack of direct evidence limits the strength of this claim.
Supporting Domains
Safety and Adverse Effects
Blinatumomab is reported to have a good safety profile, with the clinical experts indicating that it is generally well tolerated compared to chemotherapy. The adverse events associated with blinatumomab are mostly mild to moderate, supporting a very good tolerability rating.
Comparator Selection
The comparator selected for the evaluation, which is chemotherapy, is appropriate as it reflects the standard treatment for the target population. The committee agreed that this was the most relevant comparator for the evaluation.
Patient Population and Subgroups
The trial population is broadly representative of the intended patient population, including adults aged 30 to 70 years. The committee noted that the results are likely generalizable to younger adults as well, addressing potential concerns about age-related access.
Care Pathway Integration
Blinatumomab can be integrated into existing treatment pathways with manageable adjustments. The committee noted that the treatment schedule aligns with current clinical practices, indicating a good fit within the healthcare delivery system.
Resource Use and Cost Implications
The economic model indicates that the resource use associated with blinatumomab is manageable and aligns with Healthcare planning. The committee concluded that the budget impact is justifiable given the clinical benefits.
Evidence Quality and Robustness
The evidence base is supported by a robust Phase 3 RCT (E1910) with a well-defined methodology and low risk of bias. The committee found the evidence credible and reliable for decision-making.
Uncertainty, Sensitivity, and Broader Impacts
While there are some uncertainties regarding long-term survival projections, the committee noted that these uncertainties are manageable and do not significantly undermine the overall conclusions about the treatment’s effectiveness.
