Primary Risk Drivers
Below is a snapshot of domains that materially influence the MARA Rating.
Clinical effectiveness
The clinical evidence for futibatinib comes from the FOENIX-CCA2 trial, a phase 2, single-arm study that reported an objective response rate of 41.7% and a median overall survival of 20.0 months. However, there is no direct comparison with pemigatinib, the current standard of care, leading to uncertainty about its relative effectiveness. The indirect comparison suggests similar efficacy, but the lack of robust head-to-head data limits the strength of the evidence.
Cost effectiveness
The economic model indicates that futibatinib has similar costs to pemigatinib, and the committee concluded that the cost-effectiveness estimates for futibatinib are within NICE’s acceptable range. This suggests that it can be considered a cost-effective use of Healthcare resources, although specific ICER values are confidential.
Quality of life
Patient experts indicated that futibatinib, being an oral treatment, offers a considerable quality-of-life advantage by avoiding hospital visits for chemotherapy. This aligns with the evidence suggesting that targeted treatments like futibatinib are generally well tolerated with mild toxicity, contributing to improved daily functioning.
Supporting Domains
Safety and Adverse Effects
The safety profile of futibatinib appears favorable, with patient experts noting that targeted treatments typically have mild side effects. The evidence suggests that adverse events are manageable, which supports a good tolerability rating.
Comparator Selection
The only relevant comparator identified is pemigatinib, as it is the standard treatment for this patient population. However, the lack of direct comparative trials limits the robustness of the evidence regarding futibatinib’s effectiveness relative to pemigatinib.
Patient Population and Subgroups
The trial population in FOENIX-CCA2 is relevant to the intended patient population, focusing on adults with advanced cholangiocarcinoma with FGFR2 alterations. The evidence includes insights from patient experts, enhancing the understanding of the population’s needs.
Care Pathway Integration
Futibatinib can be integrated into existing treatment pathways with minor adjustments, primarily due to its oral administration. This reduces the need for hospital visits and aligns well with current clinical practices for managing advanced cholangiocarcinoma.
Resource Use and Cost Implications
The budget impact of futibatinib is manageable, and the committee noted that it is similar in cost to pemigatinib. This suggests that the resource implications are justifiable given the potential benefits, supporting its adoption in the Healthcare.
Evidence Quality and Robustness
The evidence base is primarily derived from a single-arm trial, which introduces limitations in robustness and potential biases. While the indirect comparison provides some insights, the overall evidence quality is moderate due to the lack of direct comparative data.
Uncertainty, Sensitivity, and Broader Impacts
There is significant uncertainty regarding the comparative effectiveness of futibatinib versus pemigatinib, particularly due to the reliance on indirect comparisons. This uncertainty may impact decision-making and restrict the use of futibatinib without further evidence.
