What payers will ask
Is the clinical benefit the kind payers reward? — Clinical effectiveness
Fostamatinib has demonstrated moderate clinical effectiveness in increasing platelet counts compared to placebo in the FIT trials. The primary endpoint of stable platelet response was achieved in 18% of patients receiving fostamatinib versus 2% in the placebo group. However, the benefits appear to decrease over time, and there is no direct comparison with rituximab or mycophenolate, which limits the strength of the evidence.
Does the economic case hold at the expected price? — Cost effectiveness
The cost-effectiveness estimates for fostamatinib compared to rituximab are within acceptable thresholds for Healthcare resources. The committee noted that the revised base case included preferred assumptions and that the ICERs were within the range NICE considers acceptable, indicating a clear cost-effective profile.
Is there quality-of-life evidence payers weigh? — Quality of life
The document does not provide robust evidence on HRQoL improvements associated with fostamatinib. While there are mentions of patient preferences and the potential for reduced anxiety due to fewer immunosuppressive effects, specific validated HRQoL data is lacking, leading to a mixed impact assessment.
Does the safety profile hold up for payers? — Safety and Adverse Effects
Fostamatinib has an acceptable safety profile, with adverse events being manageable. The document indicates that adverse events are generally mild to moderate, and the company has revised its approach to model these appropriately, suggesting a good tolerability compared to existing therapies.
Was the drug compared against what payers expect? — Comparator Selection
The selection of rituximab and mycophenolate as comparators is appropriate, as they are commonly used in clinical practice after TPO-RAs. Although the company excluded some comparators, the committee acknowledged the relevance of the chosen comparators based on clinical expert input.
Is the population defined the way payers need it? — Patient Population and Subgroups
The trials included a representative population of adults with chronic ITP, and the committee concluded that the results are likely generalizable to Healthcare practice. However, there are some concerns regarding the age and risk profile of trial participants compared to the general patient population.
Does the drug fit how care is delivered and paid for? — Care Pathway Integration
Fostamatinib can be integrated into existing treatment pathways with minor adjustments. The committee noted that it fits well into the treatment sequence after TPO-RAs, which suggests a manageable integration into current clinical practice.
Are the wider system costs understood? — Resource Use and Cost Implications
The resource implications of fostamatinib are manageable, with the potential for cost savings due to its effectiveness in increasing platelet counts. The committee recognized that the budget impact is aligned with planning, indicating a good resource use profile.
Would the evidence survive payer scrutiny? — Evidence Quality and Robustness
The evidence base is primarily derived from two Phase 3 trials (FIT1 and FIT2), which are robust but have limitations regarding generalizability and the absence of direct comparisons with other treatments. The committee acknowledged the quality of the evidence but noted some methodological concerns.
How exposed is the case to uncertainty? — Uncertainty, Sensitivity, and Broader Impacts
There are notable uncertainties regarding the long-term benefits of fostamatinib and its comparative effectiveness against other treatments. The committee highlighted these uncertainties but noted that they are somewhat mitigated by the unmet need for alternative treatments in this patient population.
