What payers will ask
Is the clinical benefit the kind payers reward? — Clinical effectiveness
Foslevodopa-foscarbidopa shows moderate benefit over standard care, with a significant improvement in ‘on’ time without troublesome dyskinesia and a reduction in ‘off’ time. The primary endpoint of the phase 3 trial M15-736 demonstrated a clinically significant improvement of 1.75 hours in ‘on’ time and 1.79 hours in ‘off’ time compared to oral levodopa-carbidopa. However, the uncertainty regarding its effectiveness in patients who cannot have apomorphine limits the strength of the evidence.
Does the economic case hold at the expected price? — Cost effectiveness
The cost-effectiveness estimates for foslevodopa-foscarbidopa are within the range that NICE considers acceptable, particularly when compared to standard care. The committee noted that the ICERs were likely to be at the lower end of the acceptable range, suggesting a defensible economic value.
Is there quality-of-life evidence payers weigh? — Quality of life
The treatment is likely to improve quality of life, particularly related to sleep and daily functioning, as indicated by patient expert testimonies. However, the exact benefits related to sleep were not adequately captured in the economic modelling, leading to some uncertainty.
Does the safety profile hold up for payers? — Safety and Adverse Effects
Foslevodopa-foscarbidopa has a very good safety profile, with mostly mild or moderate adverse events reported. Serious adverse events were rare, indicating a favorable tolerability compared to existing therapies.
Was the drug compared against what payers expect? — Comparator Selection
The treatment was primarily compared to standard care and levodopa-carbidopa intestinal gel, which are appropriate comparators. However, the exclusion of patients who could have apomorphine or deep brain stimulation limits the robustness of the comparisons.
Is the population defined the way payers need it? — Patient Population and Subgroups
The population considered for the evaluation is narrower than the marketing authorization, focusing on those who cannot have apomorphine or deep brain stimulation. This raises concerns about generalizability, although it reflects a high unmet need.
Does the drug fit how care is delivered and paid for? — Care Pathway Integration
Foslevodopa-foscarbidopa can be integrated into existing care pathways with minor adjustments, as it is delivered via a subcutaneous infusion that is simpler than some existing treatments.
Are the wider system costs understood? — Resource Use and Cost Implications
The treatment is expected to have a manageable budget impact, with potential cost savings associated with improved management of Parkinson’s symptoms and reduced need for other treatments.
Would the evidence survive payer scrutiny? — Evidence Quality and Robustness
The evidence base includes a phase 3 RCT (M15-736) and supporting studies, although there are some concerns regarding the generalizability of the trial population and the potential for unblinding.
How exposed is the case to uncertainty? — Uncertainty, Sensitivity, and Broader Impacts
There are several uncertainties regarding the ICERs, the effectiveness in the narrower population, and the potential benefits not captured in the modelling. This leads to a moderate level of uncertainty in the overall assessment.
