What payers will ask
Is the clinical benefit the kind payers reward? — Clinical effectiveness
Vadadustat demonstrated comparable efficacy to erythropoiesis stimulating agents (ESAs) in increasing hemoglobin levels, meeting non-inferiority criteria. However, the evidence indicates that vadadustat does not provide a clear advantage over ESAs, as shown by the trials where the primary efficacy outcome favored darbepoetin alfa, albeit within the non-inferiority margin.
Does the economic case hold at the expected price? — Cost effectiveness
The cost-effectiveness analysis indicated that vadadustat is less effective and less costly than ESAs, with an ICER over £30,000 per QALY. This suggests low cost-effectiveness, requiring justification for its price relative to the benefits it provides.
Is there quality-of-life evidence payers weigh? — Quality of life
The committee acknowledged that anaemia significantly impacts the quality of life for patients with chronic kidney disease (CKD). While specific HRQoL data for vadadustat was not detailed, the overall context suggests a moderate improvement in quality of life due to the treatment of symptomatic anaemia.
Does the safety profile hold up for payers? — Safety and Adverse Effects
The safety profile of vadadustat was comparable to that of darbepoetin alfa, with no significant differences in major adverse cardiovascular events. This indicates an acceptable safety profile with manageable adverse effects.
Was the drug compared against what payers expect? — Comparator Selection
The trials compared vadadustat directly with darbepoetin alfa, a standard treatment for symptomatic anaemia in CKD patients on dialysis. This direct comparison is appropriate and aligns with clinical practice.
Is the population defined the way payers need it? — Patient Population and Subgroups
The trials included a diverse population of patients with dialysis-dependent CKD, which enhances the generalizability of the findings. However, some subgroup analyses were limited, but the core population was well represented.
Does the drug fit how care is delivered and paid for? — Care Pathway Integration
Vadadustat can be integrated into existing treatment pathways with minimal disruption, as it offers an oral alternative to injectable ESAs, which may be beneficial for patients with needle phobia.
Are the wider system costs understood? — Resource Use and Cost Implications
While vadadustat is less costly than ESAs, the overall resource implications are significant, and the treatment may not be sustainable at scale without careful budget management.
Would the evidence survive payer scrutiny? — Evidence Quality and Robustness
The evidence base is supported by two large non-inferiority trials, which, despite some methodological concerns, provide a robust foundation for the evaluation of vadadustat.
How exposed is the case to uncertainty? — Uncertainty, Sensitivity, and Broader Impacts
There is notable uncertainty regarding the long-term effectiveness and cost-effectiveness of vadadustat, particularly given the variability in ESA pricing and treatment discontinuation rates.
