Primary Risk Drivers
Below is a snapshot of domains that materially influence the MARA Rating.
Clinical effectiveness
The clinical evidence indicates that budesonide ODT significantly improves remission rates in eosinophilic oesophagitis compared to placebo, with a clinico-histological remission rate of 57.6% versus 0% in the placebo group (p<0.0001). However, there is no direct comparison with fluticasone or the 6-food elimination diet, which introduces some uncertainty.
Cost effectiveness
The cost-effectiveness estimates for budesonide ODT are within the range NICE considers acceptable (£20,000 to £30,000 per QALY gained). The committee noted that the estimates are sensitive to model inputs but concluded that the treatment is likely cost-effective.
Quality of life
The treatment is expected to improve quality of life for patients suffering from eosinophilic oesophagitis, as indicated by the significant symptom resolution associated with remission. However, specific validated HRQoL data were not provided in the document.
Supporting Domains
Safety and Adverse Effects
Budesonide ODT has a favorable safety profile with manageable adverse effects, as indicated by the clinical trial data. The document does not report any serious adverse events associated with its use.
Comparator Selection
The comparators selected include off-label fluticasone and dietary interventions, which are relevant but not ideal. There is no direct evidence comparing budesonide ODT with these treatments, leading to uncertainty in the indirect comparisons.
Patient Population and Subgroups
The patient population for the clinical trials is relevant, focusing on adults with active eosinophilic oesophagitis. The committee noted that the trial population is likely generalizable to the Healthcare practice.
Care Pathway Integration
Budesonide ODT is expected to fit well into existing treatment pathways for eosinophilic oesophagitis, as it addresses a significant unmet need and is likely to be used as a first-line treatment.
Resource Use and Cost Implications
The resource implications of budesonide ODT are manageable, and the committee concluded that the treatment is likely to be cost-effective, with a reasonable budget impact.
Evidence Quality and Robustness
The evidence base includes a well-conducted RCT (BUL-1/EEA) with a clear primary outcome. However, the reliance on indirect comparisons introduces some uncertainty regarding the robustness of the evidence.
Uncertainty, Sensitivity, and Broader Impacts
There is significant uncertainty in the cost-effectiveness estimates and the indirect treatment comparisons. The committee noted that the estimates are sensitive to small changes in model inputs, which could impact decision-making.
