Interventions:
: Both treatments were adequately described, lenalidomide being compared with current standard practice in the authors’ setting. Dosages for both treatments were also presented.
Effectiveness/benefits:
: The two published studies used to derive clinical data appear to have been identified selectively rather than through a review of the literature. However, one of the two studies was the pivotal randomised trial for lenalidomide, which seems the most adequate source of clinical and safety data. Key details of the two studies were provided and adjustments were appropriately carried out to deal with the heterogeneity in patient population. The choice of the two benefit measures was appropriate to reflect the impact of the two treatments on patient health. The utility weights were derived from a small sample of patients, whose representativeness might be limited.
Costs:
The analysis of the costs appears to have been consistent with the authors' stated perspective. The unit costs and their sources were extensively reported. However, resource use data were not given for all items, which might limit the possibility of replicating the analysis in other settings. Statistical analyses of the costs were not performed and the issue of variability in individual cost items was not addressed, although they were varied in the probabilistic analysis. The price year was reported, which assists reflation exercises in other time periods.
Analysis and results:
: The costs and benefits were appropriately synthesised in incremental cost-effectiveness and cost-utility ratios. The description of the results from both the base-case analysis and sensitivity analysis was satisfactory. The issue of uncertainty was well addressed, although it was mainly restricted to key model inputs. The issue of generalisability was discussed and the authors pointed out that caution is required when extrapolating these findings given the limited availability of clinical data. However, the use of wide ranges of values in the sensitivity analysis enhances the external validity of the study results. Overall, there was little information on the decision model and no graphical depiction. A longer follow-up period would have been more appropriate to capture the full impact of the treatment, as the authors acknowledged. However, the choice of a 1-year follow up was justified by the data availability from a phase II trial, the fast track designation status of lenalidomide, and the lack of data to inform longer-term extrapolation of the trial results.
Concluding remark:
: The methodology of the study appears to have been sound and clearly reported. The selection of the key model inputs was justified and appropriate, despite the poor reporting of the decision model. The conclusions reached by the authors were robust.