RESUMO
OBJECTIVES: This paper presents an Australian model that formed part of the health technology assessment for public investment in siltuximab for the rare condition of idiopathic Multicentric Castleman Disease (iMCD) in Australia. METHODS: Two literature reviews were conducted to identify the appropriate comparator and model structure. Survival gain based on available clinical trial data were modelled using an Excel-based model semi-Markov model including time-varying transition probabilities, an adjustment for trial crossover and long-term data. A 20-year horizon was taken, and an Australian healthcare system perspective was adopted, with both benefits and costs discounted at 5%. The model was informed with an inclusive stakeholder approach that included a review of the model by an independent economist, Australian clinical expert opinion and feedback from the Pharmaceutical Benefits Advisory Committee (PBAC). The price used in the economic evaluation reflects a confidential discounted price, which was agreed to with the PBAC. RESULTS: An incremental cost-effectiveness ratio of A$84,935 per quality-adjusted life-year (QALY) gained was estimated. At a willingness-to-pay threshold of A$100,000 per QALY, siltuximab has a 72.1% probability of being cost-effective compared with placebo and best supportive care. Sensitivity analyses results were most sensitive to the length of interval between administrations (from 3- to 6-weekly) and crossover adjustments. CONCLUSION: Within a collaborative and inclusive stakeholder framework, the model submitted to the Australian PBAC found siltuximab to be cost-effective for the treatment of iMCD.
RESUMO
AIM: FreeStyle Libre™ Flash Glucose Monitoring System (Flash GM), a novel, sensor-based, factory-calibrated system has been compared with self-monitoring of blood glucose in a well-controlled adult type 1 diabetes mellitus (T1D) population (HbA1c ≤ 7.5%, 58 mmol/mol), in a randomized controlled trial (RCT). The need for RCTs to recruit homogenous patients and for a well-controlled environment may not necessarily reflect use of a new technology in real clinical practice. METHODS: A random effects meta-analysis of all identified studies in T1D was performed to investigate changes in laboratory-measured HbA1c following introduction of Flash GM. RESULTS: Flash GM introduction showed a mean change from baseline to longest follow-up timepoint of -0.41% ([95% CI -0.51%, -0.31%]; P < 0.001; -4.5 [95% CI -5.6, -3.3] mmol/mol) in HbA1c in the random effects meta-analysis (34 studies comprising 5,466 participants). When the Flash GM arms of the two RCTs were excluded, there was a similar change in HbA1c of -0.41% ([95% CI -0.50%, -0.32%], P < 0.001; -4.5 [95% CI -5.4, -3.5] mmol/mol) in the 32 uncontrolled studies. Considerable heterogeneity was shown in all meta-analyses (I2 values > 85%), likely due to the inclusion of diverse populations and variations in study protocols, meaning random effects meta-analyses should be strongly preferred. CONCLUSIONS: In people with T1D, use of Flash GM for 2 to 24 months was associated with an estimated HbA1c reduction from baseline of 0.4%. A similar reduction occurred in uncontrolled studies where baseline HbA1c was generally higher compared with Flash GM arms of well-controlled studies.
