Long-term cost-effectiveness of Dexcom G6 real-time continuous glucose monitoring system in people with type 1 diabetes in Australia.

INTRODUCTION: Real-time continuous glucose monitoring (rt-CGM) allows patients with diabetes to adjust insulin dosing, potentially improving glucose control. This study aimed to compare the long-term cost-effectiveness of the Dexcom G6 rt-CGM device versus self-monitoring of blood glucose (SMBG) and flash glucose monitoring (FGM) in Australia in people with type 1 diabetes (T1D). METHODS: Long-term costs and clinical outcomes were estimated using the CORE Diabetes Model. Clinical input data for the analysis of rt-CGM versus SMBG and FGM were sourced from the DIAMOND study and a network meta-analysis, respectively. Rt-CGM and FGM were associated with quality of life (QoL) benefits due to reduced fear of hypoglycaemia (FoH) and fingerstick testing. Analyses were performed over a lifetime time horizon from an Australian healthcare payer perspective, including direct costs from published data. Future costs and clinical outcomes were discounted at 5% per annum. RESULTS: Rt-CGM was associated with an increased quality-adjusted life expectancy of 1.199 quality-adjusted life years (QALYs), increased mean total lifetime costs of AUD 21,596 and an incremental cost-effectiveness ratio (ICER) of AUD 18,020 per QALY gained compared with SMBG. Compared with FGM, rt-CGM was associated with an increased quality-adjusted life expectancy of 0.569 QALYs, increased mean total lifetime costs of AUD 11,064 and an ICER of AUD 19,455 per QALY gained. Key drivers of outcomes included HbA1c benefits and QoL benefits associated with reduced FoH and fingerstick testing. CONCLUSIONS: Due to improved clinical outcomes and QoL gains rt-CGM is highly cost-effective compared with SMBG and FGM in people with T1D in Australia.

Elicitation of utility scores in Canada for immune thrombocytopenia treated with romiplostim or watch and rescue.

OBJECTIVES: The objective of this study was to obtain utilities, or preference-based quality-of-life values, from the Canadian general public, for potential health states experienced by immune thrombocytopenia (ITP) patients receiving either romiplostim (a new thrombopoietin mimetic agent) or 'watch and rescue' therapy. Utilities are needed to conduct a cost-utility analysis of romiplostim for formulary and reimbursement decisions. METHODS: An electronic Time Trade-off (TTO) survey was developed and administered to a sample of the general public in Canada, with 12 distinct health states derived from two randomized clinical trials of romiplostim vs watch and rescue treatment. Two pilot tests assessed interpretability and respondent burden. In the final survey, each subject was administered the TTO for four randomly-selected health states. Descriptive statistics were computed for utility scores, and differences between health states were evaluated with an analysis of variance model. RESULTS: Eight hundred and twenty-one adults completed the TTO survey. Mean age was 36.4 (SD = 15) years; 63% were female. Mean (SD) utility scores ranged from 0.476 (0.271) for the most severe health state (significant bleeding) to 0.633 (0.282) for the least severe health state depicting successful treatment with romiplostim. Statistical significance was found on the mean difference between the most severe health state and five other health states (p < 0.05). After adjusting utilities for matching Canadian demographic parameters, no substantial difference was found between original utility scores and adjusted scores. CONCLUSIONS: This study provides evidence of the Canadian general public's preference for 12 ITP health states pertaining to romiplostim treatment or watch and rescue. This study had a number of limitations, the main ones being the lack of perfect match in demographics between this sample and the Canadian population, as well as the fact that the scenario descriptions were based on both published literature and expert opinion. Despite those limitations, the obtained utility scores may be used in cost-utility models of romiplostim as a treatment for ITP patients in Canada.

Reliability and validity of a patient self-administered daily questionnaire to assess impact of oral mucositis (OM) on pain and daily functioning in patients undergoing autologous hematopoietic stem cell transplantation (HSCT).

Oral mucositis (OM) is a frequent complication of myeloablative therapy and HSCT. We evaluated the feasibility, reliability, and validity of a new patient self-reported daily questionnaire on OM and its impact on daily functions. This OM Daily Questionnaire (OMDQ), containing 10 items, was developed for use in palifermin clinical trials. In a phase 3 study, 212 patients received palifermin or placebo for three consecutive days before conditioning and three consecutive days after HSCT. Compliance rates were consistently >80% for most patients. Mouth and throat soreness (MTS) and MTS-Activity Limitations (MTS-AL) (swallowing, drinking, eating, talking, and sleeping) scores on consecutive days were highly correlated (days 7,8 = 0.70-0.86; test-retest reliability). Correlations among items measuring the same construct ranged between 0.5 and 0.8 (internal consistency reliability). The WHO Oral Toxicity scale was the clinical comparator to assess the criterion, discriminative, and evaluative validities of MTS-related questions. Most correlation coefficients between the WHO and MTS ranged between 0.45 and 0.55. Patients with more severe WHO OM grades had higher MTS mean scores. Changes in MTS scores were similar, but patients detected changes 1-3 days earlier than clinicians. In conclusion, the OMDQ is a feasible, reliable, valid, and responsive patient-reported measure of OM severity.