Health state utility values for children and adolescents with type 1 diabetes: A systematic review and meta-analysis.

BACKGROUND: Type 1 diabetes mellitus (T1DM) is one of the most burdensome chronic diseases in the world. Health utility values are an important tool for quantifying this disease burden and conducting cost-utility analyses. This review aimed to derive a reference set of health utility values for children and adolescents with T1DM. METHODS: We searched MEDLINE and Embase from inception to March 2023 for health utility values of T1DM children and adolescents (aged ≤18 years) measured using direct and indirect preference elicitation approaches. Utility estimates were pooled by meta-analyses with subgroup analyses to evaluate differences by populations and elicitation approaches. RESULTS: Six studies involving 1276 participants were included in this study. Meta-analysis showed the overall utility value of children and adolescents with T1DM was 0.91 (95% CI 0.89-0.93). The utility value of T1DM children and adolescents with complications was 0.90 (95% CI 0.88-0.92), which was lower than those without complications (0.96, 95% CI 0.95-0.97). The utility value of children (aged <13 years) was higher than adolescents (aged 13-18 years) (0.90 vs. 0.85). The utility value measured by the EQ-5D-3L (0.91) was higher than the HUI3 (0.89), the SF-6Dv1 (0.83), and the time trade-off (0.81). The parent proxy-reported was similar to the patient self-reported (0.91 vs. 0.91). CONCLUSIONS: This study developed a reference set of pooled utility estimates for children and adolescents with T1DM, which is helpful for understanding the overall health status of T1DM and conducting economic evaluations. Further studies are needed to explore the utilities of T1DM with different types of complications.

Endovascular Thrombectomy with or without Bridging Thrombolysis in Acute Ischemic Stroke: A Cost-Effectiveness Analysis.

BACKGROUND: There is unclear added benefit of intravenous thrombolysis (IVT) with endovascular thrombectomy (EVT). We performed a cost-effectiveness analysis to assess the cost-effectiveness of comparing EVT with IVT versus EVT alone. METHODS: We used a decision tree to examine the short-term costs and outcomes at 90 days after the occurrence of index stroke to compare the cost-effectiveness of EVT alone with EVT plus IVT for patients with stroke. Subsequently, we developed a Markov state transition model to assess the costs and outcomes over 1-year, 5-year, and 20-year time horizons. We estimated total and incremental cost, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio. RESULTS: The average costs per patient were estimated to be $47,304, $49,510, $59,770, and $76,561 for EVT-only strategy and $55,482, $57,751, $68,314, and $85,611 for EVT with IVT over 90 days, 1 year, 5 years, and 20 years, respectively. The cost saving of EVT-only strategy was driven by the avoided medication costs of IVT (ranging from $8,178 to $9,050). The additional IVT led to a slight decrease in QALY estimate during the 90-day time horizon (loss of 0.002 QALY), but a small gain over 1-year and 5-year time horizons (0.011 and 0.0636 QALY). At a willingness-to-pay threshold of $50,000 per QALY gained, the probabilities of EVT only being cost-effective were 100%, 100%, and 99.3% over 90-day, 1-year, and 5-year time horizons. CONCLUSION: Our cost-effectiveness model suggested that EVT only may be cost-effective for patients with acute ischemic stroke secondary to large vessel occlusion.

Key Issues of Economic Evaluations for Health Technology Assessment in China: A Nationwide Expert Survey.

OBJECTIVES: Health technology assessment (HTA) is increasingly crucial in medicine price negotiations in China, yet previous appraisals revealed national discrepancies on key economic evaluation issues: willingness-to-pay threshold, pricing models for multi-indication medicines, and comparator selection principles. This study aimed to collect expert opinions on these issues for future HTA evaluations. METHODS: A nationwide anonymous web-based survey encompassing experts across academia, HTA, consultancy/contract research organization/industry, service provider, and payer. In 2023, a generic invitation containing a web link to the questionnaire was disseminated via WeChat using convenience and snowball sampling. Agreement rates for questionnaire views were analyzed using descriptive statistics. The relationship between participants' responses and demographics was examined using appropriate logistic models. RESULTS: A total of 303 responses were received from experts in 34 cities. Key expert views include a suggested base willingness-to-pay threshold ranging from 0.5 to 1.5 times gross domestic product (52.1% agreement); elevated thresholds for childhood diseases, rare diseases, end-of-life diseases, and first-in-class medicines (>78.0% agreement); a single pricing model for multi-indication medicines (60.4% agreement); consideration of multiple medicines as comparators (79.9% agreement); and avoiding the use of centrally procured medicines as comparators for medicines with a time to market of less than 3 years (71.0% agreement). Participants who are service provider had lower odds of selecting higher thresholds (odds ratio 0.26; P < .01) than responders from consultancy/contract research organization/industry. CONCLUSIONS: Expert views indicate the need for substantial changes in China's current HTA methods, highlighting the need for increased investment in HTA processes and expertise cultivation.

Comparison of the measurement properties and consistency between the EQ-5D-3L and EQ-5D-Y-3L in adolescents aged 15-17 in China.

OBJECTIVE: To evaluate and compare the measurement properties and consistency between the Chinese versions of EQ-5D-3L and EQ-5D-Y-3L among Chinese adolescent populations aged 15-17 years. METHODS: Chinese adolescents aged 15-17 studying in high school were recruited through online survey. Social-demographic characteristics and self-reported EQ-5D-3L and EQ-5D-Y-3L responses were collected in the survey. The consistency of responses between the two measures was assessed using redistribution property, and the consistency of utility values was assessed by intraclass correlation coefficients (ICC). Convergent validity and known-group validity were examined using Spearman's rank correlation, F-test and effect sizes, respectively. Sensitivity was compared using relative efficiency (RE). RESULTS: 762 respondents (48.8% male; age 15-17 years;) were recruited. The EQ-5D-3L showed a more severe ceiling effect than EQ-5D-Y-3L (78.2% vs. 66.0%). Respondents reported higher proportions of having problems in four dimensions using the EQ-5D-Y-3L than using the EQ-5D-3L. The consistency of corresponding dimensions between the two measures was relatively good, while non-negligible proportions of inconsistency were observed in "pain/discomfort" (11.4%) and "anxiety/depression" (15.7%) dimensions. The ICC of the utility values between the EQ-5D-3L and EQ-5D-Y-3L was 0.852 (p < 0.001). The Spearman's rank correlation (range: 0.385-0.620) indicated an acceptable convergent validity between the correlative dimensions of the EQ-5D-3L and EQ-5D-Y-3L. The EQ-5D-Y-3L had a higher efficiency than the EQ-5D-3L at detecting differences across EQ VAS subgroups (ES = 1.793 for EQ-5D-3L, ES = 1.920 for EQ-5D-Y-3L). Mixed results were observed in sensitivity. CONCLUSIONS: Both the EQ-5D-3L and EQ-5D-Y-3L are demonstrated to be valid and generally consistent for measuring HRQoL among adolescents aged 15-17 years in China. Respondents reported higher proportions of having problems using the EQ-5D-Y-3L than using the EQ-5D-3L. More research is warranted to compare the discriminant validity and test-retest reliability between the two measures.

Understanding Canadian stakeholders' views on measuring and valuing health for children and adolescents: a qualitative study.

OBJECTIVE: Valuing child health is critical to assessing the value of healthcare interventions for children. However, there remain important methodological and normative issues. This qualitative study aimed to understand the views of Canadian stakeholders on these issues. METHODS: Stakeholders from health technology assessment (HTA) agencies, pharmaceutical industry representatives, healthcare providers, and academic researchers/scholars were invited to attend an online interview. Semi-structured interviews were designed to focus on: (1) comparing the 3-level and 5-level versions of the EQ-5D-Y; (2) source of preferences for valuation (adults vs. children); (3) perspective of valuation tasks; and (4) methods for valuation (discrete choice experiment [DCE] and its variants versus time trade-off [TTO]). Participants were probed to consider HTA guidelines, cognitive capacity, and potential ethical concerns. All interviews were recorded and transcribed verbatim. Framework analysis with the incidence density method was used to analyze the data. RESULTS: Fifteen interviews were conducted between May and September 2022. 66.7% (N = 10) of participants had experience with economic evaluations, and 86.7% (N = 13) were parents. Eleven participants preferred the EQ-5D-Y-5L. 12 participants suggested that adolescents should be directly involved in child health valuation from their own perspective. The participants were split on the ethical concerns. Eight participants did not think that there was ethical concern. 11 participants preferred DCE to TTO. Among the DCE variants, 6 participants preferred the DCE with duration to the DCE with death. CONCLUSIONS: Most Canadian stakeholders supported eliciting the preferences of adolescents directly from their own perspective for child health valuation. DCE was preferred if adolescents are directly involved.

Mapping IWQOL-Lite onto EQ-5D-5L and SF-6Dv2 among overweight and obese population in China.

PURPOSE: To develop the mapping functions from the Impact of Weight on Quality of Life-Lite (IWQOL-Lite) scores onto the EQ-5D-5L and SF-6Dv2 utility values among the overweight and obese population in China. METHODS: A representative sample of the overweight and obese population in China stratified by age, sex, body mass index (BMI), and area of residence was collected by online survey and the sample was randomly divided into development (80%) and validation (20%) datasets. The conceptual overlap between the IWQOL-Lite and the EQ-5D-5L or SF-6Dv2 was evaluated by Spearman's correlation coefficients. Five models, including OLS, Tobit, CLAD, GLM, and PTM were explored to derive mapping functions using the development dataset. The model performance was assessed using MAE, RMSE, and the percentage of AE > 0.05 and AE > 0.1 in the validation dataset. RESULTS: A total of 1000 respondents (48% female; mean [SD] age: 51.7 [15.3]; mean [SD] BMI: 27.4 [2.8]) were included in this study. The mean IWQOL-Lite scores and the utility values of EQ-5D-5L and SF-6Dv2 were 78.5, 0.851, and 0.734, respectively. The best-performing models predicting EQ-5D-5L and SF-6Dv2 utilities both used IWQOL-Lite total score as a predictor in the CLAD model (MAE: 0.083 and 0.076 for the EQ-5D-5L and SF-6Dv2; RMSE: 0.125 and 0.103 for the EQ-5D-5L and SF-6Dv2; AE > 0.05: 20.5% and 27.5% for the EQ-5D-5L and SF-6Dv2; AE > 0.10: 9.5% and 15.0% for the EQ-5D-5L and SF-6Dv2). CONCLUSION: CLAD models with the IWQOL-Lite total score can be used to predict both the EQ-5D-5L and SF-6Dv2 utility values among overweight and obese population in China.

Cost-effectiveness of brentuximab vedotin compared with conventional chemotherapy for relapsed or refractory classic Hodgkin lymphoma in China.

BACKGROUND: Relapsed or refractory classic Hodgkin lymphoma (RRcHL) associates with poor prognosis and heavy disease burden to patients. This study evaluated the cost-effectiveness of brentuximab vedotin (BV) in comparison to conventional chemotherapy in patients with RRcHL, from a Chinese healthcare perspective. METHODS: The lifetime cost and quality adjusted life years (QALYs) were estimated through a partitioned survival model with three health states (progression free, post progression, and death). Two cohorts for each BV arm and chemotherapy arm were built, representing patients with and without transplant after BV or chemotherapy, respectively. Clinical parameters were retrieved from BV trials and the literature. Resource utilization data were mainly collected from local expert surveys and cost parameters were reflecting local unit prices. Utility values were sourced from the literature. A discount rate of 5% was employed according to the Chinese guideline. A series of deterministic and probabilistic sensitivity analyses were conducted to evaluate the robustness and uncertainty associated with the model. RESULTS: Results of the base case analysis showed that the incremental cost-effectiveness ratio (ICER) for BV versus chemotherapy was $2,867 (¥19,774). The main model driver was the superior progression-free and overall survival benefits of BV. The ICERs were relatively robust in a series of sensitivity analyses, all under a conventional decision threshold (1 time of Chinese per capita GDP). With this conventional threshold, the probability of BV being cost-effective was 100%. CONCLUSIONS: Brentuximab vedotin can be considered a cost-effective treatment versus conventional chemotherapy in treating relapsed or refractory classic Hodgkin lymphoma in China.

Evaluating the Preferences and Willingness-to-Pay for Oral Antidiabetic Drugs Among Patients with Type 2 Diabetes Mellitus in China: A Discrete Choice Experiment.

PURPOSE: To quantify the preferences for an oral antidiabetic drug (OAD) among patients with type 2 diabetes mellitus (T2DM) in China. METHODS: A discrete choice experiment (DCE) with hypothetical OAD profiles was performed among patients with T2DM recruited from both online and offline sources. Each patient completed 12 DCE choice tasks. The attributes, elicited through mixed methods, include blood glucose level decrease, blood glucose level stability, frequency of medication, gastrointestinal side effects, dose adjustment and out-of-pocket expense. The conditional logit regression model was used to analyze the data. Patients' willingness-to-pay (WTP) was also calculated. Subgroup analyses based on patient characteristics were also conducted. RESULTS: A total of 741 respondents were included in the analysis sample, covering 456 respondents online and 285 offline. The result showed that all attributes and levels were statistically significant, except one level "dose adjustment required for patients with hepatic or renal insufficiency" in the attribute of dose adjustment. WTP results showed that patients were willing to pay 12.06 and 23.20 yuan, respectively to reduce the frequency of medication from "once per day" and "three times per day" to "once every 2 weeks", respectively. Subgroup analyses showed that the frequency of medication (once versus two to three times per day) had the largest impact and influenced most coefficient estimates. CONCLUSION: The results suggest that Chinese patients with T2DM prioritized better efficacy, less frequency of medication, lower gastrointestinal side effects, no dose adjustment required for patients with hepatic or renal insufficiency, and less out-of-pocket expense of OAD treatment.