Value in Health最新文献

Objectives: In the absence of EuroQol 5-dimension (EQ-5D) utility data, the National Institute for Health and Care Excellence (NICE) recommend sourcing utility values from a proxy health condition, provided data indicate that the health-related quality of life (HRQoL) impact of the conditions is similar. Given there is no standard practice for identifying proxy conditions, this study presents a patient-centred approach for selecting a proxy condition, using alopecia areata (AA) as an example.

Methods: A structured electronic search was conducted to identify conceptual models for similar conditions with overlapping HRQoL domains with AA. Conceptual models were compared to determine the most suitable proxy condition, defined as the condition with the most overlapping domains and reported similarities between patient HRQoL. A search and comparison of utility data between AA and the selected proxy condition was conducted to validate their comparability in HRQoL impact.

Results: Seven conditions with overlapping HRQoL domains with AA were identified using conceptual models. Atopic dermatitis (AD) had the most overlapping domains (n = 6) and the greatest evidence of similarities between patient HRQoL, thus was selected as the proxy condition. Conceptual models indicated that both conditions affect patients' physical, emotional, and functional wellbeing. Furthermore, AD and AA utilities were comparable, supporting the choice of AD as the proxy condition.

Conclusions: Given the correlation observed between AA and the selected proxy condition using this methodology, this research has presented a potential approach to identify proxy conditions for diseases which may be underrepresented in terms of HRQoL data.

Objectives: To examine the cost-effectiveness of a digitally supported care management system (CMS) for caregivers of People with Dementia (PwD) compared with usual care.

Methods: The analysis was based on 192 caregivers (n=96 CMS, n=96 usual care) of PwD in a cluster-randomized, controlled trial testing a digitally supported CMS, aiming to identify and address caregivers' unmet needs and develop and implement an individualized support and care plan over six months. Incremental costs from the public-payer and societal perspective, quality-adjusted life-years (QALY), and the incremental cost-effectiveness ratio (ICER) six months after baseline were calculated using multivariate regression models. We assessed the probability of cost effectiveness using a range of willingness-to-pay thresholds (WTP).

Results: Caregivers in the intervention gained QALYs (+0.004 [CI^95%-0.003-0.012], p-value=.225) and had lower costs from the public-payer (-378€ [1,926-1,168], p-value=.630), but higher costs from the societal perspective (+1,324 [-3,634-6,284], p-value=.599). The intervention dominates usual care from the payer perspective, while the ICER was 331,000€/QALY from a societal perspective. The probability of cost-effectiveness was 72% and 79% from the public payer and 33% and 35% from a societal perspective at the WTP threshold of 40,000€ and 80,000€/QALY gained.

Discussion: CMS was likely cost-effective from the payer but not from a societal perspective, underlining the importance of informal care. The gain in QALY was marginal and could be due to the short observation period. Focusing on both the caregiver and the PwD, rather than assessing the PwD needs through the caregiver, could improve the cost-effectiveness results.

Objective: There is a progressive decline in the relative effectiveness of updated COVID-19 vaccines. Surveillance reports in Australia have also shown a wide variation in the disease severity and mortality across age groups. This study aimed to perform a cost-utility analysis of the monovalent XBB.1.5 vaccine relative to no updated booster vaccine between September 2023 to August 2024.

Methods: A cost-utility analysis was performed using a Markov model from the healthcare system perspective for three different age groups (18 - 64; 65 - 74; and ≥ 75 years). Costs and outcomes with the monovalent XBB.1.5 vaccine were compared to no updated booster dose for one year. Health outcomes were expressed as Quality-adjusted life-years (QALY), while costs were presented in 2023 Australian dollars. Aggregate distributional cost-effectiveness analysis and sensitivity analyses were performed. The willingness-to-pay threshold was set at AU$50,000/QALY.

Results: The updated vaccine was dominant for the 18 - 64 years group, and cost-effective for the 65 - 74 years group (incremental cost-effectiveness ratio [ICER] = AU$10,786/QALY), and ≥ 75 years group (ICER = AU$36,531/QALY) relative to no updated booster vaccine. The major determinants of the ICER was the vaccine uptake rate. There was inequality in health benefits between the older First Nations versus non-indigenous Australians. The results were robust to simultaneous changes in the parameters' values.

Conclusion: The monovalent XBB.1.5 vaccine is cost-effective and still relevant for all adult age groups in Australia. Based on current evidence, the study findings support the promotion of booster vaccination for Australian adults.

Objectives: To identify how machine learning (ML) approaches were implemented in mapping studies, and to what extent ML improved performance compared with regression models (RMs).

Methods: A systematic literature search was conducted in 12 databases from inception to Dec-2023 to identify studies that applied ML to develop mapping algorithms. A data template was applied to extract dataset information, source and target measures, ML approaches and RMs, mapping types (direct vs. indirect), goodness-of-fit indicators (mean absolute error [MAE], mean squared error [MSE], root mean squared error [RMSE], R-squared, and intraclass-correlation coefficient [ICC]), and validation methods. Differences in goodness-of-fit indicators between ML and RMs were summarized. Potential advantages and challenges for ML were further discussed.

Results: 13 mapping studies were identified, in which both ML and RM were adopted. Bayesian networks was the most frequently used ML approach (n=6), followed by the least absolute shrinkage and selection operator (n=4). Ordinary least square model was the most used RM (n=8), followed by censored least absolute deviation model and multinomial logit model (n=5 each). The average improvement of goodness-of-fit of ML compared with RMs by indicators were 0.007 (MAE), 0.004 (MSE), 0.058 (R-squared), 0.016 (ICC), and -0.0004 (RMSE).

Conclusion: There is an increasing number of studies using ML in developing mapping algorithms. Generally, a minor improvement of goodness-of-fit was observed compared with RMs when using mean-based comparisons. Issues such as how to interpret, apply and externally validate the ML-based outputs would affect their implementation. Future studies are warranted to verify advantages of ML approaches.

Objective: This study aimed to quantify the additional cardioprotective effects of sodium-glucose cotransporter 2 inhibitors (SGLT2is) and glucagon-like peptide-1 receptor agonists (GLP-1RAs) beyond the traditional risk factors control in individuals with type 2 diabetes (T2D). This helps calibrate the BRAVO diabetes simulation model to capture the total cardiovascular benefits of new diabetes medications accurately.

Methods: We extracted patient characteristics and treatment efficacy data from four cardiovascular outcomes trials (CVOTs) of SGLT2is and four CVOTs of GLP-1RAs completed before May 2023. Using the BRAVO diabetes simulation model, we translated reductions in traditional risk factors (i.e., glycated hemoglobin; systolic blood pressure; low-density lipids; body mass index) from the newer drugs into risk reductions in cardiovascular outcomes (i.e., myocardial infarction (MI), stroke, congestive heart failure (CHF), and mortality) assuming the drug-associated risk reductions are only driven by traditional risk factors. Then we compared the simulated risk-factor-driven risk reductions of cardiovascular outcomes with observed risk reductions from the trials and calculated drug-specific incremental benefits (DIB).

Results: After accounting for the cardiovascular effects from traditional risk factors control, SGLT2is was associated with an additional 19% risk reduction in CHF (DIB: 0.81, 95% CI: 0.72-0.90). Furthermore, the uncalibrated model predicted a risk reduction in stroke with SGLT2is, which was not observed in CVOTs. This discrepancy highlights the need for an SGLT2i-specific calibrator to align the simulation results with the observed outcomes. In contrast, no additional cardiovascular benefit was associated with GLP-1RAs after controlling for traditional risk factors.

Conclusion: Our study revealed that SGLT2is could further reduce CHF risks beyond the control of traditional risk factors but may offer additional pathways to offset overall benefits from traditional risk factor control in stroke risks. No additional cardiovascular benefits were observed for GLP-1RAs beyond traditional risk factor control. The BRAVO model calibration enhances cardiovascular outcome prediction with these newer antidiabetic therapies.

Objective: Economic evaluations of mental health problems typically only include short-term measures from an individual healthcare perspective. In perinatal mental health, which spans generations, this is likely to lead to an underestimation of interventions' potential benefits. We sought to understand the spectrum of outcomes of perinatal mental health problems that have economic consequences and how they are captured in economic evaluations.

Methods: We conducted a systematic search of the peer-reviewed literature to identify two types of evidence: (i) synthesised evidence (i.e. systematic reviews, meta-analyses) or recent cohort studies that measured the outcomes of perinatal mental health problems, (ii) economic evaluations. After presenting the evidence narratively, we derive an overview of different types of outcomes to include in economic evaluations.

Results: Evidence on the many, wide ranging adverse outcomes with short- and long-term economic consequences is rich, ranging from those that can be measured during the perinatal period (e.g., mother's employment), those that require a longer-term follow-up period (e.g., children's mental health service use) and those that can be used as predictors in modelling studies (e.g., birth weight). Only a small subset of economic consequences, and their predictors (e.g., child maltreatment, poor attachment), are currently measured in economic evaluations. We make some recommendations how more and new types of economic evaluations might start addressing the gap in knowledge.

Conclusions: To inform decisions about reducing the costs of perinatal mental health problems, economic evaluations that provide knowledge of interventions' abilities to reduce the short- and long-term economic consequences are urgently needed.

Objectives: This landscape analysis aimed to summarize the role of health equity in health technology assessment (HTA) process (topic nomination, topic prioritization, assessment, appraisal, and decision-making) in Asia.

Methods: A comprehensive literature review followed by in-depth interviews with key informants was conducted. Content analysis was performed to summarize the role of health equity in HTA in thirteen health systems in Asia, including Brunei Darussalam, Cambodia, China, Indonesia, Japan, Malaysia, Myanmar, Philippines, Singapore, South Korea, Taiwan, Thailand, and Vietnam.

Results: Health equity was reported to be considered in most health systems' HTA process, except for Cambodia and Myanmar, which do not have an established HTA process. Interviews revealed that health equity has been more frequently considered to address the unmet medical needs of specific diseases (e.g., high disease burden or severity, rare diseases, cancer, and diseases affecting children and elderly) in Brunei Darussalam, China, Japan, Malaysia, Singapore, South Korea, Taiwan, Thailand, and Vietnam or inequities in the socially disadvantaged groups (e.g., socioeconomic status and geographical location) in Indonesia and the Philippines. Equity-informative economic evaluation was still in the early stages, with only three health systems reporting their use.

Conclusions: Health equity is considered in the HTA process in most Asian health systems. However, quantitative evaluation of health equity impact is still in its infancy as few health systems have just begun to perform equity-informative economic evaluations.

Objectives: Limited knowledge exists regarding individuals' thought processes when valuing health states using the composite time trade-off (cTTO) tasks, particularly for health states considered as worse-than-dead (WTD). This study aims to explore the reasons for WTD perception, the strategies respondents employ to value WTD states, and the challenges they face during cTTO tasks.

Methods: This qualitative investigation was embedded within a methodological EQ-5D-5L valuation study involving the general public in Singapore, where respondents completed seven cTTO and seven novel TTO tasks. Semi-structured interviews were conducted with respondents who assigned WTD values to one or more health state in the cTTO tasks. Interviews were recorded, transcribed verbatim, and analysed using framework analysis.

Results: Nineteen respondents were interviewed (age range: 21-71 years old). Respondents identified a range of interconnected factors leading to WTD valuation, including health state and its duration, personal beliefs and circumstances, and consequences of poor health. While respondents usually considered these factors thoroughly and were capable of complex trading-off between life years and health, some respondents resorted to heuristic strategies to simplify the valuation process, including attribute non-attendance, affect heuristic, and elimination-by-aspects strategies. Respondents encountered various difficulties when they complete the cTTO tasks, including identifying the indifference point, transitioning from conventional TTO to lead-time TTO, and interpreting and imagining health states.

Conclusion: This study provides insights on how individuals value WTD health states in cTTO tasks. It suggests that cTTO needs improvement, and provides insights into how to improve the design of valuation tasks for severe health states.

Objective: Management of breech presentation is a subject of ongoing clinical debate. The cost-effectiveness of improving safe vaginal breech birth is unknown. This study examines potential cost-effectiveness of 'OptiBreech collaborative care' and assesses value of undertaking further research.

Methods: A decision tree was used to evaluate the potential cost-effectiveness of 'OptiBreech care' versus standard care for a hypothetical cohort of women with confirmed singleton breech pregnancy after 36+0 weeks gestation and babies born thereafter for one year. Probabilities, costs, and outcomes were obtained from literature and 'OptiBreech' pilot trial. Uncertainty and value of information were analysed to prioritise future research. The main outcomes were incremental cost-utility (ICUR) and cost-effectiveness ratios (ICER), net benefits, cost-effectiveness acceptability curve, and expected value of perfect and perfect partial information.

Results: Using pre-existing evidence, 'OptiBreech care' is less effective but sufficiently less costly i.e. cost-effective compared with standard care. Influential parameters include cephalic birth rate after external cephalic version, training costs and vaginal birth rate after opting for vaginal birth. Emerging evidence for 'OptiBreech care' significantly improved the cost-effectiveness ratio. The expected population value of perfect information (EVPI) was £31.5 million, with utilities identified as key research priority.

Conclusions: Planned vaginal birth for singleton breech pregnancy may be cost-effective despite a potential loss in health outcomes. Emerging data on the effectiveness of 'OptiBreech care' significantly increased the cost-effectiveness likelihood. Conclusions did not change for litigation cost assumptions or for sole neonatal perspective. Further research on health utilities would provide a valuable reduction in decision uncertainty.