Value in Health最新文献

Objectives: The EQ Health and Wellbeing (EQ-HWB-9) is a preference-weighted instrument with an interim value set for assessing health and wellbeing in patients, social care users and carers. This study evaluated the experimental (2022) version's psychometric properties and compared it with established measures in a UK general population sample.

Methods: Data were drawn from a large cross-sectional survey of the UK general population (n = 11,383). Ceiling/floor effects were assessed at item (>50% extreme responses) and instrument (>15% at min/max score) levels. Convergent validity was assessed using Spearman correlations between EQ-HWB-9 items and conceptually similar items from the Short Warwick-Edinburgh Mental Wellbeing Scale (SWEMWBS), ICEpop CAPability measure for Adults (ICECAP-A), Health Utilities Index Mark 3 (HUI3), Office for National Statistics Four Personal Wellbeing Questions (ONS-4) and SIPHER-7. Pearson correlations were used for utility scores. Known-group validity was assessed using effect sizes to examine differences by mental wellbeing, disability, life satisfaction, caregiving status, self-reported health, and age. Agreement with comparator instruments was assessed using Bland-Altman plots and Lin's concordance correlation coefficient (CCC).

Results: Potential ceiling effects were noted for EQ-HWB-9 mobility and activity items, but not at the instrument level. Strong correlations (rs≥0.5, p<.001) with measures hypothesised a priori to assess related constructs (SWEMWBS, ICECAP-A, HUI3, ONS-4 and SIPHER-7) supported convergent validity. The EQ-HWB-9 effectively distinguished between relevant population subgroups (effect sizes≥0.8). Agreement with HUI3 utilities was higher (CCC >0.75) than other comparators.

Conclusions: The EQ-HWB-9 shows strong psychometric performance and is supported for use in UK health and wellbeing assessments.

Objectives: The EARLY TAVR trial demonstrated improved clinical outcomes for patients with asymptomatic severe aortic stenosis (aSAS) treated with transcatheter aortic valve replacement (eTAVR) compared with clinical surveillance (CS). The cost effectiveness of an eTAVR strategy for patients with aSAS in the United States (US) is unknown.

Methods: A Markov model with 30-day cycles was developed from the US healthcare payor perspective to estimate the cost-effectiveness of eTAVR vs. CS over a lifetime horizon. Inputs for population characteristics and health outcomes were derived from the EARLY TAVR trial. Costs were derived from US Medicare reimbursement rates. Probabilistic and deterministic sensitivity analyses were performed to evaluate the effect of parameter uncertainty on model output.

Results: When compared to CS, eTAVR was associated with 0.21 additional life years (LY) and 0.24 additional quality-adjusted life years (QALYs) over a lifetime due to more time spent in the alive and well health state with eTAVR. Lifetime costs were estimated to be $8,812 lower, due primarily to reductions in costs associated with the AVR procedure, stroke, and heart failure hospitalizations. Accordingly, eTAVR was projected to be economically dominant over CS. In probabilistic sensitivity analysis, a large majority of iterations (95.9%) produced cost-effective results ($100,000 threshold) for eTAVR versus CS, with most simulations (90.3%) showing dominance, confirming the robustness of the base case results. These findings were consistent over a variety of scenario analyses.

Conclusions: An eTAVR strategy for the treatment of aSAS may be a cost-saving approach for US healthcare payors, when compared to CS.

Distributional cost-effectiveness analyses (DCEAs) help decision-makers incorporate equity considerations in healthcare resource allocation. Public aversion to health inequalities is well documented, but underlying reasonings are rarely explored. Existing studies often elicit inequality aversion across socioeconomic groups, potentially conflating aversion to inequalities in health with inequalities in household finances or wellbeing. Additionally, online surveys systematically exclude people who do not use the internet, a disadvantaged group.

Objectives: To examine whether: i) reasoning for distributional preferences depend on the domain of inequality; ii) reasoning for distributional preferences are affected by cause of inequality; iii) participants provide and explain responses that violate 'monotonicity' (the welfare economics principle that, other things being equal, social welfare improves when at least one person is better-off); and iv) the above vary across the digital divide.

Methods: We employed mixed-methods to collect qualitative and quantitative data, via online discussion groups with a survey (11 groups, n=53), and telephone interviews (n=15) with digital minority individuals. Participants considered scenarios comparing equal and unequal health and wellbeing outcomes for an imaginary island. Wellbeing was framed as 'equivalent income' (described to participants as household spending money, with other life aspects being good).

Results: Distributional preferences varied by domain and cause of inequality but not digital status. Health inequality caused by financial inequality was widely unaccepted. Some preferred equal distributions, even when violating 'monotonicity', citing fairness and social cohesion.

Conclusions: Recruiting across the digital divide and using mixed-methods enriches inequality aversion research, enhancing the inclusivity and legitimacy of DCEA.

Objectives: Sacituzumab govitecan (SG) and sacituzumab tirumotecan (sac-TMT) are 2 approved antibody-drug conjugates for metastatic triple-negative breast cancer but lack direct comparison. Given their similar indications and high costs, an indirect cost-effectiveness analysis is essential to inform pricing and reimbursement decisions from the perspective of the Chinese healthcare system.

Methods: A partitioned survival model using Bayesian network meta-analysis and flexible parametric modeling estimated long-term survival, quality-adjusted life-years (QALYs), and costs. Participants were derived from the ASCENT and OptiTROP-Breast01 randomized controlled trials. Key outcomes included time-varying hazard ratio, life-years, costs, incremental cost-effectiveness ratio (ICER), incremental net health benefit, and incremental net monetary benefit. Multiple sensitivity and scenario analyses tested model robustness.

Results: In the base-case analysis, SG yielded 0.095 additional QALYs compared with sac-TMT at an incremental cost of $68 266, resulting in an ICER of $719 726 per QALY and an incremental net monetary benefit of -$64 400. Sensitivity analyses identified SG price and model time horizon as key drivers of ICER. In all plausible scenarios, SG remained not cost-effective versus sac-TMT. Probabilistic results showed a 0% probability of cost-effectiveness at a willingness-to-pay threshold of $40 763 per QALY. Scenario analysis confirmed the robustness of findings across assumptions on time horizon, utility, wastage, and discounting.

Conclusions: SG showed slightly greater health gains but much higher costs than sac-TMT, leading to poor cost-effectiveness at current prices. Given the high substitutability between antibody-drug conjugates in metastatic triple-negative breast cancer, indirect comparisons with economic modeling can help guide payer decisions and pricing negotiations when head-to-head data are lacking.

Objectives: This study presents a novel method for estimating meaningful score differences (MSDs) and meaningful score regions (MSRs) for the Observer-Reported Communication Ability (ORCA) Measure for individuals with Angelman syndrome (AS).

Methods: The AS Natural History Study collected longitudinal data on an individual's communication abilities on the ORCA Measure, Vineland Adaptive Behavior Scales-3, and the Bayley Scales of Infant and Toddler Development-4. In a separate Delphi-based study, caregivers provided MSDs and MSRs for the Vineland-3 and Bayley-4 scores. Leveraging the strong correlations between ORCA and these measures, mixed models with random intercepts were used to estimate ORCA MSDs and MSRs based on the previously derived Vineland-3/Bayley-4 MSDs and MSRs.

Results: The ORCA Measure was completed by 192 caregivers, with 113 also completing the Bayley-4 and 159 completing the Vineland-3. ORCA T-score MSRs for low, intermediate, and high communication ability were estimated for expressive, receptive, and pragmatic communication domains using the Vineland-3 and Bayley-4 previously derived MSDs and MSRs. ORCA MSDs varied depending on the MSR and ranged from 0.38-4.55 (based on Vineland-3) and 1.79-4.62 (based on Bayley-4).

Conclusions: We present a novel anchor-based methodological approach for deriving MSD and MSR thresholds for clinical outcome assessments. Although these MSD and MSR estimates for the ORCA measure are preliminary, our methods offer a strategy for establishing meaningful change thresholds in rare disease populations in which traditional longitudinal methods may be challenging to implement.