Health economic evaluations require health state utility values (HSUVs) to estimate quality-adjusted life years (QALYs). However, HSUVs derived from different preference-based instruments, valuation methods and value sets may not be directly comparable, which can affect interpretation of economic evaluations. Although reference cases, such as that of the National Institute for Health and Care Excellence (NICE), specify a preferred instrument, such as the EQ-5D, alternative measures are frequently used in practice. Using behavioural risk factors as an applied case, this study examined variation in HSUVs across instruments and synthesised utility estimates for body mass index (BMI) and smoking categories. A systematic review of population-based observational studies published between 2009 and 2024 was conducted across PubMed, Embase, Web of Science and EconLit. Random-effects meta-analyses were used to estimate pooled HSUVs by instrument type and risk factor category. Mixed-effects meta-regression models explored associations between behavioural risk factors, health-related quality of life (HRQoL) instrument type and variation in HSUVs while accounting for clustering of multiple estimates within studies. Preference-based instruments included the EQ-5D, Health Utilities Index (HUI), Short Form 6-Dimension (SF-6D) and Quality of Well-Being (QWB) scale. Given the anticipated heterogeneity across studies, pooled estimates were used to describe variation in reported HSUVs. Forty-four articles were included, contributing 186 utility estimates across five instrument categories. Substantial variation in HSUVs was observed across instruments with consistent patterns evident across BMI and smoking categories. Relative to the EQ-5D-3L, the HUI, SF-6D and QWB yielded systematically lower utility values. The EQ-5D-5L yielded higher values in the BMI model, but the corresponding estimate in the smoking model was not statistically significant. Variation associated with instrument categories, which may also reflect related valuation and derivation differences, was comparable, and in some cases greater than, differences observed across BMI and smoking categories, although substantial between-study heterogeneity remained (I2 > 90
Hospital adoption of medical devices requires structured governance mechanisms capable of integrating clinical, economic, organizational, and patient-centered dimensions. However, hospital-based health technology assessment processes remain heterogeneous and often lack standardized yet adaptable frameworks. We present the THE Mini-HTA (‘THE’ is the acronym for the Tuscany Health Ecosystem research project within the PNRR—Piano Nazionale di Ripresa e Resilienza (National Recovery and Resilience Plan)—as part of which the tool was developed), a hospital-oriented decision-support tool for post-market medical device evaluation. The framework combines a standardized set of ten dimensions and 39 criteria, derived from the EUnetHTA® Core Model and expanded to include new criteria, with a structured governance architecture that differentiates process coordination from domain-specific expert evaluation. The decision model is based on PROMETHEE, a non-compensatory multi-criteria decision analysis approach, aimed at preventing inappropriate compensation between critical criteria. An automated platform supports the full evaluation workflow, generating individual and collective rankings and enabling sensitivity analyses. The tool was implemented for assessing two implantable cardioverter defibrillators in an Italian tertiary care hospital highly specialized in cardiology and cardiac surgery. The real-world application provides initial empirical evidence of the tool’s operational feasibility in a real decision-making context, involving domain experts with different expertise, a clinically relevant comparative problem, which represent the conditions typically encountered in hospital-based post-market medical device evaluation. The outputs of the tool can support final deliberation and reporting by clarifying where the comparative advantages originated and the magnitude of residual differences between alternatives, while reducing perceptions of a “black-box” decision process. The THE Mini-HTA provides a scientifically grounded and operationally implementable framework to strengthen hospital-level health technology assessment. Future research should validate the tool across a broader range of medical devices and healthcare settings, and examine its integration into routine health technology assessment workflows.
Enzyme replacement therapy (ERT) for late-onset Pompe disease (LOPD) requires lifelong biweekly infusions, typically delivered in hospital settings. Home-based administration has been associated with reduced treatment burden and pressure on hospital infusion services. This study evaluated the cost-effectiveness and budget impact of home-based versus hospital-based ERT administration for adults with LOPD in Greece. A decision-analytic model was developed to conduct a cost-utility analysis (CUA) and a budget impact analysis (BIA) from the perspective of the Greek public healthcare system. The CUA adopted a 50-year horizon and estimated costs and quality-adjusted life-years (QALYs) associated with home- and hospital-based ERT administration. Clinical and cost inputs were derived from published literature, Greek sources, and expert consultation. Cost categories included drug cost, infusion administration, management of severe infusion-associated reactions (IARs), and one-off home-infusion nurse training. The BIA estimated the financial consequences of gradually introducing home-based ERT for the national cohort of treated patients over a 5-year horizon. In the base-case CUA, home-based ERT administration was associated with lower costs (€6,999,500 vs €7,150,725) and modestly higher QALYs (14.342 vs 14.235 QALYs) than hospital-based administration. Cost savings were driven primarily by lower administration costs, while QALY gains reflected avoidance of process-related disutility associated with hospital attendance. The BIA projected cumulative 5-year savings of €263,667 and 1174 fewer hospital infusion visits under the base-case adoption trajectory. Scenario analyses showed that the magnitude of cost savings was sensitive to the valuation of hospital administration, while removal of hospital process-related disutility reduced the incremental QALY gain to a negligible level; the direction of the cost findings remained unchanged across the scenarios evaluated. Home-based ERT administration for adults with LOPD in Greece was projected to reduce healthcare costs while generating modest health benefits, compared to hospital-based administration, potentially improving healthcare system efficiency.
Strengthening the urban primary healthcare system is essential for delivering need-based services to the rapidly growing low-income urban population in Bangladesh. This study examined the preferences of slum dwellers for primary healthcare providers under hypothetical minor and major illness conditions. A discrete choice experiment was conducted among 304 slum dwellers in Dhaka, Bangladesh, in January-February 2024. Six final attributes (time to reach healthcare centre, waiting time to see a provider, recommendation from neighbours/relatives, type of healthcare provider, consultation fee and medicine expenditure) were identified through a systematic review, focus group discussions and key informant interviews, and finalised through a community ranking exercise and expert consultation. A Bayesian D-efficient design was used to construct choice sets, each presenting two hypothetical primary healthcare providers and an opt-out option; respondents completed each choice set for minor and major illnesses. Data were analysed using mixed logit models (in preference and willingness-to-pay space), relative importance analysis and latent class analysis. Consultation fee had the highest relative importance for minor illness and type of healthcare provider for major illness. An MBBS/qualified doctor for minor illness and a senior/experienced doctor for major illness were strongly preferred over non-qualified providers. Utility decreased significantly with longer travel time (60 min) and waiting time (30 min), and higher consultation fees (p < 0.001). Compared with no recommendation, a negative recommendation significantly reduced utility for major illness, and a positive recommendation significantly increased utility for minor illness. Free medicines and a 50
Advanced therapy medicinal products, a European Union regulatory category encompassing gene-, cell-, and tissue-based medicines, challenge conventional reimbursement frameworks, particularly when a single administration entails high upfront costs and long-term effectiveness uncertainty. This study assessed the treatment-cost budget impact under three reimbursement models for etranacogene dezaparvovec (Hemgenix®) in hemophilia B within the high-income, decentralized, tax-financed healthcare system of Sweden. Using a decision-support framework, an outcome-based annuity model was identified as a relevant reimbursement strategy for Hemgenix®. A cohort Markov model simulated per-patient treatment costs for payers for Hemgenix® and the comparator factor IX prophylaxis over 5, 10, and 15 years for a hypothetical cohort of adults with hemophilia B. Three reimbursement strategies—full upfront, 30
