Applied Health Economics and Health Policy最新文献

Background

Willingness-to-pay (WTP) studies offer a demand-side perspective on the monetary value of health gains, typically expressed as WTP per quality-adjusted life year (WTP/Q). These estimates can complement supply-side cost-effectiveness thresholds (CETs) and inform whether healthcare budgets align with public preferences. However, existing thresholds often overlook heterogeneity by condition or population characteristics.

Objective

This study sought to systematically review literature on WTP/Q estimates derived from stated preference methods by (i) mapping the broader landscape of demand-side valuations and (ii) synthesizing how WTP/Q varies by both respondent and scenario-assigned age and disease severity in general-health contexts.

Methods

A systematic review was conducted across Medline, Embase, and EconLit, following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Eligible studies estimated the monetary value of a quality-adjusted life year (QALY) from a societal, demand-side perspective. We first conducted a broad narrative mapping of all included studies to describe methodological characteristics and contextual diversity. We then focused on a subset of studies using general-health scenarios to explore how willingness to pay per QALY varied by age and severity. A structured four-axis framework was applied to distinguish between respondent characteristics and scenario-assigned attributes. Data were classified and tabulated. A formal quality assessment was conducted across the full set of studies using the National Institutes of Health (NIH) tool.

Results

A total of 67 studies met inclusion criteria. Most originated from high-income countries and used contingent valuation or discrete choice experiments. Among general-health studies, WTP/Q decreased with respondents’ actual age in 49% of studies and increased with respondents’ own health severity in 79%. In studies assessing scenario-assigned attributes, WTP/Q increased with hypothetical disease severity in 91% of studies and decreased with scenario-assigned age in 83%. Substantial heterogeneity was observed in elicitation methods, framing, and utility measurement.

Conclusions

The findings support the need for condition- and population-specific WTP thresholds, as valuations appear to vary depending on the nature of the health gain and the characteristics of the beneficiary—whether real or hypothetical.

Objective

Insomnia and hypersomnia are sleep conditions associated with significant costs to the healthcare system and society. This study aimed to review the cost-effectiveness evidence of interventions for insomnia and hypersomnia, including psychotherapy, pharmacotherapy, and complementary and alternative medicine (CAM), across the age spectrum.

Methods

A systematic search (from inception to 18th February 2025) was conducted in electronic databases (Medline, PsycINFO, CINAHL, Econlit and Embase) and Health Technology Assessment websites. Full economic evaluations and return-on-investment analyses were included if they focused on treatments targeting insomnia or hypersomnia in people aged ≥12 years. The Drummond checklist was used to evaluate the quality of eligible studies. Narrative synthesis was applied to extract study characteristics and economic evaluation outcomes.

Results

Twenty-eight studies met the pre-defined criteria, including 26 for adults and older adults with insomnia, two for adolescents with insomnia, and no studies were found for hypersomnia treatment. Cognitive behavioural therapy for insomnia (CBT-I) and pharmacotherapy were likely to be cost-effective interventions for insomnia compared to inactive controls. Digital CBT-I was found to generate healthcare and societal cost savings when compared to face-to-face CBT-I or pharmacotherapy. The cost-effectiveness of CAM interventions is under-researched and remains unclear.

Conclusion

Among insomnia interventions, CBT-I has the strongest cost-effectiveness credentials. Future studies should focus on hypersomnia, adolescent insomnia, and comorbid insomnia and related conditions.

PROSPERO Registration Number

CRD42022343067.

Objective

This study aims to evaluate the technical quality of health technology assessment (HTA) studies conducted in India. Second, we aim to identify process-related challenges across the life cycle of an HTA from commissioning to policy translation.

Methods

A mixed-methods approach was employed to assess HTA studies conducted between 2018 and 2023 conducted by ten regional resource centers. The quantitative assessment involved reviewing 26 HTA reports using the Indian HTA Quality Appraisal Checklist. The qualitative component included semi-structured interviews with staff from six regional resource centers and the Health Technology Assessment in India secretariat to explore the processes of topic selection, study conduct, stakeholder engagement, and evidence to policy translation. Quantitative data were analyzed through scoring and categorization into quality ratings, while qualitative data were analyzed thematically using the framework method.

Results

In the quantitative assessment, 14% (n = 3) of studies were found to be of excellent quality, 50% (n = 11) were deemed to be of good quality, 32% (n = 7) were of average quality, and only one (4%) of poor quality. The qualitative findings highlighted limited adherence to guidelines, challenges in framing the topic, and gaps in technical expertise for advanced analyses. Additionally, a high staff turnover, the need for better stakeholder consultations, and strategies to disseminate the evidence were also highlighted.

Conclusions

These findings emphasize the need for improvements in adherence to guidelines, transparency in topic selection, and alignment of HTA findings with policy needs. Investments in training, advanced methodology guidance, and systematic communication between researchers and policy makers are crucial to enhancing HTA’s impact in India.

Objective

The aim of this review is to identify and assess modelling approaches in published model-based economic evaluations of treatments for individuals with inherited bleeding disorders.

Methods

A literature search was performed on seven electronic databases, from database inception until 30 May, 2024. Inclusion criteria were cost-effectiveness or cost-utility analyses using decision-analytic models. The approaches from included models were identified and assessed, and these approaches were compared across bleeding disorders and treatments.

Results

This review included a total of 47 decision-analytic models. The identified models primarily evaluated treatments for severe haemophilia A and B. For haemophilia without inhibitors, factor concentrates were the most evaluated intervention (n = 21, 68%), followed by gene therapies (n = 6, 19%) and emicizumab (n = 4, 13%). For haemophilia with inhibitors, assessed interventions included emicizumab (n = 8, 50%), immune tolerance induction with factor concentrates (n = 5, 31%) and bypassing agents (n = 3, 19%). Markov models were often used as a model type (n = 27, 57%), followed by decision trees (n = 9, 19%), Markov decision trees and decision process (n = 5, 11%) and individual-level models (n = 5, 11%). Regardless of the model type, most authors used a lifetime horizon, a 1-year cycle length, and bleeding events—particularly joint bleeds—as key health states of the models.

Conclusions

As the reviewed decision-analytic models mainly assessed treatments for severe haemophilia, the identified common approaches may only be generalisable to evaluating these treatments. Further research is required to evaluate their relevance for evaluating treatments of milder forms of haemophilia or other inherited bleeding disorders.

Systematic Review Protocol Registration

PROSPERO registration number CRD42023416560.

Background

Antibiotic resistance (ABR) is a growing global health threat; reliable evidence on its impact is crucial for prioritising public health interventions.

Objective

This study provides an updated, systematic review and meta-analysis to determine the true effect size of resistant infections on economic and clinical outcomes. It also evaluates methodologies used in ABR economic literature, offering recommendations for improving future research.

Methods

Following PRISMA guidelines, 11,252 articles published between 2000 and 2022 were reviewed from several databases. Studies were included if they reported the economic costs of ABR in humans and compared resistant with susceptible infections. Meta-analyses were conducted using random intercept models; standardised mean difference (SMD) was used for length of stay, and odds ratio (OR) for mortality. The Mantel-Haenszel method was applied to obtain pooled estimates.

Results

Results showed that 73% of the studies were conducted in high-income economies, the majority were performed at tertiary care settings (71%) and 67% employed only a hospital perspective. The available evidence indicated that the attributable cost of resistant infections ranged from EUR₂₀₂₂ − 21,629 to EUR₂₀₂₂ 74,452 per patient episode (with Pseudomonas spp. causing the highest costs). The majority of studies (93%) found that patients with ABR incurred higher costs than their susceptible counterparts (72% report statistically significantly higher costs). Results from meta-analysis indicated that, on average, the excess in hospital stay attributable to resistant infections was 8.72 days (95% confidence interval (CI) [6.42; 11.02], SMD = 0.91) and the odds of premature death were significantly higher in the resistance group, with a risk increase of 65% (OR 95% CI [1.44; 1.88]).

Conclusion

The findings of this study take the first steps in providing reliable evidence; they could be valuable to researchers, policymakers and clinicians involved in ABR control and health promotion across countries. Similarly, the reported estimates may prove useful for future modelling studies aimed at assessing the long-term economic impact of ABR.

Objectives

The aim of this study was to estimate the health system cost and carbon emissions of diagnostic imaging tests undertaken by patients on different surveillance schedules for follow-up of stage III melanoma. We also aimed to demonstrate how different monetary valuations of carbon emissions affect overall cost.

Methods

We conducted a retrospective analysis of administrative data from the Melanoma Institute Australia’s Melanoma Research Database for patients diagnosed with stage III melanoma between 2000 and 2014 and followed them until 2023. Imaging tests (computed tomography [CT], positron emission tomography [PET], PET–CT, ultrasound, X-ray, and magnetic resonance imaging [MRI]) undertaken during follow-up were described. Healthcare costs were estimated per patient-year using data from the Medicare Benefits Schedule. Carbon emissions from tests and transport were estimated per patient-year using life cycle assessment and valued using New South Wales carbon values.

Results

Overall, 553 patients were included in this study: 115 in the 3–6-monthly surveillance imaging group, 273 in the 12-monthly surveillance imaging group, and 165 in the no routine imaging surveillance group. Healthcare costs and carbon emissions were highest in the 3–6-monthly group (Australian dollar [AUD] $1098 and 226 kg carbon dioxide equivalent emissions [CO₂-e] per patient-year) followed by the 12-monthly imaging group (AUD $767 and 150 kg CO₂-e per patient-year), and the no routine imaging group (AUD $319 and 50 kg CO₂-e per patient-year). When carbon emissions were valued in Australian dollars they accounted for 1.8–2.6% of total costs.

Conclusions

More frequent surveillance imaging of patients with stage III melanoma is associated with higher healthcare costs and environmental impacts, the latter of which are responsible for a small proportion of total costs when valued in dollars.

Background

Obesity represents a significant public health and economic problem worldwide. In Greece, where the prevalence of adult obesity is among the highest in Europe, no prior study has examined its economic impact among adults. This study estimates the total economic burden of obesity in Greece for 2024, adopting a societal perspective and considering both direct and indirect costs.

Methods

A prevalence-based, top-down cost-of-illness approach was used. Direct costs included obesity pharmacotherapy, bariatric surgery, weight-loss supplements, and 33 obesity-related comorbidities. Indirect costs encompassed productivity losses due to absenteeism, presenteeism, and premature mortality. Epidemiological, demographic, clinical, and cost data were retrieved from national and international sources. Population-attributable fractions were used to link obesity with comorbidity costs. All costs were adjusted to 2024 euros. Deterministic and probabilistic sensitivity analyses assessed the robustness of the base-case results.

Results

The total economic burden of obesity in Greece for 2024 was estimated at €4.92 billion, equivalent to 2.07% of GDP. Direct medical costs accounted for €2.593 billion (52.71%), with obesity-related comorbidities representing 99.6% of this amount. Indirect costs totalled €2.327 billion, driven primarily by premature mortality (€1.30 billion). Absenteeism and presenteeism costs were €469 million and €555 million, respectively. Sensitivity analyses confirmed robustness, with total costs averaging €4.89 billion (95% CI: €3.21–€7.29 billion).

Conclusion

Obesity imposes a considerable economic burden on Greece, threatening the sustainability of the healthcare system and broader social well-being. Immediate and drastic coordinated, multisectoral, and multidisciplinary strategies are needed to curb the prevalence and fiscal impact of obesity.

Background

Pricing and reimbursement (P&R) systems do not normally use public investments in research and development (R&D) as criteria when negotiating the prices and reimbursement of health technologies.

Objective

The objective was to find mechanisms that consider public investment in R&D when negotiating P&R or obtaining a fair return on this public investment

Methods

We conducted a scoping review. A total of four databases (PubMed, Embase, Scopus and Web of Science) and a grey literature information source (Google Scholar) were searched. Eligible articles were published before 2024 and described how public sector investment in R&D is considered in price negotiations or how the public sector can obtain a return on R&D investment.

Results

The review found 28 papers referring to mechanisms that take into account public investment in R&D to reduce prices in the P&R negotiation (e.g. delinkage R&D model, advance purchase agreement and government patent use), to obtain a fair return on investment (e.g. royalties and venture philanthropy) or to save costs or share risks (e.g. social impact bonds and prize fund). Examples are provided of health technologies that used these mechanisms.

Conclusions

Policymakers have several resources they can draw from to ensure a fair and efficient use of public R&D funds. However, there is little evidence that these instruments are widely used in practice, and there is no political consensus on what mechanism is the most appropriate and why. In view of the above, it is essential to create a common framework that will ensure a fairer and more affordable system for public health budgets.

Background

In the absence of long-term data, structured expert elicitation gathers expert judgments and associated uncertainties to assess the clinical plausibility of long-term extrapolations.

Objective

The objective of this study was to obtain expert estimates of expected long-term outcomes for advanced renal cell carcinoma treatments to inform cost-effectiveness analysis for National Institute for Health and Care Excellence (NICE)’s pathways pilot.

Methods

Using materials from the structured expert elicitation resources (STEER) repository, aligned with the Medical Research Council (MRC) protocol, the exercise was planned and conducted. Aiming for 5–10 oncologists from diverse UK geographies and settings, experts estimated progression-free survival (PFS) at three landmark timepoints for 21 disease-risk-prior treatment combinations and overall survival for best supportive care. Within an 8-week timeframe, we piloted with one clinician, conducted online training, collected responses via an online survey using the roulette method and mathematically aggregated results through linear opinion pooling.

Results

Nine experts participated (question response rate: 95%). For first-line intermediate/poor-risk patients, clinicians projected similar PFS for three immune oncology/tyrosine kinase inhibitor (TKI) combinations from 5 years onward and comparable PFS for two TKI monotherapies. Nivolumab + ipilimumab was anticipated to achieve the highest PFS amongst first-line therapies. Expert reasoning incorporated treatment class, clinical experience, and awareness of trial data optimism. Expert estimates were generally somewhat optimistic compared with observed UK real-world evidence and pessimistic compared with observed trial data.

Conclusions

Structured expert elicitation is a pragmatic, efficient approach for informing long-term survival extrapolations in the context of a rapidly evolving treatment pathway. We demonstrated that expert elicitation is possible even for complex decision problems in a relatively short timeframe.