Applied Health Economics and Health Policy最新文献

Background and objective: Deep brain stimulation (DBS) is an established treatment for Parkinson's disease (PD) in patients with advanced motor symptoms with an inadequate response to pharmacotherapies. Despite its effectiveness, the cost effectiveness of DBS remains a subject of debate. This systematic review aims to update and synthesize evidence on the cost effectiveness of DBS for PD.

Methods: To identify full economic evaluations that compared the cost effectiveness of DBS with other best medical treatments, a comprehensive search was conducted of the PubMed, Embase, Scopus, and Tufts Cost-Effective Analysis registry databases. The selected papers were systematically reviewed, and the results were summarized. For the quality appraisal, we used the modified economic evaluations bias checklist. The review protocol was a priori registered with PROSPERO, CRD42022345508.

Results: Sixteen identified cost-utility analyses that reported 19 comparisons on the use of DBS for PD were systematically reviewed. The studies were primarily conducted in high-income countries and employed Markov models. The costs considered were direct costs: surgical expenses, calibration, pulse generator replacement, and annual drug expenses. The majority of studies used country-specific thresholds. Fourteen comparisons from 12 studies reported on the cost effectiveness of DBS compared to best medical treatments. Eleven comparisons reported DBS as cost effective based on incremental cost-utility ratio results.

Conclusions: The cost effectiveness of DBS for PD varies by time horizon, costs considered, threshold utilized, and stage of PD progression. Standardizing approaches and comparing DBS with other treatments are needed for future research on effective PD management.

Background and objective: Adding gene expression profiles (GEPs) to the current diagnostic work-up of aggressive large B-cell lymphomas may lead to the reclassification of patients, treatment changes and improved outcomes. A GEP test is in development using TempO-Seq^® technology to distinguish Burkitt lymphoma (BL) and primary mediastinal large B-cell lymphoma (PMBCL) from diffuse large B-cell lymphoma (DLBCL), and to classify patients with DLBLC and to predict the benefit of (e.g.) adding bortezomib to R-CHOP therapy (RB-CHOP). This study aims to estimate the potential impact of a GEP test on costs and health outcomes to inform pricing and evidence generation strategies.

Methods: Three decision models were developed comparing diagnostic strategies with and without GEP signatures over a lifetime horizon using a UK health and social care perspective. Inputs were taken from a recent clinical trial, literature and expert opinion. We estimated the maximum price of the test using a threshold of Great Britain Pound (GBP) 30,000 per quality-adjusted life-year (QALY). Sensitivity analyses were conducted.

Results: The estimated maximum threshold price for a combined test to be cost effective is GBP 15,352. At base-case values, the BL signature delivers QALY gains of 0.054 at an additional cost of GBP 275. This results in a net monetary benefit at a threshold of GBP 30,000 per QALY of GBP 1345. For PMBCL, the QALY gain was 0.0011 at a cost saving of GBP 406 and the net monetary benefit was GBP 437. The hazard ratio for the impact of treating BL less intensively must be at least 1.2 for a positive net monetary benefit. For identifying patients with the DLBCL subtype responsive to bortezomib, QALY gain was 0.2465 at a cost saving of GBP 6175, resulting in a net monetary benefit of GBP 13,570. In a probabilistic sensitivity analysis using 1000 simulations, a testing strategy was superior to a treat all with R-CHOP strategy in 81% of the simulations and with a cost saving in 92% assuming a cost price of zero.

Conclusions: Our estimates show that the combined test has a high probability of being cost effective. There is good quality evidence for the benefit of subtyping DLBCL but the evidence on the number of patients reclassified to or from BL and PMBCL and the impact of a more precise diagnosis and the cost of treatment is weak. The developers can use the price estimate to inform a return on investment calculations. Evidence will be required of how well the TempO-Seq^® technology performs compared to the testing GEP technology used for subtyping in the recent clinical trial. For BL and PMBCL elements of the test, evidence would be required of the number of patients reclassified and improved costing information would be useful. The diagnostic and therapeutic environment in haematological malignancies is fast moving, which increases the risk for develop

Background: Self-reported service use informs resource utilisation and cost estimates, though its validity for use within economic evaluations is uncertain.

Objective: The aim of this study is to assess agreement in health resource-use measurement between self-reported and administrative data across different resource categories, over time and between different recall periods by subgroups among Australians living with psychosis.

Methods: Data were obtained for 104 participants with psychotic disorders from a randomised controlled trial. Agreement between self-reported resource-use questionnaires and administrative data on community-based services and medication use was assessed through estimating differences of group mean number of visits and medications used and intraclass correlation coefficients (ICC) over multiple time periods.

Results: ICC showed moderate agreement across most time periods for general practitioners, psychiatrists and mental health medications. No clear trends were discernible over time, between varying lengths of recall periods nor across participant subgroups.

Conclusion: Despite poor agreement, when measuring visits to psychologists and other health professionals, small overall differences in group mean number of visits indicate that self-reported data may still be valid for use in economic evaluations in people living with psychosis.

Background: Providing informal care has a negative effect on the caregiver's health and well-being, but little is known about how individuals respond to receiving informal care. Care recipients may improve their health behaviours to minimise the onerousness of caregiving and the stress faced by their carer from seeing a loved one in ill-health.

Objective: We aimed to examine whether informal care recipients internalise the potential for carer spillovers through changes in health behaviours.

Methods: We used data from 3250 older adults with care needs who took part in the UK Household Longitudinal Study between 2017 and 2019. We examined the response to informal care receipt in terms of the probability of engaging in four health behaviours: healthy diet, physical activity, smoking and alcohol consumption. We estimated average treatment effects using regression adjustment with inverse probability treatment weights, comparing individuals that received informal care to those receiving either formal or no care.

Results: We found that informal care receipt increased the probability of refraining from negative health behaviours (smoking and alcohol consumption) but reduced the probability of engaging in positive health behaviours (eating fruits and/or vegetables and physical activity).

Conclusions: The asymmetric effects detected suggest that the underlying mechanisms are different, and care recipients may be engaging in risk and effort compensation between negative and positive health behaviours. Failure to account for the behavioural responses from informal care recipients may lead to under-estimation or over-estimation of the extent of caregiving burden and the effectiveness of interventions impacting informal carers.

Background and Objective

Reducing the affordability of alcoholic beverages by increasing alcohol excise taxation can lead to a reduction in alcohol consumption but the impact on government alcohol excise tax revenue is poorly understood. This study aimed to (a) describe cross-country tax revenue variations and (b) investigate how changes in taxation were related to changes in government tax revenue, using data from Estonia, Germany, Latvia, Lithuania and Poland.

Methods

For the population aged 15 years or older, we calculated the annual per capita alcohol excise tax revenue, total tax revenue, gross domestic product and alcohol consumption. In addition to descriptive analyses, joinpoint regressions were performed to identify whether changes in alcohol excise taxation were linked to changes in alcohol excise revenue since 1999.

Results

In 2022, the per capita alcohol excise tax revenue was lowest in Germany (€44.2) and highest in Estonia (€218.4). In all countries, the alcohol excise tax revenue was mostly determined by spirit sales (57–72% of total alcohol tax revenue). During 2010–20, inflation-adjusted per capita alcohol excise tax revenues have declined in Germany (− 22.9%), Poland (− 19.1%) and Estonia (− 4.2%) and increased in Latvia (+ 56.8%) and Lithuania (+ 49.3%). In periods of policy non-action, alcohol consumption and tax revenue showed similar trends, but tax level increases were accompanied by increased revenue and stagnant or decreased consumption.

Conclusions

Increasing alcohol taxation was not linked to decreased but increased government revenue. Policymakers can increase revenue and reduce alcohol consumption and harm by increasing alcohol taxes.

Abstract

Background: Clinical indications for ibrutinib reimbursement in Australia should consider the inclusion of patients with chronic lymphocytic leukemia (CLL) harboring prognostically unfavorable TP53/IGHV genomic aberrations. This study assessed the cost effectiveness of five first-line treatment strategies in CLL for young (aged ≤ 65 years), fit patients without significant comorbidities: (1) no testing (fludarabine, cyclophosphamide and rituximab [FCR] for all), (2) test for del(17p) only, (3) test for TP53 gene mutation status, (4) test for TP53 and IGHV gene mutation status and (5) no testing (ibrutinib for all).

Method: A decision analytic model (decision tree and partitioned survival model) was developed from the Australian healthcare system perspective with a lifetime horizon. Comparative treatment effects were estimated from indirect treatment comparisons and survival analysis using several studies. Costs, utility and adverse events were derived from public literature sources. Deterministic and probabilistic sensitivity analyses explored the impact of modeling uncertainties on outcomes.

Results: Strategy 1 was associated with 5.69 quality-adjusted life-years (QALYs) and cost 458,836 Australian dollars (AUD). All other strategies had greater effectiveness but were more expensive than Strategy 1. At the willingness-to-pay (WTP) threshold of 100,000 AUD per QALY gained, Strategy 1 was most cost effective with an estimated probability of 68.8%. Strategy 4 was cost effective between thresholds 155,000-432,300 AUD per QALY gained, and Strategy 5 >432,300 AUD per QALY gained.

Conclusion: Population targeting using mutation testing for TP53 and IGHV when performed with del(17p) testing specifically in the context of frontline ibrutinib choice does not make a cost-ineffective treatment into a cost-effective treatment.

Background: Atrial fibrillation (AF) is a severe epidemiological and public health concern among the elderly population worldwide, with substantial economic and social burdens. Economic evaluations can play an essential role in optimizing the utilization of scarce resources. In recent years, the number of economic evaluation studies related to AF has increased due to the rising number of AF patients, the continuous updating of clinical data, and the emergence of real-world evidence. However, there are still deficiencies in model settings and parameter sources in relevant studies.

Objective: This study aims to review the existing economic evaluations of novel oral anticoagulants (NOACs) in patients with AF and summarize the evidence and methods applied.

Methods: A comprehensive and systematic search was conducted on electronic databases, including PubMed, Embase, Web of Science (WOS), and The Cochrane Library, from the date of database creation to November 2022. The reporting quality of included literature was assessed using the Consolidated Health Economic Evaluation Reporting Standards 2022 (CHEERS 2022) statement.

Results: A total of 102 studies were included in the review, with 200 comparisons between NOACs and vitamin K antagonists (VKAs), as well as 58 comparisons between different NOACs. The healthcare sector and payer perspectives were the most common, and accordingly, the majority of the evaluations considered only direct medical costs. Most studies used Markov cohort models with the number of health states ranging from 4 to 29. Of included studies, 80 (78%) considered event recurrence and complications, and 78 (76%) considered discontinuation and second-line therapy. All of the studies applied uncertainty analysis to explore the robustness of the results. Of all 200 NOACs-VKAs comparisons, 149 (75%) showed that NOACs were more cost-effective; this proportion was 84% (139 out of 165) in high-income countries but decreased to 29% (10 out of 35) in middle- and low-income countries. Most (82%) of the 28 items in the CHEERS 2022 checklist were elucidated in the majority of included studies. A minority (only 39%) of included studies demonstrated high reporting quality.

Conclusion: NOACs may be more cost-effective than VKAs in patients with AF, but this conclusion applies to high-income countries, whereas VKAs may be more cost-effective in middle- and low-income countries. The reporting quality of included studies was variable, and certain methodological issues were presented. This study highlights the economic evaluation methodology of NOACs in patients with AF and provides recommendations for modeling methods and future studies.

There has been increasing interest in including carers' health-related qualify of life (HRQoL) in decision models, but currently there is no best practice guidance as to how to do so. Models thus far have typically assumed that carers' HRQoL can be predicted from patient health states, as we illustrate with three examples of disease-modifying treatments. However, this approach limits the mechanisms that influence carers' HRQoL solely to patient health and may not accurately reflect carers' outcomes. In this article, we identify and discuss challenges associated with modelling intervention effects on carers' HRQoL: attaching carer utilities to patient disease states, the size of the caring network, aggregation of carer and patient HRQoL, patient death, and modelling longer-term carer HRQoL. We review and critique potential alternatives to modelling carers' HRQoL in decision models: trial-based analyses, qualitative consideration, cost-consequence analysis, and multicriteria decision analysis, noting that each of these also has its own challenges. We provide a framework of issues to consider when modelling carers' HRQoL and suggest how these can be addressed in current practice and future research.

Introduction: Funding decisions for many health technologies occur without undergoing health technology assessment (HTA), in particular, without assessment of cost effectiveness (CE). Immunoglobulins in Australia are an interesting case study because they have been used for a long time for various rare disorders and their price is publicly available. Undertaking an HTA enables us to assess CE for an intervention for which there is limited clinical and economic evidence. This study presents a post-market review to assess the CE of immunoglobulins for the treatment of multifocal motor neuropathy (MMN) compared with best supportive care.

Methods: A Markov model was used to estimate costs and quality-adjusted life-years (QALYs). Input sources included randomised controlled trials, single-arm studies, the Australian clinical criteria for MMN, clinical guidelines, previous Medical Services Advisory Committee (MSAC) reports and inputs from clinical experts. Sensitivity analyses were conducted to assess the uncertainty and robustness of the CE results.

Results: The cost per patient of treating MMN with immunoglobulin was AU$275,853 versus AU$26,191when no treatment was provided, with accrued QALYs of 6.83 versus 6.04, respectively. The latter translated into a high incremental cost-effectiveness ratio (ICER) of AU$317,552/QALY. The ICER was most sensitive to the utility weights and the price of immunoglobulins. MSAC advised to continue funding of immunoglobulins on the grounds of efficacy, despite the high and uncertain ICER.

Conclusions: Beyond the ICER framework, other factors were acknowledged, including the high clinical need in a patient population for which there are no other active treatments available. This case study highlights the challenges of conducting HTA for already funded interventions, and the efficiency trade-offs required to fund effective high-cost therapies in rare conditions.