Applied Health Economics and Health Policy最新文献

Background

This study aimed to assess the cost-effectiveness of non-vitamin K antagonist oral anticoagulants (NOACs) in comparison with warfarin using data from real practice based on the perspective of the health care system in Thailand.

Methods

A four-state Markov model encompassing well-controlled atrial fibrillation (AF), stroke and systemic embolism, major bleeding and death was utilised to forecast clinical and economic outcomes. Transitional probabilities, direct medical costs and utilities were derived from the real-world data of the ‘COOL-AF Thailand’ registry, Thailand’s largest nationwide registry spanning 27 hospitals. The cohort comprised AF patients. The primary outcomes assessed were total costs, life years, quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio. All costs and outcomes were subject to an annual discount rate of 3.0%. A spectrum of sensitivity analyses was conducted.

Results

The mean age of the cohort was 68.8 ± 10.7 years. The NOACs group incurred a marginally lower total lifetime cost than the warfarin group (247,857 Thai baht [THB] vs 253,654 THB or 7137 USD vs 7304 USD) and experienced gains of 0.045 life years and 0.043 QALYs over the warfarin group. Given the lower cost and higher benefits associated with NOACs, this implies that NOAC treatment is a dominant strategy compared to warfarin for AF patients. At a ceiling ratio of 160,000 THB (4607 USD) per QALY, NOACs presented a 61.2% probability of being cost effective.

Conclusions

Non-vitamin K antagonist oral anticoagulants represent a cost-saving alternative to warfarin in the real clinical practice. However, with a probability of being cost effective below 65%, it suggests some parameter uncertainty regarding their overall cost effectiveness compared to warfarin.

Background

Fostering market entry of novel antibiotics and enhanced use of diagnostics to improve the quality of antibiotic prescribing are avenues to tackle antimicrobial resistance (AMR), which is a major public health threat. Pricing, procurement and reimbursement policies may work as AMR ‘pull incentives’ to support these objectives. This paper studies pull incentives in pricing, procurement and reimbursement policies (e.g., additions to, modifications of, and exemptions from standard policies) for novel antibiotics, diagnostics and health products with a similar profile in 10 study countries. It also explores whether incentives for non-AMR health products could be transferred to AMR health products.

Methods

This research included a review of policies in 10 G20 countries based on literature and unpublished documents, and the production of country fact sheets that were validated by country experts. Initial research was conducted in 2020 and updated in 2023.

Results

Identified pull incentives in pricing policies include free pricing, higher prices at launch and price increases over time, managed-entry agreements, and waiving or reducing mandatory discounts. Incentives in procurement comprise value-based procurement, pooled procurement and models that delink prices from volumes (subscription-based schemes), whereas incentives in reimbursement include lower evidence requirements for inclusion in the reimbursement scheme, accelerated reimbursement processes, separate budgets that offer add-on funding, and adapted prescribing conditions.

Conclusions

While a few pull incentives have been piloted or implemented for antibiotics in recent years, these mechanisms have been mainly used to incentivize launch of certain non-AMR health products, such as orphan medicines. Given similarities in their product characteristics, transferability of some of these pull incentives appears to be possible; however, it would be essential to conduct impact assessments of these incentives. Trade-offs between incentives to foster market entry and thus potentially improve access and the financial sustainability for payers need to be addressed.

Graphical Abstract

Background

Inappropriate antibiotic use increases selective pressure, contributing to antimicrobial resistance. Point-of-care rapid diagnostic tests (RDTs) would be instrumental to better target antibiotic prescriptions, but widespread implementation of diagnostics for improved management of febrile illnesses is limited.

Objective

Our study aims to contribute to evidence-based guidance to inform policymakers on investment decisions regarding interventions that foster more appropriate antibiotic prescriptions, as well as to address the evidence gap on the potential clinical and economic impact of RDTs on antibiotic prescription.

Methods

A country-based cost-effectiveness model was developed for Burkina Faso, Ghana and Uganda. The decision tree model simulated seven test strategies for patients with febrile illness to assess the effect of different RDT combinations on antibiotic prescription rate (APR), costs and clinical outcomes. The incremental cost-effectiveness ratio (ICER) was expressed as the incremental cost per percentage point (ppt) reduction in APR.

Results

For Burkina Faso and Uganda, testing all patients with a malaria RDT was dominant compared to standard-of-care (SoC) (which included malaria testing). Expanding the test panel with a C-reactive protein (CRP) test resulted in an ICER of $ 0.03 and $ 0.08 per ppt reduction in APR for Burkina Faso and Uganda, respectively. For Ghana, the pairwise comparison with SoC—including malaria and complete blood count testing—indicates that both testing with malaria RDT only and malaria RDT + CRP are dominant.

Conclusion

The use of RDTs for patients with febrile illness could effectively reduce APR at minimal additional costs, provided diagnostic algorithms are adhered to. Complementing SoC with CRP testing may increase clinicians’ confidence in prescribing decisions and is a favourable strategy.

The characteristics and relative strengths and weaknesses of partitioned survival models (PSMs) and state transition models (STMs) for three state oncology cost-effectiveness models have previously been studied. Despite clear and longstanding economic modeling guidelines, more than one structure is rarely presented, and the choice of structure appears correlated more with audience or precedent than disease, decision problem, or available data. One reason may be a lack of guidance and tools available to readily compare measures of internal validity such as the model fit and efficiency of different structures, or sensitivity of results to those choices. To address this gap, methods are presented to evaluate the fit and efficiency of three structures, with an accompanying R software package, psm3mkv. The methods are illustrated by analyzing interim and final analysis datasets of the KEYNOTE-826 randomized controlled trial. At both interim and final analyses, the STM Clock Reset structure provided the best and most efficient fit. Structural uncertainties had been reduced from interim to final analysis. Beyond measures of internal validity, guidelines highlight the importance of reflecting all available data, avoiding model selection purely on the basis of goodness of fit and strongly considering external validity. The method and software allow modelers to more easily evaluate and report model fit and efficiency, examine implicit assumptions, and reveal sensitivities to structural choices.

Objective

This study evaluated, in a Swedish setting, the cost effectiveness of fenfluramine (FFA) as an add-on to standard of care (SoC) for reducing seizure frequency in Dravet syndrome, a severe developmental epileptic encephalopathy.

Methods

Cost effectiveness of FFA+SoC compared with SoC only was evaluated using a patient-level simulation model with a lifetime horizon. Patient characteristics and treatment effects, including convulsive seizures, seizure-free days and mortality, were derived from FFA clinical trials. Resource use and costs included cost of drug acquisition, routine care and monitoring, as well as ongoing and emergency resources. Quality of life (QoL) estimates for patients and their caregivers were derived from clinical trial data. Robustness was evaluated by one-way sensitivity analysis, probabilistic sensitivity analysis and scenario analyses.

Results

Lifetime cost of FFA+SoC was ~3 million SEK per patient compared with ~1.5 million SEK for SoC only. FFA+SoC generated 15% more QALYs than SoC only (21.2 vs 18.5 over a lifetime), resulting in an incremental cost-effectiveness ratio (ICER) of ~540,000 SEK. Moreover, FFA+SoC had a higher probability of being cost effective than SoC only from a willingness-to-pay threshold of 710,000 SEK. Results remained generally consistent across scenario analyses, with only few exceptions (exclusions of carer utility or FFA effect on sudden unexpected death in epilepsy).

Conclusion

Due to better seizure control, FFA is a clinically meaningful add-on therapy and was estimated to be a cost-effective addition to current SoC for patients with this rare disease in Sweden at a willingness-to-pay threshold of 1,000,000 SEK.

Background

Decision-makers require knowledge of the strengths and weaknesses of decision-analytic models used to evaluate healthcare interventions to be able to confidently use the results of such models to inform policy. A number of aspects of model validity have previously been described, but no systematic approach to assessing the validity of a model has been proposed. This study aimed to consolidate the different aspects of model validity into a step-by-step approach to assessing the strengths and weaknesses of a decision-analytic model.

Methods

A pre-defined set of steps were used to conduct the validation process of an exemplar early decision-analytic-model-based cost-effectiveness analysis of a risk-stratified national breast cancer screening programme [UK healthcare perspective; lifetime horizon; costs (£; 2021)]. Internal validation was assessed in terms of descriptive validity, technical validity and face validity. External validation was assessed in terms of operational validation, convergent validity (or corroboration) and predictive validity.

Results

The results outline the findings of each step of internal and external validation of the early decision-analytic-model and present the validated model (called ‘MANC-RISK-SCREEN’). The positive aspects in terms of meeting internal validation requirements are shown together with the remaining limitations of MANC-RISK-SCREEN.

Conclusion

Following a transparent and structured validation process, MANC-RISK-SCREEN has been shown to have satisfactory internal and external validity for use in informing resource allocation decision-making. We suggest that MANC-RISK-SCREEN can be used to assess the cost-effectiveness of exemplars of risk-stratified national breast cancer screening programmes (NBSP) from the UK perspective.

Implications

A step-by-step process for conducting the validation of a decision-analytic model was developed for future use by health economists. Using this approach may help researchers to fully demonstrate the strengths and limitations of their model to decision-makers.

Introduction

The escalating burden of catastrophic health expenditure (CHE) poses a significant threat to individuals and households in India, where out-of-pocket expenditure (OOP) constitutes a substantial portion of healthcare financing. With rising OOP in India, a proper measurement to track and monitor CHE due to health expenditure is of utmost important. This study focuses on synthesizing findings, understanding measurement variations, and estimating the pooled incidence of CHE by health services, reported diseases, and survey types.

Method

Following the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines, a thorough search strategy was employed across multiple databases, between 2010 and 2023. Inclusion criteria encompassed observational or interventional studies reporting CHE incidence, while exclusion criteria screened out studies with unclear definitions, pharmacy revenue-based spending, or non-representative health facility surveys. A meta-analysis, utilizing a random-effects model, assessed the pooled CHE incidence. Sensitivity analysis and subgroup analyses were conducted to explore heterogeneity.

Results

Out of 501 initially relevant articles, 36 studies met inclusion criteria. The review identified significant variations in CHE measurements, with incidence ranging from 5.1% to 69.9%. Meta-analysis indicated the estimated incidence of CHE at a 10% threshold is 0.30 [0.25–0.35], indicating a significant prevalence of financial hardship due to health expenses. The pooled incidence is estimated by considering different sub-groups. No statistical differences were found between inpatient and outpatient CHE. However, disease-specific estimates were significantly higher (52%) compared to combined diseases (21%). Notably, surveys focusing on health reported higher CHE (33%) than consumption surveys (14%).

Discussion

The study highlights the intricate challenges in measuring CHE, emphasizing variations in recall periods, components considered in out-of-pocket expenditure, and diverse methods for defining capacity to pay. Notably, the findings underscore the need for standardized definitions and measurements across studies. The lack of uniformity in reporting exacerbates the challenge of comparing and comprehensively understanding the financial burden on households.

Multiple, accelerating and interacting ecological crises are increasingly understood as constituting a major threat to human health and well-being. Unconstrained economic growth is strongly implicated in these growing crises, and it has been argued that this growth has now become “uneconomic growth”, which is a situation where the size of the economy is still expanding, but this expansion is causing more harm than benefit. This article summarises the multiple pathways by which uneconomic growth can be expected to harm human health. It describes how health care systems—especially through overuse, low value and poor quality care—can themselves drive uneconomic growth. Health economists need to understand not only the consequences of environmental impacts on health care, but also the significance of uneconomic growth, and pay closer attention to the growing body of work by heterodox economists, especially in the fields of ecological and feminist economics. This will involve paying closer heed to the existence and consequences of diminishing marginal returns to health care consumption at high levels; the central importance of inequalities and injustice in health; and the need to remedy health economists’ currently limited ability to deal effectively with low value care, overdiagnosis and overtreatment.