PharmacoEconomics最新文献

Background and objective: Idiopathic pulmonary fibrosis is a rare progressive respiratory disease associated with high morbidity, poor survival and substantial healthcare demands. The introduction of antifibrotic therapies in the mid-2010s has reshaped treatment patterns and may have shifted the balance of medical costs. This study aimed to systematically update and expand a 2018 systematic review of the economic burden of idiopathic pulmonary fibrosis, providing an opportunity to assess trends before and after the adoption of antifibrotic drugs.

Methods: We updated the 2018 literature review by applying the same systematic protocol and search strategy, extending coverage to August 2025. Searches were conducted in EMBASE, MEDLINE and the Cochrane Library. Eligible studies reported unit costs, total costs or healthcare resource utilisation in adult patients with idiopathic pulmonary fibrosis, regardless of treatment received. Relevant data were extracted across multiple healthcare resource utilisation and cost categories. Total medical costs from US studies were inflated to 2025 US dollars to examine temporal trends. Findings were synthesised narratively.

Results: A total of 85 studies met the inclusion criteria. Sixty-two studies reported healthcare resource utilisation and cost data, and 23 were economic evaluations. Most studies originated from Europe and North America, with two from China and one from South Korea; none was identified from low- or lower-middle-income countries. Annual per-patient costs varied widely, from approximately $1700 in South Korea to over $110,000 in recent US studies. Evidence suggests a shift in cost burden: earlier studies reported hospitalisations as the largest driver of expenditure, whereas more recent analyses identified drug acquisition as the dominant cost, accounting for over 70% of total spending in some settings. US studies that included antifibrotic costs reported total medical costs above $125,000, reaching up to $175,000. In contrast, studies excluding antifibrotic therapies reported total costs below $100,000. The annual probability of all-cause hospitalisation was reported around 23% when antifibrotic drugs were available and 26% in studies without antifibrotic drugs. Economic evaluations were heterogeneous in design, perspective and assumptions. Estimated long-term costs and incremental cost-effectiveness results varied considerably by country, limiting generalisability across healthcare systems.

Conclusions: This review confirms that idiopathic pulmonary fibrosis imposes substantial and rising healthcare costs. Antifibrotic use is associated with a shift in the distribution of costs, with higher drug expenditure and lower reported hospitalisation rates. The global picture remains heterogeneous, with major differences across countries reflecting system structure, pricing and treatment access.

Globally, advances in methods to examine equity alongside economic evaluations are being considered for application in health technology assessment (HTA) processes which inform decisions to publicly fund pharmaceuticals and medical services. In this practical application, we focus on the Australian context and explore how several of these methods might be used to incorporate equity more explicitly in HTA decisions. Specifically, we describe distributional cost-effectiveness analysis, extended cost-effectiveness analysis, equity weighting, multi-criteria decision analysis, mathematical programming and other quantitative approaches. We consider the feasibility and suitability of each method considering contextual factors, highlight challenges, describe current use in Australia and make recommendations for their application. We argue that there are opportunities to use aspects of several different methods in the Australian context which would illuminate how costs (including out of pocket costs) and benefits are distributed across the population, how normative concerns for equity influence the calculated social value of a new technology, how equity criteria complement other measures of value and how the technology impacts non-health outcomes. However, some of the stated requirements for the methods, such as using the same social groupings for all analyses, applying cost-effectiveness thresholds and the application of algorithms may restrict the usefulness of the evidence generated in a context where there are many priority populations of interest, several aspects of equity are considered important and there is a well-established deliberative approach to decision-making. We recommend that the equity implications of new health technologies should be universally evaluated. However, a tailored and transparent approach should be taken whereby specific equity analyses consider the context of the technology and the chosen methods are well justified.

Objectives: Bevacizumab was approved for first-line treatment of metastatic colorectal cancer (mCRC) in 2004. However, adding bevacizumab to treatment consistently fails to be cost-effective owing to modest response rates. Recently, the European Commission (EC) funded ANGIOPREDICT consortium ( www.angiopredict.com ) identified a link between bevacizumab treatment response and intermediate-to-high chromosomal instability (CIN) in mCRC. Thus, the objective of the current study was to compare the cost-effectiveness of adding bevacizumab with first-line chemotherapy in the bevacizumab responsive CIN subtype across three European countries (Germany, Ireland and Spain) with varying costs of care and reimbursement policies.

Methods: We developed an open-source health economic model to estimate cost-effectiveness. The ANGIOPREDICT cohort informed progression risks and cause-specific mortality. Health utilities and adverse events probabilities were obtained from the literature. Costs were derived from surveys of collaborating consortium hospitals in Germany, Ireland, and Spain that participated in the recently completed EC funded COLOSSUS translational study (ANGIOPREDICT successor initiative) and the literature. Sensitivity analyses included individual and simultaneous variation of input parameters from a priori defined distributions.

Results: Bevacizumab was not cost effective even at willingness-to-pay (WTP) thresholds that are appreciably higher than those considered realistic. The highest incremental cost-effectiveness ratio (ICER) was in Germany at €241,188 per quality-adjusted life year (QALY), while the lowest was in Ireland at €180,477 per QALY. All deterministic and probabilistic sensitivity analyses demonstrated that these results were robust.

Conclusions: Even for patients with mCRC manifesting improved outcomes, adding bevacizumab to first-line chemotherapy is invariably not cost-effective in any of the countries examined. Variability in pricing, healthcare costs and WTP thresholds across countries did not commute this result.

Background: Overweight and obesity is a prevalent and growing global health concern associated with a significant healthcare burden. With recent advancements in weight management interventions demonstrating cardioprotective benefits, there is a need for risk equations that can accurately predict cardiovascular event risk in health economic models to support healthcare decision making and resource allocation.

Objective: We aimed to derive risk equations using SELECT trial data that estimate the risk of acute coronary syndrome and stroke in people with established cardiovascular disease and overweight or obesity but without diabetes mellitus for use in health economic modelling.

Methods: Risk equations estimating first in-trial observed acute coronary syndrome and stroke were derived from patient-level data from the SELECT trial, a double-blind randomised placebo-controlled trial comparing semaglutide 2.4 mg with placebo. Risk factors were identified from the literature and by clinical experts. Risk equations were developed using cause-specific Cox proportional hazard models with all-cause mortality as a competing risk. Least absolute shrinkage and selection operator (LASSO) penalisation was applied 100 times in bootstrap samples to refine the risk equations. Final risk equations were validated with clinical experts and using SELECT trial data.

Results: Sex, coronary heart disease, a history of acute coronary syndrome and use of angina medications had the strongest associations with acute coronary syndrome (hazard ratio 1.67-1.82). For stroke, a history of atrial fibrillation, cerebrovascular disorder, stroke and transient ischaemic attack had the strongest associations (hazard ratio 1.40-1.70). In terms of discrimination, the risk equations had an area under the receiver operating characteristic curve (AUC) of 0.66-0.68 for acute coronary syndrome and 0.68-0.71 for stroke, and C-indices of 0.67-0.69 for acute coronary syndrome and 0.66-0.69 for stroke. The risk equations showed good cohort-level predictive capabilities over a 4-year time horizon.

Conclusions: These novel, treatment-specific, trial-derived risk equations are the first to predict the risk of secondary cardiovascular events in people with overweight or obesity and established cardiovascular disease but without diabetes. Incorporating these risk equations into health economic models may improve the accuracy of economic evaluations in this population.

Objective: We aimed to facilitate the comparison and communication of magnitudes of health inequality impact across interventions for different diseases, and to indicate the potential range of such impacts.

Methods: We propose rescaling the slope index of inequality to measure the health inequality impact as the change in the gap in total predicted quality-adjusted life-years between the least and most socially disadvantaged groups, with linear regression predictions used to account for effects on intermediate groups. We suggest reporting the inequality impact relative to the total health opportunity cost to facilitate comparison across interventions varying in scale and unit costs. We illustrated the approach with aggregate distributional cost-effectiveness analyses of hypothetical treatments for 1336 diseases in England. We approximated benefit shares for neighbourhood deprivation quintile groups using disease-specific hospital admissions. We tested between-group equality using generalised linear regression and constructed uncertainty intervals using Monte Carlo simulation. We assumed an equal total health opportunity cost and benefit-cost ratio of one, with alternative scenarios in a sensitivity analysis.

Results: Health inequality impacts of hypothetical treatments ranged from - 33.1% of the total health opportunity cost (inequality increasing) to + 45.3% (inequality decreasing), and were ≤ - 5% for 1.6% of diseases, ≥ + 5% for 41.8% and ≥ + 20% for 1.6%. The impact was positively associated with the benefit-cost ratio and decreased when more deprived groups were assumed to incur proportionately more total health opportunity costs.

Conclusions: Health inequality impacts can be compared using the change in the total predicted quality-adjusted life-year gap between the least and most socially disadvantaged groups as a proportion of the total health opportunity cost.

Valuing pediatric health-related quality of life (HRQOL) is essential for economic evaluations in child healthcare. Instruments like EQ-5D-Y were developed for this purpose. A key methodological innovation-though controversial-has been the use of the child perspective for valuation of EQ-5D-Y health states, where adults value health states imagining a 10-year-old child. This paper critically reviews empirical findings on this approach, examines potential biases, assesses alignment with stakeholder views, and explores alternatives. We relied on a targeted review of empirical literature, including studies comparing adults valuing their own health (adult perspective) and using child perspectives, as well as stakeholder opinion studies. Findings were synthesized into ten key learnings: (1) Child-perspective valuations are typically higher than adult ones for the same health states. (2) Adults prioritize pain/discomfort and being sad/unhappy differently for children. (3) Child age has minimal impact. Mechanisms contributing to differences between adult and child perspectives include (4) discomfort with child death, (5) different valuations of life duration, (6) psychological distance, (7) emotional difficulty deciding for others, and (8) external goals influencing results. Stakeholder engagement shows that (9) the effects of using child perspectives do not align well with societal preferences, and (10) stakeholders express a preference for approaches that directly involve children and adolescents in valuation tasks. We conclude that relying on child perspectives may introduce systematic biases, potentially undermining the validity of pediatric health utilities. A re-evaluation of current valuation methods for EQ-5D-Y may be warranted, with greater consideration for direct child involvement, mapping techniques, and group-based deliberative approaches.