Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Background: The Japanese National Immunization Program against pneumococcal disease (PD) includes 23-valent pneumococcal polysaccharide vaccine (PPSV23) but does not provide vaccination for adults aged ≥66 years. We evaluated the cost-effectiveness of 20-valent pneumococcal conjugate vaccine (PCV20) in adults aged 66-84 years with no history of PPSV23 vaccination in Japan and assessed the potential economic and health impact of introducing PCV20 on the local government (specifically, Shiga prefecture).

Research design and methods: Using a Markov model, we assessed lifetime costs, quality-adjusted life-years (QALYs), and number of prevented cases and deaths caused by PD.

Results: In national-level analysis, PCV20 was cost-effective compared with no vaccination under incremental cost-effectiveness ratio threshold of Japanese yen (JPY) 5,000,000/QALY, i.e. JPY1,677,401/QALY and JPY1,351,811/QALY from payer and societal perspectives, respectively. PCV20 was dominant (less costly and more effective) compared with PPSV23. In local-level analysis, the introduction of PCV20 required initial costs but resulted in greater cost savings related to medical expenses (-JPY424 and -JPY430 per person) and nursing care (-JPY560 and -JPY575 per person) compared to PPSV23 and no vaccination, respectively.

Conclusions: PCV20 is cost-effective compared with PPSV23 and no vaccination in adults aged 66-84 years, which could reduce the future healthcare burden in Japan.

Introduction: Preference research is crucial in shaping effective patient-centered preventive healthcare strategies. Preventive measures such as vaccinations, screenings, and proactive treatment of asymptomatic high-risk conditions aim to reduce future health risks, often in healthy individuals. Understanding patient preferences in these contexts is vital to ensure that interventions are clinically effective, acceptable, and actionable. Failing to consider patient preferences when developing preventive strategies may result in low uptake and adherence to prevention programs, ultimately limiting their impact on patient and population health.

Objectives: The objective of this study is to evaluate the cost-effectiveness of different Magnetic Resonance Imaging strategies for the early detection of prostate cancer.

Methods: A comprehensive search was conducted up to 15 December 2024, utilizing databases such as PubMed and Scopus. The focus was on full economic evaluations, excluding non-English articles and reviews. The quality of the studies was assessed using the QHES checklist. The following data were extracted: settings, interventions, populations, types of economic evaluations, health outcomes, costs, and ICERs.

Results: Of the 110 articles that were assessed, 9 were selected for qualitative analysis, resulting in an overall high-quality score. The findings demonstrated that the range of MRI costs applied differed across countries. While the financial implications of non-screening are comparatively lower, the effectiveness of using screening strategies for early detection of cancer is superior to that of non-screening. Specifically, MRI was found to be more cost-effective than conventional methods. The utilization of MRI prior to biopsy has the potential to reduce unnecessary procedures while enhancing clinical cancer detection.

Conclusion: The economic viability of integrating advanced MRI techniques into prostate cancer screening is demonstrated, leading to enhanced patient outcomes by facilitating early detection and minimizing overtreatment.

Registration: PROSPERO (CRD42024572261).

Introduction: Methodological choices need to be made during model development. These choices can influence the outcome of a National Institute for Health and Care Excellence (NICE) assessment.

Areas covered: This review aims to identify, assess, and describe possible trends within the methodological aspects of economic evaluations used in NICE assessments of treatments for metastatic breast cancer (mBC). The NICE website was searched to identify technology appraisals submitted between 1 January 2009, and 31 December 2023. In this review methodological aspects are analyzed and discussed in three clusters - input data, model settings, and model outcomes - across the following characteristics: clinical trial information, quality-of-life measures, treatments used, model structure, health states, time horizon, threshold applied, and the NICE recommendations. This review provides a reference for stakeholders who want to understand previous NICE assessments of treatments for mBC, and the settings used in those, which can optimize decisions during model development.

Expert opinion: Uniformity in the methodological choices made during model development and the economic evaluations can increase transparency, increase comparability, and reduce complexity of the NICE assessment.

Introduction: Osteoporosis-related fractures represent a significant and growing public health concern, especially in aging populations. Despite the availability of effective treatments, significant care gaps persist in both primary and secondary prevention.

Areas covered: This narrative review synthesizes key findings from economic evaluations examining the cost-effectiveness of pharmacological and non-pharmacological therapies, fracture liaison services (FLS), and FRAX®-based intervention thresholds. Relevant literature was reviewed, including systematic reviews, country specific studies, and emerging data on novel technologies. The evidence consistently demonstrates that fracture prevention strategies are cost-effective across diverse healthcare settings. Pharmacological monotherapy and sequential regimens have shown favorable economic profiles, while FLS programs, although resource-intensive, significantly reduce re-fracture rates and associated costs. Intervention thresholds should be derived clinically, with health economic modeling used to evaluate their cost-effectiveness so as to guide rational, risk-based treatment strategies.

Expert opinion: Health economic evaluations are increasingly enabling the development of osteoporosis treatment strategies, highlighting the strong cost-effectiveness potential of these interventions. However, challenges remain including heterogeneity in analytical approaches, under-representation of low- and middle-income countries, and limited incorporation of real-world (adherence) data. Maintaining methodological consistency, expanding region-specific data, and evaluating emerging technologies with robust economic frameworks are essential steps toward achieving globally equitable and cost-effective osteoporosis care.

Background: Third-generation EGFR tyrosine kinase inhibitor (EGFR-TKI) osimertinib is approved as a first-line treatment against non-small-cell lung cancer (NSCLC) harboring sensitizing EGFR mutations. Herein, we perform a retrospective analysis of real-world data on first-line osimertinib treatment among Bulgarian patients with NSCLC, comparing treatment outcomes to FLAURA results.

Research design and methods: Patient data were obtained from electronic health records over a 4-year period. Baseline characteristics and endpoints (progression-free survival [PFS], objective response rate [ORR], and clinical benefit rate [CBR]) were compared. Iterative proportional fitting was performed to balance patient characteristics prior to survival analysis.

Results: A total of 365 patients on first-line osimertinib were included. Partial responses were more frequent in the RWE cohort (24% vs 13%), while the opposite was noted for stable disease (63% vs 80%). Complete response frequency was comparable (2% vs 1%). The ORR was higher in the real world compared to in FLAURA (26% vs 14%), whereas CBR was slightly higher in the trial (89% vs 94%). The real-world PFS was higher than reported in FLAURA (19.1 vs 18.9 months), with more favorable outcomes in the RWE cohort beyond 18 months.

Conclusion: RWE closely aligns with FLAURA results, suggesting even greater benefit of first-line osimertinib in the real-world setting.

Introduction: This systematic review explores the intersectoral costs associated with pneumococcal diseases across different age groups and related impacts on societal costs.

Methods: Four databases were searched (1990-2023) for partial economic evaluation studies of pneumococcal diseases from a societal perspective. The identified intersectoral costs were presented narratively and in tabular form. Studies were appraised on quality, and the PRISMA guidelines were followed.

Results: Twenty-four studies were included with wide variation in types of analysis. Intersectoral costs were captured in five sectors: patient/family, paid labor, opportunity costs, education, and public/social services. Paid labor was the main sector considered in the included studies, followed by patient/family. Cost bearers varied according to patients' age group. Mean annual costs per child varied from $182 to $82,217 healthcare costs, $240 to $846 patient/family costs and $654 to $25,721 productivity cost. In adults, mean annual costs of $168 to $43,676 healthcare costs, and $23 to $33,149 productivity costs were reported.

Conclusions: Intersectoral costs associated with pneumococcal diseases vary widely and appear in several sectors. More cost-of-illness studies are needed with wider perspectives, particularly in developing countries, to understand true economic costs of pneumococcal diseases and support the endeavor of informing a decision on treatment/preventive strategies.

Registration: PROSPERO (CRD42023417042).

Background: Active travel (AT), particularly cycling, is increasingly recognized as a public health strategy to promote physical activity and prevent non-communicable diseases (NCDs). Given the substantial investments required to build cycling infrastructure, economic evaluations are essential to inform policy and efficient funding decisions. This systematic review aims to identify and assess economic evaluations of cycleway infrastructure, with particular emphasis on the methodological approaches employed.

Methods: A systematic review was conducted using databases including PubMed, EconLit, Business Source Premier, CINAHL Plus, and MEDLINE. Eligible studies focused on cycling-specific infrastructure, reported both costs and benefits, and included a comparator. Data were extracted on evaluation type, costs, benefits, perspective, and time horizon. Study quality was assessed using the Drummond checklist, and findings were synthesized narratively, following PRISMA guidelines.

Results: Eight studies met the inclusion criteria, using cost-benefit analysis or cost-effectiveness analysis. Interventions ranged from single cycle tracks to complex networks. Most studies were hypothetical and relied on secondary data and modeling assumptions. All reported positive economic returns. Equity impacts were rarely considered, and substantial methodological variability was observed.

Conclusion: Cycling infrastructure appears to demonstrate economic value for society. However, methodological inconsistencies and data limitations remain considerable and limit comparability and generalizability of findings.

Registration: PROSPERO Protocol:(CRD420251005334).

Background: Meibomian gland disease (MGD) is a leading cause of dry eye disease (DED), yet current treatments like over-the-counter and prescription drops do not address this root cause. TearCare is an FDA-cleared, in-office procedure that directly targets MGD, but its cost-effectiveness has not been previously evaluated.

Research design and methods: We conducted a cost-utility analysis (CUA) comparing TearCare with topical cyclosporine 0.05% (CsA) for moderate-to-severe MGD-related DED. A U.S. payer perspective and 1-year time horizon were used. A Markov model with 3-month cycles evaluated transitions across four Ocular Surface Disease Index (OSDI)-defined health states. Transition probabilities and persistence rates were derived from the SAHARA trial and literature.

Results: TearCare provided better outcomes (e.g. more patients improving to mild/no symptoms) and was less costly ($4,916 vs $5,819), with a quality-adjusted life year (QALY) gain of 0.014. This corresponds to approximately 5.1 additional days in perfect health over 1 year. The incremental cost-effectiveness ratio (ICER) showed TearCare to be dominant (more effective and less costly).

Conclusions: TearCare is a cost-effective treatment for MGD-related DED, offering both clinical benefits and cost savings over CsA.

Introduction: Prevention and quality of care are increasingly recognized as fundamental drivers of sustainable, high-performing health systems. Both have demonstrated cost-effectiveness and long-term benefits, yet remain underfunded and fragmented across many European Union Member States. The COVID-19 pandemic offered a natural stress test, revealing significant variation in investment patterns, system responsiveness, and outcome efficiency.

Areas covered: This article integrates economic theory, empirical evidence, and policy analysis to explore how prevention and quality jointly shape system value. It includes analyses of prevention expenditure trends, elasticity to GDP and health spending, and cross-country efficiency indicators across EU Member States (2019-2022). The findings draw from Eurostat data and a targeted review of economic literature on cost-effectiveness and value-based care.

Expert opinion: Empirical results confirm that prevention is income- and budget-elastic, but efficiency and impact depend on institutional capacity and governance. The underuse of economic tools in quality planning and prevention prioritization hampers performance. Embedding efficiency metrics, dynamic modeling, and performance-based allocation into policy frameworks is essential to enhance value and resilience. In the coming years, prevention and quality should be better embedded in fiscal planning and system performance, not just as public health imperatives - but as economic necessities.