Expert Review of Pharmacoeconomics & Outcomes Research最新文献

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.

Objectives: Adjuvant and neoadjuvant therapies for early-stage cancers demonstrate early clinical benefit in delaying disease recurrence. Health technology assessments require economic evaluations modeling lifetime disease trajectories. We examined modeling approaches used in Canadian health technology reviews to understand relevant challenges and identify opportunities for methodological improvements.

Methods: From CDA-AMC reimbursement recommendations for adjuvant/neo-adjuvant treatment of solid tumors, we collected outcomes and details of submitted clinical and economic evidence. We classified issues raised during economic review related to data maturity, surrogacy, treatment pathways, and assumptions surrounding extrapolation, duration of benefit and cure.

Results: Reviews from Jul/2015-Mar/2023 were identified. Reimbursement was recommended in 14/18 (78%) reviews. All assessments described OS as immature. Most (9/10, 90%) reviews with interim comparative OS data recommended reimbursement, while several (3/8, 38%) without OS data were not recommended. CDA-AMC revisions changed implications for cost-effectiveness ($50,000/QALY threshold) in 10/18 (56%) reviews. Duration of benefit assumptions was inconsistent among both submitters and reviewers. Cure-time was consistently revised to ≥5 years from initiation.

Conclusions: Despite surrogate endpoints and immature survival data, positive reimbursement recommendations were common. CDA-AMC re-analyses frequently had modest impacts on cost-effectiveness. Further guidance is needed to capture benefits and assess uncertainties with more consistency for early-stage cancers.

Objective: With increasing efforts of streamlining market access of innovative drugs, the current acceptance of health technology assessment (HTA) bodies on non-traditional trial designs is still unclear. The aim of this study was to assess the perspective of drug developers on the acceptance of non-traditional trial designs by HTA bodies in Europe.

Methods: Eleven semi-structured interviews were conducted with experts to gain insights on their perception of non-traditional trial design acceptance by HTA bodies from the Netherlands, Germany, United Kingdom, Sweden, and France. Future perspectives on and recommendations for facilitating the acceptance of non-traditional trials were provided.

Results: All experts highlighted the HTA bodies' preference for randomized controlled trials over non-traditional trial designs. The degree of acceptance varied between countries. Experts recommended multistakeholder discussions between drug developers and HTA bodies for early scientific advice and reevaluation of evidence requirements. The EU-HTA regulation was viewed as both a potential opportunity for harmonization and limiting innovative approach.

Conclusion: The results of this study highlighted significant variations in the acceptance of non-traditional trial designs by HTA bodies across Europe. Actions are needed to facilitate a more progressive view on non-traditional clinical trial designs to ensure fast patient access to highly innovative treatments.

Introduction: We aimed to assess the cost-effectiveness of non-pharmacological interventions for fatigue in patients with chronic conditions in the UK.

Methods: This systematic review of cost-effectiveness studies aligns with the Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) 2020 statement. Data sources: Electronic databases and citation searches. Inclusion criteria: Studies including adults with one or more long-term health condition, either physical or mental. Exclusion criteria: Studies associated with cancer, long-COVID, post-viral fatigue, medically unexplained conditions, developmental disorders and injuries. Assessment: A single reviewer completed a two-stage sifting process.

Results: Four studies met the inclusion criteria. They included patients with either multiple sclerosis or inflammatory rheumatic conditions, and assessed either cognitive behavioral therapy (CBT) or a personalized exercise program (PEP). CBT was either dominated by usual care or had an incremental cost-effectiveness ratio (ICER) over £30,000. PEP dominated CBT, with the ICER for PEP versus usual care ranging from £13,159 to £35,424.

Conclusions: The economic literature on this topic is much more limited than the clinical effectiveness literature, both in terms of interventions and populations covered. Future research should focus on a de novo economic evaluation to identify interventions with a high potential to be cost-effective across multiple conditions.

Registration: PROSPERO (CRD42023440141).

Introduction: Medication errors and non-adherence in older adults are major issues linked to polypharmacy, cognitive decline, and caregiver burden. Medication management robots offer promise in improving patient care but require thorough evaluation . This review synthesizes the experiences of patients and caregivers, assesses the effectiveness of MMR in enhancing medication safety and minimizing errors, and offers insights into its broader implications for elderly home care.

Methods: A mixed-methods systematic review was conducted using PRISMA guidelines. Searches were performed across six databases - PubMed, Scopus, Web of Science, Embase, CINAHL, and ProQuest - without publication date limits. Studies were appraised using the Mixed Methods Appraisal Tool (MMAT), and findings were synthesized through a convergent approach.

Results: Six studies (3 qualitative, 2 quantitative, 1 mixed-methods) from Sweden, Finland, and the U.S.A. were included. MMRs improved adherence, reduced medication errors, enhanced coordination among healthcare providers, and supported patient autonomy. Key challenges involved technical issues, such as incompatibility with liquid medications, concerns about privacy, and reluctance from users due to diminished human contact.

Conclusion: MMRs show promise in elderly care, but successful adoption depends on structured training, ethical safeguards, and user-centered design. Further research is required to validate their long-term impact and optimize implementation strategies.

Registration: The protocol for this review was registered with PROSPERO (registration number: CRD42024611310): https://www.crd.york.ac.uk/PROSPERO/view/CRD42024611310.