Expert Review of Pharmacoeconomics & Outcomes Research最新文献

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.

Background: Timely access to innovative medicines remains a policy challenge in single-payer systems. South Korea has introduced mechanisms such as economic evaluation exemptions and risk-sharing agreements (RSAs) to improve access while maintaining expenditure controls. However, empirical evidence on their real-world impact remains limited.

Research design and methods: A retrospective observational analysis was conducted using data on 85 new drugs assessed by the Health Insurance Review and Assessment Service (HIRA) between 2022 and 2024. Publicly available HTA datasets were categorized by evaluation outcome, pricing ratios, and projected budget impact. Statistical analyses included descriptive analysis, correlation testing, and multivariable linear regression.

Results: Eighty-two percent of assessed drugs were reimbursed. A full recommendation from HIRA strongly predicted listing outcomes. Drugs with moderate budget impact (1.5-10 billion Korean Won) experienced shorter timelines. International price ratios - averaging 64% of the mean level across reference countries - were the only consistent predictors across settings.

Conclusion: Evaluation outcomes, assessment complexity, and budget forecasts are key determinants of reimbursement in Korea. Refinement of HTA processes and adaptive use of RSAs may enhance access efficiency while preserving budgetary sustainability.

Background: Osteoporosis is a systemic skeletal disease and has a high prevalence in Iran. This study aimed to calculate the economic burden of osteoporosis in patients referred to the Shahid Motahari Clini in 2023.

Research design and methods: This was a cost-of-illness study conducted cross-sectionally from a societal perspective in Shiraz in 2023. Overall, 252 patients were examined through a census approach. Prevalence-based and bottom-up approach were employed. Cost data were obtained using information from patient record, as well as based on the patients' self-reports. The human capital approach was used to calculate the indirect costs.

Results: The annual cost of this disease per patient was $5,223 PPP. The largest share was attributed to direct medical costs (42.75%), with hospitalization costs accounting for the highest proportion of these expenses (53.93%). The shares of direct non-medical costs and indirect costs were 38.51% and 18.74%, respectively. Furthermore, the economic burden of the disease in the country for this year was estimated at $15,749,838,044 PPP.

Conclusions: According to the results, osteoporosis can impose a significant economic burden on society, the patients, and their families. It is recommended to manage treatment strategies, implement preventive measures, and use better insurance coverage for osteoporosis medications to reduce the costs.

Objective: New prophylaxis to reduce the burden of respiratory syncytial virus (RSV) disease are available, including a long-acting monoclonal antibody (nirsevimab) and maternal immunization with an RSV prefusion F protein vaccine (RSVpreF). We compared the potential public health impact of these strategies when implemented in Panama from the payer perspective.

Methods: A static model evaluated the use of year-round nirsevimab with/without catch-up, seasonal nirsevimab with catch-up, and RSVpreF in a birth cohort. Health, cost, quality-of-life outcomes, and the number needed to immunize (NNI) were compared to the current standard of care, followed by an indirect comparison of nirsevimab and RSVpreF.

Results: Seasonal nirsevimab with catch-up would be the most effective strategy as it would prevent 62% RSV-associated lower respiratory tract disease cases compared to RSVpreF, followed by year-round nirsevimab with catch-up that would prevent 46% of cases. Each of the nirsevimab strategies would have a greater impact on all outcomes compared to RSVpreF. The NNI to prevent an RSV infection and death was lower for the nirsevimab strategies compared to RSVpreF.

Conclusion: RSV immunization strategies would significantly reduce the disease burden in Panama. Nirsevimab would have a greater public health impact than RSVpreF due to its sustained efficacy and protection regardless of gestational age.