Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Objective: To evaluate the effects of standardized pharmaceutical service (SPS) on medication adherence, anxiety, and depression in patients with chronic obstructive pulmonary disease (COPD).

Research design and methods: A prospective, randomized controlled study design was adopted. 184 COPD patients hospitalized at the First Hospital of Putian City between March and August 2023 were randomly assigned to a control group receiving routine care or observation group receiving SPS provided by clinical pharmacists. SPS included health records establishment, education, medication guidance, psychological support and medication simplification. Patients were followed for 6 months. Eight-Item Morisky Medication Adherence Scale (MMAS-8), the Self-Rating Anxiety Scale (SAS), and the Self-Rating Depression Scale (SDS) were used to assess outcomes.

Results: At 3- and 6-month, the observation group had higher MMAS-8 scores (P < 0.05) and a greater of good-to-moderate adherence at 6 months (66.30% vs.35.87%; P < 0.01). Moderate-to-severe anxiety and depression rates were significantly lower in the observation group (53.26% and 54.35%) than in controls (P < 0.001). Fewer adverse events were recorded in the observation group (3 vs. 8), though not statistically significant.

Conclusion: SPS enhance medication adherence and reduce anxiety and depression in COPD patients, supporting its integration into routine COPD management.

Introduction: Despite decades of HIV prevention efforts, key populations - MSM, transgender people, PWUD, sex workers, and migrants - remain disproportionately affected by HIV. Although prevention tools are widely available, global targets remain unmet, largely due to persistent structural and sociocultural barriers, resulting in an important societal cost.

Areas covered: This scoping review analyzes 197 studies (2018-2022) quantitative and qualitative, from 48 countries to map barriers to HIV prevention across five key populations and five continents, using Medline and Scopus databases according to the PRISMA-ScR methodology. It reveals widespread legal and institutional obstacles: lack of health insurance, criminalization of their condition, stigma (including HIV and internalized stigma), violence, poverty, low health literacy, and healthcare mistrust. It also identifies major gaps in research coverage around the world.

Expert opinion: Despite major progress in HIV prevention, inequities persist in implementation and access. Our study highlights the urgent need of coordinated governmental and institutional efforts to ensure universal access to prevention tools, social protection, and stigma reduction interventions, as requested by UNAIDS. An intersectional approach that responds to the overlapping vulnerabilities faced by key populations is essential. Without systemic change, biomedical progress alone will not be sufficient to put an end to HIV.

Introduction: Antimicrobial resistance (AMR) is a complex global health challenge with significant, yet underutilized economic dimensions. Beyond the clinical aspect, this growing threat demands interdisciplinary solutions that bridge economic theory and practice.

Areas covered: This scoping review synthesizes economic perspectives on AMR through systematic analysis from Ovid MEDLINE, Scopus, EconLit, and PubMed (December 2023 to June 2025). We examine four critical domains: (1) foundational economic theories explaining AMR drivers through public goods theory, tragedy of commons, externalities, and market failures; (2) real-world market dynamics including supply-demand imbalances and principal-agent relationships in clinical settings; (3) policy interventions spanning regulatory frameworks, fiscal measures, and behavioral economics applications in antimicrobial stewardship; and (4) economic evaluation methodologies encompassing descriptive, evaluative, and predictive analyses. Our analysis reveals how theoretical economic frameworks arise in healthcare practice and why comprehensive multi-component interventions outperform single-approach strategies.

Expert opinion: Sustainable AMR mitigation requires fundamentally rethinking policy design through these interconnected economic lenses, transitioning from fragmented interventions to economically coherent frameworks that align short-term clinical decisions with long-term antimicrobial preservation. These changes demand unprecedented collaboration between economists, clinicians, and policymakers to align individual incentives with collective health security.

Background: Atopic dermatitis (AD) is a chronic skin disease that affects patients' quality of life. Adequate treatment is important but patients' preferences are not fully understood. This study aimed to investigate which treatment attributes are most important for adult European AD patients.

Research design and methods: A discrete choice experiment was conducted. Participants were asked to choose their preferred hypothetical treatment option in 15 choice sets. Six included attributes were identified by consulting literature, patients, physicians, and preference experts. A mixed logit model was applied to estimate preference coefficients.

Results: All attributes were important for seventy-five participants (79% female, mean age 37.8 years). Itch reduction was most relevant (55.95%), followed by infection risk (15.96%), mode of administration (9.64%), long-term diseases management (8.11%), onset time (5.33%), and availability on the market (5.01%). Participants prefer emollients without preservatives and oral pills, while emollients with preservatives and self-injections are associated with utility loss.

Conclusions: This study identified most important treatment attributes for AD patients. It could improve AD patients' access to suitable therapies as findings might support development of better aligned treatments and provide new perspectives for joint treatment decision-making. Main limitation was low number of participants, although significant results were still achieved.

Background: One approach to address the limitations of the EQ-5D-5L questionnaire is to add additional dimensions. This study investigated the effect of adding a vision dimension to the EQ-5D-5L questionnaire on health state valuations in people with visual impairments.

Methods: This cross-sectional study was conducted using both the standard EQ-5D-5L and an expanded version (EQ-5D-5L + V, including a vision dimension) through face-to-face interviews with 300 individuals with visual impairments. Twenty health states, with and without the vision dimension, were assessed using the Time Trade-Off (TTO) method according to the EQ-VT protocol. Data were analyzed using Chi-square and Fisher's exact tests, independent t-tests, and multivariate regression.

Results: The addition of the vision dimension significantly increased the differences in health state valuations, particularly for severe health states (P < 0.001). The largest differences in TTO scores were observed for severe health states 555555, 555553, 532441, and 351431.

Conclusion: Adding a vision dimension to the EQ-5D-5L instrument significantly affects health state valuations, especially for severe levels (4 and 5) and severe health states. Future studies should further explore the impact of adding vision and other bolt-on dimensions, focusing on the levels of the added dimensions.

Background: Cancer survivors face ongoing health challenges that can reduce work capacity, yet the economic impact of workplace productivity losses remains underexamined. We aim to estimate workplace productivity losses, specifically absenteeism and presenteeism, among Australian cancer survivors and quantified associated wage costs.

Methods: We analyzed four waves (2009-2021) of the Household, Income and Labour Dynamics in Australia (HILDA) Survey, including adults aged ≥15 years in the labor force reporting cancer, non-cancer serious illness, or no serious illness. Absenteeism (annual days off work) was modeled using zero-inflated Poisson regression, and presenteeism (self-reported reduced work performance) using logistic regression, adjusting for sociodemographic, health, and employment factors. Sensitivity analyses explored alternative absenteeism definitions.

Results: Cancer survivors had a 33% higher rate of absenteeism (Incidence Rate Ratio [IRR]: 1.33; 95% CI: 1.31-1.35, p-value < 0.001) and approximately double the odds of presenteeism (Adjsusted Odds Ratio [aOR] : 2.04; 95% CI: 1.71-2.43; p-value < 0.001) compared with individuals without serious illness. Estimated average annual absenteeism-related wage loss was AU$1425.45, predominantly among working-age adults (25-64 years).

Conclusion: Cancer imposes substantial workplace productivity losses with significant economic implications. Findings support the development of evidence-based workplace policies and targeted occupational health interventions to sustain employment and reduce economic burden among cancer survivors.

Background: Ribociclib plus letrozole significantly improves survival in HR+/HER2- advanced-stage or metastatic postmenopausal breast cancer (BC) patients.

Aim: To evaluate the cost-effectiveness of first-line ribociclib plus letrozole versus palbociclib plus letrozole for HR+/HER2- advanced BC in the Chilean public healthcare system.

Methods: A partitioned survival model with a 1-month cycle and 40-year horizon was utilized. Data were drawn from PALOMA-2 and MONALEESA-2 trials. Utility values were sourced from trial data and literature. Costs included drug acquisition, disease monitoring, subsequent therapies, and adverse events. Deterministic sensitivity analysis (DSA), probabilistic sensitivity analysis (PSA), and scenario analyses with varying clinical efficacy estimates were conducted to address uncertainty.

Results: Ribociclib plus letrozole consistently dominated palbociclib plus letrozole, showing better effectiveness at lower costs. In the base case, ribociclib saved $5,724 with an additional 0.506 QALYs. Scenario 1 showed savings of $4,942 and 0.398 QALYs, while Scenario 2 indicated $9,830 saved with 1.282 QALYs gained. PSA confirmed a 100% probability of ribociclib being cost-effective at a willingness-to-pay threshold of 1 GDP per capita.

Conclusion: Ribociclib plus letrozole is cost-saving and cost-effective compared to palbociclib plus letrozole for the first-line treatment of postmenopausal women with HR+/HER2- advanced BC in Chile.

Background: Functional genomics approaches, such as transcriptomics and proteomics, can provide valuable insights into rare diseases when genomic sequencing fails to yield informative findings. This study estimated the monetary value that parents, carers and individuals with undiagnosed rare diseases place on functional genomics testing.

Research design and methods: A triple-bounded dichotomous choice contingent valuation survey was completed by carers and individuals with suspected rare monogenic disorders recruited as part of the Australian Undiagnosed Disease Network. A multilevel interval regression model was used to analyze response data and estimate the monetary value of functional genomics, in terms of willingness to pay (WTP).

Results: There was a total of 57 respondents (48%), primarily carers (95%). The mean WTP for functional genomics testing was estimated to be $2,522 (95% CI: $817-$4,228) [US $1,568 (95% CI: $508-$2,629)].

Conclusions: Our findings indicate that individuals with undiagnosed rare diseases and their parents or caregivers place high value on functional genomics testing. The estimated WTP is comparable to findings from contingent valuation studies of other genomic interventions and exceeds the expected economic cost of proteomics testing. These insights can inform a preference-based evaluation of the diagnostic outcomes and net benefits achieved through functional genomics, thereby guiding decision-making and clinical implementation.

Introduction: Mechanical thrombectomy (MT) is a proven treatment for acute ischemic stroke (AIS), yet its cost-effectiveness across global settings remains unclear. This systematic review synthesizes cost-effectiveness evidence and introduces a framework for evaluating MT across different health systems.

Methods: Following PRISMA 2020 guidelines, comprehensive searches were conducted across four major databases to identify full economic evaluations comparing MT with intravenous thrombolysis (IVT). Methodological quality was assessed using the Quality of Health Economic Studies (QHES) checklist, and findings were synthesized qualitatively. Incremental cost-effectiveness ratios (ICERs) were converted to 2024 US dollars. Studies were categorized by income level, modeling approach, perspective, and real-world vs. trial-based data.

Results: Of 4550 records screened, 56 met the inclusion criteria. The mean QHES score was 0.83, indicating high quality. MT was consistently cost-effective or dominant across diverse healthcare contexts. Sensitivity analyses indicated that ICER variability was primarily influenced by device costs, patient age, time horizon, acute care costs, and input data used for functional outcomes and utility weights.

Conclusion: MT is a high-value intervention, but its cost-effectiveness is context-dependent. Our classification framework supports localized decision-making. Future research should address geographic inequities in existing economic evaluations and expand evidence from low-resource settings and also integrate real-world data.

Registration: PROSPERO (CRD42024496552).

Background: Patient-reported outcome (PRO) data capture patients' perspectives on their disease and its treatments; however, use of such data in health technology assessments (HTA) and pricing/reimbursement processes is limited. We provide considerations to support the incorporation of PROs in payer decision-making.

Research design and methods: A review of guidance documents released by payers until 2024 regarding requirements for using PRO evidence and scientific literature published during 2015-2024 on the trends of PRO use by payers informed the development of a discussion guide. Using this, 15 experts from seven countries were interviewed (one-on-one) to seek their opinions on PRO use in HTAs and identify potential barriers to its adoption.

Results: PRO-related guidance focused on the validity and reliability of PRO instruments, risk for bias, missing data, and economic modeling. Guidance varied between countries with essential details often missing. Expert interviews revealed that PRO use in payer evaluations may depend on established endpoints for indications and varied decision approaches and frameworks. Barriers to PRO use include the lack of capacity or technical expertise, instrument validity concerns, and data accuracy.

Conclusion: Barriers to the use of PRO data for evaluations require further efforts from all relevant stakeholders to promote PRO incorporation in payer decision-making.