Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Objectives: This study aims to provide a clear understanding of the relative economic evaluation of hysterectomy, first- and second-generation endometrial ablation, and levonorgestrel-releasing intrauterine device for treatment of heavy menstrual bleeding.

Methods: A comprehensive search was conducted without restrictions until October 5, 2023, across databases including PubMed, EMBASE, Scopus, Cochrane, and others. The review included studies using full economic evaluation methods to compare treatments, excluding review articles, editorials, abstracts, and non-English articles. Methodological quality of included studies was assessed using the QHES checklist and analyzed with Incremental Cost-Effectiveness Ratios.

Results: Out of 156 articles assessed for full-text evaluations, 23 publications were selected for qualitative analysis. Two studies deemed to be of low quality were excluded from the final analysis, while the majority of studies showcased high quality. The findings presented diverse perspectives on the most cost-effective treatment, with LNG-IUD frequently emerging as the preferred choice.

Conclusion: There are diverse methodologies in economic evaluations, impacting reported health economic outcomes due to varying perspectives, time horizons, and modeling approaches. The review highlighted the need for further research to establish optimal HMB treatment strategies and to guide healthcare resource allocation.

Registration: PROSPERO (CRD42024530176).

Objectives: Recently, the IMmotion151 trial evaluated the safety and efficacy of atezolizumab plus bevacizumab in metastatic renal cell carcinoma (mRCC) and found that this combination led to longer progression-free survival. However, no studies have evaluated the cost-effectiveness of atezolizumab plus bevacizumab.

Methods: We constructed a Markov model to evaluate the cost-effectiveness of atezolizumab plus bevacizumab, using costs and utilities from the published studies. We set the willingness-to-pay (WTP) threshold at $150,000. One-way and probabilistic sensitivity analyses were performed to ensure that our results were robust. We performed a threshold analysis to explore a more appropriate price for atezolizumab.

Results: Our results found that although atezolizumab plus bevacizumab provided more quality-adjusted life years (QALYs), its incremental cost-effectiveness ratio (ICER) was $1,640,532/QALY, well above the WTP threshold. One-way and probabilistic sensitivity analysis results confirmed the robust of this conclusion. Based on the threshold analysis, for atezolizumab plus bevacizumab to be cost-effective, the price of them would need to be reduced by 46.3% or more.

Conclusions: From the perspective of US payers, atezolizumab plus bevacizumab is not cost-effective for mRCC patients. To make this combination cost-effective in the future, the price of atezolizumab and bevacizumab needs to be reduced.

Introduction: Patient and Public Involvement and Engagement (PPIE) involves working 'with' or 'by' patients and the public, rather than 'to,' 'about,' or 'for' them, and is integral to neurological and digital health research. This rapid review examined PPIE integration in the development and implementation of digital health technologies for neurological conditions.

Methods: Key terms were input into six databases. Included articles were qualitative studies or PPIE activities involving patient perspectives in shaping digital health technologies for neurological conditions. Bias was evaluated using the NICE qualitative checklist, with reporting following PRISMA guidelines.

Results: 2,140 articles were identified, with 28 included. Of these, 25 were qualitative studies, and only three were focused PPIE activities. Patient involvement was mostly limited to one-off consultations during development.There was little evidence of PPIE during implementation, and minimal reporting on its impact.

Conclusions: PPIE has been inconsistently reported in this research area, highlighting the need for more guidance and best-practice examples This review used a UK-based definition of PPIE, which may have excluded relevant activities from other countries. Future reviews should broaden terminology to capture PPIE integration globally.

Background: While medical costs of chickenpox have been researched, little is known about indirect costs. Understanding total costs is important for decisions about vaccination. This study estimated the value of lost productivity of adults missing work to care for children with chickenpox.

Research design and methods: It comprised an international literature review, an online survey of 1,526 parents of children aged 1-11 years, and computation of indirect costs of chickenpox in the UK. The survey covered chickenpox episodes amongst respondents' children, time children took off school/nursery, and work absenteeism by parents/caregivers caring for them.

Results: Respondents reported on 2,283 children, of whom 52% (1185/2283) experienced chickenpox. Almost half (591/1185) missed days of school/nursery, averaging 5.6 days missed. In 260 cases of 542 adults providing data with such a child, an adult missed work to care for the child. The daily value of this lost productivity was £170. There were approximately 200,000 GP consultations for chickenpox and 625,000 births annually, suggesting the annual chickenpox incidence lies between these figures. The estimated annual UK productivity loss due to chickenpox is £20-£70 million ($25-$90 million).

Conclusions: Annual value of lost productivity due to chickenpox is in range £20 to £70 million.

Background: In Japan, medical expenses for COVID-19 treatment transitioned from full public funding support to out-of-pocket (OOP) payment by patients plus partial public support in October 2023, and public support fully ended in March 2024. This study evaluated the clinical and economic impacts of initiating OOP payments.

Research design and methods: To assess the impact on prescription rates, we compared the prescription rates of antivirals from the 4-month pre- to post-OOP payment initiation period using a claims database. Subsequently, a budget impact model assessed the impacts of a hypothetical decline in the prescription rates on COVID-19-related hospitalizations, deaths, and direct medical costs for antiviral prescription and hospitalization.

Results: The antiviral prescription rate per 100 patients decreased from 17.5 for the pre-OOP payment initiation period to 11.5 for the post-OOP payment initiation period, that is, a change of - 34.3%. With prescription rate decreases of 40%, 60%, and 80%, the hospitalizations would increase annually by 22,533 (3.3%), 33800 (5.0%), and 45,066 (6.6%), respectively. The total costs would increase by JPY9.5 billion (USD67.3 million; 0.7%), JPY14.3 billion (USD100.9 million; 1.0%), and JPY19.0 billion (USD134.5 million; 1.3%), respectively.

Conclusions: Higher OOP payment decreased the antiviral prescription rate, potentially leading to clinical and economic loss.

Objectives: In 2013, South Korea introduced risk-sharing agreements (RSAs) as a new reimbursement mechanism to enhance access to new medicines and to manage pharmaceutical expenditures. This study evaluates RSAs in South Korea from the viewpoints of key stakeholders.

Methods: In 2022, a survey and semi-structured interviews were conducted. Study participants were recruited from academia (n = 3), domestic (n = 4) and foreign (n = 6) manufacturers, and government agencies (n = 6) using a purposive sampling method.

Results: Key stakeholders perceived the objective of RSAs to be 'access to medicines' and understood RSAs to manage uncertainty about 'expenditures.' They responded that financial- and performance-based RSAs address uncertainty about 'expenditures' and 'clinical effectiveness,' respectively. All stakeholders agreed that RSAs have increased the likelihood that new medicines will be listed and have reduced out-of-pocket expenditures for patients. However, foreign manufacturers insisted that the benefits of RSAs are marginal, while the administrative burden on manufacturers is high.

Conclusion: The gaps in perception between stakeholders could be narrowed by conducting a comprehensive evaluation. Financial- and performance-based RSAs need to be clearly distinguished and aligned to address the uncertainties of a new medicine in health systems.

Background: To evaluate the cost-effectiveness of first-line sintilimab plus chemotherapy versus chemotherapy for advanced esophageal squamous cell carcinoma (ESCC) from the perspective of the Chinese health service system.

Methods: A partitioned survival model was constructed to simulate quality-adjusted life years and incremental cost-effectiveness ratios over a patient's lifetime based on a phase III clinical trial.

Results: Sintilimab plus chemotherapy increased by 0.316 QALY and 0.285 QALY with the additional cost of $5692 and $5269, which led to the ICER of $18000/QALY and $18519/QALY gained in the overall population and the patients with CPS ≥ 10, respectively.

Conclusions: Compared with chemotherapy alone, sintilimab may be a cost-effective first-line treatment choice for locally advanced or metastatic ESCC.

Background: To analyze the time from drug registration to reimbursement recommendations, we examined medicinal products, including new clinical indications, registered by the EMA between 2014 and 2019 across various therapeutic areas.

Materials and methods: The Polish Agency for Health Technology Assessment and Tariffication (AOTMiT) was compared with 11 agencies in England, Wales, Ireland, Scotland, the Netherlands, Norway, France, Germany, New Zealand, Canada, Australia. A total of 1,942 recommendations published by 12 HTA agencies were analyzed.

Results: The time from registration to recommendation in Poland was statistically significantly longer than for the other countries. The analysis revealed noticeable differences in the time it takes from drug registration to recommendation across the countries included in this analysis. Analyzing trends from 2014 to 2019 across individual countries, there appears to be a slight tendency toward a decrease in the median time from registration to recommendation in many agencies.

Conclusions: This may suggest improvements in the processes of the recommending authorities and the companies responsible for providing data for assessment. Despite Poland having one of the longest times from registration to recommendation among the countries analyzed, there has been a clear year-over-year decrease in the time to publication of reimbursement recommendations.

Objectives: Adherence to the American Diabetes Association (ADA) Standards of Medical Care is low. This study aimed to assist pharmacists in identifying patients for diabetes control interventions using unsupervised machine learning.

Methods: This study analyzed the 2021 Medical Expenditure Panel Survey and used a k-mode cluster analysis. Patient features analyzed were adherence to a select set of preventive measures from the ADA Standards of Medical Care (HbA1c test, foot examination, blood cholesterol test, dilated eye examination, and influenza vaccination) and some patient characteristics (age, gender, health insurance, insulin use, and diabetes-related complications).

Results: The study included 1,219 patients with self-reported diabetes, and the adherence rate to the ADA standards was 33.72%. Five distinct clusters emerged: (A) moderate-complexity, privately insured male; (B) moderate-complexity, publicly insured female; (C) low-complexity, privately insured female; (D) high-complexity, publicly insured female; (E) moderate-complexity, publicly insured male. Groups B, C, and E exhibited nonadherence.

Conclusions: Pharmacists can target publicly insured elderly (Groups B and E) and privately insured middle-aged females (Group C) for interventions. For instance, pharmacists may help patients in Groups B and E locate existing resources in their insurance program and remind those in Group C of the importance of adequate diabetes care.

Introduction: Probabilistic analysis, also referred to as probabilistic sensitivity analysis (PSA), is used extensively in cost-effectiveness evaluations of health technologies. We present methodological guidance for implementing probabilistic analysis and interpreting its results for policy and decision-making.

Methods: We review the methodological issues related to common practices in probabilistic analysis, explore aspects that are currently not widely addressed in the health economics literature, and provide an overview of recent methodological developments.

Results: We use examples to highlight the advantages and disadvantages of common tools used for presenting probabilistic analysis results, including the cost-effectiveness acceptability curve (CEAC), cost-effectiveness acceptability frontier (CEAF), and value of information (VOI) analysis. We raise and address issues related to using Monte Carlo standard error to determine the number of iterations required, the implications of large uncertainty, and the credibility and meaningfulness of small differences in quality-adjusted life-years (QALYs). We then discuss evolving methods in probabilistic analysis, cautious uses of probabilistic analysis, and factors impacting parameter uncertainty.

Conclusions: A deeper understanding of probabilistic analysis methods enables health economists and decision-makers to more effectively address and interpret parameter uncertainty in health economic evaluations, which is essential for making informed policy decisions.