Expert Review of Pharmacoeconomics & Outcomes Research最新文献

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.

Introduction: The standard treatment for gastroesophageal reflux disease (GERD) is proton pump inhibitors (PPIs). In selected cases, Nissen fundoplication is offered as a surgical treatment option, but alternative endoscopic and minimally invasive surgical alternatives are emerging. RefluxStop is a new technology for the treatment of GERD.

Research design and methods: A cost-effectiveness analysis of RefluxStop in comparison to PPI therapy and Nissen fundoplication in the Swedish healthcare setting was conducted using a Markov model and available comprehensive population and clinical trial-based long-term data. Benefits were measured in quality-adjusted life-years (QALYs). Uncertainty was determined by deterministic and probabilistic sensitivity analyses.

Results: The base case incremental cost-effectiveness ratios (ICERs) for RefluxStop in comparison to PPIs and Nissen fundoplications were SEK 48,152 (€ 4,531) and SEK 62,966 (€ 5,925) per QALY gained, respectively. At a cost-effectiveness threshold of SEK 500,000 per QALY gained, RefluxStop has a high likelihood of being cost-effective, with probabilities of 96% and 100% against Nissen fundoplication and PPIs, respectively. The results of the model remained robust with sensitivity analysis.

Conclusions: RefluxStop may offer a highly cost-effective long-term treatment alternative for chronic GERD patients over lifelong PPI therapy, but also in comparison with laparoscopic Nissen fundoplication.

Introduction: Traditional economic evaluations typically focus on direct health effects and costs offset to the healthcare system. However, vaccines offer significant indirect benefits beyond direct health effects, such as herd immunity, reduced force of infection, reduction in antimicrobial resistance, and others. Failure to consider these benefits while evaluating vaccines may undervalue vaccines. Therefore, it is argued that the full value of vaccines should be estimated by incorporating these broader benefits.

Areas covered: This review presents the broader value domains proposed in literature by various frameworks, and their definitions. The review of economic evidence of vaccine use in India to discuss to what extent these broader value domains have been considered in economic evaluations in India has been presented. We also discuss specific considerations that need to be taken care of while developing value frameworks or guidelines for the economic evaluation of vaccines.

Expert opinion: To develop a comprehensive framework tailored to the country needs, prioritize relevant value domains and optimal methodologies based on the country's healthcare context, and data availability. These value domains must align with people's as well as decision-makers preferences to ensure economic assessments are relevant and actionable.

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).

Background: Atopic dermatitis (AD) imposes a hidden burden through its negative effects on quality of life and productivity. We aim to estimate this hidden burden in adults and adolescents in Central and Eastern European (CEE) countries.

Methods: We created a burden of disease model to quantify AD's hidden burden. Humanistic burden was calculated by estimating the monetary value of quality-adjusted life years (QALYs) lost, using prevalence data from the Global Burden of Disease study and gross domestic product (GDP) per capita for each country. Indirect economic burden was estimated based on productivity loss from absenteeism and presenteeism, adjusted for labor force participation and unemployment rates. Total hidden burden was determined by combining productivity losses and QALYs lost.

Results: QALY loss due to AD ranged from 1,832 to 58,596 annually in CEE countries, equating to 38 million to approximately 1 billion Euros per country. Productivity losses ranged from 3.6 to 148.9 million Euros annually. The total hidden burden of AD represents 0.11% to 0.43% of the GDP.

Conclusions: Our estimates reflect significant differences in population size, prevalence, and economic strength among CEE countries. Adjusting findings to country-specific GDP provided insights into AD's true hidden burden, offering valuable information for decision-making.

Background: Cannabis-based medicinal products (CBMPs) are increasingly demonstrating effectiveness in treating a wide range of conditions, with a relatively high safety profile in clinical usage compared to other prescription pain medications and few contraindications. Consultation and other prescription-related costs are, at present, higher for CBMPs than for some other treatment options, leading to some concern around wider prescribing.

Research design and methods: An early cost-effectiveness model was developed to estimate the impact of prescribing CBMPs alone and/or in addition to analgesics, physiotherapy, and cognitive behavioral therapy for chronic pain in the UK for 1 year.

Results: Due to their comparative effectiveness, CBMPs were found to be cost saving. Various scenarios were model tested; in all scenarios where CBMPs decrease pain-level states, less resource use is required. Increased efficacy of 5% was conservatively assumed based on current Real-World Evidence. In this scenario, CBMPs were significantly more cost-effective, and as costs relating to the prescribing of these continue to fall, relative savings are predicted to increase.

Conclusion: These findings highlight the substantial cost saving that CBMPs may represent for the treatment of chronic pain patients, and the benefits for healthcare providers as a treatment for this often hard-to-treat population.

Introduction: Rheumatoid Arthritis (RA) is among the most prevalent chronic inflammatory diseases affecting millions of people with a significant expenditure of resources by National Healthcare Systems. This study aimed to analyze all available treatments exclusively for RA to highlight the costs of each treatment type and raise awareness of the use of biosimilar drugs.

Methods: In an Italian healthcare authority, all prescriptions made for the diagnosis of RA were extracted to verify consumption expressed in Defined Daily Dose (DDD) and the expenditure incurred expressed in euros. Consequently, a grouping into three major drug categories has been performed: anti - tumor necrosis factor alpha agents (TNFα), other injectable formulations, and novel oral formulations.

Results: Prescriptions for the second half-year 2022 and 2023 have been analyzed, with a total cost of almost 7 million euros in the sample considered. All pharmaceutical categories showed an increase in consumption, but only anti- TNFα recorded a decrease in costs from 25% in 2022 to 22% in 2023, thanks to the lower cost of the biosimilar drug.

Conclusion: The costs of RA may represent a significant spending commitment for central governments. As a result, actions are needed to encourage the preferential use of biosimilar drugs.

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.

Objectives: To estimate the cost-utility of duloxetine compared with that of a placebo, common traditional nonsteroidal anti-inflammatory drugs (NSAIDs) and cyclooxygenase-2 (COX-2) inhibitors for the treatment of osteoarthritis (OA) from a Chinese healthcare perspective.

Methods: A Markov model was constructed. The costs and utility inputs were obtained from the database and published literature. Incremental cost-effectiveness ratio (ICER) was the main model outputs. Subgroup analyses were also conducted for patients at high risk of gastrointestinal (GI) or cardiovascular (CV) AEs. Deterministic and probabilistic sensitivity analyses were performed.

Results: The model estimated an ICER of $3409.21/QALY for duloxetine compared with etoricoxib, with duloxetine dominating other active treatment strategies in patients at a low risk of GI and CV AEs. The ICER for duloxetine over etoricoxib was $322.21/QALY in patients at high risk of GI and CV AEs. These results were consistent with the sensitivity analyses; 53.64% and 53.93% of the patients were willing to use duloxetine comparing with etoricoxib, for which the thresholds were 1.0 and 3.0 per capita gross domestic product (GDP), respectively.

Conclusions: Duloxetine is a valuable option for patients with OA; however, uncertainties exist in the model, and these suggestions can be adopted with caution.

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.