PharmacoEconomics最新文献

Objectives: This study aims to test whether preferences for children's health states differ (a) when considering those aged 2-4 years compared with older children and (b) by parental status; we also aim to provide a value set that can be used for 2-4 years old.

Methods: Health states were described using the Child Health Utility 9D (CHU9D). A discrete choice experiment (DCE) survey was administered between September 2023 and March 2024 to a representative sample of the Australian general adult population which included a 20% quota of parents of 0-18-year-old children. Participants were randomly allocated to two study arms considering the health of a 2-4-year-old or a 10-year-old child. A conditional logit model was used to obtain the latent values from the choice responses. The differences in latent values between the two ages and between parental status were analyzed using relative attribute importance (RAI), poolability test, and pooled model with interaction terms. Visual Analogue Scale (VAS) responses were used to anchor the latent values onto a 0-1 utility scale.

Results: In all, 3112 participants were included. Results suggested that the preferences between the two age perspectives were similar, with only 1 out of 36 interaction terms being significant. Preferences of parents of children aged 0-18 years differed from those who were not, as indicated by three significant interaction terms and failure in poolability testing, having smaller disutility for severe health states in the Pain, Tired, and Joining in Activities dimensions.

Conclusion: In the valuation of CHU9D health states, asking respondents to consider a 2-4-year-old compared with a 10-year-old did not influence adults' preferences; however, the preferences of respondents who were parents of 0-18-year-olds at that time differed from those who were not. Two CHU9D value sets are reported for children 2 years and older, one derived from the general adult population and the other from parents.

Background: Local resection of early rectal cancer (RC) is a desirable treatment option compared with surgery, offering reduced morbidity, mortality, health care costs and avoidance of stoma. However, local resection is restricted to cases without suspicion of lymph node metastases (LNM). Current methods to diagnose LNM and risk estimations based on histopathology cannot reliably identify patients eligible for local resection. The NanoEcho diagnostic system is based on a novel method for lymph node staging in RC. The aim of this study was to perform a health economic analysis at an early stage of clinical development to estimate the potential value of adding NanoEcho diagnostics to current diagnostic methods in RC.

Methods: A Markov model for RC diagnosis was developed where the costs and health outcomes, including quality-adjusted life years (QALYs), for adding the NanoEcho diagnostics to current diagnostic methods were compared with current diagnostic methods alone. The diagnostic performance of the NanoEcho diagnostic system is still unknown and the base-case analysis was performed at an assumed 85% sensitivity and 85% specificity. Two testing strategies corresponding to two alternative ways of implementing the diagnostic test in clinic were evaluated: (1) examine all patients diagnosed with RC and (2) examine only patients diagnosed with clinical stages T1 and T2.

Results: Adding the NanoEcho diagnostic system resulted in a gain of 0.032 life years and 0.124 QALYs per patient in the target population compared with current diagnostic methods alone. At a cost-neutral level, the estimated justifiable price of NanoEcho diagnostics was SEK 6995 in the first testing strategy and SEK 50,658 in the second testing strategy. The justifiable price of the NanoEcho diagnostics at a willingness to pay of 500,000 SEK/QALY was SEK 10,654 in the first testing strategy and SEK 65,132 in the second testing strategy.

Conclusion: The results indicate that adding NanoEcho diagnostics to standard of care can potentially reduce healthcare costs and increase quality of life in RC patients, assuming a sensitivity and specificity of 85%.

Background: Cystic fibrosis (CF) is a rare genetic condition requiring extensive medical care, which has a significant impact on people with CF. Advances in treatment have extended life expectancy, yet there remains a significant economic burden to manage CF. Cost-effectiveness analysis (CEA) is crucial for evaluating the economic value of treatments and screening for CF. This scoping review seeks to highlight the best practices and gaps in the current evidence base, contributing to robust and comparable CEAs in CF research.

Methods: A scoping review was conducted using PubMed and Embase. Studies were included if they featured a CEA focused on CF treatment. Data extraction covered study characteristics, model inputs, and modeling assumptions. A qualitative synthesis was conducted to assess the inclusion of considerations for both healthcare and societal impacts.

Results: In total, 11 studies were included. Of these, six focused on evaluations of supportive therapies for CF and five focused on evaluation of cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Heterogeneity in comparators and drug costing methods complicated cross-study comparisons. A qualitative review revealed differences in the types of costs and outcomes considered. Studies captured long-term disease progression, health-related quality-of-life effects, and direct medical costs.

Conclusions: This review highlights the complexity of CEAs for CF treatment and underscores the need for standardized methodologies and comprehensive evaluations, including broader economic impacts, to support more robust analyses and better-informed decision-making in CF treatment.

Population-adjusted indirect comparison (PAIC) methods aim to address some of the potential shortcomings of conventional approaches to indirect treatment comparisons by adjusting for imbalances in effect modifiers or prognostic factors and allowing for unanchored indirect treatment comparisons from disconnected networks of evidence. Health technology assessment bodies have published guidance and best practice recommendations for PAICs. However, recently published reviews of published PAICs have highlighted notable variability in implementation and a lack of transparency in the decision-making process in analyses and reporting; this hinders the interpretation and reproducibility of analyses, which, in turn, could affect reimbursement decision-making. We propose a systematic framework to address these challenges by describing considerations on six key elements of analyses: (1) definition of the comparison of interest (e.g., in terms of an estimand), (2) selection of the PAIC method, (3) selection of adjustment variables, (4) application of adjustment method, (5) risk-of-bias assessment, and (6) comprehensive reporting.

Background: For patients with resected non-small cell lung cancer (NSCLC), the risk of disease recurrence and progression is associated with a substantial humanistic, clinical, and economic burden. In the phase III ALINA trial (NCT03456076), adjuvant alectinib significantly improved disease-free survival (DFS) compared with chemotherapy in patients with resected ALK-positive NSCLC.

Objective: The aim of this study was to assess the cost-effectiveness of adjuvant alectinib versus chemotherapy for patients with resected ALK-positive NSCLC in Canada.

Methods: A cost-utility model comprising eight health states was developed to estimate lifetime patient outcomes and costs of patients with resected, ALK-positive NSCLC treated with adjuvant alectinib versus platinum-based chemotherapy from a societal perspective. Patterns of disease recurrence and progression were based on ALINA and other trial data; model assumptions were consistent with existing models and validated through consultation with expert Canadian clinicians. Cost-effectiveness was assessed in terms of estimated effect on life-years, quality-adjusted life-years (QALYs), and healthcare costs. In addition, scenario and probabilistic analyses were performed to explore model uncertainty. An annual discount rate of 1.5% was applied to both costs and outcomes (evaluated for 2023).

Results: Compared with chemotherapy, alectinib was associated with greater total life-years (19.2 versus 13.1 years) and QALYs (15.0 versus 10.1). Alectinib was dominant over platinum-based chemotherapy as it yielded a lower lifetime cost (CA $480,967.00) versus chemotherapy (CA $592,959.00). Scenario analyses showed model robustness and consistent dominance in cost-effectiveness. Probabilistic analyses results were similar to those from the base case and scenario analyses; alectinib was dominant over chemotherapy in 93.6% of simulations of incremental costs versus incremental QALYs, and remained under a willingness-to-pay threshold of CA$50,000.00 per QALY gained in 99.7% of simulations.

Conclusions: Our analysis suggests that adjuvant alectinib is dominant (i.e., more effective and less costly) to platinum-based chemotherapy in Canadian patients with resected ALK-positive NSCLC. Together with the DFS benefit seen in ALINA, this analysis supports adjuvant alectinib as an important new treatment strategy.

Background and objective: Hemophilia A is a costly, lifelong illness with multiple prophylaxis options. Previously, these options were assessed using a Peterson score-based model to simulate joint damage over time. This study built a model for the economic evaluation of hemophilia A with less socioeconomic selection bias utilizing the hemophilia joint health score (HJHS).

Methods: A mechanistically defined HJHS-based state-transition microsimulation model was implemented for the cost-utility analysis conducted over a lifetime horizon from a Canadian provincial Ministry of Health perspective, with a 1.5% discount rate on (costs and outcomes), to compare the following interventions: standard half-life (SHL), extended half-life (EHL), emicizumab, and efanesocotog alfa (EA). The health states are HJHS levels, waiting for surgery, postoperative time, and death. Individuals experience bleeds, joint bleeds (increasing the HJHS), and surgery in each health state. Disutilities include injections and postoperative time. Model validation included face validity, internal validity, comparison analysis, external validity, and predictive validity. Probabilistic analysis, pricing threshold analysis, and one-way scenario analyses were completed.

Results: EA showed lower levels of hospitalizations and surgeries and an improved joint damage experience in the simulation. However, EA was not cost-effective against emicizumab, which continued to be the most cost-effective intervention. Pricing threshold analysis indicated that a price decrease would be required for EA to dominate SHL (50% decrement) and emicizumab (55% decrement).

Conclusions: This is the first cost-effectiveness model incorporating HJHS to apply sequential joint damage to hemophilia A. While EA offers clinical benefits, our analysis suggests it will not be cost-effective from a Canadian provincial Ministry of Health perspective without a significant price decrease.