Value in health regional issues最新文献

Objectives: To estimate utility values for patients with metastatic triple-negative breast cancer (mTNBC) and disutility due to treatment-related adverse events (AEs) using vignettes with the Japanese general population.

Methods: Health states were developed to characterize the impact of mTNBC symptoms and AEs on health-related quality of life (HRQL), based on a literature review and interviews with clinical experts. Preferences for 10 different health states were assessed, including a progression-free (PF) for patients with TNBC, 6 health states with AEs during treatment with PF, and progression of disease. Utility values were estimated using the lead-time trade-off (LT-TTO) method and analyzed with a generalized estimating equation model. A total of 320 Japanese individuals from the general population participated, with 160 each from Tokyo and Osaka.

Results: The mean utilities for the PF and progression of disease states were 0.702 (95% CI: 0.675-0.730) and 0.583 (95% CI: 0.547-0.618), respectively. Among AEs, nausea/vomiting had the greatest negative impact on HRQL (disutility = -0.936), while neutropenia had the least (disutility = -0.037).

Conclusions: This study quantified utility weights and clarified the magnitude of the impact of AEs in Japanese patients with mTNBC. These findings highlight the importance of considering HRQL decline when evaluating the cost-effectiveness of mTNBC treatments. These data can inform future cost-effectiveness analyses for various mTNBC drugs while considering the preferences of the Japanese population. They can also be used to propose the optimal treatment for mTNBC.

Objectives: This study aimed to estimate the direct and incremental healthcare costs associated with complications in people with diabetes and those without diabetes.

Methods: In this matched retrospective cohort study, adults with diabetes (n = 18 937) and controls (without diabetes) were identified from a linked administrative data set in Western Australia. Direct healthcare costs were estimated for 2021, with costs expressed in 2024 Australian Dollars (AUD). For each complication, the average cost per person and the incremental cost-difference between the average annual costs of people living with and without diabetes were computed.

Results: Complications accounted for about 15% of the total incremental costs of diabetes. Average annual costs attributable to diabetes, where individuals had ≥1 complication (51.4%), were AUD4629. The annual incremental costs were higher in people living with diabetes diagnosed with macrovascular complications (AUD5114), followed by metabolic (AUD4841) and microvascular (AUD4682) complications. The annual incremental cost in people living with diabetes diagnosed with at least 1 complication was higher in those with T2D (AUD5708) than in those with unspecified types of diabetes (AUD4325) and T1D (AUD4787). Annual incremental healthcare costs increased with an increase in the number of complications in people living with diabetes.

Conclusions: Complications, particularly vascular complications, are associated with substantial healthcare costs in people with diabetes. Timely and evidence-based interventions are needed to prevent or delay the development and progression of diabetes-related complications, particularly vascular complications, to drive cost savings and improve health and well-being.

Objectives: This study conducted cost-effectiveness of a targeted school-based dental check-up program compared with standard care in Victoria, Australia. Subsequently, a budget impact analysis was conducted if the intervention was implemented statewide to all preschools and state government primary schools.

Methods: Cost-effectiveness analyses were performed with a 2-year time horizon from the Victorian government payer perspective. Costs were adjusted to 2024 values in Australian dollars (AUD$). The 3 outcomes of interest were the (1) incremental cost per child who have never had a dental visit (2) incremental cost per child reached from lower socioeconomic disadvantage, and (3) incremental cost per disability-adjusted life-years averted. Uncertainty was characterized using probabilistic sensitivity analysis. Budget impact analysis considered alternative scenarios using 1-way and 2-way sensitivity analyses.

Results: The incremental cost-effectiveness ratios were dominant for all 3 outcome measures, indicating that the intervention is cost-effective. The generated incremental cost-effectiveness ratios was -AUD$1791 (-$8639; $2787) per child who never had a first dental visit, (2) -AUD$722 (-$3484; -$1124) per child reached from lower socioeconomic disadvantage, and (3) -AUD$2 989 645 (-$3 487 337; $102 049) per disability-adjusted life-years averted. The budget impact analysis showed the intervention, if implemented statewide, could cost the Victorian government $96.9 million more than the base-case scenario over 5 years but provides financial protection for an additional 121 284 children.

Conclusions: This study demonstrates evidence that a targeted school-based dental check-up is cost-effective and increases equitable access and financial protection through Victorian public oral healthcare.

Background: Treatment-resistant depression (TRD) imposes a substantial public health and economic burden. Although psilocybin-assisted therapy (PAT) has shown clinical promise, its economic value remains uncertain. This study evaluated the cost-effectiveness of PAT compared with the standard of care for TRD.

Methods: A Markov model adopting a US healthcare perspective simulated patient transitions among health states (remission, response, non-response, and relapse) every 6-week cycle. Model inputs were derived from randomized controlled trials and relevant published literature. Outcomes included quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios. Sensitivity analyses were conducted to assess uncertainty in key parameters, dosing regimens, retreatment strategies, and psilocybin prices. Scenario analyses extended the time horizon to 30 years to examine treatment persistence and efficacy waning.

Results: Compared with the standard of care, PAT was more effective and less costly, yielding approximately $7000 in cost savings and a gain of 0.10 QALYs per patient. These economic advantages persisted across variations in key parameters, dosing strategies, retreatment assumptions, and psilocybin prices in sensitivity analyses. Extending the horizon to 30 years in scenario analyses increased cumulative savings to $215 900 with gains of 9.87 QALYs. PAT remained cost-effective under all efficacy-waning assumptions over the 30-year horizon.

Conclusion: This modeling analysis provides preliminary evidence that PAT may be a cost-effective option for TRD management. Consistent findings across extended time horizons suggest that its economic value is largely driven by early clinical benefits that offset downstream chronic care costs. Longitudinal real-world evidence will be essential to validate these findings and inform sustainable integration into clinical practice.

Objectives: This study described modeling approaches for survival curve extrapolation beyond time periods studied in oncology adopted by the Brazilian National Committee for Health Technology Incorporation (Conitec).

Methods: A retrospective analysis of Conitec's recommendation reports for oncology drugs from 2019 to 2024 was conducted. Independent reviewers used a predefined form to extract data on model types, health states, time horizons, discount rates, and survival extrapolation methods. The reporting quality of the health economic models was assessed qualitatively using the Consolidated Health Economic Evaluation Reporting Standards 2022 checklist.

Results: Forty-four reports (43 drugs) addressing 20 types of cancer were identified, representing 69 individual assessments. Partitioned Survival Analysis (PartSA) was the most frequent model (67%), followed by Markov model (26%). Survival curve extrapolation was performed in 90% of the models. In PartSA, Weibull distribution was used in 44% of overall survival extrapolations, whereas Log-normal and Generalized gamma distributions (17% and 9%, respectively) were common for progression-free survival. Furthermore, methodological gaps were prominent: 8% of the models provided no justification for the choice of parametric distributions, and 42% failed to report validation methods for survival curve extrapolations. Regarding reporting quality, significant omissions were found for Consolidated Health Economic Evaluation Reporting Standards 2022 checklist items related to economic analysis plan (68%) and the reporting of results, limitations, and generalizability (41%).

Conclusions: PartSA is commonly adopted in Conitec's oncology drug evaluations, aligning with international Health Technology Assessment practices. However, significant gaps in methodological rigor and transparency were identified, highlighting that the application of these models still faces uncertainty and reporting challenges.

Objectives: The World Health Organization recommends deploying the RTS,S/AS01 malaria vaccine in high-burden regions. Although effective in reducing malaria cases, affordability challenges hinder widespread adoption in low-resource settings. This study examines the cost and budget impact of introducing the vaccine into Uganda's expanded immunization program.

Methods: A budget impact analysis based on prospective studies conducted in Uganda and sub-Saharan Africa, with unit costs per dose obtained from existing literature. The primary outcome was the number of malaria cases averted among eligible children. The budget impact was calculated by comparing total vaccination costs to savings from reduced malaria treatment costs, incorporating sensitivity analyses for varying vaccine coverage and malaria incidence levels.

Results: The treatment costs for complicated malaria in children under 5 were estimated at $1.1 million before vaccination, decreasing to $297 465 afterward. The estimated annual vaccination rollout cost was $29 million, representing 24% of Uganda's immunization budget and about 3.5% of total health budget. Over 5 years, the total budget impact was $145.5 million, with a net cost of $141 million. The impact varied with coverage levels, ranging from $106.3 million at lower coverage to $175.6 million at higher coverage.

Conclusions: Implementing the malaria vaccine would lead to significant increase in Uganda's immunization budget and the overall health sector budgets. To ensure sustainability, the government should prioritize funding and adopt cost-effective strategies, such as targeting high-transmission areas and expanding prevention efforts, to maintain affordability and achieve long-term success.

Objectives: To evaluate the cost-utility and budget impact of introducing individual tumor necrosis factor-alpha inhibitors, including adalimumab, etanercept, infliximab, and golimumab, as add-on therapies for patients aged 2 years or older with refractory nonsystemic juvenile idiopathic arthritis in Thailand.

Methods: Decision-tree and Markov models were used to conduct a cost-utility analysis from a societal perspective to estimate lifetime costs and health outcomes. Direct medical and nonmedical costs were obtained from a hospital database and a previous study, respectively. Health-related quality of life (QALY) was measured using the proxy child-friendly EQ-5D and a visual analog scale. Costs and outcomes were discounted at 3% per year. Results are presented as incremental cost-effectiveness ratios (ICERs) in Thai Baht (THB). Sensitivity analyses were conducted to assess parameter uncertainty, and a 5-year budget impact analysis was performed from a governmental perspective.

Results: Adalimumab demonstrated the lowest ICER (205 012-205 225 THB/QALY), followed by infliximab (441 077-640 657), etanercept (2 134 756-2 280 312), and golimumab (93 198 577). Price reductions of 28% for adalimumab and 43% to 74% for infliximab are required to meet the Thai cost-effectiveness threshold of 160 000 THB/QALY. For the base-case analysis, the 5-year budget impact of infliximab (175-244 million THB) was similar to that of adalimumab (239 million THB), whereas adalimumab achieved greater QALY gains (1.97 vs 0.76).

Conclusions: Although none of the tumor necrosis factor-alpha inhibitors were cost-effective at the current Thai threshold, adalimumab yielded the most favorable ICER among the evaluated options, whereas infliximab offered a lower budget impact at threshold prices.

Objectives: Heart failure (HF) imposes a significant economic burden on the health system. This study evaluated the cost-effectiveness of adding ivabradine to standard pharmacotherapy compared with standard pharmacotherapy alone for treating eligible Vietnamese patients with HF.

Methods: A monthly cycle Markov model was developed, consisting of 5 health states (NYHA class I-IV and mortality) to simulate a cohort of patients with HF with sinus rhythm, left ventricular ejection fraction below 35%, and a baseline heart rate of ≥70 beats per minute over a lifetime horizon. Treatment effectiveness, transition probabilities, and utility values were derived from the published literature and the Systolic Heart Failure Treatment with the If Inhibitor Ivabradine Trial. Direct medical costs were obtained from electronic medical records and national procurement data. An annual rate of 3% was applied for both costs and outcomes. One-way and probabilistic sensitivity analyses were performed.

Results: Ivabradine plus standard pharmacotherapy was associated with an incremental cost of VND 74 115 747 (USD 3035.04) and an incremental gain of 0.28 quality-adjusted life-years (QALYs), leading to an incremental cost-effectiveness ratio of VND 268 370 919 per QALY (USD 10 989.80 per QALY). The monthly cost of ivabradine was the most influential parameter affecting the incremental cost-effectiveness ratio. There is a 71.4% probability of ivabradine plus standard pharmacotherapy being cost-effective at a willingness-to-pay threshold of VND 305 700 000 per QALY (USD 12 518.43 per QALY).

Conclusion: From a Vietnamese health system perspective, adding ivabradine to standard pharmacotherapy is likely to be a cost-effective strategy for treating eligible patients with HF. This evidence supports value-based pricing in price negotiations and efficient resource use.

Objectives: Since the introduction of incremental cost-effectiveness ratios (ICERs) and cost-effectiveness assessments for new inpatient medicines in Denmark, no willingness-to-pay (WTP) threshold has been officially published. The objective of this study was to elicit such a threshold through a quantitative analysis of publicly available assessment reports.

Methods: The assessment reports were obtained from the Danish Medicines Council database. In the primary analysis, we used a logistic regression model to identify the impact of the ICERs on the reimbursement decision. The Youden's index identified the optimal probability cutoff point that maximized sensitivity and specificity of the model. The implicit WTP threshold was then defined as the ICER value corresponding to such an optimal probability cutoff point. Secondary analyses explored WTP thresholds by disease area (oncology vs other disease areas) and the impact of the size of the patient population.

Results: The primary analysis showed a statistically significant effect of the ICER on the reimbursement decision and resulted in an elicited WTP threshold of €116 122. The secondary analysis showed different WTP thresholds for oncology and other indications, although the impact of the ICER on the recommendation decision was not statistically significant. Finally, an increase in the number of patients was associated with a declining WTP threshold.

Conclusions: To the best of our knowledge, this is the first study to assess an implicit WTP threshold in Denmark. Through an open-access use case, we provide a quantitative approach that can be repeated as more data become available and applied in different settings.

Objectives: To share Saudi Arabia experience in developing external reference pricing (ERP) policy, selection process, and choice of basket of reference countries.

Method: A systematic literature search was conducted in relevant databases to determine methodological considerations and countries selection criteria for conducting ERP. Then, the existing Saudi Arabia national ERP policy was modified based on the identified criteria. The identified criteria were modulated to fit the Saudi context and to address national public health needs. Stakeholders' and experts' opinions were considered in ERP policy adjustment and in basket countries selection.

Results: Literature search revealed 4 important factors to consider in the development of ERP policy that were deemed relevant to Saudi Arabia's context: (1) similar socioeconomic conditions stratified by gross domestic product per capita, purchasing power parity, and manufacturing activities; (2) robust health systems and regulations; (3) pricing policies; and (4) geographic proximity. Before 2021, the initial ERP basket was composed of 30 countries, narrowed to 20 countries in 2021, and finally narrowed to 16 comparable countries based on the developed criteria in 2022.

Conclusion: The revision of the ERP system reduced the complexity of existing procedures. After the implementation of changes, less variability in outcomes was observed, and as a result, Saudi Food and Drug Authority received fewer appeals. The Saudi Arabian experience offers valuable insights for other countries planning to implement or refine ERP procedures. It serves as input for evaluating ERP as a pricing policy and for assessing the impact of policy design on various factors, such as prices and access to medicines.