Cost Effectiveness and Resource Allocation最新文献

Background: This paper presents the rationale and plan for a cost-effectiveness analysis conducted alongside an open-label, prospective, randomized, two-arm, multicenter, non-inferiority study by the Consortium for Advancing the Prevention and Management of Cancer in People with HIV (AMC) in sub-Saharan Africa. The study compares two commonly used chemotherapy agents, paclitaxel (PTX) and pegylated liposomal doxorubicin (PLD), administered intravenously with concomitant antiretroviral therapy (ART) for the treatment of adult persons living with Human Immunodeficiency Virus (HIV) (PLWH) with severe Kaposi sarcoma (KS) according to WHO guidelines. The two regimens are commonly used in high-resource settings but have not been formally compared in lower-resource settings.

Methods: This study uses a decision-tree model to evaluate the cost-effectiveness of PTX versus PLD for treating severe KS in adults living with HIV. A health system perspective and a two-year time horizon will be applied. Costs, including medications, labs, and hospitalizations, will be estimated using micro-costing and time-and-motion analyses. Health outcomes will be measured in Quality Adjusted Life years using PROMIS 29 + 2 utility scores. Sensitivity analyses will include Daily adjusted life years and Years of Life Lost.

Discussion: This research will provide valuable insights into the cost-effectiveness of these treatments in managing KS. The results of this analysis will have important implications for healthcare providers and policymakers, offering guidance on the optimal treatment approach for HIV-infected individuals with KS.

Trial registration: This study (NCT05411237) is registered on ClinicalTrials.gov, sponsored by the Consortium for Advancing the Prevention and Management of Cancer in People with HIV. It was first posted on June 9, 2022, with the latest update on January 29, 2025. The trial was prospectively registered before participant enrollment. Estimated primary completion is December 2027, with full completion in September 2028.

Introduction: Lipid-lowering therapy is central to cardiovascular disease (CVD) management. Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) inhibitors (alirocumab, evolocumab) and inclisiran offer greater low-density lipoprotein cholesterol (LDL-C) reduction and improved outcomes beyond statins and ezetimibe, but high costs raise affordability concerns in low- and middle-income countries. This study reviews their cost-effectiveness in developing settings.

Methods: PubMed, Scopus, Web of Science, Embase, and Cochrane Central Register of Controlled Trials (CENTRAL) were searched up to March 1, 2024. Studies were assessed for Quality-Adjusted Life Years (QALYs), Life Years Gained (LYGs), costs, and "Incremental Cost-Effectiveness Ratios" (ICERs). Quality and bias were evaluated using the "Integrated Health Economic Evaluation Reporting Standards" (CHEERS) and ECOBIAS checklists, following "Preferred Reporting Items for Systematic Reviews and Meta-Analyses" (PRISMA) guidelines.

Results: Of 580 records, 13 studies (2019-2024) from China (n = 8), Thailand, Mexico, Taiwan, Qatar, and Saudi Arabia met inclusion criteria, mostly evaluating evolocumab for primary and secondary prevention using Markov models (Time horizon: 2 years -lifetime, Discount rates: 3-5%). Studies were generally from a healthcare perspective. Evolocumab was not cost-effective in Chinese post-myocardial infarction (MI) patients (ICERs up to $281,762/QALY) but favorable in familial hypercholesterolemia(FH) ($57,018/QALY), and when evolocumab added to statins was cost-effective in patients with acute MI ($7,819 to $61,242 per QALY) and acute coronary syndrome (ACS) (vs. ezetimibe + statins) ($26,110/QALY) and atherosclerotic cardiovascular disease (ASCVD) (vs. placebo + statins)($4,268/QALY). Alirocumab ($490,198/QALY) and inclisiran ($335,404.88/QALY) were not cost-effective, while ezetimibe was ($22,965/QALY). In Thailand, PCSK9 inhibitors ($1,496,139/QALY), and ezetimibe ($33,246/QALY) exceeded thresholds. Evolocumab was cost-effective in Mexico ($37,925/QALY-$44,346/QALY) and Saudi Arabia ($49,502/QALY-$71,968/QALY) but not in Qatar. Sensitivity analyses identified drug prices, event risks, and utilities as key ICER drivers, with probabilistic analyses showing low cost-effectiveness in China (~ 2%) but higher in high-risk groups or at elevated thresholds.

Conclusion: Cost-effectiveness of PCSK9 inhibitors and inclisiran varies across developing countries, driven by drug prices, WTP thresholds, and healthcare perspectives. Evolocumab may be cost-effective in high-risk subgroups or in Mexico and Saudi Arabia but remains largely unaffordable elsewhere. Ezetimibe was consistently more favorable. Price reductions, tiered pricing, pooled procurement, and context-specific thresholds are essential to improve access and equity.

Objectives: New antiviral medications for hepatitis C can significantly reduce liver disease risk, and decrease mortality rates and associated costs. The cost-effectiveness of Glecaprevir/Pibrentasvir (GLE/PIB) has not yet been compared with other treatments in Iran, although it has demonstrated effectiveness and cost-effectiveness in other countries such as Japan and Brazil. Therefore, this study aimed to determine the cost-effectiveness of Glecaprevir/Pibrentasvir compared with Sofosbuvir/Daclatasvir (SOF/DCV) and Sofosbuvir/Velpatasvir (SOF/VEL) in Iran.

Matherial and methods: The analysis was conducted using a Markov model with a one-year cycle in a lifetime horizon from the perspective of the Ministry of Health. Effectiveness was calculated based on Quality-Adjusted Life Years (QALY). Costs were based on the direct medical costs (DMC) of Hepatitis C Virus treatment in Iran in 2024. The extraction of effectiveness was based on the results of published valid studies. The extraction of costs was done based on micro-costing and local costing. A cost-effectiveness comparison of the three investigated medication regimens was conducted through incremental cost-effectiveness ratio (ICER) and Incremental net benefit (INB). Finally, one-way sensitivity analysis and probabilistic sensitivity analysis (PSA) were used to evaluate the uncertainty of the model parameters.

Results: The study showed that the direct medical costs (DMC) and Quality-Adjusted Life Years (QALYs) for GLE/PIB, SOF/DCV, and SOF/VEL were $7505, $5493, $5443, and 21.053, 20.806, and 20.898 QALYs, respectively. The ICER of GLE/PIB compared with SOF/DCV and SOF/VEL was $8138 and 13,282, respectively. The ICER was below the national willingness-to-pay threshold of 18,261 PPP$ (one time the GDP per capita for 2022), indicating that GLE/PIB was a cost-effective treatment. In the sensitivity analysis, the model was most sensitive to some parameters such as the cost of Chronic Hepatitis C (CHC) state for GLE/PIB, the cost of CHC for SOF/VEL, and the Utility of CHC for GLE/PIB and SOF/VEL. In the probabilistic sensitivity analysis, the probability of GLE/PIB being cost-effective compared to SOF/DCV was 56% and compared with SOF/VEL was 53.7%. The acceptability curve also showed that GLE/PIB was the superior choice in 40.6% of simulations based on differential willingness to pay.

Conclusion: The results showed that GLE/PIB is cost-effective compared with the two common medication regimens in Iran, SOF/DCV and SOF/VEL, consistent with Iran's national willingness-to-pay threshold based on one time the GDP per capita, making it a good treatment option for patients with hepatitis C.

Introduction: Nutritional support is an integral part of treating critically ill children in paediatric intensive care units (PICUs). Early enteral nutrition (EEN) in the PICU has been shown to have greater benefits compared to delayed enteral nutrition (DEN) in reducing PICU and hospital stays and lowering mortality. In this study, we conducted a cost comparison and cost-effectiveness analysis of early versus delayed enteral nutrition during PICU and hospital stays for children aged 1 month to 12 years in Malawi.

Methods: We used primary and secondary data to cost PICU and hospital admissions from a payer perspective. We developed a stochastic model that assumed that equal cohorts of 500 critically ill children were admitted to the PICU and provided with EEN and DEN. Using the average length of stay in the PICU and hospital and cost data, we estimated total cohort costs and the average cost per patient for each strategy. We estimated disability-adjusted life years (DALYs) and used the total hospital costs to estimate the incremental cost-effectiveness ratio (ICER). Deterministic and probabilistic sensitivity analyses were conducted to assess the robustness of the results.

Results: The total cohort cost for EEN in the PICU was lower, $479,850, than that for DEN, $515,623. The total cohort costs for the hospital stay were also lower for EEN, $564,290, than for DEN, $613,902. The average cost per patient for EEN in the PICU was lower at $960 than $1031 for DEN. The average cost per patient for the hospital was also lower for EEN, $1129, than for DEN, $1228. EEN dominated DEN and the ICER was estimated as -$39.47/DALY averted. Probabilistic sensitivity analysis showed that EEN had a greater probability of being cost-effective, for a capacity to pay or cost-effectiveness threshold range of 0$-2000$/DALY averted, than DEN. Scaling up the implementation of EEN led to higher net monetary and health benefits.

Conclusion: EEN in children aged 1 month to 12 years was found to be less costly and more cost-effective than DEN in Malawi.

Background: The MONARCH 3 trial has demonstrated that Anastrozole combined with Abemaciclib is safe and effective for the treatment of postmenopausal hormone receptor-positive advanced breast cancer. However, its cost-effectiveness for long-term use has not been investigated yet. This study aims to evaluate the cost-effectiveness of Anastrozole combined with Abemaciclib versus Anastrozole used alone for patients in China.

Methods: Based on MONARCH 3 trial data, we constructed a Markov model using Treeage Pro 2022 software. The model cycle was set at 1 month over a period of 20- year time horizon, and the annual discount rate was set at 5%. The cost-utility analysis was used to assess the cost-effectiveness of Anastrozole combined with Abemaciclib for the treatment of postmenopausal hormone receptor-positive advanced breast cancer. The output indexes of the model were cost and quality-adjusted life year (QALY), and the model evaluation index was incremental cost-effectiveness ratio (ICER). The willingness to pay (WTP) threshold was set at 3 times the per-capita gross domestic product (GDP) of China in 2023 (¥268,074/QALY). Meanwhile, one-way sensitivity analysis and probabilistic sensitivity analysis were used to explore the uncertainties of the model and parameters.

Results: Anastrozole combined with Abemaciclib provided more health benefits than Anastrozole used alone, however, the total cost was higher. The incremental utility and incremental cost were 0.01 QALYs and ¥1075.51, respectively. And the ICER of the two regimens was ¥93,940.83/QALY, which was less than 3 times the per-capita GDP of China in 2023 (¥268,074). The results of the sensitivity analysis attested that the study results were robust.

Conclusions: Anastrozole combined with Abemaciclib is more cost-effective than Anastrozole used alone for the treatment of postmenopausal hormone receptor-positive advanced breast cancer.