Development of an assessment tool for designated medical institutions in China--Based on the application of an online assessment system.

Background: Due to the expanding coverage of medical insurance and the growth of medical expenses, the ability to assess the performance of designated medical institutions (DMIs) in supporting the delivery of high-quality patient care and the standardized use of funds represents a priority in China. Despite such interest, there has yet to be an operable standard and labor-saving method for assessing DMIs in China. Objective: The main objectives include two aspects: (1) establishing an evaluation index system for DMIs based on contracts; (2) designing and developing an online evaluation platform. Methods: A group of 20 experts with theoretical and practical expertise in medical insurance regulation and performance evaluation were invited to select available indicators. A combination weighting method based on analytic hierarchy process and entropy method was used to determine the weight coefficient. Shanghai was taken as the sample area, and 760 DMIs were included in the empirical research. The test-retest reliability method and criterion-related validity method was used to test the reliability and the validity of the evaluation result. Results: An assessment index system that included 6 domains and 56 indicators was established in this study. Furthermore, we developed an online platform to assist in the implementation of the assessment. The results showed that the average score of assessment was 94.39, the median was 96.92. The test-retest reliability value was 0.96 (P ≤ 0.01), which indicated high stability of the assessment. In addition, there was a significant negative relationship between assessment score and the penalty amount of DMIs (R = -0.133, P < 0.001). After adjusting for the basic characteristics of medical institutions, the number of visits and revenue, the negative relationship was still significant (B = -0.080, P < 0.05). These results are consistent with expectations, indicating that the assessment had good criterion-related validity. Conclusions: This study established an operable assessment measure and developed an online platform to assess the performance of DMIs. The results showed good feasibility and reliability in empirical research. Our research findings provided an operable Chinese solution for DMI assessment that saves manpower and time, which would have good enlightening significance in other regions of China and in low-income and middle-income countries internationally.

Cost-effectiveness of screening for amblyopia among kindergarten children in China.

Objective: Current cost-effectiveness analyses of amblyopia screening are mainly from western countries. It remains unclear whether it is cost-effective to implement a preschool amblyopia screening programme in China. Our study aimed to evaluate the cost-effectiveness of a hypothetical kindergarten-based amblyopia screening versus non-screening among 3-year-old children. Methods: We developed a decision tree combined with a Markov model to compare the cost and effectiveness of screening versus non-screening for 3-year-old children from a third-party payment perspective. The primary outcomes were quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio (ICER). Costs were obtained from expert opinions in different regions of China. Transition probabilities and health utilities were mainly based on published literature and open sources. Sensitivity analyses were performed to assess the impact of parameters' uncertainty on results. Results: Base-case analysis demonstrated that the ICER of screening versus non-screening was $17,466/QALY, well below the WTP threshold ($38,223/QALY) for China. One-way sensitivity analysis showed that the prevalence of amblyopia, the transition probability per year from untreated amblyopia to healthy, and the discount rate were the top three factors. The likelihood of cost-effectiveness of screening compared with non-screening was 92.56%, according to probabilistic sensitivity analysis. Scenario analysis also indicated that ICER was lower than the WTP threshold even if the time horizon was shortened or the screening was delayed to the age of 4 or 5. Conclusions: Amblyopia screening could be considered a cost-effective strategy compared to non-screening for 3-year-old children in China. Screening for children at the age of 4 or 5 may even yield better results.

Causality Assessment Between Idiopathic Inflammatory Myopathies and Lung Cancer: A Bidirectional 2-Sample Mendelian Randomization.

BACKGROUND: Although observational studies have revealed associations between idiopathic inflammatory myopathies (IIMs) and lung cancer (LC), they have not established a causal relationship between these 2 conditions. METHODS: We used a 2-sample Mendelian randomization approach to examine the bidirectional causal associations between IIMs and LC, using single-nucleotide polymorphisms selected from high-quality genome-wide association studies in the FinnGen database. Sensitivity analyses were conducted to assess potential heterogeneity and pleiotropy impacts on the Mendelian randomization results. RESULTS: Our analysis demonstrated a positive causal effect of genetically increased IIM risk on LC (odds ratio, 1.114; 95% confidence interval, 1.057-1.173; p = 5.63 × 10 -5 ), particularly on the lung squamous cell carcinoma subtype (odds ratio, 1.168, 95% confidence interval, 1.049-1.300, p = 0.00451), but not on lung adenocarcinoma or small cell lung cancer. No causal effect of LC on IIMs was identified. Sensitivity analyses indicated that horizontal pleiotropy was unlikely to influence causality, and leave-one-out analysis confirmed that the observed associations were not driven by a single-nucleotide polymorphism. CONCLUSION: Our findings offer compelling evidence of a positive causal relationship between IIMs and LC, particularly with regard to lung squamous cell carcinoma, in the European population. Conversely, there is no evidence of LC causing IIMs. We recommend that LC diagnosis consider the specific characteristics of IIMs.

Medical resource utilization and the associated costs of asthma in China: a 1-year retrospective study.

BACKGROUND: Despite evidence that severe and poorly controlled asthma are associated with more clinical unmet needs and intensive utilization of healthcare resources, limited data is available on severe asthma expenditure in China. The study aimed to assess Medical Resource Utilization (MRU) costs of asthma and explore the cost drivers in order to better understand the economic burden of the Chinese population suffered from asthma. METHODS: A retrospective analysis was conducted using Chinese sampled national claim database. Patients aged 6 years and above with primary diagnosis of asthma and asthma-related medical visit/hospitalization during 2015 were included. Medication was used as a proxy per the GINA and China guideline to identify asthma severity (i.e. mild, moderate, and severe). multiple linear regression model was conducted to identify MRU costs drivers. RESULTS: 7,254 patients diagnosed with asthma were included: 4,529 (62.4%), 2,200 (30.3%), and 525 (7.2%) had mild, moderate, and severe asthma, respectively. On average, each severe patient spent 6,782 Chinese Yuan (CNY) on asthma treatment and had 57.0% hospitalization rate during the year, 3.9- and 4.4-fold of the average of overall population (P < 0.001 for both). The proportion of patients experiencing exacerbation significantly higher in the severe asthma population (66.5%; P < 0.001) compared to mild (30.0%) and moderate (16.8%) groups. In subgroup with 1,660 samples had annual consecutive data, severe patients had annual cost of CNY 8,314 and 52.2% hospitalization rate. 13% of severe patients who had frequent severe exacerbation (≥ 2 events) experienced the highest annual average cost CNY 23,037, P < 0.001) whereas children aged from 6 to 14 with a lower annual cost of CNY 1,094.2, 1,660.2 and 3,020.2 for mild, moderate, and severe patients respectively. The multiple model identified degree of severity, control status, complications, age, and live region as independent drivers of MRU costs. CONCLUSIONS: Chinese asthma patients bear heavy economic burden. Severe asthma associated with higher MRU (mainly from hospitalization) and costs compared with mild to moderate asthma in China. More efforts should be devoted to the control of the disease severity and complication as the main drivers of asthma cost.

Cost-Effectiveness Analysis of Risk Factor-Based Lung Cancer Screening Program by Low-Dose Computer Tomography in Current Smokers in China.

Although the effectiveness of lung cancer screening by low-dose computed tomography (LDCT) could be shown in China, there could be variation in the evidence concerning the economic impact. Our study explores the cost-effectiveness of lung cancer screening and optimizes the best definition of a high-risk population. A Markov model consisting of the natural history and post-diagnosis states was constructed to estimate the costs and quality-adjusted life years (QALYs) of LDCT screening compared with no screening. A total of 36 distinct risk factor-based screening strategies were assessed by incorporating starting ages of 40, 45, 50, 55, 60 and 65 years, stopping ages of 69, 74 and 79 years as well as smoking eligibility criteria. Screening data came from community-based mass screening with LDCT for lung cancer in Guangzhou. Compared with no screening, all screening scenarios led to incremental costs and QALYs. When the willingness-to-pay (WTP) threshold was USD37,653, three times the gross domestic product (GDP) per capita in China, six of nine strategies on the efficiency frontier may be cost-effective. Annual screening between 55 and 79 years of age for those who smoked more than 20 pack-years, which yielded an incremental cost-effectiveness ratio (ICER) of USD35,000.00 per QALY gained, was considered optimal. In sensitivity analyses, the result was stable in most cases. The trends of the results are roughly the same in scenario analyses. According to the WTP threshold of different regions, the optimal screening strategies were annual screening for those who smoked more than 20 pack-years, between 50 and 79 years of age in Zhejiang province, 55-79 years in Guangdong province and 65-74 years in Yunnan province. However, annual screening was unlikely to be cost-effective in Heilongjiang province under our modelling assumptions, indicating that tailored screening policies should be made regionally according to the local epidemiological and economic situation.

Cost supervision mining from EMR based on artificial intelligence technology.

BACKGROUND: To effectively monitor medical insurance funds in the era of big data, the study tries to construct an inpatient cost rationality judgement model by designing a virtuous cycle of inpatient cost supervision information system and exploring a complete set of inpatient cost supervision methods. OBJECTIVE: To lay the foundation for applying artificial intelligence (AI) technology in medical insurance cost control supervision and provide feasible paths and available tools for medical insurance cost control managers. METHODS: By way of collecting and cleaning electronic medical record (EMR) data from 2016 to 2018 of a city in East China, focusing on basic patient information and cost information, and using a combination of machine learning modeling and information system construction, the study tries to form a feasible inpatient cost supervision method and operation path. RESULTS: The set of the regulatory method, applied in nursing homes of a city in East China, is compelling. The accuracy rates of rationality judgement in different main diseases are stable up to 80%, the false positive rate is steady within 10%, and rehabilitation fee days of hospitalization, and the number of complications are important factors affecting the rationality of the inpatient cost. CONCLUSION: The model construction and optimization method combining machine learning and information system can make practical cost rationality judgement on medical institution's inpatient cost data, which can directly reflect the key influencing factors of relevant inpatient costs, and achieve the effect of guiding medical behavior and improving the efficiency of medical insurance fund use.

Effect of feeding a dried distillers' grains with solubles diet on the metabolism of the intestinal wall in Guanling crossbred cattle: a preliminary assessment.

Dried distillers' grains with solubles (DDGS)-based diets are nutritious and can improve the inflammations and intestinal immunity in livestock. However, there is limited research examining the effect of feeding DDGS-based diets on changes in intestinal metabolites and related pathways in livestock. In this study, six Guanling crossbred cattle (Guizhou Guanling Yellow cattle × Simmental cattle) were selected and divided into a basal diet (BD) group and an experimental group fed with DDGS replacing 25% of the daily ration concentrates (DDGS) (n=3), respectively. Fresh jejunum (J), ileum (I) and cecum (C) tissues were collected for metabolomic analysis. Differential metabolites and metabolic pathways were explored by means of univariate and multivariate statistical analysis. In comparison to the J-BD group, 123 differential metabolites (VIP > 1, p < 0.05) were identified in the J-DDGS group, which (top 20) were mainly divided into superclasses, including lipids and lipid-like molecules, organic acids and derivatives, and organoheterocyclic compounds. Compared with the I-BD group, 47 differential metabolites were obtained in the I-DDGS group, which were mainly divided into superclasses, including lipids and lipid-like molecules and organic acids and derivatives. The C-DDGS vs. C-BD comparison revealed 88 differential metabolites, which were mainly divided into superclasses, including lipids and lipid-like molecules, organic oxygen compounds, and nucleosides. A total of 34 significant metabolic pathways were found (p < 0.05, -log(p) > 1.3). Among them, 3 significant pathways were significantly enriched in the J-DDGS group, 11 significant pathways were significantly enriched in the I-DDGS group, and 20 significant pathways were significantly enriched in the C-DDGS group. Importantly, primary bile acid biosynthesis, linoleic acid metabolism, and arachidonic acid metabolism correlated with intestinal inflammation and immunity by regulating gut microbiota, prostaglandin synthesis, and cell signaling. The data suggest that DDGS-fed cattle unregulated three metabolic pathways mentioned above and that a DDGS-based diet was able to maintain a balance of these three metabolic pathways, thus resulting in improvement of intestinal inflammation and enhanced immunity in cattle. In conclusion, the DDGS diet has the potential to improve intestinal inflammation and enhance the immunity of Guanling crossbred cattle by regulating the metabolic patterns of lipids and lipid-like molecules, organic acids and derivatives, and related metabolic pathways. These results allude to potential metabolic regulatory mechanisms of DDGS diets and also provide a theoretical basis for the application of DDGS in livestock feed.

Cost-effectiveness of folic acid therapy for primary prevention of stroke in patients with hypertension.

BACKGROUND: For hypertensive patients without a history of stroke or myocardial infarction (MI), the China Stroke Primary Prevention Trial (CSPPT) demonstrated that treatment with enalapril-folic acid reduced the risk of primary stroke compared with enalapril alone. Whether folic acid therapy is an affordable and beneficial treatment strategy for the primary prevention of stroke in hypertensive patients from the Chinese healthcare sector perspective has not been thoroughly explored. METHODS: We performed a cost-effectiveness analysis alongside the CSPPT, which randomized 20,702 hypertensive patients. A patient-level microsimulation model based on the 4.5-year period of in-trial data was used to estimate costs, life years, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) for enalapril-folic acid vs. enalapril over a lifetime horizon from the payer perspective. RESULTS: During the in-trial follow-up period, patients receiving enalapril-folic acid gained an average of 0.016 QALYs related primarily to reductions in stroke, and the incremental cost was $706.03 (4553.92 RMB). Over a lifetime horizon, enalapril-folic acid treatment was projected to increase quality-adjusted life years by 0.06 QALYs or 0.03 life-year relative to enalapril alone at an incremental cost of $1633.84 (10,538.27 RMB), resulting in an ICER for enalapril-folic acid compared with enalapril alone of $26,066.13 (168,126.54 RMB) per QALY gained and $61,770.73 (398,421.21 RMB) per life-year gained, respectively. A probabilistic sensitivity analysis demonstrated that enalapril-folic acid compared with enalapril would be economically attractive in 74.5% of simulations at a threshold of $37,663 (242,9281 RMB) per QALY (3x current Chinese per capita GDP). Several high-risk subgroups had highly favorable ICERs < $12,554 (80,976 RMB) per QALY (1x GDP). CONCLUSIONS: For both in-trial and over a lifetime, it appears that enalapril-folic acid is a clinically and economically attractive medication compared with enalapril alone. Adding folic acid to enalapril may be a cost-effective strategy for the prevention of primary stroke in hypertensive patients from the Chinese health system perspective.

Copanlisib plus rituximab combination therapy vs. rituximab monotherapy for relapsed indolent non-Hodgkin lymphoma: a cost-effectiveness analysis.

Background: In the clinical use of third-line treatment of non-Hodgkin lymphoma (NHL), the combination treatment is increasingly used due to problems such as drug resistance, and while their efficacy has been proven, whether they are economical has become a new issue. A recent trial showed copanlisib plus rituximab combination therapy (CRCT) had better efficacy in the treatment of relapsed indolent NHL (iNHL) compared to rituximab monotherapy (RM). However, the long-term cost and effectiveness of this regimen is not known. We are the first to evaluate the cost effectiveness of CRCT in third-line treatment of relapsed iNHL from the perspective of US payers. Methods: We used a Markov model to evaluate cost and quality-adjusted life years (QALYs) which included a population from CHRONOS-3 with mean age of 62.5 years and total cycle length of 16.3 years. The cycle length was 1 month, adverse reaction rates were from CHRONOS-3, mean body surface area was referenced from published literature, cost values are referenced from published literature and Drugbank, utility values were referenced from the published literature, and the primary endpoint was the incremental cost-effectiveness ratio (ICER). The willingness to pay (WTP) threshold was set at $150,000 per QALYs, and one-way sensitivity analysis and probabilistic sensitivity analysis were used to verify the robustness of the model. All costs are expressed in 2021 dollars and costs and utilities have been calculated at a discount rate of 3% per year. Results: CRCT and RM obtained 6.53 QALYs and 5.15 QALYs, respectively, and the ICER of CRCT vs. RM was $358,895.2/QALYs. Parameters having the greatest impact on the robustness of the model were the drug cost of copanlisib and the utility value of the progression-free survival (PFS) state. When the WTP threshold was $150,000, the probability of CRCT and RM being the most cost effective was 0.4% and 99.6% respectively. Conclusions: From a US payer perspective, CRCT is not cost-effective in treating relapsed iNHL at current prices compared to RM. But given its positive clinical efficacy, appropriate price discounts or assistance programs should be considered to make CRCT more affordable to patients with relapsed iNHL.

Cost-effectiveness of artificial intelligence screening for diabetic retinopathy in rural China.

BACKGROUND: Diabetic retinopathy (DR) has become a leading cause of global blindness as a microvascular complication of diabetes. Regular screening of diabetic retinopathy is strongly recommended for people with diabetes so that timely treatment can be provided to reduce the incidence of visual impairment. However, DR screening is not well carried out due to lack of eye care facilities, especially in the rural areas of China. Artificial intelligence (AI) based DR screening has emerged as a novel strategy and show promising diagnostic performance in sensitivity and specificity, relieving the pressure of the shortage of facilities and ophthalmologists because of its quick and accurate diagnosis. In this study, we estimated the cost-effectiveness of AI screening for DR in rural China based on Markov model, providing evidence for extending use of AI screening for DR. METHODS: We estimated the cost-effectiveness of AI screening and compared it with ophthalmologist screening in which fundus images are evaluated by ophthalmologists. We developed a Markov model-based hybrid decision tree to analyze the costs, effectiveness and incremental cost-effectiveness ratio (ICER) of AI screening strategies relative to no screening strategies and ophthalmologist screening strategies (dominated) over 35 years (mean life expectancy of diabetes patients in rural China). The analysis was conducted from the health system perspective (included direct medical costs) and societal perspective (included medical and nonmedical costs). Effectiveness was analyzed with quality-adjusted life years (QALYs). The robustness of results was estimated by performing one-way sensitivity analysis and probabilistic analysis. RESULTS: From the health system perspective, AI screening and ophthalmologist screening had incremental costs of $180.19 and $215.05 but more quality-adjusted life years (QALYs) compared with no screening. AI screening had an ICER of $1,107.63. From the societal perspective which considers all direct and indirect costs, AI screening had an ICER of $10,347.12 compared with no screening, below the cost-effective threshold (1-3 times per capita GDP of Chinese in 2019). CONCLUSIONS: Our analysis demonstrates that AI-based screening is more cost-effective compared with conventional ophthalmologist screening and holds great promise to be an alternative approach for DR screening in the rural area of China.

Cost-Effectiveness of Conbercept vs. Ranibizumab for Age-Related Macular Degeneration, Diabetic Macular Edema, and Pathological Myopia: Population-Based Cohort Study and Markov Model.

Background: With the advent of aging society of China, fundus diseases related to pathological neovascularization, including age-related macular degeneration (AMD), diabetic macular edema (DME), and pathological myopia (PM), have become an increasingly serious medical and health problems. As effective drugs of the treatment, conbercept and ranibizumab have been commonly used and covered by the national basic medical insurance in China. However, the pharmacoeconomic evaluation of conbercept vs. ranibizumab for DME and PM remains lacking. This study would assess the cost-effectiveness of conbercept and ranibizumab for the treatment of AMD, DME, and PM from the perspective of Chinese payers. Methods: A Markov chain model was constructed based on the visual conditions of the patient indicated by the number of letters in best corrected visual acuity (BCVA). We conducted models based on real-world scenario to calculate the cost per the quality-adjusted life-year (QALY) gained. A 1-year cycle length and a 10-year simulation treatment were applied and the number of injections of conbercept and ranibizumab was assumed to the average number within 10 years. Transition probabilities, costs, utility data, and other parameters were obtained from literature searches. A 3.5% discounting rate was applied for both the costs and utilities. Results: The incremental cost-effectiveness ratios (ICERs) were more favorable for conbercept than ranibizumab in treatment of AMD, DME, and PM, with associated ICER of 66,669 renminbi (RMB), -258,813 RMB, and -373,185 RMB per QALY gained. Compared with ranibizumab, the incremental effectiveness of conbercept in treatment of AMD, DME, and PM was -0.665 QALYs, 0.215 QALYs, and 0.029 QALYs, respectively. The sensitivity analysis showed the same findings, although the ICER is sensitive to the costs of this program. Conclusion: Under the current Chinese healthcare setting, conbercept is suitable and cost-effective in treatment of AMD, DME, and PM compared with ranibizumab.

Cost-effectiveness analysis of ribociclib plus fulvestrant for hormone receptor-positive/human EGF receptor 2-negative breast cancer.

Aim: To evaluate the cost-effectiveness of ribociclib plus fulvestrant versus fulvestrant in hormone receptor-positive/human EGF receptor 2-negative advanced breast cancer. Materials & methods: A three-state Markov model was developed to evaluate the costs and effectiveness over 10 years. Direct costs and utility values were obtained from previously published studies. We calculated incremental cost-effectiveness ratio to evaluate the cost-effectiveness at a willingness-to-pay threshold of $150,000 per additional quality-adjusted life year. Results: The incremental cost-effectiveness ratio was $1,073,526 per quality-adjusted life year of ribociclib plus fulvestrant versus fulvestrant. Conclusions: Ribociclib plus fulvestrant is not cost-effective versus fulvestrant in the treatment of advanced hormone receptor-positive/human EGF receptor 2-negative breast cancer. When ribociclib is at 10% of the full price, ribociclib plus fulvestrant could be cost-effective.

Cost-Effectiveness Analysis of Adding Daratumumab to Bortezomib, Melphalan, and Prednisone for Untreated Multiple Myeloma.

Background: To evaluate the cost-effectiveness of adding daratumumab to bortezomib, melphalan, and prednisone for transplant-ineligible newly diagnosed multiple myeloma patients. Methods: A three-state Markov model was developed from the perspective of US payers to simulate the disease development of patient's life time for daratumumab plus bortezomib, melphalan, and prednisone (D-VMP) and bortezomib, melphalan, and prednisone (VMP) regimens. The primary outputs were total costs, expected life-years (LYs), quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs). Results: The base case results showed that adding daratumumab to VMP provided an additional 3.00 Lys or 2.03 QALYs, at a cost of $262,526 per LY or $388,364 per QALY. Sensitivity analysis indicated that the results were most sensitive to utility of progression disease of D-VMP regimens, but no matter how these parameters changed, ICERs remained higher than $150,000 per QALY. Conclusion: In the case that the upper limit of willingness to pay threshold was $150,000 per QALY from the perspective of US payers, D-VMP was not a cost-effective regimen compared to VMP.

Cost-effectiveness of pembrolizumab plus axitinib as first-line therapy for advanced renal cell carcinoma.

Aim: To evaluate the cost-effectiveness of first-line treatments for advanced renal cell carcinoma with pembrolizumab plus axitinib compared with sunitinib from the US payer perspective. Patients & methods: A Markov model was developed for this purpose. The clinical data were obtained from the KEYNOTE-426 trial. Utility values and direct costs related to the treatments were gathered from the published studies. Results: The incremental cost-effectiveness ratios of pembrolizumab plus axitinib versus sunitinib was $249,704 per quality-adjusted life year, which was higher than a willingness-to-pay threshold of $150,000 per quality-adjusted life year. Conclusion: Pembrolizumab plus axitinib was not considered to be cost-effective versus sunitinib as a first-line treatment for patients with advanced renal cell carcinoma from the US payer perspective.

Cost-effectiveness analysis of nivolumab plus ipilimumab versus chemotherapy as first-line treatment in advanced NSCLC.

Aim: To evaluate the cost-effectiveness of nivolumab plus ipilimumab (NI) in the first-line treatment of patients with advanced non-small-cell lung cancer from a US-payer perspective. Materials & methods: We developed a Markov model to evaluate the cost and effectiveness of NI versus chemotherapy as first-line treatment of NSCLC. Quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs) were estimated. Results: NI provided an additional 0.715 QALYs compared with chemotherapy in all population. The corresponding ICER of NI was $180,307 per QALY gained. However, the ICER decreased to $143,434 per QALY in the programmed death ligand 1 expression level <1% population. Conclusion: From a US-payer perspective, NI is estimated to be cost-effective in the first-line setting for advanced NSCLC patients with programmed death ligand 1 expression level <1%.

Cost-effectiveness analysis of atezolizumab plus nab-paclitaxel for untreated metastatic triple-negative breast cancer.

Aim: To evaluate the cost-effectiveness of atezolizumab plus nab-paclitaxel (ANP) in the first-line treatment of metastatic triple-negative breast cancer (TNBC). Materials & methods: We developed a Markov model to evaluate the cost and effectiveness of ANP versus nab-paclitaxel in the first-line treatment of metastatic TNBC. Lifetime costs, life-years (LYs) and quality-adjusted LYs (QALYs) were estimated. Results: ANP provided an additional 0.16 QALYs (0.24 LYs) compared with nab-paclitaxel in intention-to-treat population. The corresponding incremental cost-effectiveness ratio was $786,131 per QALY gained. However, the incremental cost-effectiveness ratio decreased to $361,218 per QALY gained in the PD-L1 positive subgroup analysis. Conclusion: From the perspective of a US-payer, ANP is estimated not to be cost-effective in the first-line treatment of metastatic TNBC.

A Cost-Effectiveness Analysis: First-Line Avelumab Plus Axitinib Versus Sunitinib for Advanced Renal-Cell Carcinoma.

BACKGROUND: Compared with the standard of care with sunitinib, avelumab plus axitinib can increase progression-free survival in the first-line of advanced renal cell carcinoma (RCC), but the economic effect of the treatment is unknown. The purpose of the research was to evaluate the cost-effectiveness of the avelumab plus axitinib versus sunitinib in first-line treatment for advanced RCC from the US payer perspective. METHODS: A Markov model was developed to evaluate the economic and health outcomes of avelumab plus axitinib vs sunitinib in the first-line setting for advanced RCC. The clinical data were obtained from the JAVELIN Renal 101 Clinical Trials. Deterministic and probabilistic sensitivity analyses were performed to assess uncertainty in the model. Health outcomes were measured in quality-adjusted life-years (QALYs). RESULTS: The incremental cost-effectiveness ratio (ICER) of avelumab plus axitinib compared with sunitinib was $565,232 per QALY, the costs were $884,626 and $669,838, QALYs were 3.67 and 3.29, respectively. Sensitivity analysis demonstrated that differences in utilities in PFS and after progression were the most influential factors within the model. When avelumab was at 30% of the full price or axitinib was at 40% of the full price, avelumab and axitinib were approved to be cost-effective if the WTP threshold was $150,000 per QALY. The subgroup analysis showed the ICER of avelumab plus axitinib compared with sunitinib for the patients with PD-L1-positive tumors was $588,105. CONCLUSION: Avelumab plus axitinib in the first-line treatment was not cost-effective in comparison with sunitinib when the threshold of willingness to pay (WTP) was $150,000 per QALY.

Cost-effectiveness analysis of pembrolizumab plus chemotherapy with PD-L1 test for the first-line treatment of NSCLC.

BACKGROUND: Pembrolizumab (Pembro) in combination with chemotherapy has been approved for the treatment of pretreated advanced NSCLC in the United States and China for its significant efficacy. However, the cost-effectiveness is unknown considering Pembro's high price. The impact of programmed death ligand 1 (PD-L1) test on the cost-effectiveness is also unknown. The current study assessed the cost-effectiveness of combination therapy for nonsquamous NSCLC from the United States and China public payers' perspective. MATERIALS AND METHODS: A literature-based Markov model was conducted using KEYNOTE-189 trial data to compare cost and quality-adjusted life years (QALYs) of three treatment strategies for nonsquamous NSCLC: Pembro-chemotherapy combination and chemotherapy strategy without PD-L1 test, and treatment strategy according to their PD-L1 status. RESULTS: In base case analysis, the combination strategy generated an additional 0.78 QALYs and 0.59 QALYs over chemotherapy in the United States and China respectively, resulting in an ICER of $132 392/QALY in the United States and $92 533/QALY in China. In the PD-L1 ≥1% base case, the ICERs were $77 754/QALY and $56 768/QALY respectively in the United States and China for PD-L1 test strategy. In the PD-L1 ≥50% base case, the ICERs were $44 731/QALY and $34 388/QALY respectively in the United States and China for PD-L1 test strategy. Lowering Pembro price can also partly decrease the ICERs. CONCLUSION: Compared with chemotherapy, the combination strategy is not cost-effective for the treatment of NSCLC in the American and Chinese health care system at WTP threshold of $100 000/QALY for the United States and $27 351/QALY for China. Using PD-L1 test for patient selection and price reduction could improve the cost-effective probabilities of immunotherapy for nonsquamous NSCLC.

Cost-effectiveness analysis of gemcitabine plus cisplatin versus fluorouracil plus cisplatin for first-line treatment of recurrent or metastatic nasopharyngeal carcinoma.

PURPOSE: Compared with conventional fluorouracil plus cisplatin (FP) regimen, gemcitabine plus cisplatin (GP) can prolong survival in patients with recurrent or metastatic nasopharyngeal carcinoma, but the economic impact of this practice remains unknown. It's significant to evaluate its values by taking both efficacy and cost into consideration. METHODS: We developed a Markov model with 10 years horizon to compare the cost-effectiveness of GP and FP regimen. Clinical data came from a multicentre, randomised, open-label, phase 3 trial. Direct costs related to the treatment were estimated from the perspective of the Chinese healthcare system. Utility values were gathered from published study. Sensitivity analysis was conducted to confirm the robustness of the model. RESULTS: The total cost of FP regimen was $12,587 and yielded 0.964 QALYs, while the total cost of GP regimen was $17,920 and yielded 1.685 QALYs. The ICER of GP regimen versus FP regimen was $7,386 which was far less than the willingness-to-pay threshold ($26,508) in China. CONCLUSION: From the perspective of Chinese healthcare system, GP regimen with superior efficacy was proved to be more cost-effective than the traditional FP regimen. It is likely that GP regimen may be recommended as the primarily first-line treatment option for recurrent or metastatic nasopharyngeal carcinoma.

Health-related quality of life among rural men and women with hypertension: assessment by the EQ-5D-5L in Jiangsu, China.

PURPOSE: Hypertension is a major global public health problem, including rural China. However, studies examining health-related quality of life (HRQoL) for patients with hypertension have been mostly conducted in urban populations. This study aimed to use the EuroQol five-dimensional-five-level (EQ-5D-5L) and its recently developed Chinese value set to analyze HRQoL and its influencing factors among hypertensive population in rural China. METHODS: This is a cross-sectional population-based survey. Standard interview of participants was conducted from July to September 2016 in Donghai County's 334 villages of Jiangsu Province, China. Data collection included the EQ-5D-5L, along with sociodemographic characteristics and disease-related factors such as duration of hypertension, antihypertensive treatment and comorbid conditions. The Tobit regression model was employed to analyze potential influencing factors on HRQoL. RESULTS: A total of 16,596 adults (18 years and older) with hypertension participated in this study. 62.4% were women. The mean utility score was 0.85 (standard deviation [SD] = 0.23). The proportion of participants reporting pain/discomfort problems was highest, while least patients reported problems in self-care dimension. Females, elderly, illiterate patients, ex-smokers and patients with longer duration of hypertension or comorbidities scored lower on HRQoL than others. Stroke, heart failure and coronary heart disease were associated with a larger negative impact on HRQoL among all comorbidities. CONCLUSIONS: The HRQoL was lower in this rural hypertensive population than previously reported urban counterparts. To improve the HRQoL of hypertensive patients in rural areas, it is important to control hypertension and prevent its associated co-morbidities. More attention needs to be directed to elderly female patients with less education who scored much lower HRQoL than their male counterparts.