Barriers and facilitators to guideline for the management of pediatric off-label use of drugs in China: a qualitative descriptive study.

BACKGROUND: Despite being a global public health concern, there is a research gap in analyzing implementation strategies for managing off-label drug use in children. This study aims to understand professional health managers' perspectives on implementing the Guideline in hospitals and determine the Guideline's implementation facilitators and barriers. METHODS: Pediatric directors, pharmacy directors, and medical department directors from secondary and tertiary hospitals across the country were recruited for online interviews. The interviews were performed between June 27 and August 25, 2022. The Consolidated Framework for Implementation Research (CFIR) was adopted for data collection, data analysis, and findings interpretation to implement interventions across healthcare settings. RESULTS: Individual interviews were conducted with 28 healthcare professionals from all over the Chinese mainland. Key stakeholders in implementing the Guideline for the Management of Pediatric Off-Label Use of Drugs in China (2021) were interviewed to identify 57 influencing factors, including 27 facilitators, 29 barriers, and one neutral factor, based on the CFIR framework. The study revealed the complexity of the factors influencing managing children's off-label medication use. A lack of policy incentives was the key obstacle in external settings. The communication barrier between pharmacists and physicians was the most critical internal barrier. CONCLUSION: To our knowledge, this study significantly reduces the implementation gap in managing children's off-label drug use. We provided a reference for the standardized management of children's off-label use of drugs.

Fiscal spending and green economic growth: evidence from highly polluting Asian economic.

The study examines the link between fiscal expenditure and green economic development in heavily polluting Asian nations. The conventional wisdom that environmental sustainability and economic progress are incompatible is contested in this research. The study found a strong positive link between green fiscal policy and long-term economic development, offering empirical support for using fiscal policy to promote green growth. With an emphasis on high-pollution sectors like manufacturing and energy, the study used sophisticated econometric approaches, such as panel data regression and Granger causality tests, to evaluate the data from a few Asian nations. The findings supported the idea that increased government investment in environmental protection programs and eco-friendly technology positively impacted green economic development. The results also highlight how governmental actions influence economic trajectories and aid in the shift to a sustainable economy. The report emphasizes the need for cross-level and cross-sector policy coordination, arguing that achieving significant green economic development with comprehensive, well-integrated green fiscal policies would be more straightforward. The study also highlights the need for international collaboration and assistance while acknowledging possible restrictions on fiscal expenditure in emerging nations. Policymakers, environmental economists, and other stakeholders who want to transition to a green economy while pursuing economic growth and development should take these conclusions seriously. The linkages among fiscal investment and green economic development in heavily polluting Asian nations are substantially established in this work, but it also highlights the need for additional study. Future research could benefit from examining these patterns in a broader range of situations and over extended periods, given the intricate interaction of variables driving fiscal policy and green growth.

Nivolumab plus chemotherapy versus chemotherapy alone as first-line treatment for advanced gastric, gastroesophageal junction, and esophageal adenocarcinoma: a cost-effectiveness analysis.

BACKGROUND: The aim of the study was to evaluate the cost-effectiveness of nivolumab plus chemotherapy as first-line treatment for patients with advanced gastric, gastroesophageal junction (GEJ), or esophageal adenocarcinoma from the perspective of Chinese and US society. METHODS: To conduct the analysis, a state-transitioned Markov model, which included three mutually exclusive health states (progression-free survival (PFS), progressive disease (PD), and death), was developed. Cycle length was set at 3 weeks and lifetime horizon was set at 10 years. Costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER) were calculated in the analysis. Willingness-to-pay (WTP) thresholds in the model were set at $37,653.00/QALY in China and $100,000.00/QALY in the US, respectively. Meanwhile, one-way sensitivity analyses and probabilistic sensitivity analyses were conducted to investigate the robustness of the model. RESULTS: Over a lifetime horizon, the ICERs of nivolumab plus chemotherapy versus chemotherapy alone were $430,185.04/QALY and $944,089.78/QALY in China and the US, respectively. Cost of nivolumab and utility for the PFS state had the most significant impact on ICERs both in the US and China based on the results of the one-way sensitivity analyses. In the probabilistic sensitivity analyses, the proportions of nivolumab plus chemotherapy being cost-effective compared with chemotherapy alone were 0%. CONCLUSIONS: In conclusion, nivolumab plus chemotherapy is unlikely to be a cost-effective treatment option compared with chemotherapy alone in the first-line setting of advanced gastric, GEJ, or esophageal adenocarcinoma.

Cost-effectiveness analysis of toripalimab plus chemotherapy as the first-line treatment in patients with advanced non-small cell lung cancer (NSCLC) without EGFR or ALK driver mutations from the Chinese perspective.

Objectives: The results of a CHOICE-1 study demonstrated the superior efficacy of toripalimab (anti-PD-1 antibody) plus chemotherapy for patients with advanced non-small cell lung cancer (NSCLC), with a manageable safety profile. This study was performed to evaluate the economic value of this treatment for this patient population from the Chinese payer's perspective. Materials and methods: Basic data were derived from the CHOICE-1 study. Markov models were developed to simulate the process of advanced NSCLC, including the progression-free survival (PFS), progressive disease (PD), and death in intention-to-treat (ITT) populations, as well as patients with squamous or non-squamous NSCLC. The cost was obtained from the local institution, and the value of utilities referred to previous studies. Monte Carlo simulations were performed to depict the probabilistic scatter plots of the incremental cost-effectiveness ratio (ICER) and acceptability curves, aiming to address the uncertainty of model inputs. Results: Compared with standard chemotherapy, toripalimab plus chemotherapy yields an ICER of $21,563 per quality-adjusted life year (QALY) in the ITT population. For patients with squamous NSCLC, comparing the combined therapy with chemotherapy led to an ICER of $18,369 per QALY, while the ICER was $24,754 per QALY in patients with non-squamous NSCLC. With the threshold of willingness to pay we set ($37,653 per QALY), toripalimab plus chemotherapy was cost-effective in these patient populations. Conclusion: For patients with advanced NSCLC, toripalimab plus chemotherapy was an optimal choice as first-line treatment, regardless of histology.

Assessment of knowledge, cultural beliefs, and behavior regarding medication safety among residents in Harbin, China.

BACKGROUND: Medication misuse or overuse is significantly associated with poor health outcomes. Information regarding the knowledge, cultural beliefs, and behavior about medication safety in the general population is important. AIM: To conduct a survey on medication habits and explored the potential factors impacting medication safety. METHODS: The current survey included adults from 18 districts and counties in Harbin, China. A questionnaire on medication safety was designed based on knowledge, cultural beliefs, and behavior. Both univariate and multivariate analyses were used to explore the factors that impacted medication safety. RESULTS: A total of 394 respondents completed the questionnaires on medication safety. The mean scores for knowledge, cultural beliefs, and behavior about medication safety were 59.41 ± 19.33, 40.66 ± 9.24, and 60.97 ± 13.69, respectively. The medication knowledge score was affected by age (P = 0.044), education (P < 0.001), and working status (P = 0.015). Moreover, the cultural beliefs score was significantly affected by education (P < 0.001). Finally, education (P = 0.003) and working status (P = 0.011) significantly affected the behavior score. CONCLUSION: The knowledge, cultural beliefs, and behavior about medication safety among the general population was moderate. Health education should be provisioned for the elderly, individuals with a low education level, and the unemployed to improve medication safety in Harbin, China.

Effect of Iron Deposition on Native T1 Mapping and Blood Oxygen Level Dependent for the Assessment of Liver Fibrosis in Rabbits With Carbon Tetrachloride Intoxication.

RATIONALE AND OBJECTIVES: This study aimed to explore the effect of iron deposition on native T1 mapping and blood oxygen level-dependent (BOLD) imaging in detecting liver fibrosis (LF) in a rabbit model. MATERIALS AND METHODS: An LF group (n = 100) was established by subcutaneously injecting 50% carbon tetrachloride (CCl4) oil solution, and 20 normal rabbits composed a control group. Native T1 mapping and BOLD were performed, and the T1native and R2* quantitative parameters were analyzed by receiver operating characteristic (ROC) and multiple logistic regression analyses, with histopathological results and liver iron content (LIC) serving as reference standards. RESULTS: In total, 18, 17, 16, 18, and 15 rabbits were histopathologically diagnosed with LF stages F0, F1, F2, F3, and F4, respectively. T1native (r = 0.47), R2* (r = 0.75) and LIC (r = 0.61) increased with LF stage progression (p < 0.001). Compared to T1native values, R2* performed better in diagnosing the LF stage, especially for distinguishing F1-F2 from F3-F4 (AUC = 0.66 vs. 0.91, p = 0.01). Combined with the LIC, both T1native and R2* showed improved diagnostic value in comparison to the individual imaging techniques, particularly for diagnosing F0 vs. F1-F2 and F0 vs. F1-F4, with AUC values of 0.90 vs. 0.70 (p = 0.01) and 0.93 vs. 0.77 (p = 0.01) for T1native + LIC vs. LIC, respectively. CONCLUSION: BOLD imaging performed better than native T1 mapping in predicting and diagnosing LF stage progression. The decrease in diagnostic accuracy caused by the deposition of liver iron is a potential pitfall in the assessment of LF with BOLD imaging and native T1 mapping.

Camrelizumab in patients with advanced non-squamous non-small cell lung cancer: a cost-effective analysis in China.

OBJECTIVE: Camrelizumab is a selective, humanised, high-affinity IgG4 kappa monoclonal antibody against programmed cell death 1 that shows effective antitumour activity with acceptable toxicity in multiple tumour types. The CameL trial demonstrated that camrelizumab plus chemotherapy (CC) significantly prolonged the median progression-free survival and median overall survival versus chemotherapy alone (CA) in patients with advanced non-squamous non-small cell lung cancer (NSCLC). Our study was conducted to investigate the cost-effectiveness of the two strategies in chemotherapy-naive patients with advanced non-squamous NSCLC. DESIGN, SETTING AND PARTICIPANTS: A Markov simulation model was generated based on the CameL trial. The two simulated treatments included CC and CA. PRIMARY AND SECONDARY OUTCOME MEASURES: Utility was derived from published literature, and costs were calculated based on those at our hospital in Chengdu, China. Incremental cost-effectiveness ratios (ICERs) were calculated to compare the cost-effectiveness of the two treatment arms. RESULTS: In the overall population, the total costs were $27 223.40 and $13 740.10 for CC and CA treatment, respectively. The CC treatment produced 1.37 quality-adjusted life years (QALYs), and the CA treatment produced 1.17 QALYs. Hence, patients who were in the CC group spent an additional $13 483.30 and generated an increase of 0.20 QALYs, resulting in an ICER of $67 416.50 per QALY. CONCLUSIONS: For chemotherapy-naive patients with advanced non-squamous NSCLC, CC is not considered a cost-effective treatment versus CA in China when considering a willingness-to-pay threshold of $31 500 per QALY. TRIAL REGISTRATION NUMBER: NCT03134872.

Atezolizumab with chemotherapy in first-line treatment for metastatic urothelial cancer: a cost-effectiveness analysis.

Purpose: To evaluate the cost-effectiveness of atezolizumab plus chemotherapy as first-line treatment for metastatic urothelial cancer (mUC). Materials & methods: A Markov model was established for the analysis. Parametric survival models were used to fit to progression-free survival and overall survival data in the IMvigor130 study. A series of one-way and probabilistic sensitivity analyses were performed to test the robustness of the model. Results: The incremental cost-effectiveness ratios for atezolizumab plus chemotherapy versus chemotherapy alone were US$475,633.17 and $207,488.17 per quality-adjusted life year in the USA and China, respectively. Utility for the progression-free survival and progressive disease states, the cost of atezolizumab had the most significant impact on the incremental cost-effectiveness ratio. Conclusion: Atezolizumab plus chemotherapy is not a cost-effective treatment option as a first-line treatment for metastatic urothelial cancer.

Cost-Effectiveness Analysis of Abemaciclib plus Fulvestrant versus Placebo plus Fulvestrant in Patients with Hormone Receptor-Positive, ERBB2-Negative Breast Cancer.

Purpose: Abemaciclib is a selective and potent small-molecule inhibitor of cyclin-dependent kinase 4 and 6 (CDK4 and CDK6) which is administered orally. Compared to placebo plus fulvestrant (PF), abemaciclib plus fulvestrant (AF) significantly improved progression-free survival (PFS) and overall survival (OS). However, an economic evaluation of these two treatments is currently lacking. The purpose of this article was to evaluate the cost-effectiveness of the two treatments for HR*, HER2- advanced breast cancer (ABC) in the USA. Methods: A Markov simulation model was constructed using data from a published clinical trial (MONARCH 2). The two simulated treatment strategies included AF or PF. Costs were obtained from the clinical trials and the website, and utility was derived from the published literature. Incremental cost-effectiveness ratios (ICERs) were calculated to compare the two treatment strategies. Results: The total costs were USD 400,377.43 and USD 89,937.77 for AF and PF treatment, respectively. The AF treatment produced 2.09 long-term quality-adjusted life years (QALYs), and the PF treatment produced 1.08 QALYs. Hence, patients who received AF treatment spent an additional USD 310,439.66 and generated an increase of 1.01 QALYs, resulting in an ICER of USD 307,366 per QALY. At current prices, AF was not cost-effective assuming a willingness-to-pay threshold of USD 150,000 per QALY gained. Conclusion: Despite significant gains in PFS over AF, it is not a cost-effective treatment for HR*, HER2- ABC in the USA at current drug prices.

Lenvatinib Plus Pembrolizumab vs. Chemotherapy in Pretreated Patients With Advanced Endometrial Cancer: A Cost-Effectiveness Analysis.

Background: In the international, randomized, open-label, phase 3 study 309-KEYNOTE-775 trial, lenvatinib plus pembrolizumab (LP) showed improved progression-free survival (PFS) and overall survival (OS) compared with chemotherapy in pretreated patients with advanced endometrial cancer. This study aimed to investigate whether LP is cost-effective compared with chemotherapy. Materials and Methods: The clinical data for this model was derived from the 309-KEYNOTE-775 trial. Costs and utilities were either derived from the standard fee database or extracted from previously published literature. A three-state Markov model was developed to simulate the disease process of patients with advanced endometrial cancer. One-way sensitivity analyses were conducted to investigate the impact of variables in the analysis model. Probabilistic sensitivity analysis was performed based on 10,000 Monte-Carlo simulations. A subgroup analysis was performed to test whether LP is cost-effective in patients with mismatch repair-proficient (pMMR) disease. Results: Lenvatinib plus pembrolizumab provided an incremental 0.64 quality-adjusted life years (QALYs) with an incremental cost of $241,278.18, compared with chemotherapy, resulting in the incremental cost-effectiveness ratio (ICER) of $378,251.44/QALY, which exceeded the willingness to pay (WTP) threshold. While in the pMMR subgroup, the ICER increased to $413,256.68/QALY. The variance of the utility of PFS state, the cost of LP, and the utility of the progressive disease state were the most influential factors in the sensitivity analysis. Conclusion: Under the current WTP threshold, LP is not cost-effective compared with chemotherapy in pretreated patients with advanced endometrial cancer.

Margetuximab Versus Trastuzumab in Patients With Advanced Breast Cancer: A Cost-effectiveness Analysis.

BACKGROUND: In the international, randomized, open-label, phase III study SOPHIA trial, margetuximab plus chemotherapy showed improved progression-free survival (PFS), and overall survival (OS) compared with trastuzumab plus chemotherapy. This study aimed to investigate whether margetuximab plus chemotherapy is cost-effective compared with trastuzumab plus chemotherapy in pretreated patients with ERBB2-positive advanced breast cancer. MATERIALS AND METHODS: The clinical data for this model was derived from the SOPHIA trial. Costs and utility were either derived from the standard fee database or extracted from previously published literature. A three-state Markov model was developed to simulate the disease process of patients with advanced breast cancer. One-way sensitivity analyses were conducted to investigate the impact of variables in the analysis model. Probabilistic sensitivity analysis was performed based on 10,000 Monte-Carlo simulations. A subgroup analysis was performed to test whether margetuximab is cost-effective in CD16A-158F allele carriers. RESULTS: Margetuximab plus chemotherapy provided an incremental 0.04 QALYs with an incremental cost of $66,109.78, compared with the trastuzumab plus chemotherapy, resulting in the incremental cost-effectiveness ratio (ICER) of $1,486,442.35/QALY, which exceeded the willingness to pay (WTP) threshold. While in the CD16A-158F allele carriers subgroup, the ICER decreased to $592,669.73/QALY. The variance of the utility of PFS state, costs of margetuximab, and utility of progressive disease state were the most influential factors in the sensitivity analysis. CONCLUSION: Under current WTP threshold, margetuximab plus chemotherapy is not cost-effective compared with trastuzumab plus chemotherapy in pretreated patients with ERBB2-positive advanced breast cancer. Selecting CD16A-158F allele carriers might be a considerable option to optimize the cost-effectiveness of margetuximab.

Cost-effectiveness of enfortumab vedotin in previously treated advanced urothelial carcinoma.

BACKGROUND: Antibody-drug conjugates have recently been introduced as a treatment for advanced urothelial carcinoma. The EV-301 study demonstrated that enfortumab vedotin (EV) improved overall survival compared with conventional chemotherapy. To assess the cost-effectiveness of EV for the treatment of advanced urothelial carcinoma (UC) from a payer perspective in middle- and high-income countries. METHODS: A decision analysis model was developed to assess the efficacy and economic viability of EV as a subsequent-line treatment following disease progression in patients with advanced urothelial carcinoma already treated with PD-1 or PD-L1 inhibitors. Clinical and utility values were obtained from the published literature and available databases. Cost data were obtained from payer perspectives in the United States, United Kingdom, and China. Quality-adjusted life-years (QALYs) were used to measure health outcomes, and incremental cost-effectiveness ratios (ICERs) used to evaluate cost-effectiveness in comparison to willingness-to-pay in the United States, United Kingdom, and China. One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess the robustness of the model. RESULTS: Compared with chemotherapy, EV increased the benefit by 0.16-0.17 QALYs, resulting in ICERs of $2,168,746.71, $2,164,494.38, and $1,775,576.56 per QALY in the United States, United Kingdom, and China, respectively. One-way sensitivity analysis indicated that the largest effect on outcome was the utility value for progression-free survival. Probabilistic sensitivity analysis demonstrated that the probability of EV being cost-effective was 0%. CONCLUSIONS: EV provides an additional health benefit over chemotherapy for patients with advanced urothelial carcinoma but is not cost-effective from a payer perspective in the United States, United Kingdom, or China.

Clinical manifestations, prognosis, and treat-to-target assessment of pediatric lupus nephritis.

BACKGROUND: Pediatric lupus nephritis (pLN) is one of the most refractory secondary kidney diseases in childhood. The treat-to-target (T2T) strategy has become the standard treatment for systemic lupus erythematosus (SLE). This study reviewed clinical features, overall remission status, and factors affecting prognosis, to guide pLN management according to T2T strategy. METHODS: This single-center retrospective study studied 220 children diagnosed with LN from January 2012 to December 2018, with > 6-month follow-up data on 173 and complete data on 137 patients. Primary outcome was treatment failure (deterioration or no response) at the latest follow-up. RESULTS: The most common pLN manifestation was proteinuria (81.36%). Females presented more often with rash (P<0.001) and alopecia (P=0.026) than males. Class IV LN (33.33%) was the most common grade on kidney biopsy. Median follow-up was 27.20 months (IQR, 15.78-44.45 months). One-, 3-, and 5-year cumulative overall survival rates were 93.5%, 87.8%, and 86.5%, respectively. The 5-year cumulative kidney survival rate was 97.1%. Regarding initial therapy, efficacy of corticosteroids combined with immunosuppressive agents was significantly better than corticosteroids alone (P=0.010). Factors with P<0.05 in univariate analysis, including hypoalbuminemia, higher SCr at diagnosis, lower eGFR at diagnosis, anti-dsDNA positivity, heavy proteinuria, hypertension, nervous-system involvement, treatment non-compliance, and SLEDAI-2K score, were used for logistic regression analysis. Logistic regression analysis showed hypertension (OR=0.845, P=0.011), nervous-system involvement (OR=4.240, P=0.005), treatment non-compliance (OR=6.433, P=0.001), and lower estimated glomerular filtration rate at diagnosis (OR=1.020, P=0.021) affected prognosis. At end of follow-up, 34.31% achieved varying levels of remission, and 8.76% were in low disease activity state (LDAS). CONCLUSIONS: pLN usually presented with proteinuria, and class IV LN was the dominant pathology. Hypertension, nervous-system involvement, treatment non-compliance, and lower eGFR at diagnosis were independent risk factors for poor prognosis of kidney outcomes. Compared with renal remission rate and cumulative overall survival rate, the proportion of targets achieved was not ideal, suggesting T2T strategy should be used to guide pLN management. A higher resolution version of the Graphical abstract is available as Supplementary information.

Cost-Effectiveness Analysis of Fourth- or Further-Line Ripretinib in Advanced Gastrointestinal Stromal Tumors.

BACKGROUND: The INVICTUS trial assessed the efficacy and safety of ripretinib compared with placebo in the management of advanced gastrointestinal stromal tumors. METHOD: We used a Markov model with three health states: progression-free disease, progression disease and death. We parameterized the model from time-to-event data (progression-free survival, overall survival) of ripretinib and placebo arms in the INVICTUS trial and extrapolated to a patient's lifetime horizon. Estimates of health state utilities and costs were based on clinical trial data and the published literature. The outcomes of this model were measured in quality-adjusted life-years (QALYs), costs, and incremental cost-effectiveness ratios (ICERs). Uncertainty was tested via univariate and probabilistic sensitivity analyses. RESULTS: The base-case model projected improved outcomes (by 0.29 QALYs) and additional costs (by $70,251) and yielded an ICER of $244,010/QALY gained for ripretinib versus placebo. The results were most sensitive to progression rates, the price of ripretinib, and health state utilities. The ICER was most sensitive to overall survival. When overall survival in the placebo group was lower, the ICER dropped to $127,399/QALY. The ICER dropped to $150,000/QALY when the monthly cost of ripretinib decreased to $14,057. Probabilistic sensitivity analyses revealed that ripretinib was the cost-effective therapy in 41.1% of simulations at the willingness-to-pay (WTP) threshold of $150,000. CONCLUSION: As the fourth- or further-line therapy in advanced gastrointestinal stromal tumors, ripretinib is not cost-effective in the US. Ripretinib would achieve its cost-effectiveness with a price discount of 56% given the present effectiveness.

Comparison of Primary and Secondary Prophylaxis Using PEGylated Recombinant Human Granulocyte-Stimulating Factor as a Cost-Effective Measure in Malignant Neoplasms: A Multicenter Retrospective Study.

Purpose: The aim of the study was to evaluate the cost-effectiveness of PEGylated recombinant human granulocyte-stimulating factor (PEG-rhG-CSF) as a means of achieving primary and secondary prophylaxis against chemotherapy-induced neutropenia cancer cases. Methods: Individuals who underwent PEG-rhG-CSF therapeutics were monitored for 12 months, together with thorough examination of individual medical records for extracting medical care costs. Both prophylaxis-based therapeutic options (primary/secondary) were scrutinized for cost-effectiveness, using a decision-making analysis model which derived the perspective of Chinese payers. One-way and probabilistic sensitivity analyses were used to assess the robustness of the model. Results: In summary, 130 clinical cases treated using PEG-rhG-CSF prophylaxis were included in this study: 51 within the primary prophylaxis (PP) group and 79 within the secondary prophylaxis (SP) group. Compared with SP, PP-based PEG-rhG-CSF successfully contributed to a 14.3% reduction in febrile neutropenia. In general, PP was estimated to reduce costs by $4,701.81 in comparison to SP, with a gain of 0.02 quality-adjusted life years (QALYs). Equivalent results were found in differing febrile neutropenia (FN) risk subgroups. Sensitivity analyses found the model outputs to be most affected for the average time of hospitalization and for the cost of FN. Conclusion: From the perspective of Chinese payers, PP with PEG-rhG-CSF should be considered cost-effective compared to SP strategies in patients who received chemotherapy regimens with a middle- to high-risk of FN.

Cost-Effectiveness Analysis of First-Line Nivolumab Plus Cabozantinib for Advanced Renal Cell Carcinoma in the United States.

INTRODUCTION: Nivolumab plus cabozantinib improved progression-free survival and overall survival compared with sunitinib in the first-line treatment of advanced renal cell carcinoma (RCC) according to CheckMate 9ER study. METHODS: A Markov model was developed to compare the costs and effectiveness of nivolumab plus cabozantinib with those of sunitinib in the first-line treatment of advanced RCC. Primary outcomes were costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). Model uncertainty was assessed in univariable and probabilistic sensitivity analyses. RESULTS: The total cost per patient was $681,425 for nivolumab plus cabozantinib and $256,302 for sunitinib. The incremental QALY for nivolumab plus cabozantinib was 0.49 compared with sunitinib. The ICER for nivolumab plus cabozantinib was $863,720 per QALY gained versus sunitinib. The results remained robust in univariable and probabilistic sensitivity analyses. CONCLUSIONS: On the basis of a willingness-to-pay threshold of $150,000, nivolumab plus cabozantinib was not cost-effective under current drug pricing in the first-line treatment of advanced RCC from a US payer's perspective.

Adding Enzalutamide to First-Line Treatment for Metastatic Hormone-Sensitive Prostate Cancer: A Cost-Effectiveness Analysis.

Background: The aim of this study is to evaluate the pharmacoeconomic profile of adding enzalutamide to first-line treatment for metastatic, hormone-sensitive prostate cancer (mHSPC) from the US and Chinese payers' perspectives. Materials and Methods: A Markov model with three health states: progression-free survival (PFS), progressive disease (PD), and death, was constructed. All patients were assumed to enter the model in the PFS state and transit according to the transition structure. Efficacy data were derived from the ENZAMET trial and Weibull distribution curves were modeled to fit the survival curves. Costs in the model included cost of drugs, best-supportive care (BSC), follow-up, tests, and adverse events (AEs)-related treatments. The primary endpoint of the study was incremental cost-effectiveness ratio (ICER). In addition, the impact of several key parameters on the results of the cost-effectiveness analysis was tested with one-way sensitivity analyses and probabilistic sensitivity analyses. Results: Overall, ICERs were $430,933.95/QALY and $225,444.74/QALY of addition of enzalutamide to androgen deprivation therapy (ADT) vs. ADT from the US and Chinese payers' perspective, respectively. The most influential factors were the utility for the PFS state and the cost of enzalutamide. At the willingness-to-pay (WTP) thresholds of $100,000.00/QALY in the US and $28,988.40/QALY in China, the probability of adding enzalutamide to first-line treatment being a cost-effective option for mHSPC was 0%. Conclusions: Based on the data from the ENZAMET trial and the current price of enzalutamide, adding enzalutamide to first-line treatment is not cost-effective for patients with mHSPC from the US and Chinse payers' perspectives.

Atezolizumab and bevacizumab combination compared with sorafenib as the first-line systemic treatment for patients with unresectable hepatocellular carcinoma: A cost-effectiveness analysis in China and the United states.

BACKGROUND & AIMS: In patients with unresectable hepatocellular carcinoma (HCC), the combination of atezolizumab and bevacizumab improved progression-free survival (PFS) and overall survival compared with sorafenib in the IMbrave150 trial. However, whether the price of the combination could be affordable is unknown. The current study assessed the cost-effectiveness of the combination of atezolizumab and bevacizumab as first-line systemic therapy for patients with unresectable HCC from the Chinese and American payers' perspective. METHODS: A Markov model was built based on a global, multicentre, open-label, phase III randomized trial (IMbrave150, NCT03434379) that included three states of the patient's health: stable disease (SD), progressive disease (PD) and death. Data for all medical costs were acquired from the Red Book, published literature and West China Hospital. Quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) were the primary outcomes. Sensitivity analyses were performed to evaluate the model uncertainty. RESULTS: The treatment consisting of a combination of atezolizumab and bevacizumab yielded an additional 0.53 QALYs compared with sorafenib alone, leading to an ICER of $145,546.21 per QALY in China and $168,030.21 per QALY in the USA, both beyond the willing-to-pay threshold ($28,527.00/QALY in China and $150,000.00 /QALY in the USA). The utility of the PD state was the most influential factor in the Chinese model, and the American model was the most sensitive to the price of sorafenib. The results of the models were robust across sensitivity analyses. CONCLUSION: The combination of atezolizumab and bevacizumab was not a cost-effective strategy for the first-line systemic treatment of unresectable HCC from the Chinese and American payers' perspective.

Cost­effectiveness Analysis of Helicobacter pylori Eradication Therapy in First-Degree Relatives of Patients with Gastric Cancer.

BACKGROUND: Helicobacter pylori (H. pylori) eradication therapy has been shown to reduce the risk of gastric cancer in patients who have a family history of gastric cancer in first-degree relatives. The aim of this study was to assess the cost-effectiveness of H. pylori eradication therapy in a select population in the People's Republic of China. METHODS: A Markov model was applied to evaluate the cost-effectiveness of H. pylori eradication therapy. The long-term costs of H. pylori eradication therapy were calculated from the Chinese perspective. Health outcomes were measured by quality-adjusted life years (QALYs). Epidemiological information and health utilities used in the model were collected from published literatures or statistical bureaus. A sensitivity analysis was conducted to explore the influence of parameters on the uncertainty of the model. RESULTS: Compared with the no eradication therapy group, H. pylori eradication therapy prolonged an average of 4.52 QALYs (32.64 QALYs vs 28.12 QALYs) and saved $3227.07 ($2472.83 vs $5699.90). The cost-effectiveness analysis demonstrated that no H. pylori eradication therapy cost more and produced less QALYs. It was dominated by H. pylori eradication therapy. The one-way sensitive analyses proved that the results were robust to the fluctuations of the input parameters. CONCLUSION: H. pylori eradication therapy not only reduced the risk of gastric cancer in first-degree relatives of patients with gastric cancer but also was an economical strategy with lower costs and greater efficacy.