Chinese Healthcare Perspective Research Articles

Cost-Effectiveness Analysis of Fluocinolone Acetonide Intravitreal (FAI) Implant for Chronic Noninfectious Uveitis Affecting the Posterior Segment of the Eye (NIU-PS) in China.

Chronic non-infectious uveitis affecting the posterior segment (NIU-PS), which can be recurrent and persistent for numerous years, mainly affects people of working age and significantly increases the risk of visual impairment. This study aimed to investigate the cost-effectiveness of fluocinolone acetonide intravitreal (FAI) implant in the treatment of patients with chronic NIU-PS from the Chinese healthcare perspective. A Markov model with a 2-week cycle was constructed from the perspective of the Chinese healthcare system over a lifetime time horizon. The model consists of four health states: on-treatment, treatment failure, blindness, and death. The outcomes for effectiveness were based on the Chinese real-world study (RWS). Utilities and mortality rates were derived from published literature and standard sources. Costs were determined from the MENET website, prices of medical service items at local providers, published literature, and expert surveys. Outcomes were measured in quality-adjusted life years (QALYs). Sensitivity analyses were performed to account for the impact of uncertainty. It was estimated that in the base case, the FAI implant provided 0.43 incremental QALYs compared with the limited current practice (LCP) at an additional cost of $7503.72 (¥50,575.05), resulting in an incremental cost-effectiveness ratio (ICER) of $17,373.49 (¥117,097.33) per QALY gained. Parameters related to utility emerged as the primary influencers on the outcomes. In probabilistic sensitivity analysis (PSA), considering the willingness-to-pay (WTP) threshold of $19,072 (¥128,547) and $38,145 (¥257,094), the FAI implant had 67.70% and 99.50% probability of being cost-effective, respectively. As demonstrated in the scenario analysis, if the FAI implant aligns its price reduction with the average rate from the 2023 negotiation of the National Reimbursement Drug List (NRDL), it would result in lower costs and represent an absolute advantage. The FAI implant, which can effectively reduce the recurrence rate and maintain the incremental costs within the WTP limit, is likely to be cost-effective in treating chronic NIU-PS in China.

Cost-effectiveness of edaravone dexborneol versus dl-3-n-butylphthalide for the treatment of acute ischemic stroke: a Chinese health care perspective

BackgroundEdaravone dexborneol and dl-3-n-butylphthalide are two innovative brain cytoprotective drugs from China that have been approved and widely prescribed for acute ischemic stroke, and the cost of the two drugs are partially paid by the Chinese medical insurance system. This study aimed to investigate and compare the cost-effectiveness of edaravone dexborneol versus dl-3-n-butylphthalide for acute ischemic stroke from the Chinese healthcare system’s perspective.MethodsA model combining a short-term decision tree model with 90 days and a long-term Markov model with a life-time horizon (40 years) was developed to simulate the cost-effectiveness of edaravone dexborneol versus dl-3-n-butylphthalide for acute ischemic stroke over a lifetime horizon. Since the absence of a head-to-head clinical comparison of two therapies, an unanchored matching-adjusted indirect comparison (MAIC) was conducted by adjusting the patient characteristics using individual patient data from pivotal phase III trial of edaravone dexborneol and published aggregated data of dl-3-n-butylphthalide. Health outcomes were measured in quality-adjusted life years (QALYs). Utilities and costs (Chinese Yuan, CNY) were derived from publications and open-access database. One-way and probabilistic sensitivity analyses were performed to evaluate the robustness of results.ResultsCompared with patients in dl-3-n-butylphthalide arm, edaravone dexborneol arm was found to be cost-effective in 90 days and highly cost-effective as the study horizons extended. With a similar direct medical cost, patients in edaravone dexborneol arm slightly gained an additional 0.1615 QALYs in life-time. In the long term (40 years), patients in edaravone dexborneol arm and dl-3-n-butylphthalide arm yielded 8.0351 and 7.8736 QALYs with the overall direct medical cost of CNY 29,185.23 and CNY 29,940.28, respectively. The one-way sensitivity analysis suggested that the incremental cost-effectiveness ratio was most sensitive to the price of edaravone dexborneol and dl-3-n-butylphthalide.ConclusionEdaravone dexborneol is a cost-effective alternative compared with dl-3-n-butylphthalide for acute ischemic stroke patients in current medical setting of China.

Economic evaluation of four treatment strategies for postmenopausal patients with osteoporosis and a recent fracture in mainland China: a cost-effectiveness analysis.

To evaluate the cost-effectiveness of four anti-osteoporosis medications (denosumab, zoledronate, teriparatide, and alendronate) for postmenopausal osteoporotic women in mainland China, using a stratified treatment strategy recommended by the American Association of Clinical Endocrinologists and the American College of Endocrinology (AACE/ACE). A microsimulation Markov model was used to compare the cost-effectiveness of the four treatments in postmenopausal osteoporotic patients of different ages (65, 70, 75, and 80 years), with a recent fracture from the Chinese healthcare perspective. The primary outcome was the incremental cost-effectiveness ratio (ICER), which represent the incremental cost per quality-adjusted life-year (QALY) obtained. One-way deterministic sensitivity analysis (DSA) and probabilistic sensitivity analysis (PSA) were performed to assess the robustness of model findings. Alendronate was dominated by denosumab-to-alendronate and zoledronate at all ages examined, indicating that the costs of the two drugs were lower, but QALYs was greater. However, teriparatide-to-alendronate yielded an ICER of $76,432.07/ QALY, compared with alendronate at age 65, which exceeded the pre-determined willingness-to-pay threshold of $37,653/ QALY. The results were similar at other ages. The DSA showed that the most sensitive parameters were drug efficacy for vertebral and wrist fractures, the relative risk of vertebral fractures, and the persistence of the drugs. The PSA showed that zoledronate had a 100% probability of being the most cost-effective treatment, with a willingness-to-pay threshold of $37,653/ QALY. Stratified treatment based on very high fracture risk is more cost-effective than conventional pills in mainland China. Among the stratified treatments, zoledronate is the optimal option.

Cost-effectiveness analysis of ceftazidime-avibactam as definitive treatment for treatment of carbapenem-resistant Klebsiella pneumoniae bloodstream infection.

Ceftazidime-avibactam (CAZ-AVI) is a novel antibiotic that has been confirmed in the United States and China for use in patients with carbapenem-resistant Klebsiella pneumoniae (CRKP) bloodstream infection (BSI). However, the cost-effectiveness of CAZ-AVI is unknown in China. This study aimed to evaluate the cost-effectiveness of CAZ-AVI compared to polymyxin B (PMB) monotherapy or PMB-based therapy for the treatment of CRKP BSI from the Chinese healthcare perspective. A hybrid decision tree and Markov model were constructed for a hypothetical cohort of patients with CRKP BSI. The time horizon of the Markov model was 5 years with an annual discount rate of 5% used in both costs and quality-adjusted life-years (QALYs). The model data was derived from published literature and publicly available database. Regimens with an incremental cost-effectiveness ratio (ICER) lower than the willingness-to-pay (WTP) threshold of $ 11,600 per QALY were considered cost-effective. Deterministic and probabilistic sensitivity analyses were performed to examine the robustness of model analysis. In the base-analysis, CAZ-AVI provided an additional 60 QALYs and reduced the cost by $ 2,218,300, yielding an ICER of $ -36,730.9/QALY, well below the WTP threshold of $ 11,600 per QALY when compared with PMB-based therapy. CAZ-AVI provided an additional 350 QALYs and increased the cost of $ 208,400, producing an ICER of $ 591.7/QALY that was below the WTP threshold compared to PMB monotherapy. At a $ 11,600/QALY threshold, results were sensitive to the cost of PMB-based strategy, the cost of CAZ-AVI strategy, the probability of cure with CAZ-AVI, and the probability of cure with PMB or PMB-based therapy. CAZ-AVI was an optimal regimen in 76.9% and 80.8% of 10,000 Monte Carlo simulations at $ 11,600/QALY and $ 34,800/QALY, respectively. Meanwhile, CAZ-AVI was cost-effective at the WTP thresholds of all 31 Chinese provinces in 61.4% (Gansu) to 83.1% (Beijing) of simulations. Ceftazidime-avibactam is expected to be a cost-effective treatment compared with PMB monotherapy or PMB-based therapy for CRKP BSI from the Chinese healthcare perspective.

Cost-effectiveness of camrelizumab plus chemotherapy vs. chemotherapy in the first-line treatment of non-squamous NSCLC: Evidence from China.

We aimed to evaluate the cost-effectiveness of camrelizumab plus chemotherapy compared with chemotherapy alone as the first-line treatment for patients with metastatic or advanced non-squamous non-small cell lung cancer (NSCLC) without targetable epidermal growth factor receptor or anaplastic lymphoma kinase genetic aberrations in patients in China. A partitioned survival model was constructed to estimate the cost-effectiveness of camrelizumab plus chemotherapy vs. chemotherapy in the first-line treatment of non-squamous NSCLC from a Chinese healthcare perspective. Survival analysis was performed to calculate the proportion of patients in each state using data from trial NCT03134872. The cost of drugs was obtained from Menet, and the cost of disease management was obtained from local hospitals. Health state data were obtained from published literature. Both deterministic sensitivity analyses (DSA) and probabilistic sensitivity analysis (PSA) were adopted to verify the robustness of the results. Compared with chemotherapy alone, camrelizumab plus chemotherapy provided 0.41 incremental quality-adjusted life years (QALYs) at an incremental cost of $10,482.12. Therefore, the incremental cost-effectiveness ratio of camrelizumab plus chemotherapy was $25,375.96/QALY from the Chinese healthcare perspective, much lower than three times the GDP per capita of China in 2021 ($35,936.09) as the willingness-to-pay threshold. The DSA indicated that the incremental cost-effectiveness ratio was most sensitive to the utility value of progression-free survival, followed by the cost of camrelizumab. The PSA illustrated that camrelizumab had 80% probability of being cost-effective at the threshold of $35,936.09 per QALY gained. The results suggest that camrelizumab plus chemotherapy is a cost-effective choice in the first-line treatment for patients with non-squamous NSCLC in China. Although this study has limitations such as short time of use of camrelizumab, no adjustment of Kaplan-Meier curves and the median overall survival that has not been reached, the difference in results caused by these factors is relatively small.

Cost-Effectiveness of Evolocumab in Adult Patients with Atherosclerotic Cardiovascular Disease from Chinese Healthcare Perspective.

To assess the cost-effectiveness of evolocumab with statins versus placebo combined with statins in the treatment of adult patients with atherosclerotic cardiovascular disease (ASCVD) and low-density lipoprotein cholesterol (LDL-C) levels > 70mg/dl after the maximum tolerable dose of statin therapy in China. A Markov model, based on data from the FOURIER trial, claims databases, and published literature, was used to compare the health outcomes of the two therapies from the perspective of Chinese healthcare system. The time horizon in the model was a lifetime, the cycle length was a year, and the discount rate was 5%. The output indicators of the model included direct medical costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were conducted on critical parameters such as cost, utility, and incidence of cardiovascular events to evaluate the effect of uncertainty in parameters and the robustness of the model. In Chinese adult patients with ASCVD and LDL-C levels > 70mg/dl, evolocumab was associated with incremental QALYs of 1.25 and incremental costs of 18,714 CNY versus placebo, both with a statin therapy, resulting in an ICER of 14,969 CNY/QALY gained, which was less than the willingness to pay (WTP) threshold (80,976 CNY/QALY, a capita GDP of China, 2021). The result of one-way sensitivity analysis indicated that when the effect of evolocumab on myocardial infarction (MI) rate after the first year varied, ICER changed the most. The results of probabilistic sensitivity analysis showed that the probability of evolocumab added to statins being cost-effective at a threshold of 80,976 CNY/QALY was 100%. Compared with placebo and statin therapy combination, evolocumab added to statin therapy for adult patients with ASCVD and LDL-C > 70mg/dl in China is cost-effective.

A model-based cost-effectiveness analysis of fracture liaison services in China

SummaryThis study is a model-based cost-effectiveness analysis of fracture liaison services (FLS) in China, suggesting that FLS could potentially lead to lifetime cost-saving in patients who have experienced a fracture. However, Chinese-specific real-world data is needed to confirm the results of our study.PurposeThe study aimed to assess the potential cost-effectiveness of fracture liaison services (FLS) from the Chinese healthcare perspective with a lifetime horizon.MethodsA previously validated Markov microsimulation model was adapted to estimate the cost-effectiveness of FLS compared to no-FLS. The evaluation was conducted in patients aged 65 years with a recent fracture. Treatment pathways were differentiated by gender, FLS attendance, osteoporosis diagnosis, treatment initiation, and adherence. Given the uncertainty in FLS cost, the cost in the base-case analysis was assumed at US$200. Analyses were also performed to determine the maximum cost for making the FLS cost-saving and cost-effective at the Chinese willingness-to-pay (WTP) threshold. One-way sensitivity analyses were conducted.ResultsWhen compared with no-FLS, the FLS was dominant (lower costs, higher quality-adjusted life years) in our target population at the FLS cost of US$200 per patient. For every 100 patients who were admitted to the FLS, approximately four hip fractures, nine clinical vertebral fractures, and three wrist fractures would be avoided over their lifetimes. Our findings were robust to numerous one-way sensitivity analyses; however, the FLS was not cost-effective in patients aged 80 years and older.ConclusionFLS could potentially lead to lifetime cost-saving in patients who have experienced a fracture. Our study informs the potential cost-effectiveness of FLS and the knowledge gap in China; more future research incorporating Chinese-specific real-world data are needed to confirm the results of our study and to better evaluate the cost-effectiveness of FLS in China.

EE151 Cost Minimization Analysis Evaluating Turoctocog ALFA (NOVOEIGHT®) and Product X As Prophylaxis Treatment for Paediatric and Adult Patients with Severe Haemophilia a in China

Turoctocog Alfa (Novoeight®), a recombinant factor Ⅷ(rFⅧ), was approved in China in 2021. Product X is the most commonly used rFⅧ product among Chinese patients with haemophilia A. This study was to compare the annual cost between these two products as prophylaxis treatment for paediatric and adult patients with severe haemophilia A from the Chinese healthcare perspective.

Cost-Effectiveness of Posaconazole vs. First-Generation Triazoles for the Prevention of Invasive Fungal Infections Among High-Risk Patients With Hematological Malignancies in China.

BackgroundPosaconazole is confirmed to be more effective for preventing invasive fungal infections (IFIs) than first-generation triazoles (fluconazole and itraconazole), but its economic value has not been comprehensively evaluated in China. This study compared the cost-effectiveness of these two antifungal prophylaxis regimens in hematological-malignancy patients at high risk for IFIs from the Chinese healthcare perspective.MethodsA hybrid decision tree and Markov model were built using published data to estimate the total costs and quality-adjusted life-years (QALYs) of antifungal prophylaxis with posaconazole oral suspension and first-generation triazoles. Regimens with an incremental cost-effectiveness ratio (ICER) lower than the threshold of willingness to pay (WTP) were considered cost-effective. One-way and probabilistic sensitivity analyses were performed to assess model robustness. The regional imbalance of economic development and the tablet formulation of posaconazole were considered in the scenario analyses.ResultsIn the base-case analysis, posaconazole oral suspension provided an additional 0.109 QALYs at an incremental cost of $954.7, yielding an ICER of $8,784.4/QALY, below the national WTP threshold of $31,315/QALY. One-way and probabilistic sensitivity analyses showed that the results were robust. Scenario analyses showed that the base-case ICER was consistently below the WTP thresholds of all 31 Chinese provinces, with the likelihood of posaconazole being cost-effectiveness ranging from 78.1 to 99.0%. When the posaconazole oral suspension was replaced by the tablet formulation, the ICER increased to $29,214.1/QALY, still below the national WTP threshold and WTP thresholds of 12 provinces.ConclusionsPosaconazole oral suspension is a highly cost-effective regimen for preventing IFI in high-risk hematological-malignancy patients from the Chinese healthcare perspective. Posaconazole tablets may also be considered in some high-income regions of China.

Cost-effectiveness of empagliflozin as a treatment for heart failure with reduced ejection fraction: an analysis from the Chinese healthcare perspective.

BackgroundThe effect of empagliflozin on the cardiovascular outcome is consistent in heart failure with reduced ejection fraction (HFrEF) patients regardless of the presence or absence of diabetes. More evidence is needed regarding the cost-effectiveness of empagliflozin in HFrEF patients. This study sought to evaluate the economic outcomes of adding empagliflozin to the standard treatment for HFrEF patients from the perspective of the Chinese healthcare system, and thus to provide information for decision makers.MethodsBased on the EMPEROR-Reduced clinical trial and other published literature data, the direct medical costs and quality-adjusted life years (QALYs) of patients with HFrEF over a 15-year study period were simulated by a Markov model, and the incremental cost-effectiveness ratio (ICER) was calculated. The price of empagliflozin referred to the data released by Menet, the hospitalization expenses and utility values were derived from published studies in China. A one-way sensitivity analysis and probabilistic sensitivity analysis were conducted to evaluate the robustness of the model.ResultsThe results of the cost-effectiveness analysis showed that the cost of the combination arm was $5,220.98, with a utility of 4.86 QALYs, and the cost of the standard arm was $4,873.96, with a utility of 4.68 QALYs, which equated to an ICER of $1,893.59 per QALY gained. The subgroup analysis showed that patients with HFrEF and diabetes in empagliflozin group had a higher QALY (4.62 vs. 4.35) and a lower cost ($5,213.28 vs. $5,958.60) than standard group. The corresponding ICER for non-diabetic patients was $2,568.15 per QALY. Deterministic sensitivity analysis showed robust results. At the willingness-to-pay threshold of 3 times gross domestic product (GDP) per capita ($31,510.57), almost all of the scattered points in three scenarios were below the threshold line.ConclusionsAt a willingness-to-pay threshold of $31,510.57, adding empagliflozin to standard treatment is a very cost-effective option for HFrEF patients with or without diabetes in China.

Comparison of Evolocumab and Ezetimibe, Both Combined with Statin Therapy, for Patients with Recent Acute Coronary Syndrome: A Cost-Effectiveness Analysis from the Chinese Healthcare Perspective.

To assess the cost-effectiveness of evolocumab, a PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitor, compared with ezetimibe, both added to background statin therapy in patients with recent acute coronary syndrome (ACS) events (in the past 12 months) and low-density lipoprotein cholesterol (LDL-C) levels ≥ 100 mg/dL in China. A health economic evaluation was performed from a Chinese healthcare perspective, using a Markov model over a lifetime horizon based on a baseline cardiovascular (CV) event rate from claims database data and efficacy from the FOURIER trial. The health benefit was reflected in the decrease of LDL-C level, which led to a decrease of cardiovascular events. The costs of cardiovascular events and the utility value of each health state were derived from the published literature. Sensitivity analyses were conducted to evaluate the effects of uncertainty in parameters and the robustness of the model. The cost-effectiveness of evolocumab was also explored in patients with recent myocardial infarction (MI), at very high risk (VHR) of atherosclerotic cardiovascular disease (ASCVD), and homozygous familiar hypercholesterolemia (HoFH). In patients with recent ACS, evolocumab was associated with incremental quality-adjusted life-years (QALYs) of 1.33 and incremental costs of 115,782 yuan versus ezetimibe, both with background statin therapy, resulting in an incremental cost-effectiveness ratio (ICER) of 87,050 yuan per QALY gained. The probability of evolocumab + statins being cost-effective at a threshold of 217,341 yuan (three times per capita GDP, 2020), compared with ezetimibe + statins, was 100% in patients with recent ACS, recent MI, VHR ASCVD, and HoFH. Compared with ezetimibe + statins, the combination of evolocumab + statins was found to be cost-effective at a threshold of 217,341 yuan (three times per capita GDP, 2020) in patients with recent ACS events in China.

Cost-effective analysis of mechanical thrombectomy alone in the treatment of acute ischaemic stroke: a Markov modelling study

ObjectiveRecently, a randomised controlled trial (DIRECT-MT) demonstrated that mechanical thrombectomy (MT) was non-inferior to MT with intravenous alteplase as to the functional outcomes. This study aims to investigate whether MT...

Cost-effectiveness of brentuximab vedotin for relapsed or refractory systemic anaplastic large-cell lymphoma in China

Objectives Systemic anaplastic large-cell lymphoma (sALCL) is a rare hematological malignancy with poor prognosis, which is associated with a significant economic burden. This study aimed to evaluate the cost-effectiveness of brentuximab vedotin (BV) in comparison to conventional chemotherapy in patients with relapsed/refractory sALCL, from a Chinese healthcare perspective. Methods A partitioned survival model with three health states (progression-free survival, post-progression survival, and death) was adapted to compare BV against chemotherapy. Comparator represented a basket of commonly used chemotherapies in China. Two cohorts in each arm were estimated, representing patients receiving no transplant and autologous stem cell transplant (ASCT) after BV or chemotherapy. Clinical data was obtained from the pivotal phase-II trial (NCT00866047) for BV and also from the literature for a comparator. Resource use items covered drug acquisition and administration; concomitant medications; ASCT; treatment of adverse events; and long-term follow-up. Cost parameters were based on Chinese sources. Outcomes were measured in quality-adjusted life-years (QALYs). Both costs and effects were discounted at 5% according to Chinese guidelines. The impact of uncertainty was evaluated using deterministic and probabilistic sensitivity analyses. Results The incremental cost-effectiveness ratio (ICER) for BV vs. chemotherapy was $9,610 (¥62,084) per QALY in the base case. The main model driver was superior progression-free and overall survival benefits of BV. The ICERs were relatively robust in the majority of sensitivity analyses, ranging around ±10% of the base case. Under the conventional decision thresholds (1–3 times of Chinese per capita GDP), the probability of BV being cost-effective ranged from 56 to 100%. Limitations of the study included the lack of comparative data from the trial and the small and heterogeneous sample due to its disease nature. Conclusions BV may be a cost-effective treatment vs. chemotherapy in treating relapsed or refractory systemic anaplastic large-cell lymphoma in China.

Cost-effectiveness Analysis of Prophylaxis Versus On-demand Treatment for Children With Hemophilia B Without Inhibitors in China

PurposeHemophilia B (HB) is a hereditary bleeding disorder caused by a deficiency of coagulation factor IX (FIX), which represents 15% to 20% of all patients with hemophilia. Clinical studies have found significant benefits of prophylaxis treatment with FIX versus on-demand (OD) treatment. However, these benefits are associated with an increase in FIX consumption and a considerable increase in cost. Most Chinese children with HB receive OD treatment. Only a small proportion of patients with HB receive prophylaxis treatment in China. The patients with inhibitors could result in more complicated bleeding events, joint status, or treatment patterns. The objective of this study is to assess the cost-effectiveness of prophylaxis compared with OD treatment in children with HB without inhibitors from the Chinese health care perspective. MethodsA Markov model was used to analyze cost-effectiveness by comparing prophylaxis with OD treatment. The model uses a 17-year time horizon with a yearly cycle. Transition probabilities and utility weights were estimated using published studies. The cost data for patients with HB were collected from Beijing Children's Hospital and Capital Medical University. One-way and probabilistic sensitivity analyses were performed to assess the robustness of the results. FindingsThe model projects that prophylaxis has an incremental 1.23 quality-adjusted life-years (QALYs). The incremental cost per QALY gained for individuals with HB receiving prophylaxis was ¥155,709.80 ($23,530.36) compared with the OD group, which is under the willingness-to-pay threshold (3 times the gross domestic product per capital according to the World Health Organization recommendations) in China of ¥193,932 ($29,306.37). Moreover, 1-way sensitivity analysis found that the results were sensitive to the utility of nonarticular bleeding. The lower this parameter is, the higher the probability is of the incremental cost-effectiveness ratio for prophylaxis not being cost-effective. This finding infers that when the patients have a better QALY (higher utility) at the beginning, the cost for benefit from prophylaxis treatment is lower. The results of the probabilistic sensitivity analyses indicate that the probability of prophylaxis being cost-effective is 89.3%. ImplicationsAlthough prophylaxis is a costly treatment, the results of this study indicate that it is cost-effective compared with OD treatment for children with HB in China.

Cost-effectiveness of alirocumab for myocardial infarction in China

Background and Aims: Proprotein convertase subtilisin/kexin type 9 inhibitor alirocumab reduce ischemic events; however, the cost-effectiveness remains unknown. This study sought to evaluate its economic value in patients with myocardial infarction (MI) from the Chinese healthcare perspective.

Cost-effectiveness analysis of genotype screening and therapeutic drug monitoring in patients with inflammatory bowel disease treated with azathioprine therapy: a Chinese healthcare perspective using real-world data.

BackgroundThis study aimed to analyze the cost-effectiveness of combining screening for thiopurine methyl transferase (TPMT) and nucleotide triphosphate diphosphatase (NUDT15) defective alleles with therapeutic drug monitoring (TDM) in Chinese patients with inflammatory bowel disease (IBD) treated with azathioprine (AZA).MethodsWe evaluated the cost-effectiveness of combining screening for NUDT15 and TPMT deficiency with TDM in patients receiving AZA treatment over a 1-year horizon by developing a decision tree model. Real-world data and published literature were used to derive model inputs. The model’s primary outcomes included quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs). One-way and probabilistic sensitivity analyses were used to address uncertainty.ResultsCompared to NUDT15 genotyping, the combined TPMT/NUDT15 genotyping strategy cost an additional $13.83, yielding an ICER of $3,929.54/QALY, which was under the willingness-to-pay level of $30,425 per QALY in China. Compared to strategies with singular TPMT genotyping or no genotyping, the combined TPMT/NUDT15 genotyping strategy gained 0.00406 and 0.00782 QALYs and reduced the cost by $25.15 and $99.06, respectively. Additionally, incorporating TDM of AZA was more effective and less expensive than strategies without TDM. One-way sensitivity analysis revealed the expense attached to severe myelotoxicity to be the factor with the greatest influence in the present research. The application of the combined genotype screening strategy with TDM of AZA treatment was found to have a 91.7% chance of being cost-effective.ConclusionsFor Chinese patients with IBD who receive an AZA regimen, a strategy involving combined NUDT15/TPMT genotype screening prior to treatment initiation and incorporating TDM for treatment management is cost-effective compared to strategies involving genotyping of NUDT15 or TPMT alone or genotyping without TDM.

Cost-effectiveness Analysis of Trastuzumab Emtansine as Second-line Therapy for HER2-Positive Breast Cancer in China.

Trastuzumab emtansine (T-DM1) is the standard second-line option for the treatment of patients with human epidermal growth factor receptor-2 (HER2)-positive breast cancer for its superior clinical efficacy in prolonging progression-free survival. The objective of this study was to evaluate the cost effectiveness of T-DM1 from the Chinese healthcare perspective. Capecitabine (Cap), capecitabine + lapatinib (Cap + Lap), capecitabine + trastuzumab (Cap + Tra), capecitabine + trastuzumab + pertuzumab (Cap + Tra + Per) were selected as comparators. A three-state Markov simulation model was performed. The state transition probabilities were estimated based on the results of a published network meta-analysis, and utilities were derived from the published literature. The costs populated in the model were acquired from the local charge or previously published studies. One-way sensitive analysis and probabilistic sensitivity analyses were performed to test the robustness of the results. Compared with Cap, Cap + Lap, Cap + Tra, and Cap + Tra + Per, T-DM1 was estimated to increase the cost by US$109,699.1, $106,019.1, $97,506.3, and $67,121.9, respectively, and yield a gain of 0.544 quality-adjusted life years (QALYs), 0.383 QALYs, 0.367 QALYs, 0.087 QALYs, respectively. Corresponding incremental cost-effectiveness ratios (ICERs) were $201,652.9, $276,812.5, $265,685.0, and $771,516.1 per QALY. The probabilities of T-DM1 as the dominant option were 0% at the willingness-to-pay (WTP) threshold of $31,245.1/QALY. T-DM1, as second-line therapy in the treatment of HER2-positive breast cancer, is not a cost-effective option in China. Given the significant clinical efficacy, an appropriate price reduction of T-DM1 is required to benefit more HER2-positive breast cancer patients.

Cost-Effectiveness of Alirocumab for the Secondary Prevention of Cardiovascular Events after Myocardial Infarction in the Chinese Setting.

Background: Proprotein convertase subtilisin/kexin type 9 inhibitor alirocumab reduce ischemic events; however, the cost-effectiveness remains uncertain. This study sought to evaluate its economic value in patients with myocardial infarction (MI) from the Chinese healthcare perspective. Methods: A state-transition Markov model was developed to determine the cost-effectiveness of alirocumab for preventing recurrent MI, ischemic stroke and death. Preventative effect of the therapy was gathered from ODYSSEY OUTCOMES trial and absolute reduction of low-density lipoprotein cholesterol (LDL-C) in ODYSSEY EAST trial, respectively. The primary outcome was the incremental cost-effectiveness ratio (ICER), defined as incremental cost per quality-adjusted life-year (QALY) gained. Results: Compared with statin monotherapy, the ICER of alirocumab therapy at its present discounted price [34,355 Chinese yuan (CNY) annually, 33% rebate] based on clinical follow-up efficacy was 1,613,997 CNY per QALY gained. A willingness-to-pay threshold of 212,676 CNY per QALY would be achieved when the annual cost of alirocumab was reduced by 88% from the full official price to 6071 CNY. The therapeutic effect evaluation estimated by the magnitude of LDL-C reduction was superior to the results of clinical follow-up, but this medication was still far from cost-effective. Multiple vulnerable subgroup analyses demonstrated that the ICER for patients with polyvascular disease in 3 vascular beds was 111,750 CNY per QALY gained. Conclusion: Alirocumab is not cost-effective in general MI population based on current discounted price. High long-term costs of alirocumab may be offset by health benefit in patients with polyvascular disease (3 beds).

Cost-effectiveness analysis of fruquintinib versus regorafenib as the third-line therapy for metastatic colorectal cancer in China

Objectives The aim of this study is to assess the cost-effectiveness of fruquintinib compared to regorafenib as third-line treatment for patients with metastatic colorectal cancer (mCRC) in China. Methods A three-state Markov model with monthly cycle was constructed to estimate lifetime incremental cost-effectiveness ratio (ICER) of fruquintinib versus regorafenib as third-line treatment for patients with mCRC from Chinese health care perspective. Survival analysis was applied to calculate transition probabilities using the data from the clinical trials FRESCO and CONCUR, which were also the data sources accessing probabilities of adverse events. Background mortality rate and drug costs were derived from government published data. Costs for medical services were obtained from real-world data and published literatures. Utilities applied to calculate the quality-adjusted life years (QALYs) were obtained from literature review. One-way sensitivity analysis and probabilistic sensitivity analysis were adopted to verify the robustness of the results. Results Fruquintinib provided 0.74 QALYs at a cost of CNY 151,058 (USD 22,888), whereas regorafenib provided 0.79 QALYs at a cost of CNY 226,657 (USD 32,224). Compared to fruquintinib, the ICER of regorafenib was CNY 1,529,197/QALY (USD 231,697/QALY) from Chinese health care perspective, which was above the triple GDP per capita of China in 2019 (CNY 212,676) (USD 32,224) as the threshold to define the cost-effectiveness. One-way sensitivity analysis showed the results were generally robust. Cost-effectiveness acceptability curves derived from probabilistic sensitivity analysis demonstrated the probability that fruquintinib was more cost-effective was 100% when the threshold was the triple GDP per capita of China. Conclusions Compared to regorafenib, fruquintinib, which leads to forego about 0.05 QALYs and save about CNY 75,599 (USD 11,454), is a cost-effective choice as the third-line treatment for patients with mCRC in China.

Cost-Effectiveness Analysis of Oral Direct-Acting Antivirals for Chinese Patients with Chronic Hepatitis C.

All oral direct-acting antivirals (DAAs) have shown excellent efficacy and safety in Chinese patients with chronic hepatitis C (CHC). However, the cost of DAAs used to be expensive; therefore, large numbers of patients had no access to DAAs in China. Recently, prices have been greatly reduced. The objective of this study was to evaluate the cost-effectiveness of ledipasvir/sofosbuvir (LDV/SOF), sofosbuvir/velpatasvir (SOF/VEL), elbasvir/grazoprevir (EBR/GZR) and glecaprevir/pibrentasvir (GLE/PIB) in Chinese CHC patients stratified by hepatitis C virus (HCV) genotype (GT), cirrhosis status, and treatment history. On the basis of a Chinese healthcare perspective, a Markov model was constructed to estimate the lifetime costs and health outcomes of patients treated with different DAA regimens. Chinese-specific clinical, cost, and utility inputs were obtained or calculated from published sources and expert opinions. Costs, life-years (LYs), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were reported as primary outcomes. Base-case analysis and sensitivity analysis were conducted. At a willing-to-pay (WTP) threshold of US$30,081/QALY (calculated by three times the GDP per capita in China), SOF/VEL was cost-effective in patients with HCV GT 1, 3, and 6 infections, and the probabilities that SOF/VEL was cost-effective were 9.7-75.7%, 39.1-63.9%, and 35.6-88.0%, respectively. For GT2 patients, noncirrhotic patients, treatment-naïve patients, and treatment-experienced patients, LDV/SOF was the most cost-effective regimen, and the probabilities of cost-effectiveness for each of these groups was 92.1-99.8%, 89.9-99.0%, 61.6-91.2%, and 99.3-100.0%, respectively below the WTP threshold. The GLE/PIB regimen (12-week duration) was the most cost-effective in cirrhotic patients, whereas the probability of its cost-effectiveness varied with that of EBR/GZR (4.1-93.8% versus 6.2-93.3%) below the WTP threshold. Overall, SOF/VEL and LDV/SOF regimens are more likely to be cost-effective among various subgroups of Chinese patients with CHC.